– Completion of BLA submission expected in Q4 2026 –

– 预计于2026年第四季度完成生物制品许可申请（BLA）的提交 –

WARREN, N.J.

新泽西州沃伦

,

，

Sept. 21, 2026

2026年9月21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 completed the previously announced agreement with Sangamo Therapeutics, Inc. to acquire ST-920 – a BLA-stage, one-time administered AAV gene therapy for Fabry disease. A rolling BLA submission to FDA for accelerated approval of ST-920 is expected to be completed in Q4 2026..

）今日宣布，已完成此前公布的与Sangamo Therapeutics, Inc.的协议，收购ST-920——一种用于法布里病的BLA阶段、一次性给药的AAV基因疗法。预计将于2026年第四季度完成向美国食品药品监督管理局（FDA）提交的ST-920加速批准滚动生物制品许可申请（BLA）。

'Our team looks forward to completing the ST-920 BLA submission and potentially bringing a one-time administered, safe and effective durable disease treatment that addresses the limitations of chronic enzyme replacement therapy to the Fabry community,' said Matthew B. Klein, M.D., Chief Executive Officer..

“我们的团队期待完成ST-920的生物制品许可申请（BLA）提交，并有望为法布里病患者群体带来一种一次性给药、安全有效且持久的疾病治疗方案，从而克服慢性酶替代疗法的局限性。”首席执行官Matthew B. Klein博士表示。

About ST-920

关于 ST-920

ST-920 or isaralgagene civaparvovec is a one-time administered AAV gene therapy product candidate for the treatment of Fabry disease. In clinical studies, isaralgagene civaparvovec enabled long-term production of the deficient alpha-galactosidase A (α-Gal A) enzyme and significant reduction in globotriaosylceramide (Gb3) levels with durable clinical benefit and reduction of the burden associated with chronic Enzyme Replacement Therapy (ERT).

ST-920（即 isaralgagene civaparvovec）是一种一次性给药的腺相关病毒（AAV）基因疗法候选产品，用于治疗法布里病。在临床研究中，isaralgagene civaparvovec 实现了缺陷性α-半乳糖苷酶A（α-Gal A）的长期生成，并显著降低了球三糖神经酰胺（Gb3）水平，带来持久的临床获益，同时减轻了与慢性酶替代疗法（ERT）相关的负担。

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. Medicines and Healthcare products Regulatory Agency. .

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，以及英国药品和健康产品管理局的创新许可与准入途径（ILAP）资格。

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，而该酶对代谢球三糖神经酰胺（Gb3）至关重要。细胞内Gb3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公司

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，以造福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佳的治疗方案。

The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders..

该公司的战略是利用其科学专业知识和全球商业基础设施，为患者及其他利益相关方优化价值。

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) 618-6228

+1 (615) 618-6228

[email protected]

[email protected]

Media:

媒体：

Jeanine Clemente

珍妮·克莱门特

+1 (908) 912-9406

+1 (908) 912-9406

[email protected]

[email protected]

Forward-Looking Statements:

前瞻性声明：

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 anticipated benefits of ST-920; the timing of and potential for regulatory submissions and potential commercial launch for ST-920; 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ST-920预期获益的展望；ST-920监管申报的时间安排与可能性以及潜在的商业化上市前景；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“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”等词语及类似表述来识别。

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: PTC's ability to complete the BLA submission and obtain regulatory approval for and then commercialize ST-920; 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 ST-920, including the potential achievement of regulatory milestone payments that PTC may be obligated to make; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the po.

PTC的实际结果、业绩或成就可能与其所作前瞻性陈述中明示或暗示的内容存在重大差异，这是由于各种风险和不确定性所致，包括但不限于以下方面：PTC完成ST-920的生物制品许可申请（BLA）提交、获得监管批准并随后实现商业化的能力；针对PTC已商业化或未来可能商业化的产品或候选产品，与第三方支付方就定价、覆盖范围和报销进行的谈判结果；对Sephience的预期，包括其商业化以及PTC可能需达成的销售里程碑和可能须支付的或有付款；PTC在已获批地区维持Translarna用于治疗非杜氏肌营养不良症（nmDMD）的市场授权的能力，以及欧洲药品管理局人用医药产品委员会（CHMP）对Translarna采纳负面意见、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PTC有权从诺华获得开发、监管和销售里程碑付款、利润分享及特许权使用费，临床试验和研究的设计及预期时间安排，数据的可获得性，以及监管提交和回应（包括潜在的加速批准）；对ST-920的预期，包括PTC可能需实现的监管里程碑付款；对Upstaza/Kebilidi的预期，包括商业化、生产能力以及po。

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 ST-920..

与任何在研药物一样，新产品的开发、监管审批和商业化过程存在重大风险。无法保证任何产品（包括 Sephience、Translarna、Emflaza、Upstaza、Kebilidi、Evrysdi、Tegsedi、Waylivra 或 ST-920）能在任何地区获得或维持监管批准，或证明其在商业上取得成功。

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..

本文所含的前瞻性陈述仅反映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、估计或预测或其他情况的变化。

SOURCE PTC Therapeutics, Inc.

来源：PTC Therapeutics, Inc.

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