Skyhawk Therapeutics Expands Global Clinical Trial for SKY-0515 in Huntington's Disease to US, Canada, and UK

Skyhawk Therapeutics Expands Horizon-HD Study in the US, Canada, and UK

Skyhawk Therapeutics, a pioneering biotechnology firm focused on developing small-molecule therapies, has announced that it is extending its pivotal clinical trial, known as FALCON-HD, for SKY-0515, a promising treatment for Huntington's disease, to include participants from the United States, Canada, and the United Kingdom. This expansion allows the company to recruit patients from over 20 locations across more than ten countries, bringing fresh hope to those battling this severe neurodegenerative disorder.

The FALCON-HD study is crucial for evaluating SKY-0515's efficacy and safety and has had over 175 participants enrolled thus far. The recruitment for the phase 1/2 study is now complete, and interim results indicate that SKY-0515 has been well-tolerated among patients, showing a remarkable decrease in mutant huntingtin (mHTT) protein levels by 69% and poly(Q)76 (PMS1) protein levels by 26%. Furthermore, improvements in the Composite Unified Huntington’s Disease Rating Scale (cUHDRS) scores suggest that patients experienced positive changes instead of the expected deterioration associated with the disease's natural progression.

Sergey Paushkin, the head of R&D at Skyhawk, expressed enthusiasm about commencing patient recruitment across these new territories. “The assessments conducted after twelve months of treatment reinforce the consistent effects of SKY-0515 on key biomarkers and cUHDRS scores,” Paushkin stated. He emphasized the potential for SKY-0515 to offer a meaningful therapy to Huntington's patients, delivered conveniently as an oral tablet.

SKY-0515 operates as a small-molecule RNA splicing modifier designed specifically to suppress the expression of both mHTT and PMS1 proteins. The investigational drug has exhibited exceptional exposure within the central nervous system, ensuring patient safety across several dosage levels. The urgency for treating Huntington's disease is underscored by the fact that over 40,000 individuals in the United States alone live with symptomatic forms of the disease, with countless more affected globally, yet there are currently no approved treatments that can effectively slow or halt its progression.

The study will not only evaluate the pharmacodynamics and effectiveness of SKY-0515 but will also assess its safety profile across a variety of international sites. The FALCON-HD study (NCT06873334 and NCT07378644) employs a rigorous randomized, double-blind, placebo-controlled design and aims to determine optimal dosages through conscientious participant monitoring.

In Australia and New Zealand, 144 participants struggling with stage 2 and early stage 3 Huntington's disease have already participated in the FALCON-HD 004-ANZ study with recruitment now closed. Meanwhile, the 004-WW version of the study is set to enroll up to 400 additional participants with similar disease stages from over 40 locations worldwide, some of which are already treating patients with the drug.

To know more about how to participate in the FALCON-HD study, along with available sites and eligibility criteria, interested parties can consult ClinicalTrials.gov and visit www.FALCON-HD.com.

Skyhawk Therapeutics plans to leverage its proprietary SKYSTAR® platform beyond SKY-0515 to develop innovative RNA-targeting therapies aimed at rare neurological diseases, many of which currently lack effective treatment options. As such, the biotech company is poised to move several programs into clinical development by the close of 2027, signifying a promising era of advancement in Huntington’s disease and beyond.

For additional inquiries or insights about Skyhawk Therapeuics and its pioneering work, please visit the corporate website or reach out to the designated media contact.