Celgene Corp.

在生物医药领域，创新如海岸线上翻涌不息的浪潮一般，奔腾向前。坐落在美国加利福尼亚州南部的圣地亚哥，正是行业浪潮的引领地之一，是当之无愧的“生物医药创新海岸（Biotech Beach）”。这里的产业生态圈汇聚着超过1000家生物医药技术公司，以及80多所相关科研机构和高等学府。学术和产业创新的成果在这里碰撞出火花，衍生出一款又一款新兴疗法，造福人类健康。深耕这片创新海岸多年，药明康德圣地亚哥基地不仅为当地的医药健康产业创新作出了积极贡献，也以更多实际行动回馈圣地亚哥生态圈，共同打造一个更为绿色和健康的社区。▲药明康德圣地亚哥基地员工及其亲友参加“世界地球日”公益活动在刚刚过去的周末，药明康德圣地亚哥基地的员工和亲友一起，于圣地亚哥泻湖（San Dieguito Lagoon）共同组织公益活动，清理塑料、玻璃瓶等人造垃圾。该活动也广受当地居民关注，多人携子女一道前来参加。正如本活动的合作伙伴、非营利组织“我想要一个干净的圣地亚哥”（ILACSD）所言，这些大大小小的垃圾如果不及时清理，可能会通过溪流和雨水渠流到海边，破坏当地生物的生存环境。与此同时，药明康德圣地亚哥的员工也在基地自发组织电子设备等回收活动，进一步兑现我们的全球绿色承诺。作为负责任的企业公民，不管是守护生命健康，帮助全球合作伙伴加速研发、为患者带来好药新药，还是守护我们的家园，与社群共生共长，都是我们为自身所扎根的创新热土作出的积极贡献。▲药明康德美国圣地亚哥基地的合全药业（上）和辉源生物（下）园区事实上，早在成立之初，药明康德就与圣地亚哥结下了不解之缘——当地蓬勃发展的生物技术产业，与药明康德“一体化、端到端”的新药研发和生产服务平台有着天生的契合。“药明康德是一个值得信赖的合作伙伴，我们已经合作了20年。”圣地亚哥的行业老兵John Hood博士曾在采访中说道。他在当地先后任职于多家明星生物医药公司，2021年，其创立的Impact Biomedicines被Celgene公司以70亿美元收购。他随后创立了Endeavor Biomedicines，并于2024年拿到1.32亿美元的C轮融资。▲John Hood博士参加药明康德圣地亚哥园区扩建庆典为了更好地赋能北美乃至全球的客户，药明康德圣地亚哥研发基地于2016年投入运营，为小分子API和制剂提供从工艺研发到生产的一体化服务，以及以生物学为中心的一系列临床前药物研发服务。“药明康德团队一直是一个稳定、高效且富有成效的合作伙伴，在我们努力为癌症患者创造有意义新疗法的过程中，我们深深感谢他们的支持。”原Mirati Therapeutics高级副总裁、药物发现负责人Matt Marx博士曾多次感谢药明康德过去10年间对KRAS抑制剂研发的支持。2022年，美国FDA批准该公司的KRAS抑制剂上市。2024年，这家在圣地亚哥起步的公司以48亿美元被百时美施贵宝收购。这一切只是药明康德做对的事、为社区和产业贡献的一个缩影。今后的日子里，我们将继续与社会各界一道，践行绿色运营的道路，并持续助力全球医药健康产业创新、为全球病患带去希望。我们深信，只有在可持续发展的环境中，科学创新才能生生不息，人类未来的生命质量才能获得持久的提升。Known as the "Biotech Beach," San Diego, located in the United States, is home to a thriving life sciences ecosystem, with over 1,000 biotech companies and 80 research institutes fueling breakthroughs in human health. Since its foundation, WuXi AppTec has been proud to contribute to this vibrant innovation hub through integrated API development and manufacturing, drug product development and manufacturing, and biology-focused preclinical drug discovery services.Our long-standing collaborations with local industry leaders—including support for KRAS inhibitor research that led to FDA approval—reflect our deep commitment to advancing science from San Diego to the world.Beyond our work in the lab, we are equally dedicated to giving back. This past weekend, employees and their families organized a volunteer clean-up at San Dieguito Lagoon, collecting waste to protect local ecosystems. Internally, our team also led a site-wide recycling initiative, reinforcing our global commitment to sustainable operations.At WuXi AppTec, we believe that scientific innovation and environmental stewardship go hand in hand—laying the foundation for a healthier future for both people and the planet.免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

Government cuts, especially those to the FDA, are putting more pressure on biotechs, fueling the potential for more M&A activity, according to Andrew Goodman, a partner in the M&A practice at business law firm Paul Hastings. While it was generally assumed that the return to power of business-friendly President Donald Trump would help facilitate M&A activity, his initial marching orders to the Federal Trade Commission suggest otherwise.Trump has instructed the antitrust regulator to continue to scrutinize deals, as it did under the direction of former President Joe Biden.Despite this, there have been indications that the M&A environment is opening up, as the value of deals in the first quarter increased by 101% compared to the fourth quarter of last year, according to a report from GlobalData. The first-quarter figure, however, doesn’t measure up to the same period of 2024 when deal values were 32% higher.“Apart from a flurry of large-scale deals driven by big pharma, the industry remains cautious given the uncertainty surrounding Trump’s as-yet-unspecified policies. So far, the start of 2025 continues to be shaped primarily by bolt-on transactions,” Ophelia Chan, an analyst at GlobalData, said in the report.One Trump measure that could have the unintended consequence of triggering M&A deals is his reductions in funding to the FDA and the National Institutes of Health.According to Andrew Goodman, a partner in the M&A practice at business law firm Paul Hastings, reductions in staff at the FDA are putting pressure on biotech companies, especially those strapped for cash.“When time periods for trials, reviews, inspections that might be specific to a certain biotech get elongated, you’re basically throwing out your existing cash runway timelines and you’ve got to come up with new ones,” Goodman said in a recent interview. “To factor in elongated timelines for the FDA to complete their review, that could be months extended to a company’s plans to get a drug approved, for example.”Goodman co-authored a client alert, “FDA Funding Changes Could Impact Biotech M&A,” which lays out several key areas in which FDA funding cuts are likely to impact M&A.Clients have told Goodman that contacts they have been working with at the FDA have suddenly departed. As a result, stockholders in some companies that work with Hastings are urging boards to proactively recalculate cash runways and examine strategic alternatives.As biotechs face funding problems or FDA delays, they may be more likely to pursue M&A to gain capital from cash-rich or synergistic partners. Additionally, well-funded companies might see opportunistic value in acquiring financially distressed companies. There may also be increased interest in biotech companies from private equity firms, Goodman said.