PTC Therapeutics (PTCT) investor relations material

Strategic evolution and financial position

• Transitioned to a focused, execution-oriented company with a strong cash position exceeding $1.9 billion at Q2 close.

• Recent partnership with Novartis for Huntington’s program brought $1 billion upfront, up to $1.9 billion in milestones, and a 40% U.S. profit share.

• Confident in reaching cash flow breakeven and profitability, primarily driven by the PKU program.

• Business development will be opportunistic, with a disciplined approach to value creation.

SEPHIENCE launch and PKU opportunity

• SEPHIENCE approved in the U.S. and Europe, with a strong commercial launch targeting 104 centers of excellence.

• Early launch shows uptake across all patient segments, including therapy-naive and severe cases.

• Data indicate a 70% greater reduction in phenylalanine compared to BH4 in head-to-head studies.

• Payers are familiar with PKU, and few barriers to access are anticipated due to objective efficacy measures.

• SEPHIENCE alone is expected to drive the company to profitability, with U.S. sales potential of at least $1 billion.

Huntington’s disease program and Novartis partnership

• Phase II data for PTC518 showed dose-dependent lowering of mutant huntingtin protein and early efficacy signals, especially in stage 2 patients.

• Stage 2 patients represent a larger market opportunity than stage 3, with potential to expand to earlier-stage patients.

• Novartis partnership covers all phase III costs and positions the program for broad market access.

• Next FDA meeting in Q4 aims to align on trial design and accelerated approval pathway.