Axsome Therapeutics, Inc. · NASDAQ:AXSM
Axsome Therapeutics — Investor Presentation (November 2019)
30 slides from Axsome Therapeutics's investor presentation, Nov 7, 2019
Slide 1: Axsome Therapeutics November 2019
NASDAQ: AXSM November 2019 © Axsome Therapeutics, Inc.
Slide 2: 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 3: Addressing serious CNS disorders with novel therapeutics
3 Axsome is addressing serious CNS disorders, where current treatment options are limited or inadequate, by creating novel therapeutics to improve the lives of patients. © Axsome Therapeutics, Inc.
Slide 4: Our Technologies Overview
4 Our Technologies Overview © Axsome Therapeutics, Inc. Enabling new and innovative medicines to treat CNS conditions Chemical Synthesis & Analysis Metabolic Inhibition Chiral & Formulation Chemistry MoSEIC Delivery ™
Slide 5: Our CNS Candidates and Pipeline Overview
• Four differentiated clinical-stage product candidates targeting serious CNS conditions. • Patent protection to 2034-2036, worldwide rights. 5 Our CNS Candidates and Pipeline Overview © Axsome Therapeutics, Inc. Ongoing Ongoing Product Candidate Preclinical Phase 1 Phase 2 Phase 3 AXS-05 (DM + BUP)
Slide 6: AXS-05 novel therapy for CNS disorders
Novel therapy for CNS disorders: • Treatment Resistant Depression (TRD) • Major Depressive Disorder (MDD) • Agitation in Alzheimer’s Disease (AD) • Smoking Cessation 6 © Axsome Therapeutics, Inc. (dextromethorphan/bupropion) modulated delivery tablet AXS-05
Slide 7: AXS-05: Novel Multimodal Therapy for CNS Disorders
7 AXS-05: Novel Multimodal Therapy for CNS Disorders © Axsome Therapeutics, Inc. AXS-05 Abbreviations: σ-1 = Sigma-1; DAT = Dopamine Reuptake Transporter; nACh = Nicotinic Acetylcholine Receptor; NMDA = N-methyl-D-aspartate; NET = Norepinephrine Reuptake Transporter; SERT = Serotonin Reuptake Transp
Slide 8: AXS-05: Mechanisms of Action
8 AXS-05: Mechanisms of Action © Axsome Therapeutics, Inc. AXS-05 Relevant Indications Multimodal Activity AXS-05 Mechanisms Related Agents NMDA Receptor Antagonist • Ketamine • Memantine (Namenda®) Sigma-1R Agonist • Fluvoxamine (Luvox®) • Donepezil (Aricept®) Norepinephrine Reuptake Inhibitor • Du
Slide 9: CNS Disorders: Phase 1 Results AXS-05
28 1686 0 1700 DM 60 mg DM 60 mg + BUP 150 mg (AXS-05) AUC 0 - 12 ng*hr/mL 9 CNS Disorders: Phase 1 Results © Axsome Therapeutics, Inc. AXS-05 P<0.0001 Dextromethorphan AUC Abbreviations: DM = Dextromethorphan; BUP = Bupropion. Axsome data on file. 0 40 80 120 160 0 4 8 12 16 20 24 28 32 36 Concentr
Slide 10: CNS Disorders: Depression Overview
10 CNS Disorders: Depression Overview © Axsome Therapeutics, Inc. AXS-05 1. National Survey on Drug Use and Health (NSDUH). (2017). 2. Rush AJ, et al. Am J Psychiatry 2006;163:1905-1917. 17.3M patients in the U.S.1 Product Candidate Preclinical Phase 1 Phase 2 Phase 3 AXS-05 (DM + BUP) Treatment Res
Slide 11: Results of ASCEND Trial in MDD
11 CNS Disorders: Depression Results of ASCEND Trial in MDD AXS-05 © Axsome Therapeutics, Inc. -22 -20 -18 -16 -14 -12 -10 -8 -6 -4 -2 0 Baseline 1 2 3 4 5 6 MADRS Total Score Change from Baseline Week AXS-05 (45 mg DM/105 bupropion) modulated delivery tablet, BID, n=43 Bupropion (105 mg), BID, n=37
Slide 12: CNS Disorders: TRD Phase 3 Design
12 CNS Disorders: TRD Phase 3 Design © Axsome Therapeutics, Inc. AXS-05 BUP = Bupropion; DM = Dextromethorphan. Bupropion AXS-05 (BUP + DM) Period 1, Open-label (6 weeks) Period 2, Double-blind (6 weeks) 1:1 randomization of inadequate responders Bupropion N=250 A Phase 3 trial to assess the efficac
Slide 13: CNS Disorders: MDD Phase 3 Design
13 CNS Disorders: MDD Phase 3 Design © Axsome Therapeutics, Inc. AXS-05 BUP = Bupropion; DM = Dextromethorphan. Placebo AXS-05 (BUP + DM) Screening Double-blind (6 weeks) Arm A (n=150) Arm B (n=150) • Assessments: Montgomery-Asberg Depression Rating Scale (MADRS), safety parameters, other clinician-
Slide 14: CNS Disorders: Agitation in AD Overview
14 CNS Disorders: Agitation in AD Overview © Axsome Therapeutics, Inc. AXS-05 • Agitation seen in approximately 70% of AD patients.2 – Emotional distress, aggressive behaviors, disruptive irritability, disinhibition, and increased caregiver burden.4 • Associated with3,4: – Accelerated cognitive decl
Slide 15: Agitation in AD Phase 2/3 Design
15 CNS Disorders: Agitation in AD Phase 2/3 Design © Axsome Therapeutics, Inc. AXS-05 A Phase 2/3 trial to assess the efficacy and safety of AXS-05 in the treatment of Agitation in AD. • Primary Endpoint: Cohen-Mansfield Agitation Inventory (CMAI). • Key Inclusion Criteria: – Diagnosis of probable A
Slide 16: CNS Disorders: Smoking Cessation Overview
16 CNS Disorders: Smoking Cessation Overview © Axsome Therapeutics, Inc. AXS-05 • 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 • Positive Phase 2 trial results (Du
Slide 17: AXS-07 novel therapy for migraine
Novel therapy for: •Migraine 17 (MoSEIC™ meloxicam/rizatriptan) AXS-07 © Axsome Therapeutics, Inc.
