Interim PIVOT-HD Results Demonstrate Evidence of Favorable CNS Biomarker and Clinical Effects at Month 12 in Huntington's Disease Patients

Core Insights - The FDA has lifted the partial clinical hold on PTC518 based on positive interim results from the Phase 2 PIVOT-HD study in Huntington's disease patients [1][18] - PTC518 demonstrated dose-dependent lowering of mutant huntingtin (mHTT) protein levels in both blood and cerebrospinal fluid (CSF) after 12 months of treatment [1][2] - The treatment showed a notable slowing in the progression of motor symptoms compared to placebo, with a 2.0 points worsening for the 5mg dose and 1.3 points worsening for the 10mg dose [2] Company Overview - PTC Therapeutics, Inc. is focused on the discovery, development, and commercialization of medicines for rare disorders, leveraging its scientific and clinical expertise [5][20] - The company aims to provide access to best-in-class treatments for patients with limited options, emphasizing its commitment to innovation and a diversified pipeline [5][20] Huntington's Disease Context - Huntington's disease is a hereditary genetic disorder affecting the central nervous system, caused by a defective gene that produces a toxic form of the Huntingtin protein [19] - Currently, there are no approved drugs that can delay the onset or slow the progression of Huntington's disease, highlighting the need for effective therapies like PTC518 [19]