Axsome Therapeutics, Inc. · NASDAQ:AXSM
Axsome Therapeutics — Corporate Update (June 2022)
76 slides from Axsome Therapeutics's corporate update, Jun 28, 2022
Slide 1: Sunosi (solriamfetol) Investor Update
Sunosi® (solriamfetol) Investor Update June 28, 2022
Slide 2: Forward Looking Statements & Safe Harbor
Forward Looking Statements & Safe Harbor Certain matters discussed in this presentation are "forward-looking statements". We may, in some cases, use terms such as "predicts," "believes," "potential," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "wil
Slide 3: Sunosi Investor Update: Topics and Presenters
Sunosi® Investor Update Topic Presenter Introduction Mark Jacobson Chief Operating Officer Welcome Herriot Tabuteau, MD Chief Executive Officer Excessive Daytime Sleepiness in Narcolepsy Dr. Richard Bogan, MD, FCCP Associate Clinical Professor at the University of South Carolina School of Medicine a
Slide 4: Opening Remarks
Herriot Tabuteau, MD Chief Executive Officer Opening Remarks
Slide 5: Sunosi Introduction
Sunosi® Investor Update Introduction Sunosi® is the first and only dual-acting dopamine and norepinephrine reuptake inhibitor (DNRI) approved by the FDA to treat EDS in narcolepsy or OSA Synergistic with the rest of Axsome’s late-stage psychiatry and neurology pipeline Strong efficacy profile in cur
Slide 6: Robust, Late-Stage Neuroscience Pipeline
Robust, Late-Stage Neuroscience Pipeline Product Candidate MOA Phase 1 Phase 2 Phase 3 NDA Approved Dual-acting dopamine and norepinephrine reuptake inhibitor (DNRI) NMDA receptor antagonist with multimodal activity MoSEIC™ COX-2 pref. inhibitor + 5-HT1B/1D agonist Highly selective NE reuptake inhib
Slide 7: Excessive Daytime Sleepiness in Narcolepsy
Richard Bogan, MD, FCCP Principal, Bogan Sleep Consultants Associate Clinical Professor University of South Carolina School of Medicine and Medical University of South Carolina Excessive Daytime Sleepiness in Narcolepsy
Slide 8: Narcolepsy Overview
Narcolepsy Overview 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 / hypnopompic hallucinations (sl
Slide 9: Narcolepsy Prevalence
Narcolepsy Prevalence Estimated that between 135,000 to 200,000 people in US have narcolepsy1-4 Widely recognized to be underdiagnosed1-4 ~80% of diagnosed patients receiving treatment3,5 1. National Institute of Neurologic Disorders and Stroke. Narcolepsy Fact Sheet. NIH Publication No. 17-1637. 2.
Slide 10: EDS in Narcolepsy
EDS in Narcolepsy Excessive daytime sleepiness is the most common symptom in narcolepsy Symptom Prevalence Description EDS1,2 100% Often most debilitating symptom Patients unable to stay alert and awake during day Sleep attacks cause an uncontrollable urge to sleep Negatively influences school and w
Slide 11: Current Narcolepsy Treatments
Current Narcolepsy Treatments Excessive Daytime Sleepiness1,2 Wake promoting agents Solriamfetol (Sunosi®) Modafinil / armodafinil Stimulants Methylphenidate Amphetamines Pitolisant (Wakix™) Oxybate (Xyrem™ / Xywav™) Cataplexy1,2 Oxybate (Xyrem™ / Xywav™) Pitolisant (Wakix™) Off-label treatments: Tr
Slide 12: AASM Guidelines Strongly Recommend Solriamfetol in Narcolepsy
AASM Guidelines Strongly Recommend the use of Solriamfetol in Narcolepsy American Academy of Sleep Medicine (AASM) recently published guidelines for treating central disorders of hypersomnolence1 Solriamfetol is listed as a strong recommendation for treating patients with narcolepsy1 Recommendation
Slide 13: TONES 2, TONES 5, and SURWEY Clinical Data
