Core Insights - Blueprint Medicines Corporation has showcased its leadership in advancing care for patients with systemic mastocytosis (SM) through data presentations at major congresses, emphasizing the effectiveness of AYVAKIT®/AYVAKYT® (avapritinib) as a standard treatment for both indolent and advanced forms of SM [1][2][9]. Data Presentation Highlights - The presentations included large patient populations from the PIONEER, PATHFINDER, and EXPLORER trials, demonstrating long-term benefits of AYVAKIT with follow-up periods of up to five years for indolent systemic mastocytosis (ISM) and 6.5 years for advanced SM [2][5]. - AYVAKIT has shown transformative clinical outcomes, including sustained disease control in ISM and prolonged survival in advanced SM, leading to an expanded view among clinicians regarding appropriate candidates for disease-modifying therapy [2][7]. Clinical Efficacy and Safety - AYVAKIT demonstrated robust improvements in overall symptoms and quality of life measures over 144 weeks, with a low treatment-related adverse event discontinuation rate of 3% and common adverse events including low-grade edemas, headache, and nausea [7][23]. - The Revised Mutation-Adjusted Risk Score (MARS-R) tool was validated for assessing overall survival risks in advanced SM patients treated with AYVAKIT, showing meaningful survival benefits across all prognostic categories [7][12]. Disease Burden and Patient Impact - The PRISM study highlighted the substantial disease burden faced by patients with ISM, including physical, social, and emotional challenges that significantly disrupt daily life [6][12]. - Patients reported various disease-related impacts, such as limitations in physical activities and work, as well as issues with pain, anxiety, and adjustments in daily routines to avoid triggers [8][12]. Regulatory and Market Position - AYVAKIT is the first and only FDA-approved treatment targeting the root cause of SM, with approvals for both advanced SM and ISM, and is marketed in 16 countries globally [14][26]. - The company aims to expand its impact by advancing a broad pipeline of programs in mast cell diseases and solid tumors, leveraging its established research and commercial capabilities [26].

撰文丨王聪 编辑丨王多鱼 排版丨水成文 2025 年 6 月 2 日，法国制药巨头 赛诺菲 （Sanofi） 宣布收购美国生物制药上市公司 Blueprint Medicines ， 以扩大自身在罕见免疫疾病领域的产品组合，并增添免疫学领域的早期研发管线， 交易总金 额高达 95 亿美元 。 根据收购条款，赛诺菲将在交易完成时以每股 129.00 美元的现金支付，股权价值约为 91 亿 美元，此 外，Blueprint 股东还将获得一份不可交易的或有价值权 （CVR） ，该权利持有人将有权在 BLU-808 未 来达到研发里程碑和监管里程碑时分别获得额外里程碑付款。因此，此次收购的总股权价值在完全稀释的 基础上约为 95 亿 美元。该收购预计在 2025 年第三季度完成。 此次收购对于赛诺菲的战略意义在于： 1、强化免疫学布局 ： 2、商业化协同 ： Blueprint 在过敏、皮肤和免疫领域的渠道资源，可加速赛诺菲免疫学产品落地。 3、财务影响 ： 赛诺菲将获得 Blueprint 在 系统性肥大细胞增多症 （S ystemic Mastocytosis ） 领域的独家药物 Ayvakit/Ayvakyt ...

法国制药巨头赛诺菲(SNY.US)将以91亿美元股权价值收购美国生物制药公司Blueprint Medicines Corp. (BPMC.US)，此举将显著增强其在罕见免疫疾病领域的治疗管线。 Blueprint目前主营治疗系统性肥大细胞增多症的靶向药物(美国商品名Ayvakit/欧洲商品名Ayvakyt)，该 罕见病会导致免疫细胞异常积聚引发荨麻疹、腹痛等症状。据公司预测，该药物2025年销售额预计达7- 7.2亿美元。 该交易彰显了赛诺菲打造免疫学领域领导地位的雄心。今年早些时候，该公司已宣布拟以最高19亿美元 收购Dren Bio,Inc.的自身免疫疾病药物。 赛诺菲首席执行官Paul Hudson表示："此次收购是我们罕见病和免疫学产品组合的战略性突破，不仅丰 富了我们的研发管线，更将加速我们转型为全球领先免疫学企业的进程。" Hudson补充道："赛诺菲仍保持可观的并购能力。" 赛诺菲指出，Blueprint还拥有包括多款在研免疫药物在内的丰富产品管线，并在专科医生群体中建立了 稳固的临床网络。 根据双方周一发布的联合声明，赛诺菲将以每股129美元的现金价格进行收购，较Blueprint上周五收盘 ...

  Sanofi to acquire Blueprint Medicines, expanding portfolio in rare immunological disease and adding early-stage pipeline in immunology Adds fast-growing and only approved medicine for advanced and indolent systemic mastocytosis to the Sanofi portfolio Paris and Cambridge, MA. June 2, 2025. Sanofi and Blueprint Medicines Corporation (Blueprint), a US-based, publicly traded biopharmaceutical company specializing in systemic mastocytosis (SM), a rare immunological disease, and other KIT-driven diseases, have ...

Blueprint Medicines Driving growth and innovation with operational excellence First quarter 2025 financial and operating results INTRODUCTION AYVAKIT PERFORMANCE Kate Haviland Chief Executive Officer Philina Lee, PhD Chief Commercial Officer PROGRESS May 1, 2025 First quarter 2025 financial and operating results Becker Hewes, MD Chief Medical Officer FINANCIAL RESULTS Mike Landsittel Chief Financial Officer 2 CLINICAL Forward-looking statements This presentation contains forward-looking statements within th ...

Company Overview - Blueprint Medicines Corporation is a global, fully integrated biopharmaceutical company focused on inventing life-changing medicines [3] - The company aims to alleviate human suffering by addressing important medical problems in two core areas: allergy/inflammation and oncology/hematology [3] - Blueprint Medicines has a track record of success with two approved medicines, including AYVAKIT®/AYVAKYT® (avapritinib) for systemic mastocytosis (SM) in the U.S. and Europe [3] Upcoming Events - The company will host a live conference call and webcast on May 1, 2025, at 8:00 a.m. ET to report its first quarter 2025 financial results and provide a corporate update [1] - Access to the live conference call can be obtained by dialing 833-470-1428 (domestic) or 404-975-4839 (international) with conference ID 082088 [2] - An archived webcast will be available on the company's website approximately two hours after the call and will remain accessible for 30 days [2] Research and Development Focus - Blueprint Medicines is advancing a broad pipeline of programs ranging from early science to advanced clinical trials in mast cell diseases, including SM and chronic urticaria, as well as breast cancer and other solid tumors [3]