Axsome Therapeutics, Inc. · NASDAQ:AXSM

Axsome Therapeutics — Technical Presentation (December 2019)

26 slides from Axsome Therapeutics's technical presentation, Dec 30, 2019

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

    NASDAQ: AXSM AXSOME THERAPEUTICS MOMENTUM Phase 3 Trial of AXS-07 in Results Conference Call Migraine Topline December 30, 2019 © Axsome Therapeutics, Inc.

  2. Slide 2: Presenters and Overview

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

  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 MOMENTUM Phase 3 Trial: Summary of Topline Results

    MOMENTUM AXS-07 MOMENTUM Phase 3 Trial: Summary of Topline Results • AXS-07 is a novel, oral, multi-mechanistic investigational medicine for the acute treatment of migraine • MOMENTUM enrolled only patients with a history of inadequate response to prior acute treatments and incorporated rizatriptan

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

    Migraine Migraine: Disabling Disease in Need of New Treatments • The W orld 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 CGRP Mediated Rizatriptan vasodilation  Cyclooxygenase inhibition  PGE2 synthesis inhibition MoSEIC™ meloxicam Neuroinflammation  Decrease passage of pai

  8. Slide 8: Phase 3 Trial Design & Results

    MOMENTUM 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: MOMENTUM Phase 3 Trial: Design Summary

    MOMENTUM MOMENTUM Phase 3 Trial: Design Summary MOMENTUM: Maximizing OutcoMEs iN Treating acUte Migraine Phase 3 study of AXS-07 for the acute treatment of migraine in adults with history of inadequate response to prior treatment Screening Single dose, following a qualifying migraine (20 mg M oSEI C

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

    MOMENTUM MOMENTUM 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 moderate to severe migraines per month, on averag

  11. Slide 11: MOMENTUM Baseline Characteristics: Difficult-to-Treat Migraine

    MOMENTUM MOMENTUM Baseline Characteristics: Difficult-to-Treat Migraine Characteristics MoSEIC Meloxicam (20 mg) AXS-07 (20 mg MoSEIC Mlx / 10 mg Riz) Rizatriptan (10 mg) Placebo n=428 n=419 n=421 n=209 Total mTOQ-4 Score, mean (SD) 3.5 (2.17) 3.8 (2.14) 3.6 (2.19) 3.6 (2.25) Presence of Allodynia,

  12. Slide 12: MOMENTUM Baseline Characteristics: Demographics

    MOMENTUM MOMENTUM Baseline Characteristics: Demographics MoSEIC Meloxicam (20 mg) AXS-07 (20 mg MoSEIC Mlx / 10 mg Riz) Rizatriptan (10 mg) Placebo n=428 n=419 n=421 n=209 Age, years 41.2 (11.52) 41.0 (12.07) 40.8 (11.47) 41.4 (10.68) Female gender, n (%) 346 (80.8%) 355 (84.3%) 177 (84.7%) 353 (84.

  13. Slide 13: Co-Primary Endpoints: Pain and MBS Improvement

    MOMENTUM Co-Primary Endpoints: Pain and MBS Improvement AXS-07 (n=428) Placebo (n=209) Endpoints Difference P-Value Co-Primary Endpoint 1: Pain Freedom 2 Hours after Dose, % 19.9% 6.7% -13.2% <0.001 Co-Primary Endpoint 2: Absence of Most Bothersome Symptom 2 Hours after Dose, % 36.9% 24.4% -12.5% 0.

  14. Slide 14: Key Secondary Endpoint: 2-24 Hour Sustained Pain Freedom

    MOMENTUM Key Secondary Endpoint: 2-24 Hour Sustained Pain Freedom MoSEIC Meloxicam AXS-07 Rizatriptan Placebo (n=428) (n=419) (n=209) (n=421) Sustained Pain Freedom, Pain Freedom maintained from 2 to 24 Hours after Dose, % 16.1% 11.2% 8.8% 5.3% -4.9% Difference from AXS-07 -7.3% -10.9% 0.038 0.001 <

  15. Slide 15: Rapid Relief of Migraine Pain

    MOMENTUM Rapid Relief of Migraine Pain P=0.009 riptan 80% 70% 60% 50% 40% 30% 20% 10% 0% 0 0.5 1 1.5 2 Hour Post Dose 2.5 3 3.5 4 Placebo MoSEIC Meloxicam Rizatriptan AXS-07 © Axsome Therapeutics, Inc. 15 Subjects Reporting Pain Relief vs. Rizat P=0.019 vs. Rizatriptan P=0.040 vs. Rizatriptan • AXS-

