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© Axsome Therapeutics, Inc. February 23, 2026 4Q and Full Year 2025 Financial Results
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© Axsome Therapeutics, Inc. Forward looking statements & safe harbor 2 Certain matters discussed in this presentation 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 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 presentation, and the Company undertakes no obligation to publicly update such forward - looking statements to reflect subsequent events or circumstances . This presentation contains statements regarding the Company’s observations based upon the reported clinical data . This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and other data about the Company's industry . This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates . Neither we nor any other person makes any representation as to the accuracy or completeness of such data or undertakes any obligation to update such data after the date of this presentation . In addition, these projections, assumptions and estimates are necessarily subject to a high degree of uncertainty and risk . Axsome, AUVELITY, SUNOSI, SYMBRAVO, and MoSEIC , are trademarks or registered trademarks of Axsome Therapeutics, Inc . or its affiliates . Except as with respect to AUVELITY and SUNOSI for their approved indications, the development products referenced herein have not been approved by the FDA . 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 .
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© Axsome Therapeutics, Inc. 3 Our Mission Develop and deliver transformative medicines for the hundreds of millions of people impacted by central nervous system conditions
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© Axsome Therapeutics, Inc. 4 Deep neuroscience expertise R&D and regulatory innovation Data - driven execution Disciplined capital allocation Delivered $639M in total revenue across three commercial products, representing 66% year - over - year growth AUVELITY surpassed half a billion dollars in annual sales in third full year of launch Advancing toward potential expansion of AUVELITY into Alzheimer’s disease agitation (PDUFA date: April 30, 2026) Multiple late - stage programs progressing toward key milestones Acquired novel oral GABA A α 2,3 receptor PAM, expanding leading CNS pipeline to five novel product candidates Deep CNS portfolio across multiple stages of development Substantial commercial opportunities across large and underserved CNS markets Clear path to sustained cash flow positivity 2025 was a year of significant innovation and growth across the business How we drive growth 2025 highlights Positioned for long - term value creation © Axsome Therapeutics, Inc.
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© Axsome Therapeutics, Inc. 2026 strategic focus areas 5 Drive continued strong growth across our commercial portfolio Position AUVELITY for a successful potential launch in Alzheimer’s disease agitation Advance high - value pipeline programs and deliver on key regulatory and R&D milestones Scale efficiently and maintain operational excellence toward durable cash flow positivity © Axsome Therapeutics, Inc.
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© Axsome Therapeutics, Inc. Robust path to durable value creation and long - term growth 6 Innovative medicines Novel product candidates Late - stage clinical trials underway or initiating Programs with blockbuster potential CNS conditions of critical unmet need 3 5 7 9 11 >$16B peak sales potential Robust intellectual property portfolio with patent protection through the 2040s >150M patients affected across indications
