Axsome Therapeutics, Inc. · NASDAQ:AXSM

Axsome Therapeutics — Technical Presentation (April 2020)

27 slides from Axsome Therapeutics's technical presentation, Apr 6, 2020

  1. Slide 1: INTERCEPT Phase 3 Trial of AXS-07 Topline Results Conference Call in Migraine

    NASDAQ: AXSM INTERCEPT Phase 3 Trial of AXS-07 Topline Results Conference Call in Migraine April 6, 2020 © Axsome Therapeutics, Inc.

  2. Slide 2: Overview and Presenters

    Overview AXS-07 in Migraine Acute Treatment INTERCEPT Phase 3 Trial Topline Results Clinical Development & Medical Affairs Dave Marek, Chief Commercial Officer © Axsome Therapeutics, Inc. 2 Introduction Mark Jacobson, Chief Operating Officer Overview and Summary Herriot Tabuteau, MD, Chief Executive

  3. Slide 3: Forward-Looking Statements & Safe Harbor

    FLS Forward-Looking Statements & Safe Harbor 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

  4. Slide 4: Overview and Summary

    Overview and Summary Herriot Tabuteau, MD CHIEF EXECUTIVE OFFICER AXSOME THERAPEUTICS, INC. © Axsome Therapeutics, Inc. 4

  5. Slide 5: AXS-07 INTERCEPT Phase 3 Trial: Summary of Topline Results

    INTERCEPT AXS-07 INTERCEPT Phase 3 Trial: Summary of Topline Results • AXS-07 is a novel, oral, multi-mechanistic investigational medicine for the acute treatment of migraine • The INTERCEPT trial randomized patients to treat a single migraine attack with a single dose of AXS-07 or placebo, at the e

  6. Slide 6: Migraine: Disabling Disease in Need of New Treatments

    INTERCEPT Migraine: Disabling Disease in Need of New Treatments • The World Health Organization classifies severe migraine attacks as among the most disabling illnesses, comparable to dementia, quadriplegia and active psychosis1,2 • Debilitating pain, and the often-constant fear of the next migraine

  7. Slide 7: AXS-07 Multi-Mechanistic Treatment for Migraine

    AXS-07 AXS-07 (MoSEIC™ Meloxicam/Rizatriptan) Multi-Mechanistic Treatment for Migraine  Inhibition of CGRP release  Reversal of CGRP-mediated vasodilation CGRP Mediated Rizatriptan  Cyclooxygenase inhibition  PGE2 synthesis inhibition MoSEIC™ meloxicam Neuroinflammation  Decrease passage of pai

  8. Slide 8: Phase 3 Trial Design & Results

    INTERCEPT Phase 3 Trial Design & Results Cedric O’Gorman MD, MBA SENIOR VICE PRESIDENT, CLINICAL DEVELOPMENT AND MEDICAL AFFAIRS AXSOME THERAPEUTICS, INC. © Axsome Therapeutics, Inc. 8

  9. Slide 9: INTERCEPT Phase 3 Trial: Design Summary

    INTERCEPT INTERCEPT Phase 3 Trial: Design Summary INTERCEPT: INiTiating EaRly Control of MigrainE Pain & Associated SympToms Phase 3 trial of AXS-07 for the acute treatment of migraine Single dose Taken at earliest onset of migraine pain Screening n=152 1:1 randomization n=150 Co-Primary Endpoints:

  10. Slide 10: INTERCEPT Phase 3 Trial: Key Entry Criteria

    INTERCEPT INTERCEPT Phase 3 Trial: Key Entry Criteria Inclusion Criteria • Male or female, 18 to 65 years of age, inclusive • Established diagnosis (at least 1 year) of migraine with or without aura as defined by the ICHD-3 criteria • An average 2 to 8 migraines per month Exclusion Criteria • Cluste

  11. Slide 11: INTERCEPT Baseline Demographics

    INTERCEPT INTERCEPT Baseline Demographics Characteristics: AXS-07 (20 mg MoSEIC Mlx / 10 mg Riz) Placebo n=140 n=143 Age, years 41.7 (11.58) 41.4 (11.11) Female gender, n (%) 119 (85.0%) 122 (85.3%) Race, n (%) White Black or African American Asian Other or Multiple 118 (84.3%) 18 (12.9%) 1 (0.7%) 3

  12. Slide 12: Co-Primary Endpoints: Pain Freedom and MBS Freedom at Hour 2

    INTERCEPT Co-Primary Endpoints: Pain Freedom and MBS Freedom at 2 Hours P=0.002 P=0.003 50% 35% 30% 40% 25% 30% 20% S-07 acebo 15% 20% 10% 10% 5% 0% 0% Resolution of MBS at Hour 2 Pain Freedom at Hour 2 Difference P-Value Co-Primary Endpoints AXS-07 - Placebo 16.3% 0.002 Pain Freedom 2 Hours after D

  13. Slide 13: Rapid and Durable Freedom from Migraine Pain: Pain Freedom over Time

    INTERCEPT Rapid and Durable Freedom from Time Migraine Pain: Migraine Pain Freedom over 80% .001 70% 60% 50% 40% 30% 20% 10% 0% 0 2 4 6 8 101214 Hour 16 18 20 22 24 • • Numerical separation from placebo as early as 30 minutes after dosing 64% and 69% of AXS-07 patients pain free at 12 and 24 hours,

