LATEST COMPANY NEWS. - Free Online Library (2024)

IAM Patent - UPC's first revocation proceeding decision is another blow for Amgen in decade-long PCSK9 dispute - 17/7/2024

In its first ruling, the Munich Central Division has sided with Sanofi/Regeneron, invalidating Amgen's patent.

For the complete story, see:

https://www.iam-media.com/article/upcs-first-revocation-proceeding-decision-another-blow-amgen-in-decade-long-pcsk9-dispute

Genetic Engineering and Biotechnology News - Replacing Chemo: Vertex, Orum Launch Up to $945M+ DAC Collaboration - 17/7/2024

Orum has granted Vertex rights to conduct research using its TPD² approach to developing DACs.

For the complete story, see:

https://www.genengnews.com/topics/drug-discovery/replacing-chemo-vertex-orum-launch-up-to-945m-dac-collaboration/

Reuters - Vertex sues US over fertility support program for Casgevy gene editing therapy - 16/7/2024

Vertex Pharmaceuticals sued the U.S. Department of Health and Human Services on Monday.

For the complete story, see:

https://www.reuters.com/business/healthcare-pharmaceuticals/vertex-sues-us-over-fertility-support-program-casgevy-gene-editing-therapy-2024-07-15/

Other Stories

FiercePharma - Amgen, BMS and Merck have the most exposure as Big Pharma eyes $183B patent cliff - 15/7/2024

GlobeNewswire - Regeneron Pharmaceuticals, Inc. Investors: Company Investigated by the Portnoy Law Firm - 10/7/2024

FiercePharma - US government appeals Gilead's trial win in Truvada, Descovy patent fight - 9/7/2024

FiercePharma - Roche scores FDA nod for prefilled syringe version of Vabysmo, easing its administration - 8/7/2024

MedCity News - Sanofi & Regeneron Drug Dupixent Gets EMA Approval as the First Biologic COPD Med - 7/7/2024

Media Releases

Gilead Sciences (NASDAQ: GILD) - Chief Medical Officer Merdad Parsey to Leave Gilead Early 2025 - 17/7/2024

Latest Research

Flavor Ingredient Sustainability and Biotechnology - By Mindy Davila & Xiaofen Du

Overviews of Leading Companies

Alexion Pharmaceuticals (ALXN)

Amgen (NASDAQ: AMGN)

Biogen Idec (NASDAQ: BIIB)

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN)

Celgene Corporation (CELG)

Gilead Sciences (NASDAQ: GILD)

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN)

Seagen Inc.

Shire plc (SHPG)

Vertex Pharmaceuticals (NASDAQ: VRTX)

Senior Associate: Joseph Hang Ellision

News and Commentary

IAM Patent - UPC's first revocation proceeding decision is another blow for Amgen in decade-long PCSK9 dispute - 17/7/2024

In its first ruling, the Munich Central Division has sided with Sanofi/Regeneron, invalidating Amgen's patent.

For the complete story, see:

https://www.iam-media.com/article/upcs-first-revocation-proceeding-decision-another-blow-amgen-in-decade-long-pcsk9-dispute

Genetic Engineering and Biotechnology News - Replacing Chemo: Vertex, Orum Launch Up to $945M+ DAC Collaboration - 17/7/2024

Orum has granted Vertex rights to conduct research using its TPD² approach to developing DACs.

For the complete story, see:

https://www.genengnews.com/topics/drug-discovery/replacing-chemo-vertex-orum-launch-up-to-945m-dac-collaboration/

Reuters - Vertex sues US over fertility support program for Casgevy gene editing therapy - 16/7/2024

Vertex Pharmaceuticals sued the U.S. Department of Health and Human Services on Monday.

For the complete story, see:

https://www.reuters.com/business/healthcare-pharmaceuticals/vertex-sues-us-over-fertility-support-program-casgevy-gene-editing-therapy-2024-07-15/

FiercePharma - Amgen, BMS and Merck have the most exposure as Big Pharma eyes $183B patent cliff - 15/7/2024

At the other end of the list, Amgen has the most revenue at risk at 67%, with its top four products on the clock.

For the complete story, see:

https://www.fiercepharma.com/pharma/firepower-aplenty-and-patent-cliffs-ahead-time-right-ma-activity-report

GlobeNewswire - Regeneron Pharmaceuticals, Inc. Investors: Company Investigated by the Portnoy Law Firm - 10/7/2024

On April 10, 2024, the U.S. Department of Justice ("DOJ") issued a press release announcing that it had filed a complaint against Regeneron under the False Claims Act.

For the complete story, see:

https://www.globenewswire.com/news-release/2024/07/10/2911413/0/en/Regeneron-Pharmaceuticals-Inc-Investors-Company-Investigated-by-the-Portnoy-Law-Firm.html#

FiercePharma - US government appeals Gilead's trial win in Truvada, Descovy patent fight - 9/7/2024

As Gilead Sciences tussles with a web of HIV drug litigation, a high-stakes case tied to patents for prevention medicines is coming back to test the drugmaker.

For the complete story, see:

https://www.fiercepharma.com/pharma/gilead-faces-prep-threat-after-us-appeals-trial-win-truvada-and-descovy-patent-litigation

FiercePharma - Roche scores FDA nod for prefilled syringe version of Vabysmo, easing its administration - 8/7/2024

Since Roche gained approval for its eye disease medication Vabysmo in 2022, it has stormed a market dominated by Regeneron and Bayer's Eylea.

For the complete story, see:

https://www.fiercepharma.com/pharma/roche-scores-fda-nod-prefilled-syringe-version-vabysmo-easing-its-administration

MedCity News - Sanofi & Regeneron Drug Dupixent Gets EMA Approval as the First Biologic COPD Med - 7/7/2024

European Medicines Agency approval of Dupixent in chronic obstructive pulmonary disease makes the drug the first biologic therapy approved for treating the prevalent respiratory condition.

For the complete story, see:

https://medcitynews.com/2024/07/sanofi-regeneron-dupixent-biologic-copd-drug-ema-approval-type-2-inflammation/

Media Releases

Gilead Sciences (NASDAQ: GILD) - Chief Medical Officer Merdad Parsey to Leave Gilead Early 2025 - 17/7/2024

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) announced today that Chief Medical Officer Merdad Parsey, MD, PhD, will leave the company early next year. While the company works to identify a successor, Dr. Parsey will continue as Chief Medical Officer until the first quarter of 2025 and support this transition over the next several months.

"On behalf of everyone at Gilead, I want to thank Merdad for his significant contributions as our Chief Medical Officer over the last five years. These were pivotal years for the company as we worked to strengthen and diversify the portfolio, and Merdad played a fundamental role in our success," said Daniel O'Day, Chairman and Chief Executive Officer, Gilead. "We have more than doubled our portfolio under Merdad's leadership, and with 54 ongoing clinical trials across virology, oncology, and inflammation, we are well positioned to build on our success for the future."

Dr. Parsey joined Gilead in November 2019 and is responsible for overseeing the company's global clinical development and medical affairs organizations. During his tenure, he led the growth of the development organization, specifically with the advancement of market leading treatments in virology and the establishment of Gilead's oncology pipeline and therapeutics.

"It has been a privilege to lead the development team, especially as we have worked to deliver transformative medicines for people with HIV, COVID-19, and cancer," said Dr. Parsey. "I am profoundly excited about the work we have done to build a strong, diverse clinical pipeline that has tremendous potential to deliver on our commitment to improve health for people around the world."

https://www.gilead.com/news-and-press/press-room/press-releases/2024/7/chief-medical-officer-merdad-parsey-to-leave-gilead-early-2025

Latest Research

Flavor Ingredient Sustainability and Biotechnology

Mindy Davila & Xiaofen Du

Abstract

Current flavor production methods (predominantly natural extraction and chemical synthesis) cannot be relied upon to meet the production needs of such a rapidly growing population. There is also an increasing consumer desire for natural food products. Natural products meet consumer quality expectations but are often prohibitively priced and unsustainable due to agriculture variations and damaging farming practices. Chemically synthesized flavor ingredients are cheap, but they do not meet consumer expectations due to their use of non-renewable resources and potential environmental pollution. Flavor biotechnology, or using whole or partial living organisms to create flavor ingredients, is a promising alternative to natural extraction and chemical synthesis; it offers product isomer specificity, cost advantages, and lower environmental pollution. In fact, the biotech flavors could be labeled natural in both the EU and United States. Flavor biotechnology research is growing in popularity in the following areas: using intact microbial cells to produce flavor chemicals or using isolated enzymes, whole plants, plant cells and tissue cultures, thermal processes, agro-industrial waste, or algae for flavor production. Enzymatic or microbial biotechnology methods have been adopted to produce more than 100 commercial flavor molecules. Even so, there is much to be learned involving cell and metabolic pathways in addition to solutions for feasibility issues like product cytotoxicity and difficult downstream purification. In the future, there will likely be advances in bio-aroma technology, knowledge, interest, and cost-competitiveness, leading to an overall increased market share.

https://link.springer.com/chapter/10.1007/978-3-031-51808-9_5

The Industry

Updated: January 26, 2023

Biotechnology Industry in the US - Market Research Report

Gene therapy: Merger and acquisition activity could pick up as leading pharma companies lose patents

Biotechnology in the US industry trends (2018-2023)

Biotechnology revenue has increased at a CAGR of 2.6% to $193.1 billion over the past five years, including 1.6% growth in 2023, when profit will total 7.8%. Vaccine development drove biotech investment. Biotechnology's diverse product offerings, markets, company sizes and funding sources have protected it against volatile downstream market fluctuations. Despite COVID-19's widespread economic disruption, biotechnology had one of its best years in 2020. Biotechnology companies and pharmaceutical manufacturers rushed to develop vaccines and therapeutics, leading investors to pour capital into the field. Investment in biotech accelerated further in 2021, totaling more than $34.0 billion globally. Biotech is more insulated from econom... lock

Biotechnology in the US industry outlook (2023-2028)

Average industry growth 2023-2028: x.xlock

Biotechnology revenue will increase at a CAGR of X.X% to $XX.X billion over the next five years, when profit will total X.X%. R&D funding will be crucial as ever Biotechnology companies will need to continue to raise capital to fund research and development (R&D). But, a higher cost of capital for investors could shift investment activity moving forward. Investors will be less risk-averse as interest rates rise, seeking safer and more secure investments in established biotech over early-stage companies. Still, emerging biotech developments offer promising potential to investors, likely shielding biotech from some investor uncertainty. In XXX, President Biden signed the Inflation Reduction Act, which will reduce the price of prescription drugs by a...lock

Biotechnology in the US industry statistics

attach_money Market Size: $193bn

business Number of Businesses in: 4,364

poll Average Industry Profit Margin: x.x% lock

supervisor_account Industry Employment: 408,376

Biggest companies in the Biotechnology industry in the US

IBIS World covers 7 companies in the Biotechnology in the US industry, including Abbvie inc., Amgen Inc., Genentech Inc., Bayer Corp and Gilead Sciences, Inc.

pie_chart Abbvie inc. Market Share: x.x% lock

pie_chart Amgen Inc. Market Share: x.x% lock

pie_chart Genentech Inc. Market Share: x.x% lock

pie_chart Bayer Corp Market Share: x.x% lock

pie_chart Gilead Sciences, Inc. Market Share: x.x% lock

pie_chartAaa Aaaaaaaa Market Share: x.x%lock

Source: IBIS World

https://www.ibisworld.com/united-states/market-research-reports/biotechnology-industry/

Leading Companies

Alexion Pharmaceuticals (ALXN)

Acquisition of Alexion completed

21 July 2021 13:45 BST

NOT FOR RELEASE, PUBLICATION OR DISTRIBUTION, IN WHOLE OR IN PART, DIRECTLY OR INDIRECTLY, IN OR INTO OR FROM ANY JURISDICTION WHERE TO DO SO WOULD CONSTITUTE A VIOLATION OF THE RELEVANT LAWS OR REGULATIONS OF SUCH JURISDICTION.

AstraZeneca today completed the acquisition of Alexion Pharmaceuticals, Inc. (Alexion). The closing of the acquisition marks the Company's entry into medicines for rare diseases and the beginning of a new chapter for AstraZeneca.

AstraZeneca now has an enhanced scientific presence in immunology and, through Alexion's innovative complement-biology platform and robust pipeline, will continue to pioneer the discovery and development of medicines for patients with rare diseases. Rare diseases represent a significant unmet medical need and becomes a high-growth opportunity for the Company.

Pascal Soriot, Chief Executive Officer, said: "Today we welcome our new colleagues from Alexion to AstraZeneca and begin a new chapter that will augment our growth for years to come. Our sustained R&D investment in oncology, cardiovascular and renal, as well as respiratory and immunology, has powered AstraZeneca's transformation and now we add rare diseases, where fewer approved treatment options exist."

Marc Dunoyer, incoming Chief Executive Officer, Alexion and Chief Financial Officer, AstraZeneca, said: "I am delighted to be working alongside my new colleagues at Alexion where we will continue to discover, develop and deliver medicines that change the lives of people suffering from rare diseases. We look forward to also applying Alexion's complement-biology platform across areas of AstraZeneca's broader early stage pipeline and, significantly, to the extraordinary opportunity to extend existing and future rare disease medicines to patients in many countries where AstraZeneca already has a strong presence."

Financial considerations

The total consideration paid to the Alexion shareholders was approximately $13.3bn in cash and 236,321,411 new AstraZeneca shares (approximately 94% of which will be represented by new AstraZeneca American Depositary Shares (ADSs)).

Admission of the new AstraZeneca shares issued to Alexion shareholders to listing on the premium listing segment of the official list of the Financial Conduct Authority (FCA) and to the trading on the London Stock Exchange's main market for listed securities has been approved and will be effective at 08:00 BST on 22 July 2021. Trading of the new AstraZeneca shares on Nasdaq Stockholm is expected to commence on 22 July 2021. Trading of the new AstraZeneca ADSs on the Nasdaq Stock Market will commence at 09:30 EDT on 22 July 2021. The Alexion shares will be delisted from the Nasdaq Stock Market and Alexion will terminate its registration under the U.S. Securities Exchange Act of 1934 as soon as practicable following completion of the acquisition.

As a result of completion, the issued share capital of AstraZeneca PLC with voting rights is 1,549,116,129 ordinary shares of $0.25. No shares are held in Treasury. Therefore, the total number of voting rights in AstraZeneca PLC is 1,549,116,129. The above figure for the total number of voting rights may be used by shareholders as the denominator for the calculations by which they will determine if they are required to notify their interest in, or a change to their interest in, AstraZeneca PLC under the FCA's Disclosure and Transparency Rules.

AstraZeneca anticipates providing updated 2021 financial guidance for the new, combined entity in due course. Consolidation of Alexion will start from the closing of the transaction and the first quarter of consolidated financial reporting is expected to be the third quarter of 2021 due for announcement on Friday 12 November 2021.

Rare diseases

Over 7,000 rare diseases are known today, and only approximately 5% have treatments approved by the US Food and Drug Administration.1 Demand in medicines for rare diseases is forecasted to grow by a low double-digit percentage in the future.2

Forward-looking statements

This announcement contains certain forward-looking statements with respect to the operations, performance and financial condition of the AstraZeneca Group, including, among other things, statements about expected revenues, margins, earnings per share or other financial or other measures, as well as the ability of AstraZeneca to successfully integrate Alexion's operations, and the ability of AstraZeneca to implement its plans, forecasts and other expectations with respect to Alexion's business after Completion and realise expected synergies. Although the AstraZeneca Group believes its expectations are based on reasonable assumptions, any forward-looking statements, by their very nature, involve risks and uncertainties and may be influenced by factors that could cause actual outcomes and results to be materially different from those predicted. The forward-looking statements reflect knowledge and information available at the date of preparation of this announcement and the AstraZeneca Group undertakes no obligation to update these forward-looking statements. The AstraZeneca Group identifies the forward-looking statements by using the words 'anticipates', 'believes', 'expects', 'intends' and similar expressions in such statements. Important factors that could cause actual results to differ materially from those contained in forward-looking statements, certain of which are beyond the AstraZeneca Group's control, include, among other things: the risks set out in Part II (Risk Factors) of the AstraZeneca shareholder circular published on 12 April 2021; failure or delay in delivery of pipeline or launch of new medicines; failure to meet regulatory or ethical requirements for medicine development or approval; failure to obtain, defend and enforce effective intellectual property (IP) protection and IP challenges by third parties; competitive pressures including expiry or loss of IP rights, and generic competition; price controls and reductions; economic, regulatory and political pressures; uncertainty and volatility in relation to the UK's exit from the EU; failures or delays in the quality or execution of commercial strategies; failure to maintain supply of compliant, quality medicines; illegal trade in medicines; reliance on third-party goods and services; failure in information technology, data protection or cybercrime; failure of critical processes; uncertainty of expected gains from productivity initiatives; failure to attract, develop, engage and retain a diverse, talented and capable workforce, including following Completion; failure to adhere to applicable laws, rules and regulations; the safety and efficacy of marketed medicines being questioned; adverse outcome of litigation and/or governmental investigations, including relating to the Acquisition; failure to adhere to increasingly stringent anti-bribery and anti-corruption legislation; failure to achieve strategic plans or meet targets or expectations; failure in financial control or the occurrence of fraud; unexpected deterioration in AstraZeneca's or Alexion's financial position; the impact that the COVID-19 global pandemic may have or continue to have on these risks, on AstraZeneca's ability to continue to mitigate these risks, and on AstraZeneca's operations, financial results or financial condition; the risk that AstraZeneca is unable to achieve the synergies and value creation contemplated by the Acquisition, or that AstraZeneca is unable to promptly and effectively integrate Alexion's businesses; and the risk that management's time and attention are diverted on Acquisition-related issues or that disruption from the Acquisition makes it more difficult to maintain business, contractual and operational relationships.

Neither AstraZeneca nor any of its associates or directors, officers or advisers provides any representation, assurance or guarantee that the occurrence of the events expressed or implied in any forward-looking statements in this announcement will actually occur. You are cautioned not to place undue reliance on these forward-looking statements. Other than in accordance with their legal or regulatory obligations (including under the Listing Rules, the Disclosure and Transparency Rules and the Prospectus Regulation Rules of the FCA), AstraZeneca is under no obligation, and AstraZeneca expressly disclaims any intention or obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

AstraZeneca

AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.

Contacts

For details on how to contact the Investor Relations Team, please click here. For Media contacts, click here.

References

1. In the US, a rare disease impacts less than 200,000 patients (as defined in the US Orphan Drug Act 1983).

2. EvaluatePharma, World Preview 2020, Outlook to 2026.

Adrian Kemp

Company Secretary

AstraZeneca PLC

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/acquisition-of-alexion-completed.html

ABOUT US

Every Day We Change Lives

People living with rare and devastating diseases are our Guiding Star. We believe it is our responsibility to listen to, understand, and change the lives of patients and those who work tirelessly to help them.

Our Mission

To transform the lives of people affected by rare diseases and devastating conditions by continuously innovating and creating meaningful value in all that we do.

And we're making real progress, every day.

5 - APPROVED MEDICINES

7 - RARE DISEASES & DEVASTATING CONDITIONS

4 - DURABLE, GROWING FRANCHISES

50 - COUNTRIES WITH PATIENTS SERVED

Every Day Strengthens Our Impact

We continue to deepen our understanding of rare disease, which began with our pioneering work in complement biology. This knowledge allows us to innovate and evolve into new areas, where there is great unmet need and opportunity to help patients and families fully live their best lives.

We've delivered transformative medicines for people with paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD), generalized Myasthenia Gravis (gMG), hypophosphatasia (HPP), and lysosomal acid lipase deficiency (LAL-D).

Every Day Builds a Better Tomorrow

We invest in and value people who believe in the importance of our purpose and understand what it takes to deliver on it. Our culture is rooted in integrity, inclusiveness, and our dedication to joining and supporting the communities in which we live and work.

At Alexion, our passion drives us to continuously innovate and create meaningful value in all we do. In doing so, we change lives for the better - ours, people living with rare diseases, and the communities we serve. Every day.

3000+ TALENTED COLLEAGUES

4 PRIX GALIEN AWARDS

25+ YEARS OF LEADERSHIP IN RARE DISEASE

https://alexion.com/our-company/about-us

30/4/2021

Alexion Reports First Quarter 2021 Results

1Q21 total revenues of $1,636.5 million, a 13% increase over 1Q20

1Q21 GAAP diluted EPS of $2.86; non-GAAP diluted EPS of $3.52

Completed enrollment in Phase 3 studies of ULTOMIRIS® (ravulizumab) in NMOSD and ALS

Filed for regulatory approval of ONDEXXYA® [coagulation factor Xa (recombinant), inactivated-zhzo] in Japan

Secured UK reimbursement for ULTOMIRIS in PNH and ONDEXXYA for GI bleeds

Received U.S. Federal Trade Commission clearance for proposed acquisition by AstraZeneca; transaction expected to close in 3Q21

BOSTON--(BUSINESS WIRE)--Apr. 30, 2021-- Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced financial results for the first quarter of 2021. Total revenues in the first quarter were $1,636.5 million, a 13 percent increase compared to the same period in 2020. The positive impact of foreign currency on total revenues year-over-year was less than 1 percent inclusive of hedging activities. On a GAAP basis, diluted EPS attributable to Alexion in the first quarter of 2021 was $2.86, a 14 percent increase versus the first quarter of 2020. Non-GAAP diluted EPS attributable to Alexion for the first quarter of 2021 was $3.52, a 9 percent increase versus the first quarter of 2020.

"We are off to a strong start in 2021, with continued advancement of our LEAD-EXPAND-DIVERSIFY strategy to progress our commercial portfolio as well as our many development programs," said Ludwig Hantson, Ph.D., Chief Executive Officer of Alexion. "I am so proud of our teams' ongoing dedication and execution, which are further advancing our mission of delivering life-changing medicines to people with rare diseases and devastating conditions. We are well positioned to build on our success and momentum as the year progresses and once we become part of AstraZeneca."

First Quarter 2021 Financial Highlights

Net product sales were $1,635.7 million in the first quarter of 2021, compared to $1,444.6 million in the first quarter of 2020.

SOLIRIS net product sales were $1,027.6 million, compared to $1,022.9 million in the first quarter of 2020, representing a 0.5 percent increase.

ULTOMIRIS net product sales were $346.9 million, compared to $222.8 million in the first quarter of 2020, representing a 56 percent increase.

STRENSIQ net product sales were $197.5 million, compared to $172.2 million in the first quarter of 2020, representing a 15 percent increase.

KANUMA net product sales were $34.8 million, compared to $26.7 million in the first quarter of 2020, representing a 30 percent increase.

ANDEXXA/ONDEXXYA net product sales were $28.9 million in the first quarter of 2021.

GAAP cost of sales was $125.4 million, compared to $111.7 million in the first quarter of 2020. Non-GAAP cost of sales was $113.8 million, compared to $108.6 million in the first quarter of 2020.

GAAP R&D expense was $289.1 million, compared to $200.9 million in the first quarter of 2020. Non-GAAP R&D expense was $267.0 million, compared to $185.7 million in the first quarter of 2020.

GAAP SG&A expense was $342.9 million, compared to $319.9 million in the first quarter of 2020. Non-GAAP SG&A expense was $292.2 million, compared to $259.1 million in the first quarter of 2020.

