Axsome Therapeutics, Inc. · NASDAQ:AXSM
Axsome Therapeutics — Technical Presentation (December 2019)
26 slides from Axsome Therapeutics's technical presentation, Dec 3, 2019
Slide 1: CONCERT Phase 2 Trial of AXS-12 in Narcolepsy Topline Results Conference Call
NASDAQ: AXSM CONCERT Phase 2 Trial of AXS-12 in Narcolepsy Topline Results Conference Call December 3, 2019
Slide 2: Overview
Overview AXS-12 in Narcolepsy CONCERT Phase 2 Trial Topline Results Introduction Mark Jacobson, Senior Vice President, Operations Overview and Summary Herriot Tabuteau, MD, Chief Executive Officer Cedric O'Gorman, MD, Senior Vice President, Clinical Development CONCERT Trial Design & Results & Medic
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
Slide 4: Overview and Summary
Overview and Summary Herriot Tabuteau, MD Chief Executive Officer Axsome Therapeutics, Inc. © Axsome Therapeutics, Inc. 4
Slide 5: Summary of Topline Results
Summary of Topline Results • AXS-12 (reboxetine) met the prespecified primary endpoint resulting in a highly statistically significantly reduction in the number of cataplexy attacks as compared to placebo • AXS-12 also significantly reduced excessive daytime sleepiness, assessed by the Epworth Sleep
Slide 6: Narcolepsy background and unmet need
• Narcolepsy is a chronic, debilitating, neurologic condition characterized by: – Excessive daytime sleepiness (EDS) – Cataplexy: a sudden reduction or loss of muscle tone triggered by strong emotions – Disturbed nocturnal sleep – Sleep paralysis – Hypnagogic hallucinations • Orphan condition: nearl
Slide 7: Narcolepsy neurobiology and norepinephrine signaling
• Narcolepsy is caused by a loss of hypocretin neurons in the brain • Hypocretin neurons normally excite norepinephrine neurons which promote wakefulness and help maintain muscle tone • Hypocretin loss leads to dysregulation of norepinephrine neurons resulting in: – Decreased wakefulness during the
Slide 8: AXS-12 (reboxetine) profile and rationale
• AXS-12 (reboxetine) is a potent, highly selective norepinephrine reuptake inhibitor • Rationale for development in narcolepsy: – Norepinephrine signaling is dysregulated in narcolepsy contributing to key symptoms – AXS-12 improves regulation of norepinephrine signaling • AXS-12 is orally administe
Slide 9: CONCERT Trial Design & Results
CONCERT Trial Design &Results Cedric O’Gorman MD, MBA Senior Vice President, Clinical Development and Medical Affairs Axsome Therapeutics, Inc. © Axsome Therapeutics, Inc. 9
Slide 10: Study Design
Study Design Multi-center, Randomized, Double-blind, Placebo-controlled, 3-Week Crossover Study to Assess the Efficacy and Safety of AXS-12 for Cataplexy and Excessive Daytime Sleepiness in Subjects with Narcolepsy Screening and Baseline AXS-12 Down-titration / washout Placebo Down-titration / washo
Slide 11: Study Endpoints
Study Endpoints Multi-center, Randomized, Double-blind, Placebo-controlled, 3-Week Crossover Study to Assess the Efficacy and Safety of AXS-12 for Cataplexy and Excessive Daytime Sleepiness in Subjects with Narcolepsy • Primary Endpoint: – Change in the mean weekly number of cataplexy attacks, avera
Slide 12: Demographics and Baseline Characteristics
Demographics and Baseline Characteristics • Mean age (years): 32.6 • Mean time since diagnosis (years): 3.8 • Mean cataplexy attacks at baseline: 30.0 weekly attacks • Mean Epworth Sleepiness Scale (ESS) score at baseline: 18.1 • Ability to Concentrate at baseline: rated “very poor”, “poor”, or “ave
Slide 13: Mean Change in Weekly Cataplexy Attacks
4.0 Mean Change in Weekly Cataplexy Attacks -4.0 -8.0 AXS-12 Placebo -12.0 -16.0 0 1 2 P<0.001 8 mg daily 10 mg daily P=0.002 BaselineWeek 1Week 2
Slide 14: Patients with a ≥50% Reduction in Cataplexy Attacks
Patients with a ≥50% Reduction in Cataplexy Attacks 80% P=0.003 76.2% 70% P=0.005 60% 57.1% Placebo AXS-12 40% 30% 30.0% 20% 15.0% 10% 0% Week 1Week 2
Slide 15: Patients with a ≥75% Reduction in Cataplexy Attacks
Patients with a ≥75% Reduction in Cataplexy Attacks P=0.018 42.9% 40% 30% P=0.02 Placebo AXS-12 20% 10% 10.0% 0.0% 0% Week 1Week 2
Slide 16: Change in Epworth Sleepiness Scale
0.0 -1.0 -2.0 Change in ESS -4.0 -5.0 -3.1 -6.0 -7.0 P=0.003 -6.0 -8.0
Slide 17: Change in Frequency of Inadvertent Naps
Week 1Week 2 0% Change in Frequency of Inadvertent Naps -4.2%-5.3% -20% P=0.038 -19.3% -30% P<0.001 -31.8% -40%
Slide 18: Patients with a ≥50% Reduction in Inadvertent Naps
P=0.036 Patients with a ≥50% Reduction in Inadvertent Naps 38.1% 30% Placebo AXS-12 20% 19.0% 10% 10.0% 5.0% 0% Week 1Week 2
Slide 19: Improvement in Ability to Concentrate Score
Improvement in Ability to Concentrate Score Placebo AXS-12 -0.30 -0.55 P=0.007 P=0.002 -0.80 8 mg daily 10 mg daily 0 1 2 • Ability to concentrate was collected daily on a 5-point scale where 1= very good, 2 = good, 3 = average, 4 = poor, 5 = very poor.
Slide 20: Proportion of Patients Improving
50% 40% 38.1% 42.9% Proportion of Patients Placebo AXS-12 20% 15.0% 10% 0% 0% BaselineWeek 1Week 2
Slide 21: Improvement in Sleep Quality and Related Symptoms
60% P=0.069 55.0% Proportion of Patients Demonstrating Improvement P=0.007 45.0% P=0.365 40% P=0.044 40.0% 30% 30.0% 26.3%26.3% 20% 10% 5.3%5.3% 0% Quality of SleepNight awakeningsSleep Paralysis Episodes Hypnagogic Hallucinations
Slide 22: Safety and Tolerability
• AXS-12 was safe and well tolerated • No serious adverse events • No discontinuations due to adverse events • The overall percentage of patients experiencing adverse events was 42.9% with AXS-12 treatment and 40.0% with placebo • The most commonly reported adverse events with AXS-12 treatment were
Slide 23: Summary
Summary • AXS-12 met the pre-specified primary endpoint by significantly reducing the frequency of cataplexy attacks compared to placebo • AXS-12 also demonstrated significant improvements compared to placebo in symptoms of excessive daytime sleepiness • AXS-12 demonstrated improvements in cognitive
Slide 24: FQ3A
FQ3A E THERAPEUTICS sane wpe
Slide 25: Concluding Remarks
Concluding Remarks Herriot Tabuteau, MD Chief Executive Officer Axsome Therapeutics, Inc. © Axsome Therapeutics, Inc. 25
Slide 26: Thank you.
AXSOME THERAPEUTICS Thank you.
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