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© Axsome Therapeutics, Inc. NMDA = N - methyl - D - aspartate; CYP2D6 = Cytochrome P450 Family 2 Subfamily D Member 6; DNRI = Dopamine - norepinephrine reuptake inh ibitor; TAAR1 = Trace amine - associated receptor 1; 5 - HT = 5 - Hydroxytryptamine; NRI = Norepinephrine reuptake inhibitor; GABA = gamma - aminobutyric acid Please see full Prescribing Information for AUVELITY, SUNOSI, and SYMBRAVO at www.AUVELITY.com , www.SUNOSI.com , and www.SYMBRAVO.com , respectively. Phase 1 Phase 2 Phase 3 NDA Marketed Broad and expanding CNS pipeline 7 FDA Breakthrough Therapy Designation Major Depressive Disorder Alzheimer’s Disease Agitation Smoking Cessation Attention Deficit Hyperactivity Disorder Binge Eating Disorder Major Depressive Disorder with EDS Symptoms Excessive Daytime Sleepiness in Narcolepsy or OSA Migraine Fibromyalgia Shift Work Disorder Psychiatry Neurology AXS-05 (dextromethorphan-bupropion) NMDA antagonist, sigma - 1 agonist, and aminoketone CYP2D6 inhibitor Solriamfetol DNRI , TAAR1 agonist, 5 - HT 1A agonist AXS-12 (reboxetine) Highly selective NRI, dopamine mod. AXS-14 (esreboxetine) [S , S] - enantiomer of AXS - 12 Solriamfetol DNRI , TAAR1 agonist, 5 - HT 1A agonist Narcolepsy FDA Orphan Drug Designation AXS-17 GABA A α 2,3 subtype - selective PAM Epilepsy
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© Axsome Therapeutics, Inc. Advancing new frontiers in high - impact CNS conditions 8 1. Major Depression. NIMH 2023; 2. Rush AJ, et al. Am J Psychiatry 2006; 3. Benjafield AV, et al. Lancet Respir Med. 2020; 4. Watson N, et al. Sleep 2025; 5. American Migraine Foundation 2023; 6. Symphony Health Claims, New to Brand Patients 2022 - 2023; 7. 2025 Alzheimer’s Disease Facts and Figures; 8. “About Narcolepsy.” Narcolepsy Network 2024; 9. Swick TJ. Nat Sci Sleep 2015; 10. Vincent, et al. Arthritis Care Res (Hoboken) 2013; 11. Liu Y, et al. J Manag Care Spec Pharm. 2016; 12. Facts About ADHD in Adults. CDC 2024; 13. Sibley MH, et al. Am J Psychiatry 2022; 14. Hudson JI, et al. Biol Psychiatry 2007; 15. Sateia MJ. Chest 2014; 16. Alterman T, et al. Am J Ind Med. 2013; 17. Wickwire EM. Chest 2017; 18. Hein M, et al. J Affect Disord . 2019; 19. U.S. Department of Health and Human Services 2020; 20. Hughes JR, et al. Addiction 2004; 21. Epilepsy Facts and Stats. CDC 2025; 22. Chen Z, et al. Jama Neurol. 2018 Approved indications driving today’s growth Advanced programs in key indications Lifecycle expansion and future growth opportunities Major depressive disorder 21M+ ~ ൗ2 3 people in the U.S. live with MDD 1 of patients fail to achieve remission from initial therapy 2 Obstructive sleep apnea 22M+ ~80% U.S. adults are affected by OSA 3 of patients remain undiagnosed 4 39M+ Migraine >80% U.S. adults experience migraine 5 of patients discontinue their acute migraine treatment in the first 12 months 6 Shift work disorder 15M+ 0 working Americans may be impacted 15 - 17 new medications approved since 2007 Smoking cessation 34M+ ~70% adults in the U.S. smoke cigarettes 19 of smokers say they want to quit 20 MDD with EDS symptoms ~50% 0 of MDD patients have concomitant EDS 18 FDA - approved treatments ADHD 22M+ >90% people in the U.S. live with ADHD 12 of pediatric ADHD persist into adulthood 3 7M+ people impacted in the U.S. 14 Binge eating disorder FDA - approved treatment 1 Epilepsy ~3.4M >1/3 people in the U.S. live with epilepsy 21 of patients don’t respond to treatment 22 Alzheimer’s disease agitation 5M+ 1 U.S. individuals with Alzheimer’s disease experience agitation 7 FDA - approved treatment Narcolepsy 185K ~70% people in the U.S. are affected by narcolepsy 8 of patients suffer from cataplexy 9 >50% people in the U.S. have fibromyalgia 10 17M+ Fibromyalgia of patients discontinue treatment in the first year 11