TONES 2, TONES 5, and SURWEY Clinical Data Sunosi (solriamfetol) – EDS in Narcolepsy
Slide 14: TONES Co-Primary Endpoint: MWT Sleep Latency in Narcolepsy
TONES 2 Co-Primary Endpoint: MWT Sleep Latency in Narcolepsy Patients MWT Sleep Latency at Week 12 9.2† LS Mean (SE) Change in Mean Sleep Latency on MWT Week 10.3† 5.6* 1.3 4.7 2.2 9.8† 4.7 2.1 MWT Sleep Latency Over Time Placebo 75 mg 150 mg Solriamfetol 150mg significantly improved wakefulness as
Slide 15: TONES MWT: Effects Maintained Through Hours
TONES 2 MWT: Effects were maintained through 9 hours Trial 1 (1 hr) Trial 2 (3 hrs) Trial 3 (5 hrs) Trial 4 (7 hrs) Trial 5 (9 hrs) LS Mean (SE) Change From Baseline, Minutes MWT through the Day at Week 12 At week 12, solriamfetol 150 mg significantly increased wakefulness as early as 1 hour Effects
Slide 16: TONES Co-Primary Endpoint: ESS Change in Narcolepsy
TONES 2 Co-Primary Endpoint: ESS Change in Narcolepsy Patients Placebo 75 mg 150 mg ESS at Week 12 Solriamfetol 150mg improved ESS scores as early as week 1 and maintained effect through 12 weeks LS Mean (SE) Change From Baseline in ESS Score ESS Over Time Week *P < 0.05 and †P < 0.0001 vs placebo.
Slide 17: Treatment-Emergent Adverse Events (TONES 2)
Treatment-Emergent Adverse Events (TONES 2)1 1. Thorpy MJ, et al. Ann Neurol. 2019; 85(3):359-370. 2. Sunosi® (solriamfetol) [Prescribing Information]. TEAE, n (%) Placebo (n = 59) Solriamfetol 75 mg (n = 59) Solriamfetol 150 mg (n = 59) Any TEAE 27 (45.8) 34 (57.6) 47 (79.7) Serious TEAEs 0 0 1 (1.
Slide 18: TONES Open-Label Phase: ESS Scores Over Time
TONES 5 Open-Label Phase: ESS Scores Over Time (OSA and Narcolepsy)1 Normative range ESS score, Mean ± SD Efficacy was maintained throughout the course of the study as shown by sustained reductions in ESS scores ESS, Epworth Sleepiness Scale; OSA, obstructive sleep apnea; SD, standard deviation; TON
Slide 19: SURWEY: Real World Efficacy in Germany
SURWEY: Real World Efficacy in Germany A retrospective chart review of solriamfetol use in narcolepsy patients in Germany found that improvements in ESS scores were seen regardless of solriamfetol initiation strategy1 Efficacy results and adverse events are consistent with those seen in clinical tri
Slide 20: Excessive Daytime Sleepiness in Obstructive Sleep Apnea
Andrew Krystal, MD Professor of Psychiatry UCSF Weill Institute for Neurosciences San Francisco, CA Excessive Daytime Sleepiness in Obstructive Sleep Apnea
Slide 21: Obstructive Sleep Apnea (OSA) Overview
Obstructive Sleep Apnea (OSA) Overview Obstructive Sleep Apnea (OSA) is a common sleep disorder that results from repeated collapse of the upper airway resulting in a drop in oxygen saturation, and fragmented, non-restorative sleep1 Symptoms include: Excessive daytime sleepiness (EDS) Snoring Interr
Slide 22: Current Treatments for OSA
Current Treatments for OSA Continuous positive airway pressure (CPAP) therapy is the standard of care for the treatment of the upper airway in OSA1 Other airway modalities such as BiPAP and APAP are commonly used2,3 EDS in OSA often persists despite optimized treatment of the upper airway4 Airway th
Slide 23: EDS in OSA is Common, Persistent, and Consequential
EDS in OSA is Common, persistent, and consequential EDS is a prominent symptom of OSA, occurring in occurring in 87.2% of patients by MSLT1 Residual EDS despite adequate CPAP use is reported in 34 - 65% of patients in clinical studies3,4, and 9 - 22% of patients2,5 in population-based studies Mechan
