PTC Therapeutics reports Q4 Sephience revenue of $92.5 million

PTC Therapeutics Inc. (NASDAQ: PTCT) reported unaudited fourth-quarter 2025 global revenue of $92.5 million for its drug Sephience, according to a company statement at the J.P. Morgan Healthcare Conference.

The Warren, New Jersey-based biopharmaceutical company said its unaudited 2025 total product and royalty revenue reached approximately $823 million, exceeding previous guidance. Product revenue alone totaled approximately $587.8 million for the full year.

Sephience generated $112.1 million in total net revenue during 2025 since its launch. The company reported 946 patients on commercial therapy worldwide as of December 31, 2025, with 1,134 patient start forms received in the United States.

PTC expects additional Sephience launches in 2026 in Japan, Brazil and other regions. The company projects 2026 product revenue of $700 million to $800 million, representing 19% to 36% year-over-year growth.

In December 2025, PTC sold its remaining Evrysdi royalty to Royalty Pharma for $240 million upfront and up to $60 million in sales-based milestones. The company retains rights to a $150 million milestone based on single-year Evrysdi sales of $2.5 billion by Roche.

The company reported cash, cash equivalents and marketable securities of approximately $1.94 billion as of December 31, 2025.

PTC held meetings with the FDA regarding its votoplam Huntington's disease program, reaching alignment on a global Phase 3 trial design planned to initiate in the first half of 2026. The FDA confirmed openness to a potential accelerated approval pathway.

For 2026, PTC anticipates GAAP research and development and selling, general and administrative expenses of $775 million to $815 million.

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