PTC Therapeutics (PTCT) Wells Fargo 21st Annual Healthcare Conference summary

Strategic positioning and financial outlook

• Positioned as a diversified rare disease company with strong current and future revenue growth potential and over $2 billion in cash reserves.

• Targeting cash flow breakeven this year, with disciplined OpEx and a focus on sustainable profitability.

• Business development remains a priority, leveraging financial strength for both internal pipeline and external opportunities.

SUFIANCE (PKU) launch and market dynamics

• Achieved broad penetration in all U.S. centers of excellence and key global markets within a year of launch.

• Early adoption driven by therapy-naive and previously untreated patients, not just KUVAN switches.

• Over 2,000 U.S. prescription start forms, with significant runway remaining among 17,000 U.S. patients.

• Persistence rate stabilizing with a 20% discontinuation rate, reflecting strong adherence, especially among severe patients.

International expansion and pricing

• Ex-U.S. growth expected to accelerate in 2026–2027 as pricing and reimbursement are secured in more countries.

• Japan and Germany are key early markets, with Japan showing strong uptake and Germany awaiting final price agreement.

• Name patient programs and judicialization processes are being used in other regions, with launches planned in Brazil, Russia, and the Middle East.

• European launch sequence is strategically managed to maintain pricing corridors.