Skyhawk Therapeutics Extends Huntington's Disease Drug Trial to US, Canada, UK

Skyhawk Therapeutics, a clinical-stage biotechnology company, announced it has obtained regulatory clearance to expand its pivotal FALCON-HD clinical trial to the United States, Canada, and the United Kingdom. The trial investigates SKY-0515, an orally administered small molecule drug candidate targeting RNA modulation for the treatment of Huntington's disease.

The FALCON-HD trial, now extended to over ten countries and twenty sites, has successfully recruited more than 175 patients. Preliminary data from earlier studies indicate that SKY-0515 has been generally well-tolerated and has demonstrated a significant reduction in mutant huntingtin protein (mHTT) levels, along with a decrease in PMS1 protein and improvement in cUHDRS scores compared to baseline and natural disease progression.

Huntington's disease is a rare, inherited neurodegenerative disorder affecting tens of thousands worldwide, with no approved treatments currently available to slow or halt its progression. SKY-0515 is designed to reduce mHTT and PMS1 protein levels, addressing a key pathological mechanism of the disease.

"We are thrilled to begin patient enrollment for our pivotal Huntington's disease trial in the United States, United Kingdom, and Canada," said Sergey Paushkin, Skyhawk's Head of R&D. Paushkin noted that the drug, administered as a daily tablet, shows promise as a convenient and potentially disease-modifying therapy for Huntington's patients.