GAAP income tax expense was $113.4 million, compared to income tax expense of $106.0 million in the first quarter of 2020. Non-GAAP income tax expense was $145.7 million, compared to income tax expense of $141.2 million in the first quarter of 2020.

GAAP diluted EPS attributable to Alexion was $2.86, compared to $2.50 in the first quarter of 2020. Non-GAAP diluted EPS attributable to Alexion was $3.52, compared to $3.22 in the first quarter of 2020.

Research and Development

PHASE 3/4

SOLIRIS - Guillain-Barre Syndrome (GBS): SOLIRIS in GBS has been granted SAKIGAKE designation by Japan's Ministry of Health, Labour and Welfare (MHLW). In February 2021, Alexion initiated a Phase 3 study of SOLIRIS in GBS in Japan and dosing is underway.

ULTOMIRIS - 100 mg/mL: An application for approval of the ULTOMIRIS 100 mg/mL formulation for PNH and aHUS is under review in Japan. This higher concentration formulation is designed to reduce infusion time by more than 60 percent to approximately 45 minutes.

ULTOMIRIS - Subcutaneous: The Phase 3 study of weekly subcutaneous (SC) ULTOMIRIS demonstrated PK-based non-inferiority versus intravenous ULTOMIRIS. Pending collection of 12-month safety and drug-device combination data, Alexion plans to file for approval in the U.S. for the ULTOMIRIS SC formulation and device combination in PNH and aHUS in the third quarter of 2021, and in the EU in the first quarter of 2022.

ULTOMIRIS - Paroxysmal Nocturnal Hemoglobinuria (PNH): The U.S. FDA granted priority review for ULTOMIRIS in children and adolescents with PNH and has set a Prescription Drug User Fee Act (PDUFA) target action date of June 7, 2021.

ULTOMIRIS - gMG: Enrollment is complete in the Phase 3 study of ULTOMIRIS in adults with gMG. Study results are expected in the second half of 2021.

ULTOMIRIS - NMOSD: In March 2021, Alexion completed enrollment in the Phase 3 study of ULTOMIRIS in NMOSD.

ULTOMIRIS - Amyotrophic Lateral Sclerosis (ALS): In March 2021, Alexion completed enrollment in the Phase 3 study of ULTOMIRIS in ALS. Study results are expected in the first half of 2022.

ULTOMIRIS - Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA): Phase 3 studies of ULTOMIRIS in adults and children with HSCT-TMA are underway.

ULTOMIRIS - Complement Mediated Thrombotic Microangiopathy (CM-TMA): Alexion plans to initiate a Phase 3 study of ULTOMIRIS in CM-TMA in the second quarter of 2021.

ULTOMIRIS - Severe COVID-19: Further enrollment in a Phase 3 trial of ULTOMIRIS in adults hospitalized with severe COVID-19 requiring mechanical ventilation is paused, due to lack of efficacy, pending further analysis of the data. Alexion continues to provide ULTOMIRIS for the ongoing TACTIC-R platform study led by Cambridge University Hospitals NHS Foundation Trust, which is evaluating the potential of earlier immune modulatory treatment (hospitalized patients not requiring mechanical ventilation) in preventing progression of the virus.

ULTOMIRIS - Dermatomyositis (DM): Alexion plans to initiate a Phase 2/3 study of ULTOMIRIS in DM in the second half of 2021, pending regulatory feedback.

ALXN1840 - Wilson Disease: Enrollment and dosing are complete in a Phase 3 study of ALXN1840 in Wilson disease. Study results are expected in the third quarter of 2021.

CAEL-101 - Caelum Biosciences: Alexion and Caelum Biosciences are conducting the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care therapy in AL amyloidosis. Two parallel Phase 3 studies - one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease - are underway.

ALXN2060 (AG10): Alexion holds an exclusive license to develop and commercialize ALXN2060 (AG10) in Japan. Eidos is currently evaluating AG10 in two Phase 3 studies in the U.S. and Europe - one for ATTR cardiomyopathy (ATTR-CM) and one for ATTR polyneuropathy (ATTR-PN). Alexion is conducting a Phase 3 bridging study of ALXN2060 for patients with ATTR-CM in Japan.

ALXN2040 (Danicopan) - PNH with Extravascular Hemolysis (EVH): A Phase 3 study of ALXN2040 as an add-on therapy for PNH patients with EVH is underway.

ANDEXXA/ONDEXXYA - Acute Intracranial Hemorrhage (ICH): The Phase 4 ANNEXA-I study - designed to provide clinical data supporting full approval - is underway to assess ANDEXXA compared to usual standard of care in patients presenting with acute intracranial hemorrhage while taking an oral Factor Xa inhibitor. In addition, a supplemental Biologics License Application (sBLA) is under review by the U.S. FDA to enable the addition of edoxaban and enoxaparin to the U.S. label. In February 2021, Alexion filed for regulatory approval of ONDEXXYA in Japan.

PHASE 1/2

ULTOMIRIS - Renal Diseases: A proof-of-concept study of ULTOMIRIS in patients with IgA nephropathy and lupus nephritis is underway.

ALXN1830: Due to COVID-19, Alexion discontinued the Phase 2 study of ALXN1830, administered intravenously, in warm autoimmune hemolytic anemia (WAIHA) and the Phase 1 study of a subcutaneous formulation of ALXN1830 in healthy volunteers. In March 2021, Alexion initiated a new Phase 1 study of subcutaneous ALXN1830 in healthy volunteers. Following successful completion of this Phase 1 study, Alexion plans to initiate Phase 2 studies of subcutaneous ALXN1830 in gMG and WAIHA in 2021, pending regulatory feedback.

ALXN2040 - Geographic Atrophy (GA): In March 2021, Alexion submitted an Investigational New Drug (IND) application for ALXN2040 in GA and plans to initiate a Phase 2 study in the second half of 2021.

ALXN2040 - COVID-19: Alexion has agreed to provide ALXN2040 to the U.S. National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, for the ACTIV-5 Big Effect Trial in adults hospitalized with COVID-19. This Phase 2 platform trial is comparing different investigational therapies to a common control arm with the intent of identifying promising treatments to enter a more definitive study.

ALXN2050 - PNH: Alexion has re-initiated additional enrollment in the Phase 2 study of ALXN2050 monotherapy in PNH patients, following the receipt of Phase 1 data that support further dose escalation in the Phase 2 study.

ALXN2050 - Renal Diseases: Alexion plans to initiate a proof-of-concept study of ALXN2050 in patients with various renal diseases in 2021, pending regulatory feedback.

ALXN1720: The Phase 1 healthy volunteer study of ALXN1720, a novel anti-C5 albumin-binding bi-specific mini-body that is designed to bind and prevent activation of human C5, has been paused for a second time due to COVID-19, but is expected to resume in the second quarter of 2021. Additional cohorts have been added to the study to explore higher doses and enable the initiation of a Phase 3 study in gMG, pending successful completion of the Phase 1 study as has been agreed with the U.S. FDA. Data from the Phase 1 study are expected in the second half of 2021. Alexion also plans to initiate a study of ALXN1720 in DM.

ANDEXXA - Urgent Surgery: ANDEXXA is currently being evaluated in a single-arm, open-label Phase 2 study in patients taking apixaban, rivaroxaban, edoxaban, or enoxaparin who require urgent surgery. The results of this study will inform the design of a randomized controlled Phase 3 clinical trial to expand the label in this population.

ALXN2075 (cerdulatinib): Acquired as part of the Portola acquisition, ALXN2075 is a dual spleen tyrosine kinase and janus kinase (SYK/JAK) inhibitor being evaluated in a Phase 1/2a study in patients with relapsed/refractory chronic lymphocytic leukemia or B-cell or T-cell non-Hodgkin lymphoma. Data are expected in the second quarter of 2021.

ALXN1820: A Phase 1 study of ALXN1820, Alexion's bi-specific anti-properdin mini-body, is underway in healthy volunteers.

ALXN1850 - Hypophosphatasia (HPP): Alexion plans to initiate a Phase 1 study of ALXN1850 in adults with HPP in the second quarter of 2021, pending regulatory feedback.

COVID-19

We continue to take steps to proactively respond to the evolving COVID-19 pandemic and to plan for related uncertainties. We remain focused on continuing to serve patients, protecting the health and safety of our employees and the communities in which we live and work, and supporting patients in clinical trials. We are also focused on minimizing potential interactions that could contribute to the spread of the virus and put additional strain on healthcare systems through the use of innovative virtual means where possible.

Clinical Trials: We have implemented a pandemic response business continuity plan designed to protect patients and site staff safety while continuing our clinical trials with limited interruption to the extent we are able. The COVID-19 impact has varied by study and program, but there has been little timing impact on fully-enrolled trials and the majority of studies that had been temporarily paused due to the pandemic have resumed. A small number of clinical trial sites are restricting site visits and imposing restrictions on the initiation of new trials and patient visits to protect both site staff and patients from possible COVID-19 exposure. Based on local dynamics where these studies are being conducted, there has been, and may continue to be, an impact to the timing of trials that are enrolling patients and activating sites, or have not yet started to do so. We are actively implementing remote and local procedures under the guidance of regulatory authorities.

Business Impact: We continue to take proactive measures designed to mitigate the risk of potential interruptions in supply and/or access to patients' customary site-of-care locations. Treatment compliance rates across all our medicines have remained strong. We have also seen the predicted slowing of new patient initiations and delays in treatment starts, and we are continuing to closely monitor this environment as the pandemic continues.

Conference Call/Earnings Materials:

Given the agreement for Alexion to be acquired by AstraZeneca, Alexion will not be hosting a conference call. Earnings materials are available publicly on the Investor Relations page of our website at http://ir.alexion.com. Questions may be directed to the Investor Relations team via e-mail at [emailprotected] or the contact information below.

About Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology and acute care. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.

[ALXN-E]

Additional Information and Where to Find It

In connection with AstraZeneca's proposed acquisition of Alexion (the "proposed transaction"), AstraZeneca filed with the U.S. Securities and Exchange Commission ("SEC") a registration statement on Form F-4 which includes a proxy statement of Alexion and a prospectus of AstraZeneca. The registration statement was declared effective by the SEC on April 12, 2021, and mailing of the definitive joint proxy statement/prospectus to the shareholders of Alexion occurred on or about April 12, 2021. Each of Alexion and AstraZeneca may also file other relevant documents with the SEC regarding the proposed transaction. INVESTORS AND SECURITY HOLDERS ARE URGED TO READ THE DEFINITIVE JOINT PROXY STATEMENT/PROSPECTUS AND ANY OTHER RELEVANT DOCUMENTS THAT MAY BE FILED WITH THE SEC, AS WELL AS ANY AMENDMENTS OR SUPPLEMENTS TO THESE DOCUMENTS, CAREFULLY AND IN THEIR ENTIRETY IF AND WHEN THEY BECOME AVAILABLE BECAUSE THEY CONTAIN OR WILL CONTAIN IMPORTANT INFORMATION ABOUT THE PROPOSED TRANSACTION. Investors and security holders will be able to obtain free copies of the registration statement and the definitive proxy statement/prospectus and other documents containing important information about Alexion, AstraZeneca and the proposed transaction through the website maintained by the SEC at http://www.sec.gov. Copies of the documents filed with the SEC by Alexion will be available free of charge on Alexion's website at http://www.alexion.com or by contacting Alexion's Investor Relations Department by email at [emailprotected]. Copies of the documents filed with the SEC by AstraZeneca will be available free of charge on AstraZeneca's website at https://www.astrazeneca.com/investor-relations.html or by contacting AstraZeneca's Investor Relations department by email at [emailprotected].

Participants in the Solicitation

Alexion, AstraZeneca, their respective directors and certain of their executive officers and other employees may be deemed to be participants in the solicitation of proxies from Alexion's shareholders in connection with the proposed transaction. Information about Alexion's directors and executive officers is available in Alexion's proxy statement for its 2020 annual meeting of shareholders, which was filed with the SEC on March 26, 2020, Alexion's Annual Report on Form 10-K/A for the fiscal year ended December 31, 2020, which was filed with the SEC on February 16, 2021, and other documents subsequently filed by Alexion with the SEC. Information about AstraZeneca's directors and executive officers is available in AstraZeneca's Form 20-F filed with the SEC on February 16, 2021, and other documents subsequently filed by AstraZeneca with the SEC. Other information regarding the participants in the proxy solicitations and a description of their direct and indirect interests, by security holdings or otherwise, are contained in the definitive joint proxy statement/prospectus filed with the SEC on April 12, 2021 and other relevant materials to be filed with the SEC regarding the proposed transaction when they become available. Free copies of these documents may be obtained as described in the paragraphs above.

No Offer or Solicitation

This communication is not intended to and shall not constitute an offer to buy or sell or the solicitation of an offer to buy or sell any securities, or a solicitation of any vote or approval, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction. No offering of securities shall be made, except by means of a prospectus meeting the requirements of Section 10 of the U.S. Securities Act of 1933, as amended.

Forward Looking Statements

This communication contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. You can generally identify forward-looking statements by the use of forward-looking terminology such as "anticipate," "believe," "continue," "could," "estimate," "expect," "explore," "evaluate," "intend," "may," "might," "plan," "potential," "predict," "project," "seek," "should," or "will," or the negative thereof or other variations thereon or comparable terminology. These forward-looking statements are only predictions and involve known and unknown risks and uncertainties, many of which are beyond Alexion's and AstraZeneca's control. Statements in this communication regarding Alexion, AstraZeneca and the combined company that are forward-looking, including anticipated benefits of the proposed transaction, the impact of the proposed transaction on Alexion's and AstraZeneca's businesses and future financial and operating results, the amount and timing of synergies from the proposed transaction, the terms and scope of the expected financing for the proposed transaction, the aggregate amount of indebtedness of the combined company following the closing of the proposed transaction, are based on management's estimates, assumptions and projections, and are subject to significant uncertainties and other factors, many of which are beyond Alexion's and AstraZeneca's control. These factors include, among other things, market factors, competitive product development and approvals, pricing controls and pressures (including changes in rules and practices of managed care groups and institutional and governmental purchasers), economic conditions such as interest rate and currency exchange rate fluctuations, judicial decisions, claims and concerns that may arise regarding the safety and efficacy of in-line products and product candidates, changes to wholesaler inventory levels, variability in data provided by third parties, changes in, and interpretation of, governmental regulations and legislation affecting domestic or foreign operations, including tax obligations, changes to business or tax planning strategies, difficulties and delays in product development, manufacturing or sales including any potential future recalls, patent positions and the ultimate outcome of any litigation matter. Additional information concerning these risks, uncertainties and assumptions can be found in Alexion's and AstraZeneca's respective filings with the SEC, including the risk factors discussed in Alexion's most recent Annual Report on Form 10-K, as updated by its Quarterly Reports on Form 10-Q, in AstraZeneca's most recent Annual Report on Form 20-F and in each company's future filings with the SEC. Important risk factors could cause actual future results and other future events to differ materially from those currently estimated by management, including, but not limited to, the risks that: a condition to the closing the proposed acquisition may not be satisfied; a regulatory approval that may be required for the proposed acquisition is delayed, is not obtained or is obtained subject to conditions that are not anticipated; AstraZeneca is unable to achieve the synergies and value creation contemplated by the proposed acquisition; AstraZeneca is unable to promptly and effectively integrate Alexion's businesses; management's time and attention is diverted on transaction related issues; disruption from the transaction makes it more difficult to maintain business, contractual and operational relationships; the credit ratings of the combined company declines following the proposed acquisition; legal proceedings are instituted against Alexion, AstraZeneca or the combined company; Alexion, AstraZeneca or the combined company is unable to retain key personnel; and the announcement or the consummation of the proposed acquisition has a negative effect on the market price of the capital stock of Alexion or AstraZeneca or on Alexion's or AstraZeneca's operating results. No assurances can be given that any of the events anticipated by the forward-looking statements will transpire or occur, or if any of them do occur, what impact they will have on the results of operations, financial condition or cash flows of Alexion or AstraZeneca. Should any risks and uncertainties develop into actual events, these developments could have a material adverse effect on the proposed transaction and/or Alexion or AstraZeneca, AstraZeneca's ability to successfully complete the proposed transaction and/or realize the expected benefits from the proposed transaction. You are cautioned not to rely on Alexion's and AstraZeneca's forward-looking statements. These forward-looking statements are and will be based upon management's then-current views and assumptions regarding future events and operating performance, and are applicable only as of the dates of such statements. Neither Alexion nor AstraZeneca assumes any duty to update or revise forward-looking statements, whether as a result of new information, future events or otherwise, as of any future date.

In addition to financial information prepared in accordance with GAAP, this press release also contains non-GAAP financial measures that Alexion believes, when considered together with the GAAP information, provide investors and management with supplemental information relating to performance, trends and prospects that promote a more complete understanding of our operating results and financial position during different periods. Alexion also uses these non-GAAP financial measures to establish budgets, set operational goals and to evaluate the performance of the business. The non-GAAP results, determined in accordance with our internal policies, exclude the impact of the following GAAP items (see reconciliation tables below for additional information): share-based compensation expense, fair value adjustment of inventory acquired, amortization of purchased intangible assets, changes in fair value of contingent consideration, restructuring and related expenses, upfront payments related to licenses and other strategic agreements, acquired in-process research and development, impairment of purchased intangible assets, gains and losses related to strategic equity investments, litigation charges, gain or loss on sale of a business or asset, gain or loss related to modification of purchase options, contingent milestone payments associated with acquisitions of legal entities accounted for as asset acquisitions, acquisition-related costs and certain adjustments to income tax expense. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for, or superior to, the financial measures prepared and presented in accordance with GAAP, and should be reviewed in conjunction with the relevant GAAP financial measures. Please refer to the attached Reconciliation of GAAP to non-GAAP Financial Results for explanations of the amounts adjusted to arrive at non-GAAP net income and non-GAAP earnings per share amounts for the three month periods ended March 31, 2021 and 2020.

https://ir.alexion.com/news-releases/news-release-details/alexion-reports-first-quarter-2021-results

http://news.alexionpharma.com/press-release/alexion-reports-third-quarter-2015-results

Amgen (NASDAQ: AMGN)

ABOUT AMGEN

Amgen is one of the world's leading biotechnology companies. Amgen is a values-based company, deeply rooted in science and innovation to transform new ideas and discoveries into medicines for patients with serious illnesses.

At Amgen, we believe in a "biology first" approach. We use cutting-edge science and technology to study the subtlest biological mechanisms in search of therapies that will improve the lives of those who suffer from serious diseases. Amgen believes the cure for disease can be found inside each and every one of us.

https://www.amgen.com/about/

AMGen incorporates.

AMGen (Applied Molecular Genetics Inc.) is established in Thousand Oaks, California, on April 8, 1980, as the brainchild of venture capitalists William K. (Bill) Bowes and associates. With a staff of three, the Company occupies a shared building, now called "Building 1."

George B. Rathmann is named first CEO.

AMGen names its first CEO, scientist and businessman George B. Rathmann. Dubbed "Mr. Biotech" by Red Herring magazine, Rathmann has been called one of the great geniuses of high-tech entrepreneurialism. Working from a small trailer to free up space for scientists, Rathmann quickly establishes scientific goals and secures funding to conduct grand experiments in technology.

https://www.amgenhistory.com/

2/5/2024

Amgen reports first quarter 2024 financial results

THOUSAND OAKS, Calif., May 2, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced financial results for the first quarter 2024.

"With many of our innovative products delivering strong growth and promising new medicines advancing through our pipeline, we are excited about delivering attractive long-term growth," said Robert A. Bradway, chairman and chief executive officer.

Key results include:

For the first quarter, total revenues increased 22% to $7.4 billion in comparison to the first quarter of 2023. Product sales grew 22%, driven by 25% volume growth.

Ten products delivered at least double-digit volume growth in the first quarter, including Repatha® (evolocumab), TEZSPIRE® (tezepelumab-ekko), EVENITY® (romosozumab-aqqg), BLINCYTO® (blinatumomab), and TAVNEOS® (avacopan).

S. volume grew 29% and ex-U.S. volume grew 17%.

Our performance included $914 million of sales from our Horizon Therapeutics (Horizon) acquisition, driven by several first-in-class, early-in-lifecycle medicines, including TEPEZZA® (teprotumumab-trbw), KRYSTEXXA® (pegloticase) and UPLIZNA® (inebilizumab-cdon).

Excluding sales from Horizon, our product sales grew 6%, driven by volume growth of 9%.

GAAP loss per share was $0.21 for the first quarter of 2024 compared with GAAP earnings per share (EPS) of $5.28 for the first quarter of 2023, driven by a mark-to-market loss on our BeiGene, Ltd. equity investment and higher operating expenses, including higher amortization expense from Horizon-acquired assets and incremental expenses from Horizon, partially offset by higher revenues.

GAAP operating income decreased from $1.9 billion to $1.0 billion, and GAAP operating margin decreased 19.0 percentage points to 13.9%.

Non-GAAP EPS decreased 1% from $3.98 to $3.96, due to higher operating and interest expenses driven by the Horizon acquisition, partially offset by higher revenues.

Non-GAAP operating income increased from $2.8 billion to $3.1 billion, and non-GAAP operating margin decreased 5.1 percentage points to 43.2%.

The Company generated $0.5 billion of free cash flow for the first quarter of 2024 versus $0.7 billion in the first quarter of 2023. This decrease was driven by an $800 million tax deposit, partially offset by timing of working capital items.

Product Sales Performance

Total product sales increased 22% for the first quarter of 2024 versus the first quarter of 2023, driven by 25% volume growth.

General Medicine

Repatha® sales increased 33% year-over-year to $517 million in the first quarter, driven by 44% volume growth, partially offset by 13% lower net selling price. Repatha remains the global proprotein convertase subtilisin/kexin type 9 (PCSK9) segment leader, with over 2.9 million patients treated since launch.

Prolia® (denosumab) generated $999 million of sales in the first quarter. Sales increased 8% year-over-year primarily driven by volume growth.

EVENITY® sales increased 35% year-over-year to $342 million for the first quarter, primarily driven by volume growth.

Oncology

BLINCYTO® sales increased 26% year-over-year to $244 million for the first quarter, driven by broad prescribing across academic and community segments for patients with B-cell precursor acute lymphoblastic leukemia (B-ALL).

Vectibix® (panitumumab) generated $247 million of sales in the first quarter. Sales increased 6% year-over-year driven by higher net selling price and volume growth, partially offset by unfavorable foreign exchange impact.

KYPROLIS® (carfilzomib) sales increased 5% year-over-year to $376 million for the first quarter, primarily driven by volume growth outside the U.S.

LUMAKRAS®/LUMYKRAS[TM] (sotorasib) sales increased 11% year-over-year to $82 million for the first quarter, driven by volume growth.

XGEVA® (denosumab) sales increased 5% year-over-year to $561 million for the first quarter, primarily driven by volume growth outside the U.S. and higher net selling price, partially offset by lower volume in the U.S.

Nplate® (romiplostim) generated $317 million of sales in the first quarter. Sales decreased 12% year-over-year, primarily driven by volume decline in comparison to the first quarter of 2023, which included a U.S. government order of $82 million. Excluding the U.S. government order from this comparison, Nplate sales grew 13% year-over-year, primarily driven by volume growth.

MVASI® (bevacizumab-awwb) generated $202 million of sales in the first quarter. Sales were flat year-over-year for the first quarter. Volume growth was largely offset by lower net selling price and unfavorable changes to estimated sales deductions. Going forward we expect continued net selling price erosion driven by competition.

