The purposes of this Phase 1 study of sevasemten are to:
1. Evaluate the effect of multiple-dose administration of verapamil on the single-dose of sevasemten in healthy adults
2. Evaluate the safety and tolerability of a single dose of sevasemten administered with and without verapamil in healthy adult subjects.
3. Evaluate the safety and tolerability of a single dose of sevasemten administered with and without food in healthy adult subjects.

开始日期2025-01-08

申办/合作机构

Edgewise Therapeutics, Inc.

NCT06100887

/ Active, not recruiting临床2期

A Phase 2 Study to Evaluate the Effect of EDG-5506 on Safety, Pharmacokinetics, and Biomarkers in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

The FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized, double-blind, placebo-controlled Part A, followed by an open-label part B.

开始日期2024-03-22

申办/合作机构

Edgewise Therapeutics, Inc.

NCT06066580

/ Enrolling by invitation临床2期

An Open-Label Extension Study to Assess the Long-term Effect of EDG-5506 on Safety, Biomarkers, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy

EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy

开始日期2023-11-02

申办/合作机构

Edgewise Therapeutics, Inc.

[+1]

100 项与 Sevasemten 相关的临床结果

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100 项与 Sevasemten 相关的转化医学

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100 项与 Sevasemten 相关的专利（医药）

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项与 Sevasemten 相关的文献（医药）

2026-07-01·EBioMedicine

Effects of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults with Becker muscular dystrophy: results of a phase 1b, open-label study

Article

作者: Thaler, Elizabeth ; Massaro, Monica ; Kilburn, Nicole Rempel ; Silverman, Jeffrey A ; Amato, Maria ; MacDougall, James ; Bronson, Abby ; Barthel, Ben ; Russell, Alan J ; Madden, Molly ; Donovan, Joanne ; Phan, Han

BACKGROUND:

Sevasemten (EDG-5506) is an orally administered, investigational small molecule that selectively modulates fast muscle fibre contraction by inhibiting fast myosin ATPase. In animal models of Duchenne and Becker muscular dystrophy (DMD and BMD, respectively), sevasemten reduced the muscle contraction injury that leads to inflammation, fibrosis and muscle loss without affecting function. The aim of this study was to evaluate the long-term safety, tolerability, and pharmacokinetics (PK)/pharmacodynamics of sevasemten in adult participants with BMD who had already experienced a decline in function and would be anticipated to continue to decline based on natural history.

METHODS:

This open-label, dose escalation, phase 1 b study (NCT05160415) was conducted at a single site and enrolled ambulatory adults with BMD aged 18-55 years; the study is completed. Eligible participants received 10 mg of sevasemten once daily (QD) for 8 weeks, followed by 15 mg QD for 4 months, 20 mg QD for 9 months, and 10 mg QD for 9 months. The primary objective was to assess the safety and tolerability of sevasemten in adults with BMD; endpoints included adverse events (AEs), PK, change from baseline in circulating biomarkers of muscle injury, as well as physical function measures.

FINDINGS:

The study enrolled 12 adults with BMD. Sevasemten was well tolerated; all AEs were mild or moderate in severity and there were no serious AEs or AEs leading to discontinuation. Treatment with sevasemten was associated with reductions in circulating biomarkers of muscle injury that were evident within 4 weeks and sustained for up to 24 months. Physical function, as assessed by North Star Ambulatory Assessment (NSAA), was stable over 24 months.

INTERPRETATION:

Sevasemten treatment for up to 24 months in adults with BMD was well tolerated and associated with durable reductions in muscle injury biomarkers, consistent with preclinical studies and near maximal with the 10 mg dose, as well as functional stabilisation. Further clinical development is ongoing.

FUNDING:

Edgewise Therapeutics, Inc.

2026-07-01·JOURNAL OF CLINICAL PHARMACOLOGY

Human Disposition, Metabolism, and Excretion of Sevasemten (EDG‐5506), a Selective Modulator of Fast Myosin in Healthy Volunteers

Article

作者: Donovan, Joanne ; Evanchik, Marc ; Lane, Jonathan ; Silverman, Jeffrey A. ; Russell, Alan J. ; Madden, Molly

Abstract:

Sevasemten (EDG‐5506) is a novel, orally bioavailable, investigational small molecule designed to selectively modulate type II fast skeletal myosin with the goal of protecting dystrophic muscle from contraction‐induced injury. Sevasemten is in clinical development for the treatment of Becker and Duchenne muscular dystrophy. In the current Phase 1 study (NCT05730842), eight participants were dosed with a 20 mg single oral suspension dose of sevasemten containing approximately 500 µCi [ 14 C] sevasemten. Blood and urine samples were collected to evaluate the pharmacokinetics (PK), oral absorption, metabolism, and excretion (AME) of sevasemten and its metabolites. A second cohort of seven participants received a single 20 mg oral dose of sevasemten, followed 2 h later by a single dose of [ 14 C] sevasemten administered as a single intravenous (IV) bolus with approximately 1 µCi [ 14 C] sevasemten to determine absolute bioavailability. Sevasemten was excreted primarily in urine (52.1%) subsequent to CYP3A‐mediated metabolism, and 8.89% was eliminated in the feces, for a total of 61.0% recovered over 840 h post‐dose. Most of the recoverable radioactivity was excreted by 576 h (24 days). However, sevasemten total radioactivity in plasma and whole blood was characterized by a very slow elimination phase of 16.1–20.6 days; total recovery of radioactivity was not achieved. Sevasemten was well tolerated, with no serious adverse events or adverse events leading to study discontinuation. Sevasemten has favorable PK properties for once daily administration and continues to be investigated for the treatment of Becker and Duchenne muscular dystrophies.

