Another Vibrant Possibility for Huntington’s Disease Treatment:

Novartis Branaplam

Written by the Moving Forward Team on March 17th2022

Novartis Pharmaceuticals is recruiting participants for their phase IIb clinical trial called VIBRANT-HD. This study will investigate the huntingtin lowering drug Branaplam, which was originally developed for Spinal Muscular Atrophy, a devastating paediatric genetic disease.

Novartis will test Branaplam in patients with early manifest HD to check the safety of the drug in HD and learn what is the right dose to lower huntingtin, the harmful protein. The goal of this study is to identify the right dose of Branaplam (meaning that is safe and well-tolerated) to slow down or stop HD and promote clinical improvements in patients.  Different patients will receive different doses of the drug and will go through several clinical assessments, so that Novartis can understand what dose works best.

Branaplam will be given as a liquid for patients to drink once a week. The clinical trial will take place in Europe and North America and will involve approximately 75 patients with early HD, between 25 and 75 years. The study will evaluate the safety of lower doses before proceeding to higher doses of Branaplam. Then, after the optimal dose is defined, all participants will be given Branaplam for about 1 year while attending clinic visits every 4 weeks. After this time, the trial may be amended to provide Branaplam beyond 1 year or a separate extension study may be initiated.

Branaplam is being developed as “a potential first in class orally administered disease modifying therapy for HD”. So there are high expectations for this new drug, which was recently granted a Fast Track designation by the US regulator Food and Drug Administration. This label facilitates and accelerates the development of drugs to treat serious conditions and fill unmet medical needs – such as the case of discovering an effective drug that can slow down or stop HD.

This trial will involve three Spanish hospitals (Hospital Universitario Ramón y Cajal in Madrid, Hospital de la Santa Creu i Sant Pau and Hospital Clinic both in Barcelona). In two of them, patient recruitment has already begun – Hospital Universitario Ramón y Cajal and Hospital Clinic.  We truly thank the effort and generosity of these volunteers who, in the first phase of this trial, will go through a few invasive tests, such as regular lumbar punctures to measure the cerebrospinal fluid. This will facilitate the treatment procedures in the future, because if the trial progresses successfully, future patients can have a drug that is comfortably administered orally, through a liquid.

Interestingly, the online survey about research perceptions and experiences made by the Moving Forward team some time ago showed that the route of drug administration had a strong influence on the willingness to engage in studies and trials. We found that the invasiveness of the procedures to deliver the experimental drug was one of the main factors preventing people at risk for HD and people with premanifest HD from taking part in research. Thus, these specific HD groups would more easily comply with a treatment with a drinkable liquid than with a treatment with an injection. Therefore, we believe that Branaplam can be a breakthrough in the HD world in many ways.

After COVID restrictions and all its consequences to HD healthcare and research across the world, VIBRANT-HD is one of the most recent HD clinical trials. For this reason, it is like a breath of fresh air for the global HD community, which will always be sincerely grateful for the commitment and personal efforts of both patients and their families in the search for an effective treatment for Huntington’s Disease. These persons help us keep our hope and faith in a future without HD!

To learn more about this exciting new trial, the European Huntington Association visited Novartis in Switzerland. Check the interesting video about it!