Skyhawk's Huntington's drug gains Australian provisional approval eligibility

BOSTON, March 3, 2026 /PRNewswire/ -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate critical RNA targets for a series of challenging neurological diseases, announces that Australia's Therapeutic Goods Administration (TGA), responsible for the approval of the safety, quality, and efficacy of therapeutic goods, has determined that the Company's SKY-0515 orally-administered small molecule therapy for the treatment of Huntington's disease meets TGA eligibility criteria for registration via the provisional approval pathway.

Skyhawk delivered its application submission to the TGA for provisional approval today.

"We are grateful that the TGA has determined SKY-0515 for the treatment of Huntington's disease meets its eligibility criteria for potential provisional approval," said Bill Haney, Skyhawk's CEO. "This represents an important first step toward what could be an accelerated path to approval in Australia and the world, offering the possibility of a disease modifying treatment for Huntington's patients who urgently need therapies for this devastating disease."

Treatment with SKY-0515 results in dose-dependent reductions of mHTT and PMS1, key drivers of Huntington's disease pathology. SKY-0515 has demonstrated excellent central nervous system exposure and been generally safe and well tolerated.

Patients receiving SKY-0515 in the Phase 1C trial of SKY-0515 demonstrate mean Composite Unified Huntington's Disease Rating Scale (cUHDRS) improvement from baseline. At nine months this improvement is +0.64 points in a pooled analysis, compared to expected worsening at nine months of cUHDRS in symptomatic patients of -0.73 points, based on propensity score weighting using Enroll-HD and TRACK-HD.

Huntington's disease is a rare, inherited and ultimately fatal neurodegenerative disorder that affects hundreds of thousands of people worldwide, including more than 40,000 symptomatic patients in the United States and over 2,000 Australians.^1,2 There are currently no approved disease-modifying treatments which slow or halt disease progression.²

SKY-0515 is a small molecule RNA splicing modifier developed through Skyhawk's novel RNA-splicing platform, SKYSTAR^®.

SKY-0515 is the first Skyhawk drug in clinical trials.

Skyhawk is developing additional novel drugs to treat neurological diseases for which there are no approved disease-modifying therapies.