PTC Therapeutics (PTCT) Q4 2025 earnings summary

Executive summary

• Achieved initial global approvals and strong launch of Sephience for PKU, with $111M in revenue since launch and broad uptake across all patient segments and age groups, including approvals in Japan and Brazil.

• Advanced R&D pipeline, including positive phase II results for Votoplam, progress in RNA splicing and inflammation platforms, and a major collaboration with Novartis.

• Monetized remaining Evrysdi royalty for $240M upfront and up to $60M in milestones, ending 2025 with $1.95B in cash.

• Collaboration and license revenue of $998.4M in 2025, mainly from the Votoplam agreement with Novartis.

Financial highlights

• Full-year 2025 total net product and royalty revenue reached $831M, exceeding guidance, with total revenues for 2025 at $1.73B, up from $806.8M in 2024.

• Sephience generated $92M in Q4 and $111M since launch in 2025, with 946 patients on therapy worldwide by year-end.

• DMD franchise contributed $382M in 2025; Evrysdi full-year royalty revenue was $244M.

• Non-GAAP R&D and SG&A OpEx for 2025 was $728M, below guidance; GAAP R&D expenses were $455.2M and GAAP SG&A $347.1M.

• Cash, cash equivalents, and marketable securities totaled $1.95B at year-end 2025, up from $1.14B in 2024.

Outlook and guidance

• 2026 product revenue guidance is $700M–$800M, representing 19%–36% year-over-year growth, with most revenue from Sephience.

• 2026 GAAP R&D and SG&A expense guidance is $775M–$815M; non-GAAP guidance is $680M–$720M, excluding $95M in stock-based compensation.

• Expect commercial patients in 20–30 countries by end of 2026, with significant international expansion.