8-K: Axsome Therapeutics Receives FDA Support for AXS-05 in Alzheimer's Disease Agitation, Plans sNDA Submission

Axsome Therapeutics announces positive pre-NDA meeting feedback from the FDA, supporting a supplemental NDA submission for AXS-05 in treating agitation associated with Alzheimer's disease.

Summary

• Axsome Therapeutics has received formal pre-New Drug Application (NDA) meeting minutes from the FDA supporting a supplemental NDA (sNDA) submission for AXS-05 in Alzheimers disease agitation.
• AXS-05 is a novel, oral, investigational NMDA receptor antagonist, sigma-1 agonist, and aminoketone CYP2D6 inhibitor.
• The FDA feedback confirmed the studies and data to be presented in the planned sNDA submission.
• Axsome anticipates submitting the sNDA in the third quarter of 2025.
• AXS-05 has Breakthrough Therapy designation for Alzheimers disease agitation, potentially granting it Priority Review.
• The clinical development program includes four completed pivotal Phase 3 placebo-controlled efficacy trials and a long-term safety trial.
• AXS-05 demonstrated statistically significant improvement in Alzheimers disease agitation compared to placebo in three Phase 3 trials.
• The long-term safety and tolerability of AXS-05 was evaluated in over 300 patients treated for at least 6 months and over 100 patients treated for at least 12 months.
• AXS-05 was well tolerated with long-term dosing and was not associated with increased risk of falls, sedation, or cognitive decline.

Sentiment

Score: 8

Explanation: The document conveys a positive sentiment due to the supportive FDA feedback and the planned sNDA submission. The potential for AXS-05 to address a high unmet medical need in Alzheimers disease agitation further contributes to the positive outlook.

Positives

• FDA feedback supports the sNDA submission for AXS-05.
• AXS-05 has Breakthrough Therapy designation, potentially leading to Priority Review.
• Clinical trials demonstrated statistically significant improvement in Alzheimers disease agitation.
• AXS-05 was well-tolerated in long-term studies.
• The company anticipates submitting the sNDA in the third quarter of 2025.

Risks

• Acceptance of the final sNDA will be subject to the FDAs review of the complete filing.
• The forward-looking statements include risks and uncertainties, including, but not limited to, the commercial success of the Companys Sunosi, Auvelity, and Symbravo products and the success of the Companys efforts to obtain any additional indication(s) with respect to solriamfetol and/or AXS-05; the Companys ability to maintain and expand payer coverage; the success, timing and cost of the Companys ongoing clinical trials and anticipated clinical trials for the Companys current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Companys ability to fully fund the Companys disclosed clinical trials, which assumes no material changes to the Companys currently projected revenues or expenses), futility analyses and receipt of interim results, which are not necessarily indicative of the final results of the Companys ongoing clinical trials, and/or data readouts, 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 the Companys current product candidates; the Companys ability to fund additional clinical trials to continue the advancement of the Companys product candidates; the timing of and the Companys ability to obtain and maintain U.S. Food and Drug Administration (FDA) or other regulatory authority approval of, or other action with respect to, the Companys product candidates, including statements regarding the timing of any NDA submission; the Companys 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 Companys research and development programs and collaborations; the success of the Companys license agreements; the acceptance by the market of the Companys products and product candidates, if approved; the Companys anticipated capital requirements, including the amount of capital required for the commercialization of Sunosi, Auvelity, and Symbravo and for the Companys commercial launch of its other product candidates, if approved, and the potential impact on the Companys anticipated cash runway; the Companys ability to convert sales to recognized revenue and maintain a favorable gross to net sales; unforeseen circumstances or other disruptions to normal business operations arising from or related to domestic political climate, geo-political conflicts or a global pandemic and other factors, including general economic conditions and regulatory developments, not within the Companys control.

Future Outlook

Axsome anticipates submitting the sNDA for AXS-05 in the third quarter of 2025, with the goal of making this potential therapy available to patients as quickly as possible.

Management Comments

• Herriot Tabuteau, MD, Chief Executive Officer of Axsome, stated that they are pleased with the pre-NDA meeting feedback from the FDA, which confirmed the studies and data to be presented in their planned sNDA submission of AXS-05 for the treatment of Alzheimers disease agitation.
• Herriot Tabuteau, MD, Chief Executive Officer of Axsome, stated that if approved, AXS-05 would represent a new mechanism of action for the treatment of Alzheimers disease agitation with the potential to address this high unmet medical need.

Industry Context

This announcement is significant as Alzheimers disease agitation is a prevalent condition with limited treatment options, and AXS-05 could offer a new mechanism of action if approved.

Comparison to Industry Standards

• The document does not contain enough information to make a comparison to industry standards.
• More information would be needed to compare AXS-05 to other treatments for Alzheimers disease agitation, such as efficacy, safety, and cost-effectiveness compared to drugs like risperidone or olanzapine, which are sometimes used off-label.

Stakeholder Impact

• Positive impact on patients with Alzheimers disease and their caregivers if AXS-05 is approved.
• Potential benefit to Axsome Therapeutics shareholders through increased revenue if AXS-05 is successfully commercialized.

Next Steps

• Axsome plans to submit the sNDA for AXS-05 in the third quarter of 2025.
• The FDA will review the complete sNDA filing.

Key Dates