- Data from ongoing DELIVER trial demonstrated sustained functional improvement through 18 months; results from Registrational Expansion Cohort expected late 2025 - WALTHAM, Mass., Sept. 29, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company, today announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene that are amen ...

CAMBRIDGE, Mass., Sept. 29, 2025 (GLOBE NEWSWIRE) -- Ernexa Therapeutics (Nasdaq: ERNA), developing innovative cell therapies for the treatment of advanced cancer and autoimmune disease, today announced that its President and CEO, Sanjeev Luther, will participate in a featured panel discussion at the upcoming 5th Annual iPSC Drug Development Summit on October 1 in Boston. “Participation in the iPSC Drug Development Summit provides Ernexa the opportunity to contribute to important conversations shaping the f ...

核心事件 - Oncolytics Biotech公司宣布其候选药物pelareorep在一线胰腺导管腺癌的注册导向临床试验的拟议设计更新 [1] - 公司计划于2025年11月中旬与美国食品药品监督管理局会面以推进研究细节 [1] 临床试验设计 - 试验采用三臂设计：1) 吉西他滨+白蛋白紫杉醇对照组；2) 对照组+pelareorep；3) 对照组+pelareorep+检查点抑制剂 [8] - 主要终点为总生存期 [8] - 试验将具备足够的统计效力以检测研究组与对照组之间的统计学显著性 [8] - 计划进行中期疗效分析以便对潜在临床获益进行早期评估 [8] 临床数据依据 - 拟议设计基于一项事后汇总临床分析，该分析发现，在化疗基础上加用pelareorep实现了约22%的两年生存率，而历史第三方基准中仅接受化疗的患者两年生存率为9% [3] 公司战略与前景 - 公司首席执行官表示，该试验设计旨在为患者和监管机构提供有意义的数据，并有望确立pelareorep作为一线胰腺癌首个获批的免疫疗法 [4] - 公司认为该试验设计可能吸引潜在合作伙伴，并加速药物开发 [4] - pelareorep在转移性胰腺癌和乳腺癌的研发项目均已获得FDA的快速通道资格 [6] - 公司正积极寻求战略合作伙伴关系以加速开发并最大化商业影响 [6] 药物机制与研发进展 - pelareorep是一种研究性静脉给药的双链RNA免疫治疗剂，旨在通过激活先天性和适应性免疫反应，将免疫学上的“冷”肿瘤转化为“热”肿瘤 [5] - 该药物在一线胰腺癌研究、两项转移性乳腺癌的随机二期研究以及肛癌和结直肠癌的早期研究中均显示出令人鼓舞的结果 [5]

Driving therapeutic innovation forward Corporate Presentation September 2025 Forward-Looking Statements This presentation contains "forward-looking statements," including statements regarding expectations, beliefs, intentions or strategies for the future. These include statements regarding management's expectations, beliefs and intentions regarding, among other things, the potential success of our license agreements and joint venture, the commercial potential of motixafortide and GLIX1, expectations with re ...

Kennewick, WA, Sept. 29, 2025 (GLOBE NEWSWIRE) -- Vivos Inc. (OTCQB: RDGL) The Vivos Board granted authorization to form a wholly owned corporate subsidiary in India. Vivos Inc filed for the name of Vivos Scientific India LLP. Vivos is completing the steps remaining to obtain formal approvals from the Indian government. “This demonstrates our long-term commitment to doing business in India and supports our long-term plan of establishing our first international manufacturing center for RadioGel and IsoPet, f ...

Nes-Ziona, Israel, Sept. 29, 2025 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the “Company”), a clinical-stage macrophage reprogramming immunotherapy company, will participate in a live investor webinar, “Advancing Inflammatory Disease Treatment: Insights & Breakthroughs from Enlivex's Knee Osteoarthritis Program,” hosted by D. Boral Capital on September 30, 2025, at 10:00 AM Eastern Time. The discussion will bring together leading key opinion leaders (KOLs) in musculoskeletal disease, alon ...

U.S. stock futures were higher this morning, with the Dow futures gaining more than 100 points on Monday.Shares of Inventiva ADR (NASDAQ: IVA) fell sharply in today's pre-market trading following H1 results.Inventiva reported losses of $(1.84) per share compared to the analyst consensus estimate of loss of 41 cents per share. The company reported quarterly sales of $5.050 million which missed the analyst consensus estimate of $5.310 million.Inventiva shares dipped 5.5% to $5.72 in pre-market trading.Here ar ...

Accessibility StatementSkip Navigation Following the positive interim results in 5 patients announced earlier this year, a total of 9 patients have been enrolled as planned The study is progressing according to protocol, and no safety issues have been reported Study completion is anticipated in Q4 2025 with topline to follow HOUSTON, Sept. 29, 2025 /PRNewswire/ -- Coya Therapeutics, Inc. (NASDAQ: COYA) ("Coya" or the "Company"), a clinical-stage biotechnology company focused on developing biologics that enh ...

现金流与财务状况 - 截至2025年6月30日，Larimar的现金和投资总额为2.036亿美元，预计现金流可持续到2026年第四季度[5] 临床研究成果 - 在开放标签研究中，100%的参与者在第180天的皮肤FXN水平超过健康志愿者中位数的50%[21] - 参与者的mFARS中位数评分在1年内改善了2.25，相较于FACOMS自然历史参考人群的中位数恶化1.00[5] - 参与者的基线mFARS总分中位数为55.7，较FACOMS人群的49.7高出约12%[31] - 参与者的基线FARS-ADL整体评分中位数为17.5，较FACOMS人群的14.2高出约23%[31] - 参与者的基线9-HPT主手平均时间为95.4秒，显著低于FACOMS人群的124.8秒[31] 不良事件与耐受性 - 在39名参与者中，有7名在前6周内经历了过敏反应，所有人经过标准治疗后恢复健康[5] - Larimar的nomlabofusp在长期每日给药中总体耐受性良好，最常见的不良事件为轻度/中度局部注射反应[5] - Nomlabofusp的长期给药通常耐受良好，14名参与者接受治疗超过6个月，8名超过1年[34] 未来展望与产品开发 - Larimar计划在2026年第二季度提交生物制品许可申请（BLA），以寻求加速批准[5] - BLA提交寻求加速批准的目标时间为2026年第二季度，美国市场推出目标为2027年初[42] - Nomlabofusp的BLA提交将基于皮肤FXN水平作为潜在替代终点，长期数据显示皮肤FXN水平增加，类似于无症状携带者[43] 研究设计与招募 - 全球第3阶段双盲安慰剂对照研究的患者群体为2至40岁，预计招募100至150名参与者[40] - Nomlabofusp在mFARS评分中，基线中位数为54.75，1年后变化为-2.25，FACOMS研究中基线中位数为50.00，1年后变化为1.00[33] - 在FACOMS研究中，Nomlabofusp的FARS-ADL评分基线中位数为17.75，1年后变化为-0.50，而FACOMS研究中基线中位数为14.50，1年后变化为0.50[33] - Nomlabofusp在9-HPT评分中，基线中位数为71.95，1年后变化为-7.40，FACOMS研究中基线中位数为113.50，1年后变化为3.40[33] 药代动力学 - Nomlabofusp的药代动力学特征在不同研究中一致，30天后达到稳态[36]

R&D Spotlight: CLYM116 and the IgAN Opportunity SEPTEMBER 29, 2025 © 2025 Climb Bio 1 Forward Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including without limitation statements regarding: future expectations, plans and prospects for Climb Bio, Inc. ("Climb Bio"); expectations regarding the therapeutic benefits, clinical potential and clinical development of budoprutug and CLYM116; the trial design for ...