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Axovant, Benitec Gain on Licensing Deal for Gene Therapy

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Shares of Axovant Sciences AXON gained after it announced a licensing agreement with Benitec Biopharma BNTC.

Strategic Terms of the Agreement

Per the agreement, Axovant licensed exclusive global rights to an experimental Silence-and-Replace gene therapy program from Benitec Biopharma for the treatment of oculopharyngeal muscular dystrophy (OPMD). Axovant will obtain rights to AXO-AAV-OPMD, which will utilize proprietary Silence-and-Replace technology.

AXO-AAV-OPMD is an adeno-associated viral (AAV) vector gene therapy delivered via a one-time intramuscular administration, which both silences the mutant PABPN1 gene and replaces it with a functional copy. It is currently in preclinical development stage. Axovant plans to initiate a placebo-controlled clinical study in 2019.

The candidate enjoys orphan drug status in both the United States and Europe for the treatment of OPMD. 

Both the companies have also entered into a research collaboration for the development of five additional gene therapy products in neurological disorders. 

Financial Terms of the Agreement

Per the terms, Axovant will pay Benitec an upfront payment of $10 million for rights to the AXO-AAV-OPMD program and five additional investigational gene therapy products along with milestone payments. Additionally, Benitec is entitled for 30% of the net profits on worldwide sales of AXO-AAV-OPMD and tiered royalties on other gene therapy products that result from this collaboration.

The first additional investigational gene therapy product resulting from the collaboration will target the C9orf72 gene, which is associated with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Our Take

The agreement with Benitec will bolster Axovant’s pipeline of innovative gene therapies for serious neurological diseases. We are impressed with Axovant’s efforts to develop its pipeline in recent times.

Last month, shares of Axovant gained after the company announced that it has licensed the exclusive worldwide rights to develop and commercialize AXO-LentiPD from Oxford BioMedica for Parkinsons’ disease. AXO-LentiPD, formerly OXB-102, is an experimental gene therapy being developed for Parkinson’s disease, which delivers three genes encoding a critical set of enzymes required for dopamine synthesis in the brain. Axovant plans to initiate a phase I/II dose escalation study on AXO-LentiPD in patients with advanced Parkinson’s disease, by the end of 2018.

Axovant’s stock have gained 12.4% in the last six months compared with the industry’s decline of 3.7%. Shares of Benitec gained on the deal as well.


Gene therapy has gained a lot of attention of late as quite a few companies are investing a lot of their R&D spend for developing such candidates for the treatment of various diseases.

Last month, Solid Biosciences SLDB announced that the FDA has lifted the clinical hold on its phase I/II trial, IGNITE DMD, for its experimental candidate, SGT-001, which is a microdystrophin gene transfer being evaluated for the treatment of Duchenne muscular dystrophy (DMD).

uniQure QURE is another gene therapy company, which is advancing a pipeline of proprietary and partnered gene therapies to treat patients with liver/metabolic, central nervous system and cardiovascular diseases.

Zacks Rank

Axovant carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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