Vatiquinone

– 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

PTC Therapeutics在RNA靶向小分子药物领域拥有经临床验证的平台，但其临床管线的表现表明，此类药物的价值并非普适。该公司的核心技术——调节RNA剪接——在脊髓性肌萎缩症中取得了明确成功，却在亨廷顿病和杜氏肌营养不良症中遭遇了不同程度的挑战或失败。 这一系列案例共同揭示了一个核心逻辑：小分子RNA靶向药物的临床转化效力，必须置于具体疾病的病理机制、未满足需求与竞争格局中进行严格评估。其成功高度依赖于疾病背景的匹配度，而非单纯的技术平台本身。 剪接平台经SMA验证： 成功与可复制性限制 PTC的RNA剪接调节平台在脊髓性肌萎缩症中确立了临床可行性，但其成功可复制性依赖于SMA疾病中明确且可靶向的单一生物学节点。该平台的核心优势在于作用精准、口服便捷以及作用可逆。然而，这些优势的发挥受制于特定疾病的生物学基础，平台向其他疾病的拓展面临不确定性。 Risdiplam的作用机制直接针对SMA的单一核心致病缺陷。 SMA主要由SMN1基因功能缺失导致运动神经元存活蛋白不足引起。人体内的SMN2基因因其pre-mRNA剪接通常会跳过第7号外显子，仅产生少量全长功能蛋白。Risdiplam作为一种小分子剪接修饰剂，通过特异性稳定SMN2 pre-mRNA第7号外显子处的U1 snRNP结合，促进该外显子包含，从而系统性增加全长SMN蛋白的表达。此机制直接纠正了疾病的根本原因。 口服给药是risdiplam区别于其他SMA疗法的关键差异化优势。 在risdiplam获批前，SMA的标准治疗包括需要反复鞘内注射的nusinersen以及一次性静脉输注但价格高昂的onasemnogene abeparvovec基因疗法。Risdiplam作为每日一次的口服制剂，避免了侵入性操作及其相关风险与不便，直接提升了患者依从性与治疗可及性。此优势已反映在其商业表现上：2025年，由罗氏负责商业化的Evrysdi®全球销售额达到约17.57亿瑞士法郎。 关键临床试验数据支持了risdiplam在不同类型SMA患者中的长期疗效。 针对1型SMA婴儿的FIREFISH研究（开放标签设计）5年长期随访数据显示，91%的患者存活，81%无需永久性通气支持。 针对2/3型SMA患者的SUNFISH研究（双盲/随机/安慰剂对照）显示，治疗12个月后，risdiplam组在运动功能测量32项总分上较安慰剂组有1.55分的显著改善，且长期数据表明改善得以维持。 与基因疗法相比，risdiplam代表了一种作用可逆且无需病毒载体的治疗范式，但需要终身服药。 Risdiplam的小分子特性使其作用可逆，停药后SMN蛋白水平预计会逐渐下降，这在管理潜在安全性问题时提供了灵活性，同时避免了腺相关病毒载体可能带来的免疫原性问题。然而，与旨在提供一次性长期疗效的基因疗法不同，risdiplam需要患者每日服药以维持疗效。 PTC已通过出售Evrysdi®剩余特许权使用费权益，将此项成功资产的部分未来财务价值提前变现。2025年12月，PTC将Evrysdi®剩余的特许权使用费权益以2.4亿美元预付款加上最高6000万美元里程金的价格出售给Royalty Pharma。此举为公司带来了即时现金，优化了资产负债表，但也意味着未来除了一项基于单年销售额达到25亿美元的1.5亿美元里程碑权利外，PTC将不再从此产品中获取直接收益。 平台拓展的挑战： 亨廷顿病中的疗效信号脱节 PTC的剪接调节平台向亨廷顿病的拓展，初步展示了降低致病蛋白的能力，但此生物标志物改善尚未能明确转化为具有统计学意义的临床功能获益。 与诺华合作的votoplam代表了平台在SMA之外的重要延伸，其II期数据揭示了在复杂神经退行性疾病中将靶点抑制转化为临床获益的挑战。 后续的开放标签延伸数据虽显积极，但样本量小且采用历史对照，证据等级有限。一项对21名患者进行的为期2年的开放标签延伸研究显示，与Enroll-HD自然史数据库中的未治疗患者相比，接受votoplam治疗的患者临床评分恶化速度更慢。然而，此项研究样本量小，且为非随机设计与历史对照组比较。基于II期结果，FDA已就III期试验INVEST-HD的设计与公司达成一致，该试验计划于2026年上半年启动。 将votoplam的初步数据与risdiplam在SMA中的成功对比，凸显了疾病背景对平台效力的决定性影响。 SMA的病理相对单纯，核心是运动神经元因SMN蛋白不足而死亡，增加功能性蛋白可直接延缓进程。亨廷顿病是涉及广泛脑区与多种细胞类型的复杂神经退行性疾病，发病机制多元，单纯降低亨廷顿蛋白水平是否足以在已出现症状的患者中稳定病程仍是问题。PIVOT-HD中2期与3期患者数据的异质性提示干预时机可能至关重要。 通读机制的挫折： DMD适应症中的明确失败 Ataluren在无义突变杜氏肌营养不良症中的III期失败及随后的全球监管撤回，标志着PTC的通读机制平台遭遇明确挫折。 该案例揭示了即便作用机制在早期临床中显示潜力，其在特定疾病的复杂体内环境中可能无法转化为确凿的临床获益。 监管机构的审评结论一致认为现有数据不足以支持ataluren的疗效。 基于III期数据，FDA始终未批准该药物。