PTC Therapeutics (PTCT) Goldman Sachs 46th Annual Global Healthcare Conference summary

Strategic and financial overview

• Achieved four FDA NDA submissions in 2024, with one approval and three pending, including Sepiapterin, expected to be approved within six weeks.

• Ended Q1 with over $2 billion in cash, supporting planned launches, R&D advancement, and business development.

• Strong revenue performance and effective expense management have positioned the company for future growth.

• Strategic focus includes leveraging cash for internal development and external business development opportunities.

• Goal to reach $2 billion in top-line revenue, with PKU and other assets as key drivers.

Commercial portfolio and legacy products

• Translarna in Europe continues to generate revenue despite loss of marketing authorization, with 25%-30% of prior European revenue expected to be maintained.

• More than half of European countries have opted to continue commercializing Translarna under Article 117.

• No near-term replacement for Translarna is anticipated due to lack of alternative therapies and gene therapy limitations.

• Revenue durability for Translarna in Europe is expected to persist for the foreseeable future.

Sepiapterin and PKU market opportunity

• Sepiapterin is positioned as a highly differentiated therapy for PKU, targeting a significant unmet need among 85%-90% of U.S. patients.

• Plans to price Sepiapterin at a premium to Palynziq, supported by payer feedback indicating minimal access barriers.

• Surveys show over 80% of physicians are likely to switch patients to Sepiapterin, with over 50% aiming to try all PKU patients on the drug.

• Commercial launch in Europe is expected before the end of June, with Germany as the initial market, closely followed by the U.S.

• Early access programs and pent-up demand are expected to drive a strong initial launch, with rapid patient uptake anticipated.