Rare Daily Staff

Axovant Sciences said it has licensed exclusive global rights to an experimental “Silence-and-Replace” gene therapy program from Benitec Biopharma for the treatment of oculopharyngeal muscular dystrophy.

Oculopharyngeal muscular dystrophy is an inherited muscular disease that presents between the ages of 40-70. It is characterized by progressive swallowing difficulty, eyelid drooping, and weakness of the proximal extremities. Swallowing difficulties can have life-threating consequences, including malnutrition and aspiration pneumonia. As the disease progresses, the dysphagia becomes more severe and other muscles may become involved. OPMD affects about 15,000 people in North America and Europe. There are no products approved for the treatment of the condition.

The Silence-and-Replace gene therapy technology is designed to deliver a combination of DNA-directed RNA interference along with a functional copy of the gene in a single vector construct. The company said the approach is applicable to various genetic diseases. The AXO-AAV-OPMD program is being developed as a one-time treatment for OPMD.

The U.S. Food and Drug Administration and European Commission have granted Orphan Drug designation to AXO-AAV-OPMD for the treatment of OPMD.

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 experimental gene therapy products, as well as payments tied to development, regulatory, and commercial sales milestones.

In addition, Benitec will receive 30 percent 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 first additional investigational gene therapy product will target the C9orf72 gene, which is associated with amyotrophic lateral sclerosis and frontotemporal dementia.

“OPMD is a debilitating, potentially fatal disease that affects adults in the prime of their careers, and no approved treatment options are currently available,” said Pavan Cheruvu, CEO of Axovant. “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.”

July 9, 2018
Photo: Pavan Cheruvu, CEO of Axovant

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