Axsome Therapeutics Accelerates Completion of the ADVANCE-1 Phase 2/3 Trial of AXS-05 in Alzheimer’s Disease Agitation

Randomizations concluded and trial participation in process of concluding to ensure patient safety during COVID-19 pandemic

AXS-05 and placebo arms fully enrolled with estimated completion rate of more than 90%

Topline results now expected early 2Q 2020

NEW YORK, March 20, 2020 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a clinical-stage biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced that it is accelerating the completion of the ADVANCE-1 Phase 2/3 trial of AXS-05 in patients with Alzheimer’s disease (AD) agitation to ensure the safety of this vulnerable patient population during the COVID-19 pandemic, while maintaining the integrity of the clinical trial. AXS-05 (dextromethorphan/bupropion modulated delivery tablet) is a novel, oral, investigational NMDA receptor antagonist with multimodal activity. Randomizations into the study have concluded and patient participation is concluding. With the acceleration of trial completion, topline results from ADVANCE-1 are now expected in early second quarter of 2020, versus previous guidance of the third quarter of 2020.

The decision to accelerate the completion of this trial was made in light of the newly issued U.S. Food and Drug Administration (FDA) Guidance on Conduct of Clinical Trials of Medical Products during COVID-19 Pandemic, which discusses the potential impact of the pandemic on the conduct of clinical trials and on the resulting need to ensure the safety of trial participants. The ADVANCE-1 trial is enrolling only patients 65 years of age or older, the demographic at highest risk for COVID-19 related fatalities. The conclusion of patient randomization and participation in the ADVANCE-1 trial enables patient compliance with recent and evolving COVID-19 related national, state and local government directives and executive orders that persons 65 years and older isolate at home.

In the original design of the trial, approximately 435 patients were to be randomized in a 1:1:1 ratio to receive AXS-05, bupropion, or placebo for 5 weeks (approximately 145 patients per treatment arm). The AXS-05 and placebo arms are fully enrolled and more than 90% of patients in these arms are estimated to have completed the study. In December 2018, an independent data monitoring committee (IDMC) performed an unblinded, pre-specified interim analysis for futility on the first approximately 30% of the targeted number of patients. The IDMC recommended continuation of the AXS-05 and placebo treatment arms and no further randomization of patients to the bupropion treatment arm. Subsequently, patients were randomized in a 1:1 ratio to receive AXS-05 or placebo.