Inflammation

TEZSPIRE® generated $173 million of sales in the first quarter. Sales increased 80% year-over-year, primarily driven by volume growth. Healthcare providers recognize TEZSPIRE's unique, differentiated profile and its broad potential to treat the 2.5 million patients worldwide with severe asthma who are uncontrolled, without any phenotypic or biomarker limitation.

Otezla® (apremilast) generated $394 million of sales in the first quarter. Sales increased 1% year-over-year for the first quarter.

Enbrel® (etanercept) generated $567 million of sales in the first quarter. Sales decreased 2% year-over-year driven by volume decline, partially offset by higher inventory levels. Moving forward, we expect modest volume growth offset by declining net selling price.

Otezla and Enbrel typically have lower sales in the first quarter relative to subsequent quarters due to the impact of benefit plan changes, insurance reverifications and increased co-pay expenses as U.S. patients work through deductibles.

AMJEVITA®/AMGEVITA[TM] (adalimumab) generated $168 million of sales in the first quarter. Sales increased 2% year-over-year primarily driven by international growth, partially offset by lower inventory levels and unfavorable change to estimated sales deductions.

Rare Disease

Except for TAVNEOS®, the products listed below were added through the acquisition of Horizon on Oct. 6, 2023.

TEPEZZA® (teprotumumab-trbw) generated $424 million of sales in the first quarter. TEPEZZA is the first and only FDA-approved treatment for thyroid eye disease (TED).

KRYSTEXXA® (pegloticase) generated $235 million of sales in the first quarter. KRYSTEXXA is the first and only FDA-approved treatment for chronic refractory gout.

UPLIZNA® (inebilizumab-cdon) generated $80 million of sales in the first quarter. UPLIZNA is used to treat adults with neuromyelitis optica spectrum disorders.

TAVNEOS® generated $51 million of sales in the first quarter. Sales increased 122% year-over-year, driven by volume growth.

Ultra rare products, which consist of RAVICTI® (glycerol phenylbutyrate), PROCYSBI® (cysteamine bitartrate), ACTIMMUNE® (interferon gamma-1b), BUPHENYL® (sodium phenylbutyrate) and QUINSAIR® (levofloxacin), generated $169 million of sales in the first quarter.

Established Products

Our established products, which consist of EPOGEN® (epoetin alfa), Aranesp® (darbepoetin alfa), Parsabiv® (etelcalcetide) and Neulasta® (pegfilgrastim), generated $613 million of sales. Sales decreased 19% year-over-year for the first quarter, driven by unfavorable changes to estimated sales deductions and volume declines. In the aggregate, we expect the year-over-year volume declines for this portfolio of products to continue.

For full release:

https://www.amgen.com/newsroom/press-releases/2024/05/amgen-reports-first-quarter-2024-financial-results

Biogen Idec (NASDAQ: BIIB)

About Biogen

Caring Deeply. Working Fearlessly. Changing Lives. [TM]

At Biogen, our mission is clear: we are pioneers in neuroscience.

Since our founding in 1978 as one of the world's first global biotechnology companies by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp, Biogen has led innovative scientific research with the goal over the last decade to defeat devastating neurological diseases.

Millions of people around the world are affected by multiple sclerosis, Alzheimer's disease, Parkinson's disease and amyotrophic lateral sclerosis (ALS). Many people also suffer from less common diseases such as spinal muscular atrophy (SMA) and progressive supranuclear palsy (PSP).

We believe that no other disease area holds as much need or as much promise for medical breakthroughs as neuroscience.

Biogen has some of the world's best neurologists and neuroscientists. We engage with physicians and scientific leaders around the world with the aim to further medical research. Our focus on neuroscience, our deep scientific expertise and our courage to take risks make us leaders in the research and development of medicines to transform neuroscience to benefit society.

Our technology and engineering capabilities create novel ways to seamlessly transition products from development to manufacturing with the intent of bringing our high-quality medicines to market faster.

We respect the contributions of health care providers caring for people living with neurological diseases. We honor the important role of caregivers, families and friends who care about them.

Biogen is committed to working with advocacy and patient organizations as they serve the communities they represent.

Recognizing the challenges facing health care systems today, we collaborate with regulatory authorities and customers such as health care providers and payers, so that those in need can access our medicines.

Professional, ethical, and compliant, we hold ourselves accountable to deliver value to our shareholders.

Biogen contributes to the communities where we live. We are committed to our employees, diversity and inclusion, and environmental sustainability.

We care deeply about making a difference.

We work fearlessly. We do not give up even when challenged, pursuing innovation in all that we do.

We are humbled by the opportunity to change lives.

https://www.biogen.com/en_us/about-us.html

13/2/2024

Biogen reports fourth quarter and full year 2023 results and expects return to Non-GAAP EPS growth in 2024

Fourth quarter 2023 revenue $2.4 billion; GAAP diluted EPS $1.71; Non-GAAP diluted EPS $2.95

* GAAP and Non-GAAP diluted EPS negatively impacted by $0.35 related to previously disclosed closeout costs for ADUHELM

Full year 2023 revenue $9.8 billion; GAAP diluted EPS $7.97; Non-GAAP diluted EPS $14.72

Biogen to co-promote LEQEMBI in U.S. and Japan, building off steady launch progress; LEQEMBI approved in China

Expanding rare disease portfolio with SKYCLARYS, an innovative product in an area of high unmet medical need, recently launched in the U.S. with ~1,000 patients on drug; received European Commission approval

ZURZUVAE off to a promising start in U.S. for adults with postpartum depression

Continue to expect "Fit for Growth" to generate savings of $1 billion gross and $800 million net by 2025

Full year 2024 financial guidance: Non-GAAP EPS of $15.00 to $16.00, representing EPS growth of approximately 5% versus 2023 at the mid-point

* Expect total revenue to decline by a low- to mid-single digit percentage vs. 2023 and expect core pharmaceutical revenue (product revenue + LEQEMBI) to be flat vs. 2023

* Expect operating income to grow low-double digit percentage vs. 2023 with expected mid-single digit percentage point operating margin expansion

Biogen Inc. (NASDAQ: BIIB) today reported fourth quarter and full year 2023 financial results. Commenting on the results, President and Chief Executive Officer Christopher A. Viehbacher said:

"2023 was a year of transformation for Biogen as we saw approval for four first-in-class medicines while we realigned our cost structure, remained prudent in allocating shareholder capital, and reprioritized our pipeline. We believe with these key elements in place we are now well positioned to return Biogen to sustainable growth. As we look to 2024, our focus is on operational execution, including building upon the progress of our recent new product launches. We believe this will allow us to continue to advance our goal of a new Biogen that creates enhanced value for patients and our shareholders."

* Fourth quarter 2023 GAAP and Non-GAAP cost of sales includes approximately $52 million of idle capacity charges. Fourth quarter 2022 GAAP and Non-GAAP cost of sales includes approximately $36 million of idle capacity charges. The increase in fourth quarter 2023 GAAP and Non-GAAP cost of sales as a percentage of total revenue was driven primarily by product mix.

* Full year 2023 GAAP and Non-GAAP cost of sales includes approximately $165 million of idle capacity charges. Full year 2022 GAAP and Non-GAAP cost of sales includes approximately $119 million of idle capacity charges and approximately $286 million in charges associated with the write-off of inventory and purchase commitments in excess of forecasted demand related to ADUHELM. The increase in full year 2023 GAAP and Non-GAAP cost of sales as a percentage of total revenue was driven primarily by product mix, particularly the year-over-year increase in contract manufacturing revenue.

* Fourth quarter 2023 GAAP and Non-GAAP R&D expense includes approximately $45 million related to Biogen's portion of R&D expense related to the LEQEMBI Collaboration and approximately $60 million in close out costs related to ADUHELM.

* Full year 2023 GAAP and Non-GAAP R&D expense includes approximately $186 million related to Biogen's portion of R&D expense related to the LEQEMBI Collaboration.

* Fourth quarter 2023 GAAP and Non-GAAP SG&A includes approximately $56 million related to Biogen's portion of SG&A expense related to the LEQEMBI Collaboration.

* Full year 2023 GAAP and Non-GAAP SG&A includes approximately $152 million related to Biogen's portion of SG&A expense related to the LEQEMBI Collaboration.

* Fourth quarter and full year 2023 GAAP restructuring expense was approximately $99 million and approximately $219 million, respectively.

Other Financial Highlights

* Fourth quarter 2023 GAAP and Non-GAAP collaboration profit sharing was a net expense of approximately $54 million, which includes approximately $53 million of net profit sharing expense related to Biogen's collaboration with Samsung Bioepis, and approximately $1 million of net profit sharing expense related to Biogen's collaboration with Sage Therapeutics related to the commercialization of ZURZUVAE in the U.S.

* Full year 2023 GAAP and Non-GAAP collaboration profit sharing was a net expense of approximately $219 million, which includes approximately $224 million of net profit sharing expense related to Biogen's collaboration with Samsung Bioepis, partially offset by net reimbursement of approximately $5 million from Sage Therapeutics related to the commercialization of ZURZUVAE in the U.S.

* Fourth quarter 2023 GAAP and Non-GAAP other expense was approximately $67 million and approximately $62 million, respectively, primarily driven by net interest expense.

* Full year 2023 GAAP other expense was approximately $316 million, primarily driven by net unrealized losses on strategic equity investments of approximately $270 million. Full year 2023 Non-GAAP other expense was approximately $14 million, primarily driven by foreign exchange rate losses, partially offset by net interest income.

* Fourth quarter 2023 GAAP and Non-GAAP effective tax rates were 14.7% and 17.0%, respectively. Fourth quarter 2022 GAAP and Non-GAAP effective tax rates were 9.0% and 14.9%, respectively.

* Full year 2023 GAAP and Non-GAAP effective tax rates were 10.4% and 15.2%, respectively. Full year 2022 GAAP and Non-GAAP effective tax rates were 17.6% and 15.3%, respectively.

Financial Position

* Fourth quarter 2023 net cash flow from operations was approximately $13 million, which includes a payment of approximately $393 million for equity-based compensation attributable to the postacquisition service period related to the Reata Pharmaceuticals, Inc. (Reata) transaction. Capital expenditures were approximately $65 million, and free cash flow, defined as net cash flow from operations less capital expenditures, was a net cash outflow of approximately $53 million.

* Full year 2023 net cash flow from operations was approximately $1.5 billion, and includes the aforementioned payment of approximately $393 million related to Reata. Capital expenditures were approximately $277 million, and free cash flow, defined as net cash flow from operations less capital expenditures, was approximately $1.3 billion.

* As of December 31, 2023, Biogen had cash, cash equivalents, and marketable securities totaling approximately $1.0 billion with approximately $6.9 billion in total debt, resulting in net debt of approximately $5.9 billion. This reflects all purchase payments related to the Reata transaction and a paydown of approximately $350 million of the $1 billion term loan related to our acquisition of Reata.

* No shares of the Company's common stock were repurchased in the fourth quarter of 2023. As of December 31, 2023, there was approximately $2.1 billion remaining under the share repurchase program authorized in October 2020.

* For the fourth quarter of 2023 the Company's GAAP weighted average diluted shares were 146 million. For full year 2023 the Company's GAAP weighted average diluted shares were 146 million.

Full Year 2024 Financial Guidance

For the full year 2024, Biogen expects a Non-GAAP diluted EPS guidance range as follows:

Full Year 2024 Guidance

Non-GAAP diluted EPS

$15.00 to $16.00

Reflecting growth of ~5% at the mid-point

*Versus reported full year 2023

While total revenue is expected to decline by a low- to mid-single digit percentage, Biogen expects core pharmaceutical revenue, defined as product revenue plus Biogen's 50% share of net LEQEMBI product revenue and cost of sales, including royalties, to be relatively flat for 2024 compared to 2023 as further declines in multiple sclerosis product revenue are expected to be offset by increases in revenue from new product launches. As of December 31, 2023, batch commitments related to the 2020 sale of Hillerød, Denmark manufacturing operations to FUJIFILM have been satisfied. Biogen expects contract manufacturing revenue to be significantly lower in 2024 compared to 2023.

As a result of these dynamics affecting revenue, along with lower expected idle capacity charges, Biogen expects an improvement in the cost of sales as a percentage of total revenue for 2024 compared to 2023.

For 2024 compared to 2023, Biogen expects operating income to grow at a low-double digit percentage. This is expected to be driven by improved cost of sales as a percentage of revenue, as well as lower operating expenses as a result of the Company's Fit for Growth program. The Fit for Growth program is expected to generate approximately $1 billion in gross savings and $800 million net of reinvestment by 2025. Since the program was initiated in 2023, approximately $200 million of savings have been achieved, and Biogen expects to realize approximately half of the overall net savings by the end of 2024 with the balance by the end of 2025. These amounts do not include Biogen's 50% share of sales and marketing expenses for the LEQEMBI Collaboration.

This guidance also assumes that foreign exchange rates as of February 9, 2024, will remain in effect for the remainder of the year, net of hedging activities. Other modeling considerations will be provided on the conference call and webcast.

This financial guidance does not include any impact from potential acquisitions or large business development transactions or pending and future litigation, as all are hard to predict, or any impact of potential tax or healthcare reform. Biogen may incur charges, realize gains or losses, or experience other events or circumstances in 2024 that could cause any of these assumptions to change and/or actual results to vary from this financial guidance.

Biogen does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the Company is unable to predict with reasonable certainty the financial impact of items such as the transaction, integration, and certain other costs related to acquisitions or large business development transactions; unusual gains and losses; potential future asset impairments; gains and losses from our equity security investments; and the ultimate outcome of pending or future significant litigation without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. For the same reasons, the Company is unable to address the significance of the unavailable information, which could be material to future results.

About Biogen

Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient's lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth.

https://investors.biogen.com/static-files/febfd7f7-108d-44c3-a122-e9611872030d

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN)

Pioneers in Rare Disease

Established in 1997, BioMarin is a world leader in developing and commercializing first- or best-in-class therapies for rare genetic diseases. We take pride in going where the science leads us, pioneering breakthrough treatments for debilitating and life-threatening conditions where we can significantly improve upon the current standard of care.

Our culture revolves around the ethos that no disease should go untreated, and our people are driven to discover, develop, and commercialize medicines that give patients, their families, and their caregivers hope where there was little or none. We fuel our R&D engine by looking for opportunities that align with our strengths and competencies. And we relentlessly pursue exciting, early-stage science that has the potential to change the course of disease.

Voted one of America's Best Midsize Employer by Forbes in 2019, our employees feel connected to their work at BioMarin because they believe in our purpose: the patient. The passion and dedication that our employees bring to work each day is a testament to the inspiration our patients provide, and the knowledge of the impact we can make in their lives.

BioMarin at a Glance

Headquarters

San Rafael, California

Founded

1997

Chairman and CEO

Jean-Jacques Bienaimé

Employees

3,000

Ticker

NASDAQ: BMRN

Revenue 2021

$1.8 billion

Net Income (Loss) 2021

($64.1 million)

Focus

Genetic disorders

Products

ROCTAVIAN[TM] (valoctocogene roxaparvovec-rvox)

VOXZOGO® (vosoritide)

PALYNZIQ® (pegvaliase-pqpz) Injection

BRINEURA® (cerliponase alfa)

VIMIZIM® (elosulfase alfa)

KUVAN® (sapropterin dihydrochloride)

NAGLAZYME® (galsulfase)

Clinical Pipeline

BMN 307 AAV Gene Therapy for PKU

BMN 331 AAV Gene Therapy for Hereditary Angiodema

Partners

Genzyme Corporation

https://www.biomarin.com/our-company/about-us/

24/4/2024

BioMarin Reports Record Financial Results for the First Quarter 2024

April 24, 2024

First Quarter 2024 Total Revenues of $649 Million (+9% Y/Y and +13% at Constant Currency Y/Y); GAAP Diluted Earnings per Share (EPS) of $0.46 (+70% Y/Y) and Non-GAAP Diluted Earnings per Share of $0.71 (+18% Y/Y)

VOXZOGO® Net Product Revenues of $153 Million in Q1'24 (+74% Y/Y); Children Treated Increased Over 100% Y/Y

For Full-year 2024 Guidance, Total Revenues Reaffirmed, Non-GAAP Operating Margin and Non-GAAP EPS Raised

R&D Prioritization Results in Acceleration of Three Highest Value Programs

Conference Call and Webcast Scheduled Today at 4:30 p.m. ET

SAN RAFAEL, Calif., April 24, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the quarter ended March 31, 2024.

"During the quarter, execution across our business led to double digit revenue growth, on a constant currency basis, and an 18% increase in non-GAAP diluted earnings per share. At the same time, we made rapid progress on advancing our strategic priorities for the year, including accelerating and maximizing the VOXZOGO opportunity, focusing R&D on the most productive assets, and increasing profitability," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "We were pleased with the outcome of our strategic R&D asset review, resulting in the acceleration and prioritization of the most potentially impactful medicines for patients. By advancing and focusing on these promising programs, we have taken decisive steps to drive value creation for all our stakeholders. This approach to delivering innovative therapies is an important part of BioMarin's operational transformation, and we look forward to sharing more details at Investor Day on September 4th."

Mr. Hardy continued, "Quarterly results were driven by strong demand for VOXZOGO, the only approved treatment for children with achondroplasia, and solid contributions from our established enzyme products. During the quarter, we made significant progress executing on our top priority to maximize and accelerate the reach of VOXZOGO, in achondroplasia and beyond. Globally, the number of children treated with VOXZOGO increased by over 500 from the prior quarter, and totaled over 3,100 by quarter end. In our largest single market, the U.S., we are realizing the tremendous opportunity ahead and expect U.S. uptake trends to continue. Our registration-enabling plans with VOXZOGO in hypochondroplasia, and ongoing discussions with health authorities to align on development plans for idiopathic short stature and pathway conditions are on-track, with all three studies expected to begin enrollment this year."

Financial Highlights:

Total Revenues for the first quarter of 2024 were $648.8 million, an increase of 9% compared to the same period in 2023. The increase in Total Revenues was primarily attributed to higher VOXZOGO sales volume driven by new patients initiating therapy across all regions. The increase was also due to higher PALYNZIQ product revenues primarily driven by sales volume growth in the U.S. The increase was partially offset by lower NAGLAZYME product revenues due to timing of orders in countries that place large government orders, particularly in the Middle East, as well as lower KUVAN product revenues attributed to continued generic competition as a result of the loss of market exclusivity.

GAAP Net Income increased by $37.8 million to $88.7 million in the first quarter of 2024 compared to the same period in 2023. The increase was primarily due to higher gross profit driven by increased VOXZOGO sales volume and a decrease in Other income (expense), net, related to an impairment charge in the first quarter of 2023, which did not recur in the first quarter of 2024. The increases were partially offset by higher spend in research and development (R&D) programs to support both early-stage research and clinical activities, including new indications with VOXZOGO, and higher spend in Selling, General and Administrative (SG&A) due to the continued support of global VOXZOGO market expansion and incremental administrative expenses in the quarter.

Non-GAAP Income increased by $23.9 million to $139.7 million in the first quarter of 2024 compared to the same period in 2023. The increased Non-GAAP Income was primarily due to higher gross profit driven by increased VOXZOGO revenues, partially offset by higher R&D expenses (as noted above).

1Q Update on 2024 Strategic Priorities

In the first quarter, BioMarin executed on its four strategic priorities, first outlined in January, and focused on value creation through accelerating growth, optimizing efficiencies and driving operational excellence.

Accelerate and maximize the VOXZOGO opportunity

During the first quarter, the number of children being treated with VOXZOGO for achondroplasia accelerated. In the period, more than 500 additional children began treatment with VOXZOGO, as compared to an increase of approximately 300 new treatment starts in the fourth quarter of 2023. Over 3,100 children were benefiting from VOXZOGO treatment across 43 active markets, as of the end of the first quarter. This represents a 102% increase in the number of children being treated with VOXZOGO, year-over-year.

In the largest single market, the U.S., more families with children under the age of 5 years sought treatment with VOXZOGO. This trend is expected to continue as real-world evidence, supported by VOXZOGO's extensive safety and efficacy profile, drives awareness and confidence for those families interested in treatment. With VOXZOGO now approved for children from infancy in most regions, families have the opportunity to benefit from longer treatment and potentially greater benefit.

Enrollment in the pivotal program with VOXZOGO for the treatment of children with hypochondroplasia continued. The observational run-in study is expected to complete enrollment by year-end, and the 52-week randomized, double-blind, placebo-controlled phase of the 80-participant clinical trial, is expected to start mid-year and is expected to complete enrollment in the first half of 2025.

During the quarter, BioMarin had productive engagement with the Food and Drug Administration to align on development programs in idiopathic short stature (ISS) and multiple genetic short stature pathway conditions (PC), and expects both studies to begin enrollment in the second half of 2024.

Establish ROCTAVIAN opportunity

During the quarter, reimbursement and market access challenges continued to impact the ability of interested patients to receive ROCTAVIAN treatment. For the remainder of 2024, BioMarin's global commercial team will continue to focus on key elements critical to supporting ROCTAVIAN uptake in the U.S., Italy and Germany. In April, BioMarin treated the first patient in Italy with ROCTAVIAN following the January price approval by the Italian Medicines Agency.

Focus R&D on the most productive assets

During the quarter, BioMarin completed a strategic portfolio assessment of R&D programs to determine which have the most transformative potential for patients and value creation for shareholders. With the combined focus on patient impact and commercial opportunity, three programs will be accelerated.

The programs, BMN 333, long-acting CNP for multiple growth-disorders, BMN 349, a first-in-class, oral therapeutic for AATD-associated liver disease, and BMN 351, BioMarin's next-generation oligonucleotide for DMD, all met the highest bar for advancement.

As a result of its prioritized portfolio, four programs will be discontinued, including BMN 331, BMN 255, BMN 355 and BMN 365. None of the programs were discontinued due to safety signals.

The additional programs in BioMarin's pipeline will move forward within the revised evaluation framework that provides a high bar for consistent and ongoing assessment to determine if they fit in the company's focused portfolio.

Accelerate EPS growth and expand margins

Strong performance in the first quarter underscored BioMarin's execution of its financial strategy to drive year-over-year Non-GAAP Operating Margin expansion and Non-GAAP EPS growth twice as fast as revenues.

The company reaffirmed full-year 2024 Total Revenue guidance, and raised Non-GAAP Operating Margin and Non-GAAP Diluted EPS guidance. The improved profitability guidance is a result of the planned reduction in operating expenses for the discontinued early-stage R&D programs, in the range of $50 million and $60 million. The planned reductions in R&D expense were partially off-set by planned increases in operating expenses for the accelerated programs, resulting in planned net reductions to 2024 operating expenses of $35 million to $40 million, driving the updated guidance. BioMarin's updated full-year 2024 guidance does not reflect the impact of potential additional future business decisions that may result from its ongoing strategic business review.

