Neurim Pharmaceuticals presented top line results of a placebo-controlled phase III study of pediatric prolonged-release melatonin formulation (PedPRM) in autism spectrum disorder (ASD) children aged 2-18 years who suffer from insomnia.

The study’s primary endpoint showed a clinically meaningful and statistically significant increase in total sleep time (p=0.034). Participants slept on average 57.5 minutes longer at night with PedPRM compared to 9.14 with placebo. Sleep latency (SL) decreased by 39.6 minutes on average with PedPRM compared to 12.5 with placebo (p=0.011) without causing earlier wakeup time. Sleep maintenance, parents’ quality of life and satisfaction of child’s sleep also improved significantly. The beneficial effects of PedPRM were maintained over the long term. Furthermore, compliance with the age-appropriate PedPRM formulation was good and it had a favorable safety profile over the short and long term period.

Main study results were presented during the World Sleep Congress (Prague, October 9-12), and later will be presented at the American Academy of Child & Adolescent Psychiatry (AACAP) 64th Annual Meeting, (Washington DC, from October 23-28, 2017), and are in press in the Journal of American Academy of Child & Adolescent Psychiatry.

“Children with autism spectrum disorder suffer from significant sleep problems which exacerbate their behavioral and functional deficits, and are particularly challenging to their families. Currently, there are no approved therapeutic options for pediatric insomnia, and that is particularly problematic for children with ASD,” says Paul Gringras, MRCPCH,MSc, lead for the Children’s Sleep Medicine Dept, Evelina London Children Hospital and Principal Investigator of the study, in a release. “The results of the study indicate that PedPRM is an effective and safe treatment which is well tolerated even in this challenging population. It is effective in improving sleep problems that have been refractory to the usual behavioral interventions in this population, and furthermore improves parental quality of life and satisfaction from their child’s sleep.”

PedPRM is an age-appropriate formulation of prolonged-release melatonin designed for populations with swallowing difficulties, such as children with ASD, in response to the unmet medical need in the field of pediatric insomnia under EU-PIP and US-FDA IND.

The pivotal phase III study 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. 125 patients, who had not shown improvement after standard sleep behavioral intervention, received 2 weeks placebo run-in, and were then randomized to PedPRM (2 mg with optional increase to 5 mg) 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 baseline to the end of double-blind treatment period, in parent-reported true sleep time (daily sleep and nap diary). Additional endpoints assessed children’s sleep initiation and maintenance, behavioral and social functioning, as well as, parent’s quality of life. Patients are now being followed up to 78 weeks to assess the long-term efficacy and safety of PedPRM and results will be presented during 2018.

Neurim Pharmaceuticals Ltd is a neuroscience drug discovery and development company. Its first approved drug CIRCADIN, is approved for patients over the age of 55 who are suffering from insomnia, and is commercially available in more than 45 countries around the world.