Axsome Therapeutics Announces FDA Acceptance and Priority Review of Supplemental New Drug Application for AXS-05 for the Treatment of Alzheimer’s Disease Agitation

Rhea-AI Summary

Axsome Therapeutics (NASDAQ: AXSM) announced the FDA has accepted and granted Priority Review to its supplemental NDA for AXS-05 for treatment of Alzheimer’s disease agitation, with a PDUFA target action date of April 30, 2026.

The filing follows a comprehensive clinical program including four randomized, double-blind, controlled Phase 3 trials and a long-term safety trial. The FDA previously granted Breakthrough Therapy designation for AXS-05 in June 2020. Company commentary cites that up to 76% of people with Alzheimer’s experience agitation and that approved treatments are scarce.

Positive

• FDA Priority Review granted for AXS-05
• PDUFA target action date set for April 30, 2026
• Four Phase 3 randomized, double-blind controlled trials completed
• Breakthrough Therapy designation previously granted in June 2020

Negative

• Regulatory decision is pending until the April 30, 2026 PDUFA date
• There is a current dearth of approved treatments for Alzheimer’s agitation

News Market Reaction – AXSM

+22.75% 5.1x vol

92 alerts

+22.75% News Effect

+15.4% Peak in 6 hr 37 min

+$1.72B Valuation Impact

$9.30B Market Cap

5.1x Rel. Volume

On the day this news was published, AXSM gained 22.75%, reflecting a significant positive market reaction. Argus tracked a peak move of +15.4% during that session. Our momentum scanner triggered 92 alerts that day, indicating high trading interest and price volatility. This price movement added approximately $1.72B to the company's valuation, bringing the market cap to $9.30B at that time. Trading volume was exceptionally heavy at 5.1x the daily average, suggesting very strong buying interest.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

PDUFA action date: April 30, 2026 Priority review timeline: 6 months Standard review timeline: 10 months +3 more

6 metrics

PDUFA action date April 30, 2026 FDA target action date for AXS-05 sNDA in Alzheimer’s agitation

Priority review timeline 6 months FDA goal to act on Priority Review applications

Standard review timeline 10 months FDA goal for standard application reviews

Phase 3 trials 4 trials Randomized, double-blind, controlled Phase 3 studies in Alzheimer’s agitation

Long-term safety trial 1 trial AXS-05 long-term safety in Alzheimer’s agitation

Alzheimer’s agitation prevalence 76% People with Alzheimer’s disease who experience agitation

Market Reality Check

Price: $162.33 Vol: Volume 509,245 is 4% abov...

normal vol

$162.33 Last Close

Volume Volume 509,245 is 4% above the 20-day average of 488,270 ahead of the news. normal

Technical Shares at $148.79 are trading above the 200-day MA of $119.41, near the 6.16% below 52-week high.

Peers on Argus

AXSM dipped 1.25% while key biotech peers like LEGN, CYTK, ABVX, NUVL, and RYTM ...

AXSM dipped 1.25% while key biotech peers like LEGN, CYTK, ABVX, NUVL, and RYTM also showed modest declines, but no momentum scanner signals or shared news themes appeared.

Historical Context

5 past events · Latest: Nov 20 (Neutral)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Nov 20	Investor conferences	Neutral	-2.4%	Announced participation in December healthcare and CNS-focused investor conferences.
Nov 06	Asset acquisition	Positive	+2.7%	Acquisition of AZD7325 epilepsy program with milestone and royalty structure.
Nov 03	Earnings update	Positive	+0.6%	Reported strong Q3 revenue growth, continued net loss, and sNDA submission for AXS-05.
Oct 22	Investor conferences	Neutral	-1.3%	Outlined November conference appearances with webcast access for investors.
Oct 09	Awareness initiative	Neutral	+3.2%	Recognized Mental Illness Awareness Week and highlighted national support programs.

Pattern Detected

Recent news events, including clinical and business updates, have generally led to modest price moves with reactions largely aligned to neutral-to-positive news flow.

Recent Company History

Over the last few months, Axsome reported strong Q3 2025 revenue growth while remaining loss-making, acquired epilepsy asset AZD7325, and maintained an active investor-relations presence via multiple conferences. Community-focused mental health outreach also coincided with a positive move. These events showed mostly aligned, moderate price reactions. Today’s FDA acceptance and Priority Review for AXS-05 in Alzheimer’s agitation builds on the earlier sNDA submission referenced in the 3Q2025 update, marking progression from filing to active FDA review.

Market Pulse Summary

The stock surged +22.8% in the session following this news. A strong positive reaction aligns with t...

Analysis

The stock surged +22.8% in the session following this news. A strong positive reaction aligns with the FDA’s acceptance and Priority Review of the AXS-05 sNDA in Alzheimer’s agitation, plus a defined PDUFA date of April 30, 2026. Historically, Axsome’s clinical announcements often saw negative moves despite positive data, so a sustained advance would mark a shift from earlier patterns. Investors could monitor future regulatory updates and any additional clinical disclosures around AXS-05’s risk–benefit profile.

