Neurim Pharmaceuticals announced top-line results from its NEU_CH_7911 Phase III study. PedPRM met the primary efficacy endpoint demonstrating statistically significant improvement in total sleep time (TST) compared to placebo. In addition to TST, secondary efficacy endpoints demonstrating improvements in sleep initiation and maintenance were also met. Safety profile was similar between PedPRM and placebo-treated groups.
This was a randomized, double-blind, placebo-controlled, parallel group, multi-center (EU and USA) study in children with ASD or neurogenetic diseases and sleep disorders. Patients (125), who had not shown improvement practicing sleep hygiene, received 2 weeks placebo run-in, and then randomized to PedPRM (2mg with optional increase to 5mg) or placebo in the evening, for 13 weeks. Completers received PedPRM open-label for additional 13 weeks. Primary efficacy endpoint was defined as the difference between PedPRM and placebo in mean change from run-in to the end of double-blind treatment period, in parent-reported TST (sleep diary). Results from this study will be presented at upcoming medical congresses.
“PedPRM significantly improved sleep initiation and maintenance, while maintaining a favorable safety profile” says Tali Nir, DVM, VP Regulatory and Clinical Affairs of Neurim Pharmaceuticals, in a release. “Importantly, beyond the benefit to children’s sleep, we have observed gradual improvements in parents’ daytime alertness and children’s social functioning.”
“There are no approved sleep medications for the pediatric population” says Nava Zisapel, PhD, chief scientific officer of Neurim Pharmaceuticals. “We are proud to bring a potentially new effective and safe treatment to children with ASD living with severe sleep disturbances and their families.”