Ovid Therapeutics Inc. · NASDAQ:OVID
Ovid Therapeutics — Q4 2025 Earnings Presentation
41 slides from Ovid Therapeutics's earnings presentation, Mar 18, 2026
Slide 1: Business and pipeline update
Business and pipeline update March 18, 2026 © Copyright 2026 Ovid Therapeutics
Slide 2: Forward-looking statements
Forward-looking statements This presentation includes certain disclosures by Ovid that contain “forward-looking statements” including, without limitation statements regarding: the reproducibility and durability of any favorable results initially seen to date in clinical trials; the potential therape
Slide 3: New business highlights
New business highlights Pipeline Received regulatory approval to initiate Phase 1 study of 1st- ever, oral KCC2 direct activator, OV4071* 7 mg dose OV329 GABA-aminotransferase inhibitor demonstrated strong safety and tolerability profile, no treatment-related SAEs or AEs All programs advancing on-tr
Slide 4: Focus: Medicines to quell conditions caused by neural hyperexcitability
Focus: Medicines to quell conditions caused by neural hyperexcitability Pipeline of Fundamental Differentiated, universal highly specific biological targets mechanisms of action small molecules implicated in many conditions that may hold broad therapeutic intended to culminate driven by neural hyper
Slide 5: High-value, differentiated pipeline
High-value, differentiated pipeline Programs Potential Opportunity Preclinical Phase 1 Phase 2 Anticipated milestones* OV329, a next-generation GABA-aminotransferase (AT) inhibitor • Phase 2 initiation (Q2 2026) Distinct mechanism of action in FOS with: Phase 2 RCT Drug-resistant adult focal • Compe
Slide 6: OV329, a potential best-in-category anti-seizure medicine
OV329, a potential best-in-category anti-seizure medicine for treatment-resistant & developmental epilepsies © Copyright 2026 Ovid Therapeutics 6
Slide 7: Clinical progress & indication expansion
NEW UPDATES Clinical progress & indication expansion Focal onset seizures 7 mg dose well tolerated with no treatment-related SAEs or AEs in healthy volunteers 5 mg and 7 mg doses deliver target drug exposure in the plasma, favorable tolerability and predictable, linear pharmacokinetics (PK) On track
Slide 8: OV329 offers a potential best-in-category anti-seizure medicine profile
OV329 offers a potential best-in-category anti-seizure medicine profile Quell hyper-excited neurons by Potent, lower dosing optimally tuning synaptic and (est. OV329 human doses: extra-synaptic GABAergic inhibition 5-7 mg vs 2,000-3,000 mg for VGB) Best-in-category tolerability (no treatment-related
Slide 9: OV329: believed to optimally tune GABA & enhance tolerability & efficacy
OV329: believed to optimally tune GABA & enhance tolerability & efficacy Inhibits GABA-AT, the enzyme that degrades the inhibitory neurotransmitter, GABA1 Increases GABA in the synapse & extra-synaptic regions delivering phasic & tonic inhibition2 Creates an inhibitory neural environment that averts
Slide 10: The OV329 opportunity: A universal mechanism for treatment resistant seizures
The OV329 opportunity: A universal mechanism for treatment resistant seizures ONGOING NEW EXPANSION Capsule formulation (adult) Pediatric weight-based Indication Indications Adjunctive therapy for focal onset seizures (FOS) Tuberous sclerosis complex associated seizures (TSC) Infantile spasms (IS)wi
Slide 11: The OV329 opportunity: A universal mechanism for treatment resistant seizures
The OV329 opportunity: A universal mechanism for treatment resistant seizures ONGOING NEW EXPANSION Capsule formulation (adult) Pediatric weight-based Indication Indications Adjunctive therapy for focal onset seizures (FOS) Tuberous sclerosis complex associated seizures (TSC) Infantile spasms (IS)wi
Slide 12: New mechanisms of action are needed in focal onset seizures
New mechanisms of action are needed in focal onset seizures Drug resistant epilepsy (DREs) A crowded treatment landscape and yet— 30 anti-seizure medications >1 in 3 patients live with uncontrolled seizures approved in 15 years, but only 2 with novel mechanisms1 47% of epilepsy patients in the U.S.
