Skyhawk Reports Positive Phase 1/2 Results for Huntington’s Drug SKY-0515

Patients with Huntington’s disease treated with Skyhawk Therapeutics’ oral drug SKY-0515 showed no clinical worsening after twelve months, according to interim data from a Phase 1/2 trial. The findings, presented at the European Academy of Neurology, suggest the small molecule therapy may stabilize disease progression in early-stage participants.

Corp and Tech·June 30, 2026·reads: 391

The twelve-month analysis evaluated the drug's performance across the four subcomponents of the cUHDRS assessment: Total Functional Capacity, Total Motor Score, the Symbol Digit Modalities Test, and the Stroop Word Reading Test. In each category, participants receiving SKY-0515 demonstrated favorable trends compared to expected outcomes modeled against Enroll-HD natural history datasets. Notably, the drug achieved a mean change of +0.07 in functional capacity, whereas historical data for the disease typically shows a decline of 0.87 points.

Subjective assessments provided further evidence of the drug's potential impact. During the survey period, no clinicians or patients reported disease worsening. Half of the clinicians and 65% of the participants reported observing actual improvement in their condition. These results align with the drug’s biological activity; treatment led to dose-dependent reductions of up to 69% in mutant huntingtin protein, the primary driver of Huntington’s pathology, and up to 26% in PMS1 mRNA, which is linked to disease progression.

Sergey Paushkin, Head of R&D at Skyhawk, noted that the data confirms the potential for a convenient daily pill to address a condition that currently lacks disease-modifying treatments. The company has already moved into its pivotal FALCON-HD program, which aims to enroll hundreds of additional participants across more than 40 global sites to further validate these findings.