PTC Therapeutics (PTCT) FY Earnings Call Presentation

Financial Performance & Outlook - PTC achieved total revenue of $807 million in 2024[14] - PTC has a strong cash position of over $2 billion, enabling future revenue growth and R&D innovation[15] - PTC aims to reach cash flow break-even without additional capital[16] - PTC's vision includes a path to $2 billion in topline revenue[53] Key Product Programs & Milestones - Four U S regulatory approval applications were submitted in 2024[11] - AADC Gene Therapy BLA was approved[12] - Sephience (sepiapterin) in PKU has a potential revenue opportunity greater than $1 billion[28] - Vatiquinone for Friedreich's Ataxia (FA) has an NDA filing accepted with Priority Review, with an approval decision expected August 19, 2025[32] - PTC518 for Huntington's Disease (HD) has a development and commercialization collaboration with Novartis, including $1 billion upfront payment and up to $19 billion in milestones[45] Research & Development - PTC has innovative research platforms, including a validated splicing platform[48] - PTC has inflammation & ferroptosis programs targeting CNS and non-CNS disorders[50]