Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Investor Presentation (June 2017)
33 slides from Ovid Therapeutics's investor presentation, Jun 9, 2017
Slide 1: The Rare Neurological Disorder Company
The Rare Neurological Disorder Company Corporate Presentation June 9, 2017 Exhibit 99.1
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains projections for Ovid Therapeutics Inc. (“Ovid”) and other forward-looking information. Such projections and information are based on assumptions as to future events that are inherently uncertain and subjective. Ovid, its officers, directors or st
Slide 3: To become a leading rare neurological disorder company
To become a leading rare neurological disorder company Develop innovative therapies addressing disease-relevant pathways to transform the lives of patients and families Built on core pillars of value Rare disorders with a genetic basis & novel MOA Patient-centric approach Scalable model Compelling g
Slide 4: Our Company: Industry Innovators
OURCOMPANY Industry Innovators Jeremy Levin, DPhil, MB BChir Chairman & CEO Matthew During, MD, DSc Director, Founder, President & CSO Yaron Werber, MD, MBA Chief Business and Financial Officer & Secretary Amit Rakhit, MD, MBA Chief Medical and Portfolio Management Officer Dirk Haasner, PhD, MPM SVP
Slide 5: Robust Product Pipeline
δ-selective GABAA receptor agonist Angelman Syndrome RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Fragile X Syndrome δ-selective GABAA receptor agonist δ-selective GABAA receptor agonist δ-selective GABAA receptor agonist Oral Adolescent Oral Pediatric Oral Adolescent Oral Adult OV101 OV101 Undisclo
Slide 6: Attractive Market Opportunities
Angelman Syndrome Prevalence: ~1/12,000-20,000 No FDA-approved therapies Attractive Market Opportunities OV101 OV935 Dravet Syndrome Incidence: ~1/15,700-20,900 live births No FDA-approved therapies Tuberous Sclerosis Complex Incidence: ~1/6,000 live births Prevalence: ~50,000 in the US Fragile X Sy
Slide 7: Our Accomplishments & Key Milestones
2015 2016 2017+ In-licensed OV101 from Lundbeck Recruited experienced team Completed ~$75m series B financing Initiated manufacturing validation & fill/finish activities Submitted IND for adult AS Phase 2 STARS trial & adolescent AS/FXS Phase 1 PK trial with OV101 Granted Method of Use for OV101 Gra
Slide 8: OV101 Potentially First-in-Class Therapeutic for Angelman & Fragile X Syndromes
OV101 Potentially First-in-Class Therapeutic for Angelman & Fragile X Syndromes
Slide 9: OV101 δSEGA for Angelman and Fragile X Syndromes
Potent, δ-selective, extrasynaptic GABAA receptor agonist Validated by clinical data in >4,000 adults with insomnia (approximately 950 patient-years of exposure) Demonstrated ability to restore tonic inhibition and address several key manifestations of AS/FXS Phase 2 STARS trial recruiting adult AS
Slide 10: Target Neurodevelopment Disorders: Genetics and Clinical Manifestation
Target Neurodevelopment Disorders: Genetics and Clinical Manifestation Angelman Syndrome Monogenetic trinucleotide repeat disorder in the FMR1 gene Intellectual disability, behavior and language impairments Anxiety and repetitive behaviors No FDA-approved therapies available Current options limited
Slide 11: Tonic Inhibition is Disrupted in Neurological Disorders
TONIC INHIBITION CURRENTS Key mechanism in deciphering excitatory & inhibitory neurological signals correctly Mediated by the δ-subunit containing GABAA receptors located extrasynaptically Impaired tonic inhibition linked to Angelman and Fragile X syndromes Restoring tonic inhibition improves common
Slide 12: Reduced GABA Levels Lead to Loss of Tonic Inhibition in AS and FXS Models
Fragile X Syndrome (FXS) FMRP Deficiency2 Reduced expression of GAD65/67 Decreased GABA synthesis Decreased GABA release 1Ubiquitin protein ligase E3A (UBE3A) 2Fragile X Mental Retardation Protein (FMRP) Reduced GABA Levels Lead to Loss of Tonic Inhibition in AS and FXS Models Angelman Syndrome (AS)
Slide 13: Distinct Pharmacological Profile
δ-containing extrasynaptic GABAA receptor OV101 (Gaboxadol) Distinct Pharmacological Profile We believe OV101 provides differentiated profile over current options Triggers tonic inhibitory signaling Potentiates tonic inhibition at nM concentrations4,5 Activity in the presence of low GABA concentrati
Slide 14: OV101 Restored Tonic Inhibition & Improved Motor Behavior in AS Mouse Model
AS OV101 Restored Tonic Inhibition & Improved Motor Behavior in AS Mouse Model Animal model for AS developed1: UBE3A gene-knockout mouse model Replicates impaired motor function of Angelman Syndrome In AS mouse model, OV1011: Restored tonic inhibition in cerebellar cells Improved gait and balance on
Slide 15: OV101 Restored Tonic Inhibition & Improved Motor Behavior in FXS Mouse Model
FXS OV101 Restored Tonic Inhibition & Improved Motor Behavior in FXS Mouse Model Fmr1 gene knockout mouse model of FXS: Electrophysiological studies show loss of tonic inhibition in the amygdala Mice are hyperactive, show increased sensitivity to “audiogenic” seizures, and reduced performance on lea
Slide 16: OV101 Established Safety Profile
SAFETY Lundbeck & Merck developed gaboxadol for primary insomnia through Phase 3 trials Gaboxadol improved sleep architecture with minimal next day residual effects Over 4,000 adults treated in clinical trials (approximately 950 patient-years of exposure) Well-tolerated in Phase 2 and 3 insomnia tri
