Business
PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2025 Financial Results
– European and FDA approval of Sephience™ (sepiapterin) with broad labeling for PKU – – Global launch underway in Europe and U.S. – – Total Q2 Revenue of
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"– European and FDA approval of Sephience™ (sepiapterin) with broad labeling for PKU – \n– Global launch underway in Europe and U.S. –\n– Total Q2 Revenue of $179M –\nWARREN, N.J., Aug. 7, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ended June 30, 2025. \n\n\"We had another strong quarter highlighted by the first approvals of Sephience for the treatment of children and adults with PKU,\" said Matthew B. Klein, M.D., Chief Executive Officer. \"We have initiated the global launch and expect Sephience to be the foundational product for PTC's future growth and path to profitability.\"\nKey Corporate Updates: \nSecond quarter 2025 total net product, collaboration and royalty revenue of $179 millionSecond quarter 2025 revenue for the DMD franchise of $96 million, including net product revenue for Translarna™ of $59 million and for Emflaza® of $36 millionInitiated global launch of Sephience™ in the U.S. and Germany as well as in other countries through early access and named patient programsEntered into agreement to purchase the Sephience annual percentage-based global net sales obligation owed to former Censa shareholders in exchange for an upfront payment of $225 million and future sales milestone paymentsKey Clinical and Regulatory Milestones: \nSephience™ (sepiapterin)Marketing authorization granted by the EC on June 19, 2025 with broad label inclusive of all disease subtypes and all ages FDA approval on July 28, 2025 with broad label inclusive of all disease subtypes and all ages, from 1 month of age upwardsJapan NDA review is ongoing with decision expected in Q4 2025NDA reviews for vatiquinone (Friedreich's ataxia) and Translarna (nonsense mutation DMD) are ongoing, with regulatory action date of August 19, 2025 for vatiquinoneIn May 2025, reported positive Phase 2 PIVOT-HD study results for votoplam (PTC518) in Huntington's Disease patients. PTC continues to collaborate with Novartis on next steps and aims to meet with FDA in Q4 2025 to discuss Phase 3 clinical trial design and potential accelerated approval pathway.Second Quarter 2025 Financial Highlights:\nTotal revenues were $178.9 million for the second quarter of 2025, compared to $186.7 million for the second quarter of 2024.Total revenue includes net product revenue across the c...