Axsome Therapeutics, Inc. · NASDAQ:AXSM
Axsome Therapeutics — Q3 2024 Earnings Presentation
48 slides from Axsome Therapeutics's earnings presentation, Nov 12, 2024
Slide 1: 3Q 2024 Corporate Presentation
November 2024 3Q 2024 Corporate Presentation © Axsome Therapeutics, Inc.
Slide 2: Forward Looking Statements & Safe Harbor
Forward Looking Statements & Safe Harbor Certain matters discussed in this press release are “forward-looking statements”. The Company may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “mi
Slide 3: Our Mission
Our Mission Develop and deliver transformative medicines for the hundreds of millions of people impacted by central nervous system conditions © Axsome Therapeutics, Inc.
Slide 4: Potential to reach >150M people in the U.S. across serious CNS conditions
Potential to reach >150M people in the U.S. across 10 serious CNS conditions We focus on therapeutic areas with critical gaps in care and a significant unmet need for new treatment options… Major Depressive Disorder Alzheimer’s Disease Agitation Smoking Cessation ADHD Binge Eating Disorder Neurology
Slide 5: Lead in innovation to expand therapeutic possibilities for CNS conditions
…And lead in innovation to expand the therapeutic possibilities for CNS conditions Multi-mechanistic approaches Metabolic pharmacokinetic modulation Clinical trial innovation Molecular drug delivery First-in-class mechanisms of action
Slide 6: Multiple value-creating opportunities to enable robust, long-term growth
Multiple value-creating opportunities to enable robust, long-term growth through 2040s and beyond In-market novel mechanisms of action NDA-stage product candidates Pivotal late-stage development programs Phase 3 topline data readouts through 2025 New product/indication launches anticipated through 2
Slide 7: Advancing an industry-leading neuroscience pipeline
Phase 1 Phase 2 Phase 3 NDA Marketed Advancing an industry-leading neuroscience pipeline FDA Breakthrough Therapy Designation FDA Orphan Drug Designation Major Depressive Disorder Alzheimer’s Disease Agitation Smoking Cessation Attention Deficit Hyperactivity Disorder Binge Eating Disorder Major Dep
Slide 8: $16.5B peak sales potential driven by commercial and late-stage assets
Solriamfetol MDD Solriamfetol ADHD $1-$3B AXS-05 AD Agitation $0.5-$1B Solriamfetol BED $0.3-$0.5B Solriamfetol SWD $0.5-$1B AXS-05 Smoking Cessation $0.5-$1B AXS-07 Migraine AXS-14 Fibromyalgia AXS-12 Narcolepsy $0.3-$0.5B $1-$3B $0.5-$1B $0.5-$1B $1.5-$3B $1-$1.5B $16.5B peak sales potential drive
Slide 9: 3Q 2024 highlights
3Q 2024 highlights Poised to deliver ≥3 FDA approvals through 2025/2026 and 7 new product/indication launches through 2027 Leading CNS Innovation Total net product revenue of $104.8M represents 81% YoY growth vs. 3Q 2023 Auvelity: $80.4M Sunosi: $24.4M Strong demand for Auvelity and Sunosi expected
Slide 10: Key achievements to date with catalyst-rich path ahead
Key achievements to date with catalyst-rich path ahead 2025 & 2026 Clinical Trial Topline Results Clinical Trial Initiations & Progress Updates NDA resubmission for AXS-07 in migraine accepted for review by the FDA (3Q 2024) AXS-07 PDUFA goal date (January 31, 2025) Positive topline results from SYM
Slide 11: 3Q 2024 financial summary
Net product revenue $104.8 $57.8 81% $266.9 $133.3 100% Auvelity net product sales $80.4 $37.7 113% $198.8 $81.0 145% Sunosi net product revenue† $24.4 $20.1 21% $68.1 $52.3 30% R&D expense $45.4 $28.8 58% $132.1 $67.1 97% SG&A expense $95.6 $83.2 15% $298.1 $236.3 26% 3Q 2024 financial summary 3Q =
Slide 12: 3Q 2024 commercial highlights
3Q 2024 commercial highlights Net product sales of $80.4M represents 113% YoY growth vs. 3Q 2023 ~140,000 new patients and >28,000 unique writers since launch ~78% of all covered lives between commercial and government (Medicare and Medicaid) channels Key drivers of prescribing Auvelity – fast actin
