Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Investor Presentation (December 2025)
36 slides from Ovid Therapeutics's investor presentation, Dec 18, 2025
Slide 1: Corporate Presentation
Corporate Presentation December 18, 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 the potential therapeutic benefits of Ovid's current or future product candidates and pipeline programs; statements regardi
Slide 3: Medicines to quell conditions caused by neural hyperexcitability
Medicines to quell conditions caused by neural hyperexcitability Foundational Pipeline of highly biological targets specific small molecules implicated in conditions driven by neural intended to culminate in a fully integrated hyperexcitability and which have broad potential neurotherapeutics compan
Slide 4: Focused high-value pipeline with differentiated mechanisms of action
Focused high-value pipeline with differentiated mechanisms of action Indication Programs Preclinical Phase 1 Phase 2 Anticipated milestones opportunities GABA-aminotransferase inhibitor Findings for 7 mg dose safety, tolerability, PK & exposure in healthy volunteers (Q1 2026) Drug-resistant adult fo
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 Validated mechanism Clinical data IP through 2041 of neuronal for seizures, with Phase 1 showed safety, (composition of hyperexcitability, differentiated safety tolerability and matter; assume
Slide 6: New mechanisms of action are needed in epilepsy
New mechanisms of action are needed in epilepsy Drug resistant epilepsy (DREs) A crowded treatment landscape and yet— >1 in 3 patients live with uncontrolled seizures 30 anti-seizure medications (ASM) approved in 15 years, but only 2 with novel mechanisms1 47% of epilepsy patients in the U.S. report
Slide 7: Challenges of current GABA-modulating medicines
Challenges of current GABA-modulating medicines • Vigabatrin has no therapeutic index 1st-generation • Irreversible retinal changes & vision GABA-AT inhibitor Safety had a poor safety loss in some patients • Copious dosing (2 – 3 grams) to profile achieve anti-convulsant effects Significant opportun
Slide 8: OV329 optimally tunes GABA; potentially enhancing tolerability and efficacy
OV329 optimally tunes GABA; potentially enhancing tolerability and efficacy Inhibits GABA-AT, the enzyme that degrades the inhibitory neurotransmitter, GABA 1 Increases GABA in the synapse & extra-synaptic regions delivering phasic & tonic inhibition 2 Creates an inhibitory neural environment that a
Slide 9: OV329 offers a differentiated target product profile
OV329 offers a differentiated target product profile Quell hyper-excited neurons by optimally tuning synaptic and Potent, lower dosing compared to VGB (est. OV329 human doses: 5-7 mg vs 2-3 g for VGB) extra-synaptic GABAergic inhibition Oral, once daily dosing Sustained reduction of focal seizures C
Slide 10: Phase 1 results support rapidly advancing OV329 into a Phase 2a trial
Phase 1 results support rapidly advancing OV329 into a Phase 2a trial Strong safety profile with few treatment-related adverse events; no ophthalmic safety findings Multiple positive biomarker results support inhibitory activity and therapeutic potential Inhibitory effects on par or exceed therapeut
Slide 11: Multiple biomarkers confirm OV329's inhibitory activity
Multiple biomarkers confirm OV329’s inhibitory activity Measure of inhibition • Well accepted biomarkers to study GABAergic drugs • Quantitative clinical measure of cortical inhibition • Most optimal way of measuring inhibitory activities short of seizure trial Comparability relative to established
Slide 12: OV329 showed similar or better pharmacodynamic effects as vigabatrin
OV329 showed similar or better pharmacodynamic effects as vigabatrin in confirmatory biomarker measures Vigabatrin therapeutic dose (50 mg/kg) OV329 3 mg and 5 mg doses TMS metric Measured in healthy volunteers1,2 Measured in healthy volunteers Paired-pulse long-interval Improved ~35% Improved inhib
Slide 13: OV329 demonstrated statistically significant increases in cortical inhibition