Slide 24: EDS in OSA Estimated Prevalence
EDS in OSA Estimated Prevalence EDS in OSA Estimated Prevalence1 720K Millions of Patients 12M estimated diagnosed EDS in OSA patients in 2016 claims analysis1 Many estimates indicate an even higher prevalence of OSA2,3 5M of patients had claims showing evidence of CPAP use 2.7M of OSA patients esti
Slide 25: Depression is Common in Patients With EDS in OSA
Depression is Common in Patients With EDS in OSA Depression and other comorbidities are common in patients with OSA, particularly those with EDS1 EDS, excessive daytime sleepiness; OSA, obstructive sleep apnea; MDD, major depressive disorder. 1. Gupta MA & Simpson FC. J Clin Sleep Med. 2015;11(2):16
Slide 26: Overlapping of Symptoms in OSA and Depression
Overlapping of Symptoms in OSA and Depression Depression Sadness Anhedonia Agitation Guilt Weight loss OSA Snoring Witnessed apneas Snort arousals OSA and Depression Daytime sleepiness Fatigue/loss of energy Poor concentration Irritability Psychomotor retardation Weight gain Many symptoms of depress
Slide 27: APA Guidelines Recommend Assessing for OSA
Multiple American Psychiatric Association Guidelines Recommend Assessing for OSA “The APA recommends that the initial psychiatric evaluation of a patient include assessment of past or current sleep abnormalities, including sleep apnea”1 “Clinicians should be alert to the possibility of sleep apnea i
Slide 28: TONES Clinical Data: Sunosi – EDS in OSA
TONES 3 Clinical Data Sunosi (solriamfetol) – EDS in OSA
Slide 29: TONES 3: ESS Scores and MWT Sleep Latency in OSA
TONES 3: Solriamfetol Improved ESS Scores and MWT Sleep Latency in OSA Patients *P < 0.05 and ***P < 0.0001 vs placebo. Negative change from baseline on ESS denotes improvement. ESS, Epworth Sleepiness Scale; LS, least squares; mITT, modified intention-to-treat; MWT, multiple sleep latency test; OSA
Slide 30: TONES Post-hoc Analysis: Patients with History of Depression
TONES 3 Post-hoc Analysis: Effects of Solriamfetol in Patients with History of Depression TONES 3 ESS Scores1 P-values are nominal. Note: History of depression was identified by the terms "affective disorder", "depression", "depressed mood", "major depression", "postpartum depression", or "seasonal
Slide 31: TONES 3 Post-hoc Analysis: Concomitant Antidepressant Use
TONES 3 ESS Scores1 TONES 3 MWT Sleep Latency1 ESS Score LS Mean Change From Baseline (SE) n=21 n=8 n=18 n=32 n=93 n=48 n=40 n=95 *** n=21 n=8 n=18 n=32 n=93 n=48 n=40 n=95 *** ** ** *** ** P-values are nominal. Note: History of depression was identified by the terms "affective disorder", "depressio
Slide 32: TONES Rates of Common TEAEs
TONES 2 (Narcolepsy) & TONES 3 (OSA): Rates of Common TEAEs (≥5% in ≥1 Subgroup)1 Narcolepsy OSA History of Depression No History of Depression History of Depression No History of Depression Preferred Term, n (%) Placebo (n=17) Solriamfetol (n=48) Placebo (n=42) Solriamfetol (n=129) Placebo (n=26) S
Slide 33: Overview of Attention Deficit Hyperactivity Disorder (ADHD)
Stephen V. Faraone, PhD Distinguished Professor Departments of Psychiatry & of Neuroscience and Physiology SUNY Upstate Medical University Overview of Attention Deficit Hyperactivity Disorder (ADHD)
Slide 34: The Evidence Base for ADHD is Huge
The Evidence Base for ADHD is Huge The Consensus Statement and other resources are at: www.ADHDevidence.org Faraone S.V. et al. Neuroscience and Biobehavioral Reviews, 2021, 128:789-818.
Slide 35: What is ADHD?
axsome What is ADHD?
