Axsome Therapeutics, Inc. · NASDAQ:AXSM
Axsome Therapeutics — Investor Presentation (August 2018)
34 slides from Axsome Therapeutics's investor presentation, Aug 8, 2018
Slide 1: Forward-Looking Statements & Safe Harbor
FLS Forward-Looking Statements & Safe Harbor Certain information contained in this presentation may include “forward-looking statements” within the meani ng of the Pri vate Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “conti
Slide 2: Axsome is addressing growing markets with limited treatment options
Axsome is addressing growing markets, where current treatment options are limited or inadequate, by leveraging well-characterized compounds to create nov el therapeutics to meet unmet medical needs and improve the lives of patients. © Axsome Therapeutics, Inc. 3
Slide 3: Our Technologies
Overview Our Technologies Enabling new and innovative medicines to treat CNS conditions MoSEIC™ Delivery Chiral & Formulation Chemistry Metabolic Inhibition Chemical Synthesis & Analysis © Axsome Therapeutics, Inc. 4
Slide 4: Our Candidates and Pipeline
Overview Our Candidates and Pipeline • Five differentiated clinical-stage assets targeting significant and growing markets. • Patent protection to 2034, worldwide rights. ack Granted ack Granted Abbrev iations: BML = Bone Marrow Lesions; BUP = Bupropion; CLBP = Chronic Low Back Pain; DM = Dextrometh
Slide 5: AXS-05 Dextromethorphan + Bupropion
© Axsome Therapeutics, Inc. 6 AXS-05 Dextromethorphan (DM) + Bupropion (BUP) Novel therapy for CNS disorders: • Treatment Resistant Depression (TRD) • Agitation in Alzheimer’s Disease (AD) • Major Depressive Disorder (MDD) • Smoking Cessation
Slide 6: AXS-05 CNS Disorders: Mechanisms of Action
AXS-05 CNS Disorders: Mechanisms of Action Pharmacodynamic Synergy Relevant Indications Related Agents • • Ketamine Memantine (Namenda®) • • Fluvoxamine (Luvox®) Donepezil (Aricept®) • • Duloxetine (Cymbalta®) Venlafaxine (Effexor®) • • • Escitalopram (Lexapro®) Fluoxetine (Prozac®) Sertraline (Zolo
Slide 7: AXS-05 Mechanisms of Action and Relevant Indications
AXS-05 CNS Disorders: Mechanisms of Action and Relevant Indications Pharmacodynamic Synergy Relevant Indications1 Related Agents2 • Memantine (Namenda®) • Donepezil (Aricept®) • Venlafaxine (Effexor®) Relevant DM = Dextrom ethorphan; BUP = Bupropion. Pres ent 1. Indications listed are associated w i
Slide 8: AXS-05 Novel Therapy for CNS Disorders
AXS-05 CNS Disorders: Novel Therapy for CNS Disorders DM Alone DXO Difficult to achieve plasma levels. potential therapeutic DM DM Rapid metabolism AXS-05 (DM + BUP) DXO Inhibits DM metabolism • • • Pharmacokinetic synergy Potential pharmacodynamic synergy Potential efficacy in CNS disorders DM BUP
Slide 9: AXS-05 Phase 1 Results
AXS-05 CNS Disorders: Phase 1 Results Dextromethorphan AUC Dextromethorphan Cmax P<0.0001 P<0.0001 160.0 1700 $' DJLWDWLRQ, 'HSUHVVLRQ* 85 - 883 - 525 - $EQRUPDO ODXJKLQJ/FU\LQJ† † 53 - 0 0.0 DM 60 mg DM 60 mg + BUP 150 mg (AXS-05) DM 60 mg DM 60 mg + BUP 150 mg (AXS-05) DM concentrations associated
Slide 10: AXS-05 TRD Overview
AXS-05 CNS Disorders: TRD Overview • 63% and 44% of MDD patients have inadequate response to initial therapy and second line therapy, respectively.2 • Only 1 approved drug for TRD = unmet medical need. • AXS-05 combines the MOA of 4 distinct anti-depressant drug classes into one novel oral therapeut
Slide 11: AXS-05 TRD Clinical Rationale
AXS-05 CNS Disorders: TRD Clinical Rationale • DM and metabolic inhibitor reduce depressive symptoms in TRD and in AD. Symptom Reduction in TRD Patients Depressive Symptom Reduction in AD Agitation Patients Treated with DM and Metabolic Inhibitor2 DM+Q DM and Metabolic Inhibitor1 Treated with Placeb
