Axsome Therapeutics, Inc. · NASDAQ:AXSM
Axsome Therapeutics — Investor Presentation (January 2024)
41 slides from Axsome Therapeutics's investor presentation, Jan 8, 2024
Slide 1: Corporate Presentation January 2024
Corporate Presentation January 2024
Slide 2: Forward-looking statements
Certain information contained in this presentation may include “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “pl
Slide 3: Rapidly Growing, CNS-Focused Biopharma
Rapidly Growing, CNS-Focused Biopharma 2 Marketed Products 10 New Target Indications 5 Late-stage Product Candidates Potential Patients Targeted 150 Million >
Slide 4: Late-Stage CNS Portfolio
Late-Stage CNS Portfolio Phase 1 Phase 2 Phase 3 NDA Marketed Abbreviations: CNS = Central Nervous System; MOA = Mechanism of Action; NMDA = N-Methyl-D-aspartate; COX-2 = Cyclooxygenase-2; 5-HT = 5-Hydroxytryptamine; NE = Norepinephrine; CYP2D6 = Cytochrome P450 Family 2 Subfamily D Member 6; MoSEIC
Slide 5: CNS Portfolio with Potential to Impact >150M U.S. Patients
CNS Portfolio with Potential to Impact the Lives of >150M U.S. Patients 17 M 34 M 185 K 4 M 37 M 5 M 22 M 21 M Abbreviations: MDD = Major Depressive Disorder; OSA = Obstructive Sleep Apnea; AD = Alzheimer’s Disease; ADHD = Attention Deficit Hyperactivity Disorder; BED = Binge Eating Disorder; SWD =
Slide 6: Potentially Marketed Indications by 2025
Potentially Marketed Indications by 2025 AXS-14 Fibromyalgia AXS-07 Migraine AXS-05 AD agitation AXS-12 Narcolepsy Major depressive disorder Excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea
Slide 7: Marketed Products
axsome Marketed Products © Axsome Therapeutics, Inc.
Slide 8: Auvelity: Treating adult patients with major depressive disorder
Treating adult patients living with major depressive disorder Abbreviations: TRx = total prescriptions; NMDA = N-Methyl-D-aspartate; MDD = major depressive disorder 1. Auvelity [Prescribing Information]. Axsome Therapeutics, Inc., New York, NY 2. FDA Depression Medicines. https://www.fda.gov/media/1
Slide 9: Sunosi: Improving wakefulness in EDS with narcolepsy or OSA
Improving wakefulness in adult patients with EDS associated with narcolepsy or OSA Abbreviations: nTRx = normalized total prescriptions; EDS = excessive daytime sleepiness; OSA = obstructive sleep apnea; DNRI = dopamine-norepinephrine reuptake inhibitor 1. SUNOSI [Prescribing Information]. Axsome Th
Slide 10: Development Pipeline
axsome Develooment Pipeline © Axsome Therapeutics, Inc.
Slide 11: AXS-05 (dextromethorphan-bupropion)
orsome AXS-05 (dextromethorphan-bupropion) anew approach to treating multiple CNS conditions
Slide 12: Alzheimer's Disease Agitation: High Unmet Medical Need, Novel Approach
Alzheimer’s Disease Agitation: High Unmet Medical Need, Novel Approach 1. Alzheimer’s Association. 2023 Alzheimer’s Disease Facts and Figures. Alzheimers Dement 2023;19(4). 2. Tractenberg R, et al. J Neuropsychiatry Clin Neurosci. 2002;14:11-18. 3. Alzheimers Dement. 2021 Mar;17(3):327-406. 4. Porst
Slide 13: Alzheimer's Disease Agitation: Clinical Results and Program Status
Alzheimer’s Disease Agitation: Clinical Results and Program Status Primary endpoints met in two controlled trials: ADVANCE-1 Phase 2/3, parallel group trial ACCORD Phase 3, randomized withdrawal trial ADVANCE-2 Phase 3 trial ongoing, topline results expected 1H 2024 FDA Breakthrough Therapy Designat
Slide 14: Alzheimer's Disease Agitation: ADVANCE-2 Phase 3 Trial
Alzheimer’s Disease Agitation: ADVANCE-2 Phase 3 Trial Primary Endpoint: Efficacy of AXS-05 compared to placebo on the change from baseline in CMAI total score Key Inclusion Criteria: Male or female 65-90 years old Diagnosis of probable AD and of clinically significant agitation resulting from proba
Slide 15: Smoking Cessation
Smoking is single largest cause of preventable death in the U.S.1 70% of smokers want to quit2 Only 3-5% who attempt to quit without assistance are successful for 6-12 months2 Smoking Cessation Abbreviations: NMDA = N-methyl D-aspartate U.S. Department of Health and Human Services. The Health Conseq
