- Primary efficacy endpoint assessed by sleep laboratory video assessment was not met
- Axovant is discontinuing clinical development of nelotanserin
BASEL, Switzerland I December 10, 2018 I Axovant Sciences (NASDAQ: AXON) a clinical-stage gene therapy company, today reported topline results of the 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,” said Pavan Cheruvu, M.D., chief executive officer of Axovant. “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.”
Axovant has been focused on gene therapies since the in-licensing of AXO-Lenti-PD in June 2018, and has since then strengthened its capabilities in the development, manufacturing and commercialization of gene therapies with the addition of experts to the team and continued expansion of its pipeline. AXO-Lenti-PD is an investigational gene therapy for Parkinson’s disease that delivers three genes in vivo via a lentiviral vector to encode the set of enzymes required for dopamine synthesis in the brain. The SUNRISE-PD phase 2 clinical study of AXO-Lenti-PD is ongoing, with data expected in March 2019. In addition, Axovant is developing AXO-AAV-OPMD, a gene therapy utilizing novel Silence-and-Replace technology to restore normal muscle function in patients with oculopharyngeal muscular dystrophy (OPMD), and plans to initiate a clinical study for the therapy in the second half of 2019.
About the Nelotanserin REM Sleep Behavior Disorder Study
This multi-center, randomized, double-blind, placebo-controlled phase 2 clinical study evaluated the efficacy of nelotanserin in 34 patients with LBD who were experiencing RBD. The prespecified primary endpoint of the pilot study was to determine efficacy in reducing frequency of RBD behaviors compared to placebo through sleep laboratory video assessment. Secondary efficacy assessments in the study included measures of RBD derived from a study diary and various objective sleep parameters.
About Axovant Sciences
Axovant is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases such as Parkinson’s disease, oculopharyngeal muscular dystrophy (OPMD), amyotrophic lateral sclerosis (ALS), frontotemporal dementia, and other indications. For more information, visit www.axovant.com
SOURCE: Axovant
Post Views: 30
- Primary efficacy endpoint assessed by sleep laboratory video assessment was not met
- Axovant is discontinuing clinical development of nelotanserin
BASEL, Switzerland I December 10, 2018 I Axovant Sciences (NASDAQ: AXON) a clinical-stage gene therapy company, today reported topline results of the 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,” said Pavan Cheruvu, M.D., chief executive officer of Axovant. “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.”
Axovant has been focused on gene therapies since the in-licensing of AXO-Lenti-PD in June 2018, and has since then strengthened its capabilities in the development, manufacturing and commercialization of gene therapies with the addition of experts to the team and continued expansion of its pipeline. AXO-Lenti-PD is an investigational gene therapy for Parkinson’s disease that delivers three genes in vivo via a lentiviral vector to encode the set of enzymes required for dopamine synthesis in the brain. The SUNRISE-PD phase 2 clinical study of AXO-Lenti-PD is ongoing, with data expected in March 2019. In addition, Axovant is developing AXO-AAV-OPMD, a gene therapy utilizing novel Silence-and-Replace technology to restore normal muscle function in patients with oculopharyngeal muscular dystrophy (OPMD), and plans to initiate a clinical study for the therapy in the second half of 2019.
About the Nelotanserin REM Sleep Behavior Disorder Study
This multi-center, randomized, double-blind, placebo-controlled phase 2 clinical study evaluated the efficacy of nelotanserin in 34 patients with LBD who were experiencing RBD. The prespecified primary endpoint of the pilot study was to determine efficacy in reducing frequency of RBD behaviors compared to placebo through sleep laboratory video assessment. Secondary efficacy assessments in the study included measures of RBD derived from a study diary and various objective sleep parameters.
About Axovant Sciences
Axovant is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases such as Parkinson’s disease, oculopharyngeal muscular dystrophy (OPMD), amyotrophic lateral sclerosis (ALS), frontotemporal dementia, and other indications. For more information, visit www.axovant.com
SOURCE: Axovant
Post Views: 30
