Axovant reports negative topline data from nelotanserin trial

11th December 2018 (Last Updated December 11th, 2018 00:00)

Axovant Sciences has reported negative topline results from a Phase II clinical trial of nelotanserin for the treatment of REM sleep behavior disorder (RBD) in patients with Lewy body dementia (LBD).

Axovant reports negative topline data from nelotanserin trial
Positive staining (brown) of an intraneural Lewy-body in the Substantia nigra in Parkinson’s disease. Credit: Marvin 101.

Axovant Sciences has reported negative topline results from a Phase II clinical trial of nelotanserin for the treatment of REM sleep behavior disorder (RBD) in patients with Lewy body dementia (LBD).

The multi-centre, placebo-controlled study did not meet its primary efficacy endpoint of reducing the frequency of RBD events compared to placebo as measured by sleep laboratory video assessment.

However, nelotanserin was found to be generally well-tolerated throughout the randomised, double-blind trial.

The trial observed signals of efficacy during its secondary efficacy assessments that comprised trends in pre-specified evaluation of study diaries and certain sleep parameters on polysomnography (PSG).

This new data is similar to nelotanserin’s mechanism of action and previous clinical trial of nelotanserin for insomnia.

"We will not undertake further clinical studies with our legacy small molecule portfolio, including nelotanserin."

Axovant Sciences CEO Pavan Cheruvu said: “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.

“The completion of this study closes a chapter in the company’s history.”

In June, Axovant in-licensed the investigational gene therapy AXO-Lenti-PD for the treatment of Parkinson’s disease.

AXO-Lenti-PD is designed to deliver three genes in vivo via a lentiviral vector to encode the set of enzymes needed for dopamine synthesis in the brain.

Axovant is currently conducting the Phase II SUNRISE-PD clinical study to evaluate AXO-Lenti-PD.

The nelotanserin trial included 34 patients and results are expected to be released in March next year.