OV329 demonstrated statistically significant increases in cortical inhibition exceeding vigabatrin’s inhibition Min. LICI 150 ms change: Undrugged baseline vs post-treatment on day 71,2 inhibition Measured on abductor pollicis brevis Measured on first dorsal interosseus Mean Change in Conditioned ME
Slide 14: OV329 increases inhibition as measured by the CSP
OV329 increases inhibition as measured by the CSP Max. Untreated baseline vs Untreated baseline vs inhibition post-treatment on day 7 post-treatment on day 7 12% 12% 5 MG OV329 10% 10% Δ CSP (ms) 8% 5 MG 8% OV329 6% 6% ** ** p=0.026 p=0.0001 4% 4% Placebo Placebo 2% 2% NS2 NS2 Min. inhibition 0% 0%
Slide 15: OV329 was well-tolerated with no treatment-related SAEs
OV329 was well-tolerated with no treatment-related SAEs1 Phase 1 summary of potentially treatment-related and treatment-emergent AEs1 Dosage Adverse Event OV329 (n=51) PBO Severity Drug Related Outcome Mild Possibly related Recovered or Headache n=1 n=0 (grade 1) (x2) resolved (x2) 2 mg MAD Mild Rec
Slide 16: No ophthalmic safety findings or retinal changes associated with OV329
No ophthalmic safety findings or retinal changes associated with OV329* Ophthalmic metric* Result • Extensive ophthalmic testing closely monitored for clinical and structural Best corrected No treatment related effects (sub-clinical) changes visual acuity • No treatment related effects were noted in
Slide 17: Extensive preclinical characterization of OV329 supports anti-convulsant activity & ophthalmic safety
Extensive preclinical characterization of OV329 supports anti-convulsant activity & ophthalmic safety No accumulation detected in the eye Anticonvulsant activity demonstrated or retina compared to vigabatrin1,2 in 9 chronic & acute seizure models2 • i.v. (ivPTZ) • NMDA-induced infantile spasm • Audi
Slide 18: Proposed Phase 2a trial design in patients with focal epilepsy
Proposed Phase 2a trial design in patients with focal epilepsy Evaluating the safety, tolerability, and anti-seizure effect of OV329 OV329 7 mg Phase 2b Screening n=30 potential registrational & Follow- study Randomization Taper Baseline Up focal onset seizures 2:1 (1 week) (4 weeks) (1 week) N=240-
Slide 19: 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 20: 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 Post-surgical & Substance abuse and adults with drug developmental epileptic peri-operative pain alcohol withdrawal resistant epil
Slide 21: Portfolio of direct activators of potassium chloride cotransporter 2 (KCC2)
Portfolio of direct activators of potassium chloride cotransporter 2 (KCC2) Potentially Potential OV350 (IV) Phase 1 IP through 2046 transformational first-in-class topline readout (Composition of target that is a direct activators showed good safety matter patent - fulcrum rebalance and predictable
Slide 22: KCC2 normalizes inhibitory/excitatory balance in neurons
KCC2 normalizes inhibitory/excitatory balance in neurons and maintains GABA’s inhibitory tone1 “Master switch” for KCC2 protein expression is confined to the CNS neurological disorders Where hyperexcitability is NKCC1 KCC2 KCC3 KCC4 core to pathophysiology High Safety inherent in target • Unlike enz
Slide 23: KCC2 is the main regulator of GABA inhibition
KCC2 is the main regulator of GABA inhibition by maintaining neuronal chloride homeostasis Healthy Neuron Pathological Conditions Regulates chloride gradient Manages the passive inward entry of KCC2 KCC2 Cl− through activated GABA channels Cl- K-Cl K-Cl Cl- GABAAR GABAAR Na-Cl Na-Cl Maintaining low
Slide 24: Activating KCC2: A lynchpin to restore GABA's inhibitory tone
Activating KCC2: A lynchpin to restore GABA’s inhibitory tone in a broad range of CNS conditions Initial intended indications Other indications Psychoses in Parkinson’s disease Neurodegenerative Neurodevelopmental Psychiatric & Other & Lewy body dementia* disorders disorders • Alzheimer’s agitation
Slide 25: Direct activation matters
