Sydney-based Benitec Biopharma (ASX:BLT) has licensed to Axovant Sciences the exclusive global rights for BB-30, now named AXO-AAV-OPMD, which is in development for the treatment of oculopharyngeal muscular dystrophy (OPMD).
OPMD is a rare progressive, and often fatal, muscle-wasting disease caused by mutation in the poly(A)- binding protein nuclear 1 (PABPN1) gene, that is characterised by eyelid drooping, swallowing difficulties, and proximal limb weakness.
The deal, which Benitec said in a statement could deliver the company a total of almost US$300 million, also includes a fully funded research collaboration for the development of five additional gene therapy products in neurological disorders.
Axovant has been granted worldwide rights to AXOAAV-OPMD and will assume all future development costs.
Under the terms of the agreement, Benitec will receive an upfront cash payment of US$10 million and additional cash payments totaling US$17.5 million upon completion of four specific near-term manufacturing, regulatory and clinical milestones.
The total potential value of all of the development, regulatory and commercial milestones achievable by Benitec, of which there are eight milestones including the four near-term milestones, is US$187.5 million.
Benitec said that, upon commercialisation, it will retain 30 percent of the net profits on worldwide sales of AXO-AAV-OPMD.
“Today marks a milestone for Benitec as we believe this transaction to be transformative for our company," said Jerel Banks, MD PhD, executive chairman of Benitec Biopharma.
"In addition to bolstering our opportunity to drive broad-based, clinically meaningful patient benefit across several areas of clinical medicine with true unmet need, this partnership significantly enhances the financial, intellectual, and clinical development resources available to facilitate our efforts to build Benitec into a diversified biopharmaceutical company. The non-dilutive capital expected over the near term will allow Benitec to continue to invest in proprietary R&D programs across a range of indications,” said Dr Banks.
“Our management team is focused exclusively on expanding the research, development, and commercial opportunities for the core ‘silence-and-replace’ platform with the dual goals of enhancing patient benefit and generating shareholder value. We believe Axovant is the ideal partner to advance our OPMD program, and we look forward to working closely with them to develop AXO-AAV-OPMD as we quickly progress towards clinical trials in 2019.”
AXO-AAV-OPMD is a single vector, gene therapy construct system that uses a unique 'silence-and-replace' methodology that employs DNA directed RNA interference (ddRNAi) to silence expression of the mutant gene associated with OPMD, while simultaneously expressing a copy of the normal, healthy version of the same gene to restore the function of that gene.
Axovant said it plans to initiate a placebo-controlled clinical study in 2019 in which a one-time intramuscular administration of AXO-AAVOPMD will be given to patients to treat the dysphagia associated with OPMD.
According to Dr Pavan Cheruvu, CEO of Axovant, “The ‘silence-and-replace’ platform is a targeted approach which directly addresses the underlying genetic cause of diseases arising from expression of dysfunctional proteins, including those caused by nucleotide repeat expansion. I am excited about the potential of this platform for patients suffering from OPMD, many of whom have limited treatment options today.”
In addition to AXO-AAV-OPMD, Axovant and Benitec will collaborate on a total of five additional investigational gene therapy products for neurological disorders, with Axovant fully funding each of the research programs. Axovant will have exclusive global rights to products developed under these programs.
Dr Banks concluded, “We are extremely excited about Axovant’s collaborative and financial commitments to these five additional research programs as it plants the seeds for a long and robust partnership between our organizations.