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Disclaimers and Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "believe," "expect," "plan," "anticipate" and similar expressions (as well as other words or expressions referencing future events or circumstances) are intended to identify forward-looking statements. Forward-looking statements contained in this presentation may include statements regarding the progress, timing, development of the Company's product candidates and pipeline programs; scope of clinical trials; the potential clinical benefit of the Company's product candidates and pipeline programs; regulatory development; the success of any licensing or partnering opportunities; the potential commercialization of product candidates and pipeline programs; the potential value of the 2021 royalty, license and termination agreement with Takeda; the success of Takeda's trials in soticlestat and the potential commercialization of soticlestat; and the Company's expectations regarding its operating expenses, and use of its cash, cash equivalents and short-term investments to the development the Company's pipeline and pursue business development opportunities. Each of these forward-looking statements involves risks and uncertainties.
Strong Foundation For Successful Neurosciences Development Build a unique company that delivers first-in-class therapeutics for rare disorders of the CNS 1 As of 6/30/21
2 Excluding non-cash and non-recurring expenses
3 As of 6/30/21, on an as if converted basis THE RIGHT TEAM PROVEN TRACK-RECORD IN RARE & NEUROSCIENCE STRONG BALANCE SHEET Deep experience across R&D continuum, BD, IND filings, approvals and launches $212.2M1 in cash and cash equivalents
Up to $660M in regulatory and sales milestones, if soticlestat is approved
Expected quarterly Op Ex of $8M-$10M2through 21
Chairman, CEO Jason Tardio MBA
Chief Operating Officer Jeffrey Rona Chief Business & Financial Officer Thomas Perone J.D., MBA
GC, Corporate Secretary, and CCO Claude Nicaise M.D.
Head, Research & Development
Ovid Therapeutics Focus & Approach Developer of novel first-in-class / best-in-class therapeutics that seek to make a BOLD impact in rare neurological and related CNS disorders. 4 FOCUS Translation engine
Target identification, pathology & translation with academic partners APPROACH Modality & delivery
Pairing optimal modalities (small molecule & next generation) to deliver therapeutics across the blood-brain barrier Enabling technologies & screening tools
De-risking potential candidates earlier with enabling tools and screening technologies Clinical stage pipeline acquisitions
that complement existing pipeline and areas of focus Accelerated development
Soticlestat Agreement with Takeda Holds Potential to Generate Up to $856M Upfront Payment
$196M received at closing
All financial obligations to Takeda for soticlestat are terminated
Regulatory Milestones
Regulatory milestones
Takeda funding two comprehensive pivotal trials (LGS and Dravet)
Commercial Milestones/ Royalties
Commercial sales milestones post approval
Tiered double-digit royalties up to 20% on global soticlestat sales (all indications)
Rare Neurologic Conditions Represent Significant Opportunity Yet, CNS drug development has been historically challenged
Incomplete understanding of disease biology
Poor predictive value of animal models
Lack of reliable biomarkers and difficult-to measure endpoints
Blood-brain-barrier preventing therapeutics from reaching the brain
Recent Scientific Advances and CNS Expertise Enable Development for Previously "Undruggable" Targets Recent successes in genetic medicine paves the path for next-gen therapies:
Next-gen DNA and RNA editing tech
Immune system modulation
Understanding of functional genomics
Molecular imaging and functional MRIs can improve observation of activity level; supporting understanding of disease pathology and candidate outcomes
Supported discovery of Parkinson's subtypes
Earlier identification of high-risk TIA patients
Biomarkers to better measure therapeutic efficacy
Advances in BBB-crossing approaches can drive future growth of neuroscience therapeutics:
Novel delivery approaches
Targeting of therapies to specific cell types
Decrease the time and risk associated with new CNS directed therapies
Proprietary and differentiated enabling technologies and delivery systems to support development of next-generation CNS therapeutics
Tools that can be applied and offer utility for current pre-clinical and future CNS assets
Assays that support penetration across the blood brain barrier, and allow in vitro testing
Delivery platforms that minimize immunogenicity, enable precision targeting, and address manufacturing issues
