Oral Huntington’s drug SKY-0515 shows durable 12-month signals and dual-targeting approach
Skyhawk Therapeutics reports 12-month Phase 1/2 data for SKY-0515, an oral splice-modulator that lowers huntingtin and PMS1. Higher-dose treatment reduced expanded huntingtin by about 69% over a year, with PMS1 mRNA down by up to 26%, and small, sustained improvements in the cUHDRS clinical score versus external natural-history data. While safety appears generally favorable in this early study, the results come from a small, non-placebo-controlled cohort and rely on external comparators, so they do not prove disease modification. A larger Phase 2/3 FALCON-HD trial is underway to determine if these signals translate into true slowing of HD progression. The therapy’s oral format and dual-target approach offer a promising two-pronged strategy, with global enrollment continuing and milestones reached in Australia/New Zealand.