Axsome Therapeutics, Inc. · NASDAQ:AXSM

Axsome Therapeutics — Technical Presentation (January 2019)

22 slides from Axsome Therapeutics's technical presentation, Jan 7, 2019

  1. Slide 1: AXS-05 in MDD ASCEND Phase 2 Trial Topline Results & Medical Affairs

    Overview AXS-05 in MDD ASCEND Phase 2 Trial Topline Results & Medical Affairs © Axsome Therapeutics, Inc. 2 Introduction Mark Jacobson, Senior Vice President, Operations Overview and Summary Herriot Tabuteau, MD, Chief Executive Officer ASCEND Trial Design & Results Cedric O’Gorman, MD, Senior Vice

  2. Slide 2: 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

  3. Slide 3: Overview and Summary

    Overview and Summary Herriot Tabuteau, MD Chief Executive Officer Axsome Therapeutics, Inc. © Axsome Therapeutics, Inc. 4

  4. Slide 4: AXS-05: ASCEND Phase 2 Trial in MDD Summary of Topline Results

    AXS-05 AXS-05: ASCEND Phase 2 Trial in MDD Summary of Topline Results • AXS-05: a novel, oral, investigational NMDA receptor antagonist with multimodal activity. • Significantly and rapidly improved symptoms of depression. • Met primary endpoint—highly statistically significant improvement on MADRS

  5. Slide 5: Major Depressive Disorder Overview

    AXS-05 Major Depressive Disorder Overview (MDD): • Leading cause of disability, and a major contributor to overall disease burden worldwide. • As much as 70 % of patients have inadequate response to current first-line therapies. – Can take up to 6-8 weeks for clinically meaningful response. • All ap

  6. Slide 6: AXS-05: Novel Multimodal Therapy for CNS Disorders

    AXS-05 AXS-05: Novel Multimodal Therapy for CNS Disorders Single Target NMDA Multimodal Ketamine NMDA σ-1 NET SERT AXS-05 (Dextromethorphan/ Bupropion) NMDA σ-1 NET SERT nACh DAT Abbreviations: σ-1 = Sigma-1; DAT = Dopamine Reuptake Transporter; nACh = Nicotinic Acetylcholine Receptor; NMDA = N-meth

  7. Slide 7: ASCEND Trial Design & Results

    ASCEND Trial Design & Results Cedric O’Gorman, MD, MBA Senior Vice President, Clinical Development and Medical Affairs Axsome Therapeutics, Inc. © Axsome Therapeutics, Inc. 8

  8. Slide 8: Clinical Trial Design

    AXS-05 ASCEND: Clinical Trial Design • Phase 2, randomized, double-blind, active-controlled, multi-center, U.S. trial • N=80 adult patients with confirmed diagnosis of moderate to severe MDD • 6-week treatment period – Twice daily dosing • Dose groups (1:1 randomization): – AXS-05 (45 mg dextrometho

  9. Slide 9: Clinical Trial Design: Assessing Clinical Episodes in Depression

    AXS-05 ASCEND: Clinical Trial Design Assessing Clinical Episodes in Depression Double-blind Dosing Period (6 weeks) Week 6 Week 0 Week 1 Week 2 Week 3 Week 4 N = 43 N = 80 1:1 randomization Subjects with a confirmed diagnosis of moderate to severe MDD N = 37 BUP = Bupropion; DM = Dextromethorphan Pr

  10. Slide 10: Demographics and Baseline Characteristics

    AXS-05 ASCEND: Demographics and Baseline Characteristics • Mean age (years): 37.3 for AXS-05 group, 37.7 for bupropion group • Mean age at onset of illness (years): 26.5 for AXS-05 group, 26.6 for bupropion group • Mean MADRS total score at baseline: 31.8 for AXS-05 group, 32.2 for bupropion group •

  11. Slide 11: Primary Endpoint: Change in MADRS Total Score

    AXS-05 ASCEND: Primary Endpoint: Change in MADRS Total Score 0 -2 -4 -6 -8 -10 -12 -14 -16 -18 -20 -22 Baseline 1 2 3 4 5 6 Week AXS-05 Bupropion P-Value Primary Endpoint Change in MADRS Total Score over 6-Week Period (averaged) -13.7 -8.8 < 0.001 Change in MADRS Total Score at Week 6 -17.2 -12.1 0.

  12. Slide 12: Secondary Endpoint: Achievement of Remission

    AXS-05 ASCEND: Secondary Endpoint: Achievement of Remission P=0.004 50% AXS-05 Bupropion 45% 40% 35% 30% 25% 20% 15% 10% 5% 0% 1 2 3 Week 4 6 © Axsome Therapeutics, Inc. 13 Percent of Subjects Achieving Remission (MADRS < 10) P=0.022 47% P=0.005 40% P=0.004 30% P=0.184 26% 3% 16% 16% 5% 0% 5%

  13. Slide 13: Secondary Endpoints

    AXS-05 ASCEND: Secondary Endpoints Secondary Endpoint P-Value* MADRS Total Score Change Weeks 1-2 0.01 % Achieving Remission on MADRS at Week 2 0.004 % Achieving Remission on MADRS at Week 6 0.004 MADRS-6 Change at Week 6 0.007 % of Responders on MADRS-6 (>50% reduction from baseline) at Week 6 0.01

  14. Slide 14: Safety

    AXS-05 ASCEND: Safety • AXS-05 was safe and well tolerated with similar rates of adverse events in the AXS-05 and bupropion arms. • No serious adverse events, and no meaningful difference between the two treatment arms in discontinuations due to adverse events. • The most commonly reported adverse e

  15. Slide 15: Summary

    AXS-05 ASCEND: Summary • Statistically significant improvements on MADRS and secondary efficacy endpoints for AXS-05 in patients with MDD • Early and sustained separation from active comparator bupropion • Safe and well-tolerated with no psychotomimetic effects, weight gain, or increased sexual dysf

  16. Slide 16: Q&A

    Q&A © Axsome Therapeutics, Inc. 17

  17. Slide 17: Concluding Remarks

    Concluding Remarks Herriot Tabuteau, MD Chief Executive Officer Axsome Therapeutics, Inc. © Axsome Therapeutics, Inc. 18

  18. Slide 18: AXS-05: Conclusions and Ongoing Programs with AXS-05

    AXS-05 AXS-05: Conclusions and Ongoing Programs with AXS-05 • AXS-05 demonstrated significant and rapid antidepressant activity with a favorable safety profile in the ASCEND trial in MDD. • Data support the continued development of this novel multimodal agent in MDD and other neuropsychiatric indica

  19. Slide 19: Clinical Milestones

    Overview Clinical Milestones • Ph 3 top-line results • Ph 2 top-line results (1H 2019) Abbreviations: AD = Alzheimer’s Disease; BUP = Bupropion; DM = Dextromethorphan; MDD = Major Depressive Disorder; Mx = Meloxicam; Riz = Rizatriptan; TRD = Treatment Resistant Depression. Accomplished milestone. •

  20. Slide 20: 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. epression: Fast Track Granted anted der phan Designation Abbreviations: BUP = Bupropion; CNS = Central Nervous Syste

  21. Slide 21: 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. Product ees Preclinical Phase 1 Treatment Re Ra Agitation in Alzheit (M+ BUP) Ongoing AXS-07 (MoSEIC™ M+ Ri) AXS-12

  22. Slide 22: Thank you

    AXSOME THERAPEUTICS Thank you. axsome.com

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