PTC Therapeutics (PTCT) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

2024 achievements and financial performance

• Achieved all clinical and regulatory milestones, including four FDA approval applications and major U.S. regulatory submissions for AADC gene therapy, sepiapterin for PKU, Translarna for nmDMD, and vatiquinone for Friedreich's Ataxia.

• Kebbilidi gene therapy approved as first direct-to-brain gene therapy by FDA; other applications under review.

• 2024 unaudited revenue reached $814 million, exceeding guidance, driven by strong commercial execution and inline products such as Translarna, Emflaza, Upstaza, Tegsedi, Waylivra, and Evrysdi.

• Closed 2024 with over $1.1 billion in cash, supporting commercial launches, R&D, and business development.

• Strong cash position enables reaching cashflow breakeven without additional capital and supports commercial launches and R&D innovation.

2025 outlook and strategic plans

• Anticipates four U.S. commercial launches within 12 months, including sepiapterin, vatiquinone, and Translarna.

• Initial 2025 revenue guidance set at $600–$800 million, reflecting pending regulatory outcomes and potential new product launches.

• OpEx guidance for 2025 is $730–$760 million, including launch and clinical study costs.

• Vision for 2025 and beyond includes achieving cash flow break even and a path to $2 billion topline revenue.

• Business development and strategic partnerships to accelerate revenue growth.

Pipeline and regulatory milestones

• Multiple value-creating milestones expected in 2025, including FDA and CHMP decisions for sepiapterin (PKU), vatiquinone (FA), and Translarna (DMD), as well as pivotal study readouts for PTC518 in Huntington's disease.

• Full 12-month PIVOT-HD results for PTC518 expected in Q2 2025, supporting regulatory discussions.

• Vatiquinone poised to be the first approved treatment for children with Friedreich's Ataxia, with NDA acceptance expected in February 2025 and potential approval by August 2025.

• Sepiapterin demonstrated significant efficacy in PKU, with 84% of patients reaching target Phe levels and evidence of diet liberalization.

• Launch preparations for sepiapterin and vatiquinone are underway, with strong engagement from patient and physician communities.