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2 January 2021 April 2021
approaches to create customized
solutions for rare disease Commitment to and collaboration with
underserved patient communities to
ensure patient voices are heard Poised to fuel next-generation
neuroscience therapeutics backed
by decades of experience Clear Vision Supported by a Strong Foundation April 2021
5 April 2021 2017 Collaboration
Global for all soticlestat indications 2021 Agreement
Global for all soticlestat indications Ovid receives up to $856M in payments and double-digit royalties up to 20% on global sales
Received $196M upfront (1Q '21)
Up to $660M in regulatory and commercial milestones
R&D: 50/50 cost sharing + milestones to Takeda
Commercialization: Profit Share Financials: Development: Executed multiple clinical trials enabling pivotal studies Takeda targeting a 2024 launch
Potential multi-billion-dollar market opportunity
Ph 3 trials in Dravet and LGS expected to begin in 1H 21 Transformative $856M+ Agreement with Takeda
Advance next-generation neuroscience pipeline
Identify novel delivery technologies
Focus on differentiated MOAs
Target underlying disease pathology Conduct an assessment taking a comprehensive and disciplined approach reviewing the full ecosystem of external innovation 1 2
Focus on ecosystem of rare neurological diseases with high unmet need
Develop novel first-in-class / best-in-class therapeutics
Create a customized disease approach leveraging deep understanding of underlying pathology
Development status: IND enabling studies are underway, IND expected 1H 2022 Opportunity: Create a superior product based on a validated mechanism
OV329: Potential best-in-class Asset overview Indication(s) overview Refractory epilepsies Tuberous sclerosis complex Infantile spasms 1 in 6K individuals (~50K patients in U.S.); 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 U.S.
Current treatment options include ACTH and vigabatrin
Significant unmet need: Significant side effects associated with standard of care Opportunity and OV329 April 2021 * Source: Tuberous Sclerosis Alliance; Pellock JM, et al. Epilepsia (2010) OV329
Development status: POC* activity confirmed in vitro; currently undergoing pre-clinical validation
In collaboration with Connecticut Autism Language Lab under Associate Professor Stormy Chamberlain Opportunity: Create potential disease modifying treatment for high unmet need indication
OV882: Targets the mechanism of silencing without affecting the gene, minimizes off-target effects, and potentially increases treatment duration compared to ASOs Asset overview Indication overview OV882 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 Opportunity and OV882 Source: Foundation for Angelman Syndrome Therapeutics (FAST)
* Proof of Concept (POC), ** Antisense oligonucleotides
Silencing is mediated by a non-coding RNA sequence whose expression blocks transcription of the paternal UBE3A gene OV882 shRNA APPROACH Mechanism may cause undesirable off-target effects
Requires redosing on approximately quarterly timescale
Requires chemical modification of ASO Exclusively silences UBE3A-ATS and unsilences UBE3A
Minimizes off-target effects
Potential for longer lasting effects
>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
Development status: Currently in screening stage for aptamer and gene silencing technologies
In collaboration with Asset overview Indication(s) overview OV815 KAND ~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 Opportunity: Leverage knowledge gained from KIF1A to access the broader kinesin superfamily associated diseases Opportunity and OV815 Source: *KIF1A.org
Notes: **Amyotrophic lateral sclerosis
KIF1A is a motor protein that transports cargo for neurons
Disruption of cargo transport impacts neurotransmission and leads to progressive neurologic deficits KIF1A Primary: KAND Initial opportunity Additional opportunity in the Broader kinesin superfamily Source: Al-Bassam_2018_Malleable folding of coiled-coils regulates
Pro forma cash of over $250M*
Expected 2Q 2021 - 4Q 2021 quarterly OpEx of $8M-$10M (excluding non-cash expenses)
69M shares outstanding (as of 3-15-21) As of 12-31-20 *Cash and equivalents as of 12-31-20 together with upfront payment from Takeda
Chairman, CEO Amit Rakhit MD, MBA
President, CMO Jason Tardio MBA
Chief Commercial Officer Jeffrey Rona Chief Business Officer Thomas Perone JD, MBA
GC, Corporate Secretary, and CCO Holly Roberts MD
Vice President, Medical Affairs Claude Nicaise MD
Head, Rare Disease Strategy Suzanne Wakamoto
SVP, HR Timothy Daly EVP, Finance, Corporate Controller & Treasurer Mathews Adera
VP, Neurodevelopment Julia Tsai
VP, Epilepsy Luke Rosen
VP, Accelerated Development & Community Engagement Leadership Team With Deep Knowledge and Expertise April 2021 Deep experience across full R&D continuum, numerous BD transactions, IND filings, product approvals and launches