Axsome Therapeutics, Inc. · NASDAQ:AXSM

Axsome Therapeutics — Investor Presentation (March 2020)

28 slides from Axsome Therapeutics's investor presentation, Mar 12, 2020

  1. Slide 1: Axsome Therapeutics, March 2020

    NASDAQ: AXSM March 2020 © Axsome Therapeutics, Inc.

  2. 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

  3. Slide 3: Addressing serious CNS disorders with novel therapeutics

    3 Axsome is addressing serious CNS disorders, where current treatment options are limited or inadequate, by creating novel therapeutics to improve the lives of patients. © Axsome Therapeutics, Inc.

  4. Slide 4: Our Technologies Overview

    4 Our Technologies Overview © Axsome Therapeutics, Inc. Enabling new and innovative medicines to treat CNS conditions Chemical Synthesis & Analysis Metabolic Inhibition Chiral & Formulation Chemistry MoSEIC Delivery ™

  5. Slide 5: Our CNS Candidates and Pipeline Overview

    • Four differentiated clinical-stage CNS assets targeting significant and growing markets • Patent protection to 2034-2036, worldwide rights for most product candidates Product Candidate Preclinical Phase 1 Phase 2 Phase 3 AXS-05 (DM + BUP) AXS-07 (MoSEIC™ Mx + Riz) AXS-12 (Reboxetine) AXS-14 (Esreb

  6. Slide 6: AXS-05 (dextromethorphan/bupropion)

    Novel therapy for CNS disorders: • Major Depressive Disorder (MDD) • Treatment Resistant Depression (TRD) • Agitation in Alzheimer’s Disease (AD) • Smoking Cessation 6 © Axsome Therapeutics, Inc. (dextromethorphan/bupropion) modulated delivery tablet AXS-05

  7. Slide 7: AXS-05: Novel, Oral, NMDA Receptor Antagonist with Multimodal Activity

    7 AXS-05: Novel, Oral, NMDA Receptor Antagonist with Multimodal Activity AXS-05 NMDA Receptor Antagonism Sigma-1 Receptor Agonism Monoamine Reuptake Inhibition Modulation of DM Delivery 0 80 160 0 12 24 36 DM Plasma Concentration Time AXS-05 DM NMDA receptor Na+/Ca2+ Mg2+ DM component of AXS-05 K+ C

  8. Slide 8: AXS-05: Depression Overview

    8 AXS-05: Depression Overview © Axsome Therapeutics, Inc. AXS-05 1. National Survey on Drug Use and Health (NSDUH). (2017). 2. Rush AJ, et al. Am J Psychiatry 2006;163:1905-1917. 17.3M patients in the U.S.1 Product Candidate Preclinical Phase 1 Phase 2 Phase 3 AXS-05 (DM + BUP) Treatment Resistant D

  9. Slide 9: AXS-05: Results of GEMINI Trial in MDD

    -20 -18 -16 -14 -12 -10 -8 -6 -4 -2 0 Baseline 1 2 3 4 5 6 MADRS Total Score Change from Baseline Week AXS-05 (45 mg dextromethorphan / 105 mg bupropion BID) Placebo 9 AXS-05: Depression Results of GEMINI Trial in MDD © Axsome Therapeutics, Inc. AXS-05 P<0.001 P=0.002 P=0.007 Notes: P-values calcula

  10. Slide 10: AXS-05: TRD Phase 3 Design

    10 AXS-05: TRD Phase 3 Design © Axsome Therapeutics, Inc. AXS-05 BUP = Bupropion; DM = Dextromethorphan. Bupropion AXS-05 (BUP + DM) Period 1, Open-label (6 weeks) Period 2, Double-blind (6 weeks) 1:1 randomization of inadequate responders Bupropion N=250 A Phase 3 trial to assess the efficacy and s

  11. Slide 11: AXS-05: Agitation in AD Overview

    11 AXS-05: Agitation in AD Overview © Axsome Therapeutics, Inc. AXS-05 • Agitation seen in approximately 70% of AD patients.2 – Emotional distress, aggressive behaviors, disruptive irritability, disinhibition, and increased caregiver burden.4 • Associated with3,4: – Accelerated cognitive decline – E

  12. Slide 12: AXS-05: Agitation in AD Phase 2/3 Design

    12 AXS-05: Agitation in AD Phase 2/3 Design © Axsome Therapeutics, Inc. AXS-05 A Phase 2/3 trial to assess the efficacy and safety of AXS-05 in the treatment of Agitation in AD. • Primary Endpoint: Cohen-Mansfield Agitation Inventory (CMAI). • Key Inclusion Criteria: – Diagnosis of probable Alzheime

  13. Slide 13: AXS-05: Smoking Cessation Overview

    13 AXS-05: Smoking Cessation Overview © Axsome Therapeutics, Inc. AXS-05 • Smoking is single largest cause of preventable death in the U.S.1 • 70% of smokers want to quit and only 3-5% who attempt to quit without assistance are successful for 6-12 months.2 • Positive Phase 2 trial results (Duke Univ

  14. Slide 14: AXS-07: Migraine

    Novel therapy for: •Migraine 14 (MoSEIC™ meloxicam/rizatriptan) AXS-07 © Axsome Therapeutics, Inc.

