PTC Therapeutics Q4 Revenue $263M Fueled by $92M Sephience Sales
PTC Therapeutics posted Q4 net product and royalty revenue of $263 million, driven by $92 million in Sephience sales ($81M U.S., $11M ex-U.S.) and full-year revenue of $831 million, exceeding 2025 guidance of $750 million to $800 million. Sephience secured approvals in the U.S., EU and Japan, reached 946 patients on therapy, generated $111 million since launch, and the company expects 2026 product revenue of $700 million to $800 million with potential cash flow breakeven.
1. Q4 and Full-Year Results
PTC Therapeutics reported fourth-quarter net product and royalty revenue of $263 million, including $92 million from Sephience ($81M U.S., $11M ex-U.S.), and full-year product and royalty revenue of $831 million, exceeding guidance of $750 million to $800 million. DMD franchise revenue totaled $66 million for Q4, and Evrysdi royalties contributed $79 million.
2. Sephience Launch Success
Sephience secured approvals in the U.S., EU and Japan, generated $111 million in sales since its 2025 launch and reached 946 patients on therapy. Management noted broad age and severity uptake, high refill rates and single-digit discontinuations driven by patient choice rather than safety concerns.
3. Cash Position and 2026 Guidance
The company ended 2025 with $1.95 billion in cash following a $240 million upfront royalty sale and up to $60 million contingent payment. PTC expects 2026 product revenue of $700 million to $800 million and anticipates potential cash flow breakeven during the year.
4. Pipeline and International Expansion
Novartis is set to initiate the phase III INVEST-HD trial of Votoplam in H1 2026 with approximately 770 participants in a 3:2 randomization, while the FDA signaled Vatiquinone may require an additional single-arm study. PTC projects Sephience commercial availability in 20 to 30 countries by year-end 2026, with Japan sales beginning in Q2 and early access programs in Europe and Brazil.