Pharma Focus Europe

Skyhawk Therapeutics Progresses to Multiple Ascending Dose Phase in Phase 1 Trial of SKY-0515 Targeting Huntington's Disease

Thursday, February 29, 2024

Skyhawk Therapeutics, a biotechnology company in the clinical stage, has initiated the Phase 1 multiple ascending dose (MAD) segment of its clinical study in Australia for SKY-0515, a small molecule candidate targeting Huntington's disease (HD). This disorder is caused by a toxic 'CAG' repeat in the huntingtin (HTT) gene, leading to a mutated huntingtin protein. SKY-0515 is designed to modify HTT RNA expression, reducing the production of the mutated protein associated with disease progression in Huntington's patients.

Skyhawk Therapeutics, expressed excitement about the start of the MAD portion of Phase 1, following the successful progression of the Phase 1 single ascending dose study. Faller highlighted the potential of SKY-0515 to address the therapeutic gap in Huntington's disease, where current options fail to reverse or slow disease progression. He emphasized the robust activity demonstrated by SKY-0515 in preclinical models, suggesting promising benefits for patients.

With around 30,000 symptomatic Huntington's disease patients in the US alone and numerous pre-symptomatic individuals, there exists a significant unmet medical need. SKY-0515, an orally administered small molecule with brain penetration and distribution in peripheral tissues, holds promise as a pioneering therapeutic option. It marks the first drug developed by Skyhawk Therapeutics to enter clinical trials.

SKY-0515's Phase 1 clinical trial is a pivotal first-in-human study, comprising multiple parts, including single and multiple ascending dose segments. This randomized, double-blind trial aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamic activity of SKY-0515 monotherapy in healthy volunteers initially, followed by Huntington's patients. Further details about the trial can be accessed through the Australian New Zealand Clinical Trials Registry.

SKY-0515, harnessed through Skyhawk Therapeutics' innovative SKYSTAR® platform, functions as a small molecule RNA splicing modifier with the potential to mitigate Huntington's disease progression. Its predicted pharmacokinetics support daily oral dosing, while its design facilitates brain penetration and peripheral tissue distribution.

Huntington's disease, an inherited neurodegenerative disorder, inflicts progressive nerve cell degeneration in the brain. It severely impacts functional abilities, leading to movement, cognitive, and psychiatric impairments, ultimately proving fatal. Although medications exist to alleviate symptoms, there is currently no cure for Huntington's disease, and approved drugs capable of delaying disease onset or halting its progression are lacking.



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