PTC Therapeutics Posts $92M Sephience Q4 Sales, 946 Patients on Therapy
PTC Therapeutics reported Q4 net product and royalty revenue of $263M, with Sephience generating $92M in the quarter ($81M U.S., $11M ex-U.S.) and 946 patients on therapy. Full-year 2025 revenue hit $831M, surpassing prior guidance, and the company ends 2025 with $1.95B cash while guiding 2026 product revenue to $700–800M.
1. Q4 and Full-Year 2025 Results
PTC Therapeutics reported Q4 net product and royalty revenue of $263 million and full-year 2025 net product and royalty revenue of $831 million, exceeding its prior guidance range of $750–800 million. Fourth-quarter revenue included $66 million from the DMD franchise and $79 million from Evrysdi royalties.
2. Sephience Commercial Performance
Sephience contributed $92 million in Q4 sales, including $81 million in the U.S. and $11 million ex-U.S., with 946 patients on therapy as of December 31, 2025. Management highlighted broad age and severity uptake, single-digit discontinuation rates, and favorable reimbursement policies with six- to 12-month refills and limited step edits.
3. Pipeline and Trial Plans
The company expects Novartis to initiate the phase III INVEST-HD trial of Votoplam in H1 2026, targeting ~770 participants with a 3:2 randomization and interim efficacy analysis. The FDA indicated Vatiquinone will likely require an additional study, potentially a single-arm trial using a natural history comparator, before NDA resubmission.
4. Balance Sheet and 2026 Guidance
PTC ended 2025 with $1.95 billion in cash after monetizing Evrysdi royalties for $240 million upfront plus up to $60 million. The company guided 2026 product revenue to $700–800 million and projected potential cash flow breakeven by year-end.