PTC Therapeutics Says Interim Data For Huntington's Disease Candidate Shows Promise In Mid-Stage Study
PTC Therapeutics Inc (NASDAQ: PTCT) shared interim data from the 12-week portion of the PIVOT-HD Phase 2 study of PTC518 in Huntington's disease (HD) patients. The study demonstrated dose-dependent lowering of Huntingtin (HTT) protein levels in peripheral blood cells, reaching a mean 30% reduction in mutant HTT levels at the 10mg dose level. In addition, PTC518 exposure in the cerebrospinal fluid (CSF) was consistent with or higher than plasma unbound drug levels. PTC518 treatment was also well
Source: yahoo.com