Axsome Therapeutics Inc has expedited the development plan for AXS-12 for the treatment of narcolepsy, following a Breakthrough Therapy meeting with the US Food and Drug Administration (FDA). AXS-12 is a novel, oral, highly selective and potent norepinephrine reuptake inhibitor for the treatment of narcolepsy.

The expedited development plan includes one Phase 3 efficacy trial, which, along with the previously completed Phase 2 CONCERT trial, will be used to support the filing of an NDA (New Drug Application) for approval of AXS-12 for the treatment of cataplexy in narcolepsy. The planned Phase 3 trial will be a randomized, double-blind, placebo-controlled, parallel-group study. Axsome intends to initiate this trial in 1Q 2021. Patients completing this trial will be eligible to enroll in an open-label safety extension study.

Also, as part of the expedited development plan, it was established that the data from numerous short-term and long-term clinical trials of patients treated with reboxetine, and the data from a full range of completed nonclinical studies with reboxetine, previously obtained through an exclusive license from Pfizer, can be used to support the filing of an NDA for AXS-12.

“Axsome is very pleased with the FDA feedback from our recent Breakthrough Therapy meeting, which resulted in an expedited path to a potential NDA filing for AXS-12 in the treatment of cataplexy in narcolepsy,” says Herriot Tabuteau, MD, Axsome CEO, in a release. “Narcolepsy is a debilitating condition that interferes with almost every aspect of a patient’s life. Our approach to the development of AXS-12 for narcolepsy reflects our commitment to accelerating the innovation of potentially life-changing medicines for the many patients living with serious CNS disorders.”

In August 2020, Axsome received Breakthrough Therapy designation from the FDA for AXS-12 for the treatment of cataplexy in narcolepsy.