PTC Therapeutics (PTCT) investor relations material

Commercial performance and product launch

• Achieved $273 million in total Q1 2026 revenue, with $226 million from product sales, driven by a strong SEPHIENCE launch for PKU, generating $125 million globally.

• SEPHIENCE approved in the U.S., Europe, and Japan within six months, with launches in multiple countries and plans to reach up to 30 by year-end.

• Over 1,200 patients on SEPHIENCE in Q1, with broad penetration into 90% of U.S. centers of excellence and all key patient segments, including therapy-naive and previously lost-to-follow-up patients.

• Early discontinuation rates are in the low double digits, with strong adherence, especially among challenging patient groups.

• Pricing in Japan and Germany is on par with the U.S., with a fixed 10-year price in Japan and ongoing negotiations in Germany.

Market dynamics and growth outlook

• The addressable PKU market is estimated at 58,000 globally (17,000 in the U.S.), with significant unmet need and opportunity for further patient penetration.

• Launch momentum is described as sustained, not a short-term bolus, with continued strong patient adds and no signs of plateauing.

• About 30% of current SEPHIENCE patients are treatment-naive, with more tried-and-failed patients than current therapy switchers, indicating further growth potential.

• Time from prescription to treatment averages two to three weeks, supported by broad payer coverage and flexible policies.

• Quality-of-life improvements, including diet liberalization and cognitive benefits, are widely reported and drive patient and physician enthusiasm.

International expansion and pricing

• Leveraged established infrastructure in Europe and LATAM for rapid launches, using early access programs to build experience and revenue ahead of formal reimbursement.

• In Germany, favorable initial assessments and head-to-head data support pricing negotiations, with some discount expected.

• Early access programs in Europe allow for initial sales and physician engagement, with larger patient uptake expected post-reimbursement.