PTC Therapeutics (PTCT) Corporate presentation summary

2025 performance and strategic focus

• Achieved FDA and EMA approvals for Sephience and initiated a strong global launch, exceeding revenue guidance with $831M total revenue and $587M from product sales.

• Focused on driving revenue, managing operating expenses, and advancing early-stage R&D programs.

Sephience launch and market impact

• Sephience generated $92M in Q4 2025 global revenue, with 946 patients on therapy and 1,134 US patient start forms by year-end.

• Demonstrated dual mechanism of action, enabling broad uptake across all PKU patient segments, with a US addressable market of 17,000.

• Provided significant Phe reduction, diet liberalization, cognitive, and quality of life benefits in clinical studies.

• Launched in the US, EU, and Canada in 2025, with plans to expand to Asia and LATAM in 2026.

2026 financial guidance and operational plans

• 2026 product revenue guidance set at $700–800M, representing 19–36% YoY growth, excluding Evrysdi royalties.

• Non-GAAP OpEx guidance is $680–720M, with GAAP R&D and SG&A expenses anticipated at $775–815M.

• Aims to continue Sephience launch momentum, initiate votoplam HD Phase 3 trial, advance early-stage R&D, and move toward cash flow breakeven.