2023-05-15·The Journal of clinical investigation

Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy

Article

作者: Peter, Angela K ; Qian, Ying ; Sweeney, H Lee ; Patsalos, Andreas ; Newell-Stamper, Breanne L ; Rodriguez, Hector M ; Brooks, Susan V ; Vera, Carlos ; Nagy, Laszlo ; Juehne, Twlya I ; Lehman, Sarah ; Madden, Molly ; Rutledge, Alexis ; Hunt, Kevin ; Barthel, Ben ; Yu, Jinsheng ; Schlachter, Stephen ; Van Deusen, Ashleigh ; Leinwand, Leslie A ; Russell, Alan J ; Nghiem, Peter ; Su, Yu ; Luo, Yangyi E ; Claflin, Dennis R ; DuVall, Mike ; Barton, Elisabeth R ; Koch, Kevin ; Robertson, Ben

Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by absence of the protein dystrophin, which acts as a structural link between the basal lamina and contractile machinery to stabilize muscle membranes in response to mechanical stress. In DMD, mechanical stress leads to exaggerated membrane injury and fiber breakdown, with fast fibers being the most susceptible to damage. A major contributor to this injury is muscle contraction, controlled by the motor protein myosin. However, how muscle contraction and fast muscle fiber damage contribute to the pathophysiology of DMD has not been well characterized. We explored the role of fast skeletal muscle contraction in DMD with a potentially novel, selective, orally active inhibitor of fast skeletal muscle myosin, EDG-5506. Surprisingly, even modest decreases of contraction (<15%) were sufficient to protect skeletal muscles in dystrophic mdx mice from stress injury. Longer-term treatment also decreased muscle fibrosis in key disease-implicated tissues. Importantly, therapeutic levels of myosin inhibition with EDG-5506 did not detrimentally affect strength or coordination. Finally, in dystrophic dogs, EDG-5506 reversibly reduced circulating muscle injury biomarkers and increased habitual activity. This unexpected biology may represent an important alternative treatment strategy for Duchenne and related myopathies.

