Axsome Therapeutics, Inc. · NASDAQ:AXSM
Axsome Therapeutics — Q1 2025 Earnings Presentation
45 slides from Axsome Therapeutics's earnings presentation, May 5, 2025
Slide 1: Corporate Presentation May 5, 2025
Corporate Presentation May 5, 2025
Slide 2: Forward Looking Statements & Safe Harbor
Forward Looking Statements & Safe Harbor Certain matters discussed in this presentation 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,” “mig
Slide 3: Our Mission
Our Mission Develop and deliver transformative medicines for the hundreds of millions of people impacted by central nervous system conditions
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: And lead in innovation to expand the 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: Well-positioned to deliver significant near- and long-term value
Well-positioned to deliver significant near- and long-term value to patients and shareholders through 2040s and beyond In-market innovations NDA-stage product candidates Phase 3 development programs ongoing Potential new product/indication launches through 2027 Highly prevalent and/or difficult-to-t
Slide 7: Advancing an industry-leading neuroscience pipeline
NMDA = N-methyl-D-aspartate; CYP2D6 = Cytochrome P450 Family 2 Subfamily D Member 6; DNRI = Dopamine-norepinephrine reuptake inhibitor; TAAR1 = Trace amine-associated receptor 1; 5-HT = 5-Hydroxytryptamine; NRI = Norepinephrine reuptake inhibitor; Please see full Prescribing Information for AUVELITY
Slide 8: $16.5B peak sales potential driven by current commercial and late-stage assets
$16.5B peak sales potential driven by current commercial and late-stage assets AXS-05 AD Agitation $0.5-$1B AXS-14 Fibromyalgia AXS-12 Narcolepsy $0.5-$1B $1.5-$3B Solriamfetol MDD with EDS Solriamfetol ADHD $1-$3B $0.5-$1B Solriamfetol BED $0.3-$0.5B Solriamfetol SWD $0.5-$1B AXS-05 Smoking Cessati
Slide 9: 1Q 2025 highlights
1Q 2025 highlights Rapidly advancing late-stage pipeline 1Q 2025 total net product revenue of $121.5M represents 62% YoY growth vs. 1Q 2024 AUVELITY: $96.2M SUNOSI: $25.2M SYMBRAVO approved in the U.S. for the acute treatment of migraine with or without aura in adults; commercial launch on track for
Slide 10: Maintaining momentum into 2025 with catalyst-rich path ahead
Maintaining momentum into 2025 with catalyst-rich path ahead 2025 & 2026 Clinical Trial Topline Results Clinical Trial Initiations & Progress Updates Positive topline results from EMERGE Ph 3 trial of SYMBRAVO in oral CGRP non-responders (1Q 2025) Positive topline results from FOCUS Ph 3 trial of so
Slide 11: 1Q 2025 financial summary
Net product revenue $121.5 $75.0 62% AUVELITY net product sales $96.2 $53.4 80% SUNOSI net product revenue* $25.2 $21.6 17% R&D expense $44.8 $36.8 22% SG&A expense $120.8 $99.0 22% 1Q 2025 financial summary 1Q = three months ended March 31; *Includes royalty revenue associated with sales in out-lic
Slide 12: Financial snapshot
Cash Balance: (as of March 31, 2025) $300.9 M Debt (Face Value): (as of March 31, 2025) $180 M Market Cap: (as of May 2, 2025) $5.5 B Shares Outstanding: (as of March 31, 2025) 49.2 M Options, RSUs, and Warrants Outstanding*: 9.5 M Runway to reach cash flow positivity, based on the current operating
Slide 13: Commercial Highlights
Commercial Highlights axsome
Slide 14: Building a leading commercial portfolio in CNS
Building a leading commercial portfolio in CNS MAJOR DEPRESSIVE DISORDER EDS IN NARCOLEPSY OR OSA MIGRAINE WITH OR WITHOUT AURA Approved in the U.S. on January 30, 2025 EDS = Excessive daytime sleepiness; OSA = Obstructive sleep apnea Rx, sales, and revenue growth vs. comparable periods in 2024 +76%
Slide 15: AUVELITY: rapid acting NMDA receptor antagonist for MDD
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 week 63 Rapid symptom improvement starting at week 1, sustained at week 6 vs. placebo1 Novel and differentiated oral treatment for major depress
Slide 16: AUVELITY growth driven by robust, growing underlying demand
AUVELITY Growth driven by robust, growing underlying demand Source: Symphony METYS. Weekly TRx Launch to Date ~190,000 new patients and >36,000 unique writers since launch ~78% of all covered lives between commercial and government (Medicare and Medicaid) channels Key drivers of prescribing AUVELITY
Slide 17: 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 1Q 2024 net product sales of $96.2M represents 80% year-over-year growth +80%
Slide 18: SUNOSI: first and only wakefulness promoting agent proven through 9 hours
First and only wakefulness promoting agent proven to improve wakefulness through 9 hours1 Improvements in cognitive functioning vs. placebo demonstrated in clinical trials 90% of patients reported feeling better with SUNOSI 150 mg2 First and only DNRI approved for EDS associated with narcolepsy or O
Slide 19: SUNOSI continued success supported by strong fundamentals
Continued success supported by strong fundamentals SUNOSI Quarterly nTRx Launch to Date Source: Symphony METYS. nTRx normalizes number of pills in each Trx for 30-day period. ~85,000 new patients and >14,000 unique writers since initial launch ~83% of all covered lives between commercial and governm
Slide 20: 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 1Q 2025 net product revenue of $25.2M represents 17% year-over-year growth +17%
Slide 21: SYMBRAVO single oral dose provided rapid migraine pain freedom
Single, oral dose provided rapid migraine pain freedom and return to normal functioning within 2 hours1 Harnesses Axsome’s rapid absorption technology to target multiple pathways underlying a migraine attack Superior efficacy demonstrated across a broad range of migraine severity (mild, moderate, se
