FLINDERS MEDICAL CENTRE

Skyhawk Therapeutics Initiates Phase 2/3 Trial of SKY-0515 for Huntington's Disease

- Skyhawk Therapeutics has dosed the first patient in its Phase 2/3 FALCON-HD trial evaluating SKY-0515, an oral RNA splicing modulator for Huntington's disease treatment. - SKY-0515 demonstrated promising Phase 1 results with up to 72% reduction in huntingtin mRNA in healthy volunteers and was generally well tolerated across all doses. - The investigational drug uniquely targets both huntingtin and PMS1 proteins, two key drivers of Huntington's disease pathology, potentially offering enhanced therapeutic impact. - The randomized, placebo-controlled trial will enroll 120 participants across 10 sites in Australia and New Zealand, representing a significant milestone for disease-modifying HD therapies.