Axovant Significantly Expands Pipeline with Licensing Deal with Benitec BioPharma


Axovant, one of Vivek Ramaswamy’s biotech companies best known for its catastrophic Alzheimer’s failure in September 2017, is replenishing its pipeline with a licensing deal with Australian company Benitec BioPharma.

Axovant is licensing an investigational Silence-and-Replace gene therapy program from Benitec for the treatment of oculopharyngeal muscular dystrophy (OPMD). It has also inked a research collaboration deal for five more gene therapy products in neurological disorders.

Silence-and-Replace gene therapy is designed to deliver a combination of DNA-directed RNA interference with a functional copy of the gene in a single vector. The RNA interference is the silence part and the gene insertion is the replacement component.

The lead program is AXO-AAV-OPMD, which is in preclinical development. Axovant expects to launch a placebo-controlled clinical trial in 2019. OPMD is a neuromuscular disease caused by mutations in the gene for polyA-binding protein nuclear 1 (PABPN1). This mutation can cause the formation of intranuclear inclusion bodies that cause muscle cell problems. Patients with the disease may have difficulties swallowing, with potentially life-threatening results, including malnutrition and aspiration pneumonia. The disease is estimated to affect about 15,000 people in North America and Europe. There are no products approved to treat it.

AXO-AAV-OPMD is an adeno-associated viral (AAV) vector gene therapy. It is delivered by way of a one-time intramuscular administration. It is designed to silence the mutant PABPN1 gene and replace it with a working copy of the gene. Both the U.S. Food and Drug Administration (FDA) and European Commission (EC) have granted it Orphan Drug Designation.

Under the deal, Axovant is paying Benitec $10 million upfront for the AXO-AAV-OPMD program and five more gene therapy product. There are various milestone payments included in the deal. Also, Benetec will receive 30 percent of the net profits on worldwide sales of AXO-AAV-OPMD should it be approved, as well as tiered royalties on the other gene therapy products. Of the five additional programs, the first will target the C9orf72 gene, which is linked to amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

“This expansion of Axovant’s pipeline further demonstrates our commitment to advancing innovative gene therapies for serious neurological diseases,” said Pavan Cheruvu, Axovant’s chief executive officer, in a statement. “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-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.”

This announcement comes only weeks after the company’s reorganization at the very end of May. At that time, Gavin Corcoran joined as executive vice president of Research & Development, and Michael Hayden was appointed senior scientific advisor and chairman of its newly established Scientific Advisory Board. The reorganization had begun in February 2018 with cost-cutting and streamlining business processes. The company cut its internal headcount by about 43 percent and shifted much of its internal operations to use the Roivant platform—Ramaswamy’s parent company.

At that time, Axovant had two compounds in development. They are nelotanserin, a highly selective inverse agonist of the 5HT2A receptor and RVT-104, an early-stage asset. Nelotanserin is being evaluated in patients with Dementia with Lewy bodies (DLB) or Parkinson’s Disease Dementia (PDD) who are experiencing REM sleep behavior disorder (RBD). RVT-104 is a proposed combination of a higher than currently approved dose of cholinesterase inhibitor rivastigmine and a peripherally active muscarinic receptor antagonist. It is being studied as a possible treatment for Alzheimer’s disease and dementia with Lewy bodies.

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