Idorsia (IDIA) Study result summary

Study overview and design

• Phase II, multi-center, double-blind, randomized, placebo-controlled, dose-finding study in 165 children aged 10 to <18 years with insomnia disorder, including those with neurodevelopmental disorders (NDDs) such as ASD and ADHD.

• Children were randomized to receive placebo or daridorexant at 10 mg, 25 mg, or 50 mg, matching adult dosing regimens.

• Study stratified patients by presence of NDDs and included subthreshold ASD/ADHD traits, with a balanced male-female split and ages down to 10 years.

• Primary endpoint was total sleep time measured by polysomnography on Day 1 after baseline sleep lab nights, with a 2-week treatment period.

• Only one child discontinued, indicating strong tolerability and engagement.

Key efficacy and safety results

• Statistically significant, dose-dependent improvements in total sleep time and multiple sleep measures, with the highest dose (50 mg) showing nominal significance versus placebo.

• Efficacy was especially pronounced in children with co-morbid NDDs, with unprecedented increases in sleep duration and improved morning alertness.

• No dose-response relationship for adverse events; safety profile was comparable to placebo and mirrored that seen in adults, with no increase in somnolence, suicidality, or abuse potential.

• Most common adverse events included headache, somnolence, and nasopharyngitis, with low rates of severe or serious adverse events.

• No next-morning residual sleepiness was reported; improved alertness was noted by patients and caregivers.

Implications and next steps

• Results suggest daridorexant may impact not only insomnia but also underlying NDD symptoms, warranting further analysis and regulatory engagement.

• Plans to discuss data with FDA and EMA to define regulatory path for pediatric insomnia and potential NDD indications.

• Additional studies may target younger children (ages 4–10) and explore broader NDD applications.

• Detailed results will be presented at upcoming congresses and in peer-reviewed publications.

• Ongoing efforts to protect intellectual property and prepare for scientific publication and congress presentations.