Proximagen Limited announced its Phase 3 trial of intranasal midazolam (USL261) for the rescue treatment of seizure clusters, met its primary efficacy endpoint in a top-level analysis of the data.
Based on these findings, Proximagen will approach the U.S. Food and Drug Administration (FDA) regarding its proposal to submit a 505(b)(2) New Drug Application (NDA) in the second half of this year. USL261 has been granted orphan drug designation and fast track designation for this use by the FDA.
USL261 is a novel, investigational midazolam formulation, specifically designed for intranasal delivery, being developed for the rescue treatment of seizures in patients who require control of intermittent bouts of increased seizure activity, such as seizure clusters or acute repetitive seizures. It is intended to be delivered through the nose, without active inhalation by the patient.
"The primary efficacy results observed in the pivotal phase 3 clinical trial were both statistically and clinically significant,” Bill Pullman, MB, BS, BMedSc, PhD, FRACP, President, Proximagen said. “These findings suggest that midazolam nasal spray could be an effective rescue treatment option for patients and caregivers who live with seizure clusters, subject to FDA review."
The study, ARTEMIS1 (Acute Rescue Therapy in Epilepsy with Midazolam Intranasal Spray) was designed to evaluate the efficacy and safety of USL261, an investigational formulation of midazolam delivered intranasally, for the outpatient rescue treatment of seizure clusters. Seizure clusters are associated with poor seizure control.