Date: 2018-07-09
Type of information: Licensing agreement
Compound:
- BB-301, now named AXO-AAV-OPMD (genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1) and five additional gene therapy products in neurological disorders
Company: Benitec Biopharma (Australia) Axovant (UK)
Therapeutic area: Rare diseases - Genetic diseases - Neurological diseases
Type agreement: licensing - research - development
Action mechanism:
- gene therapy/ddRNAi. BB-301 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.
Disease: oculopharyngeal muscular dystrophy (OPMD) amyotrophic lateral sclerosis (ALS) frontotemporal dementia (FTD)
Details:
- • On July 9, 2018, Benitec Biopharma announced that it has licensed to Axovant Sciences the exclusive global rights for BB-301 (now named AXO-AAV-OPMD) intended for the treatment of oculopharyngeal muscular dystrophy (OPMD), and has also entered into a fully funded research collaboration for the development of five additional gene therapy products in neurological disorders.
- Axovant plans to initiate a placebo-controlled clinical study in 2019 in which a one-time intramuscular administration AXO-AAVOPMD will be given to patients to treat the dysphagia associated with OPMD.
- 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. The first additional investigational gene therapy product will focus on developing a single vector “Silence-and-Replace” gene therapy product targeting the c9orf72 gene, which is associated with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
Financial terms:
- Under the terms of the agreement, Benitec will receive an upfront cash payment of $10 million and additional cash payments totaling $17.5 million upon completion of four specific near-term manufacturing, regulatory and clinical milestones. Axovant has been granted worldwide rights to AXOAAV-OPMD and will assume all future development costs. 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 $187.5 million. Benitec, working in partnership with Axovant over the next few years, hopes to achieve all eight milestones and thus realize the maximum
amount of US$187.5 million. There can be no assurance as to the total amount of payments that the Company will actually receive or when they will be received. Importantly, upon commercialization, Benitec will retain 30% of the net profits on worldwide sales of AXO-AAV-OPMD.
- In addition to receiving funding for development of the new research programs, each new research program target is eligible for development, regulatory and commercial milestones totaling $93.5 million and tiered royalties on
global sales.
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