Novartis is developing an experimental HD therapy called branaplam which has the potential to lower huntingtin when taken by mouth. Branaplam was originally developed for children with a rare and severe disease called spinal muscular atrophy. It was later discovered to lower huntingtin as well, leading to Novartis testing it in healthy adults in a Phase I safety trial.  

Today Novartis shared a community letter with an update on their progress rolling out a Phase 2 clinical trial called VIBRANT-HD. The trial will take place in Europe and North America, and will involve 75 participants who have symptoms of HD. Important to note is that sites have only begun screening in Europe; so far in France, Germany, Hungary, and Spain. Additional information will be added periodically at this clinical trials link, and North American sites will be listed at in the future, as they begin screening. The potential for an oral therapy that could target the source of HD is an exciting one, and this next step will be important to determine the proper dosing of branaplam to determine if it can accomplish safe and effective huntingtin-lowering in adults with HD. 

HDBuzz breakdown: Roche shares first round of findings from GENERATION HD-1 

Last week, Roche shared the first round of findings from the GENERATION HD-1 study with the HD community. Since dosing of tominersen was halted in March of 2021, researchers have been collecting and analyzing the plethora of data that was collected in the trial. While tominersen did not achieve the primary endpoint of the study, to slow the progression of HD in its participants, the question remains as to whether tominersen may offer some benefit to a subset of participants in the trial. Representatives from Roche addressed this question and others in three webinars over the past week; you can access the recording of HDSA’s community-centered discussion here. In their most recent post, the editors of HDBuzz have broken down the information shared in these forums to give us the highlights of the conversation: what have we learned so far, and where do we go next. To read the full article from HDBuzz, click here.  

Researcher Spotlight: Richard Hickman, MD  

2019 Human Biology Project Fellow, Dr. Richard Hickman, gave HDSA the scoop on his fascinating research studying the ways brain tissue changes over the course of HD. To learn more about Richard’s findings, how he became interested in the neuropathology of HD, and where he is now, read the full spotlight article here.