Skyhawk Therapeutics today announced positive results from its Phase 1 clinical trial of SKY-0515, an oral small molecule designed to reduce mutant huntingtin (HTT) protein in patients with Huntington's Disease.
The nine-month interim data showed a 62% reduction in mutant HTT protein levels in cerebrospinal fluid, along with clinically meaningful improvements in the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 3, 6, and 9 months.