Axsome Therapeutics, Inc. · NASDAQ:AXSM
Axsome Therapeutics — Technical Presentation (March 2024)
25 slides from Axsome Therapeutics's technical presentation, Mar 25, 2024
Slide 1: SYMPHONY Topline Data
axsyme @) SYMPHONY __ topline data March 25, 2024 © Axsome Therapeutics, Inc.
Slide 2: Forward Looking Statements & Safe Harbor
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,” “
Slide 3: Today's Agenda
© Axsome Therapeutics, Inc. Mark Jacobson, COO Herriot Tabuteau, M.D., CEO Summary Narcolepsy: Clinical Perspective & Implications of SYMPHONY Study Results Concluding Remarks SYMPHONY topline data Q+A Michael J. Thorpy, M.D. Dr. Thorpy & Axsome team Herriot Tabuteau, M.D., CEO Today’s Agenda
Slide 4: Summary of Topline Results
SYMPHONY Phase 3 Trial of AXS-12 in Narcolepsy Summary of Topline Results AXS-12 met the primary endpoint by demonstrating a substantial and statistically significant reduction in weekly cataplexy attacks compared to placebo (p=0.018). AXS-12 achieved statistically significant remission of cataplexy
Slide 5: Narcolepsy: Mechanism of Disease
Narcolepsy: Mechanism of Disease Norepinephrine is important to the control of muscle tone during wakefulness. Norepinephrine and dopamine play an important in sleep-wake regulation.1-3 Loss of orexin input decreases excitation of neurons that produce norepinephrine and dopamine1,2 Decreased norepin
Slide 6: Blocking norepinephrine reuptake can reduce cataplexy
title and content slide Increased norepinephrine and dopamine signaling in PFC Blocking norepinephrine reuptake can reduce cataplexy AXS-12 (reboxetine) is a selective norepinephrine reuptake inhibitor and cortical dopamine modulator*1-3 Increased norepinephrine Signaling2,3 Increased dopamine signa
Slide 7: SYMPHONY Phase 3 Study Topline Data AXS-12 (reboxetine)
SYMPHONY Phase 3 Study Topline Data AXS-12 (reboxetine)
Slide 8: Trial Design and Endpoints
Trial Design and Endpoints A Phase 3 trial to assess efficacy and safety of AXS-12 as compared to placebo in the treatment of cataplexy in narcolepsy. ESS: Epworth Sleepiness Scale; NSAQ: Narcolepsy Symptom Assessment Questionnaire; QD: once daily dosing; BID: twice daily dosing Double-blind Phase (
Slide 9: Demographics and Baseline Characteristics
Demographics and Baseline Characteristics EDS: Excessive Daytime Sleepiness; CGI-S: Clinical Global Impressions-Severity Characteristic AXS-12 (n = 46) Placebo (n = 44) Age (years) 36 34.2 Sex (% male) 45.7% 34.1% Time since diagnosis (years, mean) 7.9 6.3 Weekly frequency of cataplexy attacks (medi
Slide 10: Cataplexy Frequency: Primary Endpoint
Cataplexy Frequency: Primary Endpoint Change in Weekly Cataplexy Attacks Change in Weekly Cataplexy Attacks Rate Ratio p=0.007 p=0.006 p=0.031 p=0.031 p=0.018 Rate Ratio is calculated as the ratio of change in the AXS-12 group divided by the ratio of change in the placebo group
Slide 11: Achievement of Cataplexy Remission
Achievement of Cataplexy Remission Patients Achieving Remission (100% Reduction) in Cataplexy Attacks p=0.008 p=0.013 p=0.008
Slide 12: Effect on Excessive Daytime Sleepiness
Effect on Excessive Daytime Sleepiness Improvement of EDS as Measured by CGI-S EDS: Excessive Daytime Sleepiness; CGI-S: Clinical Global Impression-Severity p=0.006 p=0.002 p=0.001 p=0.027 57% of AXS-12 patients vs. 33% of placebo achieved concurrent EDS (naps) and cataplexy response (p=0.029) at We
Slide 13: Improvement in Cognitive Function
