Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Investor Presentation (March 2025)
30 slides from Ovid Therapeutics's investor presentation, March 2025
Slide 1: Ovid Therapeutics Corporate Presentation March 2025
Ovid Therapeutics Corporate Presentation March 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, rapidly advancing in the clinic
Differentiated pipeline, rapidly advancing in the clinic Indication Programs Preclinical Phase 1 Phase 2 Anticipated milestones1 opportunities OV329 Drug resistant adult epilepsies Phase 1 topline Q3 2025 (includes clinical effect & GABA-aminotransferase (DREs) & developmental target engagement biom
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 patent term hyperexcitability, differentiated safety biomarkers Q3 2025; extensi
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: OV329, a next-generation GABA-AT inhibitor
OV329, a next-generation GABA-AT inhibitor Challenges of current Intended OV329 target Potential to treat GABA-enhancing medicines: product profile* • Short-acting (inhibition not Efficacy and potency • Developmental epileptic sustained) • Durable inhibition and encephalopathies • Sedating anti-conv
Slide 8: Factors contributing to OV329 safety profile differentiation
Factors contributing to OV329 safety profile differentiation OV329 Vigabatrin Molecule Potency IC50: ~0.1 - 0.3 µM IC50: ~60 – 100 µM Reduced exposure (T1/2 ~1.0 Hour) coupled with T1/2 >5.0 Hours Exposure characteristics prolonged PD duration Both R (inactive) & S (active) enantiomers Mechanism of
Slide 9: GABA-AT inhibition a validated mechanism of action
GABA-AT inhibition a validated mechanism of action • GABA is the most common inhibitory neurotransmitter in the central nervous system • GABA aminotransferase (GABA-AT) is responsible for degrading GABA1 • OV329 substantially reduces GABA-AT activity, increasing GABA in the synapse & extra-synaptic
Slide 10: 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 11: 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 12: 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 12
Slide 13: 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 14: 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 15: Biomarker strategy
Biomarker strategy Biological activity Target engagement Consistent response Transcranial magnetic stimulation Magnetic resonance spectroscopy (TMS) (MRS) Increases in the parameters that Increase in GABA in Trend across post-treatment therapeutic dose of VGB increased1,2 the medial parietal lobe pa
Slide 16: Unique MoAs are rewarded in epilepsy - even in a competitive DRE market
Unique MoAs are rewarded in epilepsy - even in a competitive DRE market Substantial opportunity within the DRE segment Generalized seizures Focal / Partial seizures Diagnosed adults with epilepsy Valproate Lamotrigine Carbamazepine Levetiracetam 2.4M adults 1L Topiramate Levetiracetam Oxcarbazepine
Slide 17: 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 18: 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 1st-in-human (assumes patent in neuronal direct activators Q1 2025 term extension) hyperexcitability 9 method-of-use patents © Copyri
Slide 19: 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 20: 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 21: Franchise of KCC2 direct activators in development
Franchise of KCC2 direct activators in development OV350 & OV4071 behave similarly in phenotypic screens OV350 OV4071 OV4041 Oral Oral Formulation Intravenous Intramuscular injection / SubQ Intramuscular injection Psychosis in Parkinson’s disease Generalized anxiety disorder Indication Proof of mech
Slide 22: Entering human trials: OV350
Entering human trials: OV350 Dose dependent exposure Safety & margins Strong activity in psychosis phenotypic & Regulatory approval to initiate Phase 1 disease models Patient dosing Q1 2025 Dose-exposure-PD relationship demonstrated in animals Permissive safety package Behaves like atypi
Slide 23: 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 24: OV350 enables rapid path to patients for oral KCC2 direct activators
OV350 enables rapid path to patients for oral KCC2 direct activators Existing regulatory path Phase 1 demonstrate acceptable safety (for a first-in-class approach) Quantitative EEG exploratory biomarker for translation Potential to establish proof-of-mechanism in patients Initiate OV4071 (
Slide 25: Entering IND-enabling studies: OV4071, an oral direct activator
Entering 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 mo
Slide 26: 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 26
Slide 27: 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 28: 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 29: 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) • Topline data anticipated Q3 2025 (biomarkers for clinical effect and target engagement, safety & tolerab
Slide 30: Thank you
Thank you © Copyright 2025 Ovid Therapeutics 30
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