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© Axsome Therapeutics, Inc. Strong pipeline execution with multiple upcoming milestones 9 Upcoming milestones Clinical Trial Topline Results Clinical Trial Initiations • Positive topline results from EMERGE Phase 3 trial of SYMBRAVO in oral CGRP non - responders • Positive topline results from FOCUS Phase 3 trial of solriamfetol in ADHD in adults • Topline results from PARADIGM Phase 3 trial of solriamfetol in MDD • ENGAGE Phase 3 trial of solriamfetol in BED (2H 2026) • SUSTAIN Phase 3 trial of solriamfetol in SWD (2027) 2025 & recent achievements • Initiate Phase 3 trial of solriamfetol in MDD with EDS symptoms (1Q 2026) • Initiate Phase 2/3 trial of AXS - 05 in smoking cessation (2Q 2026) • Initiate Phase 3 trial of solriamfetol in children with ADHD (1H 2026) • Initiate Phase 3 trial of solriamfetol in adolescents with ADHD (1H 2026) • FDA approval and launch of SYMBRAVO in the U.S. • sNDA for AXS - 05 in Alzheimer’s disease agitation granted Priority Review designation • NDA submission for AXS - 12 for cataplexy in narcolepsy (1Q 2026) • AXS - 05 in Alzheimer’s disease agitation PDUFA target action date (April 30, 2026) Regulatory & Commercial • Initiated FORWARD Phase 3 trial of AXS - 14 in fibromyalgia
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© Axsome Therapeutics, Inc. Diversified growth drivers supporting >$16B in combined peak sales potential 10 AXS - 05 AD Agitation $0.5-$1B AXS - 14 Fibromyalgia AXS - 12 Narcolepsy $0.5-$1B $1.5-$3B Solriamfetol MDD with EDS Solriamfetol $1-$3B Solriamfetol $0.3-$0.5B Solriamfetol SWD $0.5-$1B AXS - 05 Smoking Cessation $1-$1.5B $0.5-$1B $0.3-$0.5B $1-$3B $0.5-$1B AXS - 17 Epilepsy BED ADHD
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© Axsome Therapeutics, Inc. 4Q and full year 2025 financial summary 11 4Q = three months ended December 31 ; †Includes royalty revenue associated with sales in out - licensed territories Net Product Revenue $196.0 $118.8 65% $638.5 $385.7 66% AUVELITY Net Product Sales $155.1 $92.6 68% $507.1 $291.4 74% SUNOSI Net Product Revenue † $36.7 $26.2 40% $124.8 $94.3 32% SYMBRAVO Net Product Sales $4.1 ̶ ̶ $6.6 ̶ ̶ R&D Expense $48.8 $55.0 -11% $183.3 $187.1 -2% SG&A Expense $169.3 $113.3 49% $570.6 $411.4 39% 4Q 2025 4Q 2024 % Change FY 2025 FY 2024 % Change $ millions
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© Axsome Therapeutics, Inc. Cash Balance: (as of December 31, 2025) $322.9M Debt (Face Value): (as of December 31, 2025) $190M Market Cap: (as of February 20, 2026) $9.4B Shares Outstanding: (as of December 31, 2025) 50.9M Options, RSUs, and Others Outstanding*: 8.8M Runway to reach cash flow positivity, based on the current operating plan Financial snapshot 12 *Includes 7.0M options, 1.5M RSUs, and 0.2M others as of December 31, 2025
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Commercial Highlights
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© Axsome Therapeutics, Inc. Scaling commercial growth and adoption across our innovative CNS medicines 14 EDS = Excessive daytime sleepiness; OSA = Obstructive sleep apnea Rx, sales, and revenue growth vs. comparable periods in 2024 • Expanding adoption across psychiatry and primary care segments • Recently initiated sales force expansion to approximately 600 sales representatives Migraine with or without auraMajor depressive disorder EDS in narcolepsy or OSA • Continued strong growth across OSA and narcolepsy markets • High patient satisfaction supporting durable utilization • Disciplined launch driving growing awareness and advocacy among highest volume migraine prescribers • Continued progress with market access and payer coverage ~$5B combined peak sales potential across marketed products
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© Axsome Therapeutics, Inc. 0 20,000 40,000 60,000 80,000 100,000 120,000 140,000 160,000 180,000 200,000 220,000 240,000 2022-Q4 2023-Q1 2023-Q2 2023-Q3 2023-Q4 2024-Q1 2024-Q2 2024-Q3 2024-Q4 2025-Q1 2025-Q2 2025-Q3 2025-Q4 Source: Symphony METYS Quarterly TRx Launch to Date Broadening adoption and scaling growth 15 TRx = Total prescriptions >60% YoY growth in primary care TRx prescribers in 4Q 2025 >280,000 New patients since launch ~52,000 Unique writers since launch ~86% Covered lives all channels ~50% First - line or second - line use ~55% Monotherapy use ~78% and ~100% Covered lives commercial and government channels >225,000 Total prescriptions in 4Q 2025 (+42% YoY) ~50% First - or second - line use >50% Monotherapy use
