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

 Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company,  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.