Les Laboratoires Servier SAS

– Completed Enrollment of the Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease; Topline Data from 52-Week Study Expected in Late 2025 – – Received $1.1 Billion in Payments from Royalty Pharma and Servier, Following FDA Approval of Vorasidenib – – Commenced Enrollment of the Phase 2b Study of Tebapivat in Lower-Risk Myelodysplastic Syndromes (LR-MDS); Granted FDA Orphan Drug Designation for Treatment of MDS – – PYRUKYND® (Mitapivat) Net Revenue of $9.0 Million in Q3; Cash, Cash Equivalents and Marketable Securities of $1.7 Billion as of September 30, 2024 – CAMBRIDGE, Mass., Oct. 31, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today reported business highlights and financial results for the third quarter ended September 30, 2024. “We had a strong quarter, marked by several important advancements across our pipeline. We completed enrollment of our Phase 3 RISE UP study of mitapivat in sickle cell disease, on our way to sharing topline results in late 2025. Our Phase 2b study of tebapivat in lower-risk MDS was initiated, and we received orphan drug designation from the FDA to support the development of tebapivat in this indication,” said Brian Goff, chief executive officer of Agios. “Our cash position was further strengthened by the receipt of $1.1 billion in payments. This will allow us to maintain this great momentum and fuel our next phase of growth, building towards a franchise with multi-billion-dollar potential. We remain focused on progressing our promising clinical programs to address the critical needs of rare disease patients and look forward to sharing our progress in the coming months.” Third Quarter 2024 and Recent Highlights PYRUKYND® Revenues: Generated $9.0 million in net revenue for the third quarter of 2024, a 4 percent increase from the second quarter of 2024, primarily driven by increased patient demand. A total of 211 unique patients have completed prescription enrollment forms, representing an increase of 5 percent over the second quarter of 2024. A total of 127 patients are on PYRUKYND® therapy, inclusive of new prescriptions and continued therapy.Sickle Cell Disease: Completed enrollment of the 52-week Phase 3 RISE UP study of mitapivat in sickle cell disease, with more than 200 patients enrolled worldwide, and topline data expected in late 2025. Lower-risk Myelodysplastic Syndromes: Initiated patient enrollment in the Phase 2b study of tebapivat (AG-946).Granted Orphan Drug Designation from the FDA to tebapivat for the treatment of myelodysplastic syndromes (MDS). Presented topline data from Agios’ Phase 3 ACTIVATE-KidsT trial of mitapivat in pediatric patients with PKD. Observed transfusion reduction response rates were higher in the mitapivat arm compared to placebo and were clinically meaningful despite not meeting prespecified statistical criterion for the primary endpoint; secondary endpoints of transfusion-free response and normal hemoglobin response were observed only in the mitapivat arm.Safety was consistent with the profile observed in adults with PK deficiency who are regularly transfused. Presented data from the Phase 3 ENERGIZE trial as an encore session at the 19th Annual Academy for Sickle Cell and Thalassemia (ASCAT) Conference.Corporate Development: The FDA approved Servier’s vorasidenib for the treatment of IDH-mutant diffuse glioma. As a result, Agios received a $200 million milestone payment from Servier and a $905 million payment from Royalty Pharma in connection with the purchase agreement announced in May 2024.Entered into a distribution agreement with NewBridge Pharmaceuticals to advance commercialization of PYRUKYND® in the Gulf Cooperation Council (GCC) region. NewBridge, a leading specialty company headquartered in Dubai, will commercialize PYRUKYND® in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia and the United Arab Emirates. Key Upcoming Milestones & Priorities Agios expects to achieve the following key milestones by the end of 2024: Thalassemia: File sNDA for mitapivat in thalassemia based on the positive results from the Phase 3 ENERGIZE and ENERGIZE-T clinical trials.Present additional clinical data at the 66th Annual American Society of Hematology (ASH) Congress, taking place December 7-10, 2024, in San Diego. Third Quarter 2024 Financial Results Revenue: Net product revenue from sales of PYRUKYND® for the third quarter of 2024 was $9.0 million, compared to $7.4 million for the third quarter of 2023. Cost of Sales: Cost of sales for the third quarter of 2024 was $0.8 million. Research and Development (R&D) Expenses: R&D expenses were $72.5 million for the third quarter of 2024, compared to $81.8 million for the third quarter of 2023. The year-over-year decrease was primarily driven by the $17.5 million upfront payment associated with the license agreement with Alnylam, which was recorded in the prior year. Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $38.5 million for the third quarter of 2024 compared to $25.8 million for the third quarter of 2023. The year-over-year increase was primarily attributable to an increase in commercial-related activities as the company prepares for the potential approval of PYRUKYND® in thalassemia. Net Income (Loss): Net income was $947.9 million for the third quarter of 2024 compared to a net loss of $91.3 million for the third quarter of 2023, reflecting the milestone and royalty agreement income recorded in the third quarter of 2024. Cash Position and Guidance: Cash, cash equivalents and marketable securities as of September 30, 2024, were $1.7 billion compared to $806.4 million as of December 31, 2023. