SELECT-HD Update, PIVOT-HD Update, PT Guidelines

Wave Life Sciences shares SELECT-HD Update 

Earlier this week, Wave Life Sciences shared the results of SELECT-HD, a small phase 1b/2a clinical trial that tested the safety and efficacy of WVE-003. This spinally-delivered ASO drug targets a specific code, SNP3, that is present only on the allele containing the expanded copy of the huntingtin gene, which allows it to lower only the harmful form of huntingtin protein while preserving healthy huntingtin in people with HD. About 40% of people with HD have SNP3, so prospective participants had to take an additional genetic test to confirm their eligibility for the study before joining.  

Wave’s results suggest that WVE-003 is safe and was well-tolerated by individuals in the study. The data also show that WVE-003 lowered expanded huntingtin protein in participants while healthy huntingtin protein levels were stable. Excitingly, this means that the study met its primary endpoints of determining that the drug is safe and able to lower expanded huntingtin protein in people with HD, and even showed some preliminary signs of clinical benefit. Wave will work with regulators to determine the best path of development for WVE-003, and will plan a larger study of about 150 people to see whether WVE-003 slows onset of HD. You can read the community letter from Wave here, and check out the full analysis of this news from HDBuzz here.  

HD Buzz on PIVOT-HD 

Last week, PTC Therapeutics shared an exciting update from PIVOT-HD, the Phase 2 study of PTC518 for HD. We shared the news in last week’s blog, but the HD Buzz team shared additional explanation of what this news means for PTC-518 moving forward. Read their analysis here.  

New PT Guidelines from the EHDN 

The Physiotherapy Working Group of the European Huntington’s Disease Network recently developed a set of resources for people with HD. Physical Therapy experts from around the world collaborated to assemble these guidelines on movement, activity, and physical therapy for people living with HD. Guidelines for clinicians and people with HD are available on the EHDN website, along with the research that supports the Working Group’s recommendations. Read more here.