In August 2022, BrainVectis, a subsidiary of AskBio, gained approval from the French National Agency for Safety of Medicines and Health Products (ANSM) to conduct a phase 1/2 trial with a drug called BV-101.
The trial will look at the safety and efficacy (ability to produce the desired result) of BV-101 in adult patients with early-stage Huntington’s Disease. BV-101 will be given (one time) through MRI-guided neurosurgical techniques directed to target tissues in the basal structures of the brain. In preclinical studies in mice, BV-101 showed the ability to repair the essential cholesterol pathway (which is damaged in HD patients), provide neuroprotection, and restore neuronal function by delivering a crucial enzyme in the brain which is lowered in people with Huntington’s Disease.
The trial will be in 2 parts with approximately 12-18 participants. One part is to find the correct dose and 2nd part, expansion of the trial. The exact number of participants will depend on the safety of part 1. This trial is called an open-label, meaning there is no placebo group. The participants will be rigorously monitored for 52 weeks and follow up of 4 years and is expected to take place in two sites in France.