PTC Therapeutics (PTCT) investor relations material

Transaction and Acquisition Overview

• Announced acquisition of ST-920, a BLA-stage gene therapy for Fabry disease, for $111 million upfront and up to $100 million in milestone payments, following a competitive bankruptcy auction.

• The acquisition leverages existing global commercial and regulatory infrastructure to accelerate revenue growth and expand the rare disease portfolio.

• Transaction is subject to definitive documentation, bankruptcy court approval, antitrust review, and other customary closing conditions, with expected close in late Q3 or early Q4 2026.

Clinical and Regulatory Highlights

• ST-920 is a one-time AAV gene therapy for Fabry disease, showing a differentiated safety and efficacy profile, including no need for immunosuppression.

• The pivotal STAAR study demonstrated improved renal and cardiac function, sustained enzyme activity for up to 4.5 years, and withdrawal from ERT in all treated patients.

• The therapy has received RMAT, Orphan, and Fast Track designations from FDA and similar designations in Europe and the UK, with a rolling BLA submission underway and CMC package expected in Q4 2026.

• BLA submission for accelerated approval is expected to complete in Q4 2026, with potential commercial launch in 2027.

Market Opportunity and Commercial Strategy

• Estimated 11,000 Fabry patients in the U.S., with similar prevalence globally; many are concentrated in centers of excellence and covered by newborn screening.

• The therapy is positioned for broad use across Fabry subtypes, including those on or off ERT and with varying disease severity.

• Plans to pursue regulatory approval outside the US, leveraging global rare disease infrastructure for rapid integration and launch without significant new build-out.