PTC Therapeutics (PTCT) investor relations material

Scientific and market opportunity overview

• PKU is a rare disease affecting 17,000 in the US and 58,000 globally in target markets, with significant unmet need due to limitations of existing therapies.

• Sepiapterin offers once-daily oral therapy with strong safety and tolerability, enabling patients to liberalize their diet and improve quality of life.

• Clinical studies show 97% of patients increased protein intake, and 70% reached recommended daily allowance while maintaining control of phenylalanine.

• Early real-world data confirm benefits across all age groups and disease severities, including classic PKU.

• Broad addressable market as data demonstrate benefit for the full spectrum of PKU patients.

Launch performance and adoption trends

• Over 1,100 patient starts and $110 million in revenue in the first 5.5 months, primarily from the US, with early contributions from Germany.

• Global launch initiated in late Q3 with approvals in the US, Europe, and Japan; launches in Japan and Brazil expected in early Q2.

• Early adoption spans all ages and disease severities, with most uptake among patients who failed prior therapies and therapy-naive adults.

• 80% of centers of excellence have submitted multiple start forms, indicating broad and rapid uptake.

• Focus is shifting to deeper penetration in existing centers and international expansion, with commercial patients expected in 20-30 countries by end of 2026.

Adherence, discontinuation, and guidance

• Discontinuation rates remain in the low single digits, with most patients staying on therapy due to perceived benefit.

• Response rates in studies are 70-75%, with real-world data mirroring clinical trial outcomes.

• Revenue guidance for 2024 is $700-800 million, with sepiapterin as the main growth driver; mature products expected to see continued erosion.

• Global opportunity for sepiapterin is projected at $2 billion+ worldwide.