Axsome Therapeutics Initiates EMERGE Phase 3 Open-Label Trial of AXS-07 for the Acute Treatment of Migraine in Adults with a Prior Inadequate Response to an Oral CGRP Inhibitor

Rhea-AI Summary

Axsome Therapeutics (NASDAQ: AXSM) has announced the enrollment of the first patient in the Phase 3 EMERGE trial for AXS-07, targeting acute migraine treatment in patients unresponsive to oral CGRP inhibitors. This multicenter trial will treat approximately 100 patients for up to eight weeks, assessing the drug's efficacy and safety with co-primary endpoints focusing on pain relief and the absence of the most bothersome symptom post-dosing. AXS-07 is a novel investigational medicine designed to provide rapid and consistent migraine relief.

Positive

• First patient enrolled in the Phase 3 EMERGE trial for AXS-07.
• AXS-07 is designed for rapid and consistent migraine relief.
• Trial assesses both efficacy and safety, potentially paving the way for future approvals.

Negative

• AXS-07 is not FDA approved, posing risks for future commercialization.
• Trial conducted following inadequate response to CGRP inhibitors, indicating potential challenges in efficacy.

NEW YORK, Sept. 01, 2022 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company developing and delivering novel therapies for the management of central nervous system (CNS) disorders, announced that it has enrolled the first patient in the EMERGE trial of AXS-07 for the acute treatment of migraine.

EMERGE (Evaluating Outcomes of AXS-07 after Acute Gepant Failures) is a Phase 3, open-label, multicenter trial to assess the efficacy and safety of AXS-07 in the acute treatment of migraine in patients with a prior inadequate response to an oral Calcitonin Gene-Related Peptide (CGRP) inhibitor. Approximately 100 patients experiencing migraine attacks will be treated with AXS-07 for up to eight weeks, following an inadequate response to prior treatment with an oral CGRP inhibitor. The two co-primary endpoints will be pain relief and absence of the most bothersome symptom two hours after dosing. The study is being conducted to further elucidate the clinical profile of AXS-07 and is not a regulatory requirement.

About Migraine

Over 37 million Americans suffer from migraine according to the Centers for Disease Control, and it is the leading cause of disability among neurological disorders in the United States according to the American Migraine Foundation. Migraine is characterized by recurrent attacks of pulsating, often severe and disabling head pain associated with nausea, and sensitivity to light and or sound. It is estimated that migraine accounts for $78 billion in direct (e.g. doctor visits, medications) and indirect (e.g. missed work, lost productivity) costs each year in the United States [1]. Published surveys of migraine sufferers indicate that more than 70% are not fully satisfied with their current treatment, that nearly 80% would try a new therapy, and that they desire treatments that work faster, more consistently, and result in less symptom recurrence [2,3].

About AXS-07

AXS-07 is a novel, oral, rapidly absorbed, multi-mechanistic investigational medicine for the acute treatment of migraine, consisting of MoSEIC™ meloxicam and rizatriptan. Meloxicam is a new molecular entity for migraine enabled by Axsome's MoSEIC™ (Molecular Solubility Enhanced Inclusion Complex) technology, which results in rapid absorption of meloxicam while maintaining a long plasma half-life. Meloxicam is a COX-2 preferential non-steroidal anti-inflammatory drug and rizatriptan is a 5-HT1B/1D agonist. AXS-07 is designed to provide rapid, enhances and consistent relief of migraine, with reduced symptom recurrence. AXS-07 is covered by more than 80 issued U.S. and international patents which provide protection out to 2036. AXS-07 is not approved by the FDA.

About Axsome Therapeutics, Inc.

Axsome Therapeutics, Inc. is a biopharmaceutical company developing and delivering novel therapies for central nervous system (CNS) conditions that have limited treatment options. Through development of therapeutic options with novel mechanisms of action, we are transforming the approach to treating CNS conditions. At Axsome, we are committed to developing products that meaningfully improve the lives of patients and provide new therapeutic options for physicians. For more information, please visit the Company’s website at axsome.com. The Company may occasionally disseminate material, nonpublic information on the company website.