  14. Slide 14: Rapid and Durable Freedom from Migraine Pain: Sustained Pain Freedom

    INTERCEPT Rapid and Durable Freedom from Migraine Pain: Sustained Pain Freedom 24-Hour Sustained Pain Freedom 48-Hour Sustained Pain Freedom P=0.030 25% 25% 20% 20% 15% 15% AXS-07 Placebo 10% 10% 5% 5% 0% 0% Sustained Pain Freedom (Hour 2-48) Sustained Pain Freedom (Hour 2-24) © Axsome Therapeutics,

  15. Slide 15: Rapid Freedom from Most Bothersome Symptom

    INTERCEPT Rapid Freedom from Most Bothersome Symptom: Most Bothersome Symptom Freedom over Time P<0.001 60% 50% 40% AXS-07 Placebo 30% 20% 10% 0% 30 minutes Hour 1 Hour 2 Hour 4 • • Numerical separation from placebo as early as 30 minutes after dosing Most bothersome symptoms: nausea, photophobia, o

  16. Slide 16: Prevention of Worsening Migraine Pain: Freedom from Pain Progression

    INTERCEPT Prevention of Worsening Migraine Pain: Freedom from Pain Progression 2-24 Hours P<0.001 80% 70% 60% 50% S-07 acebo 40% 30% 20% 10% 0% 2-24 Hour Freedom from Pain Progression • A single dose of AXS-07 significantly prevented migraine pain progression beyond mild © Axsome Therapeutics, Inc.

  17. Slide 17: Prevention of Worsening Migraine Pain: Rescue Medication Use

    INTERCEPT Prevention of Worsening Migraine Pain: Rescue Medication Use Over Time 50% P<0.001 40% 36% 30% 20% 10% 0% Hour 2 Hour 4 Hour 12 Hour 16 Hour 24 AXS-07 Placebo © Axsome Therapeutics, Inc. 17 Percent of Patients Using Rescue Medications P<0.001 P<0.00142% 39% 15% P<0.001 24% 14% 9% 13% 4%5%

  18. Slide 18: Return to Normal Functioning: Functional Disability at Hour 24

    INTERCEPT Return to Normal Functioning: Functional Disability at Hour 24 P<0.001 80% 70% 60% 50% AXS-07 Placebo 40% 30% 20% 10% 0% No Functional Disability at Hour 24 © Axsome Therapeutics, Inc. 18 Percentage of Patients 73.5% 47.4%

  19. Slide 19: Global Improvement in Migraine Symptoms: Patient Global Impression of Change

    INTERCEPT Global Improvement in Migraine Symptoms: Patient Global Impression of Change at Hour 2 P<0.001 60% 50% 40% AXS-07 Placebo 30% 20% 10% 0% "Very Much Improved" or "Much Improved" © Axsome Therapeutics, Inc. 19 Percentage of Patients 52.4% 27.7%

  20. Slide 20: Safety of AXS-07: Adverse Events Occurring in ≥2% of Subjects

    INTERCEPT Safety of AXS-07: Adverse Events Occurring in ≥2% of Subjects AXS-07 (N = 140) Placebo (N = 143) Any Treatment-Emergent AE Somnolence Dizziness Paraesthesia 25 (17.9%) 6 (4.3%) 4 (2.9%) 3 (2.1%) 11 (7.7%) 3 (2.1%) 2 (1.4%) 0 Data presented as number of subjects (% of subjects) • There were

  21. Slide 21: INTERCEPT Phase 3 Trial Results: Summary

    INTERCEPT INTERCEPT Phase 3 Trial Results: Summary • Treatment with AXS-07 resulted in rapid, sustained, and statistically significant efficacy as compared to placebo • Early treatment with AXS-07 resulted in significant prevention of migraine pain progression • Efficacy benefits of AXS-07 translate

  22. Slide 22: Q&A

    Q&A © Axsome Therapeutics, Inc. 22

  23. Slide 23: Concluding Remarks

    Concluding Remarks Herriot Tabuteau, MD CHIEF EXECUTIVE OFFICER AXSOME THERAPEUTICS, INC. © Axsome Therapeutics, Inc. 23

  24. Slide 24: AXS-07: Clinical Development in Migraine

    AXS-05 AXS-07: Clinical Development in Migraine Indication Acute treatment of migraine Acute treatment of migraine Acute treatment of migraine History of inadequate response to prior treatments Participation in MOMENTUM or INTERCEPT Patient population All comers Migraine pain severity and treatment

  25. Slide 25: Our CNS Candidates and Pipeline

    Overview Our CNS Candidates and Pipeline • Five differentiated clinical-stage CNS assets targeting significant and growing markets • Patent protection to 2034-2036, worldwide rights for most product candidates ease: Fast Track Designation Abbreviations: BUP = Bupropion; CNS = Central Nervous System;

  26. Slide 26: Our Clinical and Regulatory Milestones

    Corporate Our Clinical and Regulatory Milestones ● NDA submission (4Q) Abbreviations: AD = Alzheimer’s Disease; BUP = Bupropion; DM = Dextromethorphan; MDD = Major Depressive Disorder; Mx = Meloxicam; Riz = Rizatriptan; TRD = Treatment Resistant Depression. ✓ Accomplished milestone. ● Upcoming miles

  27. Slide 27: For more information, please contact

    For more information, please contact Mark Jacobson Chief Operating Officer 212-332-3243 mjacobson@Axsome.com axsome.com

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