Key Terms

Priority Review, PDUFA, supplemental New Drug Application, NDA, +4 more

8 terms

Priority Review regulatory

"FDA grants AXS-05 Priority Review designation and sets PDUFA..."

Priority review is a regulatory fast-track that shortens the time an agency spends evaluating a drug, vaccine or medical device application so a decision comes sooner than normal. For investors, it matters because a faster review is like an express lane to market: it can speed revenue potential and reduce regulatory uncertainty, but it does not guarantee approval and still requires the product to meet safety and effectiveness standards.

PDUFA regulatory

"The FDA has set a Prescription Drug User Fee Act (PDUFA) target..."

PDUFA, short for the Prescription Drug User Fee Act, is a law that allows drug companies to pay fees to the government to speed up the review process for new medicines. This helps bring important drugs to market more quickly, which can impact their availability and pricing. For investors, PDUFA timelines can influence the timing of a drug’s approval and potential market success.

supplemental New Drug Application regulatory

"has accepted for filing the Company’s supplemental New Drug Application (NDA)..."

A supplemental new drug application is a request submitted to regulatory authorities to make changes to an existing approved medication, such as adding new uses, strengths, or formulations. For investors, it signals that a pharmaceutical company is seeking approval for new product developments or expanded applications, which can impact the company's future sales, market potential, and stock value.

NDA regulatory

"supplemental New Drug Application (NDA) for AXS-05..."

An NDA, or nondisclosure agreement, is a legal contract that keeps certain information private between parties. It’s like a promise not to share sensitive details, helping protect business ideas, strategies, or data from being leaked or used without permission. For investors, NDAs help ensure that confidential information remains secure, enabling trust and open communication during business discussions.

Breakthrough Therapy designation regulatory

"The FDA previously granted Breakthrough Therapy designation for AXS-05..."

A breakthrough therapy designation is a regulatory fast-track given to a drug or treatment that shows early signs of providing a major improvement over existing options for a serious condition. Think of it as a VIP lane that can speed up development and more intensive guidance from regulators, which matters to investors because it can shorten time to market, reduce development risk and potentially increase a company’s value — though it does not guarantee approval.

Phase 3 clinical trials medical

"including four randomized, double-blind, controlled Phase 3 clinical trials..."

Phase 3 clinical trials are large, late-stage studies that test a drug or medical treatment in hundreds to thousands of patients to confirm it is safe and effective and to compare it with existing options. Investors care because positive results are a key step toward regulatory approval and commercial sales, reducing uncertainty much like a full dress rehearsal before a product launch; negative results can sharply reduce a program’s value.

randomized, double-blind, controlled medical

"including four randomized, double-blind, controlled Phase 3 clinical trials..."

A randomized, double-blind, controlled study is a way to test a treatment where participants are assigned by chance to different groups, neither the participants nor the researchers know who gets which treatment, and one group serves as a comparison. Like flipping a coin and hiding the result, this design reduces bias and makes results more reliable, so investors can better judge whether a therapy is likely to work, gain approval, or reach the market.

long-term safety trial medical

"and a long-term safety trial."

A long-term safety trial is a clinical study that follows patients over an extended period to monitor whether a medical treatment causes harmful effects and to track its safety as real-world use accumulates. Like taking a car on a long road trip to reveal problems that don't show up on short drives, these trials matter to investors because their results can affect regulatory approval, ongoing sales, liability risk, labeling, and the size and timing of future revenue.

AI-generated analysis. Not financial advice.

FDA grants AXS-05 Priority Review designation and sets PDUFA action goal date of April 30, 2026

NEW YORK, Dec. 31, 2025 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s supplemental New Drug Application (NDA) for AXS-05 (dextromethorphan HBr and bupropion HCl) for the treatment of Alzheimer’s disease agitation, and has granted the application Priority Review designation. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2026.

Priority Review is granted by the FDA to applications for medicines that, if approved, would provide significant improvements in the effectiveness or safety of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. A Priority Review designation means the FDA’s goal is to take action on an application within 6 months, compared to 10 months under standard review.

“We are very pleased the FDA has accepted and granted priority review to our supplemental NDA for AXS-05 for the treatment of Alzheimer’s disease agitation,” said Herriot Tabuteau, MD, Chief Executive Officer of Axsome. “Up to 76% of people with Alzheimer’s disease experience agitation, representing a significant unmet medical need for patients and their caregivers, and currently there is a dearth of approved treatments. We look forward to continuing to work with the FDA for the remainder of the review.”

The FDA previously granted Breakthrough Therapy designation for AXS-05 for the treatment of Alzheimer’s disease agitation in June 2020. 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 supplemental NDA is the culmination of a comprehensive clinical development program of AXS-05 in Alzheimer’s disease agitation, including four randomized, double-blind, controlled Phase 3 clinical trials and a long-term safety trial.