Slide 13: 7 mg dose in adults further demonstrates potential favorable safety and tolerability profile
NEW FINDINGS 7 mg dose in adults further demonstrates potential favorable safety and tolerability profile Objective Evaluated safety and tolerability of OV329 higher dosing to potentially expand dosing optionality for future studies and indications Results Demonstrated favorable safety profile at al
Slide 14: OV329 was well-tolerated at all doses, with no treatment-related SAEs
OV329 was well-tolerated at all doses, with no treatment-related SAEs Summary of potentially treatment-related and treatment-emergent AEs reported Dosage Adverse Event OV329 PBO Severity Drug Related Outcome Mild Possibly related Recovered or Headache n=1 n=0 (grade 1) (x2) resolved (x2) 2 mg MAD Mi
Slide 15: No ophthalmic or retinal changes at all doses, including 7 mg
No ophthalmic or retinal changes at all doses, including 7 mg Ophthalmic metric* Result Best corrected visual acuity No treatment-related effects Fundus photography No treatment-related effects Indirect dilated ophthalmoscopy No treatment-related effects Automated threshold visual field perimetry No
Slide 16: OV329 achieves target exposures for pharmacology strategy & potential anti-convulsant activity
OV329 achieves target exposures for pharmacology strategy & potential anti-convulsant activity Dose modeling: Leveraged Strategy Human drug plasma multiple streams of evidence exposure by dose • OV329 human cortical inhibition Target enzyme inhibition for Demonstrated Dose mean exposure & plasma exp
Slide 17: 7 mg results reaffirmed predictable pharmacokinetic profile
7 mg results reaffirmed predictable pharmacokinetic profile aligned with pharmacology strategy • Dose dependent linear increases in AUC and Cmax demonstrated in repeat dosing 1 mg, SAD • Consistent renal clearance after single and 2 mg, SAD 2 mg, MAD repeated dosing 3 mg, SAD 3 mg, MAD 5 mg, SAD • S
Slide 18: OV329 demonstrated dose dependent GABA elevation & cortical inhibition
OV329 demonstrated dose dependent GABA elevation & cortical inhibition OV329 increases inhibition as measured by Significantly increased cortical inhibition the cortical silent period (CSP) exceeding vigabatrin’s inhibition Untreated baseline vs LICI 150 ms change: Undrugged baseline vs post-treatme
Slide 19: Advancing OV329 into a Phase 2 trial & open-label study
Advancing OV329 into a Phase 2 trial & open-label study Strong safety profile with few treatment-related adverse events; no ophthalmic safety findings Demonstrated, competitive cortical inhibition as measured by multiple well recognized biomarkers Inhibitory effects match or exceed therapeutic doses
Slide 20: Next steps in FOS: Phase 2 PoC studies
Next steps in FOS: Phase 2 PoC studies Next steps in FOS: Phase 2 PoC studies Two, parallel PoC studies to confirm anti-convulsant activity and effect size Open label seizure PoC study Phase 2 – Randomized placebo controlled Objective Objective Characterize suppression of photo-paroxysmal Characteri
Slide 21: The OV329 opportunity: A universal mechanism for treatment resistant seizures
The OV329 opportunity: A universal mechanism for treatment resistant seizures ONGOING NEW EXPANSION Capsule formulation (adult) Pediatric weight-based Indication Indications Adjunctive therapy for focal onset seizures (FOS) Tuberous sclerosis complex associated seizures (TSC) Infantile spasms (IS)wi
Slide 22: Unmet need in infantile spasms
Unmet need in infantile spasms Hallmark “Jackknife” seizures Rare, seizure manifestation in the infantile brain • Hallmark jackknife seizures typically occur between 4-7 months of age • Moderate-to-severe developmental disability • Increased mortality • Seizures evolve to Lennox-Gastaut syndrome (LG
Slide 23: Unmet need in tuberous sclerosis complex
Unmet need in tuberous sclerosis complex1 A rare autosomal-dominant disorder of TSC1 and/or TSC2 genes • Upregulation of the rapamycin (mTOR) pathway with subsequent excessive cell growth and proliferation • Benign hamartomas in multiple organ systems, most frequently in brain, skin, kidneys, lungs,
Slide 24: Potential for OV329
Potential for OV329 Profile Potential to increase use if Safety Tolerability approved Therapeutic index No anticipated sedation Infantile spasms Positive ophthalmic Clean profile at high safety record dose (7mg) in adults • 1st line use with Acthar Gel • Prolonged use to mitigate LGS No intramyelini
Slide 25: SABRIL (vigabatrin) sales under-reflect the sizeable market opportunity
SABRIL (vigabatrin) sales under-reflect the sizeable market opportunity Tuberous sclerosis complex (Addressable population 2026: U.S. = ~21K and EU5 = 20K) $90K1 avg Pricing $5 - $20K / year ~$20K/year2 ~$130K / year treatment course Average treatment duration Two courses Annually Annually 9 mos. •
