Zeleciment Basivarsen

据泓博医药（301230）消息，2026年3月10日，全球生物医药领域迎来多项关键进展。临床方面，罗氏公布了口服SERD药物在晚期乳腺癌中的三期研究最新数据，同时罕见病治疗领域取得多项突破，包括DMD激素疗法展现长期安全性，以及针对强直性肌营养不良和CIDP的两种药物进入三期关键阶段。公司动态方面，益普生因临床验证挑战撤回一款EZH2抑制剂上市许可，而施维雅则与kyron.bio合作布局抗体精准糖基化技术。临床试验1. 罗氏口服SERD乳腺癌III期研究更新：罗氏近日披露了其评估口服选择性雌激素受体降解剂（SERD）giredestrant联合哌柏西利治疗ER阳性、HER2阴性晚期乳腺癌的三期persevERA研究更新数据。尽管在特定生物标志物亚组中显示出积极趋势，但整体临床终点的达成情况仍需进一步观察。这为竞争激烈的口服SERD赛道提供了重要参考。2. Santhera公布DMD药物8年长期数据：Santhera公司在2026年MDA大会上公布了其创新类固醇药物AGAMREE （vamorolone）治疗杜氏肌营养不良症（DMD）的长期随访数据。结果显示，长达8年的治疗保持了运动功能改善的稳定性，且相较于传统糖皮质激素，在保护骨密度和维持正常生长发育方面展现出显著的安全性优势。3. Dyne启动强直性肌营养不良药物III期试验：Dyne Therapeutics宣布正式启动其药物Z-basivarsen用于治疗1型强直性肌营养不良（DM1）的三期全球临床试验（HARMONIA研究）。该研究旨在评估药物改善肌肉功能及减少剪接缺陷的潜力。Z-basivarsen基于FORCE 平台开发，有望成为该领域的首创疗法。4. Dianthus发布CIDP药物III期中期结果：Dianthus Therapeutics于3月9日详细讨论了其长效补体C1s抑制剂Claseprubart（DNTH103）治疗慢性炎性脱髓鞘性多发性神经根神经病（CIDP）的三期CAPTIVATE研究A部分中期分析结果。早期数据显示出快速、持久且具有临床意义的响应。该药为可居家自我注射的皮下给药疗法，有望改善患者依从性。公司动态1. 益普生自愿撤回EZH2抑制剂上市许可：益普生宣布，由于后续验证性临床试验未能达到预设预期结果，决定自愿撤回其EZH2抑制剂Tazverik （tazemetostat）用于滤泡性淋巴瘤和上皮样肉瘤的上市申请或许可。此次撤回反映了加速批准药物在验证阶段的监管严格要求。2. 施维雅与kyron.bio合作开发抗体糖基化技术：kyron.bio宣布与施维雅建立战略合作伙伴关系。双方将利用kyron.bio的新型生物技术平台，共同推进抗体疗法中“精准糖基化”技术的开发，旨在通过精确控制糖链结构来优化抗体药物的治疗性能。原文：今日生物医药快讯-2026.03.10（来源：泓博医药） 关注同花顺财经（ths518），获取更多机会

- HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 - - 48-week trial will enroll approximately 150 individuals, and first sites are now open for enrollment - - Primary endpoint is the five times sit to stand (5xSTS) test; secondary and exploratory endpoints will assess muscle function, CNS manifestations, and patient- and clinician-reported outcomes - - HARMONIA trial design and protocol aligned with FDA; trial intended to serve as confirmatory trial for traditional approval in the U.S. and support ex-U.S. marketing applications - WALTHAM, Mass., March 08, 2026 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc . (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced the initiation of the Phase 3 HARMONIA trial of zeleciment basivarsen (z-basivarsen, also known as DYNE-101), in individuals with myotonic dystrophy type 1 (DM1). The design of the HARMONIA trial is being presented at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference on Wednesday, March 11, 2026 at 9:30 a.m. ET. A corresponding poster is also available in the Scientific Publications & Presentations section of Dyne’s website. “We are proud to be contributing to key advances in myotonic dystrophy clinical research with the initiation of a field-defining Phase 3 study designed to demonstrate the broad potential benefits of z-basivarsen,” said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. “Building on the ongoing registrational expansion cohort of the Phase 1/2 ACHIEVE trial, which is utilizing myotonia, as measured by video hand opening time, as an early indicator of clinical benefit for potential U.S. Accelerated Approval, HARMONIA is a larger and longer-term study utilizing a clinically meaningful functional measure as the primary endpoint. HARMONIA was designed to reinforce the best-in-class potential of z-basivarsen based on the differentiated capabilities of our FORCE platform to deliver therapeutics to a broad range of muscle systems as well as the CNS.” HARMONIA is a global, randomized, placebo-controlled, double-blind, confirmatory Phase 3 trial designed to assess the multi-system efficacy, safety, and tolerability of z-basivarsen administered intravenously to individuals with DM1. The trial will enroll approximately 150 participants age 16 and older who will be randomized 1:1 to receive 6.8 mg/kg of z-basivarsen or placebo every eight weeks (Q8W). The first trial sites are activated and open to enrollment. The primary endpoint is the change from baseline in the five times sit to stand (5xSTS) test at week 49. The 5xSTS test is a reliable and responsive measure that reflects key areas of DM1 impairment, including lower extremity strength, balance and trunk strength, which are critical to performing daily activities. Secondary endpoints include video hand opening time, quantitative muscle testing, the 10-Meter Walk/Run test, the Myotonic Dystrophy Health Index, and additional patient- and clinician-reported outcomes. The trial also includes a broad set of exploratory endpoints designed to assess multiple domains of DM1 central nervous system (CNS) impact. Following the 48-week double-blind placebo-controlled treatment period, patients will be eligible to enroll in a 24-week long-term extension. Dyne has aligned with the U.S. Food and Drug Administration (FDA) on the HARMONIA Phase 3 trial design and protocol. HARMONIA is intended to serve as a confirmatory trial to support conversion of Accelerated Approval to traditional approval in the U.S. and to support ex-U.S. marketing applications. About the HARMONIA Trial HARMONIA is a global, randomized, placebo-controlled, double-blind, confirmatory Phase 3 clinical trial evaluating the efficacy, safety and tolerability of zeleciment basivarsen (z-basivarsen, also known as DYNE-101) in people living with myotonic dystrophy type 1 (DM1). The trial will enroll approximately 150 participants age 16 and older who will receive 6.8mg/kg of