欧洲药品管理局曾给予有条件批准，但在确证性试验未能提供充分证据后，于2025年3月决定不再续签其上市许可。2026年2月，PTC正式从FDA撤回了其新药申请。 Ataluren的失败揭示了通读机制在DMD这一特定疾病背景下的根本性挑战。 蛋白表达水平不足：即使通读成功，所产生的全长抗肌萎缩蛋白量可能远低于生理所需水平。 疾病病理的复杂性：DMD涉及全身肌肉的进行性损伤与纤维化，炎症过程可能削弱少量功能性蛋白带来的益处。 临床试验设计：以6分钟步行距离作为主要终点在快速进展的DMD患者中变异性较大。 模式优势与持续挑战： 小分子在罕见病中的定位 PTC的资产组合展现了小分子药物在罕见病治疗中的模式优势与固有挑战。 优势包括口服给药、作用可逆性及无需病毒载体；挑战则集中于需终身服药以及在部分适应症中疗效不足的风险。公司的管线演进反映了小分子药物在基因治疗时代需寻找高度匹配的适应症才能体现其价值。 口服给药与作用可逆性是PTC小分子平台最突出的差异化优势。 与需要鞘内注射、静脉输注或侵入性手术的治疗方案相比，口服药物极大降低了治疗门槛与患者负担。例如，Evrysdi®使得SMA患者在家即可完成每日治疗。作用可逆性意味着一旦出现不可预见的不良反应，停药即可终止药物效应，为医生提供了风险管理灵活性。 然而，终身治疗的需求构成了小分子模式固有的经济负担与长期不确定性。 大多数罕见遗传病需要持续治疗以控制病情。每日服药意味着患者和支付方将面临数十年的持续支出。从长期安全性角度看，数十年持续暴露于一种系统性调节基础细胞过程的小分子之下，其潜在的累积毒性或脱靶效应仍是一个需要长期监测的科学问题。 PTC近期获批的产品Sephience™在苯丙酮尿症中的表现，展示了小分子在代谢疾病中明确的价值主张。Sephience™是2025年获批的口服疗法，作为四氢生物蝶呤的前体，用于增强苯丙氨酸羟化酶活性。关键的APHENITY III期试验（双盲/随机/安慰剂对照）显示，治疗6周后，sepiapterin组血液苯丙氨酸水平较安慰剂组显著降低395.9 µmol/L。长期扩展研究显示，大多数患者能够显著放宽饮食限制。 另一方面，vatiquinone在弗里德赖希共济失调中的挫折，再次凸显了在复杂神经退行性疾病中证明小分子临床获益的困难。 Vatiquinone是一种选择性抑制15-脂氧合酶的小分子。其关键的MOVE-FA III期注册试验未能达到改良弗里德赖希共济失调评定量表总分变化的主要终点。尽管在后续研究中观察到了积极信号，并获得了FDA对后续研究设计的认可，但III期主要终点的失败是一个明确的疗效证据不足的标志。 公司的财务状况因重磅授权交易而强化，但核心商业产品的增长面临具体挑战。 PTC在2025年因与诺华就votoplam达成的授权交易获得9.984亿美元一次性合作收入，实现净利润6.826亿美元，现金增至19.5亿美元。然而，剔除此次收入后，其核心商业产品表现如下：Translarna收入因欧盟撤市而同比下降26.7%。Emflaza因仿制药竞争，收入同比下降29.3%。主要的增长动力来自于新上市的Sephience™，2025年实现1.11亿美元收入。 PTC的未来路径将高度依赖其剪接平台在亨廷顿病中的最终验证，以及商业化产品组合的执行力。Votoplam的III期INVEST-HD试验（计划2026年上半年启动）是公司近期最重要的临床催化剂。同时，Sephience™的全球市场渗透、vatiquinone后续研究的推进，以及基因疗法Kebilidi的商业化起步，将共同决定公司能否在2026年实现其经营现金流盈亏平衡的目标。 以上是久谦的生物医药研究团队本次针对PTC Therapeutics的主要内容。之后我们还会继续推出一系列海外生科公司前沿生物医药研究文章。获取更多深度解析，敬请关注我们的公众号。我们将为您带来更多独家的海外创新药数据解读、管线与机制拆解、真实世界研究与交易案例分析与前沿趋势研判，助您保持领先认知，掌握全球生物医药信息差。 我们自 2023 年起 开始 持续、系统性地追踪 海外生物医药领域的 创新药研发演进，重点覆盖 创新药物作用机制（MOA）、平台型技术路径 及其 临床与商业化验证进展，涵盖 上百家欧美及其他海外市场的主流 / 前沿生物科技公司。 我们已逐步构建起一套 以机制可重复性与商业可扩展性为核心 的研究分析框架，完善了针对不同 技术平台、适应症与研发阶段 的 多维度评估 指标，并基于 大量横向机制对比 与 临床、BD 及商业化场景的交叉验证，沉淀出对 技术成熟度、应用边界 与 长期价值创造潜力 的 专业洞见。 关于我们： 我们是久谦旗下的生物医药研究团队，专注于创新药研发、生命科学工具、医疗技术平台及生物医药产业链等领域的研究，提供包含但不限于商业尽职调查、管线与机制评估、竞争格局分析、行业与赛道研究等服务类型。我们致力于 对早期技术路径与行业演进过程进行系统性研究与持续跟踪，助力投资人与企业在关键技术周期中形成认知优势与高质量决策支持。我们相信，唯有基于一线研究与数据与事实驱动的真实洞察，才能为客户提供真正可落地、可验证的研究结论。 若您有意向与我们进一步交流或合作，请扫描下方二维码联系我们： 您最看好哪家生物制药公司？您对RNA 靶向小分子药物有何见解？欢迎在评论区与我们交流！