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)

Three Months Ended

March 31,

2024

2023

% Change

Total Revenues

$648.8

$596.4

9 %

Net Product Revenues by Product:

VIMIZIM ®

$192.6

$189.2

2 %

VOXZOGO

$152.9

$87.8

74 %

NAGLAZYME ®

$105.6

$123.0

(14) %

PALYNZIQ ®

$75.7

$62.4

21 %

BRINEURA ®

$39.0

$39.1

- %

KUVAN ®

$35.9

$50.5

(29) %

ALDURAZYME ®

$35.3

$34.4

3 %

ROCTAVIAN ®

$0.8

$-

nm

GAAP Net Income

$88.7

$50.9

74 %

Non-GAAP Income (1)

$139.7

$115.8

21 %

GAAP Operating Margin % (2)

13.6 %

10.5 %

Non-GAAP Operating Margin % (2)

23.8 %

22.4 %

GAAP Diluted Earnings per Share (EPS)

$0.46

$0.27

70 %

Non-GAAP Diluted EPS (3)

$0.71

$0.60

18 %

March 31, 2024

December 31, 2023

Total cash, cash equivalents & investments

$ 1,667.1

$ 1,684.9

(1)

Non-GAAP Income is defined by the company as reported GAAP Net Income, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items. The company also includes a Non-GAAP adjustment for the estimated income tax impact of reconciling items. Refer to Non-GAAP Information beginning on page 9 of this press release for a complete discussion of the company's Non-GAAP financial information and reconciliations to the comparable information reported under U.S. GAAP.

(2)

GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations divided by Total Revenues. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain specified items divided by Total Revenues.

(3)

Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP diluted weighted-average shares outstanding. Non-GAAP weighted-average diluted shares outstanding is defined by the company as GAAP weighted-average diluted shares outstanding, adjusted to include any common shares issuable under the company's equity plans and convertible debt in periods when they are dilutive under Non-GAAP.

nm

Not meaningful

2024 Full-Year Financial Guidance (in millions, except % and EPS amounts) (Updated)

BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from our strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors.

About BioMarin

Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's robust research and development capabilities have resulted in multiple innovative commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit

www.biomarin.com

.

https://investors.biomarin.com/news/news-details/2024/BioMarin-Reports-Record-Financial-Results-for-the-First-Quarter-2024/default.aspx

Celgene Corporation (CELG)

11/20/2019

Bristol-Myers Squibb Completes Acquisition of Celgene, Creating a Leading Biopharma Company

CATEGORY: Corporate/Financial News

NEW YORK--(BUSINESS WIRE)--Bristol-Myers Squibb Company (NYSE:BMY) announced today that it has completed its acquisition of Celgene Corporation (NASDAQ:CELG) following the receipt of regulatory approval from all government authorities required by the merger agreement and, as announced on April 12, 2019, approval by Bristol-Myers Squibb and Celgene stockholders.

Upon completion of the acquisition, pursuant to the terms of the merger agreement, Celgene became a wholly owned subsidiary of Bristol-Myers Squibb Company. Under the terms of the merger, Celgene shareholders received for each share, 1.00 share of Bristol-Myers Squibb common stock, $50.00 in cash without interest and one tradeable Contingent Value Right (CVR), which will entitle the holder to receive a payment of $9.00 in cash if certain future regulatory milestones are achieved. Celgene common stock ceased trading as of the close of trading today. On November 21, 2019, newly issued Bristol-Myers Squibb shares and CVRs will commence trading on the New York Stock Exchange, with the CVRs trading under the symbol "BMYRT."

"This is an exciting day for Bristol-Myers Squibb as we bring together the leading science, innovative medicines and incredible talent of Bristol-Myers Squibb and Celgene to create a leading biopharma company," said Giovanni Caforio, M.D., Chairman and Chief Executive Officer of Bristol-Myers Squibb. "With our leading franchises in oncology, hematology, immunology and cardiovascular disease, and one of the most diverse and promising pipelines in the industry, I know we will deliver on our vision of transforming patients' lives through science. I am excited about the opportunities for our current employees and the new colleagues that we welcome to the Company as we work together to deliver innovative medicines to patients."

Since announcing the transaction on January 3, 2019, there have been a number of tangible advancements toward delivering on the key value drivers for the merger, including: further progress relating to the patent estate for REVLIMID®, the U.S. Food and Drug Administration (FDA) approval of INREBIC® (fedratinib) for the treatment of certain forms of myelofibrosis, the U.S. FDA approval of REBLOZYL® (luspatercept-aamt) for the treatment of anemia in certain adult patients with beta thalassemia, and regulatory filings of luspatercept and ozanimod in the U.S. and Europe. The Company has also made substantial progress toward the planning of a successful integration. For an overview of the combined company and the milestones achieved while the transaction was pending, visit www.bestofbiopharma.com.

OTEZLA® Divestiture Update

As announced on August 26, 2019, in connection with the regulatory approval process for the transaction, Celgene entered into an agreement to divest the global rights to OTEZLA® (apremilast) to Amgen (NASDAQ:AMGN) for $13.4 billion in cash following the closing of the merger with Bristol-Myers Squibb. On November 15, 2019, Bristol-Myers Squibb announced that the U.S. Federal Trade Commission (FTC) accepted the proposed consent order in connection with the pending merger of Bristol-Myers Squibb and Celgene, thereby permitting the parties to close the merger. Bristol-Myers Squibb expects the OTEZLA divestiture to be completed promptly following the closing of the merger and plans to prioritize the use of proceeds for debt reduction.

Accelerated Share Repurchase Program

Bristol-Myers Squibb also announced that its Board of Directors has authorized the repurchase of $7 billion of Bristol-Myers Squibb common stock.

In connection with this authorization, Bristol-Myers Squibb has entered into accelerated share repurchase (ASR) agreements with Morgan Stanley & Co. LLC and Barclays Bank PLC to repurchase, in aggregate, $7 billion of Bristol-Myers Squibb common stock. Bristol-Myers Squibb expects to fund the repurchase with cash on-hand. Approximately 80 percent of the shares to be repurchased under the transaction will be received by Bristol-Myers Squibb on November 27, 2019. The total number of shares ultimately repurchased under the program will be determined upon final settlement and will be based on a discount to the volume-weighted average price of Bristol-Myers Squibb's common stock during the ASR period. Bristol-Myers Squibb anticipates that all repurchases under the ASR will be completed by the end of the second quarter of 2020.

Board Appointments

As previously announced, in connection with the closing of the transaction, Michael Bonney, Dr. Julia A. Haller and Phyllis Yale have joined the Bristol-Myers Squibb Board of Directors, expanding the size of the Board from 11 to 14. Mr. Bonney and Dr. Haller served on Celgene's Board of Directors until the closing of the transaction. All three new directors bring valuable skill sets and significant experience relevant to Bristol-Myers Squibb's business.

Advisors

Morgan Stanley & Co. LLC is serving as lead financial advisor to Bristol-Myers Squibb, and Evercore and Dyal Co. LLC are serving as financial advisors to Bristol-Myers Squibb. Kirkland & Ellis LLP is serving as Bristol-Myers Squibb's legal counsel. J.P. Morgan Securities LLC is serving as lead financial advisor and Citi is acting as financial advisor to Celgene. Wachtell, Lipton, Rosen & Katz is serving as legal counsel to Celgene.

About Bristol-Myers Squibb

Bristol-Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol-Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

Cautionary Statement Regarding Forward-Looking Statements

This press release contains certain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, regarding, among other things, the research, development and commercialization of pharmaceutical products, Bristol-Myers Squibb's acquisition of Celgene (the "Merger"), the pending sale of OTEZLA (the "Divestiture," and together with the Merger, the "Transaction"), and the execution of the ASR program. These statements may be identified by the fact they use words such as "should," "could," "expect," "anticipate," "estimate," "target," "may," "project," "guidance," "intend," "plan," "believe," "will" and other words and terms of similar meaning and expression in connection with any discussion of future operating or financial performance, although not all forward-looking statements contain such terms. One can also identify forward-looking statements by the fact that they do not relate strictly to historical or current facts. Such forward-looking statements are based on historical performance and current expectations and projections about Bristol-Myers Squibb's future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond Bristol-Myers Squibb's control and could cause Bristol-Myers Squibb's future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. Such risks, uncertainties and other matters include, but are not limited to, Bristol-Myers Squibb successfully using proceeds from the Divestiture; the combined company will have substantial indebtedness following the completion of the Transaction; Bristol-Myers Squibb is unable to achieve the synergies and value creation contemplated by the Merger; Bristol-Myers Squibb is unable to promptly and effectively integrate Celgene's businesses; management's time and attention is diverted on transaction related issues; disruption from the transaction makes it more difficult to maintain business, contractual and operational relationships; the credit ratings of the combined company decline following the Transaction; legal proceedings are instituted against Bristol-Myers Squibb, Celgene or the combined company; Bristol-Myers Squibb, Celgene or the combined company is unable to retain key personnel; and the announcement or the consummation of the Transaction and ASR program has a negative effect on the market price of the capital stock of the combined company or on the combined company's operating results. No forward-looking statement can be guaranteed.

Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol-Myers Squibb's business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol-Myers Squibb's Annual Report on Form 10-K for the year ended December 31, 2018, as updated by its subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this press release are made only as of the date of this press release and except as otherwise required by applicable law, Bristol-Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

https://news.bms.com/news/corporate-financial/2019/Bristol-Myers-Squibb-Completes-Acquisition-of-Celgene-Creating-a-Leading-Biopharma-Company/default.aspx

About

COMMITTED TO IMPROVING THE LIVES OF PATIENTS WORLDWIDE

At Celgene, we seek to deliver truly innovative and life-changing drugs for our patients. Our vision as a company is to build a major global biopharmaceutical corporation while focusing on the discovery, the development, and the commercialization of products for the treatment of cancer and other severe, immune, inflammatory conditions.

"At Celgene, we seek to deliver truly innovative and life-changing drugs for our patients."

There are more than 300 clinical trials at major medical centers using compounds from Celgene. Investigational compounds are being studied for patients with incurable hematological and solid tumor cancers, including multiple myeloma, myelodysplastic syndromes, chronic lymphocytic leukemia (CLL), non-Hodgkin's lymphoma (NHL), triple-negative breast cancer and pancreatic cancer. As committed as we are to clinical accomplishment, we are equally committed to patient support, which is a guiding principle at Celgene. We believe all who can benefit from our discoveries should have the opportunity to do so. Celgene puts patients first with industry-leading programs that provide information, support and access to our innovative therapies.

https://www.celgene.com/about/

31/10/2019

Celgene Reports Third Quarter 2019 Operating and Financial Results

- Strong total revenue of $4.5 billion, increased 16% Y/Y driven by volume

- INREBIC ® (fedratinib) granted FDA approval in myelofibrosis; EU MAA submission expected by year-end 2019

- Expected Q4 regulatory updates include: Dec. 4, 2019 FDA PDUFA date for luspatercept in transfusion-dependent beta-thalassemia; BLA submission for liso-cel in R/R B-cell NHL on-track for Q4:19

- Key data presentations at ASH, including data from the pivotal TRANSCEND [TM] NHL-001 trial with liso-cel in R/R large B-cell NHL

SUMMIT, N.J.--(BUSINESS WIRE)-- Celgene Corporation (NASDAQ: CELG) reported third quarter 2019 total revenue of $4,520 million, a 16 percent increase compared to $3,892 million in the third quarter of 2018.

Based on U.S. GAAP (Generally Accepted Accounting Principles), Celgene reported net income of $1,691 million and diluted earnings per share (EPS) of $2.32 for the third quarter of 2019. For the third quarter of 2018, GAAP net income was $1,082 million and diluted EPS was $1.50.

Adjusted net income for the third quarter of 2019 increased 33 percent to $2,184 million compared to $1,645 million in the third quarter of 2018. For the same period, adjusted diluted EPS increased 31 percent to $2.99 from $2.29.

"Across functions and around the world, our teams delivered outstanding third quarter results," said Mark J. Alles, Chairman and Chief Executive Officer of Celgene Corporation. "We are continuing to advance multiple high-potential medicines toward regulatory approvals and look forward to closing the Bristol-Myers Squibb transaction by the end of the year."

Third Quarter 2019 Financial Highlights

Unless otherwise stated, all comparisons are for the third quarter of 2019 compared to the third quarter of 2018. The adjusted operating expense categories presented below exclude share-based employee compensation expense and collaboration-related upfront expense. Please see the attached Use of Non-GAAP Financial Measures and Reconciliation of GAAP to Adjusted Net Income for further information relevant to the interpretation of adjusted financial measures and reconciliations of these adjusted financial measures to the most comparable GAAP measures, respectively.

Net Product Sales Performance

REVLIMID ® sales for the third quarter were $2,770 million, an increase of 13 percent year-over-year. U.S. sales were $1,902 million and international sales were $868 million, an increase of 14 percent and 11 percent year-over-year, respectively. REVLIMID ® sales growth was driven primarily by the adoption of triplet therapy for myeloma resulting in increases in treatment duration and market share.

POMALYST ® /IMNOVID ® sales for the third quarter were $664 million, an increase of 29 percent year-over-year. U.S. sales were $469 million and international sales were $195 million, an increase of 31 percent and 25 percent year-over-year, respectively. POMALYST ® /IMNOVID ® sales growth was driven primarily by the adoption of triplet therapy for myeloma resulting in increases in treatment duration and market share.

OTEZLA ® sales for the third quarter were $547 million, an increase of 27 percent year-over-year. U.S. sales were $445 million and international sales were $102 million, an increase of 28 percent and 21 percent year-over-year, respectively. OTEZLA ® sales growth in the U.S. was driven by increase in demand, while international sales were driven by continued uptake in key markets.

ABRAXANE ® sales for the third quarter were $318 million, an increase of 10 percent year-over-year. U.S. sales were $206 million and international sales were $112 million, an increase of 18 percent and a decrease of 2 percent year-over-year, respectively. ABRAXANE ® sales growth was driven primarily by increased demand due to immuno-oncology (IO) combinations in non-small cell lung cancer (NSCLC) and triple-negative breast cancer (TNBC).

In the third quarter, all other product sales, which include INREBIC ®, IDHIFA ® , THALOMID ® , ISTODAX ® , VIDAZA ® and an authorized generic version of VIDAZA ® drug product primarily sold in the U.S., were $219 million compared to $208 million in the third quarter of 2018.

Research and Development (R&D)

On a GAAP basis, R&D expenses were $1,167 million for the third quarter of 2019 compared to $1,081 million for the same period in 2018. Adjusted R&D expenses were $928 million for the third quarter of 2019 compared to $948 million for the third quarter of 2018. The decrease in adjusted R&D expense was driven by reductions in expenses related to certain collaboration arrangements and regulatory submission-related work on multiple programs. Additional R&D expenses (only included on a GAAP basis) increased in 2019, as outlined in the attached Reconciliation of GAAP to Adjusted Net Income.

Selling, General and Administrative (SG&A)

On a GAAP basis, SG&A expenses were $781 million for the third quarter of 2019 compared to $746 million for the same period in 2018. Adjusted SG&A expenses were $700 million for the third quarter of 2019 compared to $642 million for the third quarter of 2018. The increase was driven primarily by increased pre-launch marketing-related expenses. Additional SG&A expense (only included on a GAAP basis) decreased in 2019, as outlined in the attached Reconciliation of GAAP to Adjusted Net Income.

Cash, Cash Equivalents, Marketable Debt Securities and Publicly-Traded Equity Securities

Operating cash flow was $2.2 billion in the third quarter of 2019, compared to $1.9 billion for the third quarter of 2018. Celgene ended the quarter with approximately $10.9 billion in cash, cash equivalents, marketable debt securities and publicly-traded equity securities.

Portfolio Updates

INREBIC ® (fedratinib):

In August, Celgene announced that the U.S. Food and Drug Administration (FDA) approved INREBIC ® (fedratinib) for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis

The Marketing Authorization Application (MAA) submission in the European Union is planned by year-end 2019

Luspatercept 1 :

The U.S. FDA accepted the Biologics License Application (BLA) for luspatercept for the treatment of anemia in adult patients with beta-thalassemia who require regular red blood cell (RBC) transfusions and set a Prescription Drug User Fee Act (PDUFA) date of December 4, 2019

The U.S. FDA accepted the BLA for luspatercept for the treatment of adult patients with very low to intermediate-risk myelodysplastic syndromes (MDS)-associated anemia who have ring sideroblasts and require regular RBC transfusions and set a PDUFA date of April 4, 2020

The MAA for luspatercept for the treatment of adult patients with beta-thalassemia and MDS has been accepted for review by the European Medicines Agency (EMA)

Liso-cel (JCAR017):

The BLA submission for liso-cel in patients with relapsed or refractory large B-cell lymphoma after at least 2 prior therapies is on-track for the fourth quarter of 2019

Ide-cel (bb2121) 2 :

An update from the pivotal KarMMa [TM] trial in patients with relapsed and/or refractory multiple myeloma (RRMM) is expected by year-end 2019. The BLA submission for ide-cel in 4L+ multiple myeloma is expected in the first half of 2020

The phase II KarMMa-2 and phase III KarMMa-3 trials in patients with 2L and 3L+ multiple myeloma, respectively, are continuing to enroll

Initiation of a newly diagnosed multiple myeloma (NDMM) trial is planned for the fourth quarter of 2019

CC-486:

In September, Celgene announced that the phase III QUAZAR ® AML-001 trial evaluating CC-486 as maintenance therapy in patients with newly diagnosed acute myeloid leukemia (AML) who achieved first complete response (CR) or complete response with incomplete blood count recovery (CRi) with induction chemotherapy met the primary endpoint of prolonged overall survival (OS). Celgene plans regulatory submissions beginning in the first half of 2020. Data from QUAZAR ® AML-001 will be presented at a future medical meeting

Ozanimod:

The U.S. FDA accepted the New Drug Application (NDA) for ozanimod for the treatment of patients with relapsing forms of multiple sclerosis (RMS) and set a PDUFA date of March 25, 2020

The EMA accepted the MAA for ozanimod for the treatment of patients with relapsing-remitting multiple sclerosis (RRMS). A regulatory decision is expected in the first half of 2020

Data from the phase III TRUE NORTH [TM] trial in patients with ulcerative colitis (UC) is expected in mid-2020

At the 61 st ASH annual meeting in December, select anticipated data presentations include:

Data from the pivotal TRANSCEND [TM] NHL-001 trial evaluating liso-cel in patients with relapsed/refractory B-cell non-Hodgkin lymphoma (NHL), which includes diffuse large B-cell lymphoma (DLBCL);

Updated data, including minimal residual disease (MRD), from the ongoing phase I/II TRANSCEND CLL-004 trial evaluating liso-cel in a heavily pretreated patient population with high-risk chronic lymphocytic leukemia (CLL);

Initial results from the phase II PILOT trial evaluating liso-cel as second-line treatment in patients with transplant noneligible (TNE) relapsed and/or refractory NHL;

Data from the outpatient treatment of liso-cel in multiple ongoing clinical trials in patients with relapsed/refractory B-cell NHL;

Initial data from the phase II trial evaluating luspatercept in patients with myelofibrosis;

Updated data from the phase I trial evaluating bb21217 in patients with RRMM;

First clinical data from a phase I trial evaluating CC-93269, a T cell bispecific antibody targeting B-cell maturation antigen (BCMA) in patients with RRMM; and,

First clinical data from a phase I trial evaluating CELMoD ® agent CC-90009 in patients with relapsed or refractory AML

1 In collaboration with Acceleron Pharma 2 In collaboration with bluebird bio

Transaction Update

In June, Bristol-Myers Squibb announced the planned divestiture of OTEZLA® (apremilast) in light of concerns raised by the U.S. Federal Trade Commission ("FTC"). In August, Celgene entered into an agreement with Amgen under which Amgen would acquire the OTEZLA® (apremilast) product line and related intellectual property, including any patents that primarily cover apremilast, and other specified assets and liabilities related to the OTEZLA® (apremilast) product line for $13.4 billion in cash (the "OTEZLA® Divestiture"), which represents an important step towards completion of the pending merger between Bristol-Myers Squibb and Celgene. The closing of the OTEZLA® Divestiture is contingent on Bristol-Myers Squibb and Celgene entering into a consent decree with the Federal Trade Commission (FTC) in connection with their pending merger, the closing of the pending merger between Bristol-Myers Squibb and Celgene, and the satisfaction of other customary closing conditions. The pending merger between Bristol-Myers Squibb and Celgene is expected to close by the end of 2019, subject to the FTC's acceptance of a consent order and the satisfaction of customary closing conditions.

https://s22.q4cdn.com/728481125/files/doc_financials/2019/q3/Q3-2019-Earnings-Press-Release_FINAL_APPROVED_with-rec-tables.pdf

Gilead Sciences (NASDAQ: GILD)

Leadership

The Gilead Leadership Team and Board of Directors provide the necessary guidance to execute our mission and realize our vision.

Gilead is dedicated to developing innovative medicines for life-threatening illnesses. As we pursue this goal, we strive for positive social and environmental change within our company and our supply chain. Our Corporate Social Responsibility (CSR) program is built to focus on patients, society, the planet and our business practices.

Gilead's management approach is designed to create a sustainable future that reflects our integrity and commitment to excellence. Year after year, internal and external stakeholders work to evaluate the effectiveness of our management approach. They identify strengths and opportunities that become guideposts as we reach for new breakthroughs and look at what's next.

https://www.gilead.com/about/leadership

3/8/2023

Gilead Sciences Announces Second Quarter 2023 Financial Results

Product Sales Excluding Veklury Increased 11% Year-Over-Year to $6.3 billion

Biktarvy Sales Increased 17% Year-Over-Year to $3.0 billion

Oncology Sales Increased 38% Year-Over-Year to $728 million

Net Income Reflects $525 million Legal Settlement Accrual ($0.32 per share)

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) announced today its results of operations for the second quarter of 2023.

"It was another strong quarter for Gilead, with continued commercial and clinical momentum," said Daniel O'Day, Gilead's Chairman and Chief Executive Officer. "11% year-over-year growth across our base business was driven by our diverse portfolio of therapies for HIV, Oncology, and Liver Disease. We received positive regulatory updates for six of our therapies and presented a large body of data on our pipeline, reinforcing our growing potential to help more patients and communities worldwide."

Second Quarter 2023 Financial Results

Total second quarter 2023 revenue increased 5% to $6.6 billion compared to the same period in 2022, primarily driven by increased sales in HIV and Oncology, partially offset by lower Veklury® (remdesivir) sales.

Diluted Earnings Per Share ("EPS") decreased to $0.83 for the second quarter of 2023 compared to $0.91 for the same period in 2022, mainly driven by a $525 million litigation accrual for settlements with certain plaintiffs in the HIV antitrust litigation, representing an unfavorable $0.32 impact to diluted EPS, as well as other higher operating costs and tax expense, partially offset by higher product revenues and unrealized gains on equity investments compared to unrealized losses in 2022.

Non-GAAP diluted EPS decreased to $1.34 for the second quarter of 2023 compared to $1.58 for the same period in 2022, primarily driven by the litigation accrual referenced earlier, representing an unfavorable $0.32 impact to non-GAAP diluted EPS, as well as other higher operating costs, partially offset by higher product revenues.

As of June 30, 2023, Gilead had $8.0 billion of cash, cash equivalents and marketable debt securities, up from $7.6 billion as of December 31, 2022.

During the second quarter of 2023, Gilead generated $2.3 billion in operating cash flow.

During the second quarter of 2023, Gilead paid dividends of $944 million and repurchased $150 million of common stock.

Product Sales Performance

Total second quarter 2023 product sales increased 7% to $6.6 billion compared to the same period in 2022. Total product sales, excluding Veklury, increased 11% to $6.3 billion in the second quarter of 2023 compared to the same period in 2022, primarily due to increased sales related to HIV, Cell Therapy and Trodelvy® (sacituzumab govitecan-hziy).

