PTC Therapeutics — Third Quarter 2026 Earnings Summary

Financial Performance:

• Total revenue for Q3 2026 was $358 million, compared to $211.0 million in Q3 2025.
• Net product and royalty revenue totaled $357.6 million in Q3 2026, up from $201.7 million in Q3 2025.
• Total net product revenue reached $275.9 million in Q3 2026, representing a 100%+ increase from $131.0 million in Q3 2025.
• Sephience net product revenue was $181.9 million in Q3 2026, showing 20% growth from Q2 2026.
• Translarna net product revenue was $48.5 million in Q3 2026, down from $50.7 million in Q3 2025.
• Emflaza net product revenue was $25.1 million in Q3 2026, down from $35.2 million in Q3 2025.
• Royalty revenue from Roche's Evrysdi sales was $81.7 million in Q3 2026, up from $70.8 million in Q3 2025.
• GAAP R&D expenses were $105.4 million in Q3 2026, compared to $100.2 million in Q3 2025.
• Non-GAAP R&D expenses were $94.9 million in Q3 2026, excluding $10.5 million in stock-based compensation, compared to $91.0 million in Q3 2025.
• GAAP SG&A expenses were $80.1 million in Q3 2026, compared to $84.0 million in Q3 2025.
• Non-GAAP SG&A expenses were $66.7 million in Q3 2026, excluding $13.3 million in stock-based compensation, compared to $73.8 million in Q3 2025.
• Net loss was $51.7 million for Q3 2026, compared to net income of $15.9 million in Q3 2025.
• Acquired in-process research and development expense was $111.0 million in Q3 2026, related to the ST-920 acquisition.
• Cash, cash equivalents, and marketable securities were $2,127.9 million as of September 30, 2026, up from $1,945.4 million on December 31, 2025.
• Total debt was $536.3 million as of September 30, 2026, compared to $286.6 million on December 31, 2025.
• Total deferred revenue was $1.98 million as of September 30, 2026, compared to $2.04 million on December 31, 2025.
• Total stockholders' deficit was $(191.2) million as of September 30, 2026, compared to $(205.3) million on December 31, 2025.

Guidance and Future Outlook:

• Full-year 2026 total revenue guidance increased to $1.29 to $1.32 billion.
• Full-year 2026 product revenue guidance raised to $960 to $990 million, up from the previous $850 to $950 million range.
• GAAP R&D and SG&A expense guidance for 2026 remains $775 to $815 million.
• Non-GAAP R&D and SG&A expense guidance for 2026 remains $680 to $720 million, excluding estimated non-cash stock-based compensation of $95 million.
• Management expects to achieve cash flow breakeven in 2026.

Business Segments and Product Lines:

• Sephience (sepiapterin) generated $182 million in Q3 2026 revenue, including $145 million in the US and $37 million ex-US, up over 20% from Q2 2026.
• Sephience had over 2,000 active patients worldwide as of September 30, 2026.
• ST-920 Fabry disease gene therapy was acquired; BLA submission is expected to be completed in Q4 2026.
• ST-920 is a one-time administered AAV gene therapy with potential for FDA accelerated approval and commercial launch in 2027.
• Votoplam Huntington's disease program is advancing; a Type C meeting to discuss Phase 2 data and potential accelerated approval is scheduled for Q1 2027.
• The global Phase 3 INVEST-HD study for votoplam is actively enrolling with a target of approximately 770 early symptomatic patients.
• Vatiquinone PROVE-FA study to support NDA resubmission for Friedreich's ataxia initiated in Q3 2026, enrolling approximately 120 patients ages 7 to 21.
• Phase 2a study of PTC844, a next-generation DHODH inhibitor, was initiated.
• Phase 1 study of PTC612, an oral NLRP3 inhibitor, continues to advance.
• PTC303, a splicing development candidate targeting MSH3, is expected to enter the clinic in 2027.

Other Key Points:

• The Company 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.
• Shares issued and outstanding as of September 30, 2026, were 83,535,767.
• Translarna Q3 2026 revenue includes a government purchase order from Russia.
• The company reported a loss on extinguishment of debt of $19.3 million for the nine months ended September 30, 2026.