PTC Therapeutics reported strong third-quarter growth, led by Sephience, and raised its 2026 revenue outlook. The company also outlined progress across its drug pipeline, including an acquired Fabry disease gene therapy it expects to submit for U.S. approval in the fourth quarter.
Key details
- Revenue increased sharply: Third-quarter revenue, including product sales and royalties, was $357.6 million, up from $201.7 million a year earlier. Product revenue rose to $275.9 million from $131 million.
- Sephience continued to lead growth: The drug generated $181.9 million in third-quarter sales, up 20% from the prior quarter. Sales were $145 million in the U.S. and $37 million outside the U.S. PTC said it had more than 2,000 active patients worldwide at quarter-end.
- 2026 outlook raised: PTC now expects full-year revenue of $1.29 billion to $1.32 billion and product revenue of $960 million to $990 million. The CEO said the company remains on track to reach cash-flow breakeven this year.
- Other product and royalty revenue: Translarna sales were $48.5 million, down from $50.7 million a year earlier; the quarter’s figure included a government purchase order from Russia. Emflaza sales fell to $25.1 million from $35.2 million. Royalties from Roche’s Evrysdi rose to $81.7 million from $70.8 million.
- Fabry disease program: PTC said it acquired ST-920, a one-time gene therapy for Fabry disease, and expects to complete a U.S. biologics-license application submission in the fourth quarter. The company sees potential for accelerated FDA approval and a commercial launch in 2027. It describes the therapy as having the potential to reduce the burden of chronic enzyme-replacement treatment.
- Huntington’s disease program: PTC’s global Phase 3 INVEST-HD trial of votoplam is enrolling early symptomatic patients, with a target of about 770. A planned FDA meeting to discuss Phase 2 data and a potential accelerated-approval path has moved to the first quarter of 2027 because of FDA scheduling.
- Friedreich’s ataxia program: PTC began the PROVE-FA study of vatiquinone in the third quarter to support a planned resubmission of its application for U.S. approval. The global study is expected to enroll about 120 patients ages 7 to 21 and includes an open-label treatment arm with a matched natural-history control.
- Earlier-stage pipeline: PTC began a Phase 2a study of PTC844, a next-generation DHODH inhibitor. Its Phase 1 study of oral NLRP3 inhibitor PTC612 is continuing, and PTC expects its MSH3-targeting candidate PTC303 to enter clinical testing in 2027.
- Research spending increased modestly: GAAP research and development expense was $105.4 million, compared with $100.2 million a year earlier. Non-GAAP R&D expense, excluding stock-based compensation, was $94.9 million versus $91 million.
- Investor updates: PTC scheduled a conference call for October 29 to review results and corporate updates and posted an updated corporate presentation.
The quarter’s growth was driven principally by Sephience, while the higher full-year outlook reflects stronger expected product revenue. PTC also has several clinical and regulatory milestones ahead, including the planned ST-920 application submission and continued enrollment in its Huntington’s disease and Friedreich’s ataxia studies.