Slide 13: Commercial Products
Commercial Products axsome
Slide 14: Auvelity – novel oral treatment for major depressive disorder
Auvelity – novel and differentiated oral treatment for major depressive disorder in adults1,2 Source: Symphony METYS Weekly TRx Launch to Date Rapid acting NMDA receptor antagonist and sigma-1 receptor agonist for MDD1* Rapid remission as early as week 2, sustained and increased vs control through w
Slide 15: Auvelity quarterly net sales performance
Auvelity quarterly net sales performance 3Q 2024 net sales of $80.4M represents 113% year-over-year growth vs. 3Q 2023
Slide 16: Sunosi – first and only DNRI approved for EDS in narcolepsy or OSA
Sunosi – first and only DNRI approved for EDS associated with narcolepsy or OSA1 Quarterly nTRx Launch to Date Source: Symphony METYS. nTRx normalizes number of pills in each Trx for 30-day period. First and only wakefulness promoting agent proven to improve wakefulness through 9 hours1 Improvements
Slide 17: Sunosi quarterly net revenue performance
Sunosi quarterly net revenue performance 3Q 2024 net revenue of $24.4M represents 21% year-over-year growth vs. 3Q 2023
Slide 18: Development Pipeline
Development Pipeline axsome
Slide 19: AXS-05 modulates neurotransmitters implicated in Alzheimer's disease
AXS-05 modulates the function of neurotransmitters implicated in Alzheimer’s disease (glutamate, sigma-1, norepinephrine, and dopamine)1-4 In Alzheimer’s disease, insoluble Aβ production and accumulation triggers secondary steps leading to synaptic loss and neuronal cell death1,2 Reductions in certa
Slide 20: Alzheimer's disease agitation
Number of U.S. adults aged 65+ with Alzheimer’s dementia expected to double by 20601 Alzheimer’s disease (AD) agitation Alzheimer’s disease (AD) is the most common form of dementia, affecting approximately 7M people in the U.S.1 AD agitation is associated with accelerated cognitive decline, increase
Slide 21: Clinically meaningful improvements in symptoms of agitation
P=0.069 P=0.007 P=0.010 Clinically meaningful improvements in symptoms of agitation Primary endpoint: Change from baseline in CMAI total score at week 5 Rapid and substantial reduction in agitation with separation as early as Week 2 and statistically significant improvement at Week 3 Significantly g
Slide 22: Significant increase in time to relapse
Substantial and statistically significant increase in time to relapse Hazard Ratio for Time to Relapse Hazard Ratio (95% CI) 0.275 (0.091-0.836) P-value 0.014 AXS-05 significantly delayed time to relapse and prevented more relapses of agitation symptoms vs. placebo Patients on AXS-05 were 3.6x less
Slide 23: Comprehensive development program of AXS-05 in Alzheimer's agitation
ADVANCE-1 Phase 2/3 (N=366) ACCORD-1 Phase 3 (N=108) ACCORD-2 Phase 3 (N=140) Two completed, positive, pivotal efficacy and safety trials in >450 patients with Alzheimer’s disease agitation Two ongoing pivotal Phase 3 trials evaluating the efficacy and safety of AXS-05 vs. placebo Ongoing open-label
Slide 24: Ongoing pivotal Phase 3 trials of AXS-05 in Alzheimer's agitation
Ongoing pivotal Phase 3 trials evaluating the efficacy and safety of AXS-05 in Alzheimer’s disease agitation Consistent trial design as the ACCORD-1 Phase 3 trial Key eligibility criteria 65-90 years of age with diagnosis of probable Alzheimer’s disease (AD) and clinically significant agitation resu
Slide 25: Smoking cessation
Smoking cessation ~34M adults in the U.S. smoke cigarettes, ~50% of whom live with a smoking-related disease1 Single largest cause of preventable disease and death in the U.S., accounting for nearly 1 in 5 deaths1 70% of smokers want to quit2 Associated with over $300 billion in annual costs in the
Slide 26: AXS-07 targets four pathways implicated in a migraine attack