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND® launches in thalassemia and sickle cell disease, advance existing programs, and to opportunistically expand its pipeline through both internally and externally discovered assets. Conference Call Information Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss third quarter 2024 financial results and recent business highlights. The live webcast can be accessed under “Events & Presentations” in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company's website beginning approximately two hours after the event. About Agios Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company's deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndromes (MDS)-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera. For more information, please visit the company’s website at www.agios.com. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND® (mitapivat), tebapivat (AG-946), TMPRSS6 siRNA and AG-181, Agios’ PAH stabilizer; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, tebapivat, and AG-181; Agios’ use of proceeds from the transaction with Royalty Pharma; potential U.S. net sales of vorasidenib and potential future royalty payments; Agios’ strategic vision and goals, including its key milestones for 2024 and 2025; and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of TMPRSS6 siRNA, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Consolidated Balance Sheet Data(in thousands)(Unaudited) September 30, 2024 December 31, 2023Cash, cash equivalents, and marketable securities $1,660,646 $806,363Accounts receivable, net 3,118 2,810Inventory 26,429 19,076Total assets 1,791,794 937,118Stockholders' equity 1,626,672 811,019 Consolidated Statements of Operations Data(in thousands, except share and per share data)(Unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Revenues: Product revenue, net$8,964 $7,399 $25,768 $19,720 Total revenue 8,964 7,399 25,768 19,720 Operating expenses: Cost of sales$783 $633 $2,905 $2,295 Research and development 72,455 81,841 218,476 218,037 Selling, general and administrative 38,537 25,822 105,087 84,598 Total operating expenses 111,775 108,296 326,468 304,930 Loss from operations (102,811) (100,897) (300,700) (285,210)Gain on sale of contingent payments 889,136 — 889,136 — Milestone payment from gain on sale of oncology business 200,000 — 200,000 — Interest income, net 13,059 8,375 30,068 24,720 Other income, net 1,651 1,198 4,864 4,342 Net income (loss) before taxes 1,001,035 (91,324) 823,368 — (256,148)Income tax expense 53,120 — 53,120 — Net income (loss)$947,915 $(91,324) $770,248 $(256,148)Net income (loss) per share - basic$16.65 $(1.64) $13.58 $(4.61)Net income (loss) per share - diluted$16.22 $(1.64) $13.38 $(4.61)Weighted-average number of common shares used in computing net income (loss) per share – basic 56,939,403 55,803,663 56,709,318 55,559,766 Weighted-average number of common shares used in computing net income (loss) per share – diluted 58,432,796 55,803,663 57,581,382 55,559,766 Contacts: Investor ContactChris Taylor, VP, Investor Relations and Corporate CommunicationsAgios PharmaceuticalsIR@agios.com Media ContactEamonn Nolan, Senior Director, Corporate CommunicationsAgios Pharmaceuticalsmedia@agios.com

SOMERVILLE, Mass., Oct. 30, 2024 /PRNewswire/ -- Aitia, a biotech company pioneering the use of causal AI and Digital Twins in drug discovery and development, is expanding its collaboration with Servier, a global and independent pharmaceutical company, to tackle gliomas, a broad group of brain cancers. This collaboration aims to use Aitia's breakthrough technology which is powered by millions of data points from patient cancer tissues to find new treatments for this deadly disease. Under the terms of the collaboration, Aitia and Servier will work together to discover and, validate novel drug targets and develop novel drug candidates for glioma using Aitia's Gemini Digital Twins and Servier's therapeutic discovery and clinical development expertise in oncology. This collaboration will include exploration of the heterogeneous biology of these brain cancers, mechanisms of resistance, and drug response at the individual patient level. In a recent breakthrough in another form of glioma, specifically Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation following surgery including biopsy, sub-total resection, or gross total resection in adult and pediatric patients 12 years and older, Servier recently announced approval of the brain cancer drug, VORANIGO, the first approved drug for this disease area in almost 25 years. Gliomas, tumors that originate in the brain or spinal cord from glial cells, affects more than 190,000 people worldwide annually. The most aggressive form of glioma, glioblastoma, has an average survival time after diagnosis of approximately 15 months, with less than 7% of patients surviving beyond five years, according to the National Brain Tumor Society. Despite standard treatments like surgery, radiation and chemotherapy, glioblastoma has a high rate of recurrence and there are currently no curative therapies. "We are excited to expand our collaboration with Aitia into gliomas as we continue to prioritize brain cancer after our recent positive results and approval of VORANIGO in the US and in growing number of territories," said Fabien Schmidlin, Global Head of Translational Medicine at Servier. "At Servier, we are committed to advancing therapies for cancer patients with high unmet medical needs, and we believe that integrating Aitia's Gemini Digital Twins with our expertise in oncology, particularly in targeting molecular mutations, could open new avenues for more effective