PTC Therapeutics (PTCT) investor relations material

Launch performance and market dynamics

• Sephience launch shows strong momentum, with broad uptake across over 90% of U.S. centers of excellence within two and a half quarters, driven by its dual mechanism and benefits for a wide PKU patient spectrum.

• Early adoption is primarily among therapy-naive and previously failed therapy patients, rather than expected switchers from existing treatments.

• Prescribing decisions are influenced by multidisciplinary teams, with a focus on untreated patients due to significant unmet need.

• Positive clinical feedback, including rapid phenylalanine reduction in severe cases, is fueling broader adoption.

• Expansion is ongoing into non-center-of-excellence sites and late-adopting centers, with a sustainable growth pace anticipated.

International expansion and pricing

• Commercial infrastructure enables simultaneous launches in the U.S., Europe, and Japan, targeting up to 30 countries by year-end.

• Japanese and German list prices are on par with the U.S., with Japan’s price fixed for 10 years due to orphan drug status.

• European market entry leverages named patient programs before formal pricing and reimbursement, with head-to-head clinical data supporting premium positioning.

• Ex-U.S. markets are expected to drive accelerated growth in the second half of the year as pricing and reimbursement are finalized.

Financial guidance and outlook

• Product revenue guidance was raised following a strong quarter, with $226 million in product revenue and confidence in long-term growth for Sephience.

• Guidance adjustments will be considered as more data emerges, particularly regarding U.S. growth and ex-U.S. acceleration.

• The company maintains a robust balance sheet near $2 billion, supporting ongoing R&D and potential strategic transactions.