BREAKING: Skyhawk Therapeutics has just announced promising interim results from its Phase 1 clinical trial of SKY-0515, a potential game-changer in the treatment of Huntington’s disease (HD). At just Day 84, patients receiving the 9mg daily oral dose saw an astonishing 62% reduction in mutant huntingtin (mHTT) protein levels.
These results, revealed on September 17, 2025, indicate that SKY-0515 not only reduces mHTT protein but also demonstrates significant reductions in PMS1 mRNA, excellent brain penetration, and a favorable safety profile. This news is particularly critical as Huntington’s disease currently has no approved treatments to slow or halt its progression.
Ed Wild, Professor of Neurology at University College London, commented on the findings, stating,
“SKY-0515 is reducing mHTT protein to the most impressive extent we’ve seen so far in patients, and crucially the clinical and biomarker data show no safety concerns so far at any dose tested.”
Wild emphasized that such a treatment could be transformative for the over 40,000 symptomatic patients in the United States and hundreds of thousands worldwide affected by this devastating disorder.
Skyhawk is progressing to its Phase 2/3 FALCON-HD trial, which is currently ongoing in Australia and New Zealand. This trial aims to evaluate the pharmacodynamics, safety, and efficacy of SKY-0515 in 120 participants diagnosed with Stage 2 and early Stage 3 HD. Eligible participants will receive once-daily oral doses of SKY-0515 for at least 12 months, further exploring its potential to modulate RNA splicing and reduce the proteins linked to HD pathology.
Skyhawk’s Head of R&D, Sergey Paushkin, highlighted the significance of these interim findings, stating,
“The strength of SKY-0515’s biomarker response after just 84 days of treatment underscores its potential as a transformative therapy for HD.”
This research milestone not only supports the ongoing development of SKY-0515 but also showcases Skyhawk’s innovative approach to tackling rare neurological diseases.
The Phase 1 clinical trial is structured to evaluate safety and efficacy in three parts, with Part C focusing on individuals with early-stage HD. Enrollment is complete, with topline data from the blinded active treatment extension expected by mid-2026.
As the search for effective treatments for Huntington’s disease continues, the potential of SKY-0515 marks a significant advancement for patients and their families. The urgency of these developments cannot be overstated, as there are currently no approved therapies to halt the progression of this fatal neurodegenerative disorder.
Skyhawk Therapeutics is committed to advancing this groundbreaking research, with plans to introduce more novel drugs aimed at rare neurological diseases by the end of 2027. The excitement surrounding SKY-0515 could mean a new dawn for Huntington’s disease treatment, providing hope to countless individuals affected by this condition.
Stay tuned for further updates as this story develops.
