Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Investor Presentation (August 2017)
35 slides from Ovid Therapeutics's investor presentation, Aug 15, 2017
Slide 1: Corporate Presentation August 15, 2017
Corporate Presentation August 15, 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 neurological disorder company
To become a leading neurological disorder company Develop innovative therapies addressing to transform the lives of patients and families Built on core pillars of value Rare disorders with a genetic basis & novel MOA Driven by Business Development Scalable model Compelling growth opportunities Subst
Slide 4: 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 5: Substantial Potential Opportunity
Substantial Potential Opportunity ~100 Orphan Neurological Disorders1 ~1M patients in the US1 Few or No FDA Approved Therapies Multiple assets available for partnering Late Preclinical to Clinical Stage Assets Related Disorders Enable Scalable Model 1Boston Consulting Group and Ovid analysis
Slide 6: Multiple Upcoming Milestones in 2017-'18
OV935 Execution OV101 Announced global collaboration with Takeda for TAK-935/OV935 Initiated Phase 1b/2a trial in adults with developmental and/or epileptic encephalopathies Initiated Phase 2 OV101 STARS trial in adults with Angelman syndrome Initiated Phase 1 OV101 PK trial in adolescents with Ange
Slide 7: Our Team
OUR TEAM 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 Senior Vice President, Gl
Slide 8: OV101 Potential First-in-Class Therapeutic for Angelman & Fragile X Syndromes
OV101 Potential First-in-Class Therapeutic for Angelman & Fragile X Syndromes
Slide 9: Angelman Syndrome: A Significant Unmet Need
Angelman Syndrome A Significant Unmet Need Epidemiology and Treatments Prevalence ~1/12,000 to ~ 1/20,000 ~16,000-27,000 patients in the US Treatment No FDA-approved therapies available Current treatment options limited to symptomatic agents and anti-seizure medications Monogenetic disorder that res
Slide 10: Fragile X Syndrome: A Significant Unmet Need
Epidemiology and Treatments Prevalence ~1/3,600-4,000 males ~1/4,000-6,000 females ~ 68,000 to 85,000 patients in the US Treatment No FDA-approved therapies available Limited to physical, communication, behavior and symptomatic therapy Genetics and Clinical Manifestations Monogenetic trinucleotide (
Slide 11: OV101: Impacts Tonic Inhibition
Potential first-in-class, potent, δ-selective, extrasynaptic GABAA receptor agonist (δSEGA) Demonstrated ability to restore tonic inhibition and improve several key phenotypes in preclinical models of Angelman syndrome (AS) and Fragile X syndrome (FXS) Drug profile validated by clinical data in >4,0
Slide 12: OV101: Compelling new science drove decision to in-license
OV101 Compelling new science drove decision to in-license Gaboxadol (OV101) met endpoints and was well-tolerated in Phase 3 trials Gaboxadol improved sleep architecture versus placebo and relative to Ambien with minimal next day residual effects Over 4,000 adults treated in clinical trials (approxim
Slide 13: δ-selective extrasynaptic GABAA receptor agonist mechanism
δ-containing extrasynaptic GABAA receptor OV101 Potent, oral, δ-selective, extrasynaptic GABAA receptor agonist MOA and activity in the presence of low brain GABA concentration differentiates OV101 over available GABAergic drugs Potentiates tonic inhibition signaling at low nM concentrations1,2 Rest
Slide 14: Reduced GABA Levels Lead to Loss of Tonic Inhibition in AS and FXS
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) Angelman Syndrome (AS) UBE3A Deficiency1 GAT1 does not get ‘tagged’ Increased GAT1 Increased GAB
Slide 15: Multiple domains in AS and FXS show GABAergic hypofunction
Increased seizure susceptibility Domain Observation in AS Preclinical Models GABAergic Signaling and Tonic Inhibition are reduced in AS & FXS Observation in FXS Preclinical Models Audiogenic seizures Hyperexcitability, increased startle response Working memory deficit Abnormal neuronal firing and sy
Slide 16: OV101 Restored Tonic Inhibition in AS Mouse Model
AS OV101 Restored Tonic Inhibition & Improved Motor Behavior in AS Mouse Model AS animal model1: UBE3A gene-knockout mouse model (Ube3am-/p+ ) Replicates impaired motor function of AS Impact in AS mouse model1: Restored tonic inhibition in cerebellar cells Improved gait and balance on rotarod Decrea
Slide 17: OV101 Restored Tonic Inhibition 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 18: OV101 Clinical Development in Angelman and Fragile X Syndromes
OV101 Clinical Development in Angelman and Fragile X Syndromes Phase 2 Data (Expected 2018) 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
Slide 19: OV935 New Approach to Potentially Treat Rare Developmental and/or Epileptic Encephalopathies
OV935 New Approach to Potentially Treat Rare Developmental and/or Epileptic Encephalopathies
Slide 20: Developmental and/or Epileptic Encephalopathies: A Significant Unmet Need
Some individuals have SCN2A mutations Most cases caused by TSC1 or TSC2 mutations Developmental and/or Epileptic Encephalopathies: A Significant Unmet Need Few available therapies Refractory seizures, cognitive, behavioral and neurological deficits OV935 may address underlying pathophysiology via mo
Slide 21: OV935: A Novel Approach for dEE
OV935: A Novel Approach for Developmental and/or Epileptic Encephalopathies (dEE) Potential first-in-class CH24H inhibitor Differentiated mechanism over available anti-seizure drugs Broad activity in preclinical seizure models, including genetic refractory models Commence studies in adults with a fo
Slide 22: CH24H inhibitor: An Innovative Approach to Epilepsy
Tissue Slice Slice Tissue CH24H knock-out Wild type Potential first-in-class CH24H inhibitor, a novel target for epilepsy Highly selective and potent (nM) Favorable pharmacology CH24H predominantly expressed in brain & has a central role in cholesterol homeostasis Preclinical data demonstrates that
Slide 23: OV935 Anti-Epileptic Effects Evident Across Preclinical Models
OV935 Anti-Epileptic Effects Evident 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 threshold
Slide 24: 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 25: 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 26: OV935 Delayed Seizure Development in PTZ Kindling 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 27: 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 28: 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 29: OV935 Phase 1b/2a in Developmental and/or Epileptic Encephalopathies
Inclusion N=~20, age 18-65 years Diagnosis of dEE Treatment-resistant seizures Exclusion Frequent status epilepticus Concomitant disease that would limit participation Primary endpoint Incidence of adverse events Exploratory secondary endpoints PK Parameters 24 S-Hydroxy Cholesterol levels (24HC) We
Slide 30: Finance & Business Development
FINANCE & BUSINESS DEVELOPMENT Comprehensive Approach to Secure Early to Late Stage Assets
Slide 31: Financing Highlights
Financing Highlights ~$155M raised in equity financings to date with broad institutional investor support NASDAQ: OVID Financial History Strong Balance Sheet Shares Outstanding 2014 2015 2017 ~$75 million Series B Financing ~$5 million Series A Financing ~$75 million Initial Public Offering As of Ju
Slide 32: 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 33: A Novel OneTeam Collaboration with Takeda
COLLABORATION A Novel OneTeam Collaboration with Takeda Global OneTeam Global, equal development and commercialization collaboration for OV935 in Developmental and/or Epileptic Encephalopathies (dEE) Joint collaboration and governance driven by OneTeam approach for development and commercialization
Slide 34: 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 35: 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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