PTC Therapeutics, Inc. reported its third-quarter 2026 financial results and corporate update on October 29, 2026, disclosing total revenues of $357.8 million and the acquisition of the ST-920 Fabry disease gene therapy program for $111.0 million upfront.

Key Highlights

  • Total Q3 2026 revenues: $357.8 million, compared to $211.0 million in Q3 2025.
  • Sephience (sepiapterin) net product revenue: $181.9 million for Q3 2026, representing 20% growth compared to Q2 2026, with over 2,000 active patients worldwide as of September 30, 2026.
  • ST-920 acquisition: PTC acquired the ST-920 Fabry disease gene therapy from Sangamo Therapeutics, Inc. for $111.0 million upfront, with up to an additional $100.0 million in contingent milestone payments based on US regulatory approvals.
  • Net loss: $51.7 million for Q3 2026, compared to net income of $15.9 million for Q3 2025.
  • Full-year 2026 total revenue guidance raised: to $1.29 to $1.32 billion, with total product revenue guidance raised to $960 to $990 million from $850 to $950 million.
  • Cash, cash equivalents, and marketable securities: $2,127.9 million as of September 30, 2026, compared to $1,945.4 million as of December 31, 2025.

PTC Therapeutics Reports Record Product Revenue and Pipeline Progress in Third Quarter 2026

According to the filing, PTC Therapeutics (NASDAQ:PTCT) reported total net product revenue and royalty revenue of $357.6 million for Q3 2026, more than doubling total net product revenue to $275.9 million from $131.0 million in Q3 2025. Sephience led the commercial portfolio with net product revenues of $181.9 million, including $145 million in the US and $37 million outside the US. Translarna (ataluren) contributed $48.5 million in net product revenues, compared to $50.7 million in Q3 2025, with the filing noting that Q3 2026 revenue includes a government purchase order from Russia. Emflaza (deflazacort) generated $25.1 million, compared to $35.2 million in Q3 2025. Royalty revenue of $81.7 million was reported from Roche's Evrysdi (risdiplam), based on Roche-reported year-to-date sales of approximately CHF 1,470 million.

The company disclosed a net loss of $51.7 million for Q3 2026, compared to net income of $15.9 million for Q3 2025. The filing attributed a significant portion of this swing to $111.0 million in acquired in-process research and development expense related to the purchase of the ST-920 program from Sangamo Therapeutics, Inc., and a $19.3 million loss on extinguishment of debt. GAAP R&D expenses were $105.4 million and GAAP SG&A expenses were $80.1 million for the quarter. Non-GAAP R&D expenses, excluding $10.5 million in non-cash, stock-based compensation, were $94.9 million, and non-GAAP SG&A expenses, excluding $13.3 million in non-cash, stock-based compensation, were $66.7 million.

On the pipeline, the filing disclosed that a BLA submission for ST-920 is expected to be completed in Q4 2026, with potential FDA accelerated approval and commercial launch noted for 2027. The votoplam Huntington's disease program Type C meeting with the FDA is now scheduled for Q1 2027 due to FDA scheduling, and the global Phase 3 INVEST-HD study is actively enrolling with a target of approximately 770 early symptomatic patients. The vatiquinone PROVE-FA study, intended to support an NDA resubmission for Friedreich's ataxia, was initiated in Q3 2026 and will enroll approximately 120 patients ages 7 to 21. Shares issued and outstanding as of September 30, 2026 were 83,535,767, the filing stated.

What Was Disclosed

PTC Therapeutics filed a Q3 2026 earnings press release reporting $357.8 million in total revenues, a $111.0 million acquisition of the ST-920 Fabry disease gene therapy, a net loss of $51.7 million, and raised full-year 2026 total revenue guidance to $1.29 to $1.32 billion.