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Axsome Therapeutics Confirms Pivotal Status and Advancement of AXS-05 for the Treatment of Alzheimer’s Disease Agitation Based on Successful FDA Breakthrough Therapy Meeting
Previously completed positive, pivotal ADVANCE-1 trial sufficient with single additional Phase 3 efficacy trial for NDA in Alzheimer’s disease agitation
About this update from Axsome Therapeutics, Inc.
[{"type":"text","content":" Previously completed positive, pivotal ADVANCE-1 trial sufficient with single additional Phase 3 efficacy trial for NDA in Alzheimer’s disease agitation\n Initiation of Phase 3, placebo-controlled, randomized-withdrawal efficacy trial on track for 4Q 2020 Initiation of long-term safety trial in patients with Alzheimer’s disease agitation expected in 4Q 2020 No treatments are currently approved for the treatment of Alzheimer’s disease agitation NEW YORK, Aug. 31, 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 confirmation of the pivotal development status and plan for AXS-05 in the treatment of Alzheimer’s disease (AD) agitation following a successful Breakthrough Therapy meeting with the U.S. Food and Drug Administration (FDA). AXS-05 (dextromethorphan/bupropion modulated delivery tablet) is a novel, oral, investigational NMDA receptor antagonist and sigma-1 receptor agonist. There is currently no approved treatment for AD agitation. Results of the meeting confirm the pivotal status of the previously completed positive ADVANCE-1 trial, and the establishment of the superiority of AXS-05 over its components (component contribution) in the treatment of AD agitation. Consequently, only one additional Phase 3 efficacy trial will be needed to support the filing of an NDA (New Drug Application) for approval of AXS-05 in this indication, and only a placebo control will be required for this trial. This additional Phase 3 efficacy trial will be conducted using a randomized-withdrawal design, in which all patients are first treated with open-label AXS-05, with the patients experiencing a treatment response being subsequently randomized in a double-blind fashion to continued treatment with AXS-05 or to switch to placebo. Axsome is on track to initiate this efficacy trial in the fourth quarter of 2020. Axsome also intends to initiate in the fourth quarter an open-label safety extension trial of AXS-05 in AD agitation patients to supplement the existing AXS-05 long-term safety database. “Axsome is very pleased with the FDA feedback from our recent Breakthrough Therapy meeting, which confirms a streamlined path to NDA submission for AXS-05 in Alzheimer’s disease agitation, including the pivot...