March 2020 Corporate Presentation (NASDAQ: OVID) Exhibit 99.1
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 about the progress, timing, clinical development and scope of clinical trials and the reporting of clinical data for the Company’s product candidates; the potential clinical benefit of the Company’s product candidates; the timing and outcome of discussions with regulatory authorities; the success of any partnering opportunities; and the use of 24HC as a biomarker for target engagement. Each of these forward-looking statements involves risks and uncertainties. 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. 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. 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.
The Ovid Opportunity Experienced, Proven Leadership Team Focused on Execution Cash of ~$77M(1); fully funded through clinical data events (1) As of December 31, 2019 Pivotal Phase 3 trial for OV101 in Angelman syndrome ongoing; expect data in mid-2020 NEPTUNE OV935 program in Phase 2 studies for rare epilepsies; partnered with Takeda; Expect data in 1Q (ARCADE) and 3Q 2020 (ELEKTRA – fully enrolled) ARCADE / ELEKTRA Multiple additional clinical data readouts during 2020 ROCKET / SKYROCKET / ENDYMION
Multiple Clinical Data Readouts Expected in 2020 PRODUCT CANDIDATE RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3 INDICATION Angelman Syndrome Fragile X NEPTUNE – Ongoing ELARA OLE – Ongoing ROCKET – Ongoing SKYROCKET - Ongoing CDKL5 Deficiency Disorder / Dup15q Syndrome Dravet / LGS ARCADE(1) – Ongoing ENDYMION OLE – Ongoing ELEKTRA – Fully Enrolled ENDYMION OLE – Ongoing Treatment Resistant Epilepsy Angelman Syndrome OV101 δ-selective GABAA receptor agonist OV935 CH24H inhibitor OV329 GABA aminotransferase inhibitor OV881 microRNA KEY MILESTONES NEPTUNE Topline Data: Mid-2020 ELEKTRA & ENDYMION: Q3 2020 ARCADE (Initial) & ENDYMION: Q1 2020 ROCKET & SKYROCKET Early Q2 2020 / (1) ARCADE full data set expected early 2021
Addressing Unmet Needs in Significant Populations 1 Based on estimated prevalence. Epilepsy estimate includes only four types currently represented in Ovid clinical studies: Dravet, LGS, Dup15q, CDD., estimate patients in the US only $100K/Pt/Year $50K/Pt/Year Illustrative Example of Gross Sales 2 2 Based on illustrative orphan drug pricing Gross Sales ($MM) Patients US Patients Covered by Ovid Programs 1 ~24,000 ~70,000 ~72,000 1 ~12,000 ~30,000 Example of Other Rare Disorders Populations
NEPTUNE, if positive, will be part of a broad data set to support registrational filings for OV101 in the U.S. and rest of world Angelman Syndrome STARS study OV101 QD provides foundation for Phase 3 program given statistically significant, and clinically meaningful effect in CGI-I versus placebo Favorable safety and tolerability profile Phase 3 NEPTUNE Enrollment ongoing Topline readout mid-2020 Primary endpoint CGI-I-AS OV101 AS Neurodevelopmental disorder characterized by profound developmental delay and impairments across motor, sleep and behavioral areas ~24K patients in the US No approved therapies
Angelman Syndrome Need for effective treatment options could not be greater No approved therapies since condition first described in 19651 Treatment options are symptomatic and include anti-seizure therapies Affects 1:12,000 to 20,000 people2,3,4 Affects males and females in equal numbers3 Disease results from loss of functional UBE3A protein due to defect of UBE3A gene on maternal chromosome 15 Decreased extrasynaptic GABA levels lead to decreased tonic inhibition and resulting downstream clinical phenotype Characterized by profound developmental delay, ataxia and gait disturbance, sleep disorder, seizures, heightened anxiety & aggression, and severe speech impairments 1. Buiting K et al. Nat Rev Neurol. 2016;12:584-593. 2. NORD, NIH website, Genetics Home Reference and NCBI. 3. Bird LM. Appl Clin Genet. 2014;7:93-104. 4. Assume 24,000 US patients
