PTC Therapeutics (PTCT) Q4 2025 earnings summary

Executive summary

• Achieved initial global approvals and strong launch of Sephience for PKU, with rapid uptake across all patient segments and age groups in the U.S., E.U., Japan, and other countries, generating $111M in revenue since launch.

• Monetized remaining Evrysdi royalty for $240 million upfront and up to $60 million in milestones, strengthening cash position to $1.95 billion at year-end 2025, up from $1.14 billion in 2024.

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

• Advanced R&D pipeline with positive phase II results for Votoplam in Huntington's disease and progress in RNA splicing and inflammation platforms.

Financial highlights

• Total revenues for 2025 were $1.73B, up from $806.8M in 2024; full-year 2025 net product and royalty revenue reached $831M, exceeding guidance.

• Sephience generated $92M in Q4 and $111M since launch in 2025.

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

• Net income for 2025 was $682.6M, compared to a net loss of $363.3M in 2024.

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

Outlook and guidance

• 2026 total product revenue guidance is $700M–$800M, a 19–36% increase from 2025, with Sephience as the main driver.

• Expense guidance for 2026 is $680M–$720M non-GAAP and $775M–$815M GAAP, with potential to reach cash flow breakeven.

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