Sotatercept

May 16, 2025 7:45 am ET P reliminary results from a pooled analysis of clinical trial data evaluating the long-term safety and favorable benefit-risk profile of WINREVAIR™ to be featured as a late-breaking presentation RAHWAY, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced new clinical and outcomes research data on pulmonary arterial hypertension (PAH) to be presented at the American Thoracic Society’s (ATS) 2025 International Conference in San Francisco from May 16-21. Data from nine presentations demonstrate Merck’s commitment to advancing research for patients with this disease. Merck will present pooled data from participants who have completed the PULSAR, SPECTRA, or STELLAR studies and have continued in the ongoing open-label extension study, SOTERIA, evaluating the long-term safety, tolerability and efficacy of WINREVAIR™ (sotatercept-csrk) when added to background therapy for the treatment of PAH. These results represent the largest analysis of WINREVAIR to date. "The results from this pooled analysis add to the growing body of evidence for WINREVAIR,” said Dr. Eliav Barr, senior vice president, head of global clinical development and chief medical officer, Merck Research Laboratories. “We remain confident in the long-term potential for WINREVAIR for patients with PAH and look forward to sharing findings as we continue to evaluate WINREVAIR." Merck will also present an overall survival analysis for WINREVAIR leveraging data from the pivotal Phase 3 study, STELLAR, and interim data from participants who have continued in the ongoing open-label SOTERIA study, as well as additional outcomes research on the burden and impact of PAH in various patient populations. Details on Merck abstracts at ATS: PAH Long-term safety and exposure-adjusted incidence rates of adverse events from pooled sotatercept studies (PULSAR, SPECTRA, STELLAR, and SOTERIA). I. Preston. Session B96 Crossing the Golden Gate Bridge: clinical trials, targeted therapies and novel approaches in pulmonary arterial hypertension on Monday, May 19, 5:15 – 7:15 p.m. ET/2:15 – 4:15 p.m. PT Overall survival of patients on sotatercept: an analysis of STELLAR and SOTERIA trials. T. Thakur. Session B96 Crossing the Golden Gate Bridge: clinical trials, targeted therapies and novel approaches in pulmonary arterial hypertension on Monday, May 19, 5:15 – 7:15 p.m. ET/2:15 – 4:15 p.m. PT Long-term safety of MK-5475 in pulmonary arterial hypertension (PAH): Results from the phase 2 INSIGNIA-PAH extension period. P. Hassoun. Poster Board #505; D28 Fisherman’s Wharf: Clinical and epidemiological insights into pulmonary hypertension: trends, treatments, and outcomes on Wednesday, May 21, 11:15 a.m. – 1:15 p.m. ET/8:15 – 10:15 a.m. PT Quantifying the impact of pulmonary arterial hypertension (PAH) on health-related quality of life (HRQOL), work productivity, and economic stability for patients and their caregivers. H. Black. Poster Board #1018; B23 Diffuse lung disease and pulmonary hypertension: clinical perspectives on Monday, May 19, 12:15 –2:15 p.m. ET/9:15 –11:15 a.m. PT Comparing the burden of pulmonary arterial hypertension (PAH) among women across regions: a patient survey. T. Thakur. Poster Board #1021; B23 Diffuse lung disease and pulmonary hypertension: clinical perspectives on Monday, May 19, 12:15 – 2:15 p.m. ET/9:15 – 11:15 a.m. PT Perceptions, attitudes and behaviors among women of childbearing and childrearing age with pulmonary arterial hypertension. T. Thakur. Poster Board #1020; B23 Diffuse lung disease and pulmonary hypertension: clinical perspectives on Monday, May 19, 12:15 –2:15 p.m. ET/9:15 – 11:15 a.m. PT The impact of cardiopulmonary comorbidities on the economic burden and mortality of pulmonary arterial hypertension in the United States. H. Black. Poster Board #P253; A36 Pulmonary hypertension patient experience, health services and outcomes on Sunday, May 18, 12:15 – 7:15 p.m. ET/9:15 a.m. – 4:15 p.m. PT Caregiver network and caregiving needs in women with pulmonary arterial hypertension (PAH). T. Thakur. Poster Board #P254; A36 Pulmonary hypertension patient experience, health services and outcomes on Sunday, May 18, 12:15 – 7:15 p.m. ET/9:15 a.m. – 4:15 p.m. PT Acute inpatient utilization among Medicaid patients treated for pulmonary arterial hypertension. A. Watanabe. Poster Board #P251; A36 Pulmonary hypertension patient experience, health services and outcomes on Sunday, May 18, 12:15 – 7:15 p.m. ET/9:15 a.m. – 