Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Investor Presentation (June 2025)
30 slides from Ovid Therapeutics's investor presentation, June 2025
Slide 1: Ovid Therapeutics Corporate Presentation June 2025
Ovid Therapeutics Corporate Presentation June 2025 © Copyright 2025 Ovid Therapeutics
Slide 2: Forward looking statements
Forward looking statements This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 including, without limitation, the potential therapeutic benefits of Ovid’s current or future product candidates and pipeline programs; Ovid’s
Slide 3: Our Focus
OUR FOCUS Foundational biological targets Precision small molecules highly specific targeted small molecules intended to implicated in neuronal hyperexcitability with culminate in a fully integrated neurotherapeutics broad potential therapeutic utility company with multiple clinical-stage programs a
Slide 4: Differentiated pipeline, with multiple near-term milestones
Differentiated pipeline, with multiple near-term milestones Indication Programs Preclinical Phase 1 Phase 2 Anticipated milestones* opportunities OV329 Drug resistant adult epilepsies Phase 1 topline Q3 2025 (includes biomarkers of GABA-aminotransferase (DREs) & developmental target engagement and p
Slide 5: OV329: A next-generation, potential best-in-class GABA-AT inhibitor
OV329: A next-generation, potential best-in-class GABA-aminotransferase (AT) inhibitor Precision inhibition of Validated mechanism Clinical results IP through 2041 neuronal for seizures, with Phase 1 safety and (assumes five year hyperexcitability, differentiated safety biomarkers Q3 2025; patent te
Slide 6: A significant unmet need for a safe and well-tolerated GABA-AT inhibitor
A significant unmet need for a safe and well-tolerated GABA-AT inhibitor 50 million 30% are treatment resistant living with epilepsy across the globe1 meaning failure of two or more drugs3 47% of epilepsy patients 2.4 million in the U.S. report poly- adults living with epilepsy in the US2 pharmacy u
Slide 7: Unmet need in drug-resistant epilepsies & OV329 opportunity
Unmet need in drug-resistant epilepsies & OV329 opportunity Challenges of existing Intended OV329 target product profile* GABA-enhancing medicines • Short-acting Optimally tuned inhibition at low doses • Inhibition not sustained • Durable inhibition and anti-convulsant activity • Sedating Sustained
Slide 8: Broader lifecycle opportunity for a safe GABA-AT inhibitor is significant
Broader lifecycle opportunity for a safe GABA-AT inhibitor is significant Initial intended indication Potential follow-on indications Focal seizures in Focal seizures in adults with drug Post-surgical & Substance abuse and developmental epileptic resistant epilepsy peri-operative pain alcohol withdr
Slide 9: OV329 rationally designed to deliver potential best-in-class profile
OV329 rationally designed to deliver potential best-in-class profile OV329 Vigabatrin Molecule Potency (IC50)1 ~0.1 - 0.3 mM ~60 – 100 mM Exposure T1/2 ~1.0 Hour with prolonged PD duration T1/2 >5.0 Hours Mechanism of enzyme Electrostatic (irreversible) Covalent modification of GABA-AT (irreversible
Slide 10: Mechanism of action
Mechanism of action • Validated mechanism of action • Inhibits GABA-AT, the enzyme that catabolizes the inhibitory neurotransmitter, GABA • Increases GABA in the synapse & extrasynaptic regions • Believed to optimally tune GABA to create an inhibitory neural milieu 1. Silverman RB. Chem Rev. 2018 Ap
Slide 11: OV329 Phase 1 trial design
OV329 Phase 1 trial design Establish safety & tolerability of GABA-AT inhibitor OV329 Exploratory biomarkers measuring biological Endpoints effect and target engagement • Pharmacokinetics • Transcranial magnetic stimulation (TMS) • Safety and tolerability • Magnetic resonance spectroscopy (MRS) • Op
Slide 12: OV329 has differentiated PK, PD & ocular profile in preclinical findings
OV329 has differentiated PK, PD & ocular profile in preclinical findings Penetrates the brain, as it is found in animal plasma and brain tissues1 Anti-convulsant activity across an extensive range of seizure models >100x more potent than vigabatrin in animal studies1 Undetectable in the reti
Slide 13: Anti-convulsant activity demonstrated in 9 seizure models
Anti-convulsant activity demonstrated in 9 seizure models Seizure reduction seen in chronic & acute seizure models 1. Ovid data on file ©2025 Ovid Therapeutics | All Rights Reserved 13
Slide 14: Differentiated safety in retinal model required by regulators
Differentiated safety in retinal model required by regulators OV329 3.0 mg/kg vigabatrin 300 mg/kg Anti-convulsant activity in 9 (Therapeutic dose ) (Therapeutic dose) animal seizure models • Represent a wide range of seizures • Clinically active at low doses Ocular safety profile differentiation •
Slide 15: Head-to-head ocular accumulation study of OV329 versus vigabatrin
Head-to-head ocular accumulation study of OV329 versus vigabatrin Lack of retinal accumulation of OV329 supports differentiated ocular safety profile vs. vigabatrin1 OV329 VIGABATRIN Mean Plasma and Tissue Concentration 5.0 mg/kg/day Mean Plasma and Tissue Concentration at 80.0 mg/kg/day Concentrati
Slide 16: Comprehensive biomarker strategy evaluating OV329