HIV product sales increased 9% to $4.6 billion in the second quarter of 2023 compared to the same period in 2022, primarily driven by favorable pricing dynamics and higher demand, partially offset by lower channel inventory.

Biktarvy® (bictegravir 50mg/emtricitabine 200mg ("FTC")/tenofovir alafenamide 25mg ("TAF")) sales increased 17% year-over-year in the second quarter of 2023, primarily driven by higher demand and favorable pricing dynamics, partially offset by lower channel inventory.

Descovy® (FTC 200mg/TAF 25mg) sales increased 12% year-over-year in the second quarter of 2023, primarily driven by favorable pricing dynamics and higher demand, partially offset by lower channel inventory.

The Liver Disease portfolio sales, which includes chronic hepatitis C virus ("HCV"), chronic hepatitis B virus ("HBV"), and chronic hepatitis delta virus ("HDV"), increased 4% to $711 million in the second quarter of 2023 compared to the same period in 2022. The increase was primarily driven by higher demand, partially offset by unfavorable pricing dynamics.

Cell Therapy product sales increased 27% to $469 million in the second quarter of 2023 compared to the same period in 2022.

Yescarta® (axicabtagene ciloleucel) sales increased 29% year-over-year to $380 million in the second quarter of 2023, primarily driven by strong demand in the second- and third-line settings for relapsed or refractory ("R/R") large B-cell lymphoma ("LBCL").

Tecartus® (brexucabtagene autoleucel) sales increased 21% year-over-year to $88 million in the second quarter of 2023, primarily driven by increased demand in R/R adult acute lymphoblastic leukemia ("ALL") and R/R mantle cell lymphoma ("MCL").

Trodelvy sales increased by 63% to $260 million in the second quarter of 2023 compared to the same period in 2022, primarily driven by growing adoption in pre-treated HR+/HER2- metastatic breast cancer ("mBC") in the United States.

Veklury sales decreased by 43% to $256 million for the second quarter of 2023 compared to the same period in 2022, primarily driven by lower rates of COVID-19 related hospitalizations in all regions. Veklury sales generally reflect COVID-19 related rates and severity of infections and hospitalizations, as well as the availability, uptake and effectiveness of vaccinations and alternative treatments for COVID-19.

Second Quarter 2023 Product Gross Margin, Operating Expenses and Effective Tax Rate

Product gross margin was 78.0% for the second quarter of 2023 compared to 76.5% for the same period in 2022.

Non-GAAP product gross margin was 86.9% for the second quarter of 2023 compared to 85.6% in the same period in 2022.

Research and development ("R&D") expenses and non-GAAP R&D expenses for the second quarter of 2023 were $1.4 billion compared to $1.1 billion in the same period in 2022. The increases in GAAP and non-GAAP R&D expenses were primarily driven by higher clinical activities.

Acquired in-process R&D ("IPR&D") expenses for the second quarter of 2023 were $236 million, primarily driven by the acquisition of XinThera, Inc. ("XinThera") and the expanded collaboration with Arcus Biosciences, Inc. ("Arcus").

Selling, general and administrative ("SG&A") expenses for the second quarter of 2023 were $1.8 billion compared to $1.4 billion in the same period in 2022. Non-GAAP SG&A expenses for the second quarter of 2023 were $1.8 billion compared to $1.3 billion in the same period in 2022. The increases in GAAP and non-GAAP SG&A expenses were primarily driven by the litigation accrual referenced earlier, as well as increased commercial activities in Oncology and HIV, partially offset by lower corporate expenses.

The effective tax rate ("ETR") for the second quarter of 2023 was 34.6% compared to 24.5% for the same period in 2022, primarily driven by a remeasurement of certain deferred tax liabilities. Non-GAAP ETR for the second quarter of 2023 was 21.0% compared to 19.3% for the same period in 2022.

Guidance and Outlook

For the full-year, Gilead expects:

Total product sales between $26.3 billion and $26.7 billion, compared to $26.0 billion and $26.5 billion previously.

Total product sales, excluding Veklury, between $24.6 billion and $25.0 billion, compared to $24.0 billion and $24.5 billion previously.

Total Veklury sales of approximately $1.7 billion, compared to approximately $2.0 billion previously.

Diluted earnings per share between $4.50 and $4.85, compared to $4.75 and $5.15 previously.

Non-GAAP diluted earnings per share between $6.45 and $6.80, compared to $6.60 and $7.00 previously.

Additional information and a reconciliation between GAAP and non-GAAP financial information for the 2023 guidance is provided in the accompanying tables. Also see the Forward-Looking Statements described below. The financial guidance is subject to a number of risks and uncertainties, including uncertainty around the duration and magnitude of the COVID-19 pandemic.

Key Updates Since Our Last Quarterly Release

Virology

Received U.S. Food and Drug Administration ("FDA") and European Commission ("EC") approval to extend the use of Veklury to treat COVID-19 in people with severe renal impairment, including those on dialysis.

Presented data on Biktarvy at the International AIDS Society Conference that further demonstrate the safety and efficacy profile in different subgroups of people with HIV, such as virologically suppressed pregnant women. Also presented patient-reported outcomes from the Phase 2/3 CAPELLA study of lenacapavir in heavily treatment-experienced people with HIV as well as data from use of oral lenacapavir as a bridging regimen. Note that the use of lenacapavir for oral bridging is not approved by any regulatory authority.

Presented new long-term data at the European Association for the Study of the Liver Congress 2023 from the MYR301 Phase 3 trial evaluating bulevirtide for HDV, showing improved response rates at Week 96 compared to Week 48. Additionally, abstracts across viral hepatitis and liver fibrosis were highlighted.

Received full marketing authorization from the EC for Hepcludex® (bulevirtide) for the treatment of adults with chronic HDV and compensated liver disease. Hepcludex was initially granted conditional marketing authorization in July 2020. Bulevirtide remains the only approved treatment for HDV in the EU and is not approved in the U.S.

Announced partnerships with the Clinton Health Access Initiative and Penta to improve treatment and adherence rates among children with HIV in low and middle income countries.

Oncology

Received EC approval for Trodelvy as monotherapy for the treatment of adult patients with unresectable or metastatic HR+/HER2- mBC who have received endocrine-based therapy, and at least two additional systemic therapies in the advanced setting.

Presented longer-term overall survival ("OS") data from the Phase 3 TROPiCS-02 study evaluating Trodelvy in pre-treated HR+/HER2- mBC at the 2023 American Society of Clinical Oncology ("ASCO") meeting, demonstrating durable and clinically meaningful improvement in median OS versus comparator chemotherapy. Data were also presented from a Phase 2 trial evaluating Trodelvy as a potential therapy in advanced endometrial cancer.

Presented OS data at ASCO from the Phase 3 ZUMA-7 trial of Yescarta in second-line R/R LBCL, which demonstrated significantly longer OS versus standard of care. Additionally, real-world evidence data for Tecartus in MCL were reported, which showed consistently high complete response and overall response rates, regardless of the type of prior treatment received.

Presented data at the European Hematology Association Annual Congress evaluating Yescarta, Tecartus, and magrolimab in a number of hematologic malignancies.

Received a recommendation from the National Institute for Health and Care Excellence in the United Kingdom for use of Yescarta in the second-line setting for diffuse LBCL and high-grade B-cell lymphoma, and Tecartus in R/R B-cell precursor ALL in England's National Health Service.

Announced, through Fosun Kite Biotechnology Co., Ltd., a joint venture between Kite and Shanghai Fosun Pharmaceutical (Group) Co., Ltd., the approval of axicabtagene ciloleucel (under the trade name Yikaida®) by the China National Medical Products Administration for the treatment of adult patients with R/R LBCL who failed first-line immunochemotherapy or relapsed within 12 months after first-line immunochemotherapy.

Completed the transfer of Yescarta's marketing authorization in Japan from Daiichi Sankyo Co., Ltd. ("Daiichi Sankyo") to Gilead Sciences K.K.

Announced data from an interim analysis at ASCO from the Phase 2 ARC-7 study of domvanalimab, zimberelimab and etrumadenant in first-line, metastatic PD-L1-high non-small cell lung cancer, demonstrating consistent improvement in progression-free survival and a clinically meaningful reduction in the risk of progression or death in the domvanalimab-containing arms, as compared to the zimberelimab monotherapy arm.

Announced the Phase 3 ENHANCE trial of magrolimab in combination with azacitidine in higher-risk myelodysplastic syndromes was discontinued due to futility based on a planned analysis. Data from the trial will be presented at an upcoming medical meeting.

Announced the acquisition of XinThera, adding additional pipeline assets including rights to a portfolio of small molecule inhibitors targeting PARP1 for oncology as well as MK2 for inflammatory diseases.

Inflammation

Announced expansion of the Arcus collaboration to include research programs in inflammatory diseases.

Corporate

Appointed Cindy Perettie as Executive Vice President of Kite, who joins with more than 20 years of scientific and commercial leadership experience in global biopharmaceutical organizations.

The company's Board of Directors declared a quarterly dividend of $0.75 per share of common stock for the third quarter of 2023. The dividend is payable on September 28, 2023, to stockholders of record at the close of business on September 15, 2023. Future dividends will be subject to Board approval.

Certain amounts and percentages in this press release may not sum or recalculate due to rounding.

Conference Call

At 2:00 p.m. Pacific Time today, Gilead will host a conference call to discuss Gilead's results. A live webcast will be available on http://investors.gilead.com and will be archived on www.gilead.com for one year.

Non-GAAP Financial Information

The information presented in this document has been prepared in accordance with U.S. generally accepted accounting principles ("GAAP"), unless otherwise noted as non-GAAP. Management believes non-GAAP information is useful for investors, when considered in conjunction with Gilead's GAAP financial information, because management uses such information internally for its operating, budgeting and financial planning purposes. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of Gilead's operating results as reported under GAAP. Non-GAAP financial information generally excludes acquisition-related expenses including amortization of acquired intangible assets and inventory step-up charges, and other items that are considered unusual or not representative of underlying trends of Gilead's business, fair value adjustments of equity securities and discrete and related tax charges or benefits associated with changes in tax related laws and guidelines. Although Gilead consistently excludes the amortization of acquired intangible assets from the non-GAAP financial information, management believes that it is important for investors to understand that such intangible assets were recorded as part of acquisitions and contribute to ongoing revenue generation.Non-GAAP measures may be defined and calculated differently by other companies in the same industry. Reconciliations of the non-GAAP financial measures to the most directly comparable GAAP financial measures are provided in the accompanying tables.

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis, coronavirus disease 2019 ("COVID-19"), and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

For full release:

https://www.gilead.com/news-and-press/press-room/press-releases/2023/8/gilead-sciences-announces-second-quarter-2023-financial-results

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN)

Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to seven FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye disease, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, infectious diseases, pain and rare diseases.

Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite ® technologies, such as VelocImmune ® which produces optimized fully-human antibodies, and ambitious research initiatives such as the Regeneron Genetics Center ® , which is conducting one of the largest genetics sequencing efforts in the world.

https://www.regeneron.com/about

2/2/2024

Regeneron Reports Fourth Quarter and Full Year 2023 Financial and Operating Results

Fourth quarter 2023 revenues increased 1% to $3.43 billion versus fourth quarter 2022; excluding RonapreveTM(a)(b), revenues increased 14%

Full year 2023 revenues increased 8% to $13.12 billion versus full year 2022; excluding Ronapreve(a), revenues increased 12%

Fourth quarter 2023 Dupixent® global net sales (recorded by Sanofi) increased 31% to $3.22 billion versus fourth quarter 2022; full year 2023 Dupixent global net sales increased 33% to $11.59 billion versus 2022

Fourth quarter 2023 U.S. net sales for EYLEA® HD and EYLEA® were $1.46 billion, including $123 million from EYLEA HD; full year 2023 U.S. net sales for EYLEA HD and EYLEA were $5.89 billion, including $166 million from EYLEA HD following its August 2023 FDA approval

Fourth quarter 2023 Libtayo® global net sales increased 44% to $244 million versus fourth quarter 2022; full year 2023 Libtayo global net sales increased 50% to $869 million versus 2022(f)

Fourth quarter 2023 GAAP diluted EPS of $10.19 and non-GAAP diluted EPS(a) of $11.86; includes unfavorable $0.21 impact from acquired IPR&D charge

Dupixent sBLA for chronic obstructive pulmonary disease (COPD) with type 2 inflammatory phenotype and linvoseltamab BLA for multiple myeloma submitted to FDA

TARRYTOWN, N.Y., Feb. 02, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced financial results for the fourth quarter and full year 2023 and provided a business update.

"2023 marked another year of exceptional accomplishments for Regeneron as we further diversified our revenue base and made important progress in our robust R&D pipeline," said Leonard S. Schleifer, M.D., Ph.D., Board Co-Chair, President and Chief Executive Officer of Regeneron. "In 2024, we plan to build on this momentum with continued growth of our breakthrough products Dupixent and EYLEA HD while we bring additional new therapies to market and advance our growing pipeline. Lastly, I want to congratulate our Chief Financial Officer, Bob Landry, on the occasion of his retirement and thank him for his significant contributions to Regeneron during his ten years with the Company."

Financial Highlights

Three Months Ended December 31,

Year Ended December 31,

($ in millions, except per share data)

2023

2022

% Change

2023

2022

% Change

Total revenues

$

3,434

$

3,414

1%

$

13,117

$

12,173

8%

Total revenues excluding Ronapreve(a)(b)

$

3,436

$

3,018

14%

$

12,906

$

11,546

12%

GAAP net income

$

1,160

$

1,197

(3

%)

$

3,954

$

4,338

(9

%)

GAAP net income per share - diluted

$

10.19

$

10.50

(3

%)

$

34.77

$

38.22

(9

%)

Non-GAAP net income(a)

$

1,366

$

1,449

(6

%)

$

5,045

$

5,164

(2

%)

Non-GAAP net income per share - diluted(a)

$

11.86

$

12.56

(6

%)

$

43.79

$

44.98

(3

%)

"We were pleased with our fourth-quarter and full-year 2023 financial performance, highlighted by revenue growth of 14% and 12%, respectively, when excluding contributions from Ronapreve, reflecting continued strength across our business," said Robert E. Landry, Executive Vice President, Finance and Chief Financial Officer of Regeneron. "In 2024, we plan to continue investing heavily in internal R&D, driving commercial execution with targeted promotion, and prudently deploying capital to business development and share repurchases, all of which is expected to better position the Company to deliver sustainable growth and long-term value to shareholders."

Business Highlights

Key Pipeline Progress

Regeneron has approximately 35 product candidates in clinical development, including a number of marketed products for which it is investigating additional indications. Updates from the clinical pipeline include:

EYLEA HD (aflibercept) 8 mg

In January 2024, the European Commission (EC) and Japan's Ministry of Health, Labour and Welfare (MHLW) each approved EYLEA 8 mg (known as EYLEA HD in the United States) for the treatment of patients with wet age-related macular degeneration (wAMD) and diabetic macular edema (DME).

In January 2024, the United States Centers for Medicare & Medicaid Services (CMS) assigned a permanent and product-specific J-code (J0177) for EYLEA HD. Under the Healthcare Common Procedure Coding System (HCPCS) process, the EYLEA HD J-code will become effective on April 1, 2024. J-codes are permanent reimbursement codes used by government payers and commercial insurers in the United States to facilitate billing for Medicare Part B treatments, which must be administered by a healthcare professional. J-codes simplify and streamline the billing and reimbursement processes, allowing for efficient claims processing.

Dupixent (dupilumab)

The Company and Sanofi announced that based on results from an interim analysis, the second Phase 3 trial (NOTUS) in patients with uncontrolled COPD and evidence of type 2 inflammation met its primary endpoint and showed that Dupixent significantly reduced exacerbations by 34% (confirming positive results from the replicate Phase 3 BOREAS trial). The NOTUS trial also confirmed that treatment with Dupixent led to rapid and significant improvements in lung function by 12 weeks and were sustained at 52 weeks. In December 2023, a supplemental Biologics License Application (sBLA) was submitted to the U.S. Food and Drug Administration (FDA) based on the results of these two trials. A regulatory application has also been submitted in the European Union (EU).

In January 2024, the FDA approved Dupixent for the treatment of children aged 1 to 11 years (weighing at least 15 kg) with eosinophilic esophagitis (EoE), making Dupixent the first and only medicine specifically indicated to treat these patients. A regulatory application has also been submitted in the EU.

Oncology Programs

The Company presented updated positive data from the pivotal trial of linvoseltamab, a bispecific antibody targeting BCMA and CD3, in patients with relapsed/refractory multiple myeloma at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition.

A BLA for linvoseltamab in relapsed/refractory multiple myeloma was submitted to the FDA in December 2023 and a regulatory application is also under review in the EU.

The Company presented updated data for odronextamab, a bispecific antibody targeting CD20 and CD3, in patients with relapsed/refractory follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) at the 65th ASH Annual Meeting and Exposition. A BLA for odronextamab in relapsed/refractory FL and DLBCL is currently under review by the FDA, with a target action date of March 31, 2024, and a regulatory application is also under review in the EU.

The Company presented, at European Society for Medical Oncology Immuno-Oncology (ESMO IO) Congress 2023, initial Phase 1 dose-escalation data for REGN5668, a costimulatory bispecific antibody targeting MUC16 and CD28, in combination with Libtayo (cemiplimab) that showed encouraging initial activity in patients with recurrent ovarian cancer.

Other Programs

The EC approved Evkeeza® (evinacumab) as an adjunct to other lipid-lowering therapies to treat children with homozygous familial hypercholesterolemia (HoFH), which extended the approved indication to children as young as 5 years of age.

A Phase 3 study was initiated for NTLA-2001, a TTR gene knockout using CRISPR/Cas9, in transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM).

The FDA granted Breakthrough Therapy designation to mibavademab, an agonist antibody to leptin receptor (LEPR), for generalized lipodystrophy (for which a Phase 2 study is ongoing).

Corporate and Business Development Updates

In the Company's ongoing patent infringement lawsuit against Mylan Pharmaceuticals Inc., a wholly-owned subsidiary of Viatris Inc., and Biocon Biologics Inc. concerning Mylan's filing for FDA approval of an aflibercept 2 mg biosimilar (now owned by Biocon), the United States District Court for the Northern District of West Virginia issued a decision finding that (i) the asserted claims of one of the Company's formulation patents (U.S. Patent No. 11,084,865) were valid and infringed by Mylan and (ii) the asserted claims of two of the Company's methods of treatment patents (U.S. Patent Nos. 10,888,601 and 11,253,572) were infringed by Mylan but were invalid as obvious.

In January 2024, the Company entered into an agreement with 2seventy bio, Inc. to acquire full development and commercialization rights to its preclinical and clinical stage cell therapy pipeline and will assume ongoing program, infrastructure, and personnel costs related to these programs. The transaction is expected to close in the first half of 2024 subject to certain customary closing conditions.

The Company announced its inclusion on the Dow Jones Sustainability World Index (DJSI World) for the fifth consecutive year, alongside its fourth consecutive inclusion on the Dow Jones Sustainability North America Index (DJSI North America).

Select Upcoming 2024 Milestones

Programs

Milestones

Ophthalmology

-

Initiate pivotal retinal vein occlusion (RVO) study of EYLEA HD (mid-2024) to enable FDA submission

-

Initiate pivotal studies of pozelimab (C5 antibody) in combination with cemdisiran (siRNA therapy) in geographic atrophy (second half 2024)

Immunology & Inflammation

-

EC decision on regulatory submission for Dupixent for EoE in children (1-11 years of age) (second half 2024)

-

sBLA acceptance for Dupixent in COPD with type 2 inflammatory phenotype (first quarter 2024) and FDA decision on sBLA (mid/second half 2024); EC decision on regulatory submission (second half 2024)

-

Report results from ongoing Phase 3 study for Dupixent in chronic spontaneous urticaria (CSU) in biologic-naïve patients (fourth quarter 2024)

-

Initiate Phase 1 study in severe food allergy following transient linvoseltamab treatment (in combination with Dupixent) (2024)

-

Complete enrollment of Phase 3 studies of itepekimab (IL-33 antibody) in COPD (second half 2024)

Solid Organ Oncology

-

Conduct interim analysis from Phase 3 study of Libtayo in adjuvant cutaneous squamous cell carcinoma (CSCC) (second half 2024)

-

Report potentially pivotal initial results from Phase 2/3 study of fianlimab (LAG-3 antibody) in combination with Libtayo in first-line metastatic melanoma and initial combination data in first-line advanced non-small cell lung cancer (NSCLC) (second half 2024)

-

Initiate potentially pivotal Phase 2 study for fianlimab (in combination with Libtayo) in perioperative melanoma and Phase 2 study for fianlimab (in combination with Libtayo) in perioperative NSCLC (first half 2024)

-

Initiate dose-expansion cohorts of REGN7075 (EGFR and CD28 costimulatory bispecific antibody) in combination with Libtayo in EGFR-high tumors (first half 2024)

-

Initiate cohorts combining REGN5678 (PSMA and CD28 costimulatory bispecific antibody) and REGN4336 (PSMA and CD3 bispecific antibody) in metastatic castration-resistant prostate cancer and initiate REGN5678 monotherapy cohort in renal cell carcinoma (first half 2024)

Hematology

-

FDA decision on BLA (target action date of March 31, 2024) and EC decision on regulatory submission (second half 2024) for odronextamab in relapsed/refractory FL and DLBCL

-

BLA acceptance for linvoseltamab in relapsed/refractory multiple myeloma (first quarter 2024) and FDA decision on BLA (second half 2024)

-

Initiate Phase 1 study of linvoseltamab in combination with CD38 and CD28 costimulatory bispecific antibody in multiple myeloma (2024)

-

Report Phase 2 results for REGN9933 (Factor XI antibody) in thrombosis (second half 2024)

Genetic Medicines

-

Initiate Phase 1 study of Factor 9 gene insertion in hemophilia B (mid-2024)

-

Report additional data from Phase ½ study for DB-OTO (AAV-based gene therapy) in pediatrics with hearing loss (2024)

-

Initiate Phase 1 study of ALN-SOD (SOD1 siRNA) in amyotrophic lateral sclerosis (ALS) (2024)

Obesity

-

Initiate Phase 2 study of semaglutide in combination with trevogrumab (anti-myostatin) with and without garetosmab (anti-Activin A) (mid-2024)

Fourth Quarter 2023 Financial Results

Revenues

($ in millions)

Q4 2023

Q4 2022

% Change

FY 2023

FY 2022

% Change

Net product sales:

EYLEA HD - U.S.

$

123

$

-

*

$

166

$

-

*

EYLEA - U.S.

1,338

1,496

(11

%)

5,720

6,265

(9

%)

Total EYLEA HD and EYLEA - U.S.

1,461

1,496

(2

%)

5,886

6,265

(6

%)

Libtayo - Global**

244

152

61%

863

448

93%

Praluent®- U.S.

61

36

69%

182

130

40%

Evkeeza - U.S.

24

15

60%

77

48

60%

Inmazeb®- U.S.

