PTC Therapeutics Raises 2026 Outlook as Q3 Revenue Hits $358 Million

PTC Therapeutics (PTCT ) reported financial results for the third quarter ended September 30, 2026, on October 29, 2026, posting total revenues of $357.8 million and raising its full-year 2026 total revenue expectation to a range of $1.29 billion to $1.32 billion. The Warren, New Jersey-based company also disclosed that it acquired the ST-920 Fabry disease gene therapy from Sangamo Therapeutics during the quarter for $111.0 million upfront in cash.

According to the company’s third-quarter 2026 earnings release, total revenues of $357.8 million compared with $211.0 million in the third quarter of 2025. Net product revenue across the commercial portfolio was $275.9 million, compared with $131.0 million a year earlier, an increase the company characterized as exceeding 100%. PTC reported a net loss of $51.7 million, or $0.62 per share basic and diluted, compared with net income of $15.9 million, or $0.20 per share, in the third quarter of 2025. The quarter’s results included a $19.3 million loss on extinguishment of debt and $10.6 million of income tax expense, while income from operations was $25.1 million, compared with $3.0 million a year earlier.

Chief Executive Officer Matthew B. Klein said the company delivered “another strong quarter of record product revenue led by the continued momentum of the Sephience launch,” and said the performance has PTC on track to achieve its cash flow breakeven milestone this year. Now at the one-year mark, he said, the launch “has been a success by all measures.”

Product and Royalty Revenue Detail

Sephience (sepiapterin) generated net product revenue of $181.9 million in the third quarter of 2026, representing 20% growth compared with the second quarter of 2026, according to the company. The total included $145 million of revenue in the United States and $37 million ex-US. PTC reported more than 2,000 active Sephience patients worldwide as of September 30, 2026.

Translarna (ataluren) net product revenue was $48.5 million, compared with $50.7 million in the third quarter of 2025, and the company said the quarter’s Translarna revenue includes a government purchase order from Russia. Emflaza (deflazacort) net product revenue was $25.1 million, compared with $35.2 million a year earlier.

Roche reported year-to-date Evrysdi (risdiplam) sales of approximately CHF 1,470 million (Swiss francs), resulting in royalty revenue of $81.7 million to PTC for the third quarter of 2026, compared with $70.8 million in the same period of 2025.

ST-920 Acquisition and Pipeline Updates

During the third quarter of 2026, PTC acquired ST-920, a gene therapy for Fabry disease, from Sangamo Therapeutics for $111.0 million upfront, with up to an additional $100.0 million in contingent milestone payments based on US regulatory approvals. The upfront cash consideration was recorded as acquired in-process research and development expense in the quarter. The company describes ST-920 as a one-time administered adeno-associated virus gene therapy with the potential to reduce the burden associated with chronic enzyme replacement therapy. PTC expects the ST-920 Biologics License Application submission to be completed in the fourth quarter of 2026, with potential FDA accelerated approval and a commercial launch in 2027.

For the Huntington’s disease program, the company said an FDA Type C meeting to discuss votoplam Phase 2 data and a potential accelerated approval path is now scheduled for the first quarter of 2027 because of FDA scheduling. The global Phase 3 INVEST-HD study of votoplam is actively enrolling at sites worldwide, with target enrollment of approximately 770 early symptomatic patients.

The vatiquinone PROVE-FA study, intended to support an NDA resubmission for the treatment of Friedreich’s ataxia, was initiated in the third quarter of 2026. According to the company, the global study will enroll approximately 120 patients ages 7 to 21 and includes an open-label treatment arm with a matched natural history control arm.

The company also initiated a Phase 2a study of PTC844, a next-generation DHODH inhibitor; the Phase 1 study of PTC612, an oral NLRP3 inhibitor, continues to advance; and PTC303, a splicing development candidate targeting MSH3, is expected to enter the clinic in 2027.

The company increased its expected full-year 2026 total revenue to a range of $1.29 billion to $1.32 billion and raised total product revenue guidance to $960 million to $990 million from the previous range of $850 million to $950 million. GAAP research and development and selling, general and administrative expense guidance remains $775 million to $815 million, while non-GAAP R&D and SG&A expense guidance remains $680 million to $720 million, excluding estimated non-cash stock-based compensation expense of $95 million. The company said management uses these non-GAAP measures when assessing and identifying operational trends.

GAAP R&D expenses were $105.4 million for the third quarter of 2026, compared with $100.2 million a year earlier; non-GAAP R&D expenses were $94.9 million, excluding $10.5 million in non-cash stock-based compensation expense. GAAP SG&A expenses were $80.1 million, compared with $84.0 million in the third quarter of 2025; non-GAAP SG&A expenses were $66.7 million, excluding $13.3 million in stock-based compensation expense.

Cash, cash equivalents and marketable securities totaled $2,127.9 million at September 30, 2026, compared with $1,945.4 million at December 31, 2025. Total debt was $536.3 million, compared with $286.6 million at the end of 2025, and the liability for the sale of future royalties was $2,305.6 million, compared with $2,308.4 million. Shares issued and outstanding were 83,535,767 as of September 30, 2026.

For the nine months ended September 30, 2026, total revenues were $990.9 million, compared with $1,566.0 million in the same period of 2025, and net income was $29.0 million, or $0.35 per share basic, compared with net income of $817.6 million, or $10.40 per share basic, in the prior-year period.