Through all the challenges 2021 had in store, the resilience of the HD community has continued to inspire HDSA in our collective fight to end HD. To review this year in HD research and things to look forward to in 2022, check out HDSA’s annual research report, The Marker, as well as HDSA’s research webinars, available on our YouTube channel.
Novartis receives fast-track designation for branaplam
Last week, Novartis announced that they have received fast-track designation for branaplam, a huntingtin-lowering therapy that can be taken by mouth. This track is reserved to speed up development and review of experimental medications that have the potential to improve treatment options for dire health conditions and fill unmet medical needs.
Branaplam lowers levels of huntingtin protein in the body by interfering with the genetic message that triggers the body to make huntingtin protein. Fast Track designation will provide Novartis the opportunity to collaborate with the FDA for fast, safe, and efficient development of branaplam for treatment of HD. Novartis is planning to begin recruiting participants in the US to participate in VIBRANT-HD, a phase 2b study of branaplam in adults with early-stage manifest HD, in the near future.
Sage Therapeutics announces positive preliminary data for SAGE-718
Sage Therapeutics announced last week that they’ve received positive preliminary data for the efficacy and safety of SAGE-718, a first-in-class drug for cognition in HD. Early studies have shown that SAGE-718 was well-tolerated and led to an improvement in executive performance in participants with HD and Parkinson’s Disease (PD). A Phase 2 study of SAGE-718 will open for enrollment in the US in the new year.
Year-end wrap-up from Gene Veritas
In his most recent blog post, Gene Veritas, also known as Kenneth Serbin, recounted a year of ups and downs for the HD community. To read his summary of 2021, including reflections of another year in the COVID-19 pandemic, the halt of GENERATION HD-1, promising results from KINECT-HD, and more, click here.