Axovant Licenses Benitec Gene Therapy Candidates for OPMD, Neurological Disorders

Axovant Sciences has licensed exclusive rights to Benitec Biopharma’s preclinical gene therapy for oculopharyngeal muscular dystrophy (OPMD), set to enter the clinic next year, and five additional Benitec gene therapy candidates for neurological disorders.

The deal could generate for Benitec up to $187.5 million plus net profits for the lead candidate, the OPMD gene therapy now called AXO-AAV-OPMD (formerly BB-301); and up to $467.5 million, plus royalties, tied to development of the other five neurological gene therapy candidates through a research collaboration also inked by the companies.

AXO-AAV-OPMD is an adeno-associated viral (AAV) vector gene therapy designed for delivery via a one-time intramuscular administration, which both silences the mutant PABPN1 gene and replaces it with a functional copy.

AXO-AAV-OPMD incorporates Benitec’s Silence-and-Replace gene therapy technology, which is designed to deliver a combination of DNA-directed RNA interference or ddRNAi (silence) along with a functional copy of the gene (replace) in a single vector construct, in order to restore the function of that gene. That approach, the companies reason, can be used against numerous genetic diseases, including autosomal dominant disorders caused by nucleotide repeat expansion.

“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,” says Axovant CEO Pavan Cheruvu, M.D. “AXO-AAV-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.”

The FDA and the European Commission have granted their orphan drug designations to AXO-AAV-OPMD for the treatment of OPMD. 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.

Axovant acquired global rights to AXO-AAV-OPMD, for which a placebo-controlled clinical study is planned to launch in 2019. In that study, AXO-AAV-OPMD will be given to patients via one-time intramuscular administration to treat the dysphagia associated with OPMD.

In return, Axovant agreed to pay Benitec $10 million upfront for rights to all six gene therapy candidates, agreeing in return to assume all future development costs.

Axovant also agreed to shell out $17.5 million tied to Benitec achieving four near-term manufacturing, regulatory and clinical milestones for AXO-AAV-OPMD. Should Benitec achieve those benchmarks and four additional milestones, it stands to receive from Axovant a total $187.5 million. In addition, Benitec will retain 30% of the net profits on worldwide sales of AXO-AAV-OPMD.

Targeting ALS, Dementia Gene

Of the five additional gene therapy candidates that Axovant will partner with Benitec to research and develop, one will target the C9orf72 gene, associated with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), according to Axovant. No details have been disclosed for the other four.

Benitec said in a separate statement that Axovant agreed to pay it up to $93.5 million per research program tied to achieving development, regulatory and commercial milestones—for a total $467.5 million—plus tiered royalties on global sales.

Headquartered in Sydney with laboratories in Hayward, CA, Benitec focuses on developing ddRNAi-based therapeutics for chronic and life-threatening human conditions including OPMD, head & neck squamous cell carcinoma, retinal based diseases such as wet age-related macular degeneration, and hepatitis B.

Today marks a milestone for Benitec as we believe this transaction to be transformative for our company,” says Benitec Executive Chairman Jerel Banks, M.D., Ph.D. “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 deal with Benitec marks Axovant’s second licensing of an external gene therapy candidate in about a month. On June 6, Axovant licensed Oxford BioMedica’s gene therapy candidate for Parkinson’s disease OXB-102 (now AXO-Lenti-PD), and predecessor product ProSavin®, in return for paying Oxford BioMedica $30 million upfront, potentially more than $812 million tied to development, regulatory, and commercial milestones, and tiered royalties.

Axovant has been intent on expanding its gene therapy pipeline since the September 2017 failure of its Alzheimer’s disease (AD) candidate intepirdine in the Phase III MINDSET trial (NCT02585934) in mild-to-moderate AD patients who were receiving background donepezil therapy. Due to that failure, a related 12-month open-label extension study (NCT02586909) was terminated.

Also in June, Axovant named as its CTO for gene therapy programs Fraser Wright, previously co-founder and former CTO of Spark Therapeutics.

Reference

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