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PTC Therapeutics (PTCT) investor relations material
PTC Therapeutics Q3 2025 earnings summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Q3 2025 earnings summary4 Nov, 2025
Executive summary
Third quarter 2025 revenue reached $211 million, up from $196.8 million year-over-year, driven by the successful global launch of Sephience and continued DMD franchise contributions.
Sephience generated $19.6 million in its first quarter, with 521 US patient start forms and 341 patients on commercial therapy globally by September 30.
Achieved FDA and EC approvals for Sephience for PKU, with launches in the US and EEA and regulatory submissions in Brazil and Japan.
Entered a Rights Satisfaction Agreement with former Censa securityholders, paying $225.1 million upfront and up to $500 million in milestones, cancelling certain net sales-based royalty rights.
Novartis collaboration for votoplam (Huntington's disease) closed in January 2025, yielding a $1 billion upfront payment and potential for $1.9 billion in milestones, 40% US profit share, and ex-US royalties.
Financial highlights
Q3 2025 total revenue: $211 million, up from $196.8 million in Q3 2024; net product revenue: $131 million (down 3% YoY); royalty, collaboration, and license revenue: $80.1 million.
Sephience net product revenue was $19.6 million; DMD franchise revenue was $85.9 million (Translarna $50.7M, Emflaza $35.2M); Upstaza/Kebilidi $15.7M.
Royalty revenue from Evrysdi was $70.8 million for the quarter, up from $61.4 million YoY.
Net income of $15.9 million in Q3 2025, compared to a net loss of $106.7 million in Q3 2024.
Cash, cash equivalents, and marketable securities totaled $1.69 billion at quarter-end, up from $1.14 billion at year-end 2024.
Outlook and guidance
2025 full-year revenue guidance narrowed to $750–$800 million, at the upper end of the initial range.
Sufficient liquidity to fund operations for at least the next twelve months, supported by product sales, royalties, and milestone payments.
Projected GAAP R&D and SG&A expense for 2025: $805–$835 million; non-GAAP: $730–$760 million, excluding $75 million in stock-based compensation.
OpEx expected to decline in 2026, with detailed guidance to be provided at J.P. Morgan.
Ongoing commercialization of Sephience and continued pursuit of regulatory approvals in new territories.
What drives Sephience's strong initial uptake?
Impact of Translarna EU opinion on other regions?
What caused the Q3 R&D expense reduction?
Sephience long-term payer mix expectations?
Votoplam's path to accelerated approval?
OpEx trajectory for 2026?
How will Sephience patient retention be tracked?
What data package supports PTC518 accelerated approval?
Detail 2026 OpEx decline and profitability path.
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Next PTC Therapeutics earnings date
UBS Global Healthcare Conference 202511 Nov, 2025
Next PTC Therapeutics earnings date
UBS Global Healthcare Conference 202511 Nov, 2025
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Frequently asked questions
PTC Therapeutics Inc is a biopharmaceutical company focused on discovering, developing, and commercializing treatments for rare disorders. The company uses proprietary platforms to develop small molecules and gene therapies. Its portfolio targets genetic disorders, oncology, and neurological conditions. The company is headquartered in South Plainfield, New Jersey, and its shares are listed on the NASDAQ.
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