Skyhawk Therapeutics has announced positive interim results from its phase 1 trial of SKY-0515, an investigational treatment for Huntington’s disease.
The phase 1 clinical trial of SKY-0515 showed that patients receiving SKY-0515 had a mean Composite Unified Huntington’s Disease Rating Scale (cUHDRS) improvement from baseline of 0.64 points at nine months of treatment, where a worsening of 0.73 points would usually be expected. There was a dose dependent reductions of mHTT protein in the blood of 62% at the 9mg dose, and a dose-dependent PMS1 mRNA reduction of 26%.
Huntington’s disease is a rare, hereditary neurodegenerative disorder affecting more than 40,000 patients in the US and hundreds of thousands globally. There are no approved treatments to inhibit the progression of the disease, which is ultimately fatal.
SKY-0515 is an investigational small molecule RNA modulator developed using SKYSTAR, Skyhawk’s RNA-modulating platform.
Research has found SKY-0515 has a favourable safety and tolerability profile, and has good central nervous system exposure.
Skyhawk has announced the global expansion of its phase 2/3 FALCON-HD trial of SKY-0515. Over 90 patients have now been dosed with SKY-0515, the first drug from Skyhawk to reach clinical trial stage.
Skyhawk projects that a pipeline of other small molecule drugs, designed to treat rare neurological diseases with no current approved disease modifying therapies, will reach the clinic by late 2027.
Ed Wild, professor of neurology at University College London, said: “These open-label trial results, due to be validated in the ongoing placebo-controlled FALCON-HD trial, give an expectation of meaningful impact for people living with HD across the world – for whom an orally administered HTT-lowering treatment such as SKY-0515 will be truly transformative.”
Sergey Paushkin, head of R&D at Skyhawk, said: “This interim data represents an important milestone for SKY-0515 and highlights the power of Skyhawk’s platform to deliver first-in-class small molecules for devastating diseases with no approved disease-modifying therapies.”