138

项与 Sevasemten 相关的新闻（医药）

2026-08-06

·PRNewswire

Edgewise Therapeutics Reports Second Quarter 2026 Financial Results

– Announced positive top-line data from 12-week Phase 2 CIRRUS-HCM trial with EDG-7500 in obstructive and nonobstructive hypertrophic cardiomyopathy (HCM) – – Completed sale of sevasemten and muscular dystrophy business to Servier for up to $2.65 billion in July 2026, strengthening the balance sheet, sharpening focus on the cardiovascular pipeline – BOULDER, Colo., Aug. 6, 2026 /PRNewswire/ -- Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading clinical-stage biopharmaceutical company focused on cardiovascular diseases, today reported financial results for the second quarter of 2026 and recent business highlights . "In the second quarter, Edgewise entered into a deal to sell sevasemten and our muscular dystrophy business to Servier. Our post-deal close financial position enhances our ability to advance EDG-7500 and our cardiovascular-focused pipeline toward important clinical and regulatory milestones as an independent company," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise Therapeutics. "With positive Phase 2 CIRRUS-HCM results for EDG-7500 and continued progress with EDG-15400, we are well positioned to advance a differentiated pipeline for patients with serious cardiovascular disease." Recent Highlights Sevasemten and Muscular Dystrophy Program Sale In July 2026, Edgewise completed the previously announced transaction under which Servier, an independent international pharmaceutical group governed by a foundation, acquired sevasemten and Edgewise's muscular dystrophy business for $1.55 billion in upfront cash consideration and up to $1.1 billion in additional milestone payments, for aggregate potential consideration of up to $2.65 billion. Servier brings the global development, regulatory, and commercial capabilities required to fully realize sevasemten's potential for patients. The transaction strengthens Edgewise's balance sheet, enhances financial flexibility and sharpens the company's strategic focus on advancing its cardiovascular pipeline. This transaction marks Edgewise's transition to a cardiovascular-focused company, with a pipeline comprising EDG-7500 for hypertrophic cardiomyopathy, EDG-15400 for heart failure with preserved ejection fraction (HFpEF) and EDG-003 for an undisclosed target. Cardiovascular Programs CIRRUS-HCM Phase 2 trial in adults with symptomatic HCM: The company announced positive top-line results from the 12-week Phase 2 Part D CIRRUS-HCM open label trial of EDG-7500 in obstructive (oHCM) and nonobstructive (nHCM) HCM, which was designed to inform a Phase 3 trial. Results from the study are available in the company's June 2026 press release, which can be found here. The company expects to initiate a Phase 3 trial in the fourth quarter of 2026. To learn more about CIRRUS-HCM, visit ClinicalTrials.gov, NCT06347159. EDG-15400 and heart failure: EDG-15400 is a novel, oral, selective cardiac sarcomere modulator being developed for the treatment of heart failure. EDG-15400 has completed dosing in a Phase 1 randomized, double-blind, placebo-controlled, single and multiple ascending dose study in healthy adults evaluating safety, tolerability, pharmacokinetics and pharmacodynamics. The company expects to initiate a Phase 2 trial in participants with heart failure with preserved ejection fraction (HFpEF) in the second half of 2026. To learn more about this study, go to ClinicalTrials.gov (NCT07177066). Second Quarter 2026 Financial Results Cash, cash equivalents and marketable securities were approximately $460.7 million as of June 30, 2026. Our cash and cash equivalents at June 30, 2026 do not include the $1,550.0 million in upfront cash proceeds for the sale of sevasemten on July 10, 2026. Combining the actual balance at June 30, 2026 with the upfront cash proceeds of the sale provides for a pro forma balance of $2,010.7 million before taxes and transaction related costs. Research and development (R&D) expenses were $47.5 million for the second quarter of 2026, compared to $33.6 million for the same period in 2025. The increase was primarily driven by increased clinical development activity related to EDG‑7500, EDG‑15400 and patient rollover activity in the MESA open-label extension study, as well as higher personnel‑related costs to support the advancement of the Company's clinical‑stage programs. General and Administrative (G&A) expenses were $14.4 million for the second quarter of 2026, compared to $9.1 million for the same period in 2025. The increase was primarily due to higher personnel‑related costs, including stock‑based compensation, as the Company expanded its organizational capabilities to support clinical development, as well as increased professional fees and other administrative costs. Net loss and net loss per share were $57.3 million or $0.53 per share for the second quarter of 2026, compared to $36.1 million or $0.34 per share for the same period in 2025. About Edgewise Therapeutics Edgewise Therapeutics is a leading biopharmaceutical company focused on novel, muscle-targeted therapeutics. Leveraging its expertise in muscle biology and small molecule drug development, Edgewise is advancing a cardiovascular pipeline targeting significant unmet needs, including hypertrophic cardiomyopathy, heart failure and other serious cardiovascular and cardiometabolic conditions. To learn more, go to edgewisetx.com or follow Edgewise on LinkedIn, X, Facebook and Instagram. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things, statements regarding the potential of, and expectations regarding, Edgewise's product candidates and programs, including EDG-7500, EDG-15400 and its cardiovascular programs; statements regarding Edgewise's expectations relating to its clinical trials; statements regarding timing of Edgewise's initiation of a Phase 3 trial of EDG-7500 in HCM and a Phase 2 trial of EDG-15400 in participants with HFpEF; statements regarding Edgewise's ability to advance a differentiated pipeline for patients with serious cardiovascular disease; statements regarding the sale of sevasemten and muscular dystrophy business to Servier (Transaction), including potential payments which may become owing to Edgewise; and statements by Edgewise's President and Chief Executive Officer. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. The forward-looking statements contained herein are based upon Edgewise's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those projected in any forward-looking statements due to numerous risks and uncertainties, including but not limited to: risks associated with Edgewise's limited operating history, its products being early in development and not having products approved for commercial sale; risks associated with Edgewise not having generated any revenue to date; Edgewise's ability to achieve objectives relating to the discovery, development and commercialization of its product candidates, if approved; Edgewise's potential need for substantial additional capital to finance its operations; Edgewise's substantial dependence on the success of EDG-7500; Edgewise's ability to develop and commercialize EDG-7500 and EDG-15400, and discover, develop and commercialize product candidates in its cardiovascular, cardiometabolic and future programs; risks related to Edgewise's clinical trials of its product candidates not demonstrating safety and efficacy; risks related to Edgewise's product candidates causing serious adverse events, toxicities or other undesirable side effects; the outcome of preclinical testing and early clinical trials not being predictive of the success of later clinical trials and the risks related to the results of Edgewise's clinical trials not satisfying the requirements of regulatory authorities; delays or difficulties in the enrollment and/or maintenance of patients in clinical trials; risks related to failure to capitalize on other indications or product candidates; risks related to competition; risks relating to interim, top-line and preliminary data from Edgewise's clinical trials changing as more patient data becomes available; risks related to failure to develop a proprietary drug discovery platform; risks related to production of drugs by Edgewise's third-party manufacturers; risks related to changes in methods of product candidate manufacturing or formulation; risks related to not achieving adequate market acceptance; risks related to the regulatory approval processes of domestic and foreign authorities being lengthy, time consuming and inherently unpredictable; risks relating to disruptions at the FDA, the SEC and other government agencies; risks relating to Edgewise's ability to attract and retain highly skilled executive officers and employees; Edgewise's ability to obtain and maintain intellectual property protection for its product candidates; Edgewise's reliance on third parties; risks related to future acquisitions or strategic partnerships; risks related to Edgewise's future operations following the sale of sevasemten; risks relating to Edgewise not receiving milestone payments related to the Transaction; risks related to general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled "Risk Factors" in documents that Edgewise files from time to time with the U.S. Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Edgewise assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law . This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release. SOURCE Edgewise Therapeutics 21% more press release views with Request a Demo

