PTC Therapeutics reports Q3 2026 results, raises revenue guidance, advances pipeline

PTC Therapeutics announced third-quarter 2026 results and a corporate update on October 29, 2026. The company said it acquired the ST-920 gene therapy for Fabry disease in the quarter, paying $111.0 million upfront with as much as $100.0 million more tied to US regulatory milestones.¹ The asset was purchased from Sangamo Therapeutics, Inc., according to the filing's reconciliation note. PTC said ST-920's BLA submission is expected to be completed in Q4 2026, with potential FDA accelerated approval and commercial launch in 2027.¹

For votoplam in Huntington's disease, PTC said a regulatory meeting slipped: due to FDA scheduling, the Type C meeting to discuss Phase 2 data and a potential accelerated approval path is now set for Q1 2027, while the global Phase 3 INVEST-HD study continues enrolling with a target of about 770 early symptomatic patients.¹

The company also disclosed that its PROVE-FA study, meant to support an NDA resubmission for Friedreich's ataxia, started in Q3 2026 and will enroll about 120 patients ages 7 to 21, with an open-label arm compared against a matched natural history control arm.¹ Elsewhere in the pipeline, PTC said it initiated a Phase 2a study of PTC844, a DHODH inhibitor, continued a Phase 1 study of the NLRP3 inhibitor PTC612, and expects PTC303, targeting MSH3, to enter the clinic in 2027.¹

On financials, PTC raised its outlook, saying expected total revenue increased to $1.29 to $1.32 billion, with product revenue guidance raised to $960 to $990 million from $850 to $950 million.¹ The company reported cash, cash equivalents, and marketable securities of $2,127.9 million as of September 30, 2026, up from $1,945.4 million at the end of 2025.¹ CEO Matthew B. Klein said the Sephience launch had reached its one-year mark and called it "another strong quarter of record product revenue," adding the company is "firmly on track to achieve our cash flow breakeven milestone this year."¹