Ovid Therapeutics
Corporate presentation
NOVEMBER 2023
Forward looking statement
This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including, without limitation, statements regarding the timing and development of Ovid's product candidate pipeline and achievement of expected near- and long-term milestones; the potential therapeutic benefits of Ovid's current or future product candidates and pipeline programs; the reporting of data for the potential Phase 1 study in healthy volunteers for OV329; the potential use of OV329 to treat rare and treatment-resistant forms of epilepsy and seizures; the clinical and regulatory development of OV329, including the anticipated timing of clinical trials of OV329; the likelihood that data for OV329 will support future development and therapeutic potential; the potential development of OV350 and other KCC2 compounds in the Company's library; the potential timing of a potential IND filings for OV329 and OV350; the suitability of the Company's library of novel, direct KCC2 transporter activators for a range of formulations and administrations that would make it possible to pursue both chronic and acute epilepsies; the potential development and therapeutic opportunity of OV888 (GV101) and other Rho/Rho associated coiled-coil containing protein kinase 2 inhibitors; and the potential safety, selectivity and potency of OV888 (GV101) and other ROCK2 inhibitors; the potential use of OV888 (GV101) and other ROCK2 inhibitors to treat cavernous cerebral malformations and other rare central nervous system disorders; the potential timing of the pivotal formulation for OV888 (GV101); the potential timing of clinical studies for OV888 (GV101) and the resulting data; the timing for the completion of Takeda's two pivotal Phase 3 trials evaluating soticlestat for Lennox- Gastaut and Dravet syndromes; the potential timing of regulatory decisions on soticlestat; the success of Takeda's trials in soticlestat; the duration of the Company's cash runway, and the expectation that it will support the advancement of the Company's pipeline; Ovid's business development intentions; the success of any licensing or partnering opportunities; the success, timing, ability to attract and maintain strategic collaborations; the clinical and regulatory development and potential commercialization of soticlestat, OV329, OV350, OV888 (GV101) or any of Ovid's other current or future product candidates and pipeline programs and market opportunities. You can identify forward-looking statements because they contain words such as "will," "may," "plan," "believes," intends," "anticipates," "design," "advance," "target," "seek," "expects," "demonstrates," "observe," and "potential," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances).
Forward-looking statements are based on Ovid's current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include, without limitation, uncertainties inherent in the preclinical and clinical development and regulatory approval processes, risks related to Ovid's ability to achieve its financial objectives, the risk that Ovid may not be able to realize the intended benefits of its technology or its business strategy, risks related to Ovid's ability to identify business development targets or strategic partners, to enter into strategic transactions on favorable terms, or to consummate and realize the benefits of any business development transactions and risks to Ovid's or any of its partners' abilities to meet anticipated deadlines and milestones. Additional risks that could cause actual results to differ materially from those in the forward-looking statements are set forth under the caption "Risk Factors" in Ovid's Quarterly Report of Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 3, 2023, and in future filings Ovid makes with the SEC. Any forward-looking statements contained in this press release speak only as of the date hereof, and Ovid assumes no obligation to update any forward-looking statements contained herein, whether because of any new information, future events, changed circumstances or otherwise, except as otherwise required by law.
©2023 Ovid Therapeutics | All Rights Reserved | 2 | |
Overview
+ Strong balance sheet
$87.1M | in cash, cash |
equivalents and | |
marketable securities1 | |
$30.0M | in non-dilutive capital |
from Ligand | |
Pharmaceuticals2 | |
70.6M | shares of common |
stock outstanding3 |
Current cash runway expected into 2026
Potential for a non-dilutive capital stream from Takeda
+ Veteran team
with a track record of developing and commercializing blockbuster medicines
Differentiated epilepsy & seizure-related therapeutic franchise
- 3 small molecule programs (4th program sold to Takeda and we retain significant financial interest)
- Potential 1st-in-class or potential best-in-class medicines
- Novel, complementary mechanisms of action for the potential treatment of epilepsy & seizures
- Significant unmet need
5 potential upcoming pipeline events in next 12-15 months
- Complete multiple ascending dose study to evaluate a pivotal formulation for OV888 - H1 2024
- Initiate proof-of-concept trial for OV888 in cavernous malformation - H2 2024
- Surrogate biomarkers, safety and PK results for OV329 oral formulation - H2 2024
- IND for an intravenous formulation of OV329 - H2 2024
- Anticipated submission of 1st IND from KCC2 portfolio (OV350) - H2 2024
Anticipated from Takeda (Ovid's rights to soticlestat were out-licensed to
Takeda)
- Data from a Phase 3 pivotal study of soticlestat in Lennox-Gastaut syndrome (SKYWAY); conducted by Takeda
- Data from a Phase 3 pivotal study of soticlestat in Dravet syndrome (SKYLINEY); conducted by Takeda
- Series of potential regional regulatory filings for soticlestat; conducted by Takeda
1 As of 9/30/23 | |||||
2 | Received for 13% interest of potential milestones and royalties Ovid is eligible to receive from Takeda if soticlestat is approved and commercialized | ||||
3 | As of 9/30/23, on an as if converted basis | ||||
©2023 Ovid Therapeutics | All Rights Reserved | 3 | ||||
Expected to be first-in-class or best-in-class mechanisms of action
PROGRAMS
INDICATION/TARGET | PRECLINICAL | PHASE 1 | PHASE 2 | PHASE 3 | ANTICIPATED |
MILESTONES | |||||
SOTICLESTAT
Cholesterol 24-hydroxylase inhibitor
Out-licensed to
ROCK2 PLATFORM & OV888*
Selective ROCK2 inhibitor Collaboration with
Dravet syndrome
Lennox-Gastaut syndrome
Brainstem cavernous malformations Undisclosed rare CNS indications
- Phase 3 data from 2 registrational trials
- Filing marketing authorization submissions in
Takeda's FY 2024
- MAD completion H1 2024
- POC study initiation H2 2024
OV329
GABA-aminotransferase inhibitor
In-licensed from:
OV329
GABA-aminotransferase inhibitor
In-licensed from
KCC2 PLATFORM & OV350
KCC2 transporter activators In-licensed from:
Oral formulation for chronic epilepsies (conditions with focal seizures, tuberous sclerosis, infantile spasms)
IV formulation for acute and chronic seizures
Resistant epilepsies and other neuropathologies
- Phase 1 target engagement, PD marker & safety H2 2024
- IND in H2 2024
- IND for OV350 in H2 2024
UNDISCLOSED ROCK2 INHIBITOR PROGRAMS
*Graviton is conducting development of OV888 (formerly GV101) through Phase 2, which will be directed by a Joint Development Committee that includes members of both Graviton and Ovid.
