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PTC reports third-quarter 2026 revenue of $358 million and raises product guidance

Tickers PTCT

Sephience revenue reached $182 million in the quarter, the company acquired ST-920 for $111.0 million upfront, and it now expects total revenue of $1.29 to $1.32 billion for 2026.

Key facts

Company
PTC Therapeutics, Inc. (NASDAQ: PTCT)
News type
Company Updates
Primary source
PTC Therapeutics, Inc. Exhibit 99.1 (2026-10-29)
Reading a sequencing gel, 1980. Photo: Linda Bartlett, public domain, via Wikimedia Commons. The picture shows the field, not the companies named.

PTC Therapeutics, Inc. (NASDAQ: PTCT) reported third-quarter 2026 results and a corporate update on October 29, 2026. In a press release filed as Exhibit 99.1 to a Form 8-K, the Warren, New Jersey company said total revenue for the quarter was $358 million, including $276 million of product revenue. Net product revenue and royalty revenue totaled $357.6 million, compared with $201.7 million in the third quarter of 2025. The company reported a net loss of $51.7 million for the quarter, compared with net income of $15.9 million a year earlier.

Chief executive Matthew B. Klein, M.D., said the company is on track to reach its cash flow breakeven milestone this year.

Product sales

Total net product revenue across the commercial portfolio was $275.9 million for the quarter, compared with $131.0 million in the third quarter of 2025, an increase of more than 100%. Sephience, the company's treatment for phenylketonuria, generated $181.9 million in net product revenue, 20% growth from the second quarter of 2026. The release breaks Sephience revenue into $145 million in the United States and $37 million outside the United States, and says the company had more than 2,000 active patients worldwide as of September 30, 2026.

Translarna (ataluren) net product revenue was $48.5 million, compared with $50.7 million a year earlier. Emflaza (deflazacort) net product revenue was $25.1 million, compared with $35.2 million in the third quarter of 2025. Translarna and Emflaza both fell, so the quarter's growth rests on Sephience. The release does not explain either decline.

Royalty revenue came from Roche's sales of Evrysdi (risdiplam). Roche reported year-to-date Evrysdi sales of approximately CHF 1,470 million, which produced royalty revenue of $81.7 million for PTC in the third quarter of 2026, compared with $70.8 million a year earlier.

Expenses and the ST-920 acquisition

GAAP research and development expense was $105.4 million, compared with $100.2 million a year earlier. Excluding $10.5 million of non-cash, stock-based compensation, non-GAAP research and development was $94.9 million. GAAP selling, general and administrative expense was $80.1 million, compared with $84.0 million.

In the third quarter, PTC acquired the ST-920 gene therapy for Fabry disease for $111.0 million upfront, with up to an additional $100.0 million in contingent milestone payments based on US regulatory approvals. The company booked $111.0 million of acquired in-process research and development expense for the quarter, which explains much of the net loss. The ST-920 BLA is not yet filed, and the milestone payments depend on US approvals.

Guidance and pipeline

PTC raised its full-year 2026 product revenue guidance to $960 to $990 million, from $850 to $950 million. Expected total revenue increased to $1.29 to $1.32 billion. GAAP research and development and SG&A expense guidance is unchanged at $775 to $815 million. Non-GAAP research and development and SG&A guidance is unchanged at $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million.

The release says a Type C meeting with FDA on votoplam Phase 2 data and a potential accelerated approval path in Huntington's disease is now scheduled for the first quarter of 2027, because of FDA scheduling, so the meeting that would test whether the data can support accelerated approval has slipped. The release gives no FDA view yet, and the data it refers to are Phase 2.

Financial position

Cash, cash equivalents and marketable securities were $2,127.9 million on September 30, 2026, compared with $1,945.4 million on December 31, 2025. The quarterly report filed the same day gives cash and cash equivalents of $508.9 million at September 30, 2026, and short-term investments of $1.62 billion at that date.

The annual report filed February 19, 2026 reported net income of $682.6 million for 2025, compared with a net loss of $363.3 million for 2024. Cash from operations was $711.2 million in 2025, compared with negative $107.7 million in 2024.

What comes next

PTC expects to complete the ST-920 BLA in the fourth quarter of 2026 and to hold a Type C meeting on votoplam in the first quarter of 2027. It also started a Phase 2a study of PTC844 and expects PTC303 to enter the clinic in 2027.

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