Slide 16: AXS-07 (MoSEIC meloxicam-rizatriptan)
AXS-07 (MoSEIC™ meloxicam-rizatriotan) a multi-mechanistic approach to treating migraine © Axsome Therapeutics, Inc ~ = aS) axsome
Slide 17: Migraine: Significant Need for More Efficacious Treatments
Migraine: Significant Need for More Efficacious Treatments 1. Menken et al. Arch Neurol. 2000;57:418-420. 2. Shapiro and Goadsby. Cephalalgia. 2007;27:991-4. 3. Gooch CL, Pracht E, Borenstein AR. The burden of neurological disease in the United States: A summary report and call to action. Ann Neurol
Slide 18: Migraine: Clinical Results and Program Status
Migraine: Clinical Results and Program Status P<0.001 P<0.001 P<0.001 P<0.001 P<0.001 Rapid and sustained efficacy as compared to placebo and active comparator rizatriptan, in three positive Phase 3 trials: MOMENTUM trial, in patients with history of inadequate response, vs. placebo and rizatriptan
Slide 19: AXS-12 (reboxetine)
axsome AXS-12 (reboxetine) a potentially new treatment option for narcolepsy 19
Slide 20: Narcolepsy: Debilitating Disorder with Limited Treatment Options
Narcolepsy is a debilitating disorder characterized by excessive daytime sleepiness and cataplexy, with limited treatment options Loss of excitatory hypocretin/orexin neurons in the brain lead to dysregulation of norepinephrine resulting1: Loss of muscle tone while awake (cataplexy) Decreased wakefu
Slide 21: Narcolepsy: Clinical Results and Program Status
Positive Phase 2 results with AXS-12 Significant reduction in cataplexy attacks Significant improvement in excessive daytime sleepiness Significant improvement in cognitive function SYMPHONY Phase 3 trial ongoing, topline results expected 1Q 2024 Narcolepsy: Clinical Results and Program Status CONCE
Slide 22: Narcolepsy: SYMPHONY Phase 3 Trial
Primary Endpoint: Change in the frequency of cataplexy attacks Key Inclusion Criteria: Male or female 15-75 years old Primary diagnosis of narcolepsy with cataplexy Topline Data: 1Q 2024 A Phase 3 trial to assess efficacy and safety of AXS-12 as compared to placebo in the treatment of cataplexy in n
Slide 23: AXS-14 (esreboxetine)
AXS-14 (esreboxetine) a potentially new treatment option for fibromyalgia
Slide 24: Fibromyalgia
Debilitating, chronic, CNS disorder characterized by widespread pain, fatigue, disturbed sleep, depression, and cognitive impairment; ~90% affected are women Limited treatment options with only 3 approved agents, variable efficacy, and do not address all symptoms AXS-14 (esreboxetine) increases desc
Slide 25: Fibromyalgia: Clinical Data and Program Status
Positive Phase 3 and Phase 2 efficacy results with AXS-14 in fibromyalgia: Significant reduction in pain and improvement in function NDA submission planned for first quarter of 2024 Fibromyalgia: Clinical Data and Program Status AXS-14 Phase 3 Efficacy Data
Slide 26: Solriamfetol: a potentially differentiated option for CNS disorders
Solriamfetol a potentially differentiated option for the treatment of CNS disorders
Slide 27: Attention Deficit Hyperactivity Disorder
ADHD is a serious disorder characterized by inattention, hyperactivity or impulsivity Associated with significant impairment in social, academic, and occupational functioning or development Solriamfetol targets neurotransmitter pathways in the brain implicated in ADHD3 Attention Deficit Hyperactivit
Slide 28: ADHD: FOCUS Phase 3 Trial
Attention Deficit Hyperactivity Disorder: FOCUS Phase 3 Trial Primary Endpoint: Change in the Adult ADHD Investigator Symptom Report Scale (AISRS) Key Inclusion Criteria: Male or female 18-55 Primary diagnosis of ADHD (inattentive, hyperactive, or combined subtype) using DSM-5 criteria Target Enroll
Slide 29: Major Depressive Disorder
