- Axovant Sciences will stop developing its small molecule drug portfolio, the company said Monday, following the failure of its experimental drug candidate nelotanserin in a Phase 2 trial of patients with Lewy body dementia who experience REM sleep behavior disorder.
- The drug failed to meet its primary endpoint of reducing sleep disorder episodes, and showed only "signals of efficacy" on the study's secondary goals. Shares in Axovant closed down more than 25% yesterday on the news.
- In response, Axovant CEO Pavan Ceruvu said the company won't undertake further clinical study of its small molecule candidates, shifting its sole focus onto gene therapy instead.
Axovant has met with its fair share of setbacks.
Nelotanserin isn't the only small molecule drug to have fallen short this year at Axovant, one of an umbrella of companies established through Vivek Ramaswamy's Roivant.
Most notably, the one-time Alzheimer's hopeful intepirdine failed to meet its primary efficacy endpoints in a Phase 2b study in patients with Lewy body dementia and other pilot Phase 2 studies, leading Axovant to abandon the former GlaxoSmithKline compound.
The company began an organizational restructuring in February, appointing a new CEO, increasing its investment in clinical research and reducing the size of its global commercial team. Overall, these moves reduced headcount by more than 40%.
Continuing its pivot, Axovant in June picked up AXO-Lenti-PD, a lentiviral gene therapy candidate for Parkinson's disease, from Oxford BioMedica. Perhaps riding investor enthusiasm for the gene therapy field, Axovant's stock value shot up by 160% on that news.
Initial data from a Phase 2 study of the treatment is expected next March. And, in a boost to this program, the Food and Drug Administration has said preclinical and Phase 1/2 studies carried out with the first-generation gene therapy, ProSavin, can be considered to be part of the development plan.
Axovant has since styled itself as a gene therapy specialist, building a pipeline that includes AXO-AAV-OPMD for treatment of oculopharyngeal muscular dystrophy. The company has also added gene therapy-focused staff, including a chief technology officer for gene therapy programs and a slate of people at the senior vice president level.
Yet Axovant's work in gene therapy remains very early, and just as exposed to setback. If stock values are anything to go by, the current depressed share price for Axovant suggested investors remain pessimistic about the company's prospects.