More than ten countries and more than twenty sites are now participating in the Company's global pivotal trial for Huntington's disease, with over 175 patients enrolled.
SKY-0515's Phase 1/2 enrollment is complete and it has been generally well tolerated with twelve months of patient data. SKY-0515 has shown 69% reduction in mHTT, 26% reduction in PMS1, and cUHDRS improvements from baseline instead of natural history declines.
BOSTON, July 14, 2026 /PRNewswire/ -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate RNA targets, announces it has received additional regulatory approvals to open its Phase 2/3 FALCON-HD (004-ANZ and 004-WW) pivotal trial, with an IND acceptance in the United States and CTA acceptances in each Canada and the United Kingdom.
"We are excited to now begin enrolling patients in our pivotal trial for Huntington's in the United States, the United Kingdom and Canada," said Sergey Paushkin, head of Skyhawk R&D. "Clinician and Patient assessments done at twelve months of treatment support what the SKY-0515's compelling and consistent effects on critical biomarkers and cUHDRS scores demonstrate—the exciting possibility that SKY-0515 may offer Huntington's patients a type of therapy they have long deserved, in a convenient daily pill."SKY-0515 has demonstrated excellent central nervous system exposure and has been generally safe and well tolerated across dose levels studied.Huntington's disease is a rare, hereditary, and ultimately fatal neurodegenerative disorder affecting more than 40,000 symptomatic individuals in the United States, with hundreds of thousands more impacted worldwide. There are currently no approved therapies shown to slow or halt disease progression.
