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Merck’s Winrevair excelled in a phase 3 trial of high-risk pulmonary arterial hypertension patients, reducing the risk of death, lung transplantation and hospitalization by 76% versus placebo. The interim result was conclusive enough that Merck halted the trial and another Winrevair phase 3 study of newly diagnosed patients. The interim results of a phase 3 trial of Merck’s pulmonary arterial hypertension (PAH) treatment Winrevair were so conclusive that the company not only halted the study for efficacy, but did the same two months later in another trial of the first-in-class activin signaling inhibitor.Merck has revealed data from the study, providing further evidence of the value of Winrevair, which has been approved in more than 40 countries, including in the U.S. a year ago. The Zenith trial included 172 patients at the highest risk of mortality—those in the World Health Organization Functional Class (FC) III or IV—and achieved its primary endpoint of time to clinical worsening to first morbidity or mortality event.Winrevair added to maximum background therapy reduced the risk of all-cause death, lung transplantation and hospitalization by 76% versus placebo plus maximum background therapy. Patients received a subcutaneous dose of Winrevair every three weeks, and the median follow-up with patients was 10.6 months.“I’ve been doing clinical research for almost 30 years and have never seen a study with a hard-outcome endpoint with this effect size,” Joerg Koglin, M.D., Merck’s head of general medicine, global clinical development, said in an interview with Fierce Pharma. “There has not been a PH study that used an endpoint based on death, hospitalization or lung transplant. It also is the first PH phase 3 study that was ever stopped early. I think that puts the effect size in perspective.”Merck presented the Zenith data at the American College of Cardiology Annual Scientific Session & Expo on March 31 in Chicago. Results also were reported in The New England Journal of Medicine.   For Winrevair patients in Zenith, 17% had one or more major morbidity or mortality event, compared to 55% of patients in placebo arm. Of the 86 patients on each side of the study, there were seven deaths in the Winrevair arm (8%) compared with 13 in the placebo arm (15%).One patient on Winrevair had a lung transplant (1%) while six on placebo (7%) needed the procedure. PAH hospitalizations occurred in eight patients on Winrevair (9%) compared to 43 patients (50%) on placebo.Koglin said that Merck pursued an ambitious endpoint—not undertaken in other trials—because Winrevair is the first medicine that targets the underlying cause of PAH, while other therapies for the disorder ameliorate its symptoms.One secondary endpoint where the study came up short was overall survival. While there were nearly twice as many deaths (13) on the placebo side of the trial compared to Winrevair (seven), the difference did not achieve statistical significance.“When you run an interim analysis, you are using a much higher statistical bar because the interim analysis is much smaller,” Koglin explained.Setting up Winrevair for its FDA approval was the Stellar phase 3 study, which used exercise capacity as its primary endpoint. Patients who were on Winrevair added to standard of care improved their distance in a six-minute walk test by an average of 41 meters.While the Stellar trial included longtime PAH patients at a lower risk of mortality (from the FC II and III classes), Merck’s phase 3 Hyperion study focused on those who were diagnosed with the disorder within the previous 12 months.Hyperion was halted two months ago after a review of the interim results of the Zenith study. Merck’s chief medical officer Eliav Barr, M.D., said in a release that it would be unethical to give patients placebo when the superiority of Winrevair was clearly established.All 300 patients in both the Zenith and Hyperion trials were offered to receive Winrevair through the Soteria open-label extension study. Koglin said the company will reveal Hyperion data later this year.PAH is a rare, rapidly progressing disease in which the blood vessels in the lungs become thickened and narrower due to a dangerous buildup of cells, leading to shortness of breath and difficulty in the heart’s ability to pump blood to the lungs, which increases blood pressure and can lead to heart failure. Winrevair works by rebalancing the cell signaling.Roughly 40,000 people in the U.S. have the disorder. PAH strikes more women than men and most are in middle age, killing nearly half within five years of diagnosis. The condition begins as a more common disorder, pulmonary hypertension (PH), then can progress into PAH. Winrevair was initially developed by Acceleron and Celgene. Merck gained its rights with its $11.5 billion buyout of Acceleron in 2021. The company makes royalty payments on sales of Winrevair to Bristol Myers Squibb, which bought out Celgene in 2019.Merck reported 2024 sales of Winrevair at $419 million, with nearly half of that, $200 million, coming in the fourth quarter alone.Analysts from Citi said that the full dataset from Zenith shows the "paradigm shifting impact of Winrevair in PAH. ” Citi has boosted its 2025 sales projection of Winrevair from $1.3 billion to $1.5 billion, while lifting its 2030 peak sales estimate from $5.7 billion to $6.1 billion. Johnson & Johnson controls the PAH market with endothelin receptor agonist Opsumit and prostacyclin receptor Uptravi, which were approved in 2013 and 2015, respectively, and garnered combined sales of $4 billion in 2024.Last year, the FDA approved a combination tablet Opsynvi, which combines Opsumit and United Therapeutics’ Adcirca, which are often prescribed together for PAH. These therapies dilate the blood vessels to alleviate PAH symptoms.