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© Axsome Therapeutics, Inc. 0 5,000 10,000 15,000 20,000 25,000 30,000 35,000 40,000 45,000 50,000 55,000 60,000 Q3-2019 Q4-2019 Q1-2020 Q2-2020 Q3-2020 Q4-2020 Q1-2021 Q2-2021 Q3-2021 Q4-2021 Q1-2022 Q2-2022 Q3-2022 Q4-2022 Q1-2023 Q2-2023 Q3-2023 Q4-2023 Q1-2024 Q2-2024 Q3-2024 Q4-2024 Q1-2025 Q2-2025 Q3-2025 Q4-2025 Durable demand driving continued strong performance 16 nTRx = Normalized total prescriptions Source: Symphony METYS. nTRx normalizes number of pills in each Trx for 30 - day period. ~98,000 New patients since launch ~15,600 Unique writers since launch ~82% Covered lives all channels >50% Of patients who switch from or add on to their current treatment with SUNOSI come from other WPA agents >54,000 Total prescriptions in 4Q 2025 (+11% YoY) Quarterly nTRx Launch to Date
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© Axsome Therapeutics, Inc. 0 200 400 600 800 1,000 1,200 1,400 6/13 6/20 6/27 7/4 7/11 7/18 7/25 8/1 8/8 8/15 8/22 8/29 9/5 9/12 9/19 9/26 10/3 10/10 10/17 10/24 10/31 11/7 11/14 11/21 11/28 12/5 12/12 12/19 12/26 1/2 Growing awareness and improving patient access 17 TRx = Total prescriptions Source: Symphony METYS >13,000 Total prescriptions in 4Q 2025 ~5,300 New patient starts in 4Q 2025 ~52% Covered lives all channels Weekly nTRx Launch to Date ~49% Covered lives commercial ~57% Covered lives government
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Development Pipeline
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© Axsome Therapeutics, Inc. AXS - 05 is believed to modulate the function of neurotransmitters and receptors i mplicated in Alzheimer’s disease (glutamate, sigma - 1, norepinephrine, and dopamine) 1 - 4 19 In Alzheimer’s disease, insoluble A β production and accumulation triggers secondary steps leading to synaptic loss and neuronal cell death 1,2 Reductions in certain neurotransmitters are thought to contribute to cognitive and behavioral symptoms including agitation and aggression 1 - 4 Potentially first - in - class, best - in - class treatment for Alzheimer’s disease agitation AXS - 05 (dextromethorphan - bupropion) 1. Cummings JL, N Engl J Med. 2004; 2. Querfurth HW & LaFerla FM, N Engl J Med. 2010; 3. Porsteinsson AP & Antonsdottir IM, Expert Opin Pharmacother . 2017; 4. Rosenberg PB, Nowrangi MA, & Lyketsos CG, Mol Aspects Med. 2015; 5. Stahl SM, CNS Spectr . 2019; 6. Cheng W, et al. Mol Med Rep. 2015 5,6
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© Axsome Therapeutics, Inc. Alzheimer’s disease (AD) agitation 20 1. 2025 Alzheimer’s Disease Facts and Figures; 2. Trachtenberg et al. J Neuropsychiatry Clin Neurosci . 2002 7M 14M 2024 2030 2040 2050 2060 Number of U.S. adults aged 65+ with Alzheimer’s dementia expected to double by 2060 1 20 Alzheimer’s disease (AD) is the most common form of dementia, affecting over 7M people in the U.S. 1 AD agitation is characterized by emotional distress, verbal and physical aggressiveness, disruptive irritability, and disinhibition 1,2 Agitation is one of the most common and debilitating neuropsychiatric symptoms affecting up to 7 6% of people 1,2
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© Axsome Therapeutics, Inc. sNDA for AXS-05 in Alzheimer’s disease agitation granted Priority Review with PDUFA target action date of April 30, 2026 Statistically significant and clinically meaningful improvements in Alzheimer’s disease agitation 21 CMAI = Cohen - Mansfield Agitation Inventory -18 -16 -14 -12 -10 -8 -6 -4 -2 0 Baseline 1 2 3 4 5 CMAI Total Score Change from Baseline Week ADVANCE - 1 AXS-05 Bupropion Placebo p =0.069 p =0.007 p =0.010 0 Weeks from Randomization Probability of Freedom from Relapse (%) ACCORD - 2 100 75 50 25 0 2 4 6 8 10 12 14 16 18 20 22 24 26 + Censored Log - Rank p - value: 0.001 AXS - 05 Placebo Hazard Ratio for Time to Relapse Hazard Ratio (95% CI) 0.276 (0.119 - 0.641) p-value 0.001
