PTC Therapeutics (PTCT) Bank of America Global Healthcare Conference 2026 summary

Commercial performance and product launch

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

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

• Penetrated over 90% of U.S. centers of excellence, with broad uptake across all 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.

• Over 1,200 patients on SEPHIENCE in Q1, with expectations for continued growth and potential blockbuster status.

Market dynamics and physician feedback

• No evidence of a launch bolus; instead, observed an acceleration phase followed by sustained momentum in patient adds.

• Claims data are not considered reliable due to the closed specialty pharmacy network; internal signals indicate ongoing strong demand.

• Physicians report meaningful improvements in quality of life, including diet liberalization, cognition, and mood.

• About 30% of patients are treatment-naive, with a significant portion being tried-and-failed or previously untreated.

• Average time from prescription to treatment remains two to three weeks, supported by broad and flexible insurance coverage.

International expansion and pricing

• Leveraged established infrastructure in Europe and LATAM for SEPHIENCE launch, using early access programs while formal pricing is negotiated.

• Achieved pricing in Japan and Germany on par with the U.S., with a fixed price in Japan for 10 years.

• 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 revenue and experience-building before full reimbursement.