Slide 36: Main Features of ADHD Diagnosis
Main Features of ADHD Diagnosis (American Psychiatric Association, 20131 & 2 other sources2,3) The presence of developmentally inappropriate levels of hyperactive-impulsive and/or inattentive symptoms for at least 6 months. Six or more symptoms in one or both domains required for youth Five or more
Slide 37: Changes in Clinical Presentation of ADHD Across the Lifespan
Changes in the Clinical Presentation of ADHD Across the Lifespan Expression of ADHD Symptoms Changes With Time Hyperactivity Easily distracted Makes careless errors Blurts out answers Often interrupts Fails to wait turn Often out of seat Oppositional School failure Inattention Disorganized Fails to
Slide 38: The Real-World Impact of ADHD on Patients
The Real-World Impact of ADHD on Patients Throughout a patient’s lifetime, ADHD can increase the risk of: Other psychiatric disorders, including depression and suicide Educational underachievement Occupational failure Accidents Criminality Social disability Addictions Faraone, S. V. et al. (2015) At
Slide 39: Association between ADHD and Other Disorders
Association between ADHD & other Disorders (Bernardi et al. Psychol Med. 2012 & 6 other sources) Many large epidemiologic and clinical studies show that ADHD often co-occurs with other psychiatric disorders, especially: depression bipolar disorder autism spectrum disorders anxiety disorders oppositi
Slide 40: ADHD is a Common Disorder in Childhood and Adulthood
ADHD is a Common Disorder in Childhood and Adulthood Given the population prevalence of ADHD in youth (5.9%)1 and adults (2.8%)2, we can compute the number of affected in the USA: 4,307,000 youth 7,232,400 adults The prevalence of ADHD has not changed over the past three decades that have data avail
Slide 41: Fronto-striatal and Fronto-parietal Networks in ADHD
Fronto-striatal and Fronto-parietal Networks in ADHD Under-activation of fronto-striatal and fronto-parietal networks consistent with impaired goal-directed executive processes Under-activation of frontal control over the limbic system consistent with the emotional dysregulation seen in ADHD Faraone
Slide 42: Fronto-striatal and Fronto-parietal Networks in ADHD
Lower activation of the ventral striatum in ADHD in anticipation of reward leads to poor executive control over reward regulation Under-activation of ventral attention networks leads to poor executive control of attention to behaviorally relevant external stimuli Fronto-striatal and Fronto-parietal
Slide 43: ADHD Medications and Brain Networks
The medicines that treat ADHD work in the pathways implicated by neuroimaging studies ADHD Medications and the Brain Networks Implicated in ADHD Faraone, S. V. et al. Attention-deficit/hyperactivity disorder. Nat. Rev. Dis. Primers. 2015
Slide 44: Current Treatments for ADHD
axsome Current Treatments for ADHD
Slide 45: Current Treatment Approach to ADHD
Current Treatment Approach to ADHD 50% discontinuation after six months leads to low response rates Faraone, S. V. et al. Attention-deficit/hyperactivity disorder. Nat. Rev. Dis. Primers. 2015.
Slide 46: Medications Approved by FDA for ADHD
Medications Approved by FDA for ADHD FDA-Approved Stimulants Methylphenidate and Amphetamine IR and ER formulations Duration range 4 to 16 hours Liquid and chewable forms available FDA-Approved Non-Stimulants Atomoxetine (NET reuptake inhibitor) Extended-release Viloxazine (NET and DAT reuptake inhi
Slide 47: Magnitude of Treatment Effects
Magnitude of Treatment Effects (Faraone & Antshel, Child Psych Clinics, 2014) Faraone SV, Antshel KM. Child Adolesc Psychiatr Clin N Am. 2014;23(4):965-72. ADHD Treatment Effect Sizes (Larger is Better) Red box indicates statistical significance in meta-analyses
Slide 48: Psychopharmacology of ADHD: Unmet Needs
Psychopharmacology of ADHD: Unmet Needs
Slide 49: Psychopharmacology of ADHD: Unmet Needs