UBE3A, ubiquitin protein ligase E3A; GABA, ɣ-aminobutyric acid. Background on Angelman Syndrome and OV101 Restores impaired tonic inhibition Highly selective extrasynaptic GABAA receptor (GABAAR) agonist, which binds to the δ-subunit of GABAAR as an orthosteric agonist Differentiated from all other GABAA modulators Restores deficits in tonic inhibition in a preclinical mouse model of Angelman syndrome Has the potential to normalize tonic inhibition in humans OV101 Presynaptic Neuron GABA production GABA transporter GABA Synaptic Extrasynaptic HEALTHY ANGELMAN δ δ Post-synaptic Neuron OV101
Assessment Points (weeks) Placebo (placebo morning, placebo night) OV101 BID (10 mg morning, 15 mg evening) OV101 QD (placebo morning, 15 mg evening) -2 0 6 12 Screening Baseline & Randomize (1:1:1) STARS: A Phase 2 Clinical Trial in Angelman Syndrome * Excluding, poorly controlled seizure activity, concomitant use of minocycline, levodopa, zolpidem, zaleplon, eszopiclone, ramelteon, or cannabinoid derivatives or any other investigational agent, device, and/or procedure. BID=twice a day; QD=every day. Outcome Measures Trial Design and Key Inclusion Criteria 88 adults and adolescents 12 sites in US, 1 site in Israel Age 13–49 years, inclusive Genetic diagnosis of Angelman syndrome Receiving a stable regimen of concomitant medications for at least 4 weeks prior to baseline* Primary Outcome Measure: Safety and tolerability of OV101 vs placebo Exploratory Outcome Measures: Efficacy measures of OV101 vs placebo
CGI-I Captures the Improvement in the Totality of a Patient’s Symptoms Validated measure used in neurology and psychiatry trials Co-primary or key secondary endpoint in several FDA-approved and ongoing clinical trial programs including: Nuplazid (Psychosis), Intuniv (ADHD) Clinician assessed Captures the improvement in the totality of a patient’s symptoms Patients Rated on a Likert 7-Point Scale 7 = very much worse 6 = much worse 5 = minimally worse 4 = no change 3 = minimally improved 2 = much improved 1 = very much improved Changes of 0.5 point in the scale represent a clinically meaningful change for this patient population STARS - Achieved a Clinically and Statistically Meaningful Change of 0.79 vs placebo in CGI-I P=0.3446 P=0.0006 Value of 4 represents no change on CGI-I *Least-square mean difference [Drug-Placebo] (95% CI) was -0.78 (-1.22, -0.35) with OV101 QD and -0.21 (-0.64, 0.22) with OV101 BID. BID=twice a day; CGI-I=Clinical Global Impressions–Improvement; QD=every day. Bird LM et al. Poster presented at: AACAP 2018.
74% of STARS Study Responders Experienced Clinical Improvement in Multiple Domains 1 3 Domains Improvement Driver(s): Domains: 2 Domains 1 Domain (1) Responder analysis from the QD patient arm of STARS study n=27. Effect over multiple domains in this heterogeneous population is best captured by CGI-I
STARS Data Supports Once a Day Dosing for the Phase 3 NEPTUNE Trial AS=Angelman syndrome; BID=twice a day; CGI-I=Clinical Global Impressions–Improvement; QD=every day. Bird LM et al. Poster presented at: AACAP 2018. Subjects in Each CGI-I Score Category (%) 22.2 66.6 42.8 Effect Increases Over Time and in the Younger Patient Population Note: Age Group and Duration were both post-hoc analyses Age Group
Consistent Positive Results from the Phase 2 STARS Study Strong safety and tolerability profile Statistically significant results in CGI-I 74% of responders experienced clinical meaningful improvement in multiple domains QD dosing arm had 3X number of responders vs. placebo (66% vs. 22%) 1 Effect of OV101 increases over time Effect is strongest in younger patients Provides Foundation for Phase 3 Design and Regulatory Pathway to Approval (1) QD dosing arm with 18 responders out of 27 patients; placebo arm with 6 responders out of 27 patients.