临床结果并购临床1期财报临床2期

2026-08-05

·上药睿尔

7月速览（三）罕见病药研发之投资并购

罕见病药研发之投资并购 ☞ 施维雅26.5亿美元收购Edgewise Therapeutics肌营养不良业务板块 ☞ Celea完成1.8亿美元融资 ☞ 福泰制药100亿美元并购Crinetics Pharmaceuticals ☞ Jasper Therapeutics与Kira Pharmaceuticals合并 01 施维雅26.5亿美元收购Edgewise Therapeutics肌营养不良业务板块 7月13日，施维雅宣布已完成对Edgewise Therapeutics肌营养不良业务板块的收购，交易总额最高可达26.5亿美元，其中包括15.5亿美元预付款，另设上限11亿美元的监管及商业化里程碑付款。收购完成后，施维雅将获得一款治疗肌营养不良的在研药物Sevasemten，该药物正在开展贝克型肌营养不良（BMD）关键队列临床试验，同时推进杜氏肌营养不良（DMD）的II期临床研究。根据交易条款约定，施维雅将获得Sevasemten的全球独家开发与商业化权益，同时受让Edgewise全部肌营养不良相关知识产权、专有技术、临床数据、监管申报材料及核心商业合作协议。原负责肌营养不良管线的Edgewise核心研发团队已并入施维雅，确保药物临床开发与全球商业化工作平稳过渡。 Sevasemten是一款全球首创的口服快速骨骼肌球蛋白抑制剂，可选择性地调节快缩肌纤维收缩，从而减轻肌营养不良症中的肌肉损伤，旨在选择性抑制因功能性抗肌萎缩蛋白缺乏或丧失所引发的过度肌肉损伤。 02 Celea完成1.8亿美元融资 7月2日，Celea Therapeutics宣布已完成1.8亿美元融资，所融资金将主要用于支持一款新一代抗纤维化药物LYT-100（氘代吡非尼酮）的SURPASS-IPF III期临床试验，为特发性肺纤维化（IPF）患者提供全新的标准治疗方案。 SURPASS-IPF是一项全球性、随机、双盲、头对头对照研究，针对未接受过背景治疗的成人IPF患者，直接对比氘代吡非尼酮与传统吡非尼酮的临床疗效。其中，氘代吡非尼酮给药剂量为825mg/次、每日三次，对照组吡非尼酮给药剂量为801mg/次、每日三次。研究以治疗第52周用力肺活量绝对值较基线的变化为主要疗效终点，旨在验证氘代吡非尼酮相较于传统吡非尼酮的临床优效性。氘代吡非尼酮是吡非尼酮的氘代衍生物，在保持良好耐受性的同时有望实现更高的药物暴露水平，目前已获得FDA及欧盟委员会的孤儿药资格认定。 03 福泰制药100亿美元并购Crinetics Pharmaceuticals 7月6日，福泰制药（Vertex Pharmaceuticals）宣布将以总价约100亿美元现金收购专注于开发内分泌疾病疗法的Crinetics Pharmaceuticals。交易完成后，福泰制药将获得Crinetics的两款核心药物——已上市的PALSONIFY®（帕妥索汀，paltusotine）和III期临床的Atumelnant。帕妥索汀是一款高选择性生长抑素受体2型（SST2）激动剂，已获FDA批准用于成人肢端肥大症的一线治疗，适用于手术应答不佳或不适合手术的患者。其对SST2的选择性超4000倍，通过抑制生长激素（GH）和胰岛素样生长因子1（IGF-1）分泌起效。 Atumelnant是一种每日口服一次的促肾上腺皮质激素（ACTH）受体拮抗剂，直接阻断肾上腺皮质对ACTH的应答，理论上可同时降低皮质醇和肾上腺雄激素。目前正在推进针对先天性肾上腺增生症（CAH）的III期临床试验。 04 Jasper Therapeutics与Kira Pharmaceuticals合并 7月16日，Jasper Therapeutics宣布已完成对专注于开发免疫介导疾病补体疗法的Kira Pharmaceuticals（科越医药）的全股票收购，募资总额约1.32亿美元，所募资金将用于支持合并后公司管线开发。合并后，公司的管线矩阵将重点推进以下创新疗法： KP-104（Vensobafusp alfa）：一款双功能生物制剂，用于治疗阵发性睡眠性血红蛋白尿症（PNH）及罕见肾病。公司计划于2026年第四季度公布罕见肾病2期篮式试验第一阶段的中期数据，并计划于2027年上半年公布PNH临床2期结束会议的后续计划。 Briquilimab：一款抗KIT抗体，具有广泛的移植及免疫适应症治疗潜力。基于在重症联合免疫缺陷病（SCID）中的积极数据，公司正推进与FDA的BLA前会议，预计2027年第一季度公布后续计划。 KP-701：一款新型双重作用抗CD79BxCD32B单克隆抗体，用于治疗自身抗体介导性疾病。预计将于2027年第一季度提交IND申请，并于同年第三季度公布首次人体试验数据。在推进核心管线的同时，合并后公司也进行了资产优化。Kira已将其长效抗C5a单克隆抗体KP-301及小分子C5a受体拮抗剂KP-402对外授权给Mirador Therapeutics，获得1200万美元首付款及潜在里程碑付款。