Slide 26: GABA-AT is a proven mechanism in TSC & IS
GABA-AT is a proven mechanism in TSC & IS Proven efficacy Short treatment Copious dosing Onerous Priced for a duration monitoring broad population Infantile spasms • 2nd or 3rd line • Starting dose: 2,000 • On label - risk of • $14K per/mo list price mg – 3,000 mg irreversible blindness • Freedom fr
Slide 27: OV329 holds significant potential in TSC & IS
OV329 holds significant potential in TSC & IS Anticipate earlier-line use, OV329 Peak Sales Potential* longer treatment duration Increased Validated MoA with an duration of improved profile: Earlier line use treatment Enhanced safety, efficacy and tolerability $320M** Ex-US sales Differentiated pedi
Slide 28: OV329 shows compelling anti-convulsant effects in infantile spasms, focal and broad seizure models
OV329 shows compelling anti-convulsant effects in infantile spasms, focal and broad seizure models OV329 reduced seizure score in a mouse Repeat, low doses reduce seizure frequency model of infantile spasms even at low doses1 & duration in animal models of focal seizures2 5 Vehicle (Cohort 2) OV329,
Slide 29: OV329 does not accumulate in the retina like vigabatrin (VGB)
OV329 does not accumulate in the retina like vigabatrin (VGB) No accumulation Of OV329 detected in VGB shown to cause retinal cell dysregulation in < 45 days, the eye or retina, vigabatrin OV329 shows no ocular effects1 accumulates1,2 OV329 3.0 mg/kg vigabatrin 300 mg/kg (Therapeutic dose) (Therapeu
Slide 30: Potential path to registration
Potential path to registration Regulatory interaction 2026 2027 2028 Next steps Phase 2 Pivotal Phase 3 N=60, 8-week study in adults N = 150 – 180; 12-week study in adults FOS PoC Open Label Pivotal Phase 3 N=12-20 N = 150 – 180; 12-week study in adults TSC and IS efforts are additive and non-compet
Slide 31: A potential win for patients, shareholders and Ovid
A potential win for patients, shareholders and Ovid Additive expansion programs in de-risked indications; running parallel with FOS programs Compelling orphan commercial market in population with high need & few treatments Increases Ovid’s mid-term PoC milestones and catalyst cadence Differentiated
Slide 32: OV4071, a potential first-in-class, direct activator of KCC2 for chronic conditions
OV4071, a potential first-in- class, direct activator of KCC2 for chronic conditions © Copyright 2026 Ovid Therapeutics 33
Slide 33: OV4071, cleared for Phase 1 clinical trial
NEW UPDATES OV4071, cleared for Phase 1 clinical trial Potential first-in-class oral KCC2 direct activator, OV4071, cleared for Phase 1 studies On-track to initiate Phase 1 in Australia Intend to initiate proof-of-mechanism ketamine study in H2 2026 Proof-of-concept patient studies to begin in late
Slide 34: Opportunity: OV4071 has broad syndromic psychoses applications
Opportunity: OV4071 has broad syndromic psychoses applications Parkinson’s Alzheimer’s Huntington’s Generalized Lewy Body Rett syndrome disease psychosis & hallucinations Schizophrenia anxiety dementia psychosis agitation & delusions Psychoses Anxiolytic Anti-convulsant © Copyright 2026 Ovid Therape
Slide 35: 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 36: KCC2 is the main regulator of GABA inhibition by maintaining neuronal chloride homeostasis
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 37: OV4071 overview
OV4071 overview Pharmacology Pharmacodynamics • Orally available • Profile comparable to atypical antipsychotic in phenotypic screens • Highly potent: 20x potency over OV350 • Strong effect in hyperlocomotion-induced psychoses models, with comparable activity to marketed agents • 60x half-maximal ef
Slide 38: KCC2 portfolio
NEW UPDATES KCC2 portfolio Initiating OV4071 Established class safety & first-in-human study (FIH) in Q2 2026 potential signal in humans (Tool program findings reported Dec. 2025) Potent, 15 – 17x Manufacturing pharmacodynamic safety margins complete 25 (PD) activity + proof of mechanism and PD stud
Slide 39: The potential of KCC2 direct activation
The potential of KCC2 direct activation A fulcrum in neural excitation/inhibition; Highly selective, precision Expansive therapeutic fundamental small molecules applicability biological target Potential for oral, Preferable tolerability Only confirmed injectable and IV & safety (no sedation) direct
Slide 40: Plan potentially unlocks OV329 full value creating a catalyst rich runway
Funded by Ovid runway Plan potentially unlocks OV329 full value Pivotal and OLE trials that would require funding post PoC Indication expansion with financing creating a catalyst rich runway Funded assuming full exercise of warrants 2026 2027 2028 Program Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Phase 2
Slide 41: www.ovidrx.com
www.ovidrx.com © Copyright 2026 Ovid Therapeutics