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- Skyhawk Therapeutics has advanced to the multiple ascending dose portion of its Phase 1 study evaluating SKY-0515, a small molecule RNA targeting for Huntington's disease
SKY-0515 is a small molecule RNA splicing modifier developed through the company's novel SKYSTAR ® platform. Having successfully progressed through a single ascending 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, Mass., Feb. 28, 2024/PRNewswire/ -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule RNA splicing modifiers, today announced that Phase 1 multiple ascending dose (MAD)'s Phase 1 clinical study has already begun in Australia for the company's SKY-0515. Small Molecule Candidate Targeting Huntington's Disease (HD) Huntington's disease is caused by a toxic 'CAG' repeat in the Huntingtin (HTT) gene, which causes mutated Huntingtin protein. SKY-0515 is designed to modify HTT RNA expression, reducing production of the mutated protein that leads to disease progression in Huntington's patients.
"We are pleased to announce that after successfully advancing our Phase 1 single ascending dose study SKY-0515, the safety review committee has recommended initiation of the multiple ascending dose portion of our Phase 1," said Douglas Faller. , chief medical officer of Skyhawk Therapeutics. "Following this, we hope to begin enrolling Huntington's patients, who currently have few therapeutic options and none that reverse or slow disease progression. SKY-0515 has demonstrated strong activity in our preclinical models that we believe will translate into benefit for the patient.
There are approximately 30,000 symptomatic patients with Huntington's disease in the US alone and many more who are presymptomatic. There are currently no approved medications that reverse or slow disease progression in Huntington's patients. SKY-0515 is a small molecule that directly targets HTT RNA, is dosed orally, and offers brain penetration and peripheral tissue distribution, which may provide significant benefits to patients. It is the first drug developed by Skyhawk to enter clinical trials.
Skyhawk Therapeutics' Phase 1 SKY-0515 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. 0515 as monotherapy in healthy volunteers and, subsequently, in Huntington's patients. Additional information about the trial can be found at: Australian and New Zealand Clinical Trials Registry (https://www.anzctr.org.au).
About SKY-0515
Skyhawk Therapeutics is developing a small molecule RNA splicing modifier for the 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 causes disease progression. The anticipated pharmacokinetics of SKY-0515 allow for daily oral dosing, and the molecule is designed for brain penetration and distribution into peripheral tissue.
About Huntington's disease
Huntington's disease is an inherited disease that causes progressive degeneration of nerve cells in the brain. Huntington's disease has a broad impact on a person's functional abilities and typically results in movement, cognitive and psychiatric disorders and is ultimately fatal. Symptoms of Huntington's disease can appear at any time, but often first appear when people are in their 30s and 40s. There are medications available to help manage the symptoms of Huntington's disease, but there is no cure for the disease and there are currently no approved medications that delay the onset of the disease or slow its progression.
About Skyhawk
Skyhawk develops therapies using its novel SKYSTAR® platform, building small molecules that modify RNA expression to provide innovative treatments to patients. Skyhawk has productive collaborations in a wide range of disease areas, from neurodegenerative diseases to oncology.
For more information, visit: www.skyhawktx.com https://www.linkedin.com/company/skyhawk-therapeutics/ https://twitter.com/Skyhawk_Tx
SKYHAWK MEDIA CONTACT: Maura McCarthy maura@skyhawktx.com
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