Skyhawk Therapeutics has announced final 15-month results from its Phase 1/2 clinical trial of SKY-0515, an investigational oral treatment for HD.Â
After 15 months, participants on the active medication were doing better on the cUHDRS (Composite Unified Huntington’s Disease Rating Scale) than a comparison group of untreated people. This difference was statistically significant and was seen across daily function, movement, processing speed and reading speed.
For the HD community, these results are encouraging because they suggest that SKY-0515 may have the potential to slow or alter the progression of Huntington’s disease, rather than only treating individual symptoms. The study also showed consistent reductions of more than 60% in mutant huntingtin protein (mHTT), an important biological marker of HD.
The Composite Unified Huntington’s Disease Rating Scale (cUHDRS) is a metric designed to capture several important aspects of how HD affects a person’s life. By combining measures of motor symptoms, daily functioning and cognitive abilities, it provides a broader picture of disease progression. That’s why researchers use it to judge whether a treatment could change the course of HD.
This was a small, early-stage study and SKY-0515 remains investigational. However, the findings represent an important step in the search for treatments that can meaningfully change the course of the disease. In Dina’s words: I am feeling super encouraged about this, the fact that it is a pill that we can take once a day, and if your mental clarity is improved, if you’re able to write checks a little longer, or able to cook on your own a little longer, fantastic.
And further encouraging is that the larger FalconHD trial is already underway. 400 participants, worldwide, now to determine if this findings can be verified in a larger population.
Read the full press release.
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