Axovant Sciences 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 company has also entered into a research collaboration for the development of five additional gene therapy products in neurological disorders.
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, including autosomal dominant disorders caused by nucleotide repeat expansion.
The lead program – AXO-AAV-OPMD – is in preclinical development and Axovant plans to initiate a placebo-controlled clinical study in 2019.
OPMD is a neuromuscular disease that is caused by mutations in the gene coding for polyA-binding protein nuclear 1 (PABPN1), which can lead to formation of intranuclear inclusion bodies causing muscle cell pathology.
Patients with OPMD may have swallowing difficulties with potentially life-threating consequences, including malnutrition and aspiration pneumonia. OPMD is estimated to affect at least 15,000 patients in North America and Europe, and there are no products approved for treatment of the disease.
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.
The US Food & Drug Administration and European Commission have granted Orphan Drug Designation to AXO-AAV-OPMD for the treatment of OPMD.
As per 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.
In addition, 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 first additional investigational gene therapy product will target the C9orf72 gene, which is associated with amyotrophic lateral sclerosis and frontotemporal dementia.