Single Phase 3 trial to be conducted to support an NDA filing of AXS-12 in the treatment of narcolepsy; trial initiation anticipated 1Q 2021
Extensive patient safety database, previously obtained through Pfizer exclusive license, to support NDA filing
Extensive array of completed nonclinical studies sufficient to support NDA filing
Company to host conference call today at 8:00 AM Eastern
NEW YORK, Sept. 21, 2020 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced that the development plan for AXS-12 for the treatment of narcolepsy has been expedited following a Breakthrough Therapy meeting with the U.S. 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,” said Herriot Tabuteau, MD, Chief Executive Officer of Axsome. “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.”
Breakthrough Therapy Meeting Outcomes
- One Phase 3 trial of AXS-12 in patients with narcolepsy will be conducted to support, along with the completed Phase 2 CONCERT trial, an NDA filing for AXS-12 in the treatment of cataplexy in narcolepsy.
- The existing short-term and long-term safety database of more than 2,500 patients treated with reboxetine, previously obtained through an exclusive license from Pfizer, along with the safety data from the completed and planned studies of AXS-12 in patients with narcolepsy, would be sufficient to support an NDA filing for AXS-12.
- Certain existing, completed clinical pharmacology (e.g. human pharmacokinetic) studies of reboxetine, previously obtained through an exclusive license from Pfizer, are considered sufficient to support an NDA filing for AXS-12.
- The extensive package of completed nonclinical studies of reboxetine, previously obtained through an exclusive license from Pfizer, are considered sufficient to support an NDA filing for AXS-12.
In August 2020, Axsome received Breakthrough Therapy designation from the FDA for AXS-12 for the treatment of cataplexy in narcolepsy. A Breakthrough Therapy designation is granted to potentially expedite development and review timelines for a promising investigational medicine when preliminary clinical evidence indicates it may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies for a serious or life-threatening condition. The Breakthrough Therapy designation for AXS-12 for the treatment of cataplexy in narcolepsy was supported by the positive results from the Phase 2 CONCERT study, a randomized, double-blind, placebo-controlled, crossover, multicenter U.S. trial. In the trial, 21 patients with a diagnosis of narcolepsy with cataplexy were treated for 2 weeks with AXS-12 or with placebo, followed by a crossover to the other treatment after a 1-week down-titration and washout period. AXS-12 met the primary endpoint demonstrating a highly statistically significant reduction from baseline in the mean weekly number of cataplexy attacks, averaged for the 2-week treatment period (overall treatment effect), as compared to placebo (p