STIFEL 2026 HEALTHCARE CONFERENCE - OVID THERAPEUTICS INC

Explicit Guidance:

• No formal quantitative financial guidance (revenue, earnings, margins, capex) or specific cash balance targets provided beyond a qualitative statement that the company has sufficient cash to operate through 2029.

Business-Segment Outlook:

• Opioid/Neurotherapeutics Pipeline: Management anticipates "four to five major data milestones" or "card turnovers" over the next 6 to 12 months.
• OV329 (Epilepsy):
  • Phase 2 Focal Epilepsy: Expected to read out in the second half of next year.
  • Pediatric Tuberous Sclerosis Complex (TSC): Phase 2 open-label study launching this year to assess signal and safety.
  • Photosensitivity Study: Currently running; results expected soon to validate dosing for focal epilepsies.
  • Long-term Safety: Regulatory strategy includes perpetual open-label extension studies following the core Phase 2 to build a robust safety database and avoid retinal toxicity labeling associated with Vigabatrin.
• OV4071 (KCC2 Activator):
  • Phase 1 Completion: Expected to read out in Q1 of next year, including exploratory electrophysiology data.
  • Ketamine Challenge: Planned to be triggered soon during the Phase 1 study to assess brain penetration and mechanism-of-action via biomarkers (EEG, ERP).
  • Acute Schizophrenia: A proof-of-concept (PoC) study for acute schizophrenia is targeted to initiate around the middle of next year.
  • Future Expansion: If PoC signals are met, the program is positioned to expand into broader neurodegenerative psychoses, bipolar mania, Alzheimer's psychosis/delusions, Alzheimer's agitation, and Parkinson's disease psychosis.

Market and Competitive Outlook:

• KCC2 Target: Management positions the KCC2 direct activator mechanism as a "transformative target" with the potential to be "five or six times" more effective than the recently approved co-benefit (COR332/Cobenfi).
• Epilepsy Market: OV329 targets the "holy grail" of a new mechanism that offers better tolerability, once-daily dosing without titration, and no drug-drug interactions, addressing unmet needs in pediatric developmental epileptic encephalopathies where current mechanisms "don't really work very well."
• Competitive Landscape: No other companies currently have a GABA aminotransferase inhibitor (GABA-T) in development for epilepsy, and the only prior competitor (Lundbeck) exited due to compound-specific toxicity; Ovid claims a "clean" safety profile relative to Vigabatrin.

Macro / Industry Outlook:

• Regulatory Environment: Management emphasizes a proactive strategy to secure a "clean" regulatory label for OV329, explicitly aiming to avoid the severe ocular toxicity warnings (retinal degeneration) attached to the reference drug, Vigabatrin.
• Scientific Validation: The outlook relies heavily on the validation of the KCC2 target's broad utility across multiple indications (seizures, pain, psychosis) and the confirmation of biomarkers in humans that predict clinical efficacy, a capability management notes is "not an easy thing to get out of a phase one program."

Strategic Initiatives:

• Clinical Program Advancement: Aggressive sequencing of data readouts to establish Ovid as the definitive "KCC2 company" within the next two years.
• Mechanism Expansion: Leveraging the confirmed efficacy in schizophrenia to drive a broader franchise into various forms of psychosis and neurodegenerative conditions.
• Safety Differentiation: Completion of extensive preclinical "killer experiments" and Phase 1 human safety data (100+ subjects) to de-risk the retinal safety profile of OV329 compared to first-generation GABA-T inhibitors.

Risks and Headwinds to Outlook:

• Clinical Failure: The entire strategy hinges on successful Phase 2 readouts for OV329 (focal epilepsy, TSC) and the upcoming Phase 1/PoC results for OV4071 in schizophrenia; failure in these studies could halt expansion.
• Long-term Safety: Potential for long-term retinal toxicity in OV329 remains a risk, mitigated only by the planned perpetual open-label extension studies.
• Mechanism Translation: While animal models show profound efficacy for OV4071, success must be replicated in human biomarkers and clinical symptom reduction; management notes the need to "make smart decisions about the indications" based on these early insights.
• Dose Selection: The efficacy of OV329 is dose-dependent ("if we're at the right dose"); incorrect dosing could result in suboptimal seizure control.

Management Tone on the Future:

• High Confidence: Management describes the time as "exciting," with the company in the "playoffs," citing "undeniable" and "wildly consistent" data supporting the KCC2 mechanism.
• Strategic Pivot: Expresses a deliberate shift in focus toward schizophrenia due to the strength of the data signal, stating it would be "irresponsible" not to pursue this path.
• Differentiation: Emphasizes the unique safety profile and precision of the KCC2 target, noting its "self-limiting" nature prevents over-modulation compared to other CNS targets.

Miscellaneous:

• Cash Runway: Confirmed funding sufficient through 2029.
• Target Profile: Company focuses exclusively on small molecule medicines to ensure ease of patient use.
• Key Metrics: OV329 is characterized by "superb safety margins" and "clean" data in 100 humans; OV4071 shows "highly consistent" potency and "profound activity" in over 40 animal studies across psychosis domains.