Skyhawk Therapeutics Receives Australian Regulatory Approval to Extend SKY-0515 Treatment Duration in Phase 1 Huntington's Disease Patient Trial

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Australian Human Research Ethics Committees approves SKY-0515 treatment for up to 12 weeks in the company's ongoing Phase 1 trial in patients with Huntington's Disease

Volumetric MRI measurement is also approved as exploratory study endpoint

BOSTON, Dec. 9, 2024 /PRNewswire/ --  Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company, today announced that the Australian Human Research Ethics Committees (HREC) have  approved SKY-0515 treatment for up to 12 weeks in the Company's ongoing Phase 1 trial in patients with Huntington's Disease (HD). HREC also agreed to the inclusion of Volumetric MRI (vMRI) measurements as an early exploratory endpoint in the trial. vMRI measurement of the brain's regions affected in HD can provide additional data on SKY-0515's effect in HD patients.

"We're excited to announce that after demonstrating 72% huntingtin (HTT) mRNA reduction with SKY-0515 in the healthy volunteer portion of this study, we have been approved to extend the duration of treatment in Huntington's patients," said Sergey Paushkin, Head of R&D, Skyhawk Therapeutics. "We're thankful to the HREC for their careful review of our data and approval of this extension, as well as their approval of our use of vMRI measurements as an exploratory endpoint. Both approvals broaden our opportunity to demonstrate the performance of SKY-0515 and may enable us to progress to pivotal trials faster than previously expected."

SKY-0515 is Skyhawk's investigational, oral small molecule RNA splicing modifier developed through the company's novel RNA-splicing platform. SKY-0515 is designed to reduce both HTT protein and PMS1 protein, a key driver of somatic CAG-repeat expansion and HD pathology.

About SKY-0515 Phase 1 Clinical Study

SKY-0515 is currently being evaluated in a Phase 1 clinical trial. The Phase 1 clinical trial is a first-in-human trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of SKY-0515 in healthy volunteers and individuals with early-stage Huntington's disease (HD). The trial is separated into three parts.

Topline pharmacodynamic results for Parts A (single-ascending dose) and B (multiple-ascending dose), which evaluated SKY-0515 in healthy volunteers, demonstrated a dose-dependent reduction of HTT mRNA and reached an average reduction of 72% at a daily oral dose of 9mg. SKY-0515 was generally well tolerated at all doses tested.

Part C is a double-blind placebo-controlled parallel design study of two dose levels of SKY-0515 and placebo in individuals with early-stage HD, which aims to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamic parameters. Recruitment for Part C is ongoing, and topline data are expected in mid-2025.

Additional information for the SKY-0515 Phase 1 study, including participating sites and eligibility criteria, can be found at the Australian New Zealand Clinical Trials Registry.

About Skyhawk Therapeutics
Skyhawk Therapeutics is a clinical-stage biotechnology company focused on the discovery and development of novel small molecule therapies designed to modulate critical RNA targets and revolutionize patient treatment for some of the world's most intractable diseases. Skyhawk's discovery expertise is rooted in its proprietary drug discovery platform, which assesses, identifies, and tests RNA splicing targets and small molecules across a broad range of therapeutic areas and disease states. Skyhawk's novel platform applies to a broad range of disease areas, including neurodegenerative disease, metabolic disease, autoimmune disease, fibrosis and oncology. For more information visit www.skyhawktx.com. 

Skyhawk Contact
Kyle Dow, VP Corporate Development
[email protected] 

SOURCE Skyhawk Therapeutics