PTC THERAPEUTICS, INC. Q3 FY2024 earnings call

Commercial Performance

• Achieved total revenue of $197 million in Q3, with $124 million from the DMD franchise, including $52 million in Emflaza revenue.

Pipeline Execution

• Submitted two NDAs to the FDA in Q3: one for sepiapterin (accepted for filing, FDA action date July 29, 2025) and one for Translarna (under review).
• PTC518 for Huntington's disease received Fast Track Designation from the FDA and requested Type C meetings with the FDA.
• Utreloxastat program on track for topline results from the CardinALS trial in the fourth quarter.
• AADC Gene Therapy BLA has an FDA action date of November 13.

Guidance Update

• Raised 2024 total revenue guidance to $750 million to $800 million.

In the third quarter, total revenue was $197 million. The DMD franchise contributed $124 million. Translarna net product revenue was $72 million, and Emflaza net product revenue was $52 million. Evrysdi generated $61 million in royalty revenue for PTC.

Forward-Looking Statements

• Raised 2024 total revenue guidance to a range of $750 million to $800 million.

Risks

• Regulatory uncertainties regarding NDA reviews and approvals for various programs.
• Potential impact of European Commission decisions on Translarna sales in Europe.

Q: Thanks for the update on the Type C meeting in Huntingtin's. Do you have a sense for when you could update the community on any clarity that might come out of the meeting?

A: We expect to have meetings with FDA in December of this year. As soon as we have clarity, we'll share it with the broader community.

Q: Given the FDA resubmission for Translarna, can you talk about how you think the FDA might weigh the STRIDE registry data and Study 041 that were submitted?

A: The NDA resubmission for Translarna is based on significant benefit in Study 041 and confirmatory evidence from the STRIDE registry, which shows significant benefits over several years.

Q: Ahead of the CardinALS top line in 4Q, do you plan to share like baseline characteristics, maybe like time since diagnosis or can you comment on maybe what percent of patients are going into the OLE?

A: Typically, with top line results, we'll give key baseline characteristics and results. We've seen excellent continuation of patients rolling into the OLE once they finish the CardinALS.

Q: For Translarna, what percent of the current ex-US sales would not be affected by any potential European Commission decision?

A: Roughly 46% of Translarna revenue was from Europe itself, which would be at risk if the EC adopts a negative opinion, but other regions have exclusivity out to 2029.

Q: On sepiapterin, can you talk a little bit more about the reactions from providers, patients and payers to the Lancet data?

A: Physicians are very excited about sepiapterin's differentiated efficacy, payers see a premium pricing strategy due to high unmet need and rapid efficacy measurement, and patients are optimistic about diet liberalization.

Q: For vatiquinone, what's outstanding for the submission and have the FDA reviewed the statistical analysis plan?

A: We're finishing up summary reports from long-term extension studies. We had pre-NDA interaction with the agency in October, and they've seen some data but no additional feedback expected until after NDA submission.