4:15 p.m. PT About WINREVAIR ™ (sotatercept-csrk) for injection, for subcutaneous use, 45 mg, 60 mg WINREVAIR is FDA-approved for the treatment of adults with pulmonary arterial hypertension (PAH, WHO Group 1) to increase exercise capacity, improve WHO functional class (FC) and reduce the risk of clinical worsening events. WINREVAIR is the first activin signaling inhibitor therapy approved to treat PAH. WINREVAIR improves the balance between pro-proliferative and anti-proliferative signaling to modulate vascular proliferation. In preclinical models, WINREVAIR induced cellular changes that were associated with thinner vessel walls, partial reversal of right ventricular remodeling, and improved hemodynamics. WINREVAIR is the subject of a licensing agreement with Bristol Myers Squibb. Selected Safety Information for WINREVAIR in the U.S. WINREVAIR may increase hemoglobin (Hgb). Severe erythrocytosis may increase the risk of thromboembolic events or hyperviscosity syndrome. Monitor Hgb before each dose for the first 5 doses, or longer if values are unstable, and periodically thereafter, to determine if dose adjustments are required. WINREVAIR may decrease platelet count. Severe thrombocytopenia may increase the risk of bleeding. Thrombocytopenia occurred more frequently in patients also receiving prostacyclin infusion. Do not initiate treatment if platelet count is <50,000/mm3. Monitor platelets before each dose for the first 5 doses, or longer if values are unstable, and periodically thereafter to determine whether dose adjustments are required. In clinical studies, serious bleeding (e.g., gastrointestinal, intracranial hemorrhage) was reported in 4% of patients taking WINREVAIR and 1% of patients taking placebo. Patients with serious bleeding were more likely to be on prostacyclin background therapy and/or antithrombotic agents, or have low platelet counts. Advise patients about signs and symptoms of blood loss. Do not administer WINREVAIR if the patient is experiencing serious bleeding. WINREVAIR may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use an effective method of contraception during treatment with WINREVAIR and for at least 4 months after the final dose. Pregnancy testing is recommended for females of reproductive potential before starting WINREVAIR treatment. Based on findings in animals, WINREVAIR may impair female and male fertility. Advise patients on the potential effects on fertility. The most common adverse reactions occurring in the phase 3 clinical trial (≥10% for WINREVAIR and at least 5% more than placebo) were headache (24.5% vs 17.5%), epistaxis (22.1% vs 1.9%), rash (20.2% vs 8.1%), telangiectasia (16.6% vs 4.4%), diarrhea (15.3% vs 10.0%), dizziness (14.7% vs 6.2%), and erythema (13.5% vs 3.1%). Because of the potential for serious adverse reactions in the breastfed child, advise patients that breastfeeding is not recommended during treatment with WINREVAIR, and for 4 months after the final dose. About PAH Pulmonary arterial hypertension (PAH) is a rare, progressive and life-threatening blood vessel disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. Approximately 40,000 people in the U.S. are living with PAH. The disease progresses rapidly for many patients. PAH results in significant strain on the heart, leading to limited physical activity, heart failure and reduced life expectancy. The five-year mortality rate for patients with PAH is approximately 43%. About Merck At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on X (formerly Twitter) , Facebook , Instagram , YouTube and LinkedIn . Forward-Looking Statement of Merck & Co., Inc., Rahway, N.J., USA This news release of Merck & Co., Inc., Rahway, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s Annual Report on Form 10-K for the year ended December 31, 2024 and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site ( www.sec.gov ). Please see Prescribing Information for WINREVAIR (sotatercept-csrk) at http://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_pi.pdf , Patient Information for WINREVAIR at http://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_ppi.pdf , and Instructions for Use for WINREVAIR (1-vial kit, 2-vial kit) at https://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_ifu_1-vial_2-vial_kits.pdf . Media Contacts: Julie Cunningham (617) 519-6264 Courtney Ronaldo (908) 442-5695 Investor Contacts: Peter Dannenbaum (732) 594-1579 Steven Graziano (732) 594-1583 Source: Merck & Co., Inc.