Comprehensive biomarker strategy evaluating OV329 Magnetic resonance spectroscopy Transcranial magnetic stimulation Electroencephalogram Tool (MRS) (TMS) (EEG) Cortical excitability and Brain waves linked Measures Neurotransmitter concentrations brain circuit function to inhibitory (GABAergic) activ
Slide 17: Unique, well-tolerated MoAs are rewarded in epilepsy, even in a competitive DRE market
Unique, well-tolerated MoAs are rewarded in epilepsy, even in a competitive DRE market Voltage-gated Synaptic Other / Mixed Key: GABAergic Substantial opportunity within the DRE segment Ion Channel Target Target / Unknown Focal / Partial seizures Diagnosed adults with epilepsy 2.4M adults Carbamazep
Slide 18: Continued unmet need across a range of rare epilepsies
Continued unmet need across a range of rare epilepsies; limited treatment options indicated for LGS, TSC, CDD with drug resistant seizures Substantial opportunity within the DEE segment TSC LGS CDD Overall U.S. developmental Vigabatrin Valproate Valproate epileptic encephalopathies (DEE) 1L populati
Slide 19: Portfolio of unique KCC2 direct activators
Portfolio of unique KCC2 direct activators Potassium chloride cotransporter 2 (KCC2) Fundamental target Potential OV350 IP through 2046 “master switch” first-in-class initiated Phase 1 (assumes five year in neuronal direct activators Q1 2025 patent term extension) hyperexcitability 9 method-of-use p
Slide 20: KCC2 maintains inhibitory/excitatory balance & normalizes neuronal activity
KCC2 maintains inhibitory/excitatory balance & normalizes neuronal activity1 Precise MoA Final “common Impossible to exclusively expressed pathway” “over modulate” “Master switch” in the brain for a broad spectrum of neurological disorders To rebalance network Downstream of many Energetically challe
Slide 21: KCC2 regulates GABA inhibition by maintaining neuronal chloride homeostasis
KCC2 regulates GABA inhibition by maintaining neuronal chloride homeostasis1 Pathological Conditions Healthy Neuron KCC2 regulates chloride gradient Intracellular chloride • Chloride gradient is necessary for GABA-ergic inhibition at the synapse KCC2 KCC2 Manages the passive inward flux of Cl- K-Cl
Slide 22: Franchise of KCC2 direct activators in development
Franchise of KCC2 direct activators in development OV350 & OV4071 behave similarly in phenotypic screens OV350 OV4071 OV4XXX Oral Oral Formulation Intravenous Intramuscular injection / SubQ Intramuscular injection First-in-human potential Psychosis in Parkinson’s disease Generalized anxiety disorder
Slide 23: In Phase 1: OV350
In Phase 1: OV350 Dose dependent exposure Safety & margins Strong activity in psychosis phenotypic & Started Phase 1 dosing Q1 2025 disease models Permissive safety package Dose-exposure-PD relationship demonstrated in animals No observed adverse effect level (NOAEL) in rats & dogs Behav
Slide 24: OV350 elicits potent, robust, rapid and reversible antipsychotic activities
OV350 elicits potent, robust, rapid and reversible antipsychotic activities1 Behaves as atypical antipsychotic Phencyclidine-induced psychosis (PCP) model with a clean profile in SmartCube® • Onset of effect within 15 minutes • Model is characterized by: Confusion, excitation, aggression, paranoia,
Slide 25: OV350 enables 1st in class characterization & rapid path to patients
OV350 enables 1st in class characterization & rapid path to patients for oral KCC2 direct activators Phase 1 demonstrate acceptable safety (for a first-in-class approach) Exploratory quantitative EEG biomarker for translation Enables ability to potentially establish PK/PD relationship & apply
Slide 26: IND-enabling studies: OV4071, an oral direct activator
IND-enabling studies: OV4071, an oral direct activator Potent, dose dependent Safety & margins Large opportunity PK/PD • >20x potency than OV350 • 60x in vivo EC50 reached in • Similar to OV350 repeat dosing • Strong activity in psychosis • Psychosis with Parkinson’s phenotypic & disease models • Ex
Slide 27: OV4071 dose dependent response in schizophrenia psychosis model
OV4071 dose dependent response in schizophrenia psychosis model Uses an NMDAR antagonist (MK801) to elicit dopamine release in the basal ganglia Pharmacodynamic effects of OV4071 1. Ovid data on file ©2025 Ovid Therapeutics | All Rights Reserved 27
Slide 28: OV4071 indication represents a significant unmet need
OV4071 indication represents a significant unmet need Psychoses associated with neuronal synuclein disorders (NSD) Parkinson’s disease psychosis Target population Lewy body dementia psychosis (both NSDs)1 Regulation of chloride balance is lost and GABA inhibitory signaling does not occur normally, K
Slide 29: A large opportunity for OV4071: Parkinson's disease psychosis and Lewy body disease psychosis
A large opportunity for OV4071: Parkinson’s disease psychosis and Lewy body disease psychosis • LBD and PD prevalence growing as baby boomers 2.4M U.S. patients5 age and life span increases (1.4M LBD and 1M PD) • Nearly all LBD patients (75%)1 and up to half of ~1.4M U.S. patients with psychosis sym
Slide 30: Delivering a differentiated and rapidly advancing pipeline
Delivering a differentiated and rapidly advancing pipeline • Potential for significant opportunity with OV329 in adult drug-resistant epilepsies or developmental epileptic encephalopathies (DEEs) • Multiple, unique KCC2 programs enable the potential for optionality with co-development, transactions
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