#USA #Boston #Huntington's Disease #Skyhawk Therapeutics #SKY-0515

Promising Developments in Huntington's Disease Treatment with SKY-0515

Skyhawk Therapeutics, a clinical-stage biotechnology firm, has recently unveiled encouraging findings from the first interim analysis of its Phase 1 clinical trial for SKY-0515, aimed at treating Huntington's disease, a serious neurodegenerative disorder. Huntington's disease, characterized by the progressive degeneration of nerve cells in the brain, currently lacks approved treatments that can slow or halt its progression. With over 40,000 symptomatic patients in the U.S. alone, the need for effective therapies remains critical.

In this Phase 1 clinical trial, SKY-0515 has shown a dose-dependent decrease in the levels of mutant huntingtin (mHTT) protein in the bloodstream, achieving a 62% reduction at the 9 mg daily oral dose by Day 84 of treatment. These findings underscore the drug's potential effectiveness in addressing the underlying pathophysiology of Huntington's disease. Dr. Ed Wild, a Neurology professor at University College London, emphasized the impressive extent of mHTT reduction observed in patients and noted no significant safety concerns across the doses tested.

Additionally, treatment with SKY-0515 related to reductions in PMS1 mRNA, another significant factor in Huntington's disease pathology. The drug demonstrated excellent penetration into the brain, enhancing its potential therapeutic impact. With a favorable safety profile established so far, the results pave the way for continued development of SKY-0515.

Dr. Sergey Paushkin, Head of Research and Development at Skyhawk Therapeutics, remarked on the strength of the biomarker responses after merely 84 days of treatment. He stressed that this interim analysis marks a key milestone for the therapy, illustrating the efficacy of Skyhawk's proprietary platform for delivering innovative therapies to treat devastating conditions.

The Phase 1 trial, designed as a first-in-human study, evaluates the safety and pharmacodynamics of SKY-0515 in healthy volunteers and individuals in the early stages of Huntington's disease. It consists of three parts; Parts A and B assessed the drug in healthy participants while Part C involves a double-blind, placebo-controlled approach focusing on patients with early-stage Huntington's disease. Part C’s enrollment is complete, and topline data from the subsequent active treatment phase is anticipated by mid-2026.

Skyhawk is also conducting the Phase 2/3 FALCON-HD trial, which aims to explore the pharmacodynamics and safety of SKY-0515 among 120 participants at various stages of Huntington's disease across multiple sites in Australia and New Zealand. Patients will receive daily oral doses of the investigational therapy or a placebo for at least 12 months.

Huntington's disease is an inherited condition that can be devastating, progressively diminishing patients' physical and cognitive abilities. Without effective treatment options approved by regulatory bodies, many families are left to navigate the emotional and physical tolls of the disease. SKY-0515, designed to target the mechanisms of disease at a molecular level, represents hope for many.

Skyhawk is committed to expanding its research initiatives, intending to introduce several novel therapies for rare neurological disorders by the end of 2027. The startup's integrated platform, SKYSTAR®, allows for the identification and development of unique small molecules designed to modulate RNA splicing—creating possibilities for groundbreaking treatments where none exist.

For those affected by Huntington's disease and the medical community at large, the interim findings from the Phase 1 study of SKY-0515 represent an encouraging step toward addressing this challenging disorder. As researchers continue to investigate the full potential of SKY-0515 and its impact on the disease's trajectory, the hope for effective treatment options grows stronger.