Ataluren

医道社：传承和发展民间中医 来源：海外网站 作者：医道社整理  版权归原作者所有，如有违规、侵权请联系我们删除！ 👆关注不迷路，更多创新药前沿资讯 大家好，我是微信公众号创新药新前沿的博主老汤。作为一名专注生物制药和创新疗法的从业者，我每天泡在临床试验数据和监管文件中。今天，我想和各位临床医生、药品研发人员聊聊Capricor Therapeutics（纳斯达克：CAPR）这家公司。为什么选它？因为它刚上演了一出生物科技界的“凤凰涅槃”：2025年7月FDA发出完整回应函（CRL），股价一度崩盘30%，公司市值缩水到2.5亿美元；转眼2025年11月下旬，HOPE-3三期试验顶线数据一出，股价从约2.50美元（部分报道中为6.36美元）直冲15-40美元，单日涨幅超500%，市值暴增超9.5亿美元，空头头寸（高达流通股25-34.6%）被彻底挤压。 这不是单纯的资本狂欢，而是科学数据和战略执行的胜利。DMD（杜氏肌营养不良症）这种致命遗传病，全球影响约25万男孩，心肌病是90%以上死亡主因，现有的基因疗法（如Sarepta的Elevidys）多针对早期可行走儿童，且覆盖有限。Capricor的Deramiocel（CAP-1002）却直击晚期、非行走型患者的心肌和上肢功能痛点。作为医生和研发者，你们肯定好奇：它怎么做到的？下面，我基于公司公告、Reuters、BioPharm Dive、Seeking Alpha等真实来源，结合临床细节，一层层拆解。走起！一、时间线回顾：从“濒死”到“重生”，Capricor的危机公关教科书 先别急着看数据，咱们把故事拉直。Capricor不是一夜成名，它是靠一步步硬扛过来的。 2025年7月11日，FDA对Deramiocel的首次BLA（生物制品许可申请）发出CRL，理由直白：现有数据（基于HOPE-2和开放标签延长期）“未构成有效性的实质证据”。股价当天早盘暴跌超30%，空头们欢呼雀跃，甚至有“医药恶童”Martin Shkreli公开做空，断言试验必败。公司当时现金有限，单资产高度集中，看起来像“高风险赌注”。 但Capricor没慌。两个月后（2025年9月），他们召开A类会议（Type A Meeting），这是CRL后药企与FDA的顶级沟通渠道。结果？FDA同意HOPE-3作为“补充研究”，直接支撑现有BLA框架重新提交（Type II resubmission，最长6个月审查）。他们还确认了主要终点（PUL v2.0上肢功能）和关键次要终点（LVEF左心室射血分数），并承诺“进一步的监管灵活性”。这等于FDA在说：“你去把HOPE-3做扎实，我给你第二次机会。” 为什么这转折震撼？因为CRL后，很多小盘Biotech要么重启BLA（耗时1-2年），要么融资稀释股权。Capricor却化险为夷，路径清晰：2025 Q1提交完整回应，PDUFA目标2026年中。加上Deramiocel的RMAT（再生医学高级疗法）、孤儿药、罕见儿科病指定（可获优先审评券，价值上亿美元），监管“绿色通道”已开。临床医生们，你们知道的，RMAT意味着更密集互动和灵活性，尤其对DMD这种高未满足需求的罕见病。 市场反应？2025年11月26日，HOPE-3顶线数据公布：双终点全中！股价开盘暴涨300%，盘中触及40美元，成交量超1亿股（日均仅200万）。Reuters直呼“生物科技今年最剧烈反弹”，BioPharm Dive称“deramiocel击中DMD目标”。空头惨遭“爆锤”，做空比例超1/3的流通股瞬间蒸发。这不是情绪投机，而是数据驱动的“空头屠宰场”。二、HOPE-3试验深度剖析：严谨设计+硬核数据，针对晚期DMD的“双重胜利” 作为医生和研发者，你们最关心的肯定是试验质量。HOPE-3不是“刷数据”的边缘试验，而是DMD领域少见的“严谨而不偷懒”3期研究。咱们用数据说话。 试验设计要点： ·类型： 随机、双盲、安慰剂对照、多中心（美国20个中心）。 ·人群： 106名男孩/年轻男性，平均年龄15岁，>75%基线已有心肌病——多为晚期、非行走型患者。这不是挑“容易刷分”的早期儿童，而是高难度队列（难度远超早期行走患者）。 ·给药： Deramiocel 1.5×10^8细胞/次 vs 安慰剂，每3个月静脉输注一次，共4次，12个月疗程。所有患者在稳定糖皮质激素和标准心脏药物基础上。 ·终点选择： 主要：PUL v2.0（上肢功能总分，DMD事实标准）；关键次要：心脏MRI测LVEF（结构+功能硬终点）。这些是FDA预先认可的，不玩花活。 结果详解：震撼的双重终点+多维度一致性 ·主要终点（上肢功能）： Deramiocel组12个月衰退仅为安慰剂的46%，减缓54%（p=0.029，统计显著）。首席研究员Craig McDonald博士（加州大学戴维斯分校，DMD大牛）评价：“在如此严重的晚期队列中，54%功能保留是非同寻常的，能显著延长患者独立生活时间——想想自理进食、操作轮椅。” ·关键次要终点（心脏功能）： LVEF恶化减缓91%（p=0.041）。心脏并发症是DMD 90%+死亡主因（WHO 2023数据），这几乎“冻结”了心衰进程，潜在延长生存5-10年（基于Lancet Neurology 2022自然史）。 ·额外亮点： 所有I型错误控制下的次要终点均显著；安全性完美，无新信号（3年HOPE-2 OLE随访，严重不良事件<5%，远低于基因疗法的免疫排斥）。 为什么这数据“王炸”？DMD患者中位生存<30岁，现无专用心肌疗法。HOPE-3在晚期人群中实现骨骼肌+心脏的“协同效应”，生物学一致性强（非单点偶发）。对FDA来说，这是“pivotal级”证据：效应量大、临床相关、安全稳定。相比Sarepta的Elevidys（2023批准，仅针对4-5岁儿童，价格>300万美元，未针对心肌），Deramiocel覆盖95%+患者（突变无关），适用晚期，成本估算50-100万美元/疗程。 患者价值？一个15岁男孩，本该20岁前心衰离世，现在能多抱妈妈、多玩游戏。这不是营销，是真实影响——肌营养不良症家长项目已公开欢迎，强调上肢+心脏的双重保护对生活质量的提升。三、技术黑科技大解密：Deramiocel的CDC+外泌体机制，为什么是DMD“游戏改变者”？ 核心竞争力在这里：Capricor的专利心脏来源干细胞（Cardiosphere-Derived Cells, CDCs）不是普通干细胞，而是“智能递送系统”。研发同行们，你们懂的，DMD根源是肌营养蛋白基因突变，导致持续炎症+纤维化，心肌病青少年期致命。 机制深度：抗纤维化+免疫调节的双管齐下Deramiocel是从健康心脏提取CDCs，静脉注射后不直接替换组织，而是释放外泌体（纳米级“小包裹”，携miRNA/蛋白质）。这些外泌体精准调控：  ·免疫重编程： 将促炎M1巨噬细胞转为修复M2型，减少炎症。 ·抗纤维化： 抑制成纤维细胞激活，胶原I/III表达降70%+（Biomedicines 2025.11论文，体外实验）。 这高度契合DMD病理：慢性损伤→炎症→纤维化→功能丧失。论文还验证：>100制造批次中，抗纤维化效应一致，非“小样本巧合”。AAEV 2025会议海报显示，电穿孔技术优化siRNA/PMO加载，产量提升10倍。 生产与可扩展性：工业级壁垒圣迭戈工厂通过FDA预审批检查（CMC问题解决，2025.9 A类会议）。异体（allogeneic）设计实现标准化、“即取即用”，避开自体疗法的复杂/高成本。相比基因疗法（免疫排斥风险高，NEJM 2023比较），Deramiocel安全记录稳（无排斥）。 为什么震撼？它“突变不限”（mutation-agnostic），不依赖特定基因亚型；“多面手”机制，跨骨骼肌+心肌；“无细胞”优势（外泌体避免疫）。BioPharm Dive评论：“外泌体平台是DMD‘多面手’，或成首个心肌特异标准疗法。”四、公司实力全景：小身板，大护城河——财务稳、伙伴强、平台广 Capricor市值8-10亿美元（2025.11后），但实力超预期。不是“实验室公司”，而是商业化ready。 财务稳健：跑道直通20262025 Q3财报：现金1.02亿美元（Q2增长20%），烧钱率2000万美元/季度，支撑至2026 Q4（无稀释压力）。NS Pharma协议锁定5000万美元预付款+里程碑，批准后版税15-20%。分析师预测：2026峰值销售>5亿美元（美国+日本，DMD基数2万+，Cantor Fitzgerald 2025.11）。 监管+商业布局：绿色通道+专业外包RMAT+孤儿药+罕见儿科券，满配标签。NS Pharma（武田子公司）独家授权美国/日本，减轻上市负担（他们有Viltepso等DMD产品，成熟网络）。Cedars-Sinai合作源于诺贝尔级心脏再生研究（2023 Pioneer Award）。 平台扩展：StealthX外泌体帝国不止DMD！StealthX将外泌体从“副产品”升级独立平台：  ·NIAID资助1期COVID疫苗（2025.8启动，2025 Q1数据），用工程化外泌体展示SARS-CoV-2蛋白，替代mRNA思路。 ·AAEV 2025：siRNA/PMO加载，规模化电穿孔，效率高、低免疫原。 ·未来：扩展Becker肌营养不良（2期2025）、癌症、神经病。Seeking Alpha（2025.11.27）称：“平台价值超Deramiocel，潜在并购目标（如辉瑞/诺华）。” Roth Capital重申“买入”，目标25美元（从13上调）；共识（6分析师）：强烈买入，平均28美元（50%+上涨空间）。五、竞争对比+独特定位：不是硬刚Sarepta，而是填补DMD“空白战场” DMD赛道卷：Sarepta（基因疗法Elevidys、外显子跳跃eteplirsen等）针对早期/特定突变，价格高、副作用大；PTC（ataluren）类似局限；Pfizer的Fordadistrogene 2025失败但迭代中。 Capricor聪明在“差异化”：专注晚期、非行走、心肌病人群（超基因疗法窗口）。PUL v2.0+LVEF锚定自理+生存；91%心脏保护是领域最强数据。不是抢蛋糕，而是开辟“后半段病程”标准疗法（临床指南/支付方话语权大）。市场规模>100亿美元（Grand View 2023），渗透20%即市值翻倍。六、理性思考：高回报机会，但别忽略“生物科技陷阱” 作为医生/研发者，你们知道创新药二元性。Deramiocel震撼：证明非基因编辑细胞疗法攻克心肌痛点，填补空白；外泌体“无细胞”+规模化，从跟跑到领跑。 但风险实锤：  ·监管： FDA二元（RMAT批准率70%），可能审亚组/持久性，要求上市后研究。 ·竞争/执行： 基因疗法迭代；CMC商业化挑战（虽预通过，但规模需验证）。 ·财务： 单资产依赖，潜在增发；股价波动（+500%后或-30%回吐）。 我的观点：长线看好，批准概率80%（HOPE-3后）。DMD市场大，Deramiocel+StealthX期权价值高。但仓位<5%，关注2025 Q1疫苗数据/BLA提交。不是稳健股，是高beta事件驱动。结语：Capricor，不是赌注，是DMD黎明与平台未来的火种 HOPE-3胜利，让Capricor从“死刑”到“封神”，技术+策略双轮驱动，2026或见证DMD心肌新时代。想象：男孩多活10年，自理生活——这价值万亿！基于真实数据（非虚构），我强烈看好：买入持有，目标30美元+。临床医生们，这对患者指南有何启发？研发者们，外泌体平台怎么优化？欢迎评论/私信，我继续追踪。关注创新药新前沿，前沿不负你！ 参考文献： 1.Capricor Therapeutics Q3 2025 Earnings & HOPE-3 Topline (公司官网，2025.11)。 2.Reuters: "Capricor shares soar on positive DMD trial data" (2025.11.26)。 3.BioPharm Dive: "Capricor’s deramiocel hits DMD goals" (2025.11)。 4.Biomedicines: "Potency Assay for CAP-1002" (2025.11)。 5.Seeking Alpha: "CAPR: HOPE-3 Success Changes Everything" (2025.11.27)。 6.FDA RMAT Designation & CRL Summary (2025.7-9)。 7.AAEV Conference Abstracts (2025.11)。 （老汤原创，基于公开数据。如需投资/临床建议，咨询专业人士！本文不构成投资建议，生物科技风险高，请谨慎。）

