uniQure Updates, SkyHawk Updates, NIH ENGAGE

uniQure shares update on AMT-130   

Earlier this week, uniQure shared an update on their clinical study of AMT-130, an experimental gene therapy in development to treat HD. AMT-130 is delivered directly to brain tissue in a single dose by an MRI-guided brain surgery. 39 participants are enrolled in the Phase I/II studies that are taking place in the U.S. and Europe and have received either a low dose or high dose of AMT-130, or a placebo surgery to confirm the procedure is safe. uniQure shared a 24-month data update on 21 of these participants who have received either a low or high dose of the drug.  

The main goal of a Phase I/II trial is to test safety, and so far, AMT-130 has a strong safety profile. The study also looked at NfL, a structural protein found in brain cells that can be an indicator of brain injury which tends to increase over the course of HD. After a spike in NfL from the surgery, NfL levels fell below what would be expected from the natural progression of HD. This indicates that AMT-130 is not causing harm, and may in fact be slowing the decline in brain health that is expected in HD. Lastly, the data shared show early signs that clinical progression of HD may be slowed by AMT-130 in individuals who received the highest does of the drug. To read the full press release from uniQure, click here. For a fuller picture of what this update means, check the summary from HDBuzz here.  

SkyHawk Therapeutics announces early positive results of SKY-0515 

Also this week, Skyhawk Therapeutics announced positive topline results in their Phase 1 study of SKY-0515, an experimental treatment for HD. This drug is a splicing modifier that targets both huntingtin (HTT) protein and PMS1 protein, which is a known driver of somatic expansion in HD. The drug lowers levels of these proteins by interfering with the message that triggers their production, mRNA. The phase 1 study is taking place in three parts with the goal of testing the safety and target engagement of SKY-0515. Parts A and B tested different doses of the drug in people who do not have HD. All doses were well tolerated, and strong, dose-dependent lowering of HTT mRNA was observed. With these positive results, Skyhawk will move forward with Part C of the Phase 1 study in Q3 of 2024 to assess the safety and target engagement of SKY-0515 in people with early-stage HD. Results are expected in Q2 of 2025. To read the full press release from Skyhawk Therapeutics, click here. For more details about this news, check out this article from HDBuzz.

NIH Announces ENGAGE Initiative 

Last month, the NIH Office of Science Policy announced the launch of the ENGAGE Initiative with the goal of incorporating public voices in the development of clinical trials. By amplifying input from patients, caregivers, advocates, and other research stakeholders, the NIH aims to design more meaningful research studies with maximal long-term benefit. The NIH will host a webinar and virtual listening session about this initiative on July 17th at 1PM ET; register to join here.