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These statements are based on the Company's current expectations and projections made by management and are not guarantees of future performance. Therefore, actual events, outcomes and results may differ materially from what is expressed or forecast in such forward-looking statements. Factors that may cause actual results to differ materially from these forward-looking statements include the fact that initial data from clinical trials may not be indicative, and are not guarantees, of the final results of the clinical trials and are subject to the risk that one or more clinical outcomes may materially change as patient enrollment continues and or more patient data becomes available; Takeda's ability to successfully complete clinical development of, obtain regulatory approval for and, if approved successfully commercialize Soticlestat; and uncertainties in the development and regulatory approval. process. Additional risks that could cause actual results to differ materially from those in the forward-looking statements are discussed in the Company's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Such risks may be amplified by the COVID-19 pandemic and its potential impact on the Company's business and the global economy. Except as otherwise required under federal securities laws, we do not have any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events, changes in assumptions or otherwise.
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2 January 2021 June 2021
Strong Balance Sheet
Regulatory milestones
Takeda initially 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)
Received $196M at closing (1Q '21)
All financial obligations to Takeda are terminated
Potential Non-Dilutive Cash Stream to Fund Pipeline Development
Zero Financial Obligations for Soticlestat Moving Forward $
$ 2021 Est. 2023/2024 Est. 2024 and later Up to $660M in Combined Regulatory and Commercial Milestones
Poor predictive value of animal models
Lack of reliable biomarkers and difficult to measure endpoints
Blood-brain-barrier preventing therapeutics from reaching the brain
Patient population variability and need for large trials Significant opportunity in CNS therapeutics June 2021 Source: Lee C. et al (2020); L.E.K. research and analysis
Next-gen DNA and RNA editing tech
Immune system modulation
Understanding of functional genomics 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 More precise brain imaging enables better disease understanding and patient outcomes:
Supported discovery of Parkinson's subtypes
Earlier identification of high-risk TIA patients
Biomarkers to better measure therapeutic efficacy
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 Affects 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 June 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 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 Notes: * KIF1A-Associated Neurological Disorder
KIF1A is a motor protein that transports cargo for neurons
Disruption of cargo transport impacts neurotransmission and leads to progressive neurologic deficits KAND Initial opportunity Additional opportunities in kinesin superfamily Source: Al-Bassam_2018_Malleable folding of coiled-coils regulates
rights for OV101 from Lundbeck January 2017: Soticlestat co-development agreement w/Takeda December 2016: License agreement w/Northwestern for OV329 June 2020: Strategic research collaboration w/Columbia for OV815, OV816 & other targets March 2021: Soticlestat deal w/Takeda up to $856M plus royalties July 2020: EU License Agreement w/Angelini for OV101
Rare neurological, neurometabolic, ophthalmic, seizure related, other CNS disorders Actionable assets near IND or later
Complementary to existing pipeline and strategy
Leverage core capabilities in rare CNS diseases Best- or first-in-class therapies
Disease modifying therapy / potential to establish new standard of care CNS therapeutic enabling
Supports company therapeutic area focus on genetic medicine Blood-brain barrier assays / delivery systems
BBB assays: assess penetration, evaluate targeting, and test in vitro
Delivery systems: minimize immunogenicity, enable precision targeting, and address manufacturing issues Proprietary and differentiated
Unique delivery system or differentiated technology June 2021 Clinical Stage Targets Technologies
$233M in cash and cash equivalents as of 3/31/21
No further financial obligations for the development of soticlestat, significant downstream economics if soticlestat is approved
Expected 2Q 2021 - 4Q 2021 quarterly Op Ex of $8M-$10M1
69M shares of common stock outstanding 2 1excluding non-cash and non-recurring expenses
2 as of 5/6/21, on an as if converted basis
Chairman, CEO Amit Rakhit MD, MBA
President, CMO Jason Tardio MBA
Chief Commercial Officer Jeffrey Rona Chief Business & Financial Officer Thomas Perone JD, MBA
GC, Corporate Secretary, and CCO Leadership Team With Required Track Record Deep experience across R&D continuum, BD, IND filings, approvals and launches June 2021 Claude Nicaise MD
Head, Rare Disease Strategy
Use technology to achieve a fundamental change
Leverage strong academic relationships
Invest smart and conduct R&D efficiently
Prepared and capitalized to be a major player in the next CNS wave June 2021