Slide 12: AXS-05 TRD Phase 3 Design
AXS-05 CNS Disorders: TRD Phase 3 Design A Phase 3 trial to assess the efficacy and safety of AXS-05 in the treatment of TRD. 1:1 randomization of inadequate responders Period 2, Double-blind (6 weeks) Period 1, Open-label (6 weeks) N=346 (BUP + DM) BUP = Bupropion; DM = Dextrom ethorphan. • Primary
Slide 13: AXS-05 Agitation in AD Overview
AXS-05 CNS Disorders: Agitation in AD Overview • Agitation and aggression seen in approximately 45% of AD patients during 5-year period.3 • Characterized by emotional distress, aggressive behaviors, disruptive irritability, disinhibition, and caregiver burden.4 • Associated with4,5: – – – Accelerate
Slide 14: AXS-05 Agitation in AD Clinical Rationale
AXS-05 CNS Disorders: Agitation in AD Clinical Rationale Change in Agitation/Aggression Scores in AD with DM and Metabolic Inhibitor Quinidine (Q) • Randomized, double-blind, controlled, two-stage trial. – Placebo (n=125), 30 mg quinidine (Q) (n=93), for placebo-DM + 10 stage 1. mg Placebo DM+Q 0.0
Slide 15: AXS-05 Agitation in AD Phase 2/3 Design
AXS-05 CNS Disorders: Agitation in AD Phase 2/3 Design A Phase 2/3 trial to assess the efficacy and safety of AXS-05 in the treatment of Agitation in AD. Screening Double-blind Phase (5 weeks) (BUP + DM) N=435 1:1:1 randomization BUP = Bupropion; DM = Dextrom ethorphan. • Primary Endpoint: Cohen-Man
Slide 16: AXS-05 Smoking Cessation Overview
AXS-05 CNS Disorders: Smoking Cessation Overview • Smoking is single largest cause of preventable death in the U.S.1 • 70% of smokers want to quit and only 3-5% who attempt to quit without assistance are successful for 6-12 months.2 • DM component of AXS-05 significantly reduced nicotine self-admini
Slide 17: AXS-09 (Esbupropion + DM) Overview
AXS-09 CNS Disorders: AXS-09 (Esbupropion + DM) Overview • Esbupropion and DM fixed-dose combination • Esbupropion is the chirally pure S-enantiomer of bupropion. • Phase 1 trial completed: – Pharmacokinetic trial of AXS-09, R-bupropion and dextromethorphan, single-entity S-bupropion, or single-enti
Slide 18: MoSEIC Meloxicam
© Axsome Therapeutics, Inc. 19 MoSEIC™ Meloxicam Novel therapies: • AXS-07 – Migraine • AXS-06 – OA and RA
Slide 19: MoSEIC Meloxicam Overview
MoSEIC™ Migraine, OA and RA: MoSEIC™Meloxicam Overview • MoSEIC meloxicam is a potent, oral, rapidly-absorbed, once-daily, non-opioid, COX-2 preferential, pain therapeutic. • Standard meloxicam has an extended Tmax (4-6 hours) which delays its onset of action.1,2 • Axsome’s MoSEIC (Molecular Solubil
Slide 20: MoSEIC Meloxicam Phase 1 Results
MoSEIC™ Migraine, OA and RA: MoSEIC™Meloxicam Phase 1 Results Meloxicam Tmax after 15 mg Dose Mean Meloxicam Concentrations 2,500 Mobic® 2,000 MoSEIC™ - Oral 1,500 IV (bolus) IV (infusion) 1,000 IM 500 Mobic® - Oral - 0 1 2 3 4 5 0 1 2 3 4 5 6 7 8 910 11 12 14 24 Hours Hours Mobic® (0.5 hour versus
Slide 21: AXS-07: MoSEIC Meloxicam + Rizatriptan for Migraine
MoSEIC™ AXS-07: MoSEIC™ Meloxicam + Rizatriptan for Migraine • Meloxicam is a new molecule for migraine—not currently approved or used for this indication due to prolonged Tmax • MoSEIC delivery enables its use in abortive treatment of migraine – Rapid Tmax of MoSEIC meloxicam is ideal for migraine
Slide 22: AXS-07: Differentiated Clinical Profile for Migraine
MoSEIC™ AXS-07: Differentiated Clinical Profile for Migraine Rapid absorption & onset of action Based on rapid absorption of MoSEIC meloxicam and expected additive effect of AXS-07 components Strong & consistent pain relief Potential for superior efficacy as compared to current treatments based on e
Slide 23: Barriers to Entry