– Initiated US and EU launch of Sephience™ (sepiapterin) – – Strong initial Sephience uptake with global revenue of $19.6M and 521 start forms in the US as of September 30 – – Robust Q3 performance with total revenue of $211M – – Full-year 2025 revenue guidance narrowed to $750 - $800M – WARREN, N.J., Nov. 4, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the third quarter ended September 30, 2025. "We achieved an outstanding quarter, highlighted by the strong start to the global Sephience launch," said Matthew B. Klein, M.D., Chief Executive Officer. "The broad initial uptake supports the potential of Sephience to become the standard of care for all individuals with PKU, as well as the foundational product for PTC's sustained growth and success." Key Corporate Updates Third quarter 2025 total revenue of $211.0 million Global launch of Sephience™ initiated in US and Europe Third quarter 2025 revenue of $19.6 million, including $14.4 million revenue in the US and $5.2 million revenue ex-US 521 patient start forms received from 141 unique prescribers in the US as of September 30 341 total patients on commercial therapy worldwide as of September 30 Third quarter 2025 revenue for the DMD franchise of $85.9 million, including net product revenue for Translarna™ of $50.7 million and for Emflaza® of $35.2 million R&D Day planned for Tuesday, December 2nd in New York Key Clinical and Regulatory Updates Additional Sephience marketing authorization reviews ongoing including in Japan where decision is expected in Q4 2025 FDA meeting for votoplam Huntington's disease program planned for Q4 FDA meeting planned for vatiquinone Friedreich's ataxia program in Q4 Translarna NDA remains under FDA review Third Quarter 2025 Financial Highlights Total revenue was $211.0 million for the third quarter of 2025, compared to $196.8 million for the third quarter of 2024. Total revenue includes net product revenue across the commercial portfolio of $131.0 million for the third quarter of 2025, compared to $135.4 million for the third quarter of 2024. Total revenue also includes royalty, collaboration and license revenue of $80.1 million for the third quarter of 2025, compared to $61.4 million for the third quarter of 2024. Sephience net product revenue was $19.6 million for the third quarter of 2025. Translarna net product revenue was $50.7 million for the third quarter of 2025, compared to $72.3 million for the third quarter of 2024. Emflaza net product revenue was $35.2 million for the third quarter of 2025, compared to $51.9 million for the third quarter of 2024. Roche reported Evrysdi® 2025 year-to-date sales of approximately CHF 1,293 million, resulting in royalty revenue of $70.8 million to PTC for the third quarter of 2025, as compared to $61.4 million for the third quarter of 2024. Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expense was $100.2 million for the third quarter of 2025, compared to $161.4 million for the third quarter of 2024. Non-GAAP R&D expense was $91.0 million for the third quarter of 2025, excluding $9.1 million in non-cash, stock-based compensation expense, compared to $152.0 million for the third quarter of 2024, excluding $9.4 million in non-cash, stock-based compensation expense. GAAP SG&A expense