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axsome 2Q 2026 Financial Results | August 10 , 2026
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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 to improve the brain health of millions of individuals
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© Axsome Therapeutics, Inc. Positioned for significant and durable growth 4 3 marketed products MDD Migraine AD agitation EDS in OSA or narcolepsy 6 novel product candidates Singular CNS portfolio Psychiatry + Neurology 10 pipeline indications COMMERCIAL PORTFOLIO BROAD PIPELINE Expanding franchise Continued growth Launch phase Psychiatry Neurology Smoking cessation ADHD Binge eating disorder MDD with EDS symptoms Schizophrenia Narcolepsy Fibromyalgia Shift work disorder Epilepsy Tourette syndrome
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© Axsome Therapeutics, Inc. 2Q 2026 performance 5 COMMERCIAL GROWTH PIPELINE PROGRESS UPCOMING MILESTONES • NDA submission for AXS - 12 for cataplexy in narcolepsy accepted by the FDA • Initiated FOCUS - 2 and FOCUS - 3 Phase 3 trials of solriamfetol in children and adolescent with ADHD • Clinical trials ongoing in five indications of high unmet need • Phase 2 trial - enabling activities for AXS - 17 in epilepsy underway • Phase 3 trial - enabling activities for AXS - 20 in schizophrenia underway • AXS - 12 PDUFA target action (May 1, 2027) • Initiation of pivotal Phase 2/3 trial of AXS - 05 in smoking cessation (3Q 2026) • Topline results of the ENGAGE Phase 3 trial of solriamfetol in binge eating disorder (4Q 2026) • Topline results of the SUSTAIN Phase 3 trial of solriamfetol in shift work disorder (2027) Total product revenue • Completed AUVELITY sales force expansion to ~630 representatives • Launched AUVELITY in Alzheimer’s disease agitation in June $218.4M (+46% YoY)
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© Axsome Therapeutics, Inc. Leading neuroscience pipeline with deep stratification 6 Phase 1 Phase 2 Phase 3 NDA AXS - 05 (dextromethorphan - bupropion) AXS - 12 (reboxetine) AXS - 17 NMDA antagonist and sigma - 1 agonist Highly selective NRI and dopamine modulator GABA A α 2,3 subtype - selective PAM Smoking Cessation Tourette Syndrome Psychiatry Neurology ADHD Binge Eating Disorder MDD with EDS Shift Work Disorder Narcolepsy Fibromyalgia Schizophrenia Epilepsy DNRI and TAAR1 agonist Solriamfetol Highly selective NRI and dopamine modulator AXS - 14 (esreboxetine) Selective PDE10A inhibitor AXS - 20 (balipodect) 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.SYMBR AVO .com, respectively.
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© Axsome Therapeutics, Inc. Advancing new frontiers across 10 serious CNS conditions 7 1. U.S. Department of Health and Human Services 2020; 2. Hughes JR, et al. 2004; 3. Facts About ADHD in Adults. CDC 2024; 4. Sib ley MH, et al. 2022; 5. Hudson JI, et al. 2007; 6. Hein M, et al. 2019; 7. Ringeisen H, et al. 2023; 8. Narcolepsy Network 2024; 9. Swick TJ. 2015; 10. Vincent, et al. 2013; 11. Liu Y, et al. 2016; 12. Sateia MJ. 2014; 13. Alterman T, et al. 2013; 14. Wickwire EM. 2017; 15. Epilepsy CDC 2025; 16. Chen Z, et al. 2018; 17. Tourette Syndrome. CDC 2025 PSYCHIATR Y Shift work disorder 15M+ 0 working Americans may be impacted 12 - 14 new medications approved since 2007 Smoking cessation 34M+ ~70% adults in the U.S. smoke cigarettes 1 of smokers say they want to quit 2 MDD with EDS symptoms ~50% 0 of MDD patients have concomitant EDS 6 FDA - approved treatments ADHD 22M+ >90% people in the U.S. live with ADHD 3 of pediatric ADHD persist into adulthood 4 7M+ people impacted in the U.S. 5 Binge eating disorder FDA - approved treatment 1 Epilepsy ~3.4M >1/3 people in the U.S. live with epilepsy 15 of patients don’t respond to treatment 16 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 NEUROLOGY Schizophrenia ~3.7M people in the U.S. have schizophrenia and related psychotic disorders 7 Tourette syndrome ~0.6% of children in the U.S. may suffer from Tourette syndrome 17
