Axovant buys more gene therapies, setting it up for a busy 2019

Axovant has licensed two gene therapies from the University of Massachusetts Medical School (UMMS). The deal accelerates Axovant’s evolution into a gene remedy specialist by giving it management of two belongings which might be in or close to the clinic.

Following the failures of intepirdine and nelotanserin, Axovant purged its pipeline of small molecules and got down to purchase a roster of gene therapies. The pivot obtained underway with a deal for clinical-phase Parkinson’s illness gene remedy AXO-Lenti-PD in June and continued with the licensing of a gene remedy therapy for oculopharyngeal muscular dystrophy (OPMD) the next month.

While the OPMD gene remedy is because of enter human testing subsequent yr, the pivot nonetheless left Axovant’s medical pipeline wanting a little gentle. The newest gene remedy deal goes a way towards addressing that difficulty.


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Axovant is paying UMMS $10 million upfront for the rights to 2 gene therapies designed to deal with GM1 and GM2 gangliosidosis, lysosomal storage problems that trigger the doubtless deadly construct up of poisonous gangliosides. The deal commits Axovant to up to $24.5 million in growth and regulatory milestones, plus up to $39.8 million in industrial milestones and tiered mid-single-digit royalties. 

The more superior of the 2 gene therapies, AXO-AAV-GM2, is already in medical growth in sufferers with GM2 gangliosidosis, a group of uncommon problems often known as Tay-Sachs and Sandhoff illnesses. Axovant expects to have information from the trial within the first quarter of subsequent yr. 

UMMS’ different gene remedy, AXO-AAV-GM1, is because of transfer into the clinic within the first half of subsequent yr. The trial will likely be carried out on the NIH, which has accrued a repository of pure historical past information on the illnesses Axovant thinks it may use as a management in registrational research. Axovant expects to have preliminary information from the GM1 trial within the second half of subsequent yr.

The addition of two medical readouts to Axovant’s schedule for 2019 means the corporate’s calendar is now pretty nicely stocked with probably enterprise boosting occasions. Axovant’s different huge second will are available March, when it is because of launch medical information on its Parkinson’s gene remedy.  

With medical trial exercise ramping up once more, Axovant is making a public offering of its stock. At the time of writing, Axovant is but to say how a lot it hopes to boost, however the shelf registration masking the providing permits the sale of up to $750 million in stock. Axovant ended September with $91 million in money, having run up working bills of $81 million over the primary six months of the yr.

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