Skyhawk Reports Positive Phase 1/2 Data for Huntington's Drug SKY-0515

The twelve-month results from the Phase 1/2 trial demonstrate consistent benefits for participants receiving the investigational small molecule. Data across the cUHDRS subcomponents—Total Functional Capacity, Total Motor Score, Symbol Digit Modalities Test, and Stroop Word Reading Test—showed favorable trends compared to natural history datasets. Specifically, participants recorded a mean change of +0.07 in functional capacity, contrasting with an expected decline of -0.87 points.

Subjective assessments reinforced these clinical findings. No clinicians or patients reported disease worsening at the one-year mark, with 65% of patients and 50% of clinicians noting actual improvement. Mechanistically, the treatment achieved dose-dependent reductions of up to 69% in mutant huntingtin protein and up to 26% in PMS1 mRNA, both of which are central to disease pathology. The therapy has remained well-tolerated throughout the study, which currently includes over 175 participants across five countries. Skyhawk is now advancing its broader FALCON-HD pivotal program to further evaluate the drug’s potential as a disease-modifying treatment for the more than 40,000 symptomatic individuals in the United States.