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PTC Therapeutics Provides Corporate Update and Reports First Quarter 2025 Financial Results
– Strong revenue performance of $190 million – – Positive CHMP opinion for Sephience™ (sepiapterin) received in April 2025, NDA review remains on track for
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"– Strong revenue performance of $190 million – \n– Positive CHMP opinion for Sephience™ (sepiapterin) received in April 2025, NDA review remains on track for July 29, 2025 PDUFA date – \n– Global Sephience launch activities progressing well – \n– Strong cash position of over $2.0 billion as of March 31, 2025 –\nWARREN, N.J., May 6, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ended March 31, 2025. \n\n\"Following a year of outstanding execution across every part of the Company, we have built on this positive momentum with solid revenue performance in the first quarter, allowing us to narrow our full-year revenue guidance,\" said Matthew B. Klein, M.D., Chief Executive Officer. \"Our strong cash balance supports all of our planned commercial and R&D activities and provides the ability to reach cashflow breakeven without raising additional capital. The positive CHMP opinion for Sephience kickstarts our anticipated global launch for what we see as a significant revenue opportunity.\"\nKey Corporate Updates: \nFirst quarter 2025 total net product and royalty revenue of $190 million.First quarter 2025 revenue for the DMD franchise of $134 million, including net product revenue for Translarna™ of $86 million and for Emflaza® of $48 million.Key Clinical and Regulatory Milestones: \nSephiencePositive CHMP opinion on Sephience MAA for adult and pediatric PKU patients received on April 25, 2025; the opinion includes a broad label inclusive of all ages and disease severities. PTC expects the EC to adopt the opinion in approximately two monthsNDA currently under review by the FDA, with a target regulatory action date of July 29, 2025Japan regulatory submission under review and a decision is expected in Q4 2025VatiquinoneNDA for pediatric and adult patients with Friedreich's ataxia accepted and granted Priority Review by the FDA, with a target regulatory action date of August 19, 2025TranslarnaNDA currently under review by the FDAPTC518Phase 2 PIVOT-HD study results announced on May 5, 2025:Met primary endpoint of dose-dependent blood HTT lowering at Week 12Dose-dependent trends of clinical benefit in Stage 2 patients at Month 12Signals of dose-dependent clinical benefit relative to matched natural history cohort and dose-dependent lowering of NfL i...