点击蓝字关注我们肺动脉高压（PAH）是一种罕见、进展快速且最终危及生命的进展性心血管疾病，其特征是肺血管重构，导致肺动脉管腔进行性闭塞，肺血管阻力不断升高，进而导致心力衰竭甚至死亡。       关于肺动脉高压       根据柳叶刀杂志报道，全球患肺动脉高压的人数占世界人口总量的1％，常见于30至60岁的女性，约五分之一的患者有家族病史。PAH 的诊断常被延误，因为其症状如：呼吸短促、疲劳、脚和腿肿胀、头晕或昏厥、胸痛、心悸等与常见的心脏和肺部疾病的症状相似。从病理学机制上看，肺动脉高压是多个信号通路及环境因素、基因因素共同作用的结果。绿色箭头=通路激活剂，红色箭头=通路抑制剂，蓝色方框=通路，红色方框=通路抑制剂、绿色方框=通路激活物/调节剂。  肺动脉高压现有治疗药物  在缺乏靶向药物的传统治疗时代， PAH患者预后差，存活时间短，5年生存率仅有20%左右。自靶向药物广泛用于临床后，PAH患者生存率显著提高，5年生存率达到近60%。现阶段肺动脉高压的治疗方式仍以靶向药为主，目前PAH的靶向药物治疗主要针对三大致病途径调节血管收缩或舒张，包括内皮素通路、一氧化氮通路、前列环素通路。内皮素通路:内皮素受体拮抗剂(ERA)一氧化氮通路:磷酸二酯酶抑制剂(PDE5i)、可溶性鸟苷酸环化酶(sGC) 激动剂前列环素通路:前列环素及其类似物、前列环素IP受体激动剂目前国内已上市的PAH靶向药有波生坦、安立生坦、马昔腾坦；西地那非、他达拉非、利奥西呱；吸入伊洛前列素、皮下和静脉注射曲前列尼尔、司来帕格等。  临床治疗亟需更有效药物  既然现有的靶向药已经能够改善病症，为什么患者死亡率并没有显著改善？为什么新靶点药物仍受到研究人员高度关注？肺动脉高压的特征是肺血管重构，肺动脉管腔狭窄闭塞。血流通过肺血管时的阻力增大，导致缺氧和右心功能衰竭。现有的PAH靶向药如波生坦、西地那非、利奥西呱等，通过各种途径促进肺血管扩张，让血流通过顺畅一些，缓解患者症状，使患者能维持较好的生活质量，但未从源头上抑制疾病进展，即所谓“治标不治本”。肺动脉高压的治疗亟需打破过去单纯肺血管扩张模式，聚焦逆转肺血管重构的高效的全新一代治疗药物。2024年3月26日，美国食品药品管理局（FDA）批准由Acceleron Pharma和MERCK联合开发的sotatercept（商品名：Winrevair）用于治疗成年人的肺动脉高压，以增加运动能力，改善WHO功能分级并降低临床恶化事件的风险。该药物每瓶定价14000美元，全年的费用约为24.2万美元，约175万人民币。摩根大通股票分析师 Chris Schott预测到2030年，销售额可接近50亿美元。与传统药物不同，Sotatercept是一种激活素信号抑制剂，是First-in-class激活素受体IIA-Fc型（ActRIIA Fc）融合蛋白，可选择性结合TGF-β家族配体，恢复肺动脉壁和右心室重构相关的促增殖和抗增殖信号通路之间的平衡，机制上具有抑制平滑肌细胞增殖、逆转肺动脉壁和右心室重构潜力，但仍需更多直接的临床证据证实。广州喜鹊医药有限公司独家开发的创新1类化学药物MN-08，既能拮抗NMDA受体、改善患者症状，又能在体内释放一氧化氮，扩张血管增加血流。在临床前药效学实验中显示出良好的药效，在肺和脑组织中具有很高的药物浓度分布，可以扩张肺动脉和脑血管。在肺动脉高压的病理状态下，NMDA受体过度激活导致谷氨酸超载、肺血管收缩、增生和纤维化。MN-08可借助特殊的分子结构参与到这一过程中，从而抑制谷氨酸超载。同时，MN-08分子中的一氧化氮供体基团可释放一氧化氮，有助于舒张肺血管、降低肺动脉压，改善肺血流循环、抑制肺血管重构和纤维化。MN-08治疗肺动脉高压中国临床 1 期及 2 期第一阶段单次给药开放试验结果已显示明确的肺动脉压力下降及增加心输出量的效果，目前正在开展中国临床2期第二阶段研究。本文部分内容来源于：梅斯心血管新前沿。内容仅供参考，不构成任何投资及应用建议，亦不代表任何治疗指导及用药建议。如稿件涉及版权等问题，请与我司联系，我们将在第一时间协调给予处理或删除。