Major Depressive Disorder Major depression is one of the most common mental disorders in the United States1 Solriamfetol is a dopamine and norepinephrine reuptake inhibitor, a TAAR-1 and 5HT1A agonist The combination of monoamine reuptake inhibition and TAAR1/5H1A agonism showed synergistic results
Slide 30: MDD: Planned Phase 3 Trial
Major Depressive Disorder (MDD): Planned Phase 3 Trial Primary Endpoint: Change in the Montgomery-Åsberg Depression Rating Scale (MADRS) Key Inclusion Criteria: Male or female 18-65 years old Confirmed diagnosis of moderate to severe MDD Target Enrollment: 300 AXS-05 twice daily Placebo Double-blind
Slide 31: Binge Eating Disorder
Binge eating disorder (BED) is the most common eating disorder and is thought to involve issues with food reward processing, impulse control, and appetite regulation1,2 Associated with a 2- to 3-fold increased risk of psychiatric and medical comorbidities3 Solriamfetol’s dopamine, norepinephrine, an
Slide 32: Binge Eating Disorder: Planned Phase 3 Trial
Binge Eating Disorder: Planned Phase 3 Trial Primary Endpoint: Change from baseline in binge eating Key Inclusion Criteria: Male or female 18-55 years old Meets DSM-5 criteria for BED Target Enrollment: 450 AXS-05 twice daily Solriamfetol (150 mg) Placebo Double-blind Phase (12 Weeks) Screening (up
Slide 33: Shift Work Disorder
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. workday1 Shift work has long been associated with multiple serious health complaints and a 23% higher relative risk of sustaining
Slide 34: Shift Work Disorder: Planned Phase 3 Trial
Shift Work Disorder: Planned Phase 3 Trial Primary Endpoint: Change from baseline in patient’s disease status as measured by the CGI-C Key Inclusion Criteria: Male or female 18-65 years old Diagnosis of SWD based on the ICSD-2 or -3 criteria AXS-05 twice daily Solriamfetol (150 mg) Placebo Double-bl
Slide 35: CNS portfolio with potential U.S. peak sales of up to $13.5B
CNS portfolio with potential to generate total U.S. peak sales of up to $13.5B EDS associated with OSA and narcolepsy $1- $3B $0.5 - $1B $0.5 - $1B $0.5 - $1B $1.5 - $3B 2022 2025 est. 2025 est. 2025 est. 2025 est. 2022 Rapid and substantial efficacy, as early as week 11 New differentiated oral appr
Slide 36: Strong Intellectual Property and Barriers to Entry
Strong Intellectual Property and Barriers to Entry >127 Issued U.S. Patents and >76 Issued O-U.S. Patents Claims extending to at least 2034-43; Multiple pending Proprietary drug product formulation >96 Issued U.S. Patents and >125 Issued O-U.S. Patents Claims extending to at least 2038; Multiple pen
Slide 37: Financial Snapshot
Financial Snapshot Cash Balance: (as of Sep. 30, 2023) Debt (Face Value): (as of Sep. 30, 2023) Market Cap: (as of January 5, 2024) Shares Outstanding: (as of September 30, 2023) Options, RSUs, and Warrants Outstanding1: 1 Consists of 8.15 M options, 0.8 M RSUs, and 0.080 M warrants $ 416.6 M $ 180
Slide 38: Leadership Team
Leadership Team Roger Jeffs, PhD CEO Liquidia Corporation Former President, Co-CEO, Director United Therapeutics Corp. Prior positions at Amgen and Burroughs Wellcome Mark Saad Former CFO Bird Rock Bio, Inc. Former COO of the Global Healthcare Group at UBS Mark Coleman, MD Director of Clinical Servi
Slide 39: Anticipated Upcoming Clinical and Regulatory Milestones
Anticipated Upcoming Clinical and Regulatory Milestones Regulatory and Commercial Clinical Trial Topline Results Clinical Trial Initiations AXS-05 Phase 2/3 trial in smoking cessation – 2024 AXS-12 SYMPHONY Phase 3 trial in narcolepsy – 1Q 2024 AXS-05 ADVANCE-2 Phase 3 trial in Alzheimer’s disease a
Slide 40: Rapidly Growing, CNS-Focused Biopharma
Rapidly Growing, CNS-Focused Biopharma 2 Marketed Products 10 New Target Indications 5 Late-stage Product Candidates Potential Patients Targeted 150 Million >
Slide 41: Thank you
for more information, please contact: mark jacobson chief operating officer 212-332-3243 mjacobson@axsome.com www.axsome.com thank you
Axsome Therapeutics — Investor Presentation (January 2024) on 6ix