
Financial Performance - Unaudited total revenue for 2024 was approximately $814 million, exceeding guidance[1] - Unaudited net product revenue for 2024 was approximately $601 million, driven by the DMD franchise with $340 million from Translarna and $207 million from Emflaza[3] - Cash balance as of December 31, 2024, was approximately $1.1 billion, with an additional $1.0 billion from the PTC518 collaboration agreement with Novartis[3] - PTC anticipates approximately $211 million in royalty revenue from Evrysdi for the full year 2024[8] Future Projections - PTC expects total revenues for 2025 to be between $600 million and $800 million, including in-line products and potential new product launches[8] - Anticipated GAAP R&D and SG&A expenses for 2025 are projected to be between $805 million and $835 million[8] Regulatory Approvals - Four regulatory approval applications were submitted to the FDA in 2024, including sepiapterin for PKU with an FDA action date of July 29, 2025[3] - CHMP opinion on sepiapterin MAA is expected in Q2 2025[8] Collaborations and Milestones - PTC signed a global license and collaboration agreement with Novartis for PTC518, with potential milestone payments up to $1.9 billion[3] - Results from the PIVOT-HD Phase 2 study of PTC518 are expected in Q2 2025[8]