The clinical trial tracked participants over a fifteen-month period, revealing statistically significant benefits across all four components of the disease rating scale, including motor function, processing speed, and daily living capacity. Notably, the drug achieved consistent average reductions of more than 60% in mutant huntingtin protein and 25% in PMS1 mRNA at the 9 mg dose level.
Skyhawk Therapeutics Reports Positive Phase 1/2 Data for Huntington's Drug
Patients treated with SKY-0515 showed a 1.59-point improvement on the Composite Unified Huntington's Disease Rating Scale compared to natural history controls after fifteen months. The study, which marks the final analysis of the Phase 1/2 trial, suggests the oral treatment successfully targets core mechanisms of the neurodegenerative disorder.
Experts view these findings as a significant development, particularly due to the drug's oral administration, which distinguishes it from more invasive therapies. Following these results, Skyhawk Therapeutics is advancing its global Phase 2/3 FALCON-HD pivotal program, which has already enrolled over 200 patients across ten countries. The company aims to leverage its proprietary SKYSTAR platform to address other neurological conditions by 2027.
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