Skyhawk Therapeutics has shared a press release with positive news about SKY-0515, the experimental drug they developed and are studying for Huntington’s disease (HD). This first study was a Phase 1 trial, which mainly checks whether a drug is safe and how it works in the body.

SKY-0515 is taken as a pill, which makes it easy to use. In this initial trial, patients who took SKY-0515 showed a big reduction in two harmful proteins linked to HD:

• Mutant huntingtin (mHTT) – the main protein that causes HD. SKY-0515 lowered it by as much as 62% after 84 days at the higher dose.
• PMS1 – another protein thought to make HD worse was significantly lowered too.

The drug also reached the brain well, which is crucial for treating HD, and so far it appears to be safe and well tolerated.

Because of these results, Skyhawk has already moved forward with a larger Phase 2/3 clinical trial called FALCON-HD, involving 120 people with HD in Australia and New Zealand. This study will test the drug in more patients and over a longer period. These next phases of the study are expected to be completed by the end of 2027.

While it is still early days, and much more testing is needed before SKY-0515 could become an approved treatment, these findings bring new hope for families living with HD.

Read the full press release here.