Slide 22: Positioning SYMBRAVO for immediate impact and long-term growth
Positioning SYMBRAVO for immediate impact and long-term growth 1. Symphony Health Claims, New to Brand Patients (2022-2023); 2. Lipton et al. Neurology (2015) Launch readiness High patient dissatisfaction due to limited efficacy and/or burdensome side effects >80% of patients discontinue their acute
Slide 23: Development Pipeline
Development Pipeline axsome
Slide 24: AXS-05 in Alzheimer's disease agitation
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 25: 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 26: Statistically significant improvements in Alzheimer's disease agitation
Statistically significant and clinically meaningful improvements in Alzheimer’s disease agitation CMAI = Cohen-Mansfield Agitation Inventory p=0.069 p=0.007 p=0.010 0 Weeks from Randomization Probability of Freedom from Relapse (%) ACCORD-2 100 75 50 25 0 2 4 6 8 10 12 14 16 18 20 22 24 26 + Censore
Slide 27: Smoking cessation
70% of smokers want to quit2 Only 3-5% who attempt to quit without assistance are successful for 6-12 months2 Smoking cessation Single largest cause of preventable disease and death in the U.S., accounting for nearly 1 in 5 deaths1 Associated with over $300 billion in annual costs in the U.S.1 ~34M
Slide 28: Unique pharmacology of solriamfetol supports potential utility in CNS conditions
Unique pharmacology of solriamfetol supports potential utility in a broad range of CNS conditions 1. Raony, I. et al. Prog Neuropsychopharmacol Biol Psychiatry (2022); Halff, E.F. et al. Trends Neurosci. (2023) Solriamfetol was initially developed as a dopamine and norepinephrine reuptake inhibitor
Slide 29: Solriamfetol Phase 3 development programs
Solriamfetol Phase 3 development programs Topline data 2026 Topline data 2026 Complete Complete Substantial and statistically significant improvements in ADHD symptoms and disease severity Initiation of Phase 3 pediatric trial anticipated in 2025 Solriamfetol FOCUS Phase 3 (N=516) PARADIGM Phase 3 (
Slide 30: Attention deficit hyperactivity disorder (ADHD)
Attention deficit hyperactivity disorder (ADHD) Chronic neurobiological and developmental 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
Slide 31: Statistically significant improvements in ADHD symptoms
Phase 3 pediatric trial initiation anticipated in 2025 Statistically significant improvements in ADHD symptoms Primary endpoint: Change from baseline in the AISRS total score at Week 6 Substantial improvement in ADHD symptoms observed as early as Week 1 (p=0.036, solriamfetol 150 mg) Significantly g
Slide 32: Major depressive disorder (MDD)
Major depressive disorder (MDD) ~2/3 of patients experience inadequate response to first-line treatment3 1. “Major Depression.” NIMH (2023); 2. Hasin, D.S. et al. JAMA Psychiatry (2018); 3. Rush, A.J. et al. Am J Psychiatry (2006). 4. Hein et al. J Affect Disord. (2019). Serious and chronic mental h
Slide 33: Binge eating disorder (BED)
Unmet medical need associated with a 2- to 3-fold increased risk of psychiatric and medical comorbidities3 Binge eating disorder (BED) Solriamfetol’s dopamine, norepinephrine, and TAAR1 mechanisms appear relevant to the pathophysiology of BED4-6 ~7 million people in the U.S. have BED2 BED is 1.75x m
Slide 34: 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 35: Shift work disorder (SWD)
Shift work has long been associated with multiple serious health complaints and a 23% greater risk of sustaining a work-related injury4-5 Shift work disorder (SWD) No new medications approved since 2007 and considerable residual sleepiness reported when medication is used6 ~15 million U.S. workers m
Slide 36: 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 (1-4 weeks) Double-blind Phase (12 weeks) Follow-up (1 week) Baseline SUSTAIN Phase 3 Trial N=450 Key eligibility criteria 18-65 years of age with diagnosis of SWD (ICSD-2 o
Slide 37: AXS-12 novel pharmacological approach for 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 both norepinephrine and cortical dopamine signaling in the brain The loss of orexin
Slide 38: 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 39: Statistically significant reductions in cataplexy frequency
Rate Ratio* 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 Primary endpoint: Change from baseline in weekly cataplexy attacks Statistically significant reductions in cataplexy frequency CONCERT p<0.001 p=0.002
Slide 40: AXS-14 novel approach for fibromyalgia
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-inhibitory effects AXS-14 is a more potent and selective enantiomer of racemic reboxetine that inh
Slide 41: Fibromyalgia (FM)
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 42: Positive clinical data show improvements in symptoms of fibromyalgia
Positive clinical data demonstrate statistically significant improvements in symptoms of fibromyalgia ~1,000 individuals with fibromyalgia dosed with esreboxetine across Phase 2 and Phase 3 clinical trials for up to 14 weeks Statistically significant and clinically meaningful reductions in pain scor
Slide 43: Strong intellectual property and barriers to entry
Strong intellectual property and barriers to entry Protected by a robust patent estate extending out to at least 2043; Multiple pending Proprietary drug product formulation >99 issued U.S. patents and >131 issued O.U.S. patents Claims extending to at least 2040; Multiple pending Proprietary MoSEICTM
Slide 44: 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 45: Thank you
Thank you axsome