Complement existing pre-clinical pipeline with actionable assets near IND or later
Complementary to existing pipeline and strategy
Leverage core capabilities in rare CNS diseases
Potential for first, or best-in-class medicines that are disease modifying or that establish a new standard of care
Indication(s): Refractory Epilepsies
1. Tuberous sclerosis complex
Affects 1 in 6K individuals (~50K patients in US); epilepsy present in ~85% of TSC patients*
Current treatment options include vigabatrin, everolimus, and surgery
Significant unmet need: Most patients resistant to current therapy
2-3.5 cases per 10K births in US
Current treatment options include ACTH and vigabatrin
Significant unmet need: Significant side effects associated with standard of care
Mechanism: Highly potent GABA aminotransferase (GABA-AT) oral small molecule inhibitor
Development status: IND enabling studies are underway, IND expected 1H 2022
OV329 Overview Highly Potent Inhibitor of GABA Aminotransferase * Source: Tuberous Sclerosis Alliance; Pellock JM, et al. Epilepsia (2010) 12 Opportunity:
Indication: Angelman Syndrome
Affects 1 in 15K individuals
Characterized by developmental delay, ataxia, sleep disorder, seizures, and speech impairments
Current treatment options are symptomatic (e.g., anti-seizure)
Significant unmet need:
No specific treatments available which target the neuropathophysiology of Angelman syndrome
ASOs** are being investigated by others; approach may have challenges
Mechanism: Short hairpin RNA that interacts with non-coding RNA to inhibit the silencing of paternal UBE3A gene
Development status: POC* activity confirmed in vitro; currently undergoing pre-clinical validation
Collaboration with Connecticut Autism Language Lab under Associate Professor Stormy Chamberlain
OV882 Overview Potential Disease-Modifying Genetic Therapy for Angelman Syndrome Source: Foundation for Angelman Syndrome Therapeutics (FAST)
* Proof of Concept (POC), ** Antisense oligonucleotides 14 Opportunity: Potential disease modifying treatment
OV882 Approach to the Treatment of Angelman Syndrome DISEASE STATE ASO APPROACH OV882 shRNA APPROACH Mechanism may cause undesirable off-target effects
Requires redosing on approximately quarterly timescale
Requires chemical modification of ASO Angelman syndrome is caused by a mutation in the maternal copy of the UBE3A gene and silencing of the paternal copy
Silencing is mediated by a non-coding RNA sequence whose expression blocks transcription of the paternal UBE3A gene Exclusively silences UBE3A-ATS and un-silences UBE3A
Minimizes off-target effects
OV882 Appears Active in Pre-Clinical AS Neuron Cell Model OV882 demonstrates activity in AS neuronal cell system:
>2x increase in UBE3A mRNA expression when compared to SCRAM control
Reduction in UBE3A-ATS expression further demonstrates the potential mechanism and efficacy of OV882
~200** patients worldwide with documented diagnoses; total number of affected patients likely in the thousands
Broader kinesin superfamily opportunity
Symptoms associated with KAND include hereditary spastic paraplegia, ataxia, epilepsy, hypotonia, autism, and ADHD
Current treatment options are symptomatic
Significant unmet need: No specific treatments available Overview
Mechanism: Genetic / molecular approach targeting KIF1A
Development status: Currently in screening stage for aptamer and gene silencing technologies
In collaboration with
OV815 Overview Potential Advanced Genetic Therapy for KAND and KIF-associated Diseases Notes: * KIF1A-Associated Neurological Disorder
Source: **KIF1A.org 17 Opportunity:
OV815 Has Potential For Broader Applicability Within the Kinesin Superfamily Impact of KIF1A on neurotransmission
KIF1A is a motor protein that transports cargo for neurons
Disruption of cargo transport impacts neurotransmission and leads to progressive neurologic deficits
Strong history of identifying promising targets in hard-to-treat diseases Proven Business Development Track Record Monetized CNS expertise to strengthen the balance sheet and create a potential cash stream to build the next major player in CNS DECEMBER 2016
License agreement w/Northwestern for OV329 January 2017 Soticlestat co-development agreement w/Takeda FEBRUARY 2020
Strategic research collaboration with Columbia for additional neurodevelopmental disorder targets JULY 2020 License agreement w/UConn for OV882 March 2021:
The Ingredients to Be a Major Player in CNS 20 Modality & delivery mechanisms to cross the BBB Enabling technologies & screening tools increase efficiency Effective prosecution of R coupled with pipeline acquisitions THE OVID APPROACH
Apply insights from small molecule neurologic therapeutics to expand