EX-99.2 3 ovid-ex992_7.htm EX-99.2 ovid-ex992_7.pptx.htm

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Phase 2 ELEKTRA Trial Positive Topline Results August 25, 2020 ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED Exhibit 99.2

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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 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 licensing or partnering opportunities; the commercialization of the Company’s product candidates; 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. Such risks may be amplified by the COVID-19 pandemic and its potential impact on Ovid’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. ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED

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Advanced Pipeline Focused on Rare Neurological Diseases PRODUCT CANDIDATE INDICATION RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3 OV101 δ-selective GABAA receptor agonist Angelman  Syndrome Fragile X OV935 CH24H inhibitor Dravet Syndrome (DS) / Lennox-Gastaut Syndrome (LGS) CDKL5 Deficiency Disorder / Dup15q Syndrome OV329 GABA aminotransferase inhibitor  Treatment Resistant Epilepsy OV881 microRNA Angelman Syndrome KIF1A and other non-disclosed targets shRNA-based therapeutic  Angelman Syndrome NEPTUNE – Enrollment Complete ELARA OLE – Ongoing ARCADE – Positive Initial Data Announced ENDYMION OLE – Ongoing ELEKTRA – Positive Topline Data Announced Today ENDYMION OLE – Interim Data Presented Today (Ongoing) ROCKET – Positive Topline Data Announced SKYROCKET – Topline Data Announced ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED

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PRODUCT CANDIDATE INDICATION RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3 OV101 δ-selective GABAA receptor agonist Angelman  Syndrome Fragile X OV935 CH24H inhibitor Dravet Syndrome (DS) / Lennox-Gastaut Syndrome (LGS) CDKL5 Deficiency Disorder / Dup15q Syndrome OV329 GABA aminotransferase inhibitor  Treatment Resistant Epilepsy OV881 microRNA Angelman Syndrome KIF1A and other non-disclosed targets shRNA-based therapeutic  Angelman Syndrome NEPTUNE – Enrollment Complete ELARA OLE – Ongoing ARCADE – Positive Initial Data Announced ENDYMION OLE – Ongoing ELEKTRA – Positive Topline Data Announced Today ENDYMION OLE – Interim Data Presented Today (Ongoing) ROCKET – Positive Topline Data Announced SKYROCKET – Topline Data Announced ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED Today’s Discussion – Focus on ELEKTRA and ENDYMION

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Soticlestat (OV935/TAK-935) ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED ELEKTRA and ENDYMION are part of a broad-based clinical development program to examine multiple types of epilepsy: Ovid:Takeda “One Team” Alliance – entered into January 2017 Ovid purchased a 50% share in soticlestat; 50:50 cost and profit share Ovid:Takeda Ovid responsible for development and commercialization North America, Europe and Israel Small molecule oral drug invented in Takeda laboratories Extensive preclinical, translational and Phase 1 data package DS & LGS (ELEKTRA): 141 patients enrolled Dravet syndrome (DS) topline data from double-blind placebo-controlled Phase 2 ELEKTRA trial announced today Lennox-Gastaut syndrome (LGS) topline data from double-blind placebo-controlled Phase 2 ELEKTRA trial announced today Open label extension (ENDYMION): all eligible patients from Phase 2 studies have rolled-over Data from ELEKTRA DS and LGS patients rolled-over to ENDYMION are presented today

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Primary Endpoint Achieved: Statistically significant reduction of seizures from baseline compared to placebo (P=0.0007) in the maintenance period for combined Dravet syndrome and Lennox-Gastaut syndrome study population Phase 2 ELEKTRA Trial: Topline Results in Dravet Syndrome (DS) & Lennox-Gastaut Syndrome (LGS) Combined DS and LGS study population: Statistically significant reduction of seizures from baseline compared to placebo (P=0.0024) during the full 20-week treatment period LGS: Numerical reductions in drop seizure frequency compared to placebo DS: 46% median difference from placebo in convulsive seizure frequency reduction (P=0.0007) during the full 20-week treatment period ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED

