PTC Therapeutics (PTCT) TD Cowen 46th Annual Health Care Conference summary

Key business highlights

• Achieved first approvals and global launch for Sephience in PKU, with $111M in revenue in 5.5 months and rapid uptake across patient subtypes and geographies.

• Exceeded revenue guidance for 2025, maintained OpEx below guidance, and ended the year with $1.95B in cash, supporting flexibility and resilience.

• Expecting continued strong Sephience momentum in 2026, targeting commercial patients in 20-30 countries and aiming for cash flow break even by year-end.

• Mature products are expected to face revenue pressure, especially from generics, but Sephience will drive the majority of 2026 guidance.

• No anticipated increase in OpEx; infrastructure is leveraged for global launch, with disciplined expense management to support profitability.

Sephience launch and market dynamics

• Early launch success driven by broad uptake across all PKU patient subtypes, including therapy-naive and previously treated patients.

• Penetrated over 80% of U.S. Centers of Excellence by end of 2025, an atypically rapid adoption rate.

• High refill rates and low discontinuation rates observed, with strong patient and physician motivation to maintain therapy.

• No payer step edits required due to compelling head-to-head data versus BH4, supporting broad access.

• Global expansion underway, with launches in Europe, Japan, Brazil, and other regions prioritized for 2026.

R&D and pipeline progress

• Advanced multiple programs from the RNA splicing platform and inflammation pipeline, with new development candidates expected soon.

• Votoplam for Huntington's disease showed dose-dependent Huntingtin lowering and early clinical effects; phase III trial design aligned with FDA, with potential for accelerated approval.

• Vatiquinone for Friedreich's ataxia progressing toward an open-label study using natural history database matching, targeting significant unmet need in pediatric and adult populations.

• Early-stage programs in splicing and inflammation platforms highlighted as future growth drivers, with potential for strategic partnerships.