PTC Therapeutics Announces New Sephience™ (sepiapterin) Data Reinforcing Broad Patient Benefits Including Sustained Metabolic Control and Diet Liberalization as well as Consistent Favorable Safety Profile

PTC Therapeutics (PTCT) presented new data on its PKU therapy Sephience at the 2026 SSIEM Symposium, showing benefits in lowering phenylalanine levels and diet liberalization across a broad PKU population. The data suggest Sephience could be relevant for a wider spectrum of PKU severity. PTC raised its full-year revenue guidance to US$1.18 billion to US$1.28 billion following stronger Q2 2026 results. Analysts have varying opinions on the company's future revenue and earnings, with some predicti

PTC Therapeutics (PTCT) presented data on Sephience at a metabolic disorders symposium. Clinical trials and real-world data showed significant reductions in blood phenylalanine levels and dietary liberalization in PKU patients. Sephience is approved in the U.S., EU, and Japan. The drug carries warnings about bleeding risk and hypophenylalaninemia. PKU affects about 58,000 people globally.

PTC Therapeutics won a bankruptcy auction for Sangamo Therapeutics' Fabry disease gene therapy, ST-920, paying $111M upfront and up to $100M in milestones. PTC outbid Astellas and TerSera. The deal requires court and regulatory approval. ST-920 showed positive clinical data, supporting an accelerated FDA approval pathway. Eli Lilly also acquired other Sangamo assets for $50M.

PTC Therapeutics (NASDAQ:PTCT) agreed to expand its Fabry disease pipeline with ST-920, a one-time AAV gene therapy. The deal includes $111 million upfront and up to $100 million in contingent milestones tied to regulatory approvals. PTC expects a rolling FDA BLA for accelerated approval to finish in Q4 2026. PTCT shares were about $72.55.

PTC Therapeutics (PTCT) agreed to acquire ST-920, a BLA-stage one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics for $111 million upfront plus milestones. PTC cites Phase 1/2 STAAR data and plans an accelerated approval BLA submission, with CMC due Q4 2026 and a 2027 launch. Close expected in Q3 2026.

PTC Therapeutics (PTCT) reported Q2 2026 revenue of $360.52M and net income of $83.5M, and raised its full-year 2026 total revenue outlook to $1.18B to $1.28B. The company also appointed Hege Sollie-Zetlmayer to its board. The update may affect investor views on profitability and guidance.