treatments." "Glioma is the among the deadliest forms of cancer, and the lack of broadly effective treatments highlights the urgent need for new approaches that leverage the growth of genetic and multi-omic patient data and breakthroughs in AI," said Colin Hill, CEO and co-founder of Aitia. "We believe that our Gemini Digital Twins, along with Servier's drug discovery and clinical development expertise in hard-to-treat cancers can lead to new breakthrough therapies for this disease." This collaboration represents the fourth area of research Aitia and Servier are collaborating on. The companies began their partnership in 2022, to advance drug discovery, translational, and clinical development efforts in Multiple Myeloma, then expanding in 2023 to include drug discovery and development for pancreatic cancer, and again in January 2024 to discover patient responders for Servier's LRRK2 inhibitor in Parkinson's disease. Aitia's approach centers on two key innovations: causal AI and Gemini Digital Twins. Causal AI goes beyond correlative associations to identify which genetic or molecular changes drive diseases and patient response to therapies. Gemini Digital Twins are virtual models that are reverse-engineered from huge quantities of genetic, multi-omic, and clinical data from patients that reveal how genes, proteins, and other molecules interact within cells and tissues to drive clinical outcomes. These Gemini Digital Twins of a specific disease allow researchers to rapidly test billions of experiments such as gene and protein knockdowns or application of a drug candidate in a human patient context versus animal models or cells in petri dishes. These high-throughput "virtual experiments" in patient-derived Gemini Digital Twins leads to discovering and selecting drug candidates that are more likely to succeed in clinical trials in specific patient populations. About Aitia Aitia is an AI-enabled biotechnology company deploying causal AI and creating Gemini Digital Twins to discover the next generation of breakthrough drugs in neurodegenerative diseases and oncology, including a lead program in Huntington's disease. By leveraging the convergence of multi-omic patient data, high-performance computing, and causal AI, Aitia's Gemini Digital Twins reveal the hidden biological mechanisms of disease to transform the discovery and development of new therapeutics. To date, Gemini Digital Twins have been used to accelerate R&D in oncology, neurodegeneration, cardiometabolic, and immunology. Media Contact: Isabelle Caffry Senior Director, Strategy & Corporate Development [email protected] +1-617-374-2300 Ryan Flinn In Like Flinn Communications [email protected] +1 510-207-7616 SOURCE Aitia WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Alvotech (NASDAQ: ALVO), a global biotech company specializing in the development and manufacture of biosimilar medicines for patients worldwide, announced today its participation in the Jefferies London Healthcare Conference, November 19-20, 2024. Members of the management team will host one-on-one meetings on both days. Alvotech will be presenting at the conference on Tuesday, November 19, 2024 at 9:00-9:25 am GMT. An audio webcast of the conference presentation will also be available to investors and the general public and can be accessed in the Investors Section of Alvotech’s website under News and Events – Events and Presentations . After the event, a recording will also be available for 90 days. About Alvotech Alvotech is a biotech company, founded by Robert Wessman, focused solely on the development and manufacture of biosimilar medicines for patients worldwide. Alvotech seeks to be a global leader in the biosimilar space by delivering high quality, cost-effective products, and services, enabled by a fully integrated approach and broad in-house capabilities. Alvotech has launched two biosimilars. The current development pipeline includes nine disclosed biosimilar candidates aimed at treating autoimmune disorders, eye disorders, osteoporosis, respiratory disease, and cancer. Alvotech has formed a network of strategic commercial partnerships to provide global reach and leverage local expertise in markets that include the United States, Europe, Japan, China, and other Asian countries and large parts of South America, Africa and the Middle East. Alvotech’s commercial partners include Teva Pharmaceuticals, a US affiliate of Teva Pharmaceutical Industries Ltd. (US), STADA Arzneimittel AG (EU), Fuji Pharma Co., Ltd (Japan), Advanz Pharma (EEA, UK, Switzerland, Canada, Australia and New Zealand), Dr. Reddy’s (EEA, UK and US), Biogaran (FR), Cipla/Cipla Gulf/Cipla Med Pro (Australia, New Zealand, South Africa/Africa), JAMP Pharma Corporation (Canada), Yangtze River Pharmaceutical (Group) Co., Ltd. (China), DKSH (Taiwan, Hong Kong, Cambodia, Malaysia, Singapore, Indonesia, India, Bangladesh and Pakistan), YAS Holding LLC (Middle East and North Africa), Abdi Ibrahim (Turkey), Kamada Ltd. (Israel), Mega Labs, Stein, Libbs, Tuteur and Saval (Latin America) and Lotus Pharmaceuticals Co., Ltd. (Thailand, Vietnam, Philippines, and South Korea). Each commercial partnership covers a unique set of product(s) and territories. Except as specifically set forth therein, Alvotech disclaims responsibility for the content of periodic filings, disclosures and other reports made available by its partners. For more information, please visit Please visit our investor portal , and our website or follow us on social media on LinkedIn , Facebook , Instagram , X and YouTube . ALVOTECH INVESTOR RELATIONS AND GLOBAL COMMUNICATIONS Benedikt Stefansson, VP alvotech.ir@alvotech.com