Improvement in Cognitive Function FOSQ-10 Cognitive Function, Concurrent Ability to Concentrate Response p=0.016 p=0.004
Slide 14: Effect on Narcolepsy Overall, Function and Quality of Life
Effect on Narcolepsy Overall, Function and Quality of Life Narcolepsy Overall AXS-12 treatment resulted in significant reduction in overall narcolepsy severity (CGI-S for narcolepsy overall) compared to placebo at Week 5 (p=0.007). Improvement in the CGI-S for narcolepsy overall for AXS-12 compared
Slide 15: Anxiety / Depression
Anxiety / Depression Percentage with Improvement on EQ-5D-5L Anxiety/Depression Domain p=0.146
Slide 16: Safety and Tolerability
Safety and Tolerability GI: gastrointestinal AXS-12 was well tolerated in the trial The most commonly reported adverse events in the AXS-12 arm were dry mouth (n=6), nausea (n=6), and constipation (n=4) which were overall mild to moderate. The rates of discontinuation due to adverse events was low (
Slide 17: Narcolepsy: Clinical Perspective and Implications of SYMPHONY Study Results
Dr. Michael Thorpy Albert Einstein College of Medicine NARCOLEPSY Clinical Perspective and Implications of SYMPHONY Study Results © Axsome Therapeutics, Inc.
Slide 18: Narcolepsy Clinical Overview
© Axsome Therapeutics, Inc. Narcolepsy Clinical Overview 185,000 in the U.S. with narcolepsy4,5 Serious, debilitating, lifelong, neurologic disorder that disrupts the boundaries between sleep and wake states.1-3 Characterized by excessive daytime sleepiness (EDS), cataplexy, hypnagogic hallucination
Slide 19: Narcolepsy is associated with significant morbidity, mortality, and healthcare utilization
Narcolepsy is associated with significant morbidity, mortality, and healthcare utilization Mortality rate3 Motor vehicle accident injuries2 Hospital and physician visits1 Medication use1 Healthcare use and costs1 People with narcolepsy have: Narcolepsy leads to impaired psychosocial development, edu
Slide 20: Cataplexy poses significant patient burden
Cataplexy poses significant patient burden Results from CRESCENDO survey of treated patients1 1. CRESCENDO Survey, Axsome data on file Sudden and transient loss or reduction of muscle tone while awake, typically triggered by strong emotions (e.g., laughter, elation, surprise, anger) 77% of patients
Slide 21: Narcolepsy current treatment landscape
Narcolepsy current treatment landscape Current Treatment Types Used by NT1 Patients (n=203)* Results from CRESCENDO survey of treated patients1 Currently Take Wake Promoting Agents* 53% Oxybates* 47% Stimulants* 42% Antidepressants (for narcolepsy)* 38% Other (nighttime medication)* 10% Other (dayti
Slide 22: High rates of symptoms despite current treatments
High rates of symptoms despite current treatments Percentage of Patients With Persistent Symptoms While on Treatment* Results from CRESCENDO survey of treated patients1 Narcolepsy patients have significant pre- and post-treatment unmet needs 1. CRESCENDO Survey, Axsome data on file * Excessive dayti
Slide 23: Implications of SYMPHONY Study
Implications of SYMPHONY Study AXS-12 treatment was associated with a rapid and robust efficacy on cataplexy compared to placebo, demonstrating a significant reduction in weekly attacks, achievement remission and cataplexy-free days. AXS-12 improved EDS severity as measured by the CGI-S, which was s
Slide 24: Thank You
Q+A Thank You cxtome
Slide 25: SYMPHONY Topline Data
axsyme @) SYMPHONY __ topline data March 25, 2024 © Axsome Therapeutics, Inc.
Axsome Therapeutics — Technical Presentation (March 2024) on 6ix