Bayer has become the latest in a long line of drug developers to give up on Huntington’s disease after the German pharma giant canned its clinical-stage gene therapy for the disease.
The company is ending work on the recombinant adeno-associated virus gene therapy, dubbed BV-101, it revealed in its third-quarter earnings presentation (PDF). Bayer’s AskBio unit launched a phase 1/2 trial of the med in adults with the disease back in 2022.
At the time, AskBio touted BV-101 as standing out from the crowd by aiming to restore cholesterol metabolism, reduce mutant huntingtin and improve neuronal function without affecting levels of normal huntingtin protein in cells.
When asked this morning by Fierce Biotech why the program had been scrapped, a spokesperson for Bayer said the pharma “routinely reviews our pipeline to focus our resources on the most promising assets from a strategic point of view.”
“We remain committed to advancing improvements in the field of gene therapy,” they added.
Huntington’s is a rare inherited disease that causes the progressive degeneration of nerve cells in the brain, leading to impairment of physical and mental abilities, as well as psychiatric disorders. Treatments are available to manage symptoms, but there is currently no disease-altering therapy on the market.
Bayer is hardly the first biopharma to have given up in Huntington’s. Last month, Takeda walked away from a collaboration that was at that point centered on an antisense oligonucleotide for the disease. In 2022, Novartis canned work on its splicing modulator branaplam in Huntington’s, which followed the failure of Roche and Ionis Pharmaceuticals’ antisense drug tominersen in phase 3.
Despite also suffering some tricky readouts early on its program, UniQure’s decision to persevere with its own gene therapy in Huntington’s appeared to have paid off this year when a slice of 24-month data demonstrated a slowing of disease progression.