62

-

*

70

3

*

Total net product sales

1,852

1,699

9%

7,078

6,894

3%

Collaboration revenue:

Sanofi

993

836

19%

3,800

2,856

33%

Bayer

377

355

6%

1,487

1,431

4%

Other

-

396

(100

%)

216

627

(66

%)

Other revenue

212

128

66%

536

365

47%

Total revenues

$

3,434

$

3,414

1%

$

13,117

$

12,173

8%

* Percentage not meaningful

** Effective July 1, 2022, the Company began recording net product sales of Libtayo outside the United States. Excluded from the full year 2023 is approximately $6 million of first quarter 2023 net product sales recorded by Sanofi in connection with sales in certain markets (Sanofi recorded net product sales in such markets during a transition period). Similarly, excluded from the fourth quarter and full year 2022 is approximately $17 million and $34 million, respectively, of net product sales recorded by Sanofi (see Table 5).

Net product sales of EYLEA in the U.S. decreased in the fourth quarter and full year 2023, compared to the same periods of 2022, primarily due to changing market dynamics, resulting in a lower net selling price and lower volumes. EYLEA volumes in the fourth quarter of 2023 were impacted by the August 2023 launch of EYLEA HD and subsequent transition of EYLEA patients to EYLEA HD.

Sanofi collaboration revenue increased in the fourth quarter and full year 2023, compared to the same periods of 2022, primarily due to the Company's share of profits from commercialization of antibodies, which were $886 million and $619 million in the fourth quarter of 2023 and 2022, respectively, and $3.137 billion and $2.082 billion for the full year 2023 and 2022, respectively. The change in the Company's share of profits from commercialization of antibodies was driven by higher profits associated with an increase in Dupixent sales. In addition, during 2023 (third quarter) the Company earned the final $50 million sales-based milestone from Sanofi based upon aggregate annual sales of antibodies outside the U.S., compared to earning two $50 million sales-based milestones in 2022 (including one in the fourth quarter of 2022).

The Company recorded collaboration revenue during 2023 and 2022 in connection with payments from Roche attributable to global gross profits from sales of Ronapreve. The decrease in other collaboration revenue was due to lower sales of Ronapreve.

Refer to Table 4 for a summary of collaboration revenue.

Other revenue for the fourth quarter and full year 2023 included the recognition of $16 million and $50 million, respectively, of revenue in connection with the Company's agreement with BARDA to fund certain costs for a next-generation COVID-19 monoclonal antibody therapy for the prevention of SARS-CoV-2 infection. The increase in other revenue in 2023 was also due to higher royalties earned in connection with sales of Novartis' Ilaris® (canakinumab).

Operating Expenses

GAAP

% Change

Non-GAAP(a)

% Change

($ in millions)

Q4 2023

Q4 2022

Q4 2023

Q4 2022

Research and development (R&D)

$

1,177

$

1,043

13%

$

1,031

$

911

13%

Acquired in-process research and development (IPR&D)

$

30

$

30

-%

*

*

n/a

Selling, general, and administrative (SG&A)

$

738

$

661

12%

$

622

$

579

7%

Cost of goods sold (COGS)

$

307

$

302

2%

$

259

$

126

106%

Cost of collaboration and contract manufacturing (COCM)

$

210

$

238

(12

%)

*

*

n/a

Other operating (income) expense, net

$

(1)

$

(7)

(86

%)

*

*

n/a

GAAP

% Change

Non-GAAP(a)

% Change

FY 2023

FY 2022

FY 2023

FY 2022

Research and development

$

4,439

$

3,593

24%

$

3,919

$

3,169

24%

Acquired in-process research and development

$

186

$

255

(27

%)

*

*

n/a

Selling, general, and administrative

$

2,631

$

2,116

24%

$

2,232

$

1,853

20%

Cost of goods sold

$

932

$

800

17%

$

770

$

507

52%

Cost of collaboration and contract manufacturing

$

884

$

760

16%

*

*

n/a

Other operating (income) expense, net

$

(2)

$

(90)

(98

%)

*

*

n/a

* GAAP and non-GAAP amounts are equivalent as no non-GAAP adjustments have been recorded.

GAAP and non-GAAP R&D expenses increased in the fourth quarter and full year 2023, compared to the same periods in the prior year, driven by additional costs incurred in connection with higher headcount and headcount-related costs, the advancement of the Company's late-stage pipeline, and increased manufacturing activity associated with the Company's product candidates.

Acquired IPR&D for the full year 2023 included a $100 million development milestone in connection with the Phase 1 ALN-APP program, which is in collaboration with Alnylam Pharmaceuticals, Inc. Acquired IPR&D for the full year 2022 included a $195 million charge related to the Company's acquisition of Checkmate Pharmaceuticals, Inc.

GAAP and non-GAAP SG&A expenses increased in the fourth quarter of 2023, compared to the fourth quarter of 2022, primarily due to higher headcount and headcount-related costs and higher commercialization-related expenses for various products, including the Company's retinal franchise, partly offset by lower contributions to an independent not-for-profit patient assistance organization. GAAP and non-GAAP SG&A expenses increased for the full year 2023, compared to full year 2022, primarily due to higher headcount and headcount-related costs, an increase in commercialization-related expenses for Libtayo, and, to a lesser extent, various other products, and higher contributions to an independent not-for-profit patient assistance organization.

Non-GAAP SG&A expenses excluded certain charges related to acquisition and integration-related activities primarily incurred in connection with the July 2022 acquisition of Libtayo worldwide rights.

GAAP and non-GAAP COGS for the fourth quarter and full year 2023, when compared to the same periods in the prior year, included higher start-up costs for the Company's Rensselaer, New York fill/finish facility and an increase in period costs at the Company's manufacturing facilities (resulting from lower production volumes). GAAP COGS for the fourth quarter and full year 2023, when compared to the same periods in the prior year, included lower inventory write-offs and reserves (which were primarily related to REGEN-COV® in 2022).

Non-GAAP COGS excluded certain charges related to REGEN-COV (primarily inventory write-offs and reserves) of $134 million and $197 million in the fourth quarter and full year 2022, respectively.

COCM decreased in the fourth quarter of 2023, compared to the fourth quarter of 2022, primarily due to lower Dupixent manufacturing costs as a result of the transition to a higher-yielding manufacturing process. COCM increased for the full year 2023, compared to full year 2022, primarily due to the recognition of costs in connection with manufacturing commercial supplies for Sanofi related to Praluent outside the United States and for Bayer related to EYLEA outside the United States.

Other operating (income) expense, net for the full year 2022 included the recognition of amounts previously deferred in connection with up-front and development milestone payments received in connection with the Company's previous Sanofi Immuno-Oncology, Teva, and Mitsubishi Tanabe Pharma Corporation collaborative arrangements.

Other Financial Information

GAAP other income (expense) included the recognition of net unrealized losses on equity securities of $238 million for full year 2023, compared to $40 million for full year 2022. GAAP and Non-GAAP other income (expense) also included interest income of $496 million and $160 million for the full year 2023 and 2022, respectively.

In the fourth quarter and full year 2023, the Company's GAAP effective tax rate (ETR) was (1.0%) and 5.9%, respectively, compared to 9.6% and 10.7% in the fourth quarter and full year 2022, respectively. The GAAP ETR in the fourth quarter and full year 2023, compared to the same periods in the prior year, included a higher benefit from stock-based compensation, federal tax credits for research activities, and the proportion of income earned in foreign jurisdictions with tax rates lower than the U.S. federal statutory rate. In the fourth quarter and full year 2023, the non-GAAP ETR was 2.4% and 9.1%, respectively, compared to 11.3% and 12.1% in the fourth quarter and full year 2022, respectively.

GAAP net income per diluted share was $10.19 in the fourth quarter of 2023, compared to $10.50 in the fourth quarter of 2022. GAAP net income per diluted share was $34.77 for the full year 2023, compared to $38.22 for full year 2022. Non-GAAP net income per diluted share was $11.86 in the fourth quarter of 2023, compared to $12.56 in the fourth quarter of 2022. Non-GAAP net income per diluted share was $43.79 for the full year 2023, compared to $44.98 for full year 2022. A reconciliation of the Company's GAAP to non-GAAP results is included in Table 3 of this press release.

During the fourth quarter and full year 2023, the Company repurchased shares of its common stock and recorded the cost of the shares, or $295 million and $2.215 billion, respectively, as Treasury Stock. As of December 31, 2023, $1.5 billion remained available for share repurchases under the Company's share repurchase program.

2024 Financial Guidance©

The Company's full year 2024 financial guidance consists of the following components:

2024 Guidance

GAAP R&D

$4.820-$5.070 billion

Non-GAAP R&D(a)

$4.300-$4.500 billion

GAAP SG&A

$2.890-$3.090 billion

Non-GAAP SG&A(a)

$2.500-$2.650 billion

GAAP gross margin on net product sales(d)

86%-88%

Non-GAAP gross margin on net product sales(a)(d)

89%-91%

COCM(e)*

$850-$910 million

Capital expenditures*

$825-$950 million

GAAP effective tax rate

8%-10%

Non-GAAP effective tax rate(a)

10%-12%

* GAAP and non-GAAP amounts are equivalent as no non-GAAP adjustments have been or are expected to be recorded.

About Regeneron Pharmaceuticals, Inc.

Regeneron is a leading biotechnology company that invents, develops, and commercializes life-transforming medicines for people with serious diseases. Founded and led for over 35 years by physician-scientists, Regeneron's unique ability to repeatedly and consistently translate science into medicine has led to numerous FDA-approved treatments and product candidates in development, almost all of which were homegrown in Regeneron's laboratories. Regeneron's medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, hematologic conditions, infectious diseases, and rare diseases.

Regeneron is accelerating and improving the traditional drug development process through its proprietary VelociSuite® technologies, such as VelocImmune®, which uses unique genetically humanized mice to produce optimized fully human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center®, which is conducting one of the largest genetics sequencing efforts in the world.

https://investor.regeneron.com/news-releases/news-release-details/regeneron-reports-fourth-quarter-and-full-year-2023-financial

Seagen Inc.

Seagen is a global, multi-product biotechnology company that discovers, develops and commercializes transformative medicines targeting cancer to make a meaningful difference in people's lives. We are commercializing ADCETRIS® for the treatment of several types of CD30-expressing lymphomas, PADCEV® for the treatment of locally advanced or metastatic urothelial cancer and TUKYSA® for the treatment of certain HER2-positive metastatic breast cancers. We are also advancing a pipeline of novel therapies for solid tumors and blood-related cancers designed to address unmet medical needs and improve treatment outcomes for patients. Many of our programs, including ADCETRIS and PADCEV, are based on our proprietary antibody-drug conjugate, or ADC, technology that utilizes the targeting ability of monoclonal antibodies to deliver cell-killing agents directly to cancer cells.

https://investor.seagen.com/overview/default.aspx

1/11/2023

Seagen Third Quarter 2023 Financial Results Reflect Strong Product Sales Growth, and Significant Portfolio and Pipeline Progress

-Record Net Product Sales of $571 Million in 3Q23, a 33% Increase Over 3Q22, Primarily Driven by PADCEV® First-Line Launch-

-PADCEV with Keytruda® Potentially Practice Changing for First-Line Metastatic Urothelial Cancer after EV-302 Trial Demonstrates Near Doubling of Median Overall Survival-

-Proposed Pfizer Transaction On-Track and Anticipated to Close in Late-2023 or Early-2024, Subject to Closing Conditions, with Ongoing FTC Review and Recent EC Approval-

BOTHELL, Wash.--(BUSINESS WIRE)-- Seagen Inc. (Nasdaq:SGEN) (Seagen or the Company) reported financial results today for the third quarter ended September 30, 2023.

David Epstein, Chief Executive Officer of Seagen said, "Seagen continues to build momentum in 2023 and delivered strong performance, marked by record quarterly net product sales, with significant year-over-year growth of 33%, contributing to total revenues of $649 million. Growth was primarily driven by PADCEV (enfortumab vedotin-ejfv), which grew 89% over the same quarter of last year following the successful launch in the U.S. in combination with Keytruda (pembrolizumab) as a front-line treatment for patients with advanced urothelial cancer who are not eligible to receive cisplatin-containing chemotherapy. We also announced positive trial readouts across our commercial portfolio in multiple patient settings." Highlights include:

PADCEV in combination with Keytruda demonstrated a 53% reduction in the risk of death and a near doubling in the median overall survival (OS) (31.5 months vs 16.1 months) versus chemotherapy in patients with previously untreated locally advanced or metastatic urothelial cancer (la/mUC) who were eligible for cisplatin- or carboplatin-containing chemotherapy regardless of PD-L1 status. Data from the global phase 3 study, EV-302 / KEYNOTE-A39, were presented during the Presidential Symposium at the European Society of Medical Oncology (ESMO) Congress in October.

TIVDAK (tisotumab vedotin-tftv) demonstrated superior overall survival with a 30% reduction in the risk of death in the innovaTV 301 pivotal trial for patients with recurrent or metastatic cervical cancer with disease progression on or after first-line therapy. Additionally, progression-free survival was statistically significant with a 33% reduction in the risk of disease worsening or death compared with chemotherapy and the confirmed objective response rate was also improved with TIVDAK (17.8%) compared with chemotherapy (5.2%). Results were presented during the Presidential Symposium at ESMO.

The HER2CLIMB-02 pivotal trial of TUKYSA (tucatinib) in combination with antibody-drug conjugate (ADC) ado-trastuzumab emtansine met the primary endpoint of progression-free survival in patients with previously treated HER2-positive metastatic breast cancer.

"We remain very excited about the pending acquisition of Seagen by Pfizer as we look to build a world-class oncology organization which will broaden patient reach and accelerate development of our innovative pipeline," concluded Mr. Epstein.

Roger Dansey, President of Research and Development and Chief Medical Officer, added, "We have now demonstrated an overall survival benefit in targeted indications across all four of our approved products. The PADCEV EV-302 study has the potential to be practice changing and offer a new standard of care for first-line metastatic bladder cancer. Building on our expertise and innovative platform, we are advancing next-generation ADC and immuno-oncology targeted therapies. We recently initiated a phase 3 trial of disitamab vedotin in combination with pembrolizumab in patients with previously untreated locally advanced or metastatic HER2-positive urothelial cancer, along with a phase 1 trial for SGN-EGFRd2, a novel T cell engaging bispecific antibody. We also remain on track to initiate a phase 3 trial of SGN-B6A in patients with previously treated non-small cell lung cancer and to meet our goal of submitting four Investigational New Drug (IND) applications for novel drug candidates in 2023 before year end."

PRODUCTS HIGHLIGHTS

PADCEV

Positive Results for the Phase 3 EV-302 Trial of the Combination of PADCEV and Keytruda as First-Line Treatment for Advanced Urothelial Cancer Presented at the ESMO Congress: In October 2023,data from the phase 3 EV-302 trial, which was conducted in collaboration with Merck and our partner Astellas, were presented during the Presidential Symposium at ESMO in a late-breaking oral presentation demonstrating a 53% reduction in risk of death with PADCEV and Keytruda compared to chemotherapy in patients with previously untreated locally advanced or metastatic urothelial cancer (la/mUC). The combination improved median overall survival by more than 15 months versus chemotherapy. OS results were consistent across all pre-defined subgroups, including cisplatin eligibility and PD-L1 expression level. The safety results in EV-302 were consistent with those previously reported with this combination in EV-103 in cisplatin-ineligible patients with la/mUC. No new safety signals were identified. An extension study in China continues to enroll patients. The EV-302 trial is intended to serve as the basis for global submissions and as the confirmatory trial for the U.S. accelerated approval of this combination. We intend to submit a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) this year.

Data Presented for Muscle-Invasive Bladder Cancer (MIBC) at ESMO: In October 2023, data from Cohort L of the EV-103 phase 1b/2 trial evaluating PADCEV monotherapy as perioperative treatment in cisplatin-ineligible patients with MIBC were presented demonstrating promising activity and a generally manageable safety profile.

ADCETRIS ®

European Commission Approves ADCETRIS in Combination with Chemotherapy in Previously Untreated CD30+ Stage III Hodgkin Lymphoma: In October 2023, the European Commission approved the ADCETRIS (brentuximab vedotin) combination based on updated positive overall survival results from the Phase 3 ECHELON-1 study. ADCETRIS has been previously approved as a therapy for adult patients in Europe in six distinct indications, including those with previously untreated CD30+ Stage IV Hodgkin lymphoma.

Presenting Combination Data in Metastatic Solid Tumors at the Society for Immunotherapy of Cancer (SITC) Annual Meeting: Initial data of ADCETRIS in combination with Keytruda in metastatic solid tumors will be presented at the SITC Annual Meeting being held November 3-5, 2023.

TUKYSA

Phase 3 HER2CLIMB-02 Trial Combining TUKYSA and Kadcyla® Meets Primary Endpoint of Progression-Free Survival: In August 2023, the Company announced that the phase 3 HER2CLIMB-02 clinical trial of TUKYSA in combination with the antibody-drug conjugate Kadcyla (ado-trastuzumab emtansine) met its primary endpoint of progression-free survival (PFS), including for patients with active brain metastases. Patients in the trial had unresectable locally advanced or metastatic HER2-positive breast cancer and had received previous treatment with a taxane and trastuzumab. Overall survival data, a secondary endpoint, are not yet mature. Discontinuations due to adverse events were more common in the combination arm of the trial, but no new safety signals emerged for the combination. Results will be presented at an upcoming meeting.

TIVDAK

Positive Results for Phase 3 innovaTV 301 Clinical Trial Presented at ESMO: In October 2023, data from the innovaTV 301 trial, conducted in collaboration with our partner Genmab, were presented in an oral session during the Presidential Symposium at ESMO that demonstrated a 30% reduction in risk of death compared to chemotherapy and a manageable and tolerable safety profile consistent with the known safety profile as presented in the U.S. prescribing information. No new safety signals were observed. The results from innovaTV 301 are intended to serve as the confirmatory trial for the U.S. accelerated approval and support potential global regulatory applications.

PIPELINE PROGRAMS

Initiated a Phase 3 Trial for Disitamab Vedotin for Patients with HER2-Positive, Metastatic Urothelial Cancer: The Company initiated a phase 3 trial evaluating disitamab vedotin in combination with pembrolizumab versus chemotherapy in patients with previously untreated locally advanced or metastatic HER2-positive urothelial cancer in the third quarter of 2023.

Planning to Initiate Phase 3 Trial for SGN-B6A in Patients with Previously Treated Non-Small Cell Lung Cancer (NSCLC) Before Year End: The Company expects to initiate a phase 3 trial for SGN-B6A, a novel, vedotin ADC targeting integrin beta-6, as a monotherapy in previously treated patients with NSCLC before the end of 2023.

Reported Initial Results for SGN-B7H4V at ESMO: In October 2023, initial first-in-human clinical data for SGN-B7H4V were presented at ESMO demonstrating antitumor activity across a variety of dose levels, dose schedules and tumor types with commonly observed adverse events including peripheral neuropathy, neutropenia, diarrhea, and nausea, consistent with other ADCs. Development remains ongoing in phase 1 dose expansion cohorts in breast, endometrial, ovarian and other B7H4-expressing solid tumors.

Initiated Clinical Trial for Novel Targeted Therapy and Unveiled Novel ADC Program at AACR-NCI-EORTC Conference: The Company initiated a phase 1 trial for SGN-EGFRd2, a gamma delta bispecific T-cell engager for EGFR-expressing solid tumors. Year to date, the Company has submitted INDs for three novel targeted cancer therapies, including SGN-EGFRd2, SGN-35T and SGN-CEACAM5C, with the goal of submitting one additional IND before year end. Preclinical data for SGN-35T, a next generation CD30-directed ADC with a novel tripeptide drug linker designed to improve the tolerability profile compared with ADCETRIS, were presented at the annual AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in October.

For additional information on Seagen's pipeline, visit www.seagen.com/science/pipeline.

CORPORATE HIGHLIGHTS

Update on Pfizer Acquisition: The European Commission approved the acquisition of Seagen by Pfizer unconditionally pursuant to Article 6(1)b under the EU Merger Regulation on October 19, 2023. The completion of the transaction remains subject to other customary closing conditions, including the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, relating to the consummation of the transaction, and the satisfaction or waiver of the other closing conditions specified in the Merger Agreement. The Company continues to expect that the transaction will be completed in late 2023 or early 2024.

Annual Corporate Responsibility Report Published, Detailing ESG Commitments: Seagen continues to push the boundaries of innovation while upholding its commitment to sustainable practices, as highlighted in the Company's latest corporate responsibility report, Linking Breakthroughs to Lives. Accomplishments detailed include multiple positive clinical trial results and label expansions across our commercial portfolio, new DEI strategy, publishing our Human Rights Policy and Supplier Code of Conduct, enhancing governance programs across numerous areas including compliance and information security, and new environmental initiatives. Seagen is proud to announce that Newsweek named the Company one of America's Greenest Companies 2024 and awarded it the highest rating of 5 stars, based on its ESG achievements. The report is available on Seagen's website at https://www.seagen.com/who-we-are/corporate-responsibility.

THIRD QUARTER AND NINE-MONTHS 2023 FINANCIAL RESULTS

Revenues: Total revenues for the third quarter and nine months ended September 30, 2023 were $649 million and $1,772 million, respectively, compared to $510 million and $1,434 million for the same periods in 2022, primarily driven by growth in net product sales.

Revenues included the following components:

Three months ended September 30,

Nine months ended September 30,

(dollars in millions)

2023

2022

% Change

2023

2022

% Change

Total Net Product Sales

$

571

$

428

33%

$

1,583

$

1,243

27%

ADCETRIS

$

246

$

219

13%

$

751

$

601

25%

PADCEV

$

200

$

105

89%

$

479

$

329

46%

TUKYSA

$

102

$

88

16%

$

289

$

267

8%

TIVDAK

$

23

$

16

40%

$

64

$

45

42%

Royalty Revenues

$

64

$

44

45%

$

145

$

111

30%

Collaboration and License Agreement Revenues

$

14

$

38

(63)%

$

44

$

80

(45)%

Note: Sum of product sales may not equal total net product sales due to rounding. Percent change reflects actual (unrounded) values.

Net Product Sales: The increases in net product sales for the third quarter and year-to-date of 2023 compared to the same periods in 2022 were driven by continued commercial execution. ADCETRIS demonstrated year-over-year growth of 13%, primarily attributed to volume growth from greater use in frontline advanced Hodgkin lymphoma, despite recent increased competition in this setting. PADCEV growth was driven by use as first-line treatment for patients with locally advanced or metastatic urothelial cancer who are not eligible to receive cisplatin-containing chemotherapy following its approval for this indication in April 2023. Of note, PADCEV sales in the year-to-date of 2022 included $19 million in sales to another company for a clinical trial they are conducting, while no such sales were booked in the year-to-date of 2023. TUKYSA performance reflects volume growth driven by the important role it serves in the treatment of HER2-positive metastatic breast cancer as well as contributions from its colorectal cancer indication. TIVDAK growth reflects continued uptake in its current indication.

Royalty Revenues: Growth in royalty revenues were primarily driven by royalties from sales of Polivy® (polatuzumab vedotin) by Roche, which is an ADC that uses Seagen technology, as well as by sales of ADCETRIS outside the U.S. and Canada by Takeda.

Collaboration and License Agreement Revenues: The decrease in collaboration and license agreement revenues for the third quarter of 2023 was primarily driven by an upfront payment in the prior period and the decrease for the year-to-date also due to a prior period milestone partially offset by collaboration revenue.