“There are a lot of biotechs that are trading below their cash value, and I think you’ll see acquisitions of those companies by incrementally larger companies,” Goodman noted. “They may have a program that’s strategic or complementary, but what they’re really doing is using M&A as a financing solution.”In the short term, working against a surge in M&A is the overall turbulence in the macro economy and what is going on with tariffs, according to Ben Folwell, an analyst with Citeline.“They’re big investments. They take a long time, so they tend to require stability for them to really take place,” Folwell said. “That said, we’re still in a position where there are companies—particularly big companies—that are going to be looking to refill their pipelines with looming patent cliffs getting ever closer.” Among the companies specified by Folwell that lose their patent protection for drugs this year are Johnson & Johnson (Xarelto), Novartis (Entresto), Amgen (Prolia, Xgeva) and Pfizer (Xeljanz, Inlyta).The analysts agree that once the implications of tariffs and other aspects of the transition to the Trump administration are sorted out, there will be a surge in activity.“I think we’re gonna sit here for probably a couple more months while people get comfortable and acknowledge the fact that there’s not going to be certainty,” Goodman said. “But there’s gonna have to be M&A transactions for certain companies to survive, and there’s going to be acquirers who see opportunities to pounce, and they’ll do it. I think in the second half of this year, we’ll see more M&A.” Folwell doesn't rule out a return to the splurge of huge biopharma deals late in the first Trump administration, when Bristol Myers Squibb acquired Celgene for $74 billion, Takeda snapped up Shire for $64 billion and AbbVie bought out Allergan for $63 billion. "We had a lot of companies deciding at once that they needed to look to the future and really become these global forces that they are now," Folwell said. "They were saying, 'We need to add in these big, successful companies that have got proven medicines but also very strong pipelines to really transform what the company is.'" 

– U.S. Regulatory Filing Under Active Review for Approval of PYRUKYND® (mitapivat) in Thalassemia, with PDUFA Goal Date of September 7, 2025 – – Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease On Track, with Topline Results Expected in Late 2025; Potential U.S. Commercial Launch in 2026 – – Tebapivat Advancing in Clinical Trials for Lower-Risk Myelodysplastic Syndromes (LR-MDS) and Sickle Cell Disease – – PYRUKYND Net Revenue of $8.7 Million in Q1; Cash, Cash Equivalents and Marketable Securities of $1.4 Billion as of March 31, 2025 – CAMBRIDGE, Mass. , May 01, 2025 (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 first quarter ended March 31, 2025 . “We are pleased with our strong start to 2025, highlighted by the acceptance of our sNDA for thalassemia with a PDUFA goal date of September 7, 2025 . Our engagement with the FDA is progressing as expected, and we are committed to bringing PYRUKYND to thalassemia patients, irrespective of genotype or transfusion needs,” said Brian Goff , chief executive officer at Agios. “Looking ahead, our focus is also on delivering the topline results from the Phase 3 RISE UP study in sickle cell disease, which remains on track for year-end, and continuing to advance our early and mid-stage clinical programs. Supported by our strong financial position and highly experienced team, we are driving forward PYRUKYND’s multi-billion-dollar potential while building a pipeline designed for lasting impact, with the goal of creating significant value for shareholders and delivering transformative therapies for patients.” First Quarter 2025 and Recent Highlights PYRUKYND® Revenues: Generated $8.7 million in net revenue for the first quarter of 2025, compared to $8.2 million in the first quarter of 2024. A total of 234 unique patients have completed prescription enrollment forms, representing an increase of 5 percent over the fourth quarter of 2024. A total of 136 patients are on PYRUKYND therapy, inclusive of new prescriptions and continued therapy, as compared to 130 patients at the end of the fourth quarter 2024. Thalassemia: The U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025. The FDA has communicated that at this time no advisory committee meeting is planned, and the review is ongoing. Sickle Cell Disease: The Phase 3 RISE UP study evaluating mitapivat for the treatment of sickle cell disease patients who are 16 years of age or older continued to progress as anticipated. This 52-week Phase 3 study completed enrollment in October 2024 , enrolling more than 200 patients worldwide. Advanced final preparations to initiate a Phase 2 clinical trial of tebapivat in patients with sickle cell disease in mid-2025. Pediatric