Ataluren

– First quarter 2026 total revenue of $273 million, including $226 million of product revenue, supporting full-year 2026 guidance raise – – Global Sephience™ (sepiapterin) launch momentum continues with first quarter 2026 revenue of $125 million, representing 36% quarter-over-quarter growth – – Positive topline results from 24-month interim analysis of PIVOT-HD extension study of votoplam, supporting ongoing global Phase 3 INVEST-HD study and potential regulatory interactions – – Open-label vatiquinone registration study to be initiated in Q3 2026 based on FDA feedback – WARREN, N.J., May 7, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ended March 31, 2026. "We are off to a strong start to 2026 with outstanding revenue performance this quarter that supports raising our full-year product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "The Sephience launch continues to be strong, with sustained momentum in the US and growing momentum internationally as more countries contribute to the global launch." Key Corporate Updates Q1 2026 total revenue of $273 million, with $226 million of product revenue Sephience global launch continues strong momentum Q1 2026 revenue of $125 million, including $112 million revenue in the US and $13 million revenue ex-US, up 36% from Q4 2025 1,244 total patients on commercial therapy worldwide as of March 31, 2026 Continued strong cadence of patient start forms in the US, with ~140 per month over past several months, surpassing 1,500 mark in the quarter First commercial sales in Japan in Q1 2026, with revenue expected from up to 30 countries by year-end 2026 Reported positive topline results in April 2026 from 24-month interim analysis of PIVOT-HD long-term extension study of votoplam, an oral small molecule splicing agent Dose-dependent benefit on cUHDRS in Stage 2 participants relative to natural history cohort, with 52% slowing of disease progression at 10 mg dose Continued favorable safety and tolerability with no treatment-related NfL spikes PTC and Novartis will complete data review and align on potential regulatory interactions Findings support ongoing global Phase 3 INVEST‑HD study of votoplam led by Novartis First patient dosed in Phase 3 INVEST-HD study, triggering $50 million milestone payment from Novartis to PTC in Q2 2026 Type C meeting with FDA held in April 2026 to discuss vatiquinone study to support NDA resubmission for Friedreich's ataxia program Based on meeting discussion and written feedback, PTC expects to initiate an open-label study using matched natural history control in Q3 2026 Primary endpoint will be change in mFARS from baseline to 24 months First Quarter 2026 Financial Highlights Total net product revenue and royalty revenue was $272.4 million for the first quarter of 2026, compared to $189.9 million for the first quarter of 2025. Total net product revenue across the commercial portfolio was $225.6 million for the first quarter of 2026, compared to $153.4 million for the first quarter of 2025, representing a 47% increase. Sephience net product revenues were $124.6 million for the first quarter of 2026, representing 36% growth compared to fourth quarter of 2025. Translarna™ (ataluren) net product revenues were $59.0 million for the first quarter of 2026, compared to $86.2 million for the first quarter of 2025. Translarna first quarter of 2026 revenue includes a large government purchase order from Brazil. Emflaza® (deflazacort) net product revenues were $21.5 million for the first quarter of 2026, compared to $47.8 million for the first quarter of 2025, due to continued generic erosion. Roche reported Evrysdi® (risdiplam) sales of approximately 464 CHF million, resulting in royalty revenue of $46.8 million to PTC for the first quarter of 2026, compared to $36.4 million to PTC for the first quarter of 2025. Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $100.9 million for the first quarter of 2026, compared to $109.0 million for the first quarter of 2025. Non-GAAP R&D expenses were $89.7 million for the first quarter of 2026, excluding $11.1 million in non-cash, stock-based compensation expense, compared to $100.3 million for the first quarter of 2025, excluding $8.7 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $86.2 million for the first quarter of 2026, compared to $81.0 million for the first quarter of 2025. Non-GAAP SG&A expenses were $73.9 million for the first quarter of 2026, excluding $12.3 million in non-cash, stock-based compensation expense, compared to $71.6 million for the first quarter of 2025, excluding $9.4 million in non-cash, stock-based compensation expense. Net loss was $2.8 million for the first quarter of 2026, compared to net income of $866.6 million for the first quarter of 2025. Cash, cash equivalents, and marketable securities were $1,892.5 million on March 31, 2026, compared to $1,945.4 million on December 31, 2025. Shares issued and outstanding as of March 31, 2026, were 82,882,024. PTC Updates Full-Year 2026 Financial Guidance Total product revenue guidance raised to $750 to $850 million, with expected total revenue of $1.08 to $1.18 billion GAAP R&D and SG&A expense guidance remains $775 to $815 million Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 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) cUHDRS: Composite Unified Huntington's Disease Rating Scale DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: US Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease mFARS: Modified Friedreich's Ataxia Rating Scale NDA: New Drug Application NfL: Neurofilament light chain 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 live webcast, please visit the "Events & Presentations" page 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 here to receive dial-in details. 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, Facebook and Instagram. 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 Updates Full-Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total revenue guidance and (ii) 2026 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 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; 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 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 or Waylivra. 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

氮杂环骨架是82% FDA 批准小分子药物的核心结构，如何从廉价易得的原料快速构建结构多样、立体精准的氮杂环，一直是药物合成领域的 “卡脖子” 难题。近日，中科院上海有机所张禄敏研究员团队在Science发表重磅研究，开创饱和伯胺的发散式、连续式骨架编辑新策略，实现单一底物向 15 类以上氮杂环的高效转化，为药物发现按下 “加速键”。 领域痛点：传统方法的四大局限     长期以来，饱和伯胺的骨架改造面临诸多瓶颈，传统策略难以满足药物研发需求，官能团兼容性差：经典贝克曼重排需强还原剂，不耐受腈基、酯基等敏感基团；区域选择性难控：仅纯 E/Z 型肟能实现选择性，异构体分离难度极大；骨架改造单一：仅支持线性编辑，无法实现多样化骨架跃迁；底物适用受限：早期方法依赖易爆过氧化物，仅适配特殊结构底物。核心突破：高价碘介导的 “万能骨架编辑平台”     研究团队突破传统思路，以廉价饱和伯胺为原料，借助高价碘试剂的温和氧化特性，构建了一套通用、高效的骨架编辑体系，核心逻辑如下，关键中间体生成：高价碘试剂温和氧化伯胺生成亚胺，快速实现 N - 内化，甲氧基阴离子捕获不稳定的腈鎓离子，定量生成亚胺醚关键中间体；多功能试剂筛选：优选MTIB（甲氧基 - 对甲苯磺酰氧基碘苯） 为核心试剂，百克级规模化制备收率达 91%；配套 N-HVI 试剂，适配低温高选择性反应；温和反应条件：室温、常压下即可反应，无需高温高压，后处理简单。 成果惊艳：单一底物解锁 15 + 类氮杂环     在最优条件下，该体系展现出极强的底物普适性与骨架多样性，三大核心优势颠覆认知： 1. 官能团 “百毒不侵” 完美兼容甲基、叔丁基、酯基、卤素、氰基、醇羟基、烯烃、炔烃、缩醛、芳烃、杂环等30 余种敏感基团，氧化敏感基团、酸敏感基团均不受影响。 2. 骨架多样性拉满 环胺：5-9 元单环、13 元大环氮杂环高效合成；多环骨架：螺环、稠环、桥环氮杂环（如氮杂环丁烷并氮杂䓬、金刚烷衍生胺）；链状胺：异丙基、环丙基、叔丁基等位阻型仲胺，收率最高 98%；双氮杂环：二胺双反应，双氮杂䓬产物收率 80%。 3. 区域 / 立体选择性极致     烷基迁移偏好：给电子能力越强越易迁移（甲基 < 乙基 < 异丙基）；碳选择性：叔碳 > 仲碳 > 伯碳，区域比 > 20:1；立体专一性：手性中心完全保留，非对映选择性 > 20:1。 应用落地：药物分子与天然产物的 “一键改造”     该策略不仅适用于简单底物，更在药物后期修饰、天然产物骨架跃迁中展现超强实用性：上市药物直接编辑：成功修饰阿塔鲁伦、舒林酸、阿莫沙平、己酮可可碱、金刚乙胺等10 余种药物，收率 51%-96%；天然产物骨架重塑：对冰片胺、雪松烯、蒎烯、薄荷醇、甾醇等胺化衍生物，实现碳-氮嬗变、环收缩等连续编辑；工业化价值凸显：200 倍规模化合成 2-甲基氮杂䓬，收率 90%，区域选择性 > 20:1，成本从 0.3 美元 / 克提升至 331.6 美元 / 克。 连续编辑：从 “单一改造” 到 “骨架重编程”     团队进一步开发一锅连续骨架编辑技术，实现天然产物的深度骨架改造：碳-氮嬗变：将烯烃、不饱和酮、醇等结构精准转化为氮杂环；环收缩 / 扩环串联：一步实现 “开环 异构化-环化”，构建传统方法难以合成的紧凑骨架；三步连续编辑：从苯丙酸诺龙胺化物出发，23% 收率合成 5α-还原酶抑制剂中间体，较传统 10 步合成（总收率 0.8%）效率提升近 30 倍。科学意义：填补药物合成核心空白     这项研究的价值远超方法学本身：打通原料-药物骨架链路：廉价饱和伯胺→高价值氮杂环，大幅降低药物合成成本；加速 SAR 研究：单一底物快速生成多样骨架，助力构效关系系统筛选；拓展化学空间：创造大量传统方法无法获取的 3D 饱和氮杂环，挖掘未被探索的药物靶点；工业化潜力：试剂规模化制备、反应温和安全，适配工艺放大生产。 文献：Divergent and consecutive skeletal editing of saturated primary amines；DOI: 10.1126/science.aee5416.