Skyhawk Therapeutics, a biotechnology company in the clinical stage, has recently received approval from the Australian Human Research Ethics Committees (HREC) to extend the administration of its treatment,
SKY-0515, for up to 12 weeks in patients with
Huntington's Disease (HD). This treatment is part of the company's ongoing Phase 1 clinical trial. Additionally, HREC has approved the use of Volumetric MRI (vMRI) measurements as an exploratory endpoint in the study. This method allows for collecting further data on how SKY-0515 affects the brain regions afflicted by HD.
Sergey Paushkin, the Head of Research and Development at Skyhawk Therapeutics, expressed enthusiasm over the approval. He highlighted that SKY-0515 demonstrated a significant 72% reduction in
huntingtin (HTT) mRNA during its evaluation in healthy volunteers. The prolongation of treatment in HD patients marks an important step forward. Paushkin also expressed gratitude towards HREC for their detailed assessment of the data and for authorizing this extension, as well as the use of vMRI. These approvals are anticipated to broaden the scope of demonstrating SKY-0515’s efficacy and potentially expedite progression to pivotal trials sooner than expected.
SKY-0515 is an innovative, orally administered small molecule developed through Skyhawk's proprietary RNA-splicing platform. It is specifically engineered to reduce levels of both HTT protein and
PMS1 protein, the latter being a significant contributor to somatic CAG-repeat expansion and the pathology of Huntington's Disease.
The Phase 1 clinical study of SKY-0515 is structured to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics in both healthy individuals and those with early-stage Huntington's Disease. This first-in-human trial comprises three segments. Parts A and B focused on healthy volunteers through single and multiple ascending dose studies, respectively, showing a dose-dependent reduction in HTT mRNA with a notable average reduction of 72% at a 9mg daily dose. The treatment was well-tolerated across all doses tested.
Part C of the trial adopts a double-blind, placebo-controlled parallel design to examine two different dose levels of SKY-0515 alongside a placebo in individuals with early-stage HD. This segment seeks to evaluate various parameters, including safety and pharmacodynamics, with ongoing recruitment and preliminary data expected by mid-2025.
Skyhawk Therapeutics continues its focus on discovering and developing revolutionary small molecule therapies aimed at modulating critical RNA targets. Their work seeks to transform treatment options for some of the most challenging diseases. The company leverages its advanced drug discovery platform to evaluate RNA splicing targets and small molecules across diverse therapeutic areas, including
neurodegenerative diseases, metabolic disorders, autoimmune conditions, fibrosis, and oncology.
How to obtain the latest research advancements in the field of biopharmaceuticals?
In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!