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© Axsome Therapeutics, Inc. 70% of smokers want to quit 2 Only 3 - 5% who attempt to quit without assistance are successful for 6 - 12 months 2 Smoking cessation 22 Single largest cause of preventable disease and death in the U.S., accounting for nearly 1 in 5 deaths 1 Associated with over $300 billion in annual costs in the U.S. 1 ~34M adults in the U.S. smoke cigarettes, ~50% of whom live with a smoking - related disease 1 1. U.S. Department of Health and Human Services 2020; 2. Hughes JR, et al. Addiction 2004
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© Axsome Therapeutics, Inc. 23 1. Raony I, et al. Prog Neuropsychopharmacol Biol Psychiatry 2022; 2. Halff EF, et al. Trends Neurosci . 2023 Solriamfetol was initially developed as a dopamine and norepinephrine reuptake inhibitor (DNRI) with wake - promoting effects Preclinical and clinical evidence 1,2 suggest TAAR1 plays a role in neuropsychiatric conditions related to the dysregulation of monoaminergic transmission Multimodal activity of solriamfetol selectively inhibits the reuptake of dopamine and norepinephrine and exhibits agonist activity at TAAR1 receptors in the brain Unique pharmacology supports potential utility in a broad range of CNS conditions Solriamfetol
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© Axsome Therapeutics, Inc. Solriamfetol Phase 3 development programs 24 ADHD = Attention deficit hyperactivity disorder; MDD = Major depressive disorder; BED = Binge eating disorder; SWD = Shift wo rk disorder ADHD Positive FOCUS Phase 3 trial in adults with ADHD (N=516) • Initiation of two Phase 3 trials in children and adolescents with ADHD anticipated in 1H 2026 Approved in EDS associated with OSA and narcolepsy MDD with EDS BED SWD • Initiation of Phase 3 trial in MDD with EDS symptoms anticipated in 1Q 2026 • Ongoing ENGAGE Phase 3 trial evaluating efficacy and safety of solriamfetol vs. placebo in adults with binge eating disorder (N=450) • Topline data anticipated in 2H 2026 • Ongoing SUSTAIN Phase 3 trial evaluating efficacy and safety of solriamfetol vs. placebo in adults with shift work disorder (N=450) • Topline data anticipated in 2027 Solriamfetol
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© Axsome Therapeutics, Inc. Attention deficit hyperactivity disorder (ADHD) 25 1. Facts About ADHD in Adults. CDC 2024; 2. Data and Statistics on ADHD. CDC 2024; 3. Attention - Deficit/Hyperactivity Disorder. NIMH 2024 Chronic neurobiological and developmental disorder affecting an estimated ~ 22M people in the U.S. 1 , including ~7M children aged 3 - 17 years old 2 Characterized by a persistent pattern of inattention and/or hyperactive - impulsive behaviors 3 Associated with significant impairment in social, academic, and occupational functioning and development 3
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© Axsome Therapeutics, Inc. Statistically significant improvements in ADHD symptoms with solriamfetol treatment 26 1. p - values shown for solriamfetol 150 mg dose vs. placebo only ( primary efficacy analysis) Substantial reduction in the AISRS score of 17.7 points at Week 6, representing a 45% improvement in ADHD symptoms from baseline (p=0.039, solriamfetol 150 mg) Significantly greater percentage of patients achieved a clinical response ( ≥30% reduction in AISRS) vs. placebo (p=0.024, solriamfetol 150 mg) Improvements in severity of overall ADHD as measured by the CGI - S total score at Week 6 (p=0.017, solriamfetol 150 mg) Well tolerated with a side effect profile consistent with the established safety profile of solriamfetol -20 -15 -10 -5 0 Baseline Week 1 Week 2 Week 3 Week 4 Week 6 AISRS Total Score LS Mean Change from Baseline FOCUS Phase 3 trial p =0.036 p =0.041 p =0.039 Solriamfetol 300 mg Solriamfetol 150 mg Placebo
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© Axsome Therapeutics, Inc. MDD with excessive daytime sleepiness symptoms 27 Major depressive disorder (MDD) is one of the most common mental disorders in the U.S., impacting ~21M adults each year 2,3 Approximately 50% of patients with MDD also experience excessive daytime sleepiness (EDS) 4 , for which there are no approved treatments MDD patients with EDS have difficulty maintaining wakefulness, resulting in impaired daily functioning and increased safety risks 1. Rush AJ, et al. Am J Psychiatry 2006; 2. Major Depression. NIMH 2023; 3. Hasin DS, et al. JAMA Psychiatry 2018; 4. Hein M, et al. J Affect Disord . 2019