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© Axsome Therapeutics, Inc. Solriamfetol ADHD Singular CNS portfolio with >$18B in annual peak sales potential 8 $0.5 - $ 1 B AXS - 14 Fibromyalgia AXS - 12 Narcolepsy $0.5 - $1B Solriamfetol MDD with EDS $0.5 - $1B AXS - 05 Smoking Cessation $1 - $1.5B $0.3 - $0.5B $8B+ $0.5 - $1B AXS - 17 Epilepsy AXS - 20 Schizophrenia AXS - 20 Tourette syndrome Psychiatry Neurology $1 - $3B Solriamfetol Binge eating disorder $0.5 - $1B Solriamfetol Shift work disorder $0.3 - $0.5B
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© Axsome Therapeutics, Inc. 9 Financial Highlights
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© Axsome Therapeutics, Inc. 2Q 2026 financial summary 10 2Q = three months ended June 30; * I ncludes royalty revenue associated with sales in out - licensed territories and milestone revenue Net Product Revenue $218.4 $150.0 46% AUVELITY $180.3 $119.6 51% SUNOSI * $35.8 $30.0 20% SYMBRAVO $2.3 $0.4 461% R&D Expense $46.2 $49.5 - 7% SG&A Expense $208.1 $130.3 60% 2Q 2026 2Q 2025 % Change $ millions
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© Axsome Therapeutics, Inc. AUVELITY ® quarterly net revenue performance 11 AUVELITY 2Q 2026 net produce revenue of $180.3M (51% YoY growth) +51% $5.2 $15.8 $27.6 $37.7 $49.0 $53.4 $65.0 $80.4 $92.6 $96.2 $119.6 $136.1 $155.1 $153.2 $180.3 $0 $25 $50 $75 $100 $125 $150 $175 $200 4Q '22 1Q '23 2Q '23 3Q '23 4Q '23 1Q '24 2Q '24 3Q '24 4Q '24 1Q '25 2Q '25 3Q '25 4Q '25 1Q '26 2Q '26 Millions
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© Axsome Therapeutics, Inc. SUNOSI 2Q 2026 net produce revenue of $35.8M (20% YoY growth) SUNOSI ® quarterly net revenue performance 12 $8.8 $16.8 $19.2 $13.1 $19.1 $20.1 $22.5 $21.6 $22.1 $24.4 $26.2 $25.2 $30.0 $32.8 $36.7 $33.9 $35.8 $0 $5 $10 $15 $20 $25 $30 $35 $40 2Q '22 3Q '22 4Q '22 1Q '23 2Q '23 3Q '23 4Q '23 1Q '24 2Q '24 3Q '24 4Q '24 1Q '25 2Q '25 3Q '25 4Q '25 1Q '26 2Q '26 Millions +20%
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© Axsome Therapeutics, Inc. Cash Balance: (as of June 30, 2026) $319.9M Debt (Face Value): (as of June 30, 2026) $190M Market Cap: (as of August 7, 2026) $11.0B Shares Outstanding: (as of June 30, 2026) 52.3M Options, RSUs, and Others Outstanding*: 8.1M Runway to reach cash flow positivity , based on the current operating plan Financial snapshot 13 *Includes 5.9M options, 2.0M RSUs, and 0.2M others as of June 30, 2026
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© Axsome Therapeutics, Inc. 14 Commercial Update
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© Axsome Therapeutics, Inc. Well - positioned for accelerating commercial growth 15 PEAK SALES POTENTIAL • Launched AUVELITY in Alzheimer’s disease agitation in June • Expanded sales force of ~630 representatives • Acceleration in new patient starts and new prescriber activation • Strong early NBRx growth trend among 65+ patients just weeks into launch • Consistent growth driven by durable demand • Strong underlying demand growth • Improvements in market access $8B+ $0.3B - $0.5B $0.5B - $1B HIGHLIGHTS / GROWTH DRIVERS ~$9.5B total commercial opportunity across marketed products
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© Axsome Therapeutics, Inc. First and only oral NMDA antagonist and sigma - 1 agonist for MDD and Alzheimer’s disease agitation 1,2 16 NMDA = N - methyl - D - aspartate; MDD = Major depressive disorder 1. AUVELITY [Prescribing Information]. Axsome Therapeutics, Inc., New York, NY; 2. Thomas D, Wessel C. BIO 2017; 3 . Iosifescu DV, et al. 2022 16 MAJOR DEPRESSIVE DISORDER Only oral antidepressant with rapid - acting efficacy reflected in FDA label 1 Rapid symptom improvement starting at week 1 and remission as early as week 2, sustained at week 6 1,3 Only approved treatment demonstrating substantial symptom improvement and statistically significantly longer time to relapse 1 Distinct safety/tolerability profile with no new boxed warning; most common adverse reactions were dizziness and dyspepsia 1 ALZHEIMER’S DISEASE AGITATION
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© Axsome Therapeutics, Inc. Continued momentum reflects underlying MDD growth and early impact from the sales force expansion 17 TRx = Total prescriptions ACCELERATING PRESCRIBER GROWTH +26% QoQ NBRx growth ~69,000 Unique writers since launch ~89% Total covered lives ~82% Commercial covered lives ~100% Medicare and Medicaid covered lives GROWING PATIENT ADOPTION STRONG MARKET ACCESS 0 40,000 80,000 120,000 160,000 200,000 240,000 280,000 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 2026-Q1 2026-Q2 Source: Symphony METYS Quarterly TRx Launch to Date +21% QoQ growth in activated new writers >350,000 Patients treated since launch ~266,000 TRx in 2Q 2026
