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PTC Therapeutics Reports Significant Milestones and Financial Results for 2025

In a recent announcement, PTC Therapeutics, Inc. (NASDAQ: PTCT) showcased its remarkable financial performance for the fourth quarter and full year ending December 31, 2025. This report reflects the ongoing success of the company's innovative products, notably the Sephience™ (sepiapterin), which has demonstrated tremendous uptake since its launch in the second half of 2025.

Financial Highlights

PTC Therapeutics achieved full-year product and royalty revenue of $831 million, surpassing its guidance expectations. The fourth quarter reported revenue totaled $164.7 million, with Sephience contributing $92 million to these figures. Breaking down the Sephience revenue further shows $81 million from the U.S. market and $11 million from international sales, indicating a strong global appetite for the product.

In total, Sephience generated $111 million in net revenue for 2025 since its launch, solidifying its position as a key product in PTC’s portfolio. As of December 31, 2025, 946 patients were benefiting from commercial therapy with Sephience, highlighting its crucial role in the treatment landscape for patients.

Expansion and Approvals

One of the noteworthy highlights of PTC’s report was the recent approvals for Sephience in Japan (December 2025) and Brazil (February 2026). The company's strategic plans suggest that the global footprint of Sephience is expected to expand to 20 to 30 countries by the end of 2026. This rapid expansion confirms PTC’s commitment to increasing access to innovative treatments worldwide.

Moreover, PTC Therapeutics completed the sale of its remaining Evrysdi® (risdiplam) royalties to Royalty Pharma for $240 million upfront, with the potential for additional sales-based milestones. Notably, PTC is poised to receive a significant milestone payment from Roche based on Evrysdi sales targets, demonstrating continued profitability from its collaboration efforts.

Research and Development Progress

The company held essential meetings with the FDA during the fourth quarter to discuss its ongoing projects. PTC has made strides in its Huntington’s disease program, votoplam, aligning on the design of a critical Phase 3 trial named INVEST-HD, which is set to begin in the first half of 2026. Additionally, discussions regarding the Friedreich's ataxia program indicated a pathway towards accelerated approval, underscoring PTC’s focus on addressing significant unmet medical needs.

Financial Outlook for 2026

Looking ahead, PTC has projected total product revenue of $700 to $800 million for 2026, representing an estimated 19 to 36% increase from 2025, predominantly driven by Sephience. The expected GAAP RD and SGA expenses are anticipated to be between $775 to $815 million. This forecast reflects PTC's strategy to maintain robust investment in R&D while balancing operational expenditures effectively.

As of December 31, 2025, PTC reported substantial cash reserves of $1.95 billion, providing a solid financial backbone for ongoing research and expansion efforts. The cash plus marketable securities position reflects a substantial increase from $1.14 billion at the end of 2024, ensuring PTC’s capability to navigate upcoming industry challenges while pursuing innovative pathways.

Conclusion

PTC Therapeutics continues to demonstrate impressive financial performance and commitment to growing its impact in the biotechnology field. With initiatives aimed at launching critical therapies globally and addressing rare disorders, the future looks promising for PTC and its stakeholders. Investors and stakeholders can look forward to further updates as the company advances its pipeline and expands its global presence.

For more information about PTC Therapeutics and its innovative treatment options, visit PTC's Official Website.