Warren – October 30, 2026 -- PTC Therapeutics (NASDAQ: PTCT) reported third-quarter 2026 total revenue of $358 million and raised its full-year product revenue guidance to $960 million to $990 million, up from $850 million to $950 million. Total revenue guidance for 2026 now stands at $1.29 billion to $1.32 billion.
Product revenue more than doubles on Sephience launch
Net product revenue across the commercial portfolio reached $275.9 million, against $131.0 million in the third quarter of 2025. Net product and royalty revenue combined totaled $357.6 million, compared with $201.7 million a year earlier.
Sephience (sepiapterin) generated $181.9 million, up 20% from the second quarter. US sales were $145 million and ex-US sales $37 million. More than 2,000 patients were on therapy worldwide as of September 30. CEO Matthew Klein said the launch has been a success by all measures and that PTC is on track to reach cash flow breakeven this year.
Legacy products weaken while Roche royalty grows
Translarna revenue fell to $48.5 million from $50.7 million, and the figure includes a government purchase order from Russia. Emflaza sales declined to $25.1 million from $35.2 million.
Royalty revenue from Roche's Evrysdi rose to $81.7 million from $70.8 million. Roche reported year-to-date Evrysdi sales of about CHF 1,470 million.
Acquisition charge pushes PTC to a $51.7 million net loss
PTC posted a net loss of $51.7 million, compared with net income of $15.9 million in the prior-year quarter. The company paid $111.0 million upfront for the ST-920 Fabry disease gene therapy, booked in full as acquired in-process R&D expense. Contingent milestone payments of up to $100.0 million depend on US regulatory approvals.
GAAP R&D expenses were $105.4 million, up from $100.2 million. GAAP SG&A expenses fell to $80.1 million from $84.0 million. Cash, cash equivalents and marketable securities totaled $2,127.9 million at September 30, compared with $1,945.4 million at year-end 2025.
Full-year GAAP R&D and SG&A expense guidance is unchanged at $775 million to $815 million. Non-GAAP guidance stays at $680 million to $720 million, excluding about $95 million of estimated stock-based compensation.
ST-920 BLA filing due in Q4, with possible 2027 launch
PTC expects to complete the Biologics License Application for ST-920, a one-time AAV gene therapy, in the fourth quarter of 2026. The company is targeting potential FDA accelerated approval and a commercial launch in 2027.
Votoplam FDA meeting slips to Q1 2027
Due to FDA scheduling, the Type C meeting on votoplam Phase 2 data and a potential accelerated approval path in Huntington's disease has moved to the first quarter of 2027. The global Phase 3 INVEST-HD study is enrolling and targets about 770 early symptomatic patients.
The vatiquinone PROVE-FA study began in the third quarter to support an NDA resubmission in Friedreich's ataxia. It will enroll about 120 patients aged 7 to 21 and includes an open-label arm with a matched natural history control.
PTC also started a Phase 2a study of PTC844, a next-generation DHODH inhibitor, and continues a Phase 1 study of the oral NLRP3 inhibitor PTC612. The MSH3-targeting splicing candidate PTC303 is expected to enter the clinic in 2027.