PTC Therapeutics, Inc. Q4 FY2025 earnings call

• 2025 highlights: Initial global approvals and launch of Sephience for PKU, R&D pipeline advances including Phase II PIVOT-HD study of votoplam, strong revenue performance with full year revenue exceeding guidance.
• 2026 objectives: Continue Sephience launch momentum with growth through increased penetration in current markets and expansion into additional geographies; progress in R&D portfolio including votoplam Phase III study, vatiquinone development program steps, and advancing programs from RNA splicing platform; strong commercial execution by customer-facing teams.

Fourth quarter net product and royalty revenue was $263 million, and full year 2025 total net product and royalty revenue was $831 million, exceeding guidance of $750 million to $800 million. Total revenue includes $587 million of net product revenue. Sephience achieved $92 million in revenue in the fourth quarter and $111 million since launch in 2025. DMD franchise revenue in the fourth quarter was $66 million, with Translarna net product revenue of $39 million and Emflaza net product revenue of $27 million. Evrysdi had fourth quarter royalty revenue of $79 million and full year royalty revenue of $244 million. 2026 product revenue guidance is $700 million to $800 million, representing 19% to 36% year-over-year product revenue growth. Expense guidance for 2026 is $680 million to $720 million, with potential to reach cash flow breakeven in 2026.

• 2026 product revenue guidance: $700 million to $800 million, majority from Sephience, representing 19%-36% year-over-year product revenue growth, excluding Evrysdi royalty revenue.
• 2026 expense guidance: $680 million to $720 million.
• Potential to reach cash flow breakeven in 2026.
• Sephience expected to have patients on commercial drug in 20 to 30 countries by end of 2026, including Japan launching in coming weeks.

Q: Congrats on a really strong start for the Sephience launch. What's baked into your internal and external guidance for Sephience sales this year?

A: Revenue guidance this year is $700 million to $800 million, majority from Sephience. Vast majority of revenue expected from U.S., with contributions from ex U.S. later in the year as 20-30 countries are expected to have commercial patients by end of year.

Q: In your prepared remarks, you mentioned that you are seeing some of these patients that are “ lost to follow-up ” now becoming aware and wanting to get on the therapy. How is that dynamic working?

A: “ Lost to follow-up ” adults want to be on therapies, social media shares transformative benefits of Sephience, centers are tending to try therapy naive or tried and failed other therapies first, with positive feedback from health care providers.

Q: I wanted to get a little bit of color on vatiquinone. FDA has asked for an additional study for FA. Can you provide any color on details?

A: CRL for vatiquinone was based on statistical considerations, FDA has suggested an open-label or single-arm study within natural history control. Will meet with FDA in second quarter to finalize protocol, subject number and duration of treatment.