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Skyhawk Therapeutics Secures Ethical Clearance for Phase 1 Clinical Study of SKY-0515, an RNA-Targeting Small Molecule, in the Context of Huntington's Disease

Tuesday, October 24, 2023

Skyhawk Therapeutics, Inc. has recently received approval from the Australian Human Research Ethic Committee (HREC) and Australian regulators for a clinical trial of their small molecule candidate, SKY-0515, aimed at addressing Huntington's disease (HD). This rare genetic disorder is the result of a toxic 'CAG' repeat in the huntingtin (HTT) gene, leading to the production of a mutated huntingtin protein. SKY-0515 has been specifically designed to alter the RNA expression of HTT, ultimately reducing the production of this mutated protein responsible for the progression of Huntington's disease.

Skyhawk Therapeutics, expressed optimism about the potential of SKY-0515 in benefiting Huntington's disease patients. He emphasized that the current therapeutic options for these patients are limited, with none capable of reversing or slowing down the progression of the disease. SKY-0515 has demonstrated promising results in preclinical models, raising hopes for substantial benefits for patients.

In the United States alone, around 30,000 symptomatic Huntington's disease patients exist, with many more individuals being pre-symptomatic. Currently, there are no approved drugs that can effectively reverse or slow down the disease's progression. SKY-0515 is an orally administered small molecule that directly targets HTT RNA. It possesses the ability to penetrate the brain and distribute in peripheral tissues, providing potential advantages for patients. Furthermore, SKY-0515 marks the first drug developed by Skyhawk Therapeutics to enter clinical trials.

The Phase 1 clinical trial for SKY-0515 is a groundbreaking first-in-human study with multiple parts. It will be randomized, double-blind, and involve single and multiple ascending doses. The trial is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic activity of SKY-0515 as a monotherapy in both healthy volunteers and, subsequently, in Huntington's disease patients. Additional information about the trial can be found on the Australian New Zealand Clinical Trials Registry (

SKY-0515 is part of Skyhawk Therapeutics' innovative SKYSTAR® platform, which focuses on small molecule RNA splicing modifiers. The goal of SKY-0515 is to reduce the production of the mutated huntingtin protein responsible for the progression of Huntington's disease. It is anticipated that SKY-0515 can be administered orally on a daily basis, with the molecule designed for effective penetration of the brain and distribution in peripheral tissues.

Huntington's disease is a rare inherited condition characterized by the progressive degeneration of nerve cells in the brain. It results in various functional impairments, including movement, cognitive, and psychiatric disorders. Symptoms typically appear in one's 30s or 40s, though in cases where it develops before the age of 20, it is known as juvenile Huntington's disease and presents somewhat differently. While medications are available to manage the symptoms, there is no cure for Huntington's disease, and currently, there are no approved drugs that can delay its onset or slow down its progression.



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