Axsome Therapeutics, Inc. · NASDAQ:AXSM

Axsome Therapeutics — GEMINI Phase 3 Trial of AXS-05 in MDD Topline Results Conference Call (December 2019)

22 slides from Axsome Therapeutics's conference presentation, Dec 16, 2019

  1. Slide 1: GEMINI Phase 3 Trial of AXS-05 in MDD Topline Results Conference Call

    NASDAQ: AXSM GEMINI Phase 3 Trial of AXS-05 in MDD Topline Results Conference Call December 16, 2019 © Axsome Therapeutics, Inc.

  2. Slide 2: AXS-05 in MDD GEMINI Phase 3 Trial Topline Results Overview

    2 © Axsome Therapeutics, Inc. AXS-05 in Major Depressive Disorder (MDD) GEMINI Phase 3 Trial Topline Results Overview Introduction Mark Jacobson, Senior Vice President, Operations Overview and Summary Herriot Tabuteau, MD, Chief Executive Officer GEMINI Trial Design & Results Cedric O’Gorman, MD, Se

  3. Slide 3: 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

  4. Slide 4: Overview and Summary

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

  5. Slide 5: Summary of Topline Results: Rapid and Durable Effects with AXS-05

    5 Summary of Topline Results: Rapid and Durable Effects with AXS-05 © Axsome Therapeutics, Inc. GEMINI • AXS-05: a novel, oral, investigational NMDA receptor antagonist with multimodal activity • Rapid, durable, substantial, and significant improvement in symptoms of depression • Met primary endpoin

  6. Slide 6: Major Depressive Disorder (MDD): Need for New, Innovative Treatments

    • Major depressive disorder (MDD) is a disabling and potentially life-threatening, biologically- based disorder • Majority of patients experience inadequate response to current treatments: 63% fail to achieve remission to initial therapy, and of those 69% fail second line therapy2 • Current antidepr

  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: GEMINI Phase 3 Trial Design & Results

    GEMINI Phase 3 Trial Design & Results 8 © Axsome Therapeutics, Inc. Cedric O’Gorman MD, MBA Senior Vice President, Clinical Development and Medical Affairs Axsome Therapeutics, Inc.

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

    9 GEMINI Phase 3 Trial: Design Summary GEMINI BUP = Bupropion; DM = Dextromethorphan Double-blind Dosing Period (6 weeks) N = 327 1:1 randomization Up to 4-week Screening Period Placebo (twice daily) AXS-05 (45 mg DM + 105 mg BUP, twice daily) Week 0 Week 1 Week 2 Week 3 Week 6 Week 4 © Axsome Thera

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

    10 GEMINI Phase 3 Trial: Key Entry Criteria GEMINI © Axsome Therapeutics, Inc. Inclusion criteria included: • Male or female 18-65 years of age inclusive • DSM-5 criteria for current MDD without psychotic features • Montgomery-Åsberg Depression Rating Scale (MADRS) total score of ≥ 25 • CGI-S score

  11. Slide 11: GEMINI Phase 3 Trial: Demographics and Baseline Characteristics

    11 GEMINI Phase 3 Trial: Demographics and Baseline Characteristics GEMINI © Axsome Therapeutics, Inc. AXS-05 (45 mg DM / 105 mg BUP) Placebo Age (years) 42.1 (12.71) 41.1 (13.78) Female Gender, n (%) 98 (60.1%) 117 (71.3%) Race, n (%) White Black or African American Asian Other or Not Reported 88 (5

  12. Slide 12: Improvement in Depressive Symptoms: Primary Endpoint – Change in MADRS Total Score

    -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) 12 Improvement in Depressive Symptoms: Primary Endpoint – Change in MADRS Total Score © Axsome Therapeutics, Inc. GEMINI P<0.001 P=0.002 P=0.0

  13. Slide 13: Improvement in Depressive Symptoms: Clinical Response

    15% 28% 42% 49% 54% 7% 17% 25% 27% 34% 0% 10% 20% 30% 40% 50% 60% 1 2 3 4 6 Percentage of Responders ( ≥ 50% MADRS) Week AXS-05 Placebo 13 © Axsome Therapeutics, Inc. GEMINI Improvement in Depressive Symptoms: Clinical Response (≥ 50% MADRS improvement) P=0.035 P=0.020 P=0.001 P<0.001 P<0.001

  14. Slide 14: Improvement in Depressive Symptoms: Achievement of Remission

    3% 17% 24% 32% 40% 2% 8% 11% 12% 17% 0% 5% 10% 15% 20% 25% 30% 35% 40% 45% 1 2 3 4 6 Percentage of Subjects Achieving Remission (MADRS ≤ 10) Week AXS-05 Placebo 14 © Axsome Therapeutics, Inc. GEMINI Improvement in Depressive Symptoms: Achievement of Remission (MADRS ≤ 10) P=0.013 P=0.002 P<0.001 P<0

  15. Slide 15: Rapid and Durable Improvements: Treatment Effects with AXS-05

    15 © Axsome Therapeutics, Inc. GEMINI Rapid and Durable Improvements: Treatment Effects with AXS-05 P-value Week 1 Week 2 Week 6 Depressive Symptom Improvement MADRS Total Score Change from Baseline 0.007 <0.001 0.002 Clinical Response ≥50% Reduction in MADRS from Baseline 0.035 0.020 <0.001 Remissi

  16. Slide 16: Safety of AXS-05 in MDD: Adverse Events Occurring in ≥5% of Subjects

    16 © Axsome Therapeutics, Inc. GEMINI Safety of AXS-05 in MDD: Adverse Events Occurring in ≥5% of Subjects • Rates of discontinuation due to adverse events were low in both groups, 6.2% and 0.6%, for AXS-05 and placebo, respectively • One serious adverse event in the AXS-05 arm, which was not relate

  17. Slide 17: GEMINI Phase 3 Trial Results: Summary

    17 GEMINI Phase 3 Trial Results: Summary © Axsome Therapeutics, Inc. GEMINI •AXS-05 resulted in rapid, durable, substantial, and statistically significant improvements in depressive symptomatology across multiple efficacy endpoints • Symptomatic benefits translated into statistically significant imp

  18. Slide 18: Q&A

    18 Q&A © Axsome Therapeutics, Inc.

  19. Slide 19: Concluding Remarks

    Concluding Remarks 19 © Axsome Therapeutics, Inc. Herriot Tabuteau, MD Chief Executive Officer Axsome Therapeutics, Inc.

  20. Slide 20: AXS-05: Clinical Programs in Psychiatry

    20 AXS-05: Clinical Programs in Psychiatry © Axsome Therapeutics, Inc. AXS-05 Clinical Program ASCEND GEMINI STRIDE-1 AXS-05 / OL ADVANCE-1 Indication MDD MDD TRD MDD/TRD Alzheimer's Agitation Phase Pivotal Phase 2 Pivotal Phase 3 Pivotal Phase 3 Open-label Phase 3 Pivotal Phase 2/3 Objectives Effic

  21. Slide 21: Our CNS Candidates and Pipeline Overview

    • Four differentiated clinical-stage CNS assets targeting significant and growing markets • Patent protection to 2034-2036, worldwide rights Product Candidate Preclinical Phase 1 Phase 2 Phase 3 AXS-05 (DM + BUP) AXS-07 (MoSEIC™ Mx + Riz) AXS-12 (Reboxetine) AXS-09 (DM + S-BUP) Abbreviations: BUP =

  22. Slide 22: For more information, please contact Mark Jacobson

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

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