Axovant Sciences Ltd. (NASDAQ: AXON) shares saw a handy gain on Monday after the company announced that it has licensed exclusive global rights to an investigational silence-and-replace gene therapy program from Benitec Biopharma for the treatment of oculopharyngeal muscular dystrophy (OPMD).
The firm also has entered into a research collaboration for the development of five additional gene therapy products in neurological disorders.
Under the terms of the agreement, 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, as well as payments tied to development, regulatory and commercial sales milestones. Additionally, Benitec will receive 30% of the net profits on worldwide sales of AXO-AAV-OPMD and tiered royalties on the other gene therapy products that result from this collaboration.
The silence-and-replace gene therapy technology is designed to deliver a combination of DNA-directed RNA interference (silence) along with a functional copy of the gene (replace) in a single vector construct. This approach is applicable to various genetic diseases.
Pavan Cheruvu, M.D., CEO of Axovant, commented:
This expansion of Axovant’s pipeline further demonstrates our commitment to advancing innovative gene therapies for serious neurological diseases. OPMD is a debilitating, potentially fatal disease that affects adults in the prime of their careers, and no approved treatment options are currently available. Quality-of-life in patients with OPMD can be impaired due to proximal muscle weakness, swallowing difficulties, aspiration pneumonia and malnutrition, and no approved treatment options are currently available. AXO-AAV-OPMD directly targets the underlying genetic defect that causes this disease using Silence-and-Replace technology, and I am excited about the potential of our gene therapy program for patients suffering from OPMD.
Shares of Axovant were last seen up about 4% at $2.68, with a consensus analyst price target of $6.00 and a 52-week trading range of $1.02 to $27.98.