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© Axsome Therapeutics, Inc. Binge eating disorder 28 1. Hudson JI, et al. Biol Psychiatry 2007; 2. Swanson SA, et al. Arch Gen Psychiatry 2011; 3. Kessler RM, et al. Neurosci Biobehav Rev. 2016; 4. McElroy SL, et al. J Clin Psychiatry 2020; BED is thought to involve issues with food reward processing, impulse control, cognitive control, and appetite regulation 1,3 Unmet medical need associated with a 2 - to 3 - fold increased risk of psychiatric and medical comorbidities 4 >7 million people in the U.S. have BED 1 Binge eating disorder (BED) is the most common eating disorder, affecting 2.8% of adults and 1.6% of adolescents in the US 1,2 BED is 1.75x more common in women than in men 1
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© Axsome Therapeutics, Inc. Solriamfetol inhibits the reuptake of dopamine and norepinephrine, neurotransmitters implicated in the pathophysiology of binge eating disorder 1 - 3 Pre - clinical and clinical data support potential effects of solriamfetol on appetite, food consumption, and weight 4,5 Evaluating solriamfetol as a potential treatment for binge eating disorder 29 TAAR1 = Trace amine - associated receptor 1 1. Giel KE, et al. Nat Rev Dis Primer 2022; 2. Bello NT and Hajnal A. Pharmacol Biochem Behav . 2010; 3. Pruccoli J, et al. Int J Mol Sci. 2021; 4. Malhotra A, et al. Sleep Med. 2022; 5. SUNOSI [Prescribing Information]. Axsome Therapeutics, Inc. New York, NY. Solriamfetol (150 mg) Solriamfetol (300 mg) Placebo 1:1:1 R Screening (4 weeks) Double - blind Phase (12 weeks) Follow - up (1 week) Baseline ENGAGE Phase 3 Trial N=450 Key eligibility criteria • 18 - 55 years of age with diagnosis of BED (DSM - 5) Primary endpoint • Change from baseline in days with binge eating episodes
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© Axsome Therapeutics, Inc. Shift work has long been associated with multiple serious health complaints and a 23% greater risk of sustaining a work - related injury 4 - 5 Shift work disorder 30 No new medications approved since 2007 and considerable residual sleepiness reported when medication is used 6 ~15 million U.S. workers may suffer from SWD Approximately 1 in 3 people working in the U.S. work an alternate shift 2 10 - 43% have SWD 1,3 Shift work disorder (SWD) is a combination of excessive sleepiness during wakefulness and persistent insomnia during daytime sleep when working outside a 7 a.m. to 6 p.m. workday 1 1. Sateia MJ. Chest 2014; 2. Alterman T, et al. Am J Ind Med. 2013; 3. Wickwire EM. Chest 2017; 4. Smith L, et al. Lancet 1994; 5. Akerstedt T & Wright KP. Sleep Med Clin. 2009; 6. Czeisler CA, et al. N Engl J Med. 2005
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© Axsome Therapeutics, Inc. Evaluating solriamfetol as a potential treatment for shift work disorder 31 CGI - C = Clinical Global Impression - Change Key eligibility criteria • 18 - 65 years of age with diagnosis of SWD (ICSD - 2 or ICSD - 3) Solriamfetol (150 mg) Solriamfetol (300 mg) Placebo Screening (1 - 4 weeks) Double - blind Phase (12 weeks) Follow - up (1 week) Baseline SUSTAIN Phase 3 Trial N=450 1:1:1 R Primary endpoint • Change from baseline in CGI - C score
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© Axsome Therapeutics, Inc. 32 1. Szabo ST, et al. Sleep Med Rev. 2019; 2. Krahn LE, Zee PC, & Thorpy MJ. Adv Ther. 2022; 3. Scammell TE. N Engl J Med. 2015; 4. Stahl SM & Grady MM. J Clin Psychiatry 2003; 5. Burgess CR & Peever JH. Curr Biol. 2013; 6. Wu MF, et al. Neuroscience 1999; 7. Bruinstroop E, et al. J Comp Neurol. 2012 Norepinephrine and dopamine play important roles in sleep - wake regulation (both) and in maintaining muscle tone during wakefulness (norepinephrine) 1 - 3 AXS - 12 inhibits the reuptake of both neurotransmitters, improving both norepinephrine and cortical dopamine signaling in the brain The loss of orexin input inhibits the production of these neurotransmitters 1,2 • Decreased norepinephrine signaling is thought to contribute to cataplexy, EDS, and cognitive impairment 1,4 - 7 • Decreased dopamine signaling is thought to contribute to EDS and cognitive impairment 1,4 Novel pharmacological approach for the treatment of narcolepsy AXS - 12 (reboxetine)
