Australian Human Research Ethics Committees approve SKY-0515 treatment for up to 12 weeks in the company’s ongoing Phase 1 trial in patients with Huntington Disease
MRI volumetric measurement is also approved as an endpoint for the exploratory study
BOSTON, December 10, 2024 /PRNewswire/ — Skyhawk Therapeutics, Inc.a clinical-stage biotechnology company, today announced that the Australian Human Research Ethics Committees (HREC) have approved the SKY-0515 treatment for up to 12 weeks in the phase trial 1 in progress of the company in patients with Huntington Disease (HD). HREC also agreed to the inclusion of volumetric MRI (vMRI) measurements as an early exploratory endpoint of the trial. vMRI measurement of brain regions affected by HD may provide additional data on the effect of SKY-0515 in HD patients.
“We are pleased to announce that after demonstrating a 72% reduction in huntingtin (HTT) mRNA with SKY-0515 in the healthy volunteer portion of this study, we have been approved to extend the duration of treatment. In Huntington patients,” said Sergey Paushkin, Head of R&D at Skyhawk Therapeutics. “We thank 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 expand our opportunities to demonstrate the performance of SKY-0515 and could allow us to progress toward pivotal trials more quickly than 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 the SKY-0515 Phase 1 Clinical Study
SKY-0515 is currently being evaluated in a Phase 1 clinical trial. The Phase 1 clinical trial is the first in-human trial designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of SKY -0515 in healthy volunteers and early-stage affected individuals. Huntington disease (HD). The trial is divided into three parts.
The main pharmacodynamic results for Parts A (single ascending dose) and B (multiple ascending doses), which evaluated SKY-0515 in healthy volunteers, demonstrated a dose-dependent reduction in HTT mRNA and achieved a reduction average of 72% at a daily dose. oral dose of 9 mg. SKY-0515 was generally well tolerated at all doses tested.
Part C is a double-blind, placebo-controlled parallel study of two dose levels of SKY-0515 and placebo in people with early-stage HD, which aims to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic parameters. Recruitment for Part C is ongoing and first data is expected by mid-2025.
Additional information about the SKY-0515 Phase 1 study, including participating sites and eligibility criteria, is available here Australian and 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 therapeutics designed to modulate critical RNA targets and revolutionize patient treatment for some of the world’s most incurable diseases. Skyhawk’s discovery expertise is based on its proprietary drug discovery platform, which evaluates, identifies and tests RNA splicing targets and small molecules across a broad range of therapeutic areas and disease states . Skyhawk’s new platform applies to a broad range of disease areas, including neurodegenerative diseases, metabolic diseases, autoimmune diseases, fibrosis and oncology. For more information, visit www.skyhawktx.com.
Contact Skyhawk
Kyle Dowvice president of corporate development
kyle.dow@skyhawktx.com
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