Rizatriptan inhibits the release of CGRP by stimulating 5-HT1D receptors on the pre-synaptic trigeminal nerve ending, resulting in reduced pain signal transmission Rizatriptan stimulates 5-HT1B receptors on the post-synaptic arterial smooth muscle cell, resulting in reduced vasodilation MoSEICTM mel
Slide 27: Migraine
Migraine >70% of patients are not fully satisfied with their current treatment and desire faster, more durable therapies4,5 Leading cause of disability among neurological disorders in the U.S., affecting approximately 39M people1,2 Characterized by recurrent attacks of pulsating, often severe and di
Slide 28: AXS-07 differentiated efficacy and safety profile across Phase 3 trials
Differentiated efficacy and safety profile supported by three Phase 3 clinical trials Ongoing Phase 3 trial evaluating the efficacy and safety of AXS-07 (oral CGRP antagonist non-responders) MOMENTUM Phase 3 (N=1594) Two completed, positive, registrational efficacy and safety trials in >1,800 patien
Slide 29: AXS-12 novel approach for the treatment of narcolepsy
AXS-12 (reboxetine) Novel pharmacological approach for the treatment of narcolepsy Norepinephrine and dopamine play important roles in sleep-wake regulation (both) and in maintaining muscle tone during wakefulness (norepinephrine)1-3 AXS-12 inhibits the reuptake of both neurotransmitters, improving
Slide 30: Narcolepsy
Narcolepsy Rare and debilitating neurological condition that affects approximately 185,000 people in the U.S.1 Characterized by cataplexy, excessive daytime sleepiness (EDS), hypnagogic hallucinations, sleep paralysis, and disrupted nocturnal sleep2-4 An estimated 70% of patients suffer from cataple
Slide 31: Reductions in cataplexy and EDS in two completed trials
Statistically significant reductions in cataplexy and EDS in two completed clinical trials Rate Ratio* Change in Weekly Cataplexy Attacks p=0.007 p=0.006 p=0.031 p=0.031 p=0.018 *Ratio of change in the AXS-12 group divided by the ratio of change in the placebo group SYMPHONY CONCERT (Phase 2) SYMPHO
Slide 32: ENCORE Phase 3 trial design
ENCORE Phase 3 trial design Open-label Period (24 weeks) Double-blind Phase (Up to 3 weeks) Baseline AXS-12 Placebo AXS-12 1:1 R Two-period trial evaluating long-term efficacy and safety of AXS-12 in narcolepsy TEAE = treatment emergent adverse event Key eligibility criteria 15-75 years of age with
Slide 33: AXS-14 novel approach for fibromyalgia
AXS-14 (esreboxetine) Novel pharmacological approach for the management of fibromyalgia (FM) Fibromyalgia pain is thought to be partially caused by dysregulated signaling in the descending analgesic system Norepinephrine, one of the key neurotransmitters in this pathway, has predominantly pain-inhib
Slide 34: Fibromyalgia
Fibromyalgia (FM) Chronic and debilitating neurological syndrome impacting ~17M people in the U.S.1 Characterized by widespread musculoskeletal pain, fatigue, disturbed sleep, depression, and cognitive impairment2 Limited treatment option with only 3 approved agents of variable and/or inadequate eff
Slide 35: Positive clinical data in fibromyalgia
Positive clinical data demonstrate statistically significant improvements in symptoms of fibromyalgia p<0.001 p<0.001 p=0.025 p<0.001 p<0.001 p=0.023 Phase 3 Efficacy Results (N=1,122) New Drug Application (NDA) Submission Anticipated November 2024 ~1,000 individuals with fibromyalgia dosed with esr
Slide 36: Solriamfetol Phase 3 development programs
Solriamfetol Phase 3 development programs Topline Data Anticipated 2026 Topline Data Anticipated 2025 Topline Data Anticipated 1Q 2025 Topline Data Anticipated 1Q 2025 Efficacy and safety of solriamfetol vs. placebo in adults with attention deficit hyperactivity disorder 6-week, double-blind, random
Slide 37: Attention deficit hyperactivity disorder (ADHD)
