PTC(US:PTC) Prnewswireยท2025-01-13 14:00Core Insights - PTC Therapeutics reported unaudited total revenue of approximately $814 million for 2024, exceeding guidance [1][5][11] - The company submitted four regulatory approval applications to the FDA in 2024, with significant upcoming decisions expected in 2025 [1][5][11] - PTC is preparing for the global launch of sepiapterin for PKU, targeting a market opportunity of $1 billion, with regulatory opinions expected in 2025 [1][5] - A collaboration agreement with Novartis for the PTC518 program has been finalized, providing substantial upfront proceeds and potential milestone payments [1][5][11] Financial Performance - The unaudited net product revenue for 2024 was approximately $601 million, with strong contributions from the DMD franchise, including $340 million from Translarna and $207 million from Emflaza [5][11] - The cash balance as of December 31, 2024, was approximately $1.1 billion, bolstered by $1.0 billion from the Novartis collaboration [5][11] - PTC anticipates total revenues for 2025 to range between $600 million and $800 million, including in-line products and potential new launches [12] Regulatory and Clinical Developments - PTC's regulatory submissions include Kebilidi, which was approved in November 2024, and sepiapterin, with an FDA action date set for July 29, 2025 [5][11] - The company expects results from the PIVOT-HD Phase 2 study of PTC518 in Q2 2025, with NDA acceptance for vatiquinone anticipated in Q1 2025 [11] - The CHMP opinion on sepiapterin's MAA is expected in Q2 2025, indicating ongoing regulatory engagement [5][11] Strategic Collaborations - The collaboration with Novartis includes a $1.0 billion upfront payment, potential for up to $1.9 billion in milestones, and a 40% profit share on U.S. sales [5][11] - Novartis will take over global development and commercialization responsibilities for PTC518 following the completion of the placebo-controlled portion of the PIVOT-HD study [5][11]