关注并星标CPHI制药在线 过去十年，制药与生物技术行业在创新和战略合作的推动下取得了显著增长。其中，并购（M&A）在重塑行业格局中发挥了关键作用，帮助企业加强产品组合、提升研发能力并扩大全球市场覆盖。这些高额交易往往旨在解决未满足的医疗需求、推进治疗管线并开辟新的收入来源。 本文将回顾过去十年中最具影响力的十大制药与生物技术并购案，展示这些交易如何塑造行业未来。从百时美施贵宝（Bristol-Myers Squibb）对新基（Celgene）的巨额收购，到辉瑞（Pfizer）对西雅图基因（Seagen）的收购，这些交易体现了推动生命科学创新的战略与雄心。 1. 百时美施贵宝（BMS）收购新基（Celgene） 交易价值：740亿美元 宣布日期：2019年1月3日 完成日期：2019年11月20日 关键收益： ● 获得了新基的重磅药物Revlimid（来那度胺），这是其肿瘤学产品组合的核心。 ● 获得了创新的CAR-T疗法，如Abecma（idecabtagene vicleucel）。 2019年，百时美施贵宝以现金加股票的方式收购新基，交易价值达740亿美元。这一战略收购显著增强了BMS在肿瘤学、免疫学、炎症和心血管疾病领域的产品组合。 尽管交易中需要剥离部分资产（如将Otezla以134亿美元出售给安进），但这一并购巩固了BMS在肿瘤学和免疫学领域的领先地位。 2. 阿特维斯（Actavis）收购艾尔建（Allergan） 交易价值：705亿美元 宣布日期：2014年11月17日 完成日期：2015年3月17日 关键收益：        ● 获得了艾尔建在眼科、神经科学和医美领域的强大产品组合，如Botox（肉毒杆菌毒素）和Restasis（环孢素）。 阿特维斯以现金加股票的方式收购艾尔建，交易价值达705亿美元。这一并购使阿特维斯转型为全球最大的制药公司之一，覆盖品牌药、仿制药和非处方药（OTC）领域。 3. 艾伯维（AbbVie）收购艾尔建（Allergan） 交易价值：630亿美元 宣布日期：2019年6月25日 完成日期：2020年5月8日 关键收益： ● 获得了艾尔建在医美、神经科学、眼科和胃肠病学领域的领先产品，如Botox和Juvederm。 艾伯维以现金加股票的方式收购艾尔建，交易价值达630亿美元。这一收购旨在多元化艾伯维的收入来源，减少对其旗舰药物Humira（阿达木单抗）的依赖。 4. 武田（Takeda）收购夏尔（Shire） 交易价值：620亿美元 宣布日期：2018年5月8日        完成日期：2019年1月8日 关键收益： 获得了夏尔在罕见病领域的领先疗法，如Xiidra（干眼症治疗药物）和Adynovate（血友病治疗药物）。 武田以现金加股票的方式收购夏尔，交易价值达620亿美元。这一收购使武田成为罕见病、胃肠病学、神经科学和免疫学领域的全球领导者。 5. 辉瑞（Pfizer）收购西雅图基因（Seagen） 交易价值：430亿美元 宣布日期：2023年3月13日 完成日期：2023年12月14日 关键收益： ● 获得了Seagen的ADC产品，如Adcetris（淋巴瘤治疗药物）和Padcev（膀胱癌治疗药物）。 辉瑞以现金方式收购西雅图基因，交易价值达430亿美元。这一收购强化了辉瑞在肿瘤学领域的产品组合，特别是抗体药物偶联物（ADC）技术。 6. 