PTC Therapeutics (PTCT) investor relations material

Strategic priorities and commercial performance

• Achieved record Q1 product revenue of $226 million and total revenue of $273 million, driven by SEPHIENCE launch.

• SEPHIENCE launched in Japan, with global expansion and early access programs in Europe and Brazil.

• SEPHIENCE addresses the full spectrum of PKU patients, showing broad uptake across age and treatment experience.

• Over 90% of U.S. centers of excellence have prescribed SEPHIENCE, with expectations to reach 100%.

• Global market opportunity for SEPHIENCE estimated at over $2 billion, with strong U.S. and ex-U.S. growth anticipated.

R&D pipeline and regulatory updates

• Initiating the PROVE-FA trial for vatiquinone in Friedreich's ataxia, using an open-label natural history comparator.

• Phase II-A study of PTC844 (DHODH compound) and phase I study of PTC612 (NLRP3 compound) underway.

• Expecting to announce development candidates for MSH3 splicing (Huntington's, myotonic dystrophy) and ferroptosis platform (Parkinson's, MSA).

• Votoplam phase III trial for Huntington's disease is enrolling, managed by Novartis, with significant milestone and royalty potential.

Market dynamics and patient impact

• SEPHIENCE launch driven by therapy-naive and previously untreated PKU patients, not just switches.

• Real-world feedback highlights diet liberalization and cognitive/mood improvements for patients.

• Early access programs in Europe allow patient treatment and engagement with key opinion leaders during reimbursement negotiations.

• Competitive landscape includes kidney-directed therapies, with potential for combination regimens.