PTC(US:PTC) Prnewswireยท2024-11-26 21:30Core Insights - PTC Therapeutics announced that the Phase 2 CardinALS study did not meet its primary endpoint of slowing disease progression in ALS patients, with a p-value of 0.52 indicating lack of statistical significance [1][2] - Despite showing a modest numerical benefit and correlation with a biomarker (NfL), the study's secondary efficacy endpoints also did not achieve significance [1][2] - Utreloxastat was found to be safe and well tolerated, but due to the lack of efficacy, further development is not planned [2] Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on developing and commercializing medicines for rare disorders, aiming to provide access to treatments for patients with limited options [3] - The company's strategy leverages scientific expertise and global infrastructure to maximize value for stakeholders [3]