Proximagen Limited has announced that its pivotal 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 US 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. Findings from the trial will be presented at the upcoming Antiepileptic Drug and Device Trials (AEDD) meeting, May 17-19, 2017 in Adventura, Florida.

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 intranasally, without active inhalation by the patient.

"The primary efficacy results observed in the pivotal phase 3 clinical trial were both statistically and clinically significant. 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," said Bill Pullman, MB, BS, BMedSc, PhD, FRACP, president, Proximagen. "This is an exciting development because to-date, rescue treatment options for seizure clusters have been very limited. We look forward to working with the FDA and submitting a new drug application for midazolam nasal spray later this year."

"Given the challenges and unmet needs of those living with seizures, we are very encouraged by the results of the Phase 3 trial of midazolam nasal spray. While there is still work to be done to put this medication into the hands of patients, I'd like to take this opportunity to thank our investigators, along with the teams at Upsher-Smith and Proximagen who have brought us to this major milestone," said Mark Evenstad, chief executive officer of ACOVA. "I am very proud of the important work that Proximagen is doing to help patients and their families by discovering and developing innovative therapies."

Proximagen's parent company Upsher-Smith Laboratories, Inc. recently announced an agreement to sell its generics business to Sawai, a Japanese publicly traded and family run generics pharmaceutical company. Following closing of this acquisition, Proximagen will cease to be a wholly-owned subsidiary of Upsher-Smith and will instead become a wholly-owned subsidiary of a newly created holding company, ACOVA. The Evenstad Family will continue to fund Proximagen through ACOVA.

Proximagen will retain its existing pipeline of small molecule NCE programmes, including two programs in clinical development. In addition, Upsher-Smith's intranasal midazolam program for seizure clusters (formerly known as USL261) will become part of Proximagen's pipeline.