Slide 17: OV101 Clinical Development in Angelman and Fragile X Syndromes
OV101 Clinical Development in Angelman and Fragile X Syndromes Phase 2 Adult Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Trial of OV101 Inclusion N=75, age 18-49 years Diagnosis of AS Exclusion Poorly controlled seizures Concomitant disease that would limit part
Slide 18: OV935 Our Approach to Rare Epileptic Encephalopathies
OV935 Our Approach to Rare Epileptic Encephalopathies
Slide 19: OV935 Novel CH24H Inhibitor for Epileptic Encephalopathies
Epileptic Encephalopathies Target rare epileptic disorders typically diagnosed in childhood Commence in adults with a focus to move to adolescent and pediatric patients Potent (nM), highly selective, cholesterol 24-hydroxylase inhibitor (CH24H) Anti-seizure activity across several epileptic animal m
Slide 20: Epileptic Encephalopathies: A Significant Unmet Need
No FDA-approved therapies available Seizures tend to be refractory to traditional anti-epileptic treatments 80% have mutated SCN1A gene 1-4% of childhood epilepsies Seizures do not respond well to approved anti-epileptic treatments Some individuals have de novo mutations, including SCN2A gene 60-90%
Slide 21: OV935 An Innovative Approach to Epilepsy
Tissue Slice Slice Tissue CH24H knock-out Wild type Potential first-in-class CH24H inhibitor, a target for epilepsy Highly selective and potent (nM) Favorable pharmacology CH24H predominantly expressed in brain & has a central role in cholesterol homeostasis Preclinical data demonstrated that inhibi
Slide 22: OV935 Anti-Epileptic Effects are Consistent Across Preclinical Models
OV935 Anti-Epileptic Effects are Consistent Across Preclinical Models Fring’s audiogenic seizure model SCN1A knock-in model of Dravet Syndrome APP/PS1 transgenic mouse model Mouse model of TMEV infection Pentylenetetrazol (PTZ) Kindling model Reduction in audiogenic seizures Increased temperature th
Slide 23: OV935 Shows Encouraging Activity in Acute Seizure Induction Models
OV935 Shows Encouraging Activity in Acute Seizure Induction Models Fring’s audiogenic seizure model OV935 treatment resulted in a dose-dependent reduction in seizures * p < 0.05 ** p < 0.01 *** p < 0.001 SCN1A+/- knock-in mouse model of Dravet Syndrome OV935 increased temperature threshold for hyper
Slide 24: OV935 Shows Activity in Spontaneous Seizure Models
P < 0.05 P < 0.05 OV935 Shows Activity in Spontaneous Seizure Models Mouse viral encephalitis (TMEV) inflammation-induced seizure model OV935 reduced total number of seizures and number of stage 4/5 seizures APP/PS1 transgenic mouse model show electrographic seizures, increases seizure frequency, an
Slide 25: OV935 Delayed Seizure Development in the PTZ-induced Kindling Mouse Model
TOTAL SEIZURE BURDEN OV935 Delayed Seizure Development in the PTZ-induced Kindling Mouse Model Mice dosed daily with OV935 demonstrated a significant delay in seizure development Control OV935 10 9 8 7 6 5 4 3 2 1 0 Cumulative seizure score p < 0.01 **
Slide 26: Four Phase 1 Trials Completed
Design Phase 1, randomized, double blind, placebo controlled, single ascending dose Subjects 48 Dosage 15-1,350mg QD Design Phase 1, randomized, double-blind, placebo controlled, multiple ascending dose Subjects 40 Dosage 100-600mg QD, and 300mg BID; 14 days Design Open-label, non-randomized Subject
Slide 27: Dose Dependent Reduction in 24HC Levels Seen in Phase 1
Dosing (days) % of Baseline plasma 24HC conc. Dose Dependent Reduction in 24HC Levels Seen in Phase 1 OV935 led to a dose dependent decrease in 24HC levels Correlation between plasma 24HC and enzyme occupancy (PET) may inform dose selection
Slide 28: Finance & Business Development
FINANCE & BUSINESS DEVELOPMENT Comprehensive Approach to Secure Early to Late Stage Assets
Slide 29: Business Development Strategy
Epileptic Encephalopathy Neuro- developmental Disorders Sleep Behavior Cognition Motor Seizures Seizures Behavior Cognition Motor Business Development Strategy Late stage preclinical to clinical stage assets Angelman Syndrome Fragile X Syndrome Tuberous Sclerosis Complex Lennox-Gastaut Syndrome Drav
Slide 30: OV101 Agreement with Lundbeck
OURPARTNER OV101 Agreement with Lundbeck Financial Terms with Lundbeck March 2015 exclusive license secured by Ovid Global rights Extensive patents Substantial active pharmaceutical ingredient (API) amounts Attractive financial terms to Ovid ~$4M of upfront equity to Lundbeck Up to $181M due to Lund
Slide 31: A Novel OneTeam Collaboration with Takeda
COLLABORATION A Novel OneTeam Collaboration with Takeda Global OneTeam Global, equal development and commercialization collaboration for OV935 in Epileptic Encephalopathies (EE) Joint collaboration and governance driven by OneTeam approach for development and commercialization Ovid leads clinical de
Slide 32: Investment Highlights
INVESTMENTHIGHLIGHTS VALUE TEAM VALIDATED EXECUTION OPPORTUNITY Potential to create significant value for patients & stockholders Building leading company in rare neurological disorders ~$155M in equity financing Highly experienced management team Deep expertise in translational science, drug evalua
Slide 33: Y The Rare Neurological Disorder
Y The Rare Neurological Disorder Company THERAPEUTICS BOLD M E D | Ci N E Corporate Presentation 2017
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