PTC Therapeutics Achieves $92M Q4 Sephience Sales, $831M 2025 Revenue Beat
Sephience generated $92 million in Q4, including $81 million in the U.S. and $11 million abroad, contributing to $263 million in quarterly product and royalty revenue. Full-year 2025 revenue of $831 million exceeded guidance, and PTC closed the year with $1.95 billion cash while guiding 2026 sales to $700–800 million.
1. Q4 and Full-Year Revenue Performance
PTC reported Q4 total product and royalty revenue of $263 million, driven by Sephience sales of $92 million ( $81 million U.S., $11 million ex-U.S.). Full-year 2025 net product and royalty revenue reached $831 million, surpassing guidance of $750–800 million.
2. Cash Position and 2026 Guidance
The company ended 2025 with $1.95 billion in cash after monetizing Evrysdi royalties for $240 million upfront and up to $60 million, and guided 2026 product sales to $700–800 million with potential cash flow breakeven.
3. Sephience Commercial Momentum
Sephience gained approvals in the U.S., E.U., Japan and Brazil, achieving 946 patients on therapy by year-end 2025, with broad age and severity uptake and high payer access supporting refill rates above single-digit discontinuations.
4. Pipeline Development Updates
Novartis plans to initiate the phase III INVEST-HD trial of Votoplam in H1 2026 with approximately 770 participants, while the FDA indicated Vatiquinone may require an additional study, potentially using a natural history comparator, before NDA resubmission.