2026-07-22

·会会药咖

2026年6月全球医药BD交易领域分析

2026年6月，全球医药BD市场进入明确的"并购超级周期"。据STAT News统计，2026年上半年全球医药/生物科技并购总额已达1340亿美元，超过2025年全年（1120亿美元），其中超十亿美元交易达33笔，创下2019年以来最高纪录。6月单月即落地三笔百亿美元级交易：艾伯维109亿美元收购Apogee、GSK 106亿美元收购Nuvalent、默克KGaA 113亿美元收购Bio-Techne，驱动逻辑直指总额约3000亿美元的专利悬崖。与此同时，中国创新药出海延续爆发：上半年License-out总金额约976至997亿美元（医药魔方NextPharma口径），首付款总额约63至64.5亿美元、同比增长约124%，交易金额占全球比重约59%。关键验证数据：公开披露的重点交易超30笔，覆盖肿瘤（精准小分子/DAC/MDM2）、免疫炎症（IL-13/TL1A）、罕见病（肌营养不良/出血性疾病/移植感染）、诱导邻近平台（降解剂/分子胶）及AI制药等核心领域百亿美元级交易3笔：艾伯维/Apogee（109亿美元）、默克KGaA/Bio-Techne（113亿美元）、GSK/Nuvalent（106亿美元，GSK历史上最大收购）超十亿美元交易合计达8笔以上，含Servier/Edgewise（26.5亿美元）、Incyte/Vega（最高20亿美元）、Ipsen/Kartos（最高17.5亿美元）、Biogen/RayThera（最高10亿美元）、强生/Firefly Bio（10亿美元）中国资产6月密集成交：云顶新耀EVER001授权Travere（首付1.125亿美元、总额超11亿美元）、TrueLab双抗体授权法国NewCo Bionyra（最高9.85亿美元+股权）、和誉医药与礼来多靶点发现合作（最高约19亿美元） "临批准资产"定价权确立：Nuvalent两款肺癌药PDUFA日期分别为2026年9月18日与11月27日，GSK在FDA裁决前以40%溢价锁定，Truist预计两药合计峰值销售35亿美元一、肿瘤学临批准精准小分子与下一代偶联技术战略定位：肿瘤领域呈现"批准前夜抢购"与"下一代偶联 modality 抢位"并行的格局。MNC不再等待资产获批或上市放量，而是在PDUFA日期前、Phase 3数据读出前直接出手，以确定性溢价换取时间差收益；同时，降解剂-抗体偶联物（DAC）作为ADC的迭代方向首次获得十亿美元级收购验证。 2.战略洞察： GSK以106亿美元买下"两个FDA裁决日期"：Zidesamtinib（PDUFA 2026年9月18日）与Neladalkib（PDUFA 11月27日）均获突破性疗法与孤儿药资格，若获批将于2026年内上市、自2027年起贡献收入，直接对冲多替拉韦2028至2030年专利到期风险。该交易为GSK历史上最大收购，也标志其正式重返肺癌市场，并与Phase III阶段B7-H3 ADC（risvutatug rezetecan）形成肺癌组合平台。交易于7月15日完成交割。强生10亿美元收购Firefly Bio，为DAC modality 的首个十亿美元级并购验证：Firelink平台将靶向蛋白降解剂（而非传统化疗毒素）作为载荷递送至肿瘤细胞，主攻KRAS驱动实体瘤。叠加罗氏同季度与C4 Therapeutics超10亿美元的DAC研发合作，降解剂偶联已从概念走向MNC军备竞赛。 Ipsen以最高17.5亿美元收购Kartos，卡位JAK抑制剂耐药后的骨髓纤维化二线市场：Navtemadlin（MDM2抑制剂）Phase III POIESIS研究预计2027年读出顶线数据，是Ipsen一周内第二笔收购（另一笔为Memo Therapeutics），体现法国中型药企以肿瘤+罕见病双轮驱动的激进扩张。二、免疫与炎症（I&I） 2型炎症长效抗体成最大标的 1.战略定位： I&I领域由6月最大单笔交易定义：艾伯维109亿美元收购Apogee，核心逻辑是以半衰期延长（HLE）技术挑战Dupixent（2025年全球销售约178亿美元）主导的2型炎症市场。半衰期延长带来的低给药频次成为下一代自免抗体的核心差异化要素，口服小分子自免药亦同步获得十亿美元级定价。 2.战略洞察：艾伯维109亿美元收购Apogee为当月最大交易，直指Dupixent继任者之争：Zumilokibart为半衰期延长抗IL-13单抗，Phase 2 APEX研究16周约三分之二患者实现显著皮损清除，有望实现季度甚至半年一次皮下给药，Phase 3已于特应性皮炎启动。交易结构值得注意：Apogee在5月刚获得黑石生命科学13亿美元战略融资（含最高8亿美元合成特许权交易），收购触发控制权变更条款，艾伯维实际净对价约101亿美元，预计2032年起增厚调整后EPS。 Biogen一个月内第二笔免疫交易（继3月56亿美元收购Apellis后），以最高10亿美元收购RayThera，押注口服小分子自免药，先导项目预计2026年三季度进入Phase I。自免领域"大分子收购+小分子补充"的组合式建仓成为Biogen转型主线。 TL1A靶点竞争白热化并首次出现中国资产NewCo化退出路径：TrueLab将TL-001（抗TL1A）与TL-003（TL1A×IL-23p19双抗）大中华区外权益授权给法国NewCo Bionyra，后者同步完成1.65亿美元A轮（Jeito与Sofinnova共同领投，赛诺菲风投参投）。TL-003为全球同靶点双抗中进度前三的分子。至此TL1A赛道已形成默沙东（tulisokibart）、罗氏（afimkibart）、赛诺菲/Teva（duvakitug）与中国授权资产四方竞逐格局。三、罕见病、血液与神经肌肉临床后期专科资产密集换手 1.