Corporate Barriers to Entry 24 Issued U.S. Patents 2 Issued O-U.S. Patents Claims extending to 2034 >40 pending 2 issued U.S. Patents Claims extending >25 pending to 2036 Proprietary Manufacturing Drug Product Formulation Proprietary Manufacturing Drug Product Formulation MoSEIC™ Meloxicam (AXS-07,
Slide 24: Our Team
Corporate Our Team United Therapeutics Corp. Former President, COO Merck Former CFO Group at UBS Diplomat of the American Board of Herriot Tabuteau, MD Chairman © Axsome Therapeutics, Inc. 25 Management Herriot Tabuteau, MD Founder & CEO Nick Pizzie, CPA, MBA CFO Cedric O’Gorman, MD, MBA SVP, Clinic
Slide 25: Key Financial Information
Corporate Key Financial Information Cash: $20.4 Million Debt (Face V alue)1: $8.1 Million Common Shares Outstanding: 26.3 Million Options and Warrants Outstanding2: 5.0 Million • Financial guidance: Cash anticipated to fund operating requirements into the third quarter of 2019. 1. Book value of $8.5
Slide 26: Clinical Milestones
Overview Clinical Milestones 2020) Abbreviations: AD = Alzheimer’s Disease; BUP = Bupropion; CLBP = Chronic Low Back Pain; DM = Dextromethorphan; DZT = Disodium Zoledronate Tetrahydrate; Eso = Esomeprazole; MDD = Major Depressive Disorder; Mx = Meloxicam; OA = Osteoarthritis; RA = Rheumatoid Arthrit
Slide 27: AXS-02 Disodium Zoledronate Tetrahydrate
© Axsome Therapeutics, Inc. 28 S-02 Disodium Zoledronate Tetrahydrate Novel therapy for chronic pain: • Knee Osteoarthritis (OA) with Bone Marrow Lesions (BMLs) • Chronic Low Back Pain (CLBP) with Modic Changes (MCs)
Slide 28: AXS-02 Chronic Pain: Differentiated Therapy
AXS-02 Chronic Pain: Differentiated Therapy AXS-02 Disodium Zoledronate Tetrahydrate Oral Dose Long-acting Targeted Therapy Non-opioid Nov el Mechanism Mechanisms of Action Inhibits bone-resorbing osteoclasts Downregulates acid-sensing† ion channels Reduces pro-inflammatory cytokine production Anti-
Slide 29: AXS-02 Knee OA with BMLs Overview
AXS-02 Chronic Pain: Knee OA with BMLs Overview MRI: knee side view* • Bone marrow lesions (BMLs) on MRI are associated with pain in knee osteoarthritis (OA).1 • BMLs are regions of increased bone turnover, and reduced mineral density.2,3 • Zoledronic acid inhibits bone resorption and increases mine
Slide 30: AXS-02 Knee OA with BMLs Phase 3 Design
AXS-02 Chronic Pain: Knee OA with BMLs Phase 3 Design A Phase 3 trial to assess the efficacy and safety of AXS-02 in the treatment of pain of knee OA associated with BMLs. Clinical Knee OA Symptom Treatment 1 Study Screening, Baseline Double-blind Phase (24 weeks) Special Prot ocol A ssessment (SPA
Slide 31: AXS-02 CLBP with MCs Overview
AXS-02 Chronic Pain: CLBP with MCs Overview MRI: lumbar side view* • Modic changes (MCs) type 1 (M1) on MRI are associated with chronic low back pain (CLBP).1 • Increased bone turnover on bone scan is seen in M1 lesions.2 • Increased pro-inflammatory cytokines, and vascular density seen in M1 lesion
Slide 32: AXS-06: MoSEIC Meloxicam + Esomeprazole for OA & RA
MoSEIC™ AXS-06: MoSEIC™ Meloxicam + Esomeprazole for OA & RA • AXS-06 is a fixed-dose combination of MoSEIC™ meloxicam and esomeprazole • Being developed to treat OA and RA, and to reduce the risk of NSAID-associated upper GI ulcers • Potentially best-in-class NSAID profile: – – – Oral administratio
Slide 33: For more information, please contact
For more information, please contact Mark Jacobson SVP, Operations 212-332-3243 mjacobson@A xsome.com axsome.com
Slide 34: Thank you
AXSOME THERAPEUTICS Thank you. For more information, please contact Mark Jacobson SVP, Operations 212-332-3243 mjacobson@Axsome.com axsome.com
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