PTC Therapeutics Reports Strong Third Quarter 2025 Financial Performance and Sephience Launch

PTC Therapeutics, Inc. (NASDAQ: PTCT) has announced a significant corporate update along with its financial results for the third quarter ended September 30, 2025. The quarter demonstrates the company's strength with a remarkable start to the global launch of Sephience™, a treatment for Phenylketonuria (PKU), and an overall solid financial performance.

Financial Highlights

The financial results for the third quarter of 2025 revealed total revenue of $211.0 million, marking an increase from $196.8 million in the same quarter of 2024. This revenue not only includes net product revenues from the company's portfolio but also highlights the impressive early uptake of Sephience, which yielded $19.6 million in revenue. Out of this, $14.4 million was generated in the US market, with a total of 521 patient start forms received from 141 unique prescribers.

Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, expressed his enthusiasm about the company’s performance, stating, “We achieved an outstanding quarter, highlighted by the strong start to the global Sephience launch. The broad initial uptake supports the potential of Sephience to become the standard of care for individuals with PKU.” He also noted that Sephience is set to be the foundation for the company’s sustained growth and success.

In detail, the DMD franchise contributed significantly with sales amounting to $85.9 million, which included net product revenue for Translarna™ at $50.7 million and Emflaza® at $35.2 million. The results not only signify growth in their PKU-related treatments but also a resilient performance in their broader portfolio.

Corporate Developments

PTC Therapeutics is set to hold a Rare Disease (RD) Day on December 2nd in New York, highlighting their commitment to advocacy and education within the rare disease community. Additionally, the company is pursuing ongoing marketing authorization reviews for Sephience in international markets, including Japan, where decisions are anticipated to be made in the fourth quarter of 2025.

The company has planned several meetings with the FDA for various projects, including those for votoplam, targeting Huntington's disease, and vatiquinone for Friedreich's ataxia. Their continued commitment to regulatory compliance and market expansion is evident as they await decisions on pending applications, particularly for Translarna.

Looking Forward

For the full year 2025, PTC Therapeutics has narrowed its total revenue guidance to a range of $750 million to $800 million. This forecast includes revenue from their in-line products and royalties from Evrysdi. Furthermore, the company anticipates research and development (RD) and selling, general and administrative (SGA) expenses to range between $805 million and $835 million.

It's noteworthy that PTC’s cash and cash equivalents rose to $1.68 billion as of September 30, 2025, a significant increase from $1.14 billion at the end of 2024. This growth reflects the company’s healthy cash flow and strategic investments, particularly linked to their Sephience launch.

The management views these non-GAAP financial measures as essential for understanding operational trends and providing investors with better transparency on PTC's growth metrics.

Conclusion

PTC Therapeutics has positioned itself strongly within the biopharmaceutical industry through its innovative approaches and responsive market strategies. The impressive revenue growth and successful launch of Sephience reinforce their ambition to meet the needs of patients living with rare diseases. As they continue to pursue regulatory milestones and expand their market reach, PTC Therapeutics remains a company to watch in the healthcare sector.

For more details on PTC Therapeutics, you can visit their official website or check out their latest news and updates on investor platforms.