战略定位：罕见病与专科药成为中型药企并购主战场：Servier、Incyte、Ipsen、Zymeworks集中出手，共性标的是"后期临床+监管绿色通道+无同机制竞品"的专科资产，买方以确定性现金流和差异化管线深度对冲单一治疗领域依赖。 2.战略洞察： Servier以最高26.5亿美元收购Edgewise肌营养不良业务，为其3月25亿美元收购Day One后的年内第二笔大型交易，"Servier 2030"罕见神经战略持续加码。Sevasemten若获批将成为首个Becker肌营养不良适应症药物（美欧日患者约1.2万人），BMD关键队列GRAND CANYON已完成175例入组、检验效能超98%，顶线数据预计2026年四季度读出。交易于7月13日完成交割，Edgewise保留心血管管线（EDG-7500等）并以15.5亿美元首付款支撑其至潜在获批。 Incyte以最高20亿美元收购Star Therapeutics子公司Vega，获得全球进度最快的VWD预防性抗体：VGA039通过调节Protein S双重促进血小板黏附与纤维蛋白沉积，有望成为全球首个全类型VWD皮下预防疗法（每月一次给药），对照现有每周2至3次静脉凝血因子输注是量级性改善。该资产拥有突破性疗法、快速通道、孤儿药、罕见儿科疾病四项FDA资格，Phase III VIVID-6进行中。交易于7月6日完成。 Zymeworks以9.29亿美元收购Theravance，标志ADC平台公司向"商业化现金流"转型：Yupelri为已上市每日一次雾化COPD药物，交易同时带来合作分成权益，为Zymeworks的肿瘤管线研发投入提供自我造血能力，代表2026年Biotech"平台+商业资产"混合型并购的兴起。四、中国创新药出海从双抗ADC扩散至小分子与IBD 1.战略定位： 6月中国License-out的结构性变化在于modality扩散：继PD-1/VEGF双抗与ADC之后，小分子BTK抑制剂、TL1A单抗/双抗、小分子发现平台成为新的出海主力。据医药魔方统计，2026年上半年中国License-out交易总金额约976至997亿美元、首付款约63至64.5亿美元（同比+124%），截至6月30日累计81笔，交易金额占全球约59%。 2.战略洞察：云顶新耀EVER001交易验证"引进-改良-再出海"路径：该分子由信诺维原研，云顶新耀2021年获得肾脏领域全球权益，此次以超11亿美元授权Travere（7月7日完成交割，首付款到账）。作为共价可逆BTK抑制剂，EVER001规避了不可逆BTK抑制剂的持续抑制毒性，瞄准全球尚无获批药物的原发性膜性肾病（pMN）等五个免疫介导肾病，买方Travere为美国肾病专科公司，资产与渠道高度匹配。和誉医药与礼来的多靶点发现合作（最高约19亿美元）表明，中国小分子发现平台开始进入MNC的"管线供给方"角色，而非单一资产出让。这与1月石药与阿斯利康185亿美元多肽平台合作（首付12亿美元，中国授权史上最高首付，5月29日到账）、5月恒瑞与BMS 152亿美元双向授权共同构成上半年中国BD的平台化主线。质量信号重于数量信号：上半年首付款总额同比增长124%、平均首付显著抬升，说明MNC对中国资产的付费时点从临床后期前移至早期，且6月新增交易中NewCo模式（Bionyra）与专科公司直采（Travere）并存，退出路径多元化。需要冷静看待的是，总金额中里程碑占比仍高，历史退货率约四成，首付款占比才是资产质量的真实锚点。五、平台技术诱导邻近与AI发现双线放量 1.战略定位： 6月平台型交易集中在两条主线：一是诱导邻近（induced proximity）全面开花，覆盖BTK降解剂、分子胶、DAC三种形态；二是AI制药从单笔合作转向"多伙伴并行变现"，英矽智能继3月礼来27.5亿美元合作后再签韩国SK，Alnylam携手AI初创Inceptive刷新RNAi发现模式。共同点仍是低首付、高里程碑、深度绑定。 2.战略洞察：罗氏与Nurix的BTK降解剂合作为降解剂领域史上最大交易之一：首付7亿美元、含里程碑总额最高约30亿美元。Bexobrutideg在经多线治疗CLL中实现83% ORR、中位PFS 22.1个月，且具脑渗透性，计划2026年夏启动二线CLL Phase III，并扩展至慢性自发性荨麻疹、多发性硬化等免疫与神经适应症。交易采用60/40开发成本分担、美国50/50分润、海外低至高双位数分成的深度共同开发结构，反映罗氏对降解剂跨疾病潜力的战略押注。诺华与Orionis的第二次合作（首付4000万美元、最高14亿美元）验证了"回头客"信号：MNC加深既有合作而非另觅新欢，通常意味着早期产出达到内部阈值。分子胶正从投机性modality转变为具备管线纵深确认的药物类别。 AI制药进入"多伙伴并行"变现阶段：英矽智能3个月内连签礼来（27.5亿美元）与SK（超25亿美元，其亚太区最大合作）；Alnylam以最高20亿美元引入Inceptive的AI基础模型设计siRNA；礼来再以最高19亿美元引入Ascidian的RNA外显子编辑平台切入肾病。AI/RNA平台首付占比普遍低于2%，但里程碑与分成结构将平台方与MNC长期绑定，"可持续产出能力"的定价模式已经成型。六、其他重要交易与边界说明产业链最大交易：默克KGaA于6月25日宣布以每股73美元现金收购Bio-Techne，总估值约113亿美元，为当月金额最大的交易。标的为生命科学试剂与仪器平台（细胞/基因/蛋白研究工具），属生命科学工具整合而非药物管线BD，但反映了制药产业链上游"卖铲人"资产在并购超级周期中的稀缺性定价。 CNS与疼痛：礼来收购德州神经科学公司4E Therapeutics（金额未披露），获得IND-enabling阶段的MNK抑制剂，布局非阿片类慢性疼痛机制性疗法，延续其在CNS领域的连续建仓（继3月78亿美元收购Centessa后）。