UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

 

FORM 8-K

 

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): August 25, 2020

 

OVID THERAPEUTICS INC.

(Exact name of Registrant as Specified in Its Charter)

 

 

Delaware

001-38085

46-5270895

(State or Other Jurisdiction

of Incorporation)

(Commission File Number)

(IRS Employer

Identification No.)

 

 

 

1460 Broadway, Suite 15044

New York, New York

 

10036

(Address of Principal Executive Offices)

 

(Zip Code)

Registrant’s Telephone Number, Including Area Code: 646-661-7661

Not Applicable

(Former Name or Former Address, if Changed Since Last Report)

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instructions A.2. below):

Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

 

Title of each class

 

Trading

Symbol(s)

 

Name of each exchange on which registered

Common Stock par value $0.001 per share

 

OVID

 

The Nasdaq Stock Market LLC

 

 

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company 

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. 

 

 


Item 8.01.Other Events.

 

On August 25, 2020, Ovid Therapeutics Inc. (the “Company”) and Takeda Pharmaceutical Company Limited issued a press release announcing positive topline data from the Phase 2 ELEKTRA study of soticlestat in children with Dravet syndrome and Lennox-Gastaut syndrome. A copy of the press release is filed as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.

 

The Company will host a conference call and live webcast presentation of the data on August 25, 2020 at 8:00 a.m. Eastern time. A copy of the presentation to be used during the webcast is filed as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated herein by reference. A recording of the webcast will be made available on the Investors section of the Company’s website at investors.ovidrx.com.

 

Item 9.01.Financial Statements and Exhibits

(d) Exhibit

 

Exhibit No.

  

Description

 

 

99.1

  

Press Release, dated August 25, 2020

99.2

 

Presentation, dated August 25, 2020

 

 


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

OVID THERAPEUTICS INC.

 

 

By:    

/s/ Thomas M. Perone

 

Thomas M. Perone

 

General Counsel & Corporate Secretary  

Dated: August 25, 2020

 

 

Exhibit 99.1

 

 

 

News Release

 

 

 

 

Phase 2 ELEKTRA Study of Soticlestat (TAK-935/OV935) Meets Primary Endpoint Reducing Seizure Frequency in Children with Dravet Syndrome or Lennox-Gastaut Syndrome

 

 

Primary endpoint achieved, demonstrating a statistically significant reduction of seizures from baseline compared to placebo (p=0.0007) in the combined Dravet syndrome and Lennox-Gastaut syndrome study population  

 

 

Statistically significant reduction in seizure frequency from baseline in Dravet syndrome cohort compared to placebo (p=0.0007); based on strong efficacy results, Takeda and Ovid plan to initiate a Phase 3 registrational program of soticlestat in Dravet syndrome

 

 

Data from Lennox-Gastaut syndrome cohort demonstrated numeric reductions in seizure frequency compared to placebo but did not achieve statistical significance (p=0.1279); data analysis ongoing for the Lennox-Gastaut syndrome patients

 

 

Soticlestat was well-tolerated and demonstrated a safety profile consistent with the findings of previous studies with no new safety signals identified

 

 

Ovid to host conference call and webcast today at 8:00 a.m. EDT

 

New York and Osaka, Japan, August 25, 2020 -- Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) and Ovid Therapeutics Inc. (NASDAQ: OVID) (“Ovid”), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced positive topline results from the randomized Phase 2 ELEKTRA study of soticlestat in children with Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS). Soticlestat is a potent, highly selective, oral, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H). It is being investigated by Ovid and Takeda for the treatment of rare developmental and epileptic encephalopathies (DEEs), a group of highly refractory epilepsy syndromes including DS and LGS.

 

The ELEKTRA study achieved its primary endpoint with high statistical significance, demonstrating a 27.8% median reduction from baseline in convulsive seizure (DS) and drop seizure (LGS) frequency compared to a

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3.1% median increase in patients taking placebo during the 12-week maintenance period (median placebo-adjusted reduction=30.5%; p=0.0007, based on the efficacy analysis set of 120 patients with seizure data in the maintenance period). In addition, DS and LGS patients treated with soticlestat demonstrated a 29.8% median reduction in convulsive seizure (DS) and drop seizure (LGS) frequency compared to 0.0% change in median seizure frequency in patients taking placebo during the full 20-week treatment period (titration plus maintenance) of the ELEKTRA study (placebo-adjusted reduction=25.1%; p=0.0024).