  16. Slide 16: Pain Freedom Rates Over Time

    MOMENTUM Pain Freedom Rates Over Time: Significant Improvements in Pain Freedom P<0.001 70 P<0.001 P<0.001 60 50 40 30 20 10 0 2 Hour 4 Hour 12 Hour 16 Hour Placebo MoSEIC Meloxicam Rizatriptan AXS-07 © Axsome Therapeutics, Inc. 16 Percent of Subjects Achieving Pain Freedom P<0.001 P=0.019 P=0.018 5

  17. Slide 17: Sustained Effect from 2 to 24 Hours: AXS-07 Superiority to Rizatriptan

    MOMENTUM Sustained Effect from 2 to 24 hours: AXS-07 Superiority to Rizatriptan Sustained Pain Relief Sustained Pain Freedom P<0.001 P<0.001 20% P=0.001 60% 16% 50% 40% 12% 30% 8% 20% 4% 10% 0% 0% Placebo MoSEIC Meloxicam Rizatriptan AXS-07 Placebo MoSEIC Meloxicam Rizatriptan AXS-07 • 80% of subjec

  18. Slide 18: Sustained Effect from 2 to 48 Hours: AXS-07 Superiority to Rizatriptan

    MOMENTUM Sustained Effect from 2 to 48 hours: AXS-07 Superiority to Rizatriptan Sustained Pain Relief Sustained Pain Freedom P<0.001 60% P<0.001 20% 50% 16% 40% 12% 30% 8% 20% 4% 10% 0% 0% Placebo MoSEIC Meloxicam Rizatriptan AXS-07 Placebo MoSEIC Meloxicam Rizatriptan AXS-07 • 77% of subjects treat

  19. Slide 19: Reduced Use of Rescue Medication: AXS-07 Superiority to Rizatriptan

    MOMENTUM Reduced Use of Rescue Medication: AXS-07 Superiority to Rizatriptan • 77% of subjects receiving AXS-07 did not require rescue medication 50% P<0.001 40% 30% 20% 10% 0% Placebo MoSEIC MeloxicamRizatriptan AXS-07 © Axsome Therapeutics, Inc. 19 Percentage of Subjects Who Took Rescue Medication

  20. Slide 20: Multiple Efficacy-Related Endpoints: AXS-07 Superiority Over Rizatriptan

    MOMENTUM Multiple Efficacy-Related Endpoints: AXS-07 Superiority Over Rizatriptan P-value AXS-07 vs. Rizatriptan Endpoint 1 Hour Pain Relief 2-24 Hour Sustained Pain Relief 2-48 Hour Sustained Pain Relief 2-24 Hour Sustained Pain Freedom 2-48 Hour Sustained Pain Freedom PGI-C Functional Improvement

  21. Slide 21: Safety of AXS-07: Adverse Events Occurring in ≥2% of Subjects

    MOMENTUM Safety of AXS-07: Adverse Events Occurring in ≥2% of Subjects AXS-07 (N = 441) Rizatriptan (N = 434) Meloxicam (N = 433) Placebo (N = 218) Any Treatment-Emergent AE Nausea Dizziness Somnolence 49 (11.1%) 12 (2.7%) 7 (1.6%) 6 (1.4%) 67 (15.4%) 21 (4.8%) 9 (2.1%) 9 (2.1%) 50 (11.5%) 14 (3.2%)

  22. Slide 22: MOMENTUM Phase 3 Trial Results: Summary

    MOMENTUM MOMENTUM Phase 3 Trial Results: Summary • AXS-07 resulted in rapid, sustained, substantial and statistically significant efficacy as compared to placebo and rizatriptan in the acute treatment of migraine in patients with a history of inadequate response to prior acute treatments. • The effi

  23. Slide 23: Q&A

    Q&A © Axsome Therapeutics, Inc. 23

  24. Slide 24: Concluding Remarks

    Concluding Remarks Herriot Tabuteau, MD CHIEF EXECUTIVE OFFICER AXSOME THERAPEUTICS, INC. © Axsome Therapeutics, Inc. 24

  25. Slide 25: INTERCEPT Study of AXS-07 for Acute Treatment of Migraine

    AXS-07 INTERCEPT Study of AXS-07 For the Acute Treatment of Migraine INTERCEPT STUDY: INiTiating EaRly Control of MigrainE Pain & Associated SympToms Phase 3 trial of AXS-07 for the acute treatment of migraine Screening Single dose, immediately following migraine pain onset n=300 1:1 randomization P

  26. Slide 26: Our CNS Candidates and Pipeline

    Overview Our CNS Candidates and Pipeline • Four differentiated clinical-stage CNS assets targeting significant and growing markets • Patent protection to 2034-2036, worldwide rights signation rder: Breakthrough Therap signation Protocol Assessment an Designation Abbreviations: BUP = Bupropion; CNS =

Axsome Therapeutics — Technical Presentation (December 2019) on 6ix