SKY-0515 is a small molecule RNA splicing modifier developed through the company’s novel SKYSTAR® platform. Having successfully advanced through single asending dose, SKY-0515 is advancing into the Multiple Ascending Dose portion of its phase 1 study. Huntington’s disease is a fatal neurological disease with no curative therapies.

WALTHAM, MA, USA I February 28, 2024 I Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule RNA splicing modifiers, today announced that the Phase 1 multiple ascending dose (MAD) portion of its Phase 1 clinical study has now begun in Australia for the company’s SKY-0515 small molecule candidate targeting Huntington’s disease (HD). Huntington’s is caused by a toxic ‘CAG’ repeat in the huntingtin (HTT) gene, causing mutated huntingtin protein. SKY-0515 is designed to modify the RNA expression of HTT, reducing the production of mutated protein that leads to disease progression in Huntington’s patients.

“We are excited to announce that after successfully advancing in our SKY-0515 Phase 1 single ascending dose study, the safety review committee has recommended initiation of the multiple asending dose portion of our Phase 1,” said Douglas Faller, Chief Medical Officer of Skyhawk Therapeutics. “Following this, we expect to begin enrolling Huntington’s patients, who presently have few therapeutic options and none that reverse or slow disease progression. SKY-0515 has demonstrated strong activity in our preclinical models which we believe will translate to patient benefit.”

There are approximately 30,000 symptomatic Huntington’s disease patients in the US alone and many more who are pre-symptomatic. There are currently no approved drugs that reverse or slow disease progression for Huntington’s patients. SKY-0515 is a small molecule which directly targets HTT RNA, is dosed orally and offers brain penetration and distribution in peripheral tissues which may provide important benefits to patients. It is the first Skyhawk-developed drug to enter clinical trials.

Skyhawk Therapeutics’ SKY-0515 Phase 1 clinical trial is a first-in-human, multi-part, randomized, double-blind, single and multiple ascending dose study designed to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamic activity of SKY-0515 monotherapy in healthy volunteers and, subsequently, in Huntington’s patients. Additional information about the trial can be found at: Australian New Zealand Clinical Trials Registry (

About SKY-0515
Skyhawk Therapeutics is developing a small molecule RNA splicing modifier for potential treatment of Huntington’s disease through the company’s novel SKYSTAR® platform. SKY-0515 is designed to reduce the production of the mutated huntingtin protein which results in disease progression. SKY-0515’s predicted PK allows for daily, oral dosing, and the molecule is designed for brain penetration and distribution in peripheral tissue.

About Huntington’s disease
Huntington’s disease is an inherited disease that causes the progressive degeneration of nerve cells in the brain. Huntington’s disease has a wide impact on a person’s functional abilities and usually results in movement, cognitive and psychiatric disorders, and is ultimately lethal. Huntington’s disease symptoms can develop at any time, but they often first appear when people are in their 30s or 40s. Medications are available to help manage the symptoms of Huntington’s disease, but there is no cure for Huntington’s disease, and currently no approved drugs that delay the disease onset or slow disease progression.

About Skyhawk
Skyhawk develops therapies using its novel SKYSTAR® platform, building small molecules that modify RNA expression to provide breakthrough treatments for patients. Skyhawk has productive collaborations across a broad range of disease areas, from neurodegenerative disease to oncology.

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SOURCE: Skyhawk Therapeutis