There is very little doubt that developing drugs for neuroscience disorders in general and dementias, in particular, is a high-risk business. Axovant Sciences, unfortunately, seems to be finding that out the hard way. The company announced today that its Phase II clinical trial of nelotanserin for the treatment of REM sleep behavior disorder (RBD) in patients with Lewy body dementia (LBD) failed to meet its primary efficacy endpoint. The endpoint was a reduction in the frequency of RBD events as measured by sleep laboratory video.
“While secondary measures of efficacy suggest a biologic activity for nelotanserin, Axovant has been focused on developing innovative gene therapies and we will not undertake further clinical studies with our legacy small molecule portfolio, including nelotanserin,” stated Pavan Cheruvu, Axovant’s chief executive officer. “The completion of this study closes a chapter in the company’s history. We are grateful to the patients and clinical investigators who participated in this study, and we look forward to advancing Axovant’s gene therapy pipeline through multiple important milestones in 2019.”