Skyhawk Reports 69% Huntingtin Reduction for SKY-0515 as 12-Month HD Data Support Phase 2/3 Push
Updated
Updated · en.hdbuzz.net · Jun 1
Skyhawk Reports 69% Huntingtin Reduction for SKY-0515 as 12-Month HD Data Support Phase 2/3 Push
3 articles · Updated · en.hdbuzz.net · Jun 1
12-month Phase 1/2 data showed SKY-0515 cut expanded huntingtin by about 69% at the higher dose, with no major new safety concerns and effects holding steady over a year.
Clinical readouts also stayed encouraging: treated patients posted average cUHDRS gains of 0.31 to 0.38 points from months 3 to 12, versus an expected 0.92-point decline from external natural-history datasets.
Skyhawk said the oral splice modulator also reduced PMS1 messenger RNA by up to 26%, suggesting a possible second mechanism tied to somatic CAG repeat expansion as well as huntingtin lowering.
The findings remain preliminary because the study involved only a few dozen early-stage Huntington's patients and relied on external comparators rather than a large year-long placebo group.
FALCON-HD, the pivotal Phase 2/3 trial, has enrolled 144 patients in Australia and New Zealand six months early and is expanding across eight countries toward roughly 400 participants.
With a rival drug ahead in trials, can Skyhawk's unique therapy win the race to treat Huntington's disease?
Is targeting two disease drivers at once the key to finally slowing the relentless progression of Huntington's disease?
SKY-0515’s Dual-Targeting Oral Therapy Delivers 62% mHTT Reduction in Huntington’s Disease: Latest Clinical Data and Future Prospects
Overview
SKY-0515 is an investigational oral small-molecule therapy for Huntington’s disease that has shown promising results in early clinical trials. In a 12-month Phase 1/2 study, SKY-0515 led to sustained reductions in key disease-related biomarkers and was linked to improved clinical outcomes for patients with early-stage Huntington’s disease. These positive trends suggest that SKY-0515 may impact the underlying biological mechanisms of the disease. The encouraging results from this trial provide a strong foundation for further research in larger studies to confirm its efficacy and safety.