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© Axsome Therapeutics, Inc. First and only dopamine and norepinephrine reuptake inhibitor for EDS associated with narcolepsy or OSA 1 18 EDS = Excessive daytime sleepiness; OSA = Obstructive sleep apnea; DNRI = Dopamine - norepinephrine reuptake inhibitor 1. SUNOSI [Prescribing Information]. Axsome Therapeutics, Inc., New York, NY; 2. Schweitzer PK, et al. 2019 First and only wakefulness promoting agent proven to improve wakefulness through 9 hours 1 Improvements in cognitive functioning vs. placebo demonstrated in clinical trials 90% of patients reported feeling better with SUNOSI 150 mg 2
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© Axsome Therapeutics, Inc. Durable demand continues to drive growth 19 nTRx = Normalized total prescriptions 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 65,000 3Q 2019 4Q 2019 1Q 2020 2Q 2020 3Q 2020 4Q 2020 1Q 2021 2Q 2021 3Q 2021 4Q 2021 1Q 2022 2Q 2022 3Q 2022 4Q 2022 1Q 2023 2Q 2023 3Q 2023 4Q 2023 1Q 2024 2Q 2024 3Q 2024 4Q 2024 1Q 2025 2Q 2025 3Q 2025 4Q 2025 1Q 2026 2Q 2026 Source: Symphony METYS. nTRx normalizes number of pills in each TRx for 30 - day period. Quarterly nTRx Launch to Date EXPANDING PRESCRIBER REACH ~61,000 TRx in 2Q 2026 ~109,000 Patients treated since launch >17,000 Unique writers since launch ~82% Total covered lives ~96% Commercial covered lives ~59% Medicare and Medicaid covered lives STEADY PATIENT GROWTH BROAD PAYER COVERAGE
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© Axsome Therapeutics, Inc. Novel, oral, rapidly - absorbed, multi - mechanistic approach for the acute treatment of migraine 1 20 1. SYMBRAVO [Prescribing Information]. Axsome Therapeutics, Inc., New York, NY Single, oral dose provided rapid migraine pain freedom and return to normal functioning within 2 hours 1 Harnesses Axsome’s MoSEIC rapid absorption technology to target multiple pathways underlying a migraine attack Superior efficacy demonstrated across a broad range of migraine severity (mild, moderate, severe) 1
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© Axsome Therapeutics, Inc. Prescription growth driven by increasing demand 21 TRx = Total prescriptions PRESCRIBER GROWTH ~23,500 TRx in 2Q 2026 ~19,500 Patients treated since launch ~4,700 Unique writers since launch ~57% Total covered lives ~56% Commercial covered lives ~57% Medicare and Medicaid covered lives PATIENT GROWTH ESTABLISHED MARKET ACCESS 0 200 400 600 800 1,000 1,200 1,400 1,600 1,800 2,000 Jun 2025 Jul 2025 Aug 2025 Sep 2025 Oct 2025 Nov 2025 Dec 2025 Jan 2026 Feb 2026 Mar 2026 Apr 2026 May 2026 Jun 2026 Source: Symphony METYS Weekly TRx Launch to Date
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© Axsome Therapeutics, Inc. 22 Development Pipeline
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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 AXS - 05 for smoking cessation 23 1. U.S. Department of Health and Human Services 2020; 2. Hughes JR, et al. 2004 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
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© Axsome Therapeutics, Inc. 24 1. Raony I, et al. 2022; 2. Halff EF, et al . 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 25 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) Initiated FOCUS - 2 and FOCUS - 3 Phase 3 trials in children and adolescents with ADHD A pproved in EDS associated with OSA and narcolepsy MDD with EDS BED SWD • Ongoing CLARITY Phase 3 trial in MDD with EDS symptoms • Ongoing ENGAGE Phase 3 trial in adults with binge eating disorder (N=450) • Topline data anticipated in 4Q 2026 • Ongoing SUSTAIN Phase 3 trial 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) 26 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 Associated with significant impairment in social, academic, and occupational functioning and development 3 Characterized by a persistent pattern of inattention and/or hyperactive - impulsive behaviors 3