Cost of Sales: Cost of sales for the third quarter and year-to-date in 2023 were $165 million and $458 million, respectively, compared to $108 million and $302 million for the same periods in 2022. The increases reflect higher sales of our medicines and the related gross profit share amounts owed to collaboration partners, which were $103 million and $249 million in the third quarter and year-to-date in 2023, respectively, compared to $71 million and $189 million for the same periods in 2022. Cost of sales also reflects amortization of TUKYSA acquired in-process technology costs, third-party royalties owed for PADCEV and TUKYSA net product sales, and cost of products sold. The year-to-date in 2023 cost of sales included a $47 million inventory write-off related to in-process production of one of our products that did not meet a release specification that was updated in June 2023. This inventory adjustment and new release specification are not expected to impact availability of product supply required to meet current or future demand.

Research and Development (R&D) Expenses: R&D expenses for the third quarter and year-to-date in 2023 were $449 million and $1,205 million, respectively, compared to $385 million and $987 million for the same periods in 2022 reflecting continued investment in clinical development of the Company's approved drugs and pipeline programs.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses for the third quarter and year-to-date in 2023 were $266 million and $746 million, respectively, compared to $210 million and $605 million for the same periods in 2022. The increases in 2023 were driven by ongoing commercialization efforts, as well as $40 million in expenses year-to-date associated with the pending acquisition by Pfizer and other corporate activities.

Non-cash, share-based compensation expense for the nine months ended September 30, 2023 was $288 million, compared to $157 million for the same period in 2022.

Net Loss: Net loss for the third quarter of 2023 was $216 million, or $1.15 per diluted share, and net loss for the year-to-date of 2023 was $602 million, or $3.21 per diluted share.

Net loss for the third quarter of 2022 was $191 million, or $1.03 per diluted share, and net loss for the year-to-date of 2022 was $462 million, or $2.51 per diluted share.

Cash and Investments: As of September 30, 2023, Seagen had $1.2 billion in cash and investments.

CONFERENCE CALL

Given the pending acquisition of Seagen by Pfizer, Seagen is no longer providing financial guidance for 2023 and will not be hosting its quarterly conference call and does not expect to do so for future quarters. Earnings materials are available publicly on the Investor Relations page of our website at investor.seagen.com. Please direct any questions to Seagen Investor Relations at the contact information below.

About Seagen

Founded 25 years ago, Seagen Inc. is a global biotechnology company that discovers, develops, manufactures, and commercializes targeted cancer therapeutics, with antibody-drug conjugates (ADCs) at our core. Our colleagues work together with urgency to improve and extend the lives of people living with cancer. An ADC technology trailblazer, approximately one-third of FDA-approved and marketed ADCs use Seagen technology. Seagen is headquartered in Bothell, Washington and has locations in California, Canada, Switzerland and across Europe. For additional information, visit www.seagen.com and follow us on X and LinkedIn.

https://investor.seagen.com/press-releases/news-details/2023/Seagen-Third-Quarter-2023-Financial-Results-Reflect-Strong-Product-Sales-Growth-and-Significant-Portfolio-and-Pipeline-Progress/default.aspx

Shire plc (SHPG)

Takeda Completes Acquisition of Shire, Becoming a Global, Values-based, R&D-Driven Biopharmaceutical Leader

January 8, 2019

- 8 months from deal announcement to deal close

- Strong shareholder support with high approval rates on Takeda (89.1%) and Shire (99.8%)

- Integration planning well underway

OSAKA, JAPAN, January 8, 2019 - Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") today announced the completion of its acquisition of Shire plc ("Shire"), becoming a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan.

Takeda now has an attractive, expanded geographic footprint and leading position in Japan and the U.S., bringing its highly-innovative medicines to approximately 80 countries/regions with dedicated employees worldwide. Takeda's R&D efforts are focused on its four therapeutic areas of Oncology, Gastroenterology (GI), Neuroscience and Rare Diseases, with targeted R&D investment also committed to Plasma-Derived Therapies (PDT) and Vaccines. Takeda's strengthened, highly innovative R&D engine enables the company to have a more global, robust and modality-diverse pipeline as well as to focus on breakthrough innovation.

The combined annual revenue of the company, exceeding $30 billion USD, is mainly derived from the key business areas of Oncology, GI, Neuroscience, Rare Diseases and PDT.

"We are delighted that the acquisition was approved by an overwhelming majority of our shareholders at Takeda's extraordinary general meeting on December 5th, 2018. We are also pleased to have completed the acquisition several months earlier than expected, which was enabled through the hard work of our respective organizations and the smooth receipt of regulatory clearances," said Christophe Weber, President and Chief Executive Officer of Takeda. "We appreciate the support of our employees, partners and shareholders throughout the process. This marks a significant moment in Takeda's history and is an exciting step forward as we accelerate our transformation journey to deliver highly-innovative medicines to patients around the world with expanded scale and geographical footprint."

Weber continued, "The execution of our integration begins today, and we are confident in our ability to execute a smooth integration under the leadership of our experienced and diverse Takeda Executive Team with a strong track record. The Operating Model we established in September last year has set a clear framework for our integration plans, and we have a highly skilled and dedicated integration team leading the process."

In order to fund the acquisition, Takeda has secured permanent financing with highly competitive rates, resulting in an overall blended interest rate for Takeda's total debt of approximately 2.3%. The company is confident that it will retain its investment grade credit rating and return to a net debt to EBITDA ratio of 2.0x or less within three to five years following completion.

Weber will present at the J.P. Morgan Healthcare Conference at 3:30 p.m. PST on January 8, 2019.

Notice regarding the Acquisition of the Entire Issued and To Be Issued Share of Shire plc. by Takeda Pharmaceutical Company Limited

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology (GI), Neuroscience and Rare Diseases. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions.

For more information, visit https://www.takeda.com

https://www.takeda.com/newsroom/newsreleases/2019/takeda-completes-acquisition-of-shire-becoming-a-global-values-based-rd-driven-biopharmaceutical-leader/

Shire is now part of Takeda.

Takeda is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology (GI), Neuroscience and Rare Diseases. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions.

https://www.shire.com/who-we-are

November 1, 2018 - Shire plc (Shire) (LSE: SHP, NASDAQ: SHPG), the leading global biotech company focused on rare diseases, announces unaudited results for the three months ended September 30, 2018.

Flemming Ornskov, M.D., M.P.H., Shire Chief Executive Officer, commented:

"We continue to deliver solid growth and pay down our debt while advancing our late-stage pipeline. Our focus on commercial execution led to 6% growth in product sales to $3.8 billion in the third quarter overcoming foreign exchange headwinds. Our growth was once again driven by our Immunology franchise, recently-launched products, and expansion in international markets. Proceeds from the sale of our Oncology franchise coupled with strong free cash flow allowed us to reduce net debt by $3.9 billion year to date.

"We recently launched TAKHZYRO, the first monoclonal antibody to prevent hereditary angioedema (HAE) attacks, in the U.S. We also gained approval for this innovative treatment in Canada and received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) recommending marketing authorization in Europe.

"Takeda's proposed acquisition of Shire remains on track to close in H1 2019, subject to shareholder approval of both companies and additional regulatory approvals. While integration planning is ongoing, our solid performance through the third quarter of 2018 demonstrates our continued focus on delivering for patients and executing against our key priorities."

Financial Highlights

Q3 2018 (1)

Reported Growth (1)

Non GAAP CER (1)(2)

Product sales

$3,753 million

+6%

+7%

Total revenues

$3,872 million

+5%

+6%

Operating income from continuing operations

$956 million

+35%

Non GAAP operating income (2)

$1,475 million

-2%

-1%

Net income margin (3)(4)

14%

-1ppc

Non GAAP EBITDA margin (2)(3)(4)

42%

-2ppc

Net income

$537 million

-2%

Non GAAP net income (2)

$1,119 million

-3%

Diluted earnings per ADS (5)

$1.75

-3%

Non GAAP diluted earnings per ADS (2)(5)

$3.64

-4%

-4%

Net cash provided by operating activities

$858 million

-19%

Non GAAP free cash flow (2)

$971 million

+8%

(1) Results include the Oncology franchise until the date of its sale on August 31, 2018. (2) The Non GAAP financial measures included within this release are explained on pages 27 - 28, and are reconciled to the most directly comparable financial measures prepared in accordance with U.S. GAAP on pages 19 - 22. (3) Percentage point change (ppc). (4) Calculated as a percentage of total revenues. (5) Diluted weighted average number of ordinary shares of 921.1 million.

Product sales growth

All franchises demonstrated product sales growth on a Non GAAP constant exchange rate basis, excluding Oncology which was sold during the quarter.

Encouraging early trajectory of TAKHZYRO since U.S. launch on August 23, 2018 with $51 million in initial launch stocking.

Growth of recently-launched products of 45%, primarily due to TAKHZYRO, ADYNOVATE, CUVITRU, and XIIDRA.

Operating performance

Generated Non GAAP diluted earnings per ADS of $3.64, a decrease of 4%, as product sales growth and operating expense discipline were offset by unfavorable foreign exchange, lower gross margins, and unrealized losses on equity investments.

Reported Non GAAP EBITDA margin of 42%, a slight decline from Q3 2017, primarily due to lower gross margins, as Q3 2017 reflected favorability from the timing of changes in the costs to manufacture certain products, partially offset by ongoing cost discipline and operating expense synergies.

Cash flow

Proceeds from the sale of our Oncology franchise and strong free cash flow during the year enabled a $3,915 million reduction in Non GAAP net debt since December 31, 2017.

FINANCIAL SUMMARY - THIRD QUARTER 2018 COMPARED TO THIRD QUARTER 2017

Revenues

Delivered total revenues of $3,872 million representing growth of 5%.

Product sales increased 6% to $3,753 million (Q3 2017: $3,534 million), driven by Immunology, up 12%, Neuroscience, up 6%, Genetic Diseases, up 6%, Internal Medicine, up 10%, and Ophthalmics, up 21%.

Royalties and other revenues decreased 27% to $119 million (Q3 2017: $164 million), primarily due to certain royalty expirations, the reclassification of ADDERALL XR from royalty revenue to product sales, and other changes as required under the new revenue accounting standard.

Operating results

Operating income increased 35% to $956 million (Q3 2017: $709 million), due to the gain on the sale of Shire's Oncology franchise and lower integration and acquisition costs, partially offset by increased reorganization costs.

Non GAAP operating income decreased 2% to $1,475 million (Q3 2017: $1,498 million), primarily due to lower gross margins as Q3 2017 reflected favorability from the timing of changes in the costs to manufacture certain products.

Non GAAP EBITDA margin was slightly down to 42% (Q3 2017: 44%), primarily due to lower gross margins partially offset by ongoing cost discipline and operating expense synergies.

Earnings per share (EPS)

Diluted earnings per American Depository Share (ADS) decreased 3% to $1.75 (Q3 2017: $1.81), primarily due to increased reorganization costs and income taxes, offset by the gain on the sale of Shire's Oncology franchise.

Non GAAP diluted earnings per ADS decreased 4% to $3.64 (Q3 2017: $3.81) as product sales growth and operating expense discipline were offset by unfavorable foreign exchange, lower gross margins, and unrealized losses on equity investments.

Cash flows

Net cash provided by operating activities decreased 19% to $858 million (Q3 2017: $1,055 million), driven by a $251 million contingent consideration payment to former shareholders of Dyax Corp. due to the approval of TAKHZYRO.

Non GAAP free cash flow increased 8% to $971 million (Q3 2017: $901 million). Non GAAP free cash flow includes capital expenditures of $203 million (Q3 2017: $174 million) and excludes payments relating to milestone and license arrangements of $316 million (Q3 2017: $20 million).

Debt

Non GAAP net debt as of September 30, 2018 decreased $3,915 million since December 31, 2017, to $15,154 million (December 31, 2017: $19,069 million). A combination of proceeds from the sale of Shire's Oncology franchise, Non GAAP free cash flow, and existing cash balances were utilized to repay debt during the year. Non GAAP net debt represents aggregate long and short term borrowings of $14,980 million, and capital leases of $367 million, partially offset by cash and cash equivalents of $193 million.

OUTLOOK

Our 2018 guidance, presented in the table below, has been updated to adjust for the sale of our Oncology franchise, which closed on August 31, 2018. Similarly, our projected 2020 revenue target has been updated to $16.5 - $17.5 billion, reflecting the removal of $0.5 billion of Oncology sales in our original projection. We continue to expect to achieve mid-forties Non GAAP EBITDA margin by 2020, which remains unchanged after considering the impact of the sale of our Oncology franchise.

Our Non GAAP diluted earnings per ADS outlook assumes a weighted average number of 917 million fully diluted ordinary shares outstanding for 2018.

Our U.S. GAAP diluted earnings per ADS outlook reflects anticipated amortization, integration, acquisition, and reorganization costs, as well as the gain on sale of our Oncology franchise and the impact from debt repurchase.

Risks associated with this outlook include the potential uncertainty resulting from the announcement by Takeda Pharmaceutical Company Limited (Takeda) on May 8, 2018 of a recommended offer for Shire under the U.K. Takeover Code.

RECENT DEVELOPMENTS

Corporate

The acquisition of Shire by Takeda is expected to close in H1 2019, subject to receipt of additional regulatory clearances and approval by the shareholders of both companies. Takeda has already received clearances from regulatory agencies in the U.S., Japan, China, and other countries and is in discussions with the European Commission as part of its Phase 1 review of the proposed acquisition.

Business Development

Sale of Oncology franchise

On August 31, 2018, Shire announced it had completed the sale of its Oncology franchise to Servier S.A.S. (Servier) for $2.4 billion. The franchise included the global rights to ONCASPAR and ex-U.S. and ex-Taiwan rights to ONIVYDE, as well as Oncology pipeline assets.

Acquisition of sanaplasma AG

On September 6, 2018, Shire announced the acquisition of sanaplasma AG, a source plasma collection company headquartered in Switzerland. Sanaplasma AG adds 14 new centers in the Czech Republic and Hungary to Shire's European-based plasma collection network.

Financing

On September 11, 2018, Shire completed a $2.3 billion cash tender offer to repurchase certain of its outstanding senior notes. The tender offer was funded from the proceeds of the sale of its Oncology franchise.

Products

TAKHZYRO, a first-of-its-kind monoclonal antibody (mAb) preventive treatment for HAE

On August 23, 2018, Shire announced that the U.S. Food and Drug Administration (FDA) had approved TAKHZYRO injection, for prophylaxis to prevent attacks of HAE in patients 12 years of age and older.

On September 20, 2018, Shire announced that Health Canada had authorized TAKHZYRO for routine prevention of attacks of HAE in patients 12 years of age and older.

On October 19, 2018, Shire announced that the CHMP of the European Medicines Agency (EMA) had issued a positive opinion recommending the granting of marketing authorization in the European Union (EU) for lanadelumab for the prevention of HAE attacks.

FIRAZYR for the treatment of HAE attacks in Japan

On September 21, 2018, Shire announced that the Ministry of Health, Labour and Welfare in Japan had granted manufacturing and marketing authorization for FIRAZYR, for the acute treatment of HAE attacks in adult patients with HAE.

VEYVONDI, for adults with von Willebrand disease (VWD)

On September 12, 2018, Shire announced that the European Commission had granted Marketing Authorization for VEYVONDI, for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults (age 18 and older) with VWD when desmopressin treatment alone is ineffective or not indicated.

INTUNIV, for the treatment of attention deficit hyperactivity disorder (ADHD) in adults

On August 13, 2018, Shire announced that its partner in Japan, Shionogi & Co., Ltd had submitted a New Drug Application (NDA) for the manufacture and marketing in Japan of INTUNIV.

Pipeline

Prucalopride (SHP555) for the treatment of chronic idiopathic constipation (CIC)

On October 18, 2018, Shire announced that the FDA Gastrointestinal Drugs Advisory Committee voted unanimously that the risk-benefit profile of prucalopride supports the approval of this NDA, which has a Prescription Drug User Fee Act (PDUFA) date of December 21, 2018.

Facilities

On October 25, 2018, Shire announced it had filed a second submission to the FDA for approval to manufacture albumin therapy at its new plasma manufacturing facility near Covington, Georgia.

OVERVIEW OF THIRD QUARTER 2018 FINANCIAL RESULTS COMPARED TO THIRD QUARTER 2017

Product sales

Product sales increased 6% to $3,753 million(Q3 2017: $3,534 million), driven by Immunology, up 12%, Neuroscience, up 6%, Genetic Diseases, up 6%, Internal Medicine, up 10%, and Ophthalmics, up 21%. Product sales include TAKHZYRO, which was launched on August 23, 2018, and results for Oncology through August 31, 2018, the date the sale of the franchise was completed.

Immunology

Immunology product sales were $1,197 million in Q3 2018. Immunoglobulin therapies and bio therapeutics were each up 8% mainly due to increased demand. HAE product sales were up 23%, which included $51 million of TAKHZYRO product sales for initial launch stocking. HAE product sales also reflected higher CINRYZE product sales as Q3 2017 included the impact of supply constraints, offset by destocking of FIRAZYR.

Hematology

Hematology product sales were $905 million in Q3 2018. Sales of inhibitor therapies declined 11% due to new competition, while sales of hemophilia therapies increased by 1% with continued growth of our extended half-life product, ADYNOVATE.

Neuroscience

Neuroscience product sales were $732 million in Q3 2018. VYVANSE product sales increased 11% due to a U.S. price increase and continued growth in our international markets.

Genetic Diseases

Genetic Diseases product sales increased 6%to $381 million, driven by increased sales for ELAPRASE and REPLAGAL primarily in our international markets.

Established Brands

Established Brands products sales increased 14% to $217 million, with the impact of generic competition offset by favorable sales deductions and some stocking for LIALDA in Q3 2018 compared to Q3 2017.

Internal Medicine

Internal Medicine product sales increased 10% to $177 million, driven by demand growth for GATTEX/REVESTIVE and NATPARA/NATPAR.

Ophthalmics

Ophthalmics product sales increased 21% to $93 million due to XIIDRA demand growth.

Oncology

As a result of the sale of Shire's Oncology franchise, completed on August 31, 2018, Oncology product sales decreased to $51 million from $69 million in Q3 2017.

Royalties and other revenues

Royalties and other revenues decreased 27%, primarily due to certain royalty expirations, the reclassification of ADDERALLXR from royalty revenue to product sales, and other changes as required under the new revenue accounting standard.

Financial details

Cost of sales as a percentage of total revenues increased by 3 percentage points to 30%, primarily due to lower gross margins as Q3 2017 reflected favorability from the timing of changes in the costs to manufacture certain products.

R&D

R&D increased $4 million, or 1%, primarily due to continued investment in late stage and launch programs offset by savings on discontinued programs. R&D as a percentage of total revenues remained consistent with Q3 2017. Non GAAP R&D was flat.

Non GAAP R&D as a percentage of total revenues decreased 1 percentage point.

SG&A

SG&A decreased $23 million, or 3%, primarily due to the benefits of on-going cost discipline and operating synergies partially offset by increased depreciation.

Non GAAP SG&A decreased by $40 million, or 5%, due to the benefits of on-going cost discipline and operating synergies.

Amortization of acquired intangible assets

In Q3 2018, Shire recorded amortization of acquired intangible assets of $434 million (Q3 2017: $482 million). The decrease was primarily related to amortization for CINRYZE and the sale of Oncology assets, including ONCASPAR and ONIVYDE, partially offset by amortization for TAKHZYRO, which was approved in Q3 2018.

Integration and acquisition costs

In Q3 2018, Shire recorded integration and acquisition costs of $93 million, primarily related to changes in fair value of contingent consideration and costs related to the proposed Takeda transaction.

In Q3 2017, Shire recorded integration and acquisition costs of $237 million, primarily related to the Baxalta transaction. Costs included asset impairment charges, employee severance, the acceleration of stock compensation, third-party professional fees, and expenses associated with facility consolidations.

Reorganization costs

In Q3 2018, Shire recorded reorganization costs of $255 million, primarily related to expenses associated with office facility closures. In Q3 2017, Shire recorded reorganization costs of $5 million.

Gain/loss on sale of Oncology and product rights

In Q3 2018, Shire recorded a gain from the sale of its Oncology franchise of $267 million. In Q3 2017, Shire recorded a loss on sale of product rights of less than $1 million.

Other expense, net

Other expense, net increased by $80 million, primarily due to costs related to the cash tender offer for the repurchase of $2.3 billion of Shire's outstanding senior notes.

Non GAAP other expense, net increased by $34 million, primarily due to unrealized losses in equity investments and net losses on foreign exchange revaluations.

Taxation

The effective tax rate on U.S. GAAP income in Q3 2018 was 28% (Q3 2017: 2%) and on a Non GAAP basis was 15% (Q3 2017: 15%).

The effective rate in Q3 2018 on U.S. GAAP income from continuing operations has been affected by certain provisions of the U.S. Tax Cuts and Jobs Act (Tax Act) passed in December 2017.

Income tax expense was increased by $60 million during the three months ended September 30, 2018 due to continued refinement of Shire's provisional computations under the Tax Act. The increase in the tax expense recorded during the three months ended September 30, 2018 was due to 1) an adjustment to U.S. deferred tax balances recorded as of December 31, 2017 related to the corporate income tax rate reduction of $15 million; and 2) an increase of $45 million related to the repatriation toll charge. The change in the toll charge was partially driven by an adjustment of $31 million related to the tax rates applied to certain drivers of the provisional repatriation toll charge in 2017, as well as the finalization of inputs to the calculation of the repatriation toll charge and the refinement of Shire's computation for the various guidance and regulations issued during 2018.

Shire will continue to assess the financial statement impact of the applicable provisions of the Tax Act upon enactment. It is expected that additional interpretive guidance will be issued during the measurement period that may change how Shire has computed the provisional amounts for the year ended December 31, 2017. Consequently, Shire continues to assert that all amounts recorded and disclosed to date remain provisional.

PROFIT FORECASTS

On February 14, 2018, Shire published its full year 2018 outlook for total revenue of $15.4 - $15.9 billion, GAAP diluted EPS of $7.30 - $7.90, and non-GAAP diluted EPS of $14.90 - $15.50 (the "Full Year 2018 Outlook"). Shire also announced "We are committed to achieving our projected revenue target of $17 - $18 billion in 2020" and "With the already disclosed manufacturing and SG&Acost reduction initiatives, we are on track to achieve mid-forties Non-GAAP EBITDA margin by 2020" (the "Mid-Term Outlook").

Both the Full Year 2018 Outlook and the Mid-Term Outlook comprised all Shire franchises, including Oncology. Earlier in the year, Shire announced that it had entered into an agreement with Servier for the sale of its Oncology franchise for $2.4 billion as part of its strategy to unlock value and sharpen its focus on rare disease leadership. The transaction closed on August 31, 2018. The U.S. GAAP Full Year 2018 Outlook has also been updated for the gain from the sale of the Oncology franchise, bond retirement with the proceeds of the sale and resulting interest reduction, and reorganization costs. Accordingly, the Full Year 2018 Outlook and the Mid-Term Outlook are no longer valid and Shire has updated the Full Year 2018 Outlook and the Mid-Term Outlook to adjust for the sale of the Oncology franchise as follows:

Full Year 2018 Outlook

Full year 2018 U.S. GAAP outlook for total revenue is expected to be $15.3 - $15.8 billion and diluted earnings per ADS is expected to be $7.17 - $7.77.

Full year 2018 Non GAAP outlook for total revenue is expected to be $15.3 - $15.8 billion and diluted earnings per ADS is expected to be $14.77 - $15.37.

