Skyhawk Therapeutics Reports Nine-Month Interim Results for SKY-0515 in Huntington's Disease Study

Skyhawk Therapeutics Announces Interim Results of SKY-0515

Skyhawk Therapeutics has recently disclosed the interim nine-month results of its phase 1 clinical trial for the drug SKY-0515, aimed at treating Huntington's disease (HD). This promising drug, which acts through the modulation of RNA targets, has shown positive outcomes that may significantly impact the lives of patients diagnosed with this rare neurodegenerative disorder.

The clinical study results reveal an average improvement of 0.64 points on the Composite Unified Huntington's Disease Rating Scale (cUHDRS) from the baseline, contrasting the natural disease progression, which typically sees an expected decline of -0.73 points over the same period. These findings were derived from weighted propensity analysis, utilizing prior research data from studies like Enroll-HD and TRACK-HD.

Ed Wild, a neurology professor at University College London, expressed his enthusiasm regarding the data, highlighting the treatment's ability to achieve a significant reduction in mutant huntingtin protein (mHTT) levels in the blood, alongside improvements in other biomarkers. The study indicates a 62% decrease in mHTT levels at a dose of 9 mg, alongside a 26% reduction in PMS1 mRNA levels, marking a dual mechanism of action in combating the disease.

Throughout the nine-month course, patients on SKY-0515 continually demonstrated clinical improvements compared to their baseline scores. The treatment showed overall efficacy, safety, and tolerance with minimal adverse effects reported across the various dosage levels tested in the trial.

In addition to the positive results, the company announced the global expansion of its phase 2/3 study, FALCON-HD, which has now recruited over 90 patients across multiple countries. This expanded participation signals a robust scientific interest in this promising therapeutic approach to treating Huntington's disease, a condition that currently has no approved disease-modifying therapies.

Skyhawk's approach utilizes its proprietary SKYSTAR® platform to develop small-molecule RNA-modulating drugs for various challenging diseases. The promising results of SKY-0515 highlight the company's commitment to innovate treatment options where previously none existed.

As Huntington's disease continues to affect more than 40,000 individuals in the U.S. and hundreds of thousands worldwide, the unveiling of these results brings hope to patients and families enduring the effects of this debilitating condition. Currently, no therapies are approved that can slow or halt the disease's progression, which underscores the significance of SKY-0515's potential impact.

Sergey Paushkin, Skyhawk’s R&D lead, emphasized that their phase 1 study's goal was to establish the safety and activity of biomarkers associated with SKY-0515. The retained strength in biomarker response over the nine-month interim analysis bolsters hope for the drug being a leading therapy in the treatment of Huntington’s disease.

The phase 1 clinical trial itself is designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of SKY-0515 among early-stage HD patients involving multiple study parts. Following this foundational trial, Skyhawk aims to start an additional clinical trial of new treatments targeting other rare neurological diseases by the end of 2027, maintaining their mission to deliver innovative solutions in the medical field.

As anticipation builds for the further phases of testing, including the double-blind placebo-controlled phase 2/3 trial, the medical community and patients alike are eager to witness the potential of SKY-0515 as a transformative treatment for Huntington’s disease. The outcomes of this research may not only change the course of treatment but may also fundamentally alter patients' daily lives, granting them more hope and stability than previously thought possible.