Direct activation matters Advantages Profile optimization Bind directly to KCC2 transporter—modulating Avoids tolerability & safety issues that have function independently of upstream pathways been associated with other potassium chloride transporters Effective regardless of phosphorylation, brain-
Slide 26: Ovid development-stage portfolio
Ovid development-stage portfolio OV350 OV5000 & OV6000 OV4071 OV4041 (tool program) Series Trial initiation projected Q2 Stage Phase 1 completed 2026 IND est. Q4 2026 Leads confirmed Oral and injectable Oral & injectable Oral & injectable Formulation Intravenous formulations formulations formulation
Slide 27: OV350 is a first-in-class KCC2 direct activator
OV350 is a first-in-class KCC2 direct activator Specificity Penetrance Directly binds to KCC2 (surface plasmon Small molecule (MW<500) resonance and thermal shift assay) Excellent brain penetration No off-target effects on kinases regulating KCC2 activity (PKC, SPAK, WNK) Potential for biomarker OV3
Slide 28: OV350 IV showed a good safety profile in Phase 1
OV350 IV showed a good safety profile in Phase 1 Evaluated as tool program in an exploratory randomized, placebo-controlled, single-ascending dose study • 16 healthy participants (6=active, 2=placebo cohort) • Doses 50 mg and 100 mg administered by IV infusion Showed good safety profile, with no tre
Slide 29: OV350 elicits potent, robust, rapid and reversible antipsychotic activities
OV350 elicits potent, robust, rapid and reversible antipsychotic activities Profile similar to atypical antipsychotic Phencyclidine-induced psychosis (PCP) model in SmartCube® screen • Onset of effect within 15 minutes • OV350 dose-dependently inhibited PCP induced hyperlocomotion • Dose response st
Slide 30: Overview: OV4071, an oral KCC2 direct activator
Overview: OV4071, an oral KCC2 direct activator Pharmacology Pharmacodynamics • Orally available • Strong profile comparable to atypical antipsychotic in phenotypic screens without sedation • Highly potent: 20x potency over OV350 • Strong effect in mouse model of hyperlocomotion induced by • 60x EC5
Slide 31: OV4071: IND-enabling progress
OV4071: IND-enabling progress Selected Key Properties Attribute Status Comments Potency 60x EC50 reached in repeat dosing Highly potent, 20x potency over OV350 Direct activation Highly selective direct activator of KCC2, avoids unintended off-target 0.64 µM downstream regulation of KCC2 PK/PD EC50=4
Slide 32: MK801 schizophrenia psychosis model confirmed OV4071 robust, dose dependent signal
MK801 schizophrenia psychosis model confirmed OV4071 robust, dose dependent signal 5000 Vehicle + MK-801, 0.3mg/kg OV4071, 0.3mg/kg + MK-801, 0.3mg/kg 4000 OV4071, 1mg/kg + MK-801, 0.3mg/kg 3000 OV4071, 3mg/kg + MK-801, 0.3mg/kg Activity (counts/5min) Locomotor activities OV4071, 10mg/kg + MK-801, 0
Slide 33: Mechanistic relevance in initial psychiatric indications
Mechanistic relevance in initial psychiatric indications Neurodegenerative psychoses Schizophrenia • Visual hallucinations, especially in conditions like Parkinson's • Increasing evidence suggests a contributor to executive disease (PD) and schizophrenia, are linked to disruptions in the dysfunction
Slide 34: Strategy for initial KCC2 indication – PD and LBD psychosis
Strategy for initial KCC2 indication – PD and LBD psychosis Risks to mitigate Strategy for 1st indication • Trial population heterogeneity Confirm biological underpinnings of disease pathology (via synuclein assays) and reduce clinical heterogeneity • Endpoint validation and variability (by focused
Slide 35: A large opportunity for OV4071: PD and LBD psychosis
A large opportunity for OV4071: Parkinson’s disease (PD) psychosis and Lewy body dementia (LPD) 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 P
Slide 36: Catalyst-rich pipeline set to deliver over the next 24 months
Catalyst-rich pipeline set to deliver over the next 24 months 2025 2026 2027 Program Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 OV329 PoC: Adults with focal onset seizures Next-generation 7mg HV Phase 2a placebo controlled GABA AT inhibitor Phase 2a open label OV350 (IV) Safety, tolerability First-ever KCC2 direct Pha
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