NEPTUNE – Pivotal Phase 3 Study in Angelman Syndrome Initiated; Anticipate Data by Mid-2020 Placebo (at bedtime) OV101 QD (weight based dosing, at bedtime) -2 0 6 12 Assessment Points (weeks) Screening Baseline & Randomize (1:1) Trial design Objectives Primary endpoint: CGI-I-AS at Week 12 of OV101 vs placebo Secondary endpoints: Sleep measures Communication, motor skills, socialization, daily living skills, and maladaptive behavior domains, as assessed by the Vineland Adaptive Behavior Scale 3rd edition Change in CGI-S-AS Symptoms Overall Randomized, double-blind, Phase 3 study based on FDA feedback Up to 15 sites in United States, Israel, Australia and Europe Approximately 60 subjects (ages 4 –12 years) Approximately 5 subjects ages 2 – 3 years for safety only Powered to detect a clinically meaningful difference in CGI-I-AS
Clear Regulatory Path Forward to Registration for OV101 for AS* Strategic Positioning First in disease, first-in-class, first-line treatment for individuals with Angelman syndrome* No approved therapies for Angelman syndrome Orphan drug designation in US and EU US Registration Package to Support Global Development Plan: *Assumes positive results from the Phase 3 NEPTUNE trial. Planned filing package = Phase 3 NEPTUNE study (single pivotal trial) + Phase 2 STARS study (supportive evidence) + preclinical / safety data from prior insomnia studies + other preclinical / safety data Extensive safety database Initial filing package would serve as basis for rest of world
Fragile X Syndrome OV101 normalizes anxiety, irritability, repetitive behaviors and hyperactivity in animal models of Fragile X ~70K patients in the US No approved therapies ROCKET Phase 2 signal-finding study to assess: Three different daytime dose regimens Safety and tolerability (primary endpoint) Changes in behavioral symptoms Data expected early 2Q 2020 SKYROCKET Non-drug study to assess the suitability of scales for the measurement of behavior, sleep and functioning Data expected early 2Q 2020 OV101 FXS
ROCKET: Phase 2 Signal Finding in Fragile X Syndrome to Assess Safety, Tolerability, Efficacy, and Optimal Dose OV101 5 mg QD OV101 5 mg TID OV101 5 mg BID Screening Baseline & Randomize (1:1:1) Key Inclusion Criteria Endpoints Primary Endpoint: Safety and tolerability of OV101 over 12 weeks across 3 daily dosing regimens Secondary Endpoint: Changes in behavior (ABC-C, ADAMS) at 12 weeks Adult and adolescent males with diagnosis of FXS (n~20-30) Ages: 13–22 years CGI-S score of ≥ 4 IQ under 75 Sites – 8 in US and 1 in Israel
OV101: Upcoming Data Readouts NEPTUNE Phase 3 study in Angelman syndrome Topline readout anticipated mid-2020 ROCKET & SKYROCKET Phase 2 studies in Fragile X syndrome Data anticipated early 2Q 2020
Robust clinical program for developmental and epileptic encephalopathies (DEE) patients In adult Phase 1b/2a study Good safety profile and generally well tolerated 61% reduction in median seizure frequency ENDYMION (open label extension) evaluating long-term safety and efficacy In a cohort from our Phase 1b/2a study (“2001 Study”): Continued to see favorable safety and tolerability profile Achieved a 90% median reduction in seizures in patients on drug out to 48 weeks Open-label ARCADE study for early signal finding in Dup15q and CDKL5 Data 1Q:20 ELEKTRA study is POC for DS and LGS Study is fully enrolled; data in 3Q:20 Strong preclinical data support clinical development Rare Epilepsies Novel CH24H enzyme target & unique MOA OV935 (soticlestat)
DEE Encompass Diverse Etiologies with Few or No Approved Treatments CDKL5 Deficiency Disorder (CDD) Chromosome 15q Duplication Syndrome (Dup15q) Dravet Syndrome (DS) Lennox-Gastaut Syndrome (LGS) Etiology Mutations in CDKL5 gene on X chromosome Duplication in chromosome 15q11.2-q13.1 region 80% have mutations in Scn1a gene Multiple Age of onset Birth–3 months 6 months–9 years Birth–1 year 2–5 years Prevalence ~1:40,000–1:60,000 (5.5K- 8.1K Patients in the US) ~1:30,000 (11K Patients in the US) ~1:15,000–1:21,000 (15.5K to 22K patients in the US) ~1:11,000 (30K Patients in the US) Clinical Manifestations Long, intractable seizures, periods of repeated seizures Mainly affects females Atypical hypsarrhythmia pattern on EEG over time Repetitive hand movements Hypotonia Hypotonia, motor delays, ASD symptoms, anxiety disorder Infantile spasms progressing to LGS-type syndrome Prolonged focal that can evolve to generalized convulsive tonic-clonic seizures First seizure associated with fever in 60% of cases Developmental delay Drop seizures Multiple seizure types More common in males Intellectual disabilities along with psychiatric comorbidities Distinctive EEG brain wave pattern FDA-approved Therapies None None Epidiolex® (cannabidiol), stiripentol Lamotrigine, topiramate, felbamate, rufinamide, clobazam, clonazepam, Epidiolex® (cannabidiol) Soticlestat has the potential to treat ~70K patients in the U.S. that are affected by these conditions ARCADE ELEKTRA