Attention deficit hyperactivity disorder (ADHD) Chronic neurodevelopmental disorder affecting an estimated ~22M people in the U.S.1, including ~7M children aged 3-17 years old2 Characterized by a persistent pattern of inattention and/or hyperactive-impulsive behaviors3 Associated with significant im
Slide 38: Evaluating solriamfetol as a potential treatment for ADHD
Evaluating solriamfetol as a potential treatment for ADHD Solriamfetol (150 mg) Solriamfetol (300 mg) Placebo 1:1:1 R Screening (5 weeks) Double-blind Phase (6 weeks) Follow-up (1 week) Baseline FOCUS Phase 3 Trial N=450 Key eligibility criteria 18-55 years of age with primary diagnosis of ADHD (DSM
Slide 39: Major depressive disorder (MDD)
Major depressive disorder (MDD) >70% of patients experience only a partial improvement in symptoms with first-line standard of care 1. “Major Depression.” NIMH (2023); 2. Hasin, D.S. et al. JAMA Psychiatry (2018) Serious and chronic mental health condition causing persistently low or depressed mood
Slide 40: Evaluating solriamfetol as a potential treatment for MDD
Solriamfetol (300 mg) Placebo 1:1 R Screening (5 weeks) Double-blind Phase (6 weeks) Follow-up (1 week) Baseline N=300 PARADIGM Phase 3 Trial Key eligibility criteria 18-65 years of age with confirmed diagnosis of moderate to severe MDD Primary endpoint Change from baseline in MADRS score The combin
Slide 41: Binge eating disorder (BED)
Binge eating disorder (BED) is the most common eating disorder and is thought to involve issues with food reward processing, impulse control, and appetite regulation1,2 Unmet medical need associated with a 2- to 3-fold increased risk of psychiatric and medical comorbidities3 Binge eating disorder (B
Slide 42: Evaluating solriamfetol as a potential treatment for BED
Evaluating solriamfetol as a potential treatment for BED Solriamfetol (150 mg) Solriamfetol (300 mg) Placebo 1:1:1 R Screening (4 weeks) Double-blind Phase (12 weeks) Follow-up (1 week) Baseline ENGAGE Phase 3 Trial N=450 Key eligibility criteria 18-55 years of age with diagnosis of BED (DSM-5) Prim
Slide 43: Shift work disorder (SWD)
Shift work disorder (SWD) is a combination of excessive sleepiness during wakefulness and persistent insomnia during daytime sleep when working outside a 7 a.m. to 6 p.m. workday1 Shift work has long been associated with multiple serious health complaints and a 23% greater risk of sustaining a work-
Slide 44: Evaluating solriamfetol as a potential treatment for SWD
Evaluating solriamfetol as a potential treatment for SWD Solriamfetol (150 mg) Solriamfetol (300 mg) Placebo 1:1:1 R Screening (2-4 weeks) Double-blind Phase (6 weeks) Follow-up (1 week) Baseline SUSTAIN Phase 3 Trial N=450 Key eligibility criteria 18-55 years of age with diagnosis of SWD (ICSD-2 or
Slide 45: Strong intellectual property and barriers to entry
Strong intellectual property and barriers to entry >135 issued U.S. patents and >92 issued O.U.S. patents Claims extending to at least 2034-43; Multiple pending Proprietary drug product formulation >98 issued U.S. patents and >129 issued O.U.S. patents Claims extending to at least 2038; Multiple pen
Slide 46: Financial snapshot
Cash Balance: (as of September 30, 2024) $327.3 M Debt (Face Value): (as of September 30, 2024) $180 M Market Cap: (as of November 11, 2024) $4.4 B Shares Outstanding: (as of September 30, 2024) 48.4 M Options, RSUs, and Warrants Outstanding†: 9.5 M Runway to reach cash flow positivity, based on the
Slide 47: Leadership team
Leadership team Roger Jeffs, PhD CEO, Liquidia Corporation Former President, Co-CEO, Director United Therapeutics Corp. Prior positions at Amgen and Burroughs Wellcome Herriot Tabuteau, MD Founder & CEO Management Board of Directors Nick Pizzie, CPA, MBA Chief Financial Officer Mark Jacobson, MA Chi
Slide 48: Thank you
November 2024 Thank you © Axsome Therapeutics, Inc.