PTC Therapeutics (PTCT) Corporate presentation summary

Strategic focus and execution in 2025

• Achieved US, EU, and Japan approvals for Sephience and initiated a strong global launch.

• Exceeded 2025 revenue guidance with $831M in total revenue, including $587M in product revenue.

• Advanced early-stage R&D programs and managed operating expenses effectively.

Sephience launch and performance

• Generated $125M in Q1 2026 global revenue for Sephience, with $112M from the US and $13M ex-US.

• 1,244 patients on commercial therapy worldwide as of March 31, 2026.

• Anticipates commercial patients in up to 30 countries by year-end 2026.

Product differentiation and market potential

• Sephience’s dual mechanism of action supports broad uptake, significantly increasing intracellular BH4 levels.

• Demonstrated 70.3% greater reduction in blood Phe compared to BH4 in clinical studies.

• Provided meaningful diet liberalization, cognitive, and quality of life benefits in extension studies.

• Positioned to penetrate all key PKU market segments, with a total addressable market of 17,000 US patients.