其他披露交易：日本新药与Elixirgen Therapeutics合作开发DMD mRNA疗法EXG-7001（金额未披露）；勃林格殷格翰双签Immunai（1500万美元，AI发现T细胞靶点）与Combotope（SMART-Phage糖链抗体平台）；AmMax Bio与Lonza达成血液肿瘤ADC授权；Genesis Pharma与Alnylam扩展RNAi北欧商业化合作；CNX Therapeutics与Legacy Pharma合作在美国商业化Ethyol；英国Thalia收购microRNA抑制剂公司Sanmirna（首付367.5万英镑）。边界说明：Ipsen收购Memo Therapeutics（首付2亿欧元、总额超7亿欧元，核心资产potravitug为全球首创抗BK多瘤病毒单抗，用于肾移植后BKPyV相关肾病）于7月1日宣布，不计入6月统计，但作为Ipsen一周内双收购的组成部分在第三章战略洞察中引用。7月重大事项（Vertex约100亿美元收购Crinetics、诺华最高15亿美元收购Myricx、迪哲医药舒沃替尼授权阿斯利康首付6亿美元）亦不在本月统计范围七、核心观察与战略启示 1. 并购超级周期确立，专利悬崖焦虑是最底层驱动上半年全球医药并购1340亿美元已超2025年全年，33笔超十亿美元交易创2019年以来纪录。约3000亿美元年收入面临专利到期、约70个重磅药物将在数年内失去独占权，MNC的购买逻辑已从"补充管线"升级为"替换收入"，买家愿意为确定性支付40%至60%溢价。 2. "批准前夜"与"数据读出前"成为最佳收购窗口 GSK在Nuvalent两个PDUFA日期前出手、艾伯维在Apogee Phase 3启动前出手、Ipsen在Kartos Phase III读出前出手，共同特征是以溢价换取时间差。对卖方而言，关键催化事件前的3至6个月是估值最大化的谈判窗口；对买方而言，临批准资产的风险调整后回报优于已上市资产的价格竞争。 3. 诱导邻近从技术主题升级为交易主线 6月单月覆盖降解剂（罗氏/Nurix，最高约30亿美元）、分子胶（诺华/Orionis，最高14亿美元）、DAC（强生/Firefly 10亿美元收购、罗氏/C4超10亿美元合作）三种形态。诱导邻近正在复制ADC在2023至2024年的交易轨迹，成为下一个MNC必须卡位的平台型modality。 4. 中国出海的第二增长曲线是小分子与IBD，而非双抗ADC的简单延续 6月中国三笔代表性交易分属小分子肾病（云顶新耀）、小分子发现平台（和誉）、TL1A单抗/双抗（TrueLab），加上上半年977亿美元级总量与124%的首付款增速，中国资产的出海逻辑正从"热门靶点fast-follow"转向"差异化机制+平台供给"。对BD筛选的启示是：海外买方对中国小分子与自免资产的接受度已被验证，罕见病与专科适应症的中国资产同样具备全球交易能力。 5. 法国中型药企成为最激进买家群体 Servier（Day One 25亿美元+Edgewise最高26.5亿美元）与Ipsen（Kartos最高17.5亿美元+Memo超7亿欧元）在4个月内各完成两笔大型收购，均以罕见病/专科药后期资产为标的。中型药企通过密集并购对冲规模劣势，是2026年除MNC外最确定的买方力量，也是中国后期资产值得重点覆盖的潜在交易对手。本分析来源于甄育资本，基于Endpoints News、FiercePharma/FierceBiotech、BioWorld、BioPharma Dive、Labiotech、STAT News、Pharmaceutical Executive、SEC文件（GSK 6-K、Edgewise 8-K）、公司官方新闻稿（AbbVie、GSK、Servier、Ipsen、Incyte、Edgewise、Biogen、Nurix、Insilico、TrueLab）、港交所公告（云顶新耀、石药集团）、医药魔方NextPharma及PR Newswire/GlobeNewswire披露。数据截至2026年7月20日，涵盖已公开财务条款的重点交易；未披露金额的合作按披露口径标注。建议结合具体项目需求，进一步分析相关治疗领域的竞争格局与监管动态。 Part1：关于甄育资本甄育资本（ZhenYu Capital）成立于2020年，是一家深耕全球医疗大健康领域的创新型投资机构。作为由资深科学家、投资专家组成的国际化团队，通过战略合作、跨境资本运作及产业赋能，助力中国生物医药创新企业实现全球化发展，同时推动海外优质医疗技术落地中国市场。凭借对中国及北美、欧洲、日本等海外医疗市场的深度覆盖，甄育资本主要提供以下三大核心服务： 1.跨境投行业务专注为全球Biotech及Pharma企业提供资本运作解决方案，涵盖跨境融资、并购重组（M&A）及全球权益许可（Licensing）服务，重点推进中国与欧美日等成熟医疗市场间的技术转化与产业协同。 2.战略顾问业务面向国内创新医疗项目，提供定制化的海外市场拓展策略，包括BD合作、跨境投资并购标的筛选、国际临床开发路径规划，以及全球商业化授权（License-out）的全周期咨询服务。 3.早期投资孵化聚焦生物医药领域具有突破性技术的早期项目（PCC/Feasibility），重点布局FIC（首创新药）和BIC（同类最优）潜力标的。投资标准强调科学数据的临床转化价值、合理的估值体系，以及明确的全球商业化路径，同时开展国际前沿技术的引进与本土化开发。通过构建"资本+产业+跨境"的三维服务体系，甄育资本致力于成为连接中国与全球医疗创新生态的价值枢纽。感兴趣请联系我们！欢迎医疗创新领域同仁与我们建立连接，甄育资本将依托覆盖三大洲的产业网络与专业智库，为您的技术商业化提供跨境资源精准对接。联系人：周女士（BD） Email address:houchao.zhou@two-winning.com 微信/电话：19136299173

