Focused on rebuilding the pipeline, Axovant's new crew assembles another preclinical gene therapy deal
Just a few weeks after Vivek Ramaswamy’s Axovant $AXON got a big boost out of its licensing pact with Oxford BioMedica for what is now called AXO-Lenti-PD, a gene therapy designed to spur dopamine production in the brain, the biotech is continuing its makeover with a new deal adding a fresh set of gene therapies to the pipeline.
This time Axovant is turning to the Australian biotech Benitec $BNTX for a double barreled therapy that delivers an RNAi therapy alongside a replacement gene inside the same vector to treat oculopharyngeal muscular dystrophy, a rare condition that creates severe swallowing issues. And Axovant gets 5 more programs in the package, which comes with an economy-sized upfront of $10 million.
One of those additional programs includes a treatment targeting the C9orf72 gene, which is associated with amyotrophic lateral sclerosis and frontotemporal dementia.
Benitec’s shares jumped 41% in pre-market trading.
Axovant’s share price was flattened by the recent failure of a late-stage program for Alzheimer’s, Ramaswamy’s first big bet in biotech. This time, though, Axovant is starting over in preclinical mode, building from the ground up rather than gambling on reviving a failed therapy off Big Pharma’s shelves. After Ramaswamy boasted that he had found a quick way to hurtle toward new drug approvals, the new crew at Axovant — replacing David Hung and his confederates — are taking it all one step at a time.
“The Silence-and-Replace technology is a unique approach in gene therapy, using a single vector to suppress mutant protein production while also restoring expression of the functional protein, and could be an elegant solution to tackling autosomal dominant genetic disorders,” stated Fraser Wright, chief technology officer of Axovant.