Skyhawk Therapeutics Reports Successful Interim Results for SKY-0515 in Huntington's Disease Trial

Skyhawk Therapeutics, a clinical-stage biotechnology company, has announced positive findings from the interim analysis of their Phase 1 clinical trial for SKY-0515, a novel treatment aimed at Huntington's Disease (HD). The results released on January 27, 2026, indicate significant advancements in patient conditions when treated with this investigational therapy.

Trial Overview and Results

The interim results, assessed over a nine-month treatment period, showcased a mean improvement of +0.64 points in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) among patients receiving SKY-0515. This contrasts sharply with the anticipated decline of -0.73 points based on historical data from symptomatic patients undergoing natural disease progression without treatment. This notable divergence highlights the potential of SKY-0515 as a modifying therapy for this challenging neurological disorder.

Analyzing the treatment in detail, patients demonstrated a 62% reduction in mutant huntingtin protein (mHTT) levels in the bloodstream at the 9 mg dosage, alongside a 26% reduction in PMS1 mRNA, a key factor linked to the pathological expansion of CAG repeats associated with HD. The drug also exhibited excellent CNS penetration and a safety profile that was well-tolerated across all dosage levels.

According to Ed Wild, a Neurology professor at University College London, these results present early but compelling evidence of SKY-0515's efficacy and safety. “The capacity of SKY-0515 to concurrently reduce both mHTT and PMS1 creates a formidable approach to combatting Huntington's Disease, focusing on two vital pathways involved in the disease's progression,” he stated.

Progress and Future Directions

Alongside these promising interim results, Skyhawk Therapeutics has initiated a global expansion of its Phase 2/3 FALCON-HD trial, actively including more sites worldwide after originally launching the trial in Australia and New Zealand. To date, the SKY-0515 treatment has been administered to over 90 patients, illustrating both the interest and the urgency surrounding potential HD treatments.

Sergey Paushkin, Head of R&D at Skyhawk, reinforced the significance of these findings, indicating that the response showcased an important step forward for SKY-0515, underscoring its standing as a prospective disease-modifying therapy for Huntington's Disease.

Context of Huntington's Disease

Huntington's Disease is a hereditary neurodegenerative disorder that affects an estimated 40,000 symptomatic patients in the United States alone, with many more potentially but undiagnosed cases globally. Currently, there are no approved therapies that can halt or significantly slow its progression, leaving patients and families desperate for effective treatment options. As SKY-0515 progresses through the clinical trial phases, the hope remains that it could represent a viable oral treatment to reduce huntingtin and provide much-needed relief to those impacted by this devastating disorder.

Skyhawk plans to introduce additional small-molecule treatments targeting rare neurological diseases lacking approved therapies by the end of 2027, reflecting the company's commitment to advancing novel therapies for those in dire need of effective treatment options.

Conclusion

Overall, the interim results from Skyhawk Therapeutics' trial of SKY-0515 are highly promising, suggesting that the investigational drug could play a crucial role in altering the course of Huntington's Disease. As further data is gathered from ongoing and upcoming trials, the hope for patients may finally be gathering momentum through innovative therapeutic avenues. Skyhawk Therapeutics' work exemplifies the groundbreaking efforts being made in biotechnology to combat historically challenging conditions.