Slide 18: AXS-07: MoSEIC Meloxicam + Rizatriptan for Migraine
18 AXS-07: MoSEIC™ Meloxicam + Rizatriptan for Migraine © Axsome Therapeutics, Inc. AXS-07 • AXS-07 incorporates dual mechanisms of action, rapid absorption of MoSEIC™ meloxicam, and extended half-life. – Meloxicam is a new molecule for migraine enabled by MoSEIC delivery. – Rizatriptan is a potent
Slide 19: Migraine: AXS-07 Phase 1 Results
19 Migraine: AXS-07 Phase 1 Results © Axsome Therapeutics, Inc. AXS-07 • Therapeutic AXS-07 MoSEIC™ meloxicam concentrations reached in 17 minutes. • Maximum concentrations of AXS-07 rizatriptan reached in 38 minutes. • MoSEIC meloxicam terminal half-life of 18.2 hours. Sources: Axsome data on file.
Slide 20: AXS-07 Phase 3 Trial in Patients with History of Inadequate Response
20 Migraine: AXS-07 Phase 3 Trial in Patients with History of Inadequate Response AXS-07 N=875 2:2:2:1 randomization Rizatriptan AXS-07 Arm A (n=250) Arm C (n=250) Single dose, following a qualifying migraine (up to 10 weeks) Screening MoSEIC meloxicam Arm B (n=250) Placebo Arm D (n=125) • Co-primar
Slide 21: Migraine-Treating Physician Survey: Likelihood to Prescribe AXS-07
21 Migraine-Treating Physician Survey Likelihood to Prescribe AXS-07 © Axsome Therapeutics, Inc. AXS-07 18% 38% 31% 8% 4% 2% 16% 40% 35% 7% 1% 2% Significantly greater Moderately greater Slightly greater Slightly less Significantly less 87% Likelihood of physicians to prescribe AXS-07 if it is shown
Slide 22: AXS-12 novel therapy for narcolepsy
Novel therapy for: •Narcolepsy 22 (reboxetine) AXS-12 © Axsome Therapeutics, Inc.
Slide 23: CNS Disorders: Narcolepsy Overview
Product Candidate Preclinical Phase 1 Phase 2 Phase 3 AXS-12 (Reboxetine) Narcolepsy; U.S. Orphan Designation • Debilitating sleep disorder characterized by excessive daytime sleepiness (EDS) and cataplexy. • Limited treatment options – Only one approved agent for cataplexy. – Most currently approve
Slide 24: CNS Disorders: Narcolepsy AXS-12 Effects in Mouse Model
• Reboxetine dose-dependently reduced the number of narcoleptic episodes in hypocretin (orexin)-deficient mice (P<0.0001) 24 CNS Disorders: Narcolepsy AXS-12 Effects in Mouse Model AXS-12 Number of narcoleptic episodes Total duration of narcoleptic episodes Adapted from Schmidt et al. Behav Brain Re
Slide 25: CNS Disorders: Narcolepsy Phase 2 Design
25 CNS Disorders: Narcolepsy Phase 2 Design AXS-12 © Axsome Therapeutics, Inc. AXS-12 Placebo Screening and Baseline • Primary Endpoint: Frequency of cataplexy attacks • Other Assessments: Measures of other symptoms of narcolepsy including excessive daytime sleepiness. • Key Inclusion Criteria: – Di
Slide 26: Barriers to Entry
26 Barriers to Entry © Axsome Therapeutics, Inc. Corporate MoSEIC™ Meloxicam (AXS-07, AXS-06) AXS-05 AXS-09 33 Issued U.S. Patents 6 Issued O-U.S. Patents Claims extending to 2034 >45 pending Proprietary Manufacturing Drug Product Formulation 17 issued U.S. Patents 2 issued O-U.S. Patent Claims exte
Slide 27: Our Team
27 Our Team © Axsome Therapeutics, Inc. Corporate Management Herriot Tabuteau, MD Founder & CEO Nick Pizzie, CPA, MBA Chief Financial Officer Dave Marek Chief Commercial Officer Cedric O’Gorman, MD, MBA SVP, Clinical Development & Medical Affairs Mark Jacobson, MA SVP, Operations Board of Directors
Slide 28: Key Financial Information
28 Key Financial Information © Axsome Therapeutics, Inc. Corporate • Financial guidance: Cash anticipated to fund operating requirements into the second quarter of 2021. As September 30, 2019 Cash: $43.6 Million Debt (Face Value): $20.0 Million Common Shares Outstanding: 34.5 Million Options and War
Slide 29: Clinical Milestones Overview
29 Clinical Milestones © Axsome Therapeutics, Inc. Overview Product Candidate Indication 2019 2020 AXS-05 (DM + BUP) TRD ● STRIDE-1 topline results (1Q 2020) AD Agitation ● ADVANCE-1 topline results (1H 2020) MDD ✓ ASCEND topline results ✓ FDA Breakthrough Therapy Designation ✓ GEMINI trial start ●
Slide 30: For more information, please contact
For more information, please contact Mark Jacobson SVP, Operations 212-332-3243 mjacobson@Axsome.com axsome.com
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