Ataluren

– Planned acquisition leverages existing regulatory and commercial infrastructure and leadership's experience in Fabry therapy commercialization – – 计划中的收购将利用现有的监管和商业基础设施，以及管理层在法布里疗法商业化方面的经验 –– ST-920 is a one-time administered AAV gene therapy for the enzyme deficient in Fabry disease with demonstrated long-term clinical benefits and safety profile – – ST-920 是一种一次性给药的 AAV 基因疗法，针对法布里病中缺乏的酶，已证实具有长期的临床获益和良好的安全性特征 –– BLA submission expected to be completed in Q4 2026 with potential for commercial launch in 2027 – – 预计将于2026年第四季度完成生物制品许可申请（BLA）的提交，并有望于2027年启动商业化上市 –– PTC will host a conference call today, Aug. 12, at 5 p.m. ET – – PTC 将于今天（8月12日）美国东部时间下午5点举行电话会议 –WARREN, N.J. 新泽西州沃伦, ，Aug. 12, 2026 2026年8月12日/PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: /美通社/ -- PTC Therapeutics, Inc.（纳斯达克股票代码：PTCT PTCT) today announced that it was selected as the winning bidder to acquire ST-920 – a BLA-stage one-time administered AAV gene therapy for Fabry disease – from Sangamo Therapeutics in a competitive bankruptcy auction. The terms include $111 million upfront and up to $100 million in contingent milestone payments based on certain regulatory approvals.A rolling BLA submission to FDA for accelerated approval of ST-920 is expected to be completed in Q4 2026. ）今日宣布，在一场竞争性破产拍卖中，其被选为中标方，将从Sangamo Therapeutics收购ST-920——一种处于生物制品许可申请（BLA）阶段、一次性给药的AAV基因疗法，用于治疗法布里病。交易条款包括1.11亿美元的预付款，以及基于某些监管批准情况、最高可达1亿美元的或有里程碑付款。预计向美国食品药品监督管理局（FDA）提交的ST-920加速批准滚动式BLA申请将于2026年第四季度完成。The BLA is based on evidence of meaningful favorable clinical effect on renal function and safety and tolerability profile over 52 weeks in the Phase 1/2 STAAR study.. 该生物制品许可申请（BLA）基于1/2期STAAR研究中为期52周的证据，证明其对肾功能具有具有临床意义的有利影响，且安全性和耐受性良好。'This transaction advances our strategy of leveraging our accomplished existing rare disease global commercial infrastructure to accelerate short- and intermediate-term revenue growth,' said Matthew B. Klein, M.D., Chief Executive Officer. 'The ST-920 gene therapy program puts another innovative and valuable product in the demonstrated capable hands of our customer-facing teams. “此次交易推进了我们的战略，即利用我们成熟的现有罕见病全球商业化基础设施，加速短期和中期的收入增长。”首席执行官Matthew B. Klein博士表示。“ST-920基因治疗项目将另一款创新且具有高价值的产品，交予我们已证明具备卓越能力的面向客户团队手中。”This was a unique opportunity with the potential for significant return on investment without the need for any development or commercial build and without impacting our objective of reaching cashflow break even in 2026. We look forward to working to bring ST-920 to all individuals who may benefit from this therapy as quickly as possible.'. 这是一次独特的机会，具有显著投资回报的潜力，且无需进行任何开发或商业化建设，也不会影响我们实现2026年现金流盈亏平衡的目标。我们期待尽快推动ST-920惠及所有可能从该疗法中受益的患者。Fabry disease is a rare, inherited lysosomal storage disorder caused by mutations in the GLA gene, resulting in deficiency of the alpha-galactosidase A (α-Gal A) enzyme and causing a range of serious signs and symptoms that require lifelong treatments. It is estimated that there are 11,000 people living with Fabry disease in the United States with similar prevalence rates in markets where PTC has the potential to commercialize.. 法布里病是一种罕见的遗传性溶酶体贮积症，由GLA基因突变引起，导致α-半乳糖苷酶A（α-Gal A）缺乏，并引发一系列需要终身治疗的严重体征和症状。据估计，美国有11,000名法布里病患者，在PTC具有商业化潜力的市场中，其患病率与之相似。ST-920 is designed as a one-time administered AAV gene therapy that enables long-term production of the deficient α-Gal A enzyme and significant reduction in globotriaosylceramide (Gb3) levels with demonstrated durable clinical benefit and reduction of the burden associated with chronic Enzyme Replacement Therapy (ERT). ST-920 是一款设计为单次给药的腺相关病毒（AAV）基因疗法，能够实现缺陷性 α-Gal A 酶的长期产生，并显著降低球三糖神经酰胺（Gb3）水平，已证实其具有持久的临床获益，并可减轻与慢性酶替代疗法（ERT）相关的负担。