  15. Slide 15: AXS-07: MoSEIC Meloxicam + Rizatriptan for Migraine

    15 AXS-07: MoSEIC™ Meloxicam + Rizatriptan for Migraine © Axsome Therapeutics, Inc. AXS-07 • AXS-07 is a novel, oral, rapidly absorbed, multi-mechanistic investigational medicine for the acute treatment of migraine • Positive results from MOMENTUM Phase 3 trial in patients with history of inadequate

  16. Slide 16: AXS-07: Results of MOMENTUM Trial in Acute Migraine

    16 AXS-07: Results of MOMENTUM Trial in Acute Migraine © Axsome Therapeutics, Inc. AXS-07 5.3% 6.8% 11.2% 16.1% 0% 4% 8% 12% 16% 20% Percentage of Subjects Placebo MoSEIC Meloxicam Rizatriptan AXS-07 P<0.001 P=0.038 P=0.001 Sustained Pain Freedom 2-24hrs Key Secondary 33.5% 42.0% 43.9% 53.3% 0% 10%

  17. Slide 17: AXS-07: Migraine INTERCEPT Phase 3 Trial Design

    17 AXS-07: Migraine INTERCEPT Phase 3 Trial Design © Axsome Therapeutics, Inc. AXS-07 INTERCEPT: INiTiating EaRly Control of MigrainEPain & Associated SympToms Phase 3 trial of AXS-07 for the acute treatment of migraine 1:1 randomization Single dose, immediately following migraine pain onset Screeni

  18. Slide 18: AXS-12: Narcolepsy

    Novel therapy for: •Narcolepsy 18 (reboxetine) AXS-12 © Axsome Therapeutics, Inc.

  19. Slide 19: AXS-12 (reboxetine): Narcolepsy Overview

    Product Candidate Preclinical Phase 1 Phase 2 Phase 3 AXS-12 (Reboxetine) Narcolepsy; U.S. Orphan Designation • Debilitating sleep disorder characterized by excessive daytime sleepiness (EDS) and cataplexy • Limited treatment options – Only one approved agent for cataplexy – Most currently approved

  20. Slide 20: AXS-12: Results of CONCERT Trial in Narcolepsy

    -20.0 -16.0 -12.0 -8.0 -4.0 0.0 4.0 0 1 2 Mean Change in Weekly Cataplexy Attacks AXS-12 Placebo 20 © Axsome Therapeutics, Inc. AXS-12 P<0.001 P=0.002 Primary Endpoint AXS-12 Placebo P-value Overall Change in Average Weekly Cataplexy Attacks -13.0 -0.3 <0.001 Week 2 Change in Average Weekly Cataplex

  21. Slide 21: AXS-14: Fibromyalgia

    Novel therapy for: •Fibromyalgia 21 esreboxetine AXS-14 © Axsome Therapeutics, Inc.

  22. Slide 22: AXS-14 (esreboxetine): Fibromyalgia Overview

    22 AXS-14 (esreboxetine): Fibromyalgia Overview © Axsome Therapeutics, Inc. AXS-14 • Debilitating, chronic, CNS disorder characterized by widespread pain, fatigue, disturbed sleep, depression, and cognitive impairment; ~90% affected are women • Limited treatment options—only 3 approved agents: – Cur

  23. Slide 23: AXS-14: Phase 3 Efficacy Data in Fibromyalgia

    23 AXS-14 (esreboxetine): Phase 3 Efficacy Data in Fibromyalgia © Axsome Therapeutics, Inc. AXS-14 -1.11 -1.76 -1.85 -1.55 -2 -1.8 -1.6 -1.4 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 Placebo Esreboxetine 4 mg/day Esreboxetine 8 mg/day Esreboxetine 10 mg/day Mean Change in Pain Score Scale: 0 = no pain; 10 = wor

  24. Slide 24: Barriers to Entry

    MoSEIC™ Meloxicam (AXS-07, AXS-06) AXS-05 / AXS-09 24 Barriers to Entry © Axsome Therapeutics, Inc. Corporate 34 Issued U.S. Patents 8 Issued O-U.S. Patents Claims extending to 2034 >45 pending Proprietary Manufacturing Drug Product Formulation 24 issued U.S. Patents 3 issued O-U.S. Patent Claims ex

  25. Slide 25: Our Team

    25 Our Team © Axsome Therapeutics, Inc. Corporate Management Herriot Tabuteau, MD Founder & CEO Nick Pizzie, CPA, MBA Chief Financial Officer Dave Marek Chief Commercial Officer Mark Jacobson, MA Chief Operating Officer Cedric O’Gorman, MD, MBA SVP, Clinical Development & Medical Affairs Board of Di

  26. Slide 26: Key Financial Information

    26 Key Financial Information © Axsome Therapeutics, Inc. Corporate • Financial guidance: Cash anticipated to fund operating requirements for at least two years. As December 31, 2019 Cash: $220.0 Million Debt (Face Value): $20.0 Million Common Shares Outstanding: 36.9 Million Options and Warrants Out

  27. Slide 27: Our Clinical and Regulatory Milestones

    27 Our Clinical and Regulatory Milestones Corporate Product Candidate Indication 2019 2020 AXS-05 (DM + BUP) TRD ● STRIDE-1 Phase 3 topline results (1Q) AD Agitation ● ADVANCE-1 Phase 2/3 topline results (3Q) MDD ✓ ASCEND Phase 2 topline results ✓ FDA Breakthrough Therapy Designation ✓ GEMINI Phase

  28. Slide 28: For more information, please contact

    For more information, please contact Mark Jacobson SVP, Operations 212-332-3243 mjacobson@Axsome.com axsome.com

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