PTC THERAPEUTICS, INC. Q1 FY2025 earnings call

Revenue Performance - Achieved $190 million in Q1 revenue, narrowing 2025 full-year guidance to $650 million to $800 million. ### SUFIANCE - Positive CHMP opinion on marketing authorization with broad label expected, preparing for European launch with priority on Germany; FDA NDA review progressing well with no impact from recent FDA changes, expecting over $1 billion in revenue. ### PTC518 - Positive top line results for PIVOT HT Phase 2 study, showing dose-dependent HTT lowering, safety, and early clinical effect signals. ### Other Approvals - Vatiquinone NDA in priority review at typical cadence, Translarna NDA receiving information requests and clinical site inspections. ### Cash Position - Closed Q1 with over $2 billion in cash, enabling support for commercial and R&D activities.

In the first quarter, total products and royalty revenue was $190 million. The DMD franchise contributed $134 million, with Translarna net product revenue at $86 million and Emflaza net product revenue at $48 million. Roche's first quarter global revenue for Evrysdi was approximately $470 million, resulting in a royalty revenue of $36 million for PTC. The DMD franchise's revenue of $134 million made up about 70.53% of the total Q1 revenue of $190 million.

Revenue - Narrowed 2025 full-year revenue guidance to $650 million to $800 million, pending regulatory actions and Emflaza performance. ### SUFIANCE - Anticipates over $1 billion in revenue from SUFIANCE. ### Approvals - Vatiquinone NDA in priority review, Translarna NDA under review with information requests.

Macro Uncertainties - Company believes insulated from global macro uncertainties. ### FDA Changes - No observed impact on SUFIANCE NDA or other approval applications from recent FDA changes, but macro uncertainties exist.

Q: On SUFIANCE, comments on FDA review progress and patient inbound traffic?

A: Matthew Klein mentioned FDA review of SUFIANCE NDA is progressing well with no impact from recent FDA changes, and Eric Pauwels noted patient activity via social media and disease awareness site.

Q: On SUFIANCE launch in Europe and US?

A: Matthew Klein and Eric Pauwels discussed similar launch timelines in Germany and US, with pre-marketing work and enrollment in programs.

Q: On Huntington's disease data?

A: Matthew Klein discussed PIVOT HT Phase 2 results, learning on optimal patient population for efficacy, and accelerated path considerations.

Q: On PKU diet liberalization and reimbursement?

A: Matthew Klein and Eric Pauwels talked about feed tolerance data, differentiated profile, and positive payer reactions.

Q: On Emflaza volumes and defense?

A: Eric Pauwels discussed Emflaza's brand loyalty, minimal impact from generics, and team's white glove service.

Q: On Translarna in ex EU countries?

A: Matthew Klein and Eric Pauwels mentioned continued revenue generation in ex EU countries with no disruption.

Q: On vatiquinone patient breakdown?

A: Matthew Klein discussed pediatric and adult patient breakdown expectations.

Q: On macro factors impact?

A: Matthew Klein stated minimal impact on business from tariffs and macro factors.

Q: On vatiquinone natural history for approval?

A: Matthew Klein discussed comparison with FACOMS natural history database as part of confirmatory evidence.