PTC Raises 2026 Product Revenue Outlook After Sephience Gain

The Tip Desk · November 10, 2026 · 3 min read

Third-quarter product revenue reached $275.9 million as Sephience sales hit $181.9 million and the company lifted full-year product guidance to $960 million to $990 million.

PTC Therapeutics (PTCT) raised its 2026 product revenue outlook after Sephience sales continued to climb in the third quarter, even as a $111.0 million in-process research and development charge on a Fabry gene-therapy acquisition flipped the quarter to a net loss.

The rare-disease biopharmaceutical company reported total revenue of $358 million for the quarter ended September 30, 2026, including $276 million of product revenue. It increased expected full-year 2026 total revenue to $1.29 billion to $1.32 billion and lifted full-year product revenue guidance to $960 million to $990 million from $850 million to $950 million.

Sephience (sepiapterin) net product revenue was $181.9 million, up 20% from the second quarter of 2026. US sales were $145 million and ex-US sales were $37 million. Active patients worldwide reached more than 2,000 as of September 30, compared with 1,647 as of June 30. Chief Executive Officer Matthew B. Klein said the company was putting cash-flow breakeven on track for the year.

Total net product revenue and royalty revenue was $357.6 million, compared with $309.9 million in the second quarter. Commercial-portfolio product revenue was $275.9 million, versus $239 million in the second quarter.

Other commercial products contributed less. Translarna net product revenue was $48.5 million, including a government purchase order from Russia. Emflaza net product revenue was $25.1 million. Roche-reported Evrysdi year-to-date sales of approximately CHF 1,470 million produced $81.7 million of royalty revenue to PTC.

GAAP research and development expense was $105.4 million. Non-GAAP R&D was $94.9 million after excluding $10.5 million of stock-based compensation. GAAP selling, general and administrative expense was $80.1 million; non-GAAP SG&A was $66.7 million after excluding $13.3 million of stock-based compensation.

The company acquired the ST-920 Fabry disease gene therapy from Sangamo Therapeutics for $111.0 million upfront, with up to $100.0 million in contingent milestone payments tied to US regulatory approvals. It recorded $111.0 million of acquired in-process R&D expense in the third quarter. Net loss was $51.7 million, or $0.62 a share, versus net income of $15.9 million, or $0.20 a share, a year earlier.

Cash, cash equivalents and marketable securities were $2,127.9 million on September 30, compared with $1,945.4 million on December 31, 2025. Total debt was $536.3 million. Shares outstanding were 83,535,767.

ST-920 is a one-time administered AAV gene therapy. PTC said a BLA submission is expected to be completed in the fourth quarter of 2026, with potential FDA accelerated approval and commercial launch in 2027.

The votoplam Huntington’s disease program continued to advance. Due to FDA scheduling, a Type C meeting to discuss Phase 2 data and a potential accelerated-approval path is now scheduled for the first quarter of 2027. The global Phase 3 INVEST-HD study is actively enrolling toward a target of approximately 770 early symptomatic patients.

The vatiquinone PROVE-FA study to support an NDA resubmission for Friedreich’s ataxia was initiated in the third quarter, enrolling approximately 120 patients ages 7 to 21. PTC also initiated a Phase 2a study of PTC844, a next-generation DHODH inhibitor, and continued a Phase 1 study of PTC612, an oral NLRP3 inhibitor. PTC303, an MSH3 splicing candidate, is expected to enter the clinic in 2027.

GAAP R&D and SG&A expense guidance for full-year 2026 remains $775 million to $815 million. Non-GAAP R&D and SG&A guidance remains $680 million to $720 million, excluding an estimated $95 million of stock-based compensation.