100 项与 Sevasemten 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D12902	-	-	-

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
贝克型肌营养不良	临床3期	-	Edgewise Therapeutics, Inc.	-
糖原贮积病Ⅴ型	临床2期	丹麦	Edgewise Therapeutics, Inc.	2022-12-06
肢带型肌营养不良	临床2期	丹麦	Edgewise Therapeutics, Inc.	2022-12-06
杜氏肌营养不良症	临床2期	美国	Edgewise Therapeutics, Inc.	2022-10-24

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT06066580 (MESA, NEWS) 人工标引	临床2期	贝克型肌营养不良	-	Sevasemten (ARCH trial participants)	網淵膚鬱網製鹽鑰願製(製窪繭繭餘襯廠觸製餘) = Sustained stabilization over 3.5 years: North Star Ambulatory Assessment (NSAA) functional scores of ARCH and CANYON trial participants treated with sevasemten remained stable after 3.5 years and 2 years, respectively. The CANYON participants on placebo who rolled into the MESA study also had NSAA functional scores that trended upward during the first year after switching to sevasemten. Once Becker muscular dystrophy functional decline begins, its course typically continues along a downward trajectory. Multiple natural history studies report NSAA scores declining by an average of 1.0 to 1.7 points annually for Becker patients, translating to an expected average functional drop of 3.0 to 5.1 points across 3 years. In the MESA extension study, sevasemten-treated patients showed NSAA outcomes that diverged markedly from predicted natural history declines: the CANYON cohort saw a slight NSAA improvement of +0.1 over 2 years versus a predicted natural decline of −2.9 points, while the ARCH cohort maintained a +0.1 NSAA benefit over 3.5 years compared with an expected historical decline of −5.3 points. 鬱蓋餘糧衊繭艱艱觸壓 (淵壓齋齋膚鏇觸蓋繭衊 )	积极	2026-03-10
				Sevasemten (CANYON trial participants)
NCT05160415 (ARCH, MDA2026)	临床1期	贝克型肌营养不良	97	Sevasemten (Becker muscular dystrophy)	獵觸觸襯製餘顧獵築壓(鹽蓋繭膚顧積網願壓繭) = 願構簾憲築襯顧鹹簾願齋鑰襯製遞艱衊糧憲製 (鏇齋夢憲糧製廠選憲鬱, -0.4 ~ 0.8) 更多	积极	2026-03-08
NCT05160415 (ARCH, CTgov)	临床1期	贝克型肌营养不良	12	Sevasemten	築衊網憲獵鑰艱繭鑰醖 = 遞鏇襯淵夢淵蓋夢願範範網鹽餘築醖蓋餘淵艱 (築選醖鏇襯築襯窪築積, 糧餘獵膚蓋遞鏇膚鏇夢 ~ 淵簾醖鹹範鑰鑰獵獵糧) 更多	-	2025-06-24
NCT05291091 (CANYON, MDA2025)	临床2期	贝克型肌营养不良	40	Sevasemten	膚艱廠壓鹽構觸蓋繭鑰(糧醖製鏇糧壓憲窪淵齋): P-Value = 0.02 更多	积极	2025-03-16
				Placebo
NCT05160415 (ARCH, MDA2025)	临床1期	贝克型肌营养不良	12	Sevasemten	顧獵鏇遞獵淵築鹹構壓(蓋築願廠齋憲獵衊構願): Difference = 2.3 (95.0% CI, 1.0 ~ 3.6), P-Value = < 0.001 更多	积极	2025-03-16
				Sevasemten (Natural History Controls)
NCT05291091 (CANYON, BusinessWire) 人工标引	临床2期	贝克型肌营养不良	-	Sevasemten	顧膚衊壓衊繭鏇窪衊膚(鹽餘蓋襯繭築鑰淵糧艱): Difference (%) = -28, P-Value = 0.02 达到更多	积极	2024-12-16
				Placebo
NCT05160415 (ARCH, MDA2023) 人工标引	临床1期	杜氏肌营养不良症 CK \| TNNI2	12	EDG-5506 10-15 mg	鬱夢蓋範衊窪簾願獵憲(構積構遞憲繭積鑰鬱觸) = 醖獵糧範餘鹹築壓齋齋醖淵夢觸廠憲襯醖鑰衊 (鏇築憲餘衊鑰網構簾齋 ) 更多	积极	2023-03-19
NCT05160415 (ARCH, Corporate Publications) 人工标引	临床1期	贝克型肌营养不良	12	EDG-5506	衊衊鹽構願鏇顧衊遞鑰(夢膚範網繭觸製艱廠糧) = 25% Dizziness; 25% Somnolence; 25% Headache; 17% Fall; 8% Gastroenteritis virus 選壓網廠製蓋顧繭廠鏇 (鹽壓範獵鬱製廠夢遞遞 )	积极	2022-09-12
NCT05160415 (ARCH, Corporate Publications) 人工标引	临床1期	贝克型肌营养不良	12	EDG-5506	繭鹹網鑰壓鹹壓醖夢鹹(積蓋獵鬱憲鹹壓構簾鏇) = 構簾願夢鏇繭構範製蓋廠淵簾廠獵齋遞廠築範 (餘齋廠築遞廠鹽壓簾鑰 ) 更多	积极	2022-06-20
NCT04585464 (BusinessWire) 人工标引	临床1期	贝克型肌营养不良	7	EDG-5506	鑰蓋餘遞構壓選積膚範(淵製廠選築蓋齋窪餘蓋) = 顧願鹽製觸膚艱膚積壓窪膚廠衊壓選構襯糧醖 (鹹鏇夢獵築構膚網糧鬱 ) 更多	积极	2022-01-05
				Placebo	-