SAN FRANCISCO—J.P. Morgan week one year ago wasn’t Axovant’s finest hour. But since Pavan Cheruvu—formerly Roivant’s chief people officer—took Axovant’s helm in February 2018, the company has been plugging away at becoming a gene therapy player.
“When I joined, at the outset, I had a vision of building a gene therapy company that would be an entirely new company than what Axovant had been before … The small molecule past is now squarely behind us,” said Cheruvu at the J. P. Morgan Healthcare Conference.
It is a change from the “incremental” approach Axovant previously took: in-licensing Alzheimer’s castoffs from Big Pharma. This time around, Axovant wants to be transformative (the word I’ve heard most frequently at J. P. Morgan).
So what does that mean?
Axovant picked gene therapies because of the potential of a one-time curative therapy that could improve upon current treatments for neurodegenerative disease and rare, fatal pediatric diseases.
In June, Axovant licensed a gene therapy for Parkinson’s disease, known as AXO-Lenti-PD from Oxford Biomedica and hired on a new chief technology officer, Fraser Wright, who cofounded Spark Therapeutics, where he was involved in the design and development and ultimately, the commercialization of the gene therapy Luxturna. With AXO-Lenti-PD, Axovant hopes to turn patients’ brain cells into “native factories” of dopamine, which is deficient in the brains of Parkinson’s patients. This would avoid certain problems with the current treatment. Oral levodopa boosts a patient’s dopamine levels, but can lead to dyskinesia—uncontrolled involuntary movements—and other off-target effects, such as psychosis, Cheruvu said.
In December, the company dumped the last vestige of the old Axovant—nelotanserin, which had proved disappointing as a treatment for Lewy body dementia. In the intervening months, Axovant built up its pipeline and team, picking up gene therapies for the pediatric diseases GM1 gangliosidosis and GM2 gangliosidosis, as well as for oculopharangeal muscular dystrophy. It also stacked its leadership with gene therapy veterans from Spark, Novartis and Sarepta.
“The team today has decades of gene therapy expertise … we’ve assembled folks who’ve gotten gene therapies approved and on the market,” Cheruvu said.
In 2019, Cheruvu hopes to “pull forward” all of Axovant’s clinical-stage programs, with data coming for its two most advanced therapies in the first quarter. And beyond that?
“In 10 years’ time, Axovant will be the leader in not only gene therapies, but gene editing and related modalities that could offer one-time cures,” Cheruvu said. Eventually, he wants Axovant to be the leader in gene therapy and gene editing in a "modality-agnostic way."
What he means is, instead of making a particular approach—like gene therapy—the centerpiece of the company, Axovant will view those methods as a means to drive the real focus: "meaningful change in patients’ lives.” Cheruvu acknowledged that virtually everyone will say that, adding that Axovant would "make it real" by making patients, advocacy groups and family members a part of its drug development process. We'll check back in on Axovant at J.P. Morgan 2020.