ST-920 has received Regenerative Medicine Advanced Therapy (RMAT) designation as well as Orphan Drug and Fast Track designations from FDA.. ST-920 已获得美国食品药品监督管理局（FDA）授予的再生医学先进疗法（RMAT）认定，以及孤儿药和快速通道认定。The Phase 1/2 STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks following ST-920 administration, as well as evidence of favorable effect on other aspects of Fabry disease including cardiac function and quality of life. The finding of improved eGFR over 52 weeks is differentiated from other Fabry therapies which demonstrated improved renal function but still negative eGFR slope from baseline. 1/2期STAAR研究显示，在给予ST-920后52周时，年化估计肾小球滤过率（eGFR）斜率呈阳性均值，并且有证据表明对法布里病的其他方面（包括心脏功能和生活质量）具有有利影响。在52周内eGFR改善的发现与其他法布里病疗法不同，后者虽显示出肾功能改善，但自基线起的eGFR斜率仍为负值。Furthermore, all study participants on ERT at study start were withdrawn from ERT. Durability of effect has been demonstrated with sustained increased α-Gal A activity maintained for up to 4.5 years for the earliest treated study participant, and evidence of maintained improvements in renal function across the study population. 此外，所有在研究开始时接受酶替代疗法（ERT）的参与者均停止了ERT。疗效的持久性已得到证实：最早接受治疗的参与者在长达4.5年的时间内持续保持α-半乳糖苷酶A（α-Gal A）活性升高，且研究人群整体显示出肾功能改善得以维持的证据。In addition, ST-920 has demonstrated an encouraging safety and tolerability profile and there is no requirement for routine prophylactic or post-infusion systemic immunosuppressive agents.. 此外，ST-920已显示出令人鼓舞的安全性和耐受性特征，且无需常规使用预防性或输注后全身免疫抑制剂。The BLA submission for accelerated approval is based on the intermediate clinical endpoint of annualized eGFR at Week 52 as aligned with FDA, with 104-week results from the STAAR study planned to provide confirmatory evidence to support traditional approval. The nonclinical and clinical BLA modules have already been submitted as part of a rolling submission, with the CMC package expected to be submitted in Q4 2026. 用于加速批准的生物制品许可申请（BLA）基于与FDA商定的第52周年化估算肾小球滤过率（eGFR）这一中间临床终点，并计划提供STAAR研究的104周结果作为确证性证据，以支持传统批准。非临床和临床BLA模块已作为滚动提交的一部分递交，预计化学、制造和控制（CMC）资料包将于2026年第四季度提交。PTC will also pursue regulatory approval outside of the United States, again leveraging existing regulatory and commercial rare disease infrastructure.. PTC 还将寻求在美国以外的监管批准，再次利用现有的罕见病监管和商业基础设施。The acquisition remains subject to definitive documentation, bankruptcy court approval, antitrust review, and other customary closing conditions. It is expected to close in late Q3 or early Q4 2026. 该收购仍须以最终协议的签署、破产法院的批准、反垄断审查以及其他惯例交割条件为前提。预计交易将于2026年第三季度末或第四季度初完成。Conference Call and Webcast Details 电话会议及网络直播详情PTC will hold a conference call today at 5 p.m. ET to discuss this news. To access the live webcast, please visit PTC 将于今日美国东部时间下午 5 点举行电话会议，讨论此新闻。如需收听网络直播，请访问Events & Presentations 活动与演示within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance 在PTC网站的投资者关系板块。网络直播的回放将在活动结束后的30天内于PTC网站提供。如需通过电话参与，请提前注册here 这里to receive dial-in details. 以获取电话拨入详情。About the STAAR Study 关于STAAR研究The Phase 1/2 STAAR study was a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate isaralgagene civaparvovec, or ST-920, a gene therapy product candidate in patients with Fabry disease. Isaralgagene civaparvovec requires a one-time infusion without preconditioning. STAAR 1/2期研究是一项全球性、开放标签、单剂量、剂量递增的多中心临床研究，旨在评估基因疗法候选产品isaralgagene civaparvovec（又称ST-920）在法布里病患者中的疗效。Isaralgagene civaparvovec只需一次性输注，无需预处理。The STAAR study enrolled patients who were on ERT, were ERT pseudo-naïve (defined as having been off ERT for six or more months), or who were ERT-naïve. The FDA has granted Orphan Drug, Fast Track, and RMAT designations to isaralgagene civaparvovec, which has also received Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and Innovative Licensing and Access Pathway from the U.K. STAAR 研究入组了正在接受酶替代疗法（ERT）的患者、ERT 伪初治患者（定义为已停用 ERT 六个月或更长时间）或 ERT 初治患者。