Axovant Enters Global Licensing Deal with Benitec for Investigational AXO-AAV-OPMD Program to Treat OPMD

Benitec Biopharma recently granted Axovant Sciences worldwide rights to its investigational AXO-AAV-OPMD gene therapy program for the treatment of oculopharyngeal muscular dystrophy (OPMD) under a global licensing agreement.

AXO-AAV-OPMD is currently in preclinical development, and Axovant plans to launch a placebo-controlled trial testing the therapy candidate for OPMD treatment in 2019.

Under the agreement, Avoxant also gained global rights from Benitec for five more investigational gene therapies for the treatment of other neurological disorders.

“This agreement with Axovant further demonstrates the importance of Benitec’s core technology and our strategic focus on rapidly progressing these programs into the clinic,” Jerel Banks, MD, PhD, executive chairman of Benitec, said in a press release. “We believe Axovant is the ideal partner to take these programs forward and look forward to working closely with them to develop AXO-AAV-OPMD and other neurological gene therapies.”

OPMD is a neuromuscular disorder caused by defects in the PABPN1 gene that provides instructions for a protein called polyA-binding protein nuclear 1 (PABPN1). Abnormal production of this protein leads to protein aggregates that cause damage in the muscle cells. There are no approved treatments for this disease yet.

AXO-AAV-OPMD is an investigational, one-time intramuscular gene therapy injection using Benitec’s Silence-and-Replace technology. This technology is a combination of two different molecules, one designed to stop the production of the mutant protein, while the other is a correct gene copy to replace the damaged one and restore the normal production of PABPN1.

“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,” said Fraser Wright, PhD, chief technology officer of Axovant. “I look forward to collaborating with the research and manufacturing teams at Benitec to advance the progress of the platform and bring additional therapies into the clinic.”

Both the U.S. Food and Drug Administration and the European Commission granted AXO-AAV-OPMD orphan drug status for the treatment of OPMD.

According to the release, Axovant will pay Benitec $10 million for the rights to the AXO-AAV-OPMD program and five other gene therapies. The agreement also includes payments related to specific milestones in the development, regulatory, and commercial sale processes.

Benitec will also receive 30 percent of the worldwide sales of AXO-AVV-OPMD, as well as associated royalties on the other gene therapy products.

“This expansion of Axovant’s pipeline further demonstrates our commitment to advancing innovative gene therapies for serious neurological diseases,” said Pavan Cheruvu, MD, CEO of Axovant.

“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-AVV-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,” he said.

Axovant announced the licensing agreement via a recent conference call that is available on the company’s website until Aug. 8.