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© Axsome Therapeutics, Inc. -20 -15 -10 -5 0 Baseline Week 1 Week 2 Week 3 Week 4 Week 6 Significant improvements in overall ADHD severity vs. placebo (CGI - S, p=0.017) c AISRS Total Score LSM Change ( ± SE) FOCUS Phase 3 Trial a 27 CMAI = Cohen - Mansfield Agitation Inventory; LSM = least - squares mean; SE = standard error; SR = sustained release a. p - values shown for solriamfetol 150 mg p - values are nominal; b. Clinical response defined as ≥30% reduction in AISRS; c. solr iamfetol 150 mg Axsome Therapeutics, Inc. Data on file. 17.7 - point reduction in the AISRS total score at Week 6 with solriamfetol vs. 14.3 - point reduction with placebo (p=0.039) b Significantly greater percentage of patients receiving solriamfetol achieved a clinical response b vs. placebo (p=0.024) c Well tolerated with a side effect profile consistent with the established safety profile of solriamfetol Statistically significant improvements in ADHD symptoms with solriamfetol treatment Primary endpoint: Change from baseline in AISRS total score at Week 6 Solriamfetol 300 mg Solriamfetol 150 mg Placebo p =0.036 p =0.041 p =0.039
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© Axsome Therapeutics, Inc. Solriamfetol in ADHD pediatric Phase 3 trial designs 28 ADHD - RS - 5 = ADHD Rating Scale Solriamfetol ( D ose 1) Solriamfetol ( D ose 2) Placebo 1:1:1 R Screening (5 weeks) Double - blind Phase ( 6 weeks) Follow - up (1 week) Baseline FOCUS - 3 Phase 3 Trial N=468 Key eligibility criteria • 12 to <18 years of age with diagnosis of ADHD (DSM - 5) Primary endpoint • Change from baseline in ADHD - RS - 5 total score Solriamfetol ( D ose 1) Solriamfetol ( D ose 2) Placebo 1:1:1 R Screening ( 5 weeks) Double - blind Phase ( 6 weeks) Follow - up (1 week) Baseline FOCUS - 2 Phase 3 Trial N=468 Key eligibility criteria • 6 to <12 years of age with diagnosis of ADHD (DSM - 5) Primary endpoint • Change from baseline in ADHD - RS - 5 total score
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© Axsome Therapeutics, Inc. MDD with excessive daytime sleepiness symptoms 29 1. Rush AJ, et al. 2006; 2. Major Depression. NIMH 2023; 3. Hasin DS, et al. 2018; 4. Hein M, et al. 2019 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
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© Axsome Therapeutics, Inc. Placebo Solriamfetol (150 mg) CLARITY Phase 3 trial design 30 Key eligibility criteria • 18 - 65 years of age with diagnosis of MDD (DSM - 5 criteria) • Excessive daytime sleepiness symptoms Open - label Period Double - blind Phase Baseline CLARITY Phase 3 Trial 1:1 R Primary endpoint • Time from randomization to relapse of depressive symptoms Solriamfetol (150 mg) 1:1 Randomization (Patients achieving treatment response)
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© Axsome Therapeutics, Inc. Binge eating disorder 31 1. Hudson JI, et al. 2007; 2. Swanson SA, et al. 2011; 3. Kessler RM, et al. 2016; 4. McElroy SL, et al. 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 32 TAAR1 = Trace amine - associated receptor 1 1. Giel KE, et al. 2022; 2. Bello NT, Hajnal A. 2010; 3. Pruccoli J, et al. 2021; 4. Malhotra A, et al. 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 33 1. Sateia MJ. 2014; 2. Alterman T, et al. 2013; 3. Wickwire EM. 2017; 4. Smith L, et al. 1994; 5. Akerstedt T, Wright KP. 2009; 6. Czeisler CA, et al. 2005 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
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© Axsome Therapeutics, Inc. Placebo Solriamfetol (150 mg) Evaluating solriamfetol as a potential treatment for shift work disorder 34 MWT = Maintenance of Wakefulness Test Key eligibility criteria • 18 - 65 years of age with diagnosis of SWD (ICSD - 2 or ICSD - 3) Screening (1 - 4 weeks) Double - blind Phase (up to 12 weeks) Baseline SUSTAIN Phase 3 Trial 1:1 R Primary endpoint • Change from baseline in MWT N=450 Follow - up (1 week)