About Alzheimer’s Disease Agitation

Alzheimer’s disease (AD) is the most common form of dementia, affecting approximately 7 million people in the United States.¹ Agitation is reported in up to 76% of patients with AD and is characterized by emotional distress, verbal and physical aggressiveness, disruptive irritability, and disinhibition.^1,2 AD agitation has been associated with accelerated cognitive decline, increased caregiver burden, earlier nursing home placement, and increased mortality.³

About AXS-05

AXS-05 (dextromethorphan-bupropion) is a novel, oral, investigational N-methyl-D-aspartate (NMDA) receptor antagonist, sigma-1 agonist, and aminoketone CYP2D6 inhibitor under development for the treatment of Alzheimer’s disease (AD) agitation and smoking cessation. AXS-05 utilizes a proprietary formulation and dose of dextromethorphan and bupropion, and Axsome’s metabolic inhibition technology, to modulate the delivery of the components. The dextromethorphan component of AXS-05 is an uncompetitive NMDA receptor antagonist, also known as a glutamate receptor modulator, and a sigma-1 receptor agonist. The bupropion component of AXS-05 serves to increase the bioavailability of dextromethorphan and is a norepinephrine and dopamine reuptake inhibitor. AXS-05 is covered by a robust patent estate extending out to at least 2043. AXS-05 was granted U.S. FDA Breakthrough Therapy designation for the treatment of Alzheimer’s disease agitation in June 2020. AXS-05 (AUVELITY^®) is approved in the U.S. for the treatment of major depressive disorder in adults. AXS-05 is not approved by the FDA for Alzheimer’s disease agitation.

About Axsome Therapeutics

Axsome Therapeutics is a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) conditions. We deliver scientific breakthroughs by identifying critical gaps in care and develop differentiated products with a focus on novel mechanisms of action that enable meaningful advancements in patient outcomes. Our industry-leading neuroscience portfolio includes FDA-approved treatments for major depressive disorder, excessive daytime sleepiness associated with narcolepsy and obstructive sleep apnea, and migraine, and multiple late-stage development programs addressing a broad range of serious neurological and psychiatric conditions that impact over 150 million people in the United States. Together, we are on a mission to solve some of the brain’s biggest problems so patients and their loved ones can flourish. For more information, please visit us at www.axsome.com and follow us on LinkedIn and X.

Forward Looking Statements

Certain matters discussed in this press release are “forward-looking statements”. The Company 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 commercial success of the Company’s SUNOSI^®, AUVELITY^®, and SYMBRAVO^® products and the success of the Company’s efforts to obtain any additional indication(s) with respect to solriamfetol and/or AXS-05; the Company’s ability to maintain and expand payer coverage; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund the Company’s disclosed clinical trials, which assumes no material changes to the Company’s currently projected revenues or expenses), futility analyses and receipt of interim results, which are not necessarily indicative of the final results of the Company’s 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 Company’s current product candidates; the Company’s ability to fund additional clinical trials to continue the advancement of the Company’s product candidates; the timing of and the Company’s 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 Company’s product candidates, including statements regarding the timing of any NDA submission; 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 Company’s ability to successfully resolve any intellectual property litigation, and even if such disputes are settled, whether the applicable federal agencies will approve of such settlements; 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 products and product candidates, if approved; the Company’s anticipated capital requirements, including the amount of capital required for the commercialization of SUNOSI, AUVELITY, and SYMBRAVO and for the Company’s commercial launch of its other product candidates, if approved, and the potential impact on the Company’s anticipated cash runway; the Company’s 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 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 circumstances.

Investors:
Ashley Dong
Director, Investor Relations
(929) 687-1614
adong@axsome.com

Media:
Darren Opland
Senior Director, Corporate Communications
(929) 837-1065
dopland@axsome.com

References

1. Alzheimer’s Association. 2025 Alzheimer’s Disease Facts and Figures.
2. Van der Mussele, S. et al. Agitation-associated behavioral symptoms in mild cognitive impairment and Alzheimer's dementia. Aging Ment Health. 2015;19(3):247-57.
3. Porsteinsson, A.P. and Antonsdottir, I.M. An update on the advancements in the treatment of agitation in Alzheimer’s disease. Expert Opin Pharmacother. 2017 Apr;18(6):611-620.
   

FAQ

What did Axsome (AXSM) announce about AXS-05 on December 31, 2025?

Axsome announced the FDA accepted its supplemental NDA for AXS-05 and granted Priority Review with a PDUFA date of April 30, 2026.

What is the PDUFA action date for Axsome's AXS-05 (AXSM)?

The FDA set a PDUFA target action date of April 30, 2026 for the AXS-05 supplemental NDA.

Why did the FDA grant Priority Review to AXS-05 (AXSM)?

Priority Review was granted because the application could provide significant improvements in treating a serious condition versus standard applications.

What clinical data supported Axsome's AXS-05 supplemental NDA (AXSM)?

The supplemental NDA reflects a program including four randomized, double-blind, controlled Phase 3 trials and a long-term safety trial.

Does AXS-05 have any prior regulatory designations (AXSM)?

Yes; the FDA previously granted Breakthrough Therapy designation for AXS-05 for Alzheimer’s disease agitation in June 2020.

How common is agitation in Alzheimer’s patients according to Axsome (AXSM)?

Axsome cites that up to 76% of people with Alzheimer’s disease experience agitation.