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Soticlestat Targets Cholesterol Metabolism in the Brain Reduced 24HC levels cause reduced glutamatergic signaling and modulates glial function, resulting in reduced inflammation Cholesterol-24-Hydroxylase metabolizes cholesterol and generates 24 Hydroxycholesterol (24HC) Soticlestat has the potential to reduce seizure susceptibility and improve seizure control ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED CH24H 24HC NMDA Receptor Modulation Seizure Susceptibility Cholesterol

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Reduced 24HC levels cause reduced glutamatergic signaling and modulates glial function, resulting in reduced inflammation Soticlestat inhibits CH24H, resulting in a  dose-dependent reduction in 24HC levels Soticlestat has the potential to reduce seizure susceptibility and improve seizure control Soticlestat is a Potent, Selective, Brain-Penetrating CH24H Inhibitor In Development to Help Improve Seizure Control ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED Soticlestat may provide benefit through mechanisms that are not targets of conventional AEDs Epileptogenic Insult CH24H ↓ 24HC ↓ NMDA Receptor Activation ↓ Glial Inflammation ↓ Seizure Susceptibility Therapeutic Potential in Epilepsy Cholesterol Soticlestat

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Dravet Syndrome and Lennox-Gastaut Syndrome are Different Disorders with Few Approved Treatments Dravet Syndrome (DS) Lennox-Gastaut Syndrome (LGS) Etiology 80% have mutations in Scn1a gene Heterogeneous condition which can be caused by any number of reasons including: several gene mutations, perinatal insults, congenital infections, brain tumors/malformations, and genetic disorders such as tuberous sclerosis and West syndrome Age of Onset Birth–1 year 2–5 years Prevalence ~1:15,000–1:21,000 (15.5K to 22K patients in the US) ~1:11,000 (30K Patients in the US) Clinical Manifestations Prolonged focal seizures 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 Epidiolex® (cannabidiol), Fintepla® (fenfluramine),  Diacomit® (stiripentol) Lamotrigine, topiramate, felbamate, rufinamide, clobazam, clonazepam, Epidiolex® (cannabidiol) 45-52K patients in the U.S. are affected by these conditions ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED

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Key Inclusion Criteria Aged ≥2 and ≤17 years  Clinical diagnosis of DS or LGS Failed at least ≥2 AEDs Currently taking 1–4 AEDs at a stable dose Minimum of at least 3 convulsive (DS) or 4 drop (LGS) seizures during a minimum 4-wk prospective baseline Trial Design Global, multicenter, randomized, double-blind, placebo-controlled Final enrollment: n=141 Titrated: 100 mg BID, 200 mg BID, 300 mg BID (mg/kg dosing <60 kg) oral tablets Phase 2 ELEKTRA Trial Design Screening/ Baseline DS LGS Follow-up (4 Weeks) Entry into ENDYMION End of trial Dose Optimization Period (8 Weeks) Maintenance Period (12 Weeks) Randomization Soticlestat BID Placebo BID ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED Protocol Amendment 1 (14 wk full treatment)- PA1 Protocol Amendment 2 (20 wk full treatment)- PA2

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A total of 141 patients were enrolled and 126 completed the study Modified intent to treat (mITT) analysis of 139 patients was performed to evaluate the efficacy endpoints1 Patient Baseline Demographics & Disease Characteristics ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED DS LGS Soticlestat (N=26) Placebo (N=25) Soticlestat (N=43) Placebo (N=45) Age (years) Mean (SD) Min, Max 8.7 (3.9) 4, 17 8.8 (4.5) 2, 16 10.0 (4.2) 2, 17 9.8 (3.6) 3, 17 Sex, n (%) Male Female 17 (65) 9 (35) 14 (56) 11 (44) 30 (70) 13 (30) 28 (62) 17 (38) Race, n (%) American Indian or Alaska Native Asian African American White 0 11 (42) 0 15 (58) 1 (4) 5 (20) 0 19 (76) 0 11 (26) 0 32 (74) 0 16 (36) 1 (2) 28 (62) Ethnicity, n (%) Hispanic or Latino Non-Hispanic and Latino 2 (8) 24 (92) 6 (24) 19 (76) 8 (19) 35 (81) 4 (9) 41 (91) DS LGS Soticlestat (N=26) Placebo (N=25) Soticlestat (N=43) Placebo (N=45) Weight (kg) Mean (SD) Min, Max 29 (11) 15, 61 30 (15) 14, 68 36 (17) 10, 73 34 (16) 12, 82 Number of AEDs2, n (%) Any 1 2 3 4 5 26 (100) 0 11 10 5 0 25 (100) 1 6 11 7 0 43 (100) 2 12 18 10 1 45 (100) 1 11 19 13 1 Seizure Frequency3 Mean (SD) Median Min, Max 13.8 (11.0) 9.1 2.6, 40.3 13.2 (23.9) 6.0 2.5, 125.0 441.0 (1133.5) 67.3 8.1, 5187.7 150.0 (203.8) 89.8 4.0, 1040.1 1. mITT includes any patient who enrolled in the study and received at least one dose of study drug and has at least one post-randomization seizure assessment. 2. Ongoing AEDs are included if start date is prior to first dose date or missing. 3. Seizure frequency is based on convulsive for DS and drop seizures for LGS.

