Picking up the pieces left from an Alzheimer’s implosion, Axovant is starting over by diving into gene therapy

After getting blasted by the failure of its lead drug for Alzheimer’s, Vivek Ramaswamy’s Axovant $AXON is going to try and blaze a new path forward by developing a gene therapy for Parkinson’s.

Pavan Cheruvu

The biotech has unveiled a deal in which it will hand over $30 million in cash to Oxford BioMedica for what will now be called AXO-Lenti-PD, a gene therapy designed to spur dopamine production in the brain. And to help fund the next phase, parent company Roivant is buying $25 million worth of shares.

There’s a hefty set of milestones on the table for Oxford BioMedica, adding up to $812 million for their success. And Axovant execs say they’ll be ready to take this gene therapy into the clinic before the end of the year.

Axovant has a long way to go before it can win back the trust of the investors who lost big on its high-wire act on Alzheimer’s. But they are taking the first steps in the journey. And the investors cheered on the move this morning, with Axovant’s shares rocketing up 160%. Oxford Biomedica $OXB,meanwhile, jumped 17% on the news.

“I really see this as the start of pipeline expansion,” Axovant CEO Pavan Cheruvu tells me.

“This” is a troika of genes designed to turn cells into “dopamine factories,” says the CEO. And it makes them one of the few biotechs in gene therapy — alongside Voyager — to go after a neurosciences target.

They’ll be starting on this new drug several years after Oxford BioMedica published positive results from a small study of the gene therapy, which they say demonstrated its ability to provide a durable assistance to patients.

Axovant imploded with spectacular effect following the recent complete failure of its lead effort on Alzheimers, which has taken down one player after the next that has tried to tackle the disease. CEO David Hung subsequently left, taking his crew with him and wiping his hands of the entire company, which went through a painful restructuring. Now Cheruvu is bringing in a new crew of his own to help lead the company in a new direction.

Fraser Wright

The CEO has recruited Fraser Wright — who had a senior role at gene therapy pioneer Spark Therapeutics — as chief technology officer. A former Penn investigator, Wright is also a veteran of the gene therapy program at The Children’s Hospital of Philadelphia, which provided Spark with its initial pipeline in gene therapy.

He’ll be working with other new recruits, including ex-Teva R&D chief Michael Hayden, who is coming on board as a senior scientific adviser at Roivant as well as head of Axovant’s scientific advisory board. Allergan $AGN CMO Gavin Corcoran, meanwhile, is jumping ship to become the new head of R&D at Axovant.


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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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