阿斯利康（AstraZeneca）收购亚力兄（Alexion） 交易价值：390亿美元 宣布日期：2020年12月12日 完成日期：2021年7月21日 关键收益： ● 获得了亚力兄的重磅药物Soliris和Ultomiris，用于治疗补体介导的罕见病。 阿斯利康以现金加股票的方式收购亚力兄，交易价值达390亿美元。这一收购标志着阿斯利康进军罕见病领域。 7. 强生（J&J）收购爱可泰隆（Actelion） 交易价值：300亿美元 宣布日期：2017年1月26日 完成日期：2017年6月16日 关键收益： ● 获得了爱可泰隆的PAH药物，如Tracleer、Opsumit和Uptravi。 强生以现金方式收购爱可泰隆，交易价值达300亿美元。这一收购强化了强生在肺动脉高压（PAH）领域的市场地位。 8. 安进（Amgen）收购 Horizon Therapeutics 交易价值：278亿美元 宣布日期：2022年12月12日 完成日期：2023年10月6日 关键收益： ● 获得了Horizon的罕见病疗法，如Tepezza（甲状腺眼病治疗药物）和Krystexxa（慢性痛风治疗药物）。 安进以现金方式收购Horizon Therapeutics，交易价值达278亿美元。这一收购显著增强了安进在罕见病和炎症领域的产品组合。 9. 艾伯维（AbbVie）收购 Pharmacyclics 交易价值：210亿美元 宣布日期：2015年3月4日 完成日期：2015年5月26日 关键收益： ● 获得了Pharmacyclics的重磅药物Imbruvica（依鲁替尼），用于治疗多种血液癌症。 艾伯维以现金加股票的方式收购Pharmacyclics，交易价值达210亿美元。这一收购强化了艾伯维在肿瘤学领域的产品组合。 10. 吉利德（Gilead）收购 Immunomedics 交易价值：210亿美元       宣布日期：2020年9月13日 完成日期：2020年10月23日 关键收益： ● 获得了Immunomedics的ADC技术及其重磅药物Trodelvy，用于治疗三阴性乳腺癌。 吉利德以现金方式收购Immunomedics，交易价值达210亿美元。这一收购显著增强了吉利德在肿瘤学领域的产品组合。 结语 过去十年的制药与生物技术并购浪潮，不仅是资本与资源的整合，更是一场关于创新、战略与未来的深度博弈。这些动辄数百亿美元的交易，映射出行业的核心趋势： ● 创新驱动：从CAR-T疗法到ADC技术，并购成为企业快速获取尖端科技、填补管线空缺的关键手段。 ● 风险对冲：面对专利悬崖与仿制药冲击，巨头们通过收购多元化布局，降低对单一产品的依赖。 ● 聚焦高壁垒领域：罕见病、肿瘤、免疫疾病等高壁垒领域成为并购焦点，推动更多“无药可医”的困境被打破。 然而，高额交易也伴随挑战——巨额债务、整合风险、文化冲突，无一不考验着企业的战略定力。未来，随着基因治疗、AI药物发现等新兴领域的崛起，并购或将更加精准化、技术化，成为行业重塑的常态。 参考资料： 1、https://xtalks.com/category/blogs/life-science-blogs/pharma-blogs/ END 2025金笔奖征文活动开启，来投稿吧！ 领取CPHI & PMEC China 2025展会门票 智药研习社直播预告 来源：CPHI制药在线 声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。 投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~