Forward Looking Statements

Certain matters discussed in this press release are “forward-looking statements”. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. In particular, the Company’s statements regarding trends and potential future results are examples of such forward-looking statements. The forward-looking statements include risks and uncertainties, including, but not limited to, the continued commercial success of our Sunosi® product and the success of our efforts to obtain any additional indication(s) with respect to Sunosi;   the commercial success of our Auvelity™ product and the success of our efforts to obtain any additional indication(s) with respect to AXS-05, the success, timing and cost of our ongoing clinical trials and anticipated clinical trials for our current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including our ability to fully fund our disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses and receipt of interim results, which are not necessarily indicative of the final results of our ongoing clinical trials, and the number or type of studies or nature of results necessary to support the filing of a new drug application (“NDA”) for any of our current product candidates; our ability to fund additional clinical trials to continue the advancement of our product candidates; the timing of and our ability to obtain and maintain U.S. Food and Drug Administration (“FDA”) or other regulatory authority approval of, or other action with respect to, our product candidates (including, but not limited to,; whether issues identified by FDA in the complete response letter may impact the potential approvability of the Company’s NDA for AXS-07 for the acute treatment of migraine in adults with or without aura, pursuant to our special protocol assessment for the MOMENTUM clinical trial; the Company’s ability to successfully defend its intellectual property or obtain the necessary licenses at a cost acceptable to the Company, if at all; the successful implementation of the Company’s research and development programs and collaborations; the success of the Company’s license agreements; the acceptance by the market of the Company’s product candidates, if approved; the Company’s anticipated capital requirements, including the amount of capital required for the successful commercialization of Sunosi and Auvelity and for the Company’s commercial launch of its other product candidates, and the potential impact on the Company’s anticipated cash runway; unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19; and other factors, including general economic conditions and regulatory developments, not within the Company’s control. The factors discussed herein could cause actual results and developments to be materially different from those expressed in or implied by such statements. The forward-looking statements are made only as of the date of this press release and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstance.

Axsome Contact:

Mark Jacobson
Chief Operating Officer
Axsome Therapeutics, Inc.
22 Cortlandt Street, 16th Floor
New York, NY 10007
Tel: 212-332-3243
Email: mjacobson@axsome.com
www.axsome.com

References

1. Gooch CL, Pracht E, Borenstein AR. The burden of neurological disease in the United States: A summary report and call to action. Ann Neurol. 2017 Apr; 81(4):479-484.
2. Smelt AF, Louter MA, Kies DA, Blom JW, Terwindt GM, van der Heijden GJ, De Gucht V, Ferrari MD, Assendelft WJ. What do patients consider to be the most important outcomes for effectiveness studies on migraine treatment? Results of a Delphi study. PLoS One. 2014 Jun 16;9(6):e98933.
3. Lipton RB, Stewart WF. Acute migraine therapy: do doctors understand what patients with migraine want from therapy? Headache. 1999;39(suppl 2):S20-S26.

FAQ

What is the purpose of the EMERGE trial for AXS-07?

The EMERGE trial aims to evaluate the efficacy and safety of AXS-07 in treating acute migraines in patients who did not respond adequately to oral CGRP inhibitors.

How many patients will be enrolled in the EMERGE trial for AXS-07?

Approximately 100 patients will be treated in the EMERGE trial.

What are the primary endpoints of the EMERGE trial for AXS-07?

The co-primary endpoints are pain relief and the absence of the most bothersome symptom two hours after dosing.

Is AXS-07 approved for use?

No, AXS-07 is not currently approved by the FDA.

What is the significance of the Phase 3 EMERGE trial for AXSM's future?

Success in the EMERGE trial could enhance the clinical profile of AXS-07 and support future regulatory approval.