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© Axsome Therapeutics, Inc. Narcolepsy 33 Rare and debilitating neurological condition that affects approximately 185,000 people in the U.S. 1 Characterized by cataplexy, excessive daytime sleepiness (EDS), hypnagogic hallucinations, sleep paralysis, and disrupted nocturnal sleep 2 - 4 An estimated 70% of patients suffer from cataplexy, or the sudden reduction or loss of muscle tone while awake 5 1. “About Narcolepsy.” Narcolepsy Network 2024; 2. Sateia MJ. Chest 2014; 3. “Narcolepsy.” NINDS 2024; 4. España RA & Scammell TE. Sleep 2011; 5. Swick TJ. Nat Sci Sleep 2015
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© Axsome Therapeutics, Inc. 0 5 10 15 20 25 Baseline Week 1 Week 2 Week 3 Week 4 Week 5 Weekly cataplexy attacks (median) Placebo AXS-12 -20 -16 -12 -8 -4 0 4 Baseline Week 1 Week 2 Change in weekly cataplexy attacks (median) Placebo AXS-12 Rate ratio* 0.65 0.49 0.53 0.53 0.49 0.0 0.5 1.0 Week 1 Week 2 Week 3 Week 4 Week 5 p=0.007 p=0.006 p=0.031 p=0.031 p=0.018 p<0.001 p=0.002 *Ratio of change in the AXS - 12 group divided by the ratio of change in the placebo group (rate ratio of 1 = no difference) CONCERT SYMPHONY Rapid and robust reductions in cataplexy with AXS - 12 treatment 34 New Drug Application (NDA) submission anticipated in 1Q 2026
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© Axsome Therapeutics, Inc. 35 Fibromyalgia pain is thought to be partially caused by dysregulated signaling in the descending analgesic system Norepinephrine, one of the key neurotransmitters in this pathway, has predominantly pain - inhibitory effects AXS - 14 is a more potent and selective enantiomer of racemic reboxetine that inhibits the reuptake of norepinephrine, resulting in increased norepinephrine activity and decreased pain signaling AXS - 14 ( esreboxetine ) Novel pharmacological approach for the management of fibromyalgia (FM) Adapted from Siracusa, R. et al. Int. J. Mol. Sci. (2021)
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© Axsome Therapeutics, Inc. Fibromyalgia 36 1. Vincent, et al. Arthritis Care Res (Hoboken) 2013; 3. Choy E, et al. BMC Health Serv Res. 2010; 3. Arnold LM, et al. Patient Educ Couns. 2008; 4. Bair MJ & Krebs EE. Ann Intern Med. 2020; 5. Clauw DJ. Ann Rheum Dis. 2024 Chronic and debilitating neurological pain syndrome resulting from a dysfunction in central pain processing 2,3 Characterized by widespread musculoskeletal pain, fatigue, disturbed sleep, mood disturbances, cognitive impairment, and hypersensitivity to sensory sitmuli 4,5 Associated with substantial physical disability and reduced emotional and social wellbeing, financial burden, and reduced quality of life 2,3 An estimated ~17 million people in the U.S. are impacted by fibromyalgia 1
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© Axsome Therapeutics, Inc. 37 -2.0 -1.5 -1.0 -0.5 0.0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 Weekly mean pain score LS mean change from baseline (SE) Week Placebo 8 mg Baseline * * *** * ** *** *** ** *** *** **** **** **** *** *** **** -2.0 -1.5 -1.0 -0.5 0.0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 Weekly mean pain score LS mean change from baseline (SE) Week Placebo 4 mg Baseline * ** *** *** *** ** *** *** ** **** p<0.05 * p<0.01 ** p<0.001 *** p<0.0001 **** -2.0 -1.5 -1.0 -0.5 0.0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 Weekly mean pain score LS mean change from baseline (SE) Week Placebo 10 mg Baseline * * Pain reduction Phase 3 efficacy results (N=1,122) Efficacy and safety of AXS - 14 compared to placebo evaluated in >1,000 individuals with fibromyalgia across Phase 2 and Phase 3 clinical trials for up to 14 weeks Rapid and significant reductions in pain scores , improvements in patient - reported global functioning, fatigue, and overall symptom severity Rapid and robust improvements in fibromyalgia symptoms with AXS - 14 treatment FORWARD Phase 3 trial initiated in January 2026
