PTC Therapeutics (PTCT) Leerink Global Healthcare Conference 2026 summary

Recent accomplishments and financial performance

• Achieved initial global approvals and strong launch of Sephience for PKU, generating over $111 million in revenue in the first 5.5 months of 2025, exceeding guidance.

• Ended 2025 with approximately $1.95 billion in cash, with expenses below guidance, positioning for continued commercial and R&D investment.

• Projecting continued revenue growth in 2026, with commercial patients expected in 20-30 countries and potential to reach cash flow break-even.

Sephience launch dynamics and patient uptake

• Broad uptake across all patient segments, including therapy-naive, previously treated, and adults previously lost to follow-up.

• Early launch momentum driven more by new patients than by switches from existing therapies.

• Over 80% of centers of excellence had prescribed Sephience by end of Q4, indicating wide center participation.

• High prescription refill rates and adherence, with low single-digit discontinuations, mainly due to taste/texture in adolescents.

Clinical outcomes and payer landscape

• Real-world response rates and benefits align with clinical trial data, including improved quality of life and cognitive function.

• Clinical trials showed a 70-75% response rate for Phe lowering; 84% of patients achieved target Phe levels, enabling increased protein intake.

• Payer coverage is broad, with no step edits required; response defined by Phe lowering, increased protein intake, or overall medical benefit.