PTC Therapeutics (PTCT) Morgan Stanley 24th Annual Global Healthcare Conference summary

Commercial performance and product launch

• Six marketed products with a focus on rare diseases and a robust R&D pipeline, including two scientific platforms.

• Sephience launch is exceeding expectations, with revenue annualizing above previous PKU therapies and targeting $2B+ in revenue.

• Achieved 100% penetration in original U.S. centers of excellence, with 80% of PKU patients tied to these centers and additional centers identified.

• Discontinuation rates for Sephience are around 20%, better than anticipated, with strong adherence even among difficult-to-treat patients.

• Favorable payer policies and broad reauthorization criteria have supported compliance and access.

Clinical data and patient impact

• Real-world and clinical data show Sephience benefits the full spectrum of PKU patients, including severe and therapy-naive cases.

• Majority of responders achieve normalized phenylalanine levels, with improved diet liberalization and protein intake.

• Presentations by independent KOLs reinforce Sephience’s value proposition.

• Early uptake includes both treatment-experienced and naive patients, with 30% of new starts from the naive segment.

• Physicians are considering Sephience as first-line therapy, including for newborns.

Global expansion and growth outlook

• Rapid global launch with approvals in the U.S., Europe, and Japan within six months; strong early trajectory in Japan.

• Ongoing launches and access programs in Germany, France, Southeastern Europe, Latin America, Middle East, North Africa, and Canada.

• Pricing and reimbursement discussions are underway in major markets; inflection points expected as access is secured.

• U.S. growth expected to be steady, with global growth driven by new market access and patient starts.

• Raised full-year revenue guidance to $850M–$950M, primarily driven by Sephience.