Sep 15, 2026 —
Skyhawk Therapeutics has reported final 15-month results from a Phase 1/2 trial of SKY-0515, an oral small molecule for Huntington’s disease that works by modulating RNA splicing to lower the disease-causing protein.
At the 15-month primary timepoint, treated patients showed a 1.59-point advantage on the composite Unified Huntington’s Disease Rating Scale (cUHDRS) versus an external natural-history comparison, with statistically significant differences across all four components. The company reported average reductions in mutant huntingtin protein of more than 60% at the 9 mg dose.
Skyhawk said its Phase 1/2 and global Phase 2/3 FALCON-HD program have now enrolled more than 200 patients across ten countries. Huntington’s is a fatal, inherited neurodegenerative disease with no approved disease-modifying therapy.