美国食品药品监督管理局（FDA）已授予 isaralgagene civaparvovec 孤儿药、快速通道和再生医学先进疗法（RMAT）认定；该药物还获得了欧洲药品管理局的孤儿药产品认定和优先药物（PRIME）资格，以及英国的创新许可与准入途径（ILAP）资格。Medicines and Healthcare products Regulatory Agency.. 药品和保健品监管局About Fabry Disease 关于法布里病Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). The buildup of Gb3 in the cells can cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin. 法布里病是一种溶酶体贮积症，由α-半乳糖苷酶基因（GLA）突变引起，导致α-半乳糖苷酶A（α-Gal A）活性不足，而该酶对于代谢三己糖基神经酰胺（Gb3）至关重要。Gb3在细胞内的蓄积可对肾脏、心脏、神经、眼睛、肠道和皮肤等重要器官造成严重损害。Symptoms of Fabry disease can include decreased or absent sweat production, heat intolerance, angiokeratoma (skin blemishes), vision problems, kidney disease, heart failure, gastrointestinal disturbance, mood disorders, neuropathic pain and tingling in the extremities.. 法布里病的症状可能包括汗液分泌减少或缺失、不耐热、血管角皮瘤（皮肤瑕疵）、视力问题、肾脏疾病、心力衰竭、胃肠道紊乱、情绪障碍、神经性疼痛以及四肢麻木和刺痛感。About PTC Therapeutics, Inc. 关于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. PTC 是一家全球性生物制药公司，致力于发现、开发和商业化具有临床差异化优势的疗法，服务于患有罕见病的儿童和成人患者。作为其使命的一部分，PTC 正在推进一个强大且多元化的变革性药物研发管线，旨在为存在未满足医疗需求的患者提供同类最优的治疗方案。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 . 公司的战略是利用其科学专业知识和全球商业基础设施，为患者和其他利益相关者优化价值。如需了解更多关于PTC的信息，请访问。www.ptcbio.com www.ptcbio.comand follow us on LinkedIn, X, Facebook and Instagram. 并在 LinkedIn、X、Facebook 和 Instagram 上关注我们。For more information please contact: 如需更多信息，请联系：Investors: 投资者：Ellen Cavaleri 艾伦·卡瓦莱里+1 (615) 618-8228 +1 (615) 618-8228[emailprotected] [email protected]Media: 媒体：Jeanine Clemente 让妮娜·克莱门特+1 (908) 912-9406 +1 (908) 912-9406[emailprotected] [email protected]Forward-Looking Statements 前瞻性陈述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 Company's expectations regarding the proposed acquisition, including the expectation of finalizing definitive documentation for the transaction and the entry of an bankruptcy court order approving the transaction; the Company's ability to complete the acquisition; the anticipated benefits of ST-920; the timing of and potential for regulatory submissions and potential commercial launch for ST-920, if acquired; and PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. 本新闻稿包含经1995年《私人证券诉讼改革法案》界定的前瞻性陈述。除历史事实陈述外，本新闻稿中的所有陈述均为前瞻性陈述，包括公司对拟议收购的预期（包括完成交易最终文件的签署以及获得破产法院批准交易的命令的预期）、公司完成收购的能力、ST-920的预期收益、若完成收购后ST-920监管申报的时间安排和潜力以及潜在的商业发布时间表、PTC的战略、未来运营、未来财务状况、未来收入、预计成本以及管理层的战略目标。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.. 其他前瞻性陈述可通过以下词语识别：“指引”、“计划”、“预期”、“相信”、“估计”、“期望”、“打算”、“可能”、“目标”、“潜在”、“将”、“将会”、“可以”、“应该”、“继续”、“旨在”以及类似表述。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:uncertainty surrounding the bankruptcy's court entry of an order approving the acquisition and the possibility that the acquisition is not completed; 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 commercialization and the potential achievement of 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 geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the US on other regulatory bodies; 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; exp. PTC的实际结果、业绩或成就可能与其前瞻性陈述中明示或暗示的内容存在重大差异，这是由多种风险和不确定性导致的，包括但不限于以下方面：围绕破产法院批准收购的指令下达的不确定性，以及收购可能无法完成的风险；与第三方支付方就PTC已商业化或未来可能商业化的产品或候选产品进行定价、覆盖范围和报销谈判的结果；对Sephience的预期，包括其商业化进程，以及PTC可能需履行的销售里程碑达成情况和或有付款义务；PTC在已获批地区维持Translarna治疗非杜氏肌营养不良症（nmDMD）的市场营销授权的能力，以及欧洲药品管理局人用药品委员会（CHMP）对Translarna采取负面意见、美国撤回Translarna新药申请（NDA）对其他监管机构产生的影响；对PTC与诺华制药公司就votoplam治疗亨廷顿病达成的许可与合作协议的预期，包括其获得开发、监管和销售里程碑付款、利润分成及特许权使用费的权利，临床试验和研究的设计及预期时间表，数据可用性，监管提交及回应（包括潜在的加速批准）；对Upstaza/Kebilidi的预期，包括其商业化、生产能力，以及PTC可能需履行的销售里程碑达成情况和或有付款义务；exp.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.. 本文所载前瞻性陈述仅反映PTC在本新闻稿发布之日的观点。除非法律另有要求，PTC不承担也不计划更新或修订任何此类前瞻性陈述，以反映实际结果或本新闻稿发布之日后发生的计划、前景、假设、估计或预测的变化，或其他情况。SOURCE PTC Therapeutics, Inc. 来源：PTC Therapeutics, Inc.21 二十一% %more press release views with 更多新闻稿浏览量，使用Request a Demo 申请演示