©2023 Ovid Therapeutics | All Rights Reserved | 4 | |
Scientific & pipeline strategy
- Exclusively targeted small molecules
- Expected to be first-in-class or best-in-class mechanisms of action
- Under-addressedbiological targets expressed in CNS
- Mechanisms to modulate metabolic, enzymatic, intracellular & structural causes of hyperexcitability
- Potential application in multiple indications for which hyperexcitability is the cause for the pathology
CONFIDENTIAL - NOT FOR DISTRIBUTION © 2022 | 5 | ||
The goal of many CNS medicines: Neuronal homeostasis (balance)
Glutamate>GABA
Appropriately Regulated
GABA>Glutamate
Extracellular Neuronal Environment
Pathologic Conditions:
• Epilepsy
• Pain
• Schizophrenia
Physiological homeostasis Optimal efficacy, safety & tolerability
Pathologic Conditions:
- Depression
- Sedation
Intrinsic Neuronal Excitability
Hypo-excitability | Balanced | Hyperexcitability |
©2023 Ovid Therapeutics | All Rights Reserved | 6 | |
Ovid strategy:
Unique mechanisms to achieve neuronal balance (homeostasis)
Glutamate>GABA
Appropriately Regulated
GABA>Glutamate
Extracellular Neuronal Environment
EXTRINSIC | OV329 | SOTICLESTAT: | OV888: | ||
Elevation of | Reduced 24HC modulation | Integrity of | |||
GABA levels | of glutamate transmission1 | cell structure/ | |||
vasculature | |||||
Physiological homeostasis Optimal efficacy, safety & tolerability
KCC2 & OV350 Restoration of intraneuronal Cl- concentration
INTRINSIC | ||
Intrinsic Neuronal Excitability | ||
Hypo-excitability | Balanced | Hyperexcitability |
©2023 Ovid Therapeutics | All Rights Reserved | 7 | |
1) Out-licensed rights to Takeda | ||
Ovid mechanisms of action
Seeking to mitigate extrinsic & intrinsic causes of neuronal hyperexcitability
Metabolic & enzymatic:
Modulate extracellular neurotransmitter balance
Cholesterol 24 hydroxylase inhibition (soticlestat): Reduces glutamatergic signaling and glial cell inflammation
GABA-ATinhibition (OV329): Chronic and persistent elevation of GABA leads to profound tonic inhibitory activities in neurons
Structural & cellular integrity:
Restore endothelial cell junction integrity (ROCK2 inhibition)
Cellular intrinsic properties:
Restore chloride homeostasis in neurons
ROCK2 inhibition (OV888, previously GV101): Improving endothelial integrity to reduce leakage & bleeds
KCC2 direct activation (OV350): Restoring proper chloride homeostasis by activating the K+/Cl- co-transporter leads to rebalance of excitatory/inhibitory
response of neurons
Specific mechanism references on pages 11, 20, 26, 34 | CONFIDENTIAL - NOT FOR DISTRIBUTION © 2022 | 8 |
Soticlestat
- first-in-classcholesterol 24 hydroxylase inhibitor for the potential treatment of Lennox-Gastaut syndrome & Dravet syndrome
CONFIDENTIAL - NOT FOR DISTRIBUTION © 2022 | 9 |
Overview
UPDATE
Phase 3 global studies (ongoing)
- SKYLINE trial - Dravet syndrome
- SKYWAY trial - Lennox- Gastaut syndrome
Anticipated global regulatory filings in Takeda's FY2024
Trial completion estimates and site updates available on www.ClinicalTrials.gov
Soticlestat
- First-in-class,selective inhibitor of cholesterol 24-hydroxylase
- Believed to decrease glutamatergic signaling with the potential to reduce seizure susceptibility and improve seizure control
- Studied in one of the most robust development programs for developmental and epileptic encephalopathies (DEE)
- To-date,has demonstrated sustained seizure reduction efficacy, safety & tolerability with no anticipated monitoring in clinical trials
- Planned indications:
- Lennox-Gastautsyndrome o Dravet syndrome
Evidence of clinical findings included on subsequent slides. | ©2023 Ovid Therapeutics | All Rights Reserved | 10 |
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Ovid Therapeutics Inc. published this content on 03 November 2023 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 03 November 2023 12:35:18 UTC.