Drug Development

Plowing ahead after its latest setback, Axovant nabs two assets to beef up gene therapy pipeline

Fresh from the spectacular failure of its dementia drug, nelotanserin, which has been pushed to the scrap heap along with its much-hyped Alzheimer’s asset, Axovant $AXON is moving on fast at the behest of its deal hungry patron Vivek Ramaswamy.

On Thursday, the company said it had struck a deal to license the global rights to develop and market two gene therapy programs designed to address two progressive and fatal pediatric lysosomal storage disorders: GM1 gangliosidosis and GM2 gangliosidosis – also known as Tay-Sachs and Sandhoff diseases – from the University of Massachusetts Medical School.

The two assets add heft to the London-based company’s existing pipeline of gene therapies. A mid-stage trial testing its existing gene therapy for Parkinson’s is set to read out data in March.

Meanwhile, a program evaluating the newly licensed AXO-AAV-GM2 in GM2 gangliosidosis is already underway, with initial data expected in the first quarter of 2019.  The first GM1 gangliosidosis patient to be dosed with AXO-AAV-GM1, the other asset, will likely occur next year in an investigator-initiated clinical trial conducted at the NIH.

Concurrently, Axovant also announced an offering of 30 million shares, at $1 a pop – a far cry from the over $20 a share it was trading at before the announcement of the nelotanserin failure.


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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
Marketing Associate - Demand Generation
Catalytic Data Science Charleston, SC
Associate Principal, Life Sciences Partnerships
Flatiron Health New York City or San Francisco

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