Psychopharmacology of ADHD: Unmet Needs Stimulants Schedule II controlled substances Misuse, abuse and diversion risk Irritability/rebound phenomena; Loss of ‘sparkle’ Non-Stimulants Low effect sizes for all Clonidine and guanfacine: sedation, lack of data in adults with ADHD Atomoxetine: black box
Slide 50: ADHD Summary
ADHD Summary ADHD is a common, impairing disorder in children and adults ADHD is caused by the confluence of many genetic and environmental risk factors Pathophysiologic studies of ADHD implicate dopaminergic and noradrenergic circuits in the brain Many treatments are available, but many unmet needs
Slide 51: Ongoing and Planned Clinical Development of Solriamfetol
Ongoing and Planned Clinical Development of Solriamfetol Amanda Jones, PharmD Senior Vice President Clinical Development
Slide 52: Clinical Development of Sunosi
Clinical Development of Sunosi in ADHD Evaluation of Effect of Sunosi on Cognition Investigator Initiated Trials in Other Indications
Slide 53: Development of Solriamfetol for the Treatment of ADHD
Development of Solriamfetol for the Treatment of ADHD* *Solriamfetol is not approved by FDA to treat ADHD. Safety and effectiveness have not been established. Rationale for Developing Sunosi in ADHD Dopamine (DA) and norepinephrine (NE) have been implicated in the pathophysiology of ADHD1 Sunosi (so
Slide 54: Solriamfetol for ADHD: Planned Clinical Trial
Solriamfetol for ADHD*: Planned Clinical Trial Solriamfetol (titrated to 150 mg once daily) Placebo (once daily) Randomization (1:1) Treatment Period 4 weeks Follow up 1 week Follow up Visit Screening Up to 4 weeks n=100 n=100 Randomized, Double-blind, 4-week, Placebo-controlled Study to Evaluate th
Slide 55: Evaluation of Effects of Sunosi on Cognition in OSA
Evaluation of Effects of Sunosi on Cognition in Patients with EDS with OSA OSA and EDS in OSA are associated with impairments in attention, memory, and executive functions1,2 About 40% of OSA patients (with or without positive airway pressure use) complain of cognitive difficulties3 Cognitive diffic
Slide 56: SHARP Study: Effect of Solriamfetol on Cognition
SHARP Study: Evaluating the Effect of Solriamfetol on Cognition Solriamfetol (titrated to 150 mg) Placebo Placebo Solriamfetol (titrated to 150 mg) Randomization (1:1) Period 1 2 weeks Washout 1 week Period 2 2 weeks Follow up 1 week Follow up Visit Screening Up to 4 weeks Randomized, Double-blind,
Slide 57: Investigator Initiated Trials
Investigator Initiated Trials *Solriamfetol is not approved by FDA to treat conditions under study that are investigational. Safety and effectiveness have not been established. Research Focus Study Type Institution Status Solriamfetol in Binge Eating Disorder* Clinical Lindner Center of HOPE Solriam
Slide 58: Clinical Development Summary
Clinical Development Summary The pharmacology of solriamfetol is relevant to multiple new potential indications Near and intermediate term clinical development milestones for solriamfetol include: SHARP study in cognition results, 3Q 2022 Phase 3 Adult ADHD trial initiation, 4Q 2022
Slide 59: Q&A
axsome Q&A
Slide 60: Commercial Update
Commercial Update Lori Englebert, MBA Executive Vice President Commercial and Business Development
Slide 61: Acquisition Update
Acquisition Update U.S. portion of Sunosi acquisition from Jazz Pharmaceuticals was completed on May 9th Axsome ensured no disruption and patient continuity through the U.S. transition Conveyed sales reps were trained and operational within 48 hours of deal close Axsome is utilizing our first-in-cla
Slide 62: Narcolepsy with EDS U.S. Opportunity
Narcolepsy with EDS U.S. Opportunity U.S. Individuals with Narcolepsy5 2021 100% of narcolepsy patients have EDS ~50% of people living with narcolepsy are undiagnosed Orphan condition ~185 thousand people in the U.S. have Narcolepsy1-4 1. National Institute of Neurologic Disorders and Stroke. Narcol
Slide 63: OSA with EDS U.S. Opportunity