Epileptogenic insult ↑ Modulation of NMDA Receptor ↑ Modulation of Glial Function Epilepsy ↑ 24HC Cholesterol ↑ Glutamate Release ↑ Inflammation ↑ Tissue Excitability CH24H Mechanism of OV9351,2 OV935 (soticlestat): A Highly-selective First-in-Class CH24H Inhibitor in Development to Reduce Glutamatergic Signaling and Help Improve Seizure Control Thus, OV935 has the potential to reduce seizure susceptibility and improve seizure control1 Reducing 24HC levels mediates reduced glutamatergic signaling, modulation of glial (ie, astrocyte and microglia) function and activation, and reduced inflammation1 OV935 inhibits CH24H resulting in a dose-dependent reduction in plasma 24HC levels1 OV935 may provide benefit through mechanisms that are not targets of conventional AEDs1,2 1. Salamone A et al. Poster presented at: ECE 2018. 2. Nishi T et al. Poster presented at: AAN 2018. OV935 T
Phase 1b/2a Placebo-Controlled 12-week Adult DEE Trial Results Soticlestat Was Well Tolerated with Reduced Seizure Frequency Over Time Soticlestat achieved primary endpoint of safety and tolerability as measured by incidence of adverse events (AEs)* The majority of AEs were mild Seizure Frequency 61% reduction in median seizure frequency observed at scheduled Day 92 Two of 11 patients became seizure-free (last 28 days of treatment to Day 92) Median Reduction from Baseline in Total Seizures** per 28 Days (FSA, Part 2, excluding patients on perampanel)a *AEs that occurred more frequently in the soticlestat-treatment group vs placebo group were dysarthria, insomnia, lethargy, seizure cluster, and upper respiratory infection. **Data represent per protocol analyses; ITT group (n=18) demonstrated median reduction in total seizures of 36% at Day 85. Two patients withdrew in Part 1 (weakness (n=1); difficulty with walking/worsening lethargy (n=1)) and two patients withdrew in Part 2 (seizure cluster (n=2)). a Based on an interval averaged over 28 days, excluding myoclonic seizures. The first dose day is Study Day 1. Safety And Tolerability Patient Diagnosis at Trial Entry LGS (n=6), Dravet (n=1), DEE (n=6), Tuberous sclerosis (n=1), All other (n=4)
ENDYMION: An OLE Study to Evaluate Long-Term Safety and Efficacy of Soticlestat Trial Design Prospective, interventional, open-label, multi-site, extension trial Endpoints Aged ≥2 and ≤65 years Has participated in a previous soticlestat trial Soticlestat Treatment (104 weeks) (Up to 2 weeks titration followed by ~102 weeks maintenance)* Completion of Previous Soticlestat Study *Patients who roll over from an open-label trial will continue on their current dose for 104 weeks without titration. Key Inclusion Criteria Primary: Safety and tolerability Secondary: % change in median seizure frequency
ENDYMION: Median Seizure Frequency Reduction Increased Over Time Chart shows data from the 6 patients in adult DEE trial who continued in ENDYMION after the 2001 Study Break between end of Adult DEE trial and ENDYMION (range 6-52 weeks) Baseline seizure frequency of median 11.5 seizures per 28 days in ENDYMION Longest seizure free interval 264 days (out of total treatment duration 350 days) in ENDYMION -48% -90% -84% -65% -59% Last 28 days in adult DEE Study Median % change in seizure frequency including the patient with concomitant perampanel (n=7) per 12 weeks: -31%, -46%, -82%, -90% *two patients have not yet completed 48 weeks of dosing
ENDYMION: Summary of Findings and Interpretations Safety Soticlestat continues to show a favorable safety and tolerability profile No new safety signals were identified with longer term exposure Majority of AEs are mild; no SAEs reported Consistent with prior observations in 12-week Phase 1b/2a adult DEE trial Efficacy Consistent with prior observations in 12-week Phase 1b/2a adult DEE trial Data suggest increasing activity with longer duration of soticlestat treatment Reduction of seizure activity appears to occur progressively Some patients experience prolonged intervals of seizure freedom Encouraging data despite small number of patients
Soticlestat: Upcoming Data Readouts ARCADE (Ultra Rare DEEs) All completed patients have rolled over into ENDYMION ARCADE trial initial data anticipated for Q1 2020 If positive, will engage global regulatory authorities to discuss registration path ELEKTRA (LGS and Dravet) Interest in trial has been strong and all completed patients have rolled over into ENDYMION Fully enrolled, ahead of initial expectations ELEKTRA trial topline results anticipated in 3Q 2020
2020: A Transformative Year for Ovid We anticipate significant value-creating events over the coming year 1 As of December 31, 2019. 2 As of March 4, 2020. Financial highlights ~$76.7 million in cash, cash equivalents and short term investments1 ~54.7 million shares of common stock outstanding2 OV101: Fragile X syndrome (early 2Q 2020) Angelman syndrome (mid-2020) OV935: ARCADE initial data (1Q 2020), ENDYMION data ELEKTRA topline data (3Q 2020), ENDYMION data
Ovid Therapeutics: Our Inspiration to Succeed