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

Skyhawk Therapeutics Expands Treatment Protocol for Huntington's Disease in Australia

In a significant advancement for Huntington's Disease (HD) research, Skyhawk Therapeutics, Inc., a pioneering clinical-stage biotechnology firm, received vital regulatory approval from the Australian Human Research Ethics Committees (HREC). This approval permits the company to extend the administration of its investigational treatment, SKY-0515, in ongoing Phase 1 trials, allowing for treatment durations of up to 12 weeks. This marks a promising step forward in the fight against HD, a neurodegenerative disorder characterized by progressive motor control decline and cognitive challenges.

The extension comes after remarkable results were generated from the earlier phases of the study, with SKY-0515 demonstrating a substantial 72% reduction in huntingtin (HTT) mRNA levels following treatment in healthy volunteers. Sergey Paushkin, the Head of Research and Development at Skyhawk Therapeutics, expressed excitement over the approval, emphasizing the company's gratitude to HREC for their thorough evaluation and support in extending the trial duration. The inclusion of Volumetric MRI (vMRI) measurements as an exploratory endpoint is also noteworthy, as it promises to provide additional insights into the treatment's efficacy by examining how SKY-0515 impacts specific brain regions affected by HD.

SKY-0515 is recognized for its innovative use as an oral small molecule RNA splicing modifier derived from Skyhawk's proprietary drug discovery platform. The mechanism of action aims to minimize both HTT protein and PMS1 protein levels, the latter being a significant contributor to the somatic CAG-repeat expansion that underpins HD pathology. The Phase 1 trial is structured into three distinct parts:

• - Part A and B: These phases focus on single-ascending and multiple-ascending doses, confirmed the safety and tolerability along with pharmacokinetic evaluations in healthy volunteers.
• - Part C: A double-blind, placebo-controlled segment currently underway, aimed at determining safety and pharmacodynamics in individuals exhibiting early-stage HD.

Within these trials, interim results point toward a successful reduction of HTT, alongside promising tolerability profiles for the tested doses of SKY-0515. Ongoing recruitment for Part C is expected to contribute to the accumulation of pivotal data, with topline outcomes anticipated in mid-2025.

Skyhawk’s continual commitment to its innovative approach embodies its dedication not only to Huntington's Disease but also to a wider array of challenging diseases including autoimmune disorders, metabolic anomalies, and certain cancers. With the competitive biotechnology landscape constantly evolving, the advancements seen with SKY-0515 could establish a new standard of care for HD patients.

As Skyhawk Therapeutics prepares for additional further trials, the findings from the Phase 1 study will be crucial to defining the drug's potential and impact. More detailed information regarding the SKY-0515 Phase 1 study can be accessed via the Australian New Zealand Clinical Trials Registry, providing transparency in eligibility criteria, recruitment status, and upcoming milestones.

In conclusion, the approval of extended treatment durations coupled with new exploratory endpoints showcases a proactive approach to enhancing the understanding and management of Huntington's Disease. Skyhawk’s exploration into RNA splicing modifications has now set the stage for potential breakthroughs that may alleviate the burden faced by those afflicted with this debilitating condition.

For further insights and development updates concerning Skyhawk Therapeutics and their ongoing research efforts, please visit their official website at www.skyhawktx.com.