PTC Therapeutics, Inc. reported its financial results for the third quarter and nine months ended September 30, 2024, highlighting a mixed performance in revenue and significant strategic developments. The company recorded total revenues of $196.8 million for Q3 2024, slightly up from $196.6 million in Q3 2023. This increase was driven by a notable rise in royalty revenue, which reached $61.4 million, up 22% from $50.2 million in the prior year, primarily due to higher sales of Evrysdi. However, net product revenue decreased to $135.4 million, down 6% from $144.0 million in Q3 2023, with Emflaza sales declining significantly by 23% to $51.9 million, attributed to the expiration of orphan drug exclusivity in February 2024.

For the nine months ended September 30, 2024, total revenues were $446.2 million, a decrease of 12% from $506.2 million in the same period in 2023. Net product sales outside the U.S. totaled $289.5 million, down from $318.5 million, while U.S. sales also fell to $156.7 million from $187.7 million. Translarna net revenues for the nine months were $246.2 million, down from $280.6 million in 2023.

The company reported a net loss of $106.7 million for Q3 2024, an improvement from a net loss of $133.0 million in Q3 2023. For the nine months, the net loss was $297.4 million, compared to $470.8 million in the prior year. The reduction in losses was attributed to lower operating expenses, which decreased to $251.3 million in Q3 2024 from $314.7 million in Q3 2023, reflecting strategic cost management and a reduction in research and development expenses.

PTC Therapeutics has been actively pursuing regulatory approvals for its products. The FDA accepted the resubmission of the New Drug Application (NDA) for Translarna in October 2024, while the Biologics License Application (BLA) for Upstaza was submitted in March 2024 and accepted for priority review in May 2024, with a target action date of November 13, 2024. The company also submitted a Marketing Authorization Application (MAA) for sepiapterin for PKU, which was validated in May 2024.

In terms of financial position, as of September 30, 2024, PTC Therapeutics had cash and cash equivalents of $526.0 million, down from $594.0 million at the end of 2023. The company’s accumulated deficit increased to approximately $3.58 billion. The company anticipates that cash flows from product sales, along with existing cash and marketable securities, will be sufficient to fund operations for at least the next twelve months.

Strategically, PTC Therapeutics sold its gene therapy manufacturing business in June 2024, which is expected to impact future revenue from manufacturing services. The company also amended its Royalty Purchase Agreement with Royalty Pharma, receiving $241.8 million in cash consideration, which will help bolster its financial resources.