Skyhawk Therapeutics has dosed the first subject in the randomised Phase II/III FALCON-HD trial of the oral small-molecule RNA splicing modulator, SKY-0515, for Huntington’s disease (HD) treatment.
The placebo-controlled, double-blind trial plans to enrol 120 individuals across New Zealand and Australia across 10 sites, with the first dosing occurring at Flinders Medical Centre in Adelaide.
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It aims to assess the safety, efficacy, and pharmacodynamics of the therapy in those with Stage 2 and early Stage 3 HD.
Also, it will evaluate changes in huntingtin (HTT) protein levels, the effects on HD-related brain atrophy and clinical features, and the long-term safety of the therapy.
The FALCON-HD study comprises three periods: a screening period of up to four weeks for eligibility evaluations, a 12-month double-blind treatment duration where subjects will take the therapy or a placebo daily, and a four-week follow-up period post-treatment completion.
Regular clinic visits will help monitor efficacy and safety via blood tests, magnetic resonance imaging (MRI), and clinical evaluations using the Unified Huntington’s Disease Rating Scale (UHDRS).
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By GlobalDataThe trial will be overseen by an independent Data Safety Monitoring Board (DSMB) to ensure the safety of the subjects.
Developed through the company’s platform, SKY-0515 is designed to minimise the production of HTT and PMS1 homolog 1 (PMS1) proteins, which are significant contributors to HD pathology.
In a previous Phase I trial involving healthy volunteers, the therapy showed a dose-dependent decrease in HTT mRNA, achieving an average lowering of 72% at the highest dose evaluated.
The therapy was found to be generally well tolerated across every dose. Commenced in January 2025, the company’s Phase I trial in HD patients concluded enrolment ahead of schedule in March.
Skyhawk Therapeutics CEO and founder Bill Haney said: “Dosing the first patient in our FALCON-HD trial marks a significant milestone in our mission to develop a disease-modifying therapy for Huntington’s patients.
“Building on our compelling Phase I data, we are eager to assess SKY-0515’s potential to make a meaningful difference in the lives of patients affected by this devastating condition.”
