Interim PIVOT-HD Results Demonstrate Evidence of Favorable CNS Biomarker and Clinical Effects at Month 12 in Huntington’s Disease Patients
– FDA lifts PTC518 partial clinical hold based on PIVOT-HD data –
– Conference call and webcast to be held June 20th at 8:00 am EDT –
WARREN, N.J., June 20, 2024 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared interim results from the Phase 2 PIVOT-HD study of PTC518 in Huntington’s disease (HD) patients. At Month 12, PTC518 treatment resulted in dose-dependent lowering of mutant huntingtin (mHTT) protein in the blood and cerebrospinal fluid (CSF) in the interim cohort of patients. In addition, favorable trends were demonstrated on several relevant HD clinical assessments including Total Motor Score (TMS) and Composite Unified Huntington’s Disease Rating Scale (cUHDRS). Furthermore, following 12 months of treatment, PTC518 continues to be safe and well tolerated.