Shares in Axovant lost more than a quarter of their value yesterday after the Swiss biotech revealed that a trial of a drug intended for use in dementia failed a mid-stage trial.
The 34-patient study of nelotanserin – the last active programme in its small-molecule development pipeline after other project failures – failed to meet its main efficacy goal and will be discontinued.
Nelotanserin was being tested as a treatment for REM sleep behaviour disorder in patients with Lewy body dementia, but wasn’t able to reduce sleep disturbances compared to placebo.
There were signs of efficacy on some other secondary endpoints but not enough to extend its commitment to the programme, and there is a palpable sense that the company is keen to move onto the next phase of its development.
The decision to scrap nelotanserin comes after Axovant’s Alzheimer’s disease candidate intepirdine joined the chorus of failed drug candidates in that indication, and draws a line under the company’s original business model of licensing dormant drugs from other developers and re-purposing them for new uses. Nelotanserin stemmed from a $4m licensing deal with Arena Pharma signed in 2015.
Basel-based Axovant – which is part of the ‘vant’ group or companies set up by entrepreneur Vivek Ramaswamy under umbrella company Roivant Sciences – said earlier this year that it was pulling back from that approach and re-inventing itself as a gene therapy player, once again by licensing rights to technologies developed at other companies.
“Axovant has been focused on developing innovative gene therapies and we will not undertake further clinical studies with our legacy small molecule portfolio,” said Pavan Cheruvu, the company’s chief executive.
Pavan Cheruvu, Axovant CEO
Having attracted a gene therapy veteran to lead the programme in Fraser Wright – a co-founder and former chief technological officer at Spark Therapeutics – in June it licensed rights to a gene therapy for Parkinson’s disease (PD) from UK biotech Oxford BioMedica for $30m upfront.
That was followed swiftly by a $10m upfront deal with Benitec Biopharma for rights to a preclinical stage gene therapy for rare disease oculopharyngeal muscular dystrophy (OPMD), due to start trials next year, and five other early-stage programmes for neurological disorders.
The first two patients to be treated with PD candidate Axo-Lenti-PD in Axovant’s phase 2 SUNRISE-PD trial was dosed last month, and the company is hoping for preliminary data next March.
After recent meeting with the FDA, the firm as said it will be able to draw on earlier clinical data with a first-generation version of the gene therapy which was published in 2014, which should shorten its development timeline for the project.
The earlier version achieved improvements in PD symptoms that have been sustained for up to six years in some patients, it said.