z-basivarsen or placebo once every eight weeks for 48 weeks, and participants who complete the placebo-controlled period may enter a long-term extension during which all will receive 6.8mg/kg of z-basivarsen every eight weeks for up to 24 additional weeks. The primary endpoint of HARMONIA is the change from baseline in the five times sit to stand (5xSTS) test at week 49. The 5xSTS test is a reliable and responsive measure that reflects key areas of DM1 impairment, including lower extremity strength, balance and trunk strength, which are critical to performing daily activities. Secondary endpoints include video hand opening time, quantitative muscle testing, the 10-Meter Walk/Run test, the Myotonic Dystrophy Health Index, and additional patient- and clinician-reported outcomes. The trial also includes a broad set of exploratory endpoints designed to assess multiple domains of DM1 central nervous system impact. About zeleciment basivarsen (z-basivarsen, formerly known as DYNE-101) Z-basivarsen is an investigational therapeutic being evaluated in the Phase 1/2 global ACHIEVE clinical trial for people living with DM1. Z-basivarsen consists of an antisense oligonucleotide (ASO) conjugated to an antigen-binding fragment (Fab) that binds to the transferrin receptor 1 (TfR1) to enable delivery to muscle and the central nervous system. It is designed to deliver functional improvement in individuals living with DM1 by reducing toxic nuclear DMPK RNA to release splicing proteins and allow normal mRNA processing. Z-basivarsen has been granted Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA), as well as Orphan Drug designation from the European Medicines Agency (EMA) and the Ministry of Health, Labour and Welfare (MHLW) in Japan for the treatment of DM1. About Myotonic Dystrophy Type 1 (DM1) Myotonic dystrophy type 1 (DM1) is a rare, progressive, genetic neuromuscular disease with high morbidity and early mortality. DM1 affects ~40,000 people in the U.S. and ~55,000 people in the EU. The severity of symptoms and rate of progression varies. Symptoms can begin at any point in an affected person’s life, depending on the DM1 subtype. Adult-onset DM1 symptoms typically appear between 20 to 40 years of age. DM1 is caused by mutations in the DMPK gene, leading to a widespread disruption of RNA splicing, known as spliceopathy, which drives the multi-system manifestations of the disease. People experience a broad spectrum of symptoms, including: muscle weakness throughout the body, myotonia or difficulty relaxing muscles, excessive daytime sleepiness, fatigue, dysregulated sleep, cognitive impairments, cardiac arrhythmias, respiratory issues and gastrointestinal dysfunction. Although the genetic cause of DM1 is well understood, there are currently no approved disease-modifying treatments for DM1. About Dyne Therapeutics Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases. We are developing therapeutics that target muscle and the central nervous system (CNS) to address the root cause of disease. The company is advancing clinical programs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) as well as preclinical programs for facioscapulohumeral muscular dystrophy (FSHD), Pompe disease and multiple DMD mutations. At Dyne, we are on a mission to deliver functional improvement for individuals, families and communities. Learn more at , and follow us on X , LinkedIn and Facebook . Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Dyne’s strategy, future operations, prospects and plans, objectives of management, the potential of the FORCE platform, the clinical potential of zeleciment basivarsen (z-basivarsen, also known as DYNE-101), the potential of video hand opening time to serve as an intermediate clinical endpoint for U.S. accelerated approval, and the capability of Dyne’s FORCE platform to deliver therapeutics to a broad range of muscle systems as well as the central nervous system, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” “will” or “would,” or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Dyne may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the initiation and completion of preclinical studies and clinical trials; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; the timing of and Dyne’s ability to enroll patients in clinical trials; uncertainties as to the FDA’s and other regulatory authorities’ interpretation of the data from Dyne's clinical trials and the regulatory approval process; whether Dyne’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Dyne’s filings with the Securities and Exchange Commission (SEC), including the Company’s most recent Form 10-K and in subsequent filings Dyne may make with the SEC. In addition, the forward-looking statements included in this press release represent Dyne’s views as of the date of this press release. Dyne anticipates that subsequent events and developments will cause its views to change. However, while Dyne may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Dyne’s views as of any date subsequent to the date of this press release. Contacts: Investors Mia Tobias ir@dyne-tx.com 781-317-0353 Media Stacy Nartker snartker@dyne-tx.com 781-317-1938