Axovant Sciences yesterday reported topline results of a 34-patient, exploratory phase 2 clinical study of nelotanserin for the treatment of REM sleep behavior disorder (RBD) in patients with Lewy body dementia (LBD). The primary efficacy endpoint of reduction in frequency of RBD events as measured by sleep laboratory video assessment was not met.
Nelotanserin was generally well-tolerated in the study. Signals of efficacy were observed on secondary measures, including trends in prespecified analyses of study diaries and certain sleep parameters on polysomnography (PSG). These findings are consistent with nelotanserin’s mechanism of action and previous clinical studies of nelotanserin in patients with insomnia.
“While secondary measures of efficacy suggest 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,” says Pavan Cheruvu, MD, CEO of Axovant, in a release. “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.”