On February 13, 2017, Axovant Sciences, a specialist clinical stage biopharmaceutical company, announced the positive preliminary data from their Phase II dementia study for their 5-HT2A inverse antagonist drug, nelotanserin. The results show that 11 patients with Lewy body dementia (LBD) who were treated with nelotanserin have shown significant improvement in extrapyramidal symptoms, as measured by the Unified Parkinson’s Disease Rating Scale (UPDRS) Parts II and III.
The ongoing Phase II study is a double blind, randomized, placebo-controlled, crossover design study involving the random assignment of patients to a treatment sequence of either placebo followed by nelotanserin or nelotanserin followed by placebo. The final results of the study are expected to be announced later this year, but based on the promising preliminary data, Axovant Sciences is already preparing to start a Phase III trial in H2 2017. Axovant expects to evaluate UPDRS Parts II and III, as well as safety, as potential end points for LBD patients in Phase III. One slight downside from the preliminary data is that the secondary end point, tracking visual hallucinations, was not met in the preliminary data; this slightly dampened the optimistic mood at Axovant.
Currently, there are no drugs specifically approved for the treatment of LBD, and nelotanserin would therefore become a best-in-class drug. If approved, the market size would be considerable, as LBD is considered to be the second most common cause of dementia after Alzheimer’s disease. Axovant believes that the drug has the potential to meet a considerable unmet need in the dementia market due to its success in controlling Parkinsonism symptoms without exacerbating psychiatric symptoms, which it does with higher efficacy than any other drug on the market.
The positive results from nelotanserin’s preliminary data, coupled with Axovant’s intepiridine standing as the leading pipeline drug within its class for the treatment of Alzheimer’s, means the company is feeling positive for the future as they look to prove themselves as a major player in the neurodegenerative drug market. The next steps for company are to press on with Phase II trials and hope for positive top line data to support a New Drug Application (NDA) filing.