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© Axsome Therapeutics, Inc. 35 1. Szabo ST, et al. 2019; 2. Krahn LE, Zee PC, Thorpy MJ. 2022; 3. Scammell TE. 2015; 4. Stahl SM, Grady MM. 2003; 5. Burgess CR , Peever JH. 2013; 6. Wu MF, et al. 1999; 7. Bruinstroop E, et al. 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 36 1. Narcolepsy Network 2024; 2. Sateia MJ. 2014; 3. Narcolepsy. NINDS 2024; 4. España RA, Scammell TE. 2011; 5. Swick TJ. 2015 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 Up to 70% of patients suffer from cataplexy, or the sudden reduction or loss of muscle tone while awake 5
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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 37 NDA for AXS - 12 for cataplexy in narcolepsy accepted by the FDA with PDUFA target action date of May 1, 2027
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© Axsome Therapeutics, Inc. 38 Adapted from Siracusa, R. et al. 2021 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 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)
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© Axsome Therapeutics, Inc. Fibromyalgia 39 1. Vincent, et al. 2013; 3. Choy E, et al. 2010; 3. Arnold LM, et al. 2008; 4. Bair MJ, Krebs EE. 2020; 5. Clauw DJ. 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. Rapid and robust improvements in fibromyalgia symptoms with AXS - 14 treatment 40 -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 * ** *** *** *** ** *** *** ** **** -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 a 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 FORWARD Phase 3 trial ongoing a. p - values are defined as: *p<0.05, **p<0.01, ***<0.001, ****p<0.0001
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© Axsome Therapeutics, Inc. Placebo AXS - 14 (8 mg) FORWARD Phase 3 trial design 41 Key eligibility criteria • ≥18 years of age with diagnosis of fibromyalgia 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) 1:1 Randomization (Patients achieving treatment response)
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© Axsome Therapeutics, Inc. 42 1. Epilepsy. CDC 2025; 2. Chen Z, et al. 2018 Despite currently available treatment options, <1/3 of patients do not response to treatment 2 AXS - 17 was safe and well tolerated in clinical studies in >700 patients to date and demonstrated compelling anti - convulsant activity in preclinical seizure models Phase 2 trial - enabling activities underway Epilepsy is a chronic and debilitating neurological disorder affecting ~3.4M people in the U.S. 1 Novel oral GABA A receptor α 2,3 subtype - selective positive allosteric modulator (PAM) for epilepsy AXS - 17
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© Axsome Therapeutics, Inc. 43 1. Ringeisen H, et al. 2023; 2. Tourette Syndrome. CDC 2025 Potential first - in - class selective PDE10A inhibitor for neuropsychiatric conditions AXS - 20 Schizophrenia • Demonstrated a favorable safety and tolerability profile in clinical studies in >360 individuals to date • Completed a proof - of - concept Phase 2 trial in patients with schizophrenia • Phase 3 trial - enabling activities for AXS - 20 in schizophrenia underway Tourette syndrome • Evaluating AXS - 20 as a potential treatment for Tourette syndrome ~3.7M people in the U.S. have schizophrenia and related psychotic disorders 1 1 out of every 162 children in the U.S. may suffer from Tourette syndrome 2
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© Axsome Therapeutics, Inc. Strong intellectual property portfolio 44 AUVELITY AXS - 12 SUNOSI AXS - 14 SYMBRAVO Product Highlights • Protected by a robust patent estate extending to at least 2043, multiple pending • Proprietary 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 • Protected by a robust patent estate extending to at least 2045, multiple pending • Proprietary MoSEIC TM formulation, drug product formulation, and methods of treatment • Orphan Drug Designation • Claims extending to at least 2039; 10 issued U.S. patents and 6 issued O.U.S. patents, multiple pending • Proprietary drug substance, drug product formulation, and methods of treatment • Claims extending to at least 2045; 1 issued U.S. patent, multiple pending • Proprietary drug substance, drug product formulation, and methods of treatment
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© Axsome Therapeutics, Inc. Leadership team 45 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. 46 Thank you