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© Axsome Therapeutics, Inc. FORWARD Phase 3 trial design 38 CGI - C = Clinical Global Impression - Change Key eligibility criteria • ≥18 years of age with diagnosis of fibromyalgia (ACR 2016 criteria) Open - label Period (12 weeks) Double - blind Phase (up to 12 weeks) Baseline FORWARD Phase 3 Trial 1:1 R Primary endpoint • Time from randomization to loss of therapeutic response AXS - 14 (8 mg) AXS - 14 (8 mg) Placebo 1:1 Randomization (Patients achieving treatment response)
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© Axsome Therapeutics, Inc. 39 *In - licensed AXS - 17 (formerly AZD7325) via the acquisition of Baergic Bio, Inc., a subsidiary of Avenue Therapeutics, Inc., and concurrent amendment to the License Agreement between Baergic Bio and AstraZeneca, in November 2025 1. Epilepsy Facts and Stats. CDC 2025; 2. Chen Z, et al. Jama Neurol. 2018 Expanding our innovative CNS portfolio with a complementary early - stage product candidate AXS - 17 Acquired AXS-17*, a novel oral GABAA receptor α2,3 subtype-selective positive allosteric modulator (PAM), licensed from AstraZeneca AXS-17 was safe and well tolerated in clinical studies • Favorable safety and tolerability profile demonstrated in clinical studies including >700 patients to date Compelling anti-convulsant activity observed in preclinical seizure models AXS-17 to be evaluated for the treatment of epilepsy • Epilepsy is a chronic and debilitating neurological disorder affecting ~3.4M people in the U.S. 1 • Despite currently available treatment options, <1/3 of patients do not response to treatment 2 Phase 2 trial-enabling activities underway
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© Axsome Therapeutics, Inc. Strong intellectual property and barriers to entry 40 • Protected by a robust patent estate extending to at least 2043; M ultiple pending • Proprietary drug product formulation and methods of treatment • Protected by a robust patent estate extending to at least 2045; Multiple pending • Proprietary MoSEIC TM formulation, drug product formulation, and methods of treatment • Protected by a robust patent estate extending to at least 2042; Multiple pending • Proprietary drug substance, drug product formulation, and methods of treatment • Orphan Drug Designation • Claims extending to at least 2039 • 9 issued U.S. patents and 5 issued O.U.S. patents ; Multiple pending • Proprietary drug substance, drug product formulation, and methods of treatment • Claims extending to at least 2043 • >150 issued U.S. patents and > 130 issued O . U.S. patents ; Multiple pending • Proprietary drug product formulation and methods of treatment • Multiple p ending U.S. patents • Proprietary drug substance, drug product formulation, and methods of treatment AXS - 05 AXS - 12 AXS - 14
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© Axsome Therapeutics, Inc. Leadership team 41 Roger Jeffs, PhD CEO, Liquidia Corporation Former President, Co - CEO, Director United Therapeutics Corp. Prior positions at Amgen and Burroughs Wellcome Herriot Tabuteau, MD Founder & CEO Management Board of Directors Nick Pizzie, CPA, MBA Chief Financial Officer Mark Jacobson, MA Chief Operating Officer Hunter Murdock, JD General Counsel Ari Maizel Chief Commercial Officer Mark Saad CEO, NuLids , LLC Former COO of the Global Healthcare Group at UBS Mark Coleman, MD M edical Director, National Spine and Pain Centers Diplomat of the American Board of Anesthesiology Susan Mahony, PhD Former SVP of Eli Lilly and President Lilly Oncology Prior positions at BMS, Amgen and Schering - Plough Herriot Tabuteau, MD Chairman
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© Axsome Therapeutics, Inc. Thank you