BOSTON – November 06, 2026 -- Skyhawk Therapeutics has dosed the first U.S. patients in its global pivotal FALCON-HD study of SKY-0515 in Huntington's disease (HD). More than 50,000 doses have been administered to date across more than 30 study sites in 10 countries.
CenExel Colorado site opens U.S. dosing, with Beth Israel Deaconess next
Dosing in the U.S. began at the CenExel Movement Disorder Center in Colorado. Additional U.S. sites, including Beth Israel Deaconess Medical Center, are expected to begin dosing in the coming weeks. Samuel Frank, MD, Director of the HDSA Center of Excellence at Beth Israel Deaconess and a FALCON-HD investigator, expects dosing there before the end of 2026. He described Phase 1/2 results to date as very encouraging.
SKY-0515 targets two disease proteins with a once-daily oral dose
SKY-0515 is an investigational oral small molecule RNA splicing modifier designed to reduce both mutant huntingtin (mHTT) and PMS1. Mutant huntingtin is the primary protein responsible for HD pathology, while PMS1 drives somatic CAG repeat expansion associated with disease progression. CEO Bill Haney said the company's goal is a once-daily oral, "potentially disease-modifying treatment."
FALCON-HD runs two placebo-controlled, dose-ranging studies
The program (NCT06873334 and NCT07378644) comprises two randomized, double-blind, placebo-controlled studies. Participants receive once-daily oral SKY-0515 at one of three dose levels or placebo.
FALCON-HD 004-ANZ enrolled 144 participants with Stage 2 and Stage 3 HD in Australia and New Zealand, and enrollment is complete. FALCON-HD 004-WW is actively recruiting Stage 2 and Stage 3 participants and plans to enroll across more than 40 sites worldwide.
No disease-modifying therapy exists for more than 40,000 symptomatic U.S. patients
More than 40,000 people in the U.S. live with symptomatic HD, and hundreds of thousands are affected worldwide. No approved therapy has been shown to slow or stop the underlying progression of the disease.