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ELEKTRA Efficacy Endpoints ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED Primary Efficacy Endpoint Median percent change from baseline in seizure frequency (based only on convulsive seizures for DS patients and drop seizures for LGS patients) during 12-week maintenance period Secondary Efficacy Endpoints Percent change from baseline in seizure frequency during 20-week treatment period Percent change from baseline in seizure frequency in each respective DS and LGS stratum Proportion of responders during maintenance period (≥50% reduction in seizure frequency from baseline in each respective DS (convulsive seizures) and LGS (drop seizures) stratum) Investigator & Caregiver reported Clinical Global Impression of Change (CGI-C; Caregiver GI-C), respectively Plasma levels of 24HC

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Efficacy Endpoint Results: DS & LGS ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED (Primary Endpoint) Efficacy Set (Secondary Endpoint) mITT (2) (2) 1 Rank Transformed ANCOVA adjusting for baseline seizure frequency and protocl amendment cohort 2Hodges-Lehmann Estimation of the median in treatment difference (percent change from baseline in soticlestat vs percent change from baseline in placebo) Soticlestat (n=64) Placebo (n=56) Placebo-Adjusted

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Efficacy Endpoint Results (mITT): DS Cohort (Convulsive Seizures) ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED (2) (2) 1 Rank Transformed ANCOVA adjusting for baseline seizure frequency and protocol amendment cohort 2Hodges-Lehmann Estimation of the median in treatment difference (percent change from baseline in soticlestat vs percent change from baseline in placebo) Soticlestat (n=26) Placebo (n=24) Placebo-Adjusted

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Efficacy Endpoint Results (mITT): LGS Cohort (Drop Seizures) ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED (2) (2) 1 Rank Transformed ANCOVA adjusting for baseline seizure frequency and protocol amendment cohort 2Hodges-Lehmann Estimation of the median in treatment difference (percent change from baseline in soticlestat vs percent change from baseline in placebo) Soticlestat (n=43) Placebo (n=42) Placebo-Adjusted

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Proportion of Responders During Full Treatment Period ≥50% reduction in seizure frequency from baseline ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED DS LGS % reduction from baseline in convulsive seizure frequency % reduction from baseline in drop seizure frequency

