Skyhawk Therapeutics Expands Pivotal Trial for Huntington's Disease to U.S., Canada, U.K.
Skyhawk Therapeutics has received regulatory approvals to expand its global FALCON-HD clinical trial for SKY-0515 to the United States, Canada, and the United Kingdom. Patient enrollment will now begin in these countries.
Biotechnology company Skyhawk Therapeutics announced it has secured regulatory approvals to broaden its Phase 2/3 FALCON-HD pivotal clinical trial for the investigational drug SKY-0515. The expansion adds the United States, Canada, and the United Kingdom to the global study, with patient enrollment set to commence in these new regions.
The FALCON-HD trial is evaluating SKY-0515, an oral small molecule therapy designed to modulate RNA targets, for the treatment of Huntington's disease. The global study now involves over ten countries and more than twenty sites, with over 175 patients already enrolled. Previous Phase 1/2 trials indicated SKY-0515 achieved a 69% reduction in mutant HTT protein and a 26% reduction in PMS1, alongside improvements in cUHDRS scores from baseline.
Huntington's disease is a rare, inherited neurodegenerative disorder for which there are currently no approved treatments that slow its progression. SKY-0515 is developed using Skyhawk's proprietary SKYSTAR platform and has demonstrated favorable central nervous system exposure and tolerability in early studies.
The company plans for the FALCON-HD pivotal program to enroll up to 400 additional participants across more than 40 sites worldwide. Skyhawk Therapeutics is also developing other therapies for rare neurological diseases using its platform, with several expected to enter clinical development by the end of 2027.