On February 19, 2026 PTC Therapeutics, Inc., (NASDAQ: PTCT) reported a corporate update and financial results for the fourth quarter and full year ending December 31, 2025.

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"We delivered another strong quarter and finish to 2025, building on the successful global launch of Sephience," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "With our robust commercial engine, innovative R&D programs, and strong financial position, we look forward to continued success as we approach cash flow breakeven."

Key Corporate Highlights

Full-year 2025 product and royalty revenue of $831 million, exceeding guidance
Global launch of Sephience off to strong start
Q4 2025 revenue of $92 million, including $81 million revenue in the US and $11 million revenue ex-US
Sephience total net revenue of $111 million in 2025 since launch
946 total patients on commercial therapy worldwide as of December 31, 2025
1,134 patient start forms received in the US as of December 31, 2025
Approval in Japan in December 2025; approval in Brazil in February 2026
Sephience global footprint expected to increase to 20 to 30 countries by end of 2026
In December 2025, PTC sold the remainder of its Evrysdi (risdiplam) royalty to Royalty Pharma for $240 million upfront and up to $60 million in sales-based milestones; PTC maintains the right to receive a $150 million milestone from Roche based on single-year Evrysdi sales of $2.5 billion
End-of-Phase 2 meeting with FDA held in Q4 2025 to discuss the votoplam Huntington’s disease (HD) program
Alignment reached on design of global Phase 3 trial, INVEST-HD, which is planned to initiate in 1H 2026
FDA confirmed openness for potential Accelerated Approval pathway given significant unmet need
Type C meeting with FDA held in December 2025 to discuss the vatiquinone Friedreich’s ataxia program; FDA indicated that an additional study would be necessary to support NDA resubmission and meeting minutes stated that this could be an open-label study with a natural history control group
Fourth Quarter and Full Year 2025 Financial Highlights

Total revenues were $164.7 million for the fourth quarter of 2025, compared to $213.2 million for the fourth quarter of 2024. Total revenues were $1,730.7 million for full year 2025, compared to $806.8 million for full year 2024. Included in total revenues is collaboration and license revenue of $998.4 million for the full year 2025, related to the votoplam license and collaboration agreement with Novartis, which closed in January 2025.
Total net product revenues were $184.0 million for the fourth quarter of 2025, compared to $150.1 million for the fourth quarter of 2024. Total net product revenues were $586.7 million for full year 2025, compared to $582.1 million for full year 2024.
Translarna (ataluren) net product revenues were $39.0 million for the fourth quarter of 2025, compared to $89.1 million for the fourth quarter of 2024. Translarna net product revenues were $235.3 million for full year 2025, compared to $321.1 million for full year 2024.
Emflaza (deflazacort) net product revenues were $27.1 million for the fourth quarter of 2025, compared to $50.5 million for the fourth quarter of 2024. Emflaza net product revenues were $146.4 million for full year 2025, compared to $207.2 million for full year 2024.
Roche reported Evrysdi full year 2025 sales of approximately 1,757 CHF million, resulting in royalty revenue of $244.2 million to PTC for full year 2025, compared to $203.9 million to PTC for full year 2024.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $133.1 million for the fourth quarter of 2025, compared to $124.8 million for the fourth quarter of 2024. GAAP R&D expenses were $455.2 million for full year 2025, compared to $534.5 million for full year 2024.
Non-GAAP R&D expenses were $124.3 million for the fourth quarter of 2025, excluding $8.8 million in non-cash, stock-based compensation expense, compared to $116.0 million for the fourth quarter of 2024, excluding $8.8 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $419.6 million for full year 2025, excluding $35.7 million in non-cash, stock-based compensation expense, compared to $497.9 million for full year 2024, excluding $36.6 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $96.9 million for the fourth quarter of 2025, compared to $84.7 million for the fourth quarter of 2024. GAAP SG&A expenses were $347.1 million for full year 2025, compared to $300.9 million for full year 2024.
Non-GAAP SG&A expenses were $87.2 million for the fourth quarter of 2025, excluding $9.7 million in non-cash, stock-based compensation expense, compared to $76.3 million for the fourth quarter of 2024, excluding $8.4 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $308.3 million for full year 2025, excluding $38.9 million in non-cash, stock-based compensation expense, compared to $262.9 million for full year 2024, excluding $38.0 million in non-cash, stock-based compensation expense.
Net loss was $135.0 million for the fourth quarter of 2025, compared to net loss of $65.9 million for the fourth quarter of 2024. Net income was $682.6 million for full year 2025, compared to net loss of $363.3 million for full year 2024.
Cash, cash equivalents, and marketable securities were $1,945.4 million on December 31, 2025, compared to $1,139.7 million on December 31, 2024.
Shares issued and outstanding as of December 31, 2025, were 81,474,366.
Full Year 2026 Financial Guidance

Total product revenue of $700 to $800 million, representing a 19 to 36% increase from 2025, with the majority from Sephience
GAAP R&D and SG&A expense of $775 to $815 million
Non-GAAP R&D and SG&A expense of $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million

(Press release, PTC Therapeutics, FEB 19, 2026, View Source [SID1234662790])