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Skyhawk Therapeutics Announces Twelve-Month Interim Results from Phase 1/2 Clinical Trial of SKY-0515 in Huntington's Disease
Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate RNA targets, today announced results from the twelve-month interim analysis of patients in its Phase 1/2 clinical trial evaluating SKY-0515, an investigational treatment for Huntington's disease (HD).
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Twelve-month findings showed improvement in Composite Unified Huntington's Disease Rating Scale (cUHDRS) scores from baseline of +0.38, compared to an expected decline of -0.92 points in propensity score-weighted natural history analyses of symptomatic patients SKY-0515 patients achieved reductions of 69% mutant huntingtin protein (mHTT) at the 9 mg dose at twelve months The Company also announces that the Australia and New Zealand (004-ANZ) portion of its Phase 2/3 FALCON-HD pivotal study completed enrollment six months ahead of schedule with 144 patients enrolled, and that the worldwide (004-WW) Phase 2/3 FALCON-HD pivotal study has expanded to eight countries More than 175 patients are now enrolled across the SKY-0515 Phase 1/2 and FALCON-HD pivotal studies BOSTON, June 1, 2026 /PRNewswire/ -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate RNA targets, today announced results from the twelve-month interim analysis of patients in its Phase 1/2 clinical trial evaluating SKY-0515, an investigational treatment for Huntington's disease (HD). Treatment with SKY-0515 resulted in dose-dependent reductions in mutant huntingtin (mHTT) protein in blood of up to 69% as well as reductions in PMS1 mRNA of up to 26%. Mutant huntingtin is the primary protein responsible for HD pathology, while PMS1 is a key driver of somatic CAG repeat expansion associated with disease progression. SKY-0515 has demonstrated excellent central nervous system exposure and has been generally safe and well tolerated across dose levels studied. At three, six, nine and twelve months, patients receiving SKY-0515 demonstrated positive mean changes from baseline Composite Unified Huntington's Disease Rating Scale (cUHDRS) scores ranging from +0.31 to +0.38. These findings compared favorably with an expected worsening of -0.92 points over twelve months from propensity score-weighted analyses which use Enroll-HD and TRACK-HD natural history datasets. Favorable trends for patients on SKY-0515 were also observed across all cUHDRS subcomponents, including Total Motor Score (TMS), Total Functional Capacity (TFC), Symbol Digit Modalities Test (SDMT), and Stroop Word Reading Test (SWRT). "Skyhawk's results show the extraordinary power of the ability to modulate RNA splicing with a small molecule...