was $84.0 million for the third quarter of 2025, compared to $73.5 million for the third quarter of 2024. Non-GAAP SG&A expense was $73.8 million for the third quarter of 2025, excluding $10.3 million in non-cash, stock-based compensation expense, compared to $63.1 million for the third quarter of 2024, excluding $10.3 million in non-cash, stock-based compensation expense. Net income was $15.9 million for the third quarter of 2025, compared to net loss of $106.7 million for the third quarter of 2024. Cash, cash equivalents, and marketable securities were $1,687.8 million as of September 30, 2025, compared to $1,139.7 million as of December 31, 2024. The third quarter cash balance reflects the PTC agreement to purchase approximately 90% of the Sephience annual percentage-based global net sales obligation owed to former Censa shareholders in exchange for an upfront payment of $225.0 million and future sales-based milestone payments. Shares issued and outstanding as of September 30, 2025, were 79,931,766. PTC Full-Year 2025 Financial Guidance For the full year 2025, PTC anticipates: Total revenue of $750 to $800 million, which includes in-line products and royalty revenue from Evrysdi. GAAP R&D and SG&A expense of $805 to $835 million. Non-GAAP R&D and SG&A expense of $730 to $760 million, excluding estimated non-cash, stock-based compensation expense of $75 million. Non-GAAP Financial Measures In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms: CHF: Confoederatio Helvetica Francs (Swiss francs) DMD: Duchenne Muscular Dystrophy FDA: U.S. Food and Drug Administration GAAP: Generally Accepted Accounting Principles NDA: New Drug Application nmDMD: Nonsense mutation Duchenne muscular dystrophy PKU: Phenylketonuria R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder: To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in for the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investors section of the PTC website at . A replay of the call will be available approximately two hours after completion and will be archived on the company's website for 30 days. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X and Facebook. For more information please contact: Investors: Ellen Cavaleri +1 (615) 618-8228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "PTC Full-Year 2025 Financial Guidance", including with respect to (i) 2025 total revenue guidance and (ii) 2025 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of regulatory and sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia and other regions; the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna on other regulatory bodies; PTC's ability to use the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from its trials in Translarna; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi, Waylivra or vatiquinone. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo