BOSTON, September 17, 2025 — Leads & Copy — Skyhawk Therapeutics Inc. announced positive results from an interim analysis of the Phase 1 clinical trial of SKY-0515, a treatment for Huntington’s disease (HD). Patients receiving SKY-0515 showed dose-dependent reductions of mutant huntingtin (mHTT) protein in blood, including a 62% reduction at the 9mg dose, at Day 84. The drug has been generally well tolerated.
Treatment with SKY-0515 also resulted in dose-dependent PMS1 mRNA reduction and demonstrated central nervous system penetration. The overall safety profile supports continued clinical development.
Ed Wild, Professor of Neurology at University College London, noted the impressive mHTT protein reduction and the absence of safety concerns. Sergey Paushkin, Head of R&D at Skyhawk Therapeutics, highlighted the potential of SKY-0515 as a transformative therapy for HD.
Huntington’s disease is a rare, hereditary neurodegenerative disorder affecting over 40,000 symptomatic patients in the U.S. SKY-0515, an orally-administered small molecule RNA splicing modifier, is designed to reduce both HTT protein and PMS1 protein.
SKY-0515’s Phase 1 clinical trial evaluates safety, tolerability, pharmacokinetics, and pharmacodynamics. Part C is a double-blind, placebo-controlled study of SKY-0515 in individuals with early-stage HD. Topline data from the blinded active treatment extension are expected in mid-2026.
FALCON-HD (NCT06873334) is a Phase 2/3 study across 10 sites in Australia and New Zealand to evaluate the pharmacodynamics, safety, and efficacy of SKY-0515 in 120 participants with Stage 2 and early Stage 3 HD.
Skyhawk Therapeutics is a clinical-stage biotechnology company developing small molecule RNA modulating therapies.
Contact: Maura McCarthy, maura@skyhawktx.com
Source: Skyhawk Therapeutics
