Axovant licenses Benitec muscular dystrophy drug, continuing pivot toward gene therapies  | Bio Tech

Axovant has picked up global rights to Benitec Biopharma’s gene therapy against oculopharyngeal muscular dystrophy (OPMD). The gene therapy, AXO-AAV-OPMD, is designed to suppress expression of a mutated protein and encourage production of a functional version, thereby lessening muscular problems that affect an estimated 15,000 people in Europe and North America.

Benitec’s silence-and-replace approach has two components. First, the one-time gene therapy seeks to silence the mutated PABPN1 gene using DNA-directed RNA interference. In parallel, the therapy tries to insert a functional copy of the PABPN1 gene, enabling the patient to produce a protein found in healthy individuals. Preclinical studies suggest neither the silence nor replace mechanisms improve performance in isolation. But together, the mechanisms cut muscle fibrosis and increase strength.

“The silence-and-replace technology is a unique approach in gene therapy, using a single vector to suppress mutant protein production while also restoring expression of the functional protein, and could be an elegant solution to tackling autosomal dominant genetic disorders,” Fraser Wright, Ph.D., chief technology officer at Axovant, said in a statement.

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Having taken a look at Benitec’s preclinical data, Axovant has paid $10 million upfront for the global rights to the OPMD gene therapy. Axovant has also committed to milestones of undisclosed size and promised Benitec a 30% cut of any net profits generated by AXO-AAV-OPMD. Axovant plans to start a placebo-controlled trial of the drug next year.

AXO-AAV-OPMD is the near-term focus of the deal but Axovant has also bought itself a source of future candidates. The deal covers five more gene therapy programs, including a candidate against the C9orf72 gene that is involved in amyotrophic lateral sclerosis and frontotemporal dementia. The $10 million upfront covers the rights for all these therapies.

RELATED: Axovant pivots with new gene therapy, Spark co-founder as CTO

News of the deal comes one month after Axovant picked up the rights to a Parkinson’s disease gene therapy from Oxford Biomedica and named Spark Therapeutics co-founder Wright as CTO. The deals and appointment are central to Axovant’s strategy for recovering from the phase 3 Alzheimer’s disease failure that crushed its stock and near-term commercial prospects. 

“Axovant is trying to re-invigorate the company and the stock by re-inventing itself and has in-licensed two key neurological gene therapy programs in two months and with new management leading the way,” Jefferies analyst Michael Yee wrote in a note to investors.  

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