OSA with EDS U.S. Opportunity U.S. Individuals with OSA3 2021 1. Peppard AE, et al. Increased Prevalence of Sleep-Disordered Breathing in Adults. Am J Epidemiol. 2013;177(9):1006-1014.; 2. Seneviratne U, Puvanendran K. Sleep Med. 2004;5(4):339-343.; 3. Symphony Health 2021 Large population with up t
Slide 64: Differentiated Profile with Strong Efficacy and Prescriber Satisfaction
Differentiated Profile with Strong Efficacy and Prescriber Satisfaction 1st and only DNRI approved for EDS in narcolepsy and OSA 82% improvement in wakefulness in OSA vs. 0% for placebo at week 121,3 118% improvement in wakefulness in narcolepsy vs. 5% for placebo at week 122,3 90% of Sunosi-treated
Slide 65: Strong Market Access Position
Strong Market Access Position 96% of Commercial Lives covered Sunosi has broad payer coverage with 96% of commercial lives covered 83% of all lives are covered (commercial plus Medicare and Medicaid) Sunosi has a robust patient support program including prior authorization support and copay assistan
Slide 66: Sunosi Weekly nTRx Performance
Source: Symphony Health nTRx (Normalized TRx) = (Number of pills in a prescription/30) Weekly Prescriptions Since Launch SUNOSI Weekly nTRx Performance July 5, 2019 through June 17, 2022 COVID Restrictions Transition (Mar 28th – May 9th) Sunosi scripts were maintained during the transition period an
Slide 67: Growth Potential for Sunosi in EDS in OSA and Narcolepsy
Growth Potential for Sunosi in EDS in OSA and Narcolepsy is Significant 95,000 7% Patient Share Source: Symphony Health Apr 2022 800,000 2% Patient Share
Slide 68: Psychiatrists Treat the Vast Majority of Specialist-treated Patients
Psychiatrists Treat the Vast Majority of Specialist-treated OSA and Narcolepsy Patients % of OSA and Narcolepsy Patients Treated by Specialists Source: Symphony Health April 2022 40% of patients with OSA treated by psychiatrists are on pharmacotherapy vs. 3% for PCPs % of OSA and Narcolepsy Patients
Slide 69: Commercialization Strategy
Expand opportunity with Psychiatrist by leveraging synergies with AXS-05 MDD targets Target highest potential prescribers Build on strong launch foundation laid in both OSA and Narcolepsy 4 3 2 EDS = excessive daytime sleepiness, OSA = obstructive sleep apnea Commercialization Strategy 1 5 Deplo
Slide 70: Financial Update
Financial Update Nick Pizzie, MBA Chief Financial Officer
Slide 71: Sunosi Performance Since Launch
Sunosi Performance Since Launch Commercial strategy and market potential expected to contribute to continued growth 9.1 8.6 Sunosi Quarterly Net Sales 2019 2020 2021 Q1 2022 US $3.7M $26.7M $51.7M $13.5M EU - $1.6M $6.2M $2.4M Total $3.7M $28.3M $57.9M $15.9M In $M USD 15.9 18.8 15.8 12.1 11.6 8.7
Slide 72: Potential Sunosi U.S. Peak Sales for EDS in Narcolepsy or OSA
Potential Sunosi U.S. Peak Sales for EDS in Narcolepsy or OSA Market Growth through Promotional Efforts Designed to: Increase market share of Sunosi in EDS in OSA Increase market share of Sunosi in EDS in Narcolepsy Disease Education Efforts for HCPs and Patients Designed to: Increase EDS in OSA tre
Slide 73: ADHD Potential U.S. Opportunity
ADHD Potential U.S. Opportunity 1% ≈ $900Mb 103 Million annual ADHD Rx’s dispensed in the U.S.a a2018 U.S. Rx’s for ADHD – adult and pediatric (Source: IQVIA Institute, Medicine Use and Spending in the U.S., May 2019), grown 1.1%/year to estimated Year 5 of potential launch. bResulting potential est
Slide 74: Sunosi Has Substantial Revenue Potential
Sunosi Has Substantial Revenue Potential in Current and Potential Future Indications >$1 billion peak potential with current and potential future indications Growth in EDS EDS in Narcolepsy or OSA Potential New Neuroscience Indications
Slide 75: Sunosi Financial Update
Sunosi Financial Update Wholesaler Acquistion Cost (WAC) and Gross to Net (GTN) WAC - $755 for 75mg and 150mg Tablets (30 days supply) Axsome Sales commenced on deal closing (May 9, 2022) GTN – expected to be approximately 50% (potentially favorable in Q2 2022) Gross Margin Expected to be in the mid
Slide 76: Q&A
axsome Q&A