Mid-Term Outlook

"Our projected 2020 revenue target has been updated to $16.5 - $17.5 billion."

"We continue to expect to achieve mid-forties Non GAAP EBITDA margin by 2020."

Assumptions and basis of preparation

The Full Year 2018 Outlook and the Mid-Term Outlook (as updated) (the "Profit Forecasts") include "profit forecasts" for the purposes of Rule 28 of the City Code on Takeovers and Mergers (the "Code").

In accordance with Rule 28.1(c) of the Code, the directors of Shire confirm that: (i) the basis of accounting used to prepare the Profit Forecasts is consistent with the accounting policies of Shire (or in the case of the Non GAAP Profit Forecasts, or in the case of the Non GAAP guidance, as adjusted in accordance with Shire's established Non GAAP policy, which excludes the items set out on pages 27 - 28 in the section "Non GAAP measures", including their tax effect); and (ii) each of the Profit Forecasts has been properly compiled on the basis of the following assumptions:

Assumptions outside the Directors' control

the Profit Forecasts exclude any future effect resulting from the announcement by Takeda on May 8, 2018 of a recommended offer for Shire under the Code;

there will be no material change to existing global macroeconomic and political conditions during the year ending December 31, 2018;

there will be no material changes in market conditions within the pharmaceutical industry over the forecast period to December 31, 2018, in relation to either customer demand or the competitive environment which could impact Shire's products;

there will be no product shortages caused by unanticipated production issues, such as contamination, which could result in prolonged supply shortages;

there will be no material changes to Shire's obligations to customers or governments, its ability to negotiate new business, resolve contract disputes, or the retention of key management;

the Euro, British Pound, and Swiss Franc and other exchange rates against the U.S. dollar, together with inflation, tax, and interest rates in Shire's principal markets, will remain relatively unchanged from the rates underpinning the Profit Forecasts;

there will be no material adverse events that will have a significant impact on Shire's financial position or performance;

there will be no material change in legislation, regulatory requirements, or the position of any regulatory bodies impacting Shire's operations or its accounting and tax conclusions, policies, and procedures;

there will be no significant increases or decreases in the value of publicly-held investments resulting in recognition of material gains or losses; and

there will be no material change in tax law and practice, including interpretive guidance issued by the IRS with respect to U.S. tax reform, impacting Shire's operations and the jurisdictions in which it earns significant amounts of income, whether earned from third parties or from intercompany transactions

Assumptions within the Directors' control

there will be no material change in the present management of Shire or its existing operational strategy prior to the closing of the recommended offer by Takeda announced on May 8, 2018;

there will be no material future acquisitions, disposals, or licensing arrangements;

there will be no material change in the debt structure of the Shire Group, other than planned repayments of existing borrowings;

there will be no material change to the number of diluted shares in issue; and

Shire's accounting and tax policies, including those related to determining Shire's effective tax rate, will be consistently applied in the financial year to December 31, 2018.

https://www.shire.com/-/media/shire/shireglobal/shirecom/pdffiles/newsroom/2018/pr-01-11-2018.pdf?la=en&hash=82626AA57B9538AC0D264E97AE843436404F538C

Vertex Pharmaceuticals (NASDAQ: VRTX)

Who We Are

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. We have three approved medicines that treat the underlying cause of cystic fibrosis (CF) - a rare, life-threatening genetic disease - and have several ongoing clinical and research programs in CF. Beyond CF, we have a robust pipeline of investigational medicines in other serious diseases where we have deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, we have research and development sites and commercial offices in North America, Europe, Australia and Latin America.

Vertex is consistently recognized as one of the industry's top places to work, including nine consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.

https://www.vrtx.com/about-us

6/5/2024

Vertex Reports First Quarter 2024 Financial Results

- Product revenue of $2.69 billion, a 13% increase compared to Q1 2023; reiterated full year 2024 financial guidance, including product revenue guidance of $10.55 to $10.75 billion

- Submitted NDA and MAA filings for vanzacaftor triple in CF to FDA and EMA, respectively

- Initiated rolling NDA submission for suzetrigine (VX-548) in moderate-to-severe acute pain and on track to complete this quarter

- Entered into agreement to acquire Alpine Immune Sciences, including povetacicept, a Phase 3-ready asset in IgA nephropathy and potential pipeline-in-a-product

BOSTON--(BUSINESS WIRE)--May 6, 2024-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the first quarter ended March 31, 2024, and reiterated full year 2024 financial guidance.

"Vertex delivered a strong start to 2024 with 13 percent product revenue growth and outstanding execution across the business. This quarter, we continued to expand our leadership in CF including completion of the regulatory submissions for the vanzacaftor triple, advanced the global launch of CASGEVY[TM], and initiated the rolling submission for suzetrigine in moderate-to-severe acute pain, while progressing our broad and deep pipeline of potentially transformative medicines," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "We also recently announced the acquisition of Alpine Immune Sciences, a compelling strategic fit. We look forward to welcoming the Alpine team and together accelerating the development of povetacicept, a potential best-in-class treatment for IgAN, and fully exploring povetacicept's pipeline-in-a-product potential."

First Quarter 2024 Results

Product revenue increased 13% to $2.69 billion compared to the first quarter of 2023, primarily driven in ex-U.S. markets by the continued strong uptake of TRIKAFTA®/KAFTRIO®, including label extensions in younger age groups, and in the U.S. by the continued performance of TRIKAFTA, including the uptake in children with CF 2 to 5 years of age. Net product revenue in the first quarter of 2024 increased 8% to $1.52 billion in the U.S. and increased 21% to $1.17 billion outside the U.S., compared to the first quarter of 2023.

Combined GAAP and Non-GAAP R&D, Acquired IPR&D and SG&A expenses were $1.2 billion and $1.0 billion, respectively, compared to $1.3 billion and $1.2 billion, respectively, in the first quarter of 2023. The decreases were due to lower Acquired IPR&D expenses partially offset by increased investments to support launches of Vertex's therapies globally and continued investment in support of multiple programs that have advanced in mid- and late-stage clinical development.

GAAP effective tax rate was 14.0% compared to 21.5% for the first quarter of 2023, primarily due to a discrete adjustment to Vertex's income tax reserves as well as tax benefits related to stock-based compensation.

Non-GAAP effective tax rate was 17.4% compared to 21.3% for the first quarter of 2023, primarily due to a discrete adjustment to Vertex's income tax reserves. Please refer to Note 1 for further details on Vertex's GAAP to Non-GAAP tax adjustments.

GAAP and Non-GAAP net income increased by 57% and 56%, respectively, compared to the first quarter of 2023, primarily due to higher product revenues and lower Acquired IPR&D expenses.

Cash, cash equivalents and total marketable securities as of March 31, 2024 were $14.6 billion, compared to $13.7 billion as of December 31, 2023. The increase was primarily due to cash from operations driven by strong revenue growth, partially offset by business development payments, and repurchases of Vertex's common stock.

Full Year 2024 Financial Guidance

Vertex today reiterated its full year 2024 financial guidance, including product revenue guidance of $10.55 to $10.75 billion. Vertex's product revenue guidance includes expectations for continued growth in CF as well as for the launch of CASGEVY in approved indications and geographies. Vertex's combined Non-GAAP R&D, Acquired IPR&D and SG&A expense guidance of $4.3 billion to $4.4 billion includes expectations for continued investment in multiple mid- and late-stage clinical development programs, commercial and manufacturing capabilities, and approximately $125 million of upfront and milestone payments. The recently announced acquisition of Alpine Immune Sciences is expected to close in the second quarter. Vertex does not anticipate adjusting its guidance for Alpine's operating expenses, other than the potential impact of purchase accounting.

Vertex's financial guidance is summarized below:

Current FY 2024

Previous FY 2024

Total product revenues

Unchanged

$10.55 to $10.75 billion

Combined GAAP R&D, Acquired IPR&D and SG&A expenses (2)

Unchanged

$4.9 to $5.1 billion

Combined Non-GAAP R&D, Acquired IPR&D and SG&A expenses (2)

Unchanged

$4.3 to $4.4 billion

Non-GAAP effective tax rate

Unchanged

20% to 21%

Marketed Products and Potential Near-Term Launch Opportunities

Cystic Fibrosis (CF) Portfolio

Vertex anticipates the number of CF patients taking our medicines will continue to grow through new approvals and reimbursement for the treatment of younger patients. Recent and anticipated progress includes:

The European Commission has granted approval for KALYDECO for the treatment of infants with CF ages 1 month to less than 4 months with specific mutations in the CFTR gene. KALYDECO now represents the first and only medicine approved in Europe to treat the underlying cause of CF for this age group.

In the first quarter, Vertex shared positive data from the pivotal studies for the next-generation triple combination of vanzacaftor/tezacaftor/deutivacaftor (the "vanzacaftor triple"), showing that the two randomized studies in patients 12 years and older met the primary endpoint and all key secondary endpoints, and the results in the single-arm study in children ages 6 to 11 were even more pronounced.

Vertex has submitted regulatory marketing applications for the once-daily vanzacaftor triple in people with CF 6 years and older to the U.S. Food and Drug Administration (FDA), using a priority review voucher, and to the European Medicines Agency (EMA). Vertex intends to complete regulatory submissions to the MHRA in Great Britain, Health Canada, SwissMedic, the Australian Therapeutic Goods Administration (TGA) and the New Zealand Ministry of Health for people with CF 6 years of age and older later this year.

CASGEVY for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT):

CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT that has been shown to reduce or eliminate vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT.

CASGEVY is approved in the U.S., Great Britain, the European Union (EU), the Kingdom of Saudi Arabia (KSA), and the Kingdom of Bahrain (Bahrain) for the treatment of both SCD and TDT. Vertex has completed regulatory submissions for CASGEVY for SCD and TDT in Switzerland and Canada; the submission in Canada was granted priority review.

As of mid-April, Vertex has activated more than 25 authorized treatment centers (ATCs) globally and multiple patients across all regions have initiated cell collection.

Vertex has signed multiple agreements with both commercial and government health insurance providers in the U.S. to provide access to CASGEVY.

Vertex has also secured reimbursed access for people with SCD or TDT in KSA and Bahrain, as well as for people with TDT in France through an early access program.

Suzetrigine (VX-548) for the treatment of moderate to severe acute pain:

Vertex has discovered multiple selective small molecule inhibitors of NaV1.8 with the goal of creating a new class of pain medicines that has the potential to provide effective pain relief across a variety of pain states, without the limitations of opioids and other currently available medicines.

In the first quarter, Vertex shared positive results from the three Phase 3 trials of suzetrigine in moderate-to-severe acute pain.

The FDA has granted a rolling New Drug Application (NDA) submission to suzetrigine in moderate-to-severe acute pain, and Vertex has started the rolling submission process. The submission is on track to be completed in the second quarter of 2024. Suzetrigine has also been granted FDA Fast Track and Breakthrough Therapy designations in moderate-to-severe acute pain.

Select Clinical-Stage R&D Pipeline

Cystic Fibrosis

Vertex continues to pursue next-in-class, small molecule, oral CFTR modulators for the ~90% of patients who may benefit from such an approach, as well as a nebulized mRNA therapy for the more than 5,000 people with CF who do not make CFTR protein and cannot benefit from CFTR modulators.

Vanzacaftor/tezacaftor/deutivacaftor, the next-in-class triple oral small molecule combination, in cystic fibrosis Vertex plans to initiate a new cohort in the Phase 3 study, RIDGELINE, in the second half of 2024 in children with cystic fibrosis ages 2 to 5 years who have at least one F508del mutation or a mutation responsive to triple combination CFTR modulators.

Nebulized mRNA therapy:

Vertex continues to enroll and dose patients in the multiple ascending dose (MAD) portion of the Phase 1/2 study of VX-522 in people with CF.

Vertex expects to share data from this study in late 2024 or early 2025.

Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Vertex has completed enrollment in two global Phase 3 studies of CASGEVY in people 5 to 11 years of age with SCD or TDT.

Vertex continues to work on preclinical assets for gentler conditioning for CASGEVY, which could broaden the eligible patient population to more than 150,000 people in the U.S. and Europe.

Acute Pain

Vertex plans to initiate a Phase 2 study with an oral formulation of VX-993, a next-generation selective NaV1.8 inhibitor, for the treatment of moderate to severe acute pain in 2024.

The FDA cleared the investigational new drug (IND) application for an intravenous formulation of VX-993 for the treatment of moderate to severe acute pain, and Vertex has initiated a Phase 1 trial.

Consistent with its commitment to serial innovation and leadership in pain, Vertex also continues to develop NaV1.7 inhibitors, for stand-alone use or in combination with NaV1.8 inhibitors, for both acute and peripheral neuropathic pain.

Peripheral Neuropathic Pain (PNP)

Following a successful end-of-Phase 2 meeting with the FDA, Vertex is on track to initiate the Phase 3 pivotal program of suzetrigine in patients with painful diabetic peripheral neuropathy (DPN), a type of PNP, in the second half of 2024. The FDA recently granted suzetrigine Breakthrough Therapy designation in this indication.

Vertex continues to enroll and dose patients in its Phase 2 study of suzetrigine in painful lumbosacral radiculopathy (LSR), representing ~40% of the PNP category. Vertex is on track to complete enrollment in the Phase 2 study by the end of 2024.

Vertex anticipates initiating a Phase 2 study with an oral formulation of VX-993 for the treatment of PNP in 2024.

APOL1-Mediated Kidney Disease (AMKD)

Vertex has discovered and advanced multiple oral, small molecule inhibitors of APOL1 function, pioneering a new class of medicines that targets an underlying genetic driver of this kidney disease.

Based on the totality of the unblinded data reviewed by the Independent Data Safety Monitoring Committee (IDMC), Vertex advanced into the Phase 3 portion of the global Phase 2/3 pivotal clinical trial in patients with AMKD, in which a 45 mg once-daily oral dose of inaxaplin will be compared to placebo, on top of standard of care.

In addition, based on the IDMC review, the trial has been expanded to include adolescents 10 to 17 years of age with AMKD.

The study is designed to have a pre-planned interim analysis at Week 48 evaluating eGFR slope, supported by a percent change from baseline in proteinuria, in the inaxaplin arm versus placebo. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval in the U.S.

Type 1 Diabetes (T1D)

Vertex is evaluating cell therapies using stem cell-derived, fully differentiated, insulin-producing islet cells to replace the endogenous insulin-producing islet cells that are destroyed in people with T1D, with the goal of developing a potential one-time functional cure for this disease.

VX-880, fully differentiated islet cells with standard immunosuppression:

Based on the totality of evidence reviewed by the IDMC, the Phase 1/2 study in people with T1D and impaired awareness of hypoglycemia and recurrent hypoglycemic events has resumed dosing.

Vertex has completed enrollment in Parts A, B, and C of the global 17-patient study and expects to complete dosing soon.

Vertex plans to present updated data from the ongoing Phase 1/2 study at the American Diabetes Association 84th Scientific Sessions Conference in June 2024.

VX-264, fully differentiated islet cells encapsulated in an immunoprotective device:

The clinical trial for VX-264, which encapsulates the same VX-880 islet cells in a novel device designed to eliminate the need for immunosuppressants, is a multi-part, Phase 1/2 study.

Vertex has completed Part A of the study and initiated Part B.

Hypoimmune, edited fully differentiated islet cells:

Vertex's hypoimmune cell program involves using CRISPR/Cas9 to gene edit the same stem cell-derived, fully differentiated islet cells used in the VX-880 and VX-264 programs to cloak the cells from the immune system. This program is progressing through the research stage.

Myotonic Dystrophy Type 1 (DM1)

Vertex is evaluating multiple approaches that target the underlying cause of DM1, the most prevalent muscular dystrophy in adults, with ~110,000 people living with the disease in the U.S. and Europe, and no approved therapies. Vertex's lead approach, VX-670, was in-licensed from Entrada Therapeutics in February 2023 and is an oligonucleotide connected to a cyclic peptide to promote effective delivery into cells, which holds the potential to address the underlying cause of DM1.

The IND in the U.S. for VX-670 has cleared, as have the clinical trial applications (CTAs) in Canada, the U.K. and the EU, and the clinical trial notification (CTN) in Australia.

Enrollment and dosing are underway.

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Vertex is developing small molecule correctors that restore function to PC1 missense variants, with the goal to address the underlying cause of ADPKD, the most common genetic kidney disease, affecting approximately 250,000 people in the U.S. and Europe.

Vertex has initiated a Phase 1 clinical trial in healthy volunteers for VX-407, a first-in-class small molecule corrector that targets the underlying cause of ADPKD in patients with a subset of PKD1 variants, estimated at ~25,000 (or ~10 percent) of the overall ~250,000 ADPKD patient population.

Investments in External Innovation

Vertex and Alpine Immune Sciences entered into a definitive agreement under which Vertex will acquire Alpine for approximately $4.9 billion in cash. Vertex has commenced a tender offer to purchase all of the outstanding shares of common stock of Alpine for $65 per share in cash. The transaction is anticipated to close in the second quarter of 2024. Alpine's lead molecule, povetacicept, is a highly potent and effective dual antagonist of BAFF (B cell activating factor) and APRIL (a proliferation-inducing ligand). Through Phase 2 development, povetacicept has shown potential best-in-class efficacy in IgA nephropathy (IgAN), a serious, progressive, autoimmune disease of the kidney that can lead to end-stage-renal disease. There are currently no approved therapies that target the underlying cause of IgAN, which is the most common cause of primary (idiopathic) glomerulonephritis worldwide, affecting approximately 130,000 people in the U.S. alone. Povetacicept is on track to enter Phase 3 clinical development in IgAN in the second half of 2024. Phase 1b/2 studies in autoimmune renal diseases and cytopenias are ongoing with data expected later this year.

Vertex achieved a clinical milestone for VX-670 in DM1 in the first quarter of 2024, resulting in a $75 million milestone payable to Entrada.

Non-GAAP Financial Measures

In this press release, Vertex's financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results and guidance exclude from Vertex's pre-tax income (i) stock-based compensation expense, (ii) intangible asset amortization expense, (iii) gains or losses related to the fair value of the company's strategic investments, (iv) increases or decreases in the fair value of contingent consideration, (v) acquisition-related costs, and (vi) other adjustments. The company's non-GAAP financial results also exclude from its provision for income taxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items. These results should not be viewed as a substitute for the company's GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures help indicate underlying trends in the company's business, are important in comparing current results with prior period results and provide additional information regarding the company's financial position that the company believes is helpful to an understanding of its ongoing business. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, to manage the company's business and to evaluate its performance. The company's calculation of non-GAAP financial measures likely differs from the calculations used by other companies. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information.

The company provides guidance regarding combined R&D, Acquired IPR&D and SG&A expenses and effective tax rate on a non-GAAP basis. Unless otherwise noted, the guidance regarding combined GAAP and non-GAAP R&D, Acquired IPR&D and SG&A expenses does not include estimates associated with any potential future business development transactions, including collaborations, asset acquisitions and/or licensing of third-party intellectual property rights. The company does not provide guidance regarding its GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax benefits related to stock-based compensation and the possibility of certain discrete items, which could be material.

Vertex Pharmaceuticals Incorporated

Consolidated Statements of Income

(in millions, except per share amounts)(unaudited)

Three Months Ended March 31,

2024

2023

Product revenues, net

$

2,690.6

$

2,374.8

Costs and expenses:

Cost of sales

342.6

266.9

Research and development expenses

789.1

742.6

Acquired in-process research and development expenses

76.8

347.1

Selling, general and administrative expenses

342.7

241.1

Change in fair value of contingent consideration

(0.1)

(1.9)

Total costs and expenses

1,551.1

1,595.8

Income from operations

1,139.5

779.0

Interest income

181.2

122.6

Interest expense

(10.4)

(11.4)

Other (expense) income, net

(31.2)

1.3

Income before provision for income taxes

1,279.1

891.5

Provision for income taxes

179.5

191.7

Net income

$

1,099.6

$

699.8

Net income per common share:

Basic

$

4.26

$

2.72

Diluted

$

4.21

$

2.69

Shares used in per share calculations:

Basic

258.2

257.4

Diluted

261.1

260.3

Vertex Pharmaceuticals Incorporated

Product Revenues

(in millions)(unaudited)

Three Months Ended March 31,

2024

2023

TRIKAFTA/KAFTRIO

$

2,483.6

$

2,096.7

Other CF products

207.0

278.1

Product revenues, net

$

2,690.6

$

2,374.8

Vertex Pharmaceuticals Incorporated

Reconciliation of GAAP to Non-GAAP Financial Information

(in millions, except percentages)(unaudited)

Three Months Ended March 31,

2024

2023

GAAP cost of sales

$

342.6

$

266.9

Stock-based compensation expense

(1.8)

(1.9)

Intangible asset amortization expense

(5.0)

-

Non-GAAP cost of sales

$

335.8

$

265.0

GAAP research and development expenses

$

789.1

$

742.6

Stock-based compensation expense

(119.4)

(76.3)

Acquisition-related costs

-

(2.8)

Non-GAAP research and development expenses

$

669.7

$

663.5

Acquired in-process research and development expenses

$

76.8

$

347.1

GAAP selling, general and administrative expenses

$

342.7

$

241.1

Stock-based compensation expense

(70.7)

(44.2)

Non-GAAP selling, general and administrative expenses

$

272.0

$

196.9

Combined non-GAAP R&D, Acquired IPR&D and SG&A expenses

$

1,018.5

$

1,207.5

GAAP other (expense) income, net

$

(31.2)

$

1.3

Decrease (increase) in fair value of strategic investments

27.0

(6.4)

Non-GAAP other expense, net

$

(4.2)

$

(5.1)

GAAP provision for income taxes

$

179.5

$

191.7

Tax adjustments (1)

81.6

22.7

Non-GAAP provision for income taxes

$

261.1

$

214.4

GAAP effective tax rate

14.0%

21.5%

Non-GAAP effective tax rate

17.4%

21.3%

Vertex Pharmaceuticals Incorporated

Reconciliation of GAAP to Non-GAAP Financial Information (continued)

(in millions, except per share amounts)(unaudited)

Three Months Ended March 31,

2024

2023

GAAP operating income

$

1,139.5

$

779.0

Stock-based compensation expense

191.9

122.4

Intangible asset amortization expense

5.0

-

Decrease in fair value of contingent consideration

(0.1)

(1.9)

Acquisition-related costs

-

2.8

Non-GAAP operating income

$

1,336.3

$

902.3

GAAP net income

$

1,099.6

$

699.8

Stock-based compensation expense

191.9

122.4

Intangible asset amortization expense

5.0

-

Decrease (increase) in fair value of strategic investments

27.0

(6.4)

Decrease in fair value of contingent consideration

(0.1)

(1.9)

Acquisition-related costs

-

2.8

Total non-GAAP adjustments to pre-tax income

223.8

116.9

Tax adjustments (1)

(81.6)

(22.7)

Non-GAAP net income

$

1,241.8

$

794.0

Net income per diluted common share:

GAAP

$

4.21

$

2.69

Non-GAAP

$

4.76

$

3.05

Shares used in diluted per share calculations:

GAAP and Non-GAAP

261.1

260.3

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases - cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia - and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency.

Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 14 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and Twitter/X.

https://news.vrtx.com/news-releases/news-release-details/vertex-reports-first-quarter-2024-financial-results

ACQ_REF: IS/44477/20240718/USA/14/4

ACQ_AUTHOR: Senior Associate/Joseph Hang Ellision