On January 12, 2026 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported an update on the Company’s progress and its outlook for 2026. Matthew B. Klein, M.D., Chief Executive Officer of PTC, will discuss these updates at the Company’s presentation at the J.P. Morgan 2026 Healthcare Conference on Monday, January 12, 2026, at 9:00 a.m. PST / 12:00 p.m. EST.
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"2025 was a highly successful year for PTC, highlighted by the initial regulatory approvals of Sephience and strong start to the global launch," Dr. Klein said. "In 2026, we look forward to continuing the Sephience launch momentum, advancing our innovative earlier-stage programs and moving the company towards becoming cash flow breakeven."
Key Corporate Highlights
Unaudited 2025 product and royalty revenue of approximately $823.4 million, exceeding guidance, and unaudited 2025 product revenue of approximately $587.8 million
Unaudited Sephience Q4 2025 net revenue of approximately $92.5 million, including $81.6 million in the US and $10.9 million ex-US
Unaudited Sephience total net revenue of approximately $112.1 million in 2025 since launch
1,134 patient start forms received in the US as of December 31, 2025
946 total patients on commercial therapy worldwide as of December 31, 2025
Additional Sephience launches expected in 2026, including in Japan, Brazil and other geographies
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 based on single-year Evrysdi sales of $2.5 billion by Roche
Cash, cash equivalents, and marketable securities of approximately $1.94 billion as of December 31, 2025
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, planned to initiate in H1 2026
FDA confirmed openness to potential Accelerated Approval pathway given significant unmet need
Type C meeting with FDA held in December 2025 to discuss vatiquinone Friedreich’s ataxia program; FDA requested additional information from MOVE-FA Phase 3 trial prior to providing guidance on next steps
Translarna (ataluren) NDA remains under FDA review
Unaudited 2025 Financial Results
Total unaudited product and royalty revenue for full-year 2025 was approximately $823.4 million
Total unaudited product revenue for full-year 2025 was approximately $587.8 million
Sephience unaudited total net revenue for 2025 was approximately $112.1 million
DMD franchise unaudited revenue for full-year 2025 was approximately $381.8 million, including unaudited product revenue for Translarna of approximately $235.5 million and for Emflaza (deflazacort) of approximately $146.3 million
PTC expects to report approximately $235.7 million of full-year 2025 royalty revenue associated with Evrysdi
PTC is finalizing its financial results for the 2025 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2025, subject to the completion of PTC’s financial closing procedures. Evrysdi royalty revenue estimates are based on internal estimates and pending accounting treatment of Evrysdi royalty following the December 2025 transaction.
2026 Financial Guidance
For the full year 2026, PTC anticipates:
Total product revenue of $700 to $800 million, excluding Evrysdi royalty revenue and collaboration revenue
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, JAN 12, 2026, View Source [SID1234661966])