PTC Therapeutics (PTCT) investor relations material

Commercial launch and product performance

• Sephience, an oral therapy for PKU, received U.S. and EU approval in 2025 and has shown strong early commercial momentum, with 341 patients on therapy in the first quarter and continued growth into Q4.

• The product addresses a significant unmet need in PKU, offering a dual mechanism of action that benefits both classical and non-classical patients, including those not responsive to existing therapies.

• Early adoption spans all patient segments, including therapy-naive individuals and those previously considered lost to follow-up, with rapid onboarding averaging two to four weeks from prescription to drug delivery.

• Social media and patient testimonials have played a key role in driving awareness and enthusiasm, highlighting transformational impacts such as diet liberalization and improved quality of life.

• The company has expanded manufacturing capacity to meet anticipated global demand, adding additional API and drug product manufacturers.

Global expansion and market access

• The launch was nearly simultaneous in the U.S. and Europe, with infrastructure and teams prepared for upcoming launches in Japan and Brazil.

• Pricing strategies are carefully managed to maintain a narrow corridor across markets, with early access programs supporting patient uptake outside Germany.

• Market access in Europe and other regions involves navigating reimbursement, guidelines, and payer negotiations, leveraging strong clinical data on Phe lowering and diet liberalization.

• Patient engagement teams work globally to foster disease awareness and support, utilizing well-organized patient communities and social media.

Clinical data and competitive landscape

• Sephience demonstrated 70% greater Phe lowering compared to BH4 in head-to-head studies, with 84% of trial patients achieving target Phe levels and 97% able to liberalize their diet.

• The therapy is effective across all age groups, with documented benefits in executive function, cognitive function, and mood.

• New therapies targeting renal transporters are in development but are expected to be used in combination with Sephience; broad market penetration is anticipated before these competitors launch.