Pyruvate Kinase (PK) Deficiency: Reported positive topline results from the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused. Safety was consistent with the profile for mitapivat previously observed in adults with PK deficiency who are not regularly transfused. ACTIVATE-Kids is the first study to demonstrate efficacy of an oral therapy for children with PK Deficiency who are not regularly transfused. Lower-risk Myelodysplastic Syndromes (LR-MDS): Progressed patient enrollment in the Phase 2b study of tebapivat in LR-MDS. Corporate: Krishnan Viswanadhan , Pharm. D, joined Agios as Chief Corporate Development and Strategy Officer, responsible for leading the company’s corporate strategy, business development, and long-term growth initiatives. Previously, he served as President and Chief Operating Officer of Be Biopharma and at various senior roles at both Bristol Myers Squibb and Celgene. The U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025. The FDA has communicated that at this time no advisory committee meeting is planned, and the review is ongoing. The Phase 3 RISE UP study evaluating mitapivat for the treatment of sickle cell disease patients who are 16 years of age or older continued to progress as anticipated. This 52-week Phase 3 study completed enrollment in October 2024 , enrolling more than 200 patients worldwide. Advanced final preparations to initiate a Phase 2 clinical trial of tebapivat in patients with sickle cell disease in mid-2025. Reported positive topline results from the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused. Safety was consistent with the profile for mitapivat previously observed in adults with PK deficiency who are not regularly transfused. ACTIVATE-Kids is the first study to demonstrate efficacy of an oral therapy for children with PK Deficiency who are not regularly transfused. Progressed patient enrollment in the Phase 2b study of tebapivat in LR-MDS. Key Upcoming Milestones & Priorities Agios expects to achieve the following key milestones in 2025: Thalassemia: Receive FDA regulatory decision for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia (PDUFA goal date is September 7, 2025). Continue progressing the review of regulatory applications with health authorities in the European Union , Kingdom of Saudi Arabia and United Arab Emirates . Sickle Cell Disease: Announce topline results from the Phase 3 RISE UP study of mitapivat in sickle cell disease in late 2025, with a potential U.S. commercial launch in 2026. Additionally, begin patient enrollment for the Phase 2 study of tebapivat in sickle cell disease in mid-2025. LR-MDS: Complete patient enrollment in the Phase 2b study of tebapivat for LR-MDS in late 2025. Early-Stage Pipeline: File an Investigational New Drug Application for AG-236, an siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera, in mid-2025. First Quarter 2025 Financial Results Revenue: Net product revenue from sales of PYRUKYND for the first quarter of 2025 was $8.7 million , compared to $8.2 million for the first quarter of 2024. Cost of Sales: Cost of sales for the first quarter of 2025 was $1.1 million . Research and Development (R&D) Expenses: R&D expenses were $72.7 million for the first quarter of 2025, compared to $68.6 million for the first quarter of 2024. The year-over-year increase was primarily attributed to an increase in workforce-related expenses and costs associated with clinical trials of tebapivat in LR-MDS and sickle cell disease, partially offset by lower costs associated with the clinical trials of mitapivat in thalassemia and pediatric PKD. Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $41.5 million for the first quarter of 2025 compared to $31.0 million for the first quarter of 2024. The year-over-year increase was primarily attributable to an increase in commercial-related activities, including headcount, as the company prepares for the potential approval of PYRUKYND in thalassemia. Net Loss: Net loss was $89.3 million for the first quarter of 2025 compared to $81.5 million for the first quarter of 2024. Cash Position and Guidance: Cash, cash equivalents and marketable securities as of March 31, 2025 , were $1.4 billion compared to $1.5 billion as of December 31, 2024 . 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 today at 8:00 a.m. ET to discuss the company’s first quarter 2025 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company's website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast will be available on the company’s website 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-236 and AG-181; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, tebapivat, AG-236 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 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 AG-236, 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. Contacts: Investor Contact Chris Taylor , VP, Investor Relations and Corporate Communications Agios Pharmaceuticals IR@agios.com Media Contact Eamonn Nolan , Senior Director, Corporate Communications Agios Pharmaceuticals media@agios.com Source: Agios Pharmaceuticals, Inc.