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NOTE: TEAEs are the adverse events reported following randomization and initial dose of study drug Favorable Safety Profile Consistent with Previous Studies No new safety signals identified ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED Soticlestat (N=71) n (%) Placebo  (N=70) n (%) Total  (N=141)   n (%) TEAEs 57 (80.3) 52 (74.3) 109 (77.3)  Mild TEAE 47 (66.2) 47 (67.1) 94 (66.7)  Moderate 25 (35.2) 19 (27.1) 44 (31.2)  Severe TEAE 10 (14.1) 11 (15.7) 21 (14.9) AE-related withdrawal 4 (5.6) 3 (4.3) 7 (5.0) Dravet 1 1 2 LGS 3 2 5 SAEs 11 (15.5) 13 (18.6) 24 (17.0) Deaths 0 (0) 0 (0) 0 (0) Most common TEAEs (>5%) Soticlestat (N=71) n (%) Placebo (N=70) n (%) Total (N=141) n (%) Upper Resp tract infection 13 (18.3) 12 (17.1) 25 (17.7) Pyrexia 11 (15.5) 8 (11.4) 19 (13.5) Seizure (worsening or new) 6 (8.5) 9 (12.9) 15 (10.6) Nasopharyngitis 6 (8.5) 6 (8.6) 12 (8.5) Decreased appetite 6 (8.5) 5 (7.1) 11 (7.8) Vomiting 6 (8.5) 4 (5.7) 10 (7.1) Somnolence 6 (8.5) 3 (4.3) 9 (6.4) Diarrhea 5 (7.0) 4 (5.7) 9 (6.4) Lethargy * 5 (7.0) 0 5 (3.5) Fatigue 4 (5.6) 3 (4.3) 7 (5.0) Pneumonia 4 (5.6) 2 (2.9) 6 (4.3) Irritability 4 (5.6) 2 (2.9) 6 (4.3) Constipation **4 (5.6) 0 4 (2.8) Soticlestat was generally well-tolerated and demonstrated a safety profile consistent with the findings of previous studies with no new safety signals identified Incidence of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs) were similar in both the treatment and placebo groups; most frequent TEAEs reported in soticlestat treated patients with ≥5% difference from placebo were lethargy and constipation *Number (No) is the number of subjects with at least one adverse event reported *Lethargy 4 mild, 1 severe; **Constipation 3 mild, 1 moderate

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Clinical Global Impression of Change (CGI-C; Investigator) Responses at Last Visit ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED m=  the number of subjects with non-missing value at the specific analysis visit in the modified intent-to-treat analysis set

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ENDYMION: DS Cohort – Positive Response in Placebo Crossover and Continued Efficacy in Soticlestat Patients ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED Median Percent Change from ELEKTRA Study Baseline in 28-days Convulsive Seizure Frequency Soticlestat Placebo 25 0 -25 -50 -75 -100 Overall Treatment Period in ELEKTRA Month 1 Month 3 Month 6 18 18 18 17 10 13 n = Median % Change from Baseline in 28-days Convulsive Seizure Frequency 18 18 Placebo Crossover to Soticlestat ENDYMION Treatment Period

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ENDYMION: LGS Cohort – Reduction in Drop Seizure Frequency in Placebo Crossover and Continued Effect in Soticlestat Patients ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED 50 25 0 -25 -50 -75 Overall Treatment Period in ELEKTRA Month 1 Month 3 Month 6 25 25 32 25 29 15 20 n = Median Percent Change from ELEKTRA Study Baseline in 28-days Drop Seizure Frequency Median % Change from Baseline in 28-days Drop Seizure Frequency 32 Soticlestat Placebo Placebo Crossover to Soticlestat ENDYMION Treatment Period

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Summary and Next Steps ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED ELEKTRA Summary  ELEKTRA study achieved its primary endpoint with high statistical significance (P=0.0007) Strong efficacy results in Dravet syndrome with a 46% median difference from placebo in convulsive seizure frequency reduction (P=0.0007)  Numerical reductions in drop seizure frequency in Lennox-Gastaut syndrome vs. placebo  Soticlestat continues to be well-tolerated with no new safety signals identified Reduction of plasma 24HC levels was observed with soticlestat treatment vs. placebo Next Steps Initiate Phase 3 registrational program of soticlestat in Dravet syndrome  Data analysis ongoing from the Lennox-Gastaut syndrome patients Full data to be presented at future scientific meetings

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2020: A Transformative Year for Ovid ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED OV101:  Fragile X syndrome – Phase 2 ROCKET trial positive topline data  4Q 2020: Angelman syndrome - Pivotal Phase 3 NEPTUNE trial topline data Soticlestat:  ARCADE initial data ELEKTRA topline data, ENDYMION data  3Q 2020: ARCADE data, ENDYMION data Significant Value-Creating Events During 2020

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Q&A ©2020 OVID THERAPEUTICS INC. | ALL RIGHTS RESERVED