 

In the ELEKTRA DS cohort (n=51), patients treated with soticlestat demonstrated a 33.8% median reduction in convulsive seizure frequency compared to a 7.0% median increase in patients taking placebo during the full 20-week treatment period of the study (median placebo-adjusted reduction in seizure frequency is 46.0%; p=0.0007). Based on these data, the companies plan to meet with regulatory authorities to discuss initiation of a Phase 3 registrational program for soticlestat in patients with DS.

 

In the ELEKTRA LGS cohort (n=88), patients treated with soticlestat demonstrated a 20.6% median reduction in drop seizure frequency compared to a 6.0.% median reduction in patients taking placebo during the full 20-week treatment period of the study (median placebo-adjusted reduction in seizure frequency is 14.8%; p=0.1279). Additional analyses are being conducted to better understand the potential next steps for the development of soticlestat in this highly heterogenous patient population.

 

Soticlestat was generally well-tolerated in the ELEKTRA study and demonstrated a safety profile consistent with those of previous studies, with no new safety signals identified. All patients who completed the ELEKTRA study elected to enroll into the ENDYMION open-label extension study and findings from ENDYMION are also reported today.

 

“We are extremely encouraged by these results, which show a clear statistically significant reduction of seizures in Dravet syndrome patients treated with soticlestat, as well as a trend for seizure reduction in Lennox-Gastaut patients,” said Amit Rakhit, M.D., MBA, President and Chief Medical Officer of Ovid. “We look forward to continuing our collaboration with Takeda to initiate a Phase 3 registrational program for soticlestat in patients with DS, while continuing to analyze the data from patients with LGS in the ELEKTRA and ENDYMION studies to define potential next steps. We also expect to report data from the open-label Phase 2 ARCADE study with soticlestat in patients with CDKL5 deficiency disorder and Dup15q syndrome, two other types of highly-refractory DEEs, later this quarter.”

 

“It is exciting to see these positive results and to advance soticlestat into late stage clinical development -- initially for the potential treatment of children with Dravet syndrome who need more options to manage treatment-resistant seizures,” said Sarah Sheikh, M.D., M.Sc., MRCP, Head, Neuroscience Therapeutic Area Unit at Takeda. “Soticlestat and its novel mechanism of action were discovered at Takeda and we are

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enthusiastic about continuing to advance the science and clinical programs as one aligned team, in strong partnership with Ovid Therapeutics.”

 

“Children with developmental epileptic encephalopathies like DS and LGS need more options to manage their treatment-resistant seizures,” said Dr. Cecil Hahn, M.D., MPH, a Child Neurologist at The Hospital for Sick Children and Associate Professor of Pediatrics at the University of Toronto. “The results of the ELEKTRA study are very promising, particularly for children with DS and represent a clinically significant reduction in seizure burden. Moreover, soticlestat was well-tolerated in this study."  

 

Phase 2 ELEKTRA Study Design and Patient Baseline Demographics

ELEKTRA was an international, multi-center, randomized, double-blind, placebo-controlled study designed to evaluate treatment with soticlestat in pediatric patients, aged 2 to 17 years, with highly refractory epileptic seizures associated with DS (convulsive seizures) or LGS (drop seizures). The study consisted of a four- to six-week screening period to establish baseline seizure frequency, followed by a 20-week double-blind treatment period, including an 8-week dose optimization period and a 12-week maintenance period. During the 8-week dose optimization period, patients were titrated from 100mg twice daily (BID), to 200mg BID to 300mg BID (mg/kg dosing for <60 kg) of orally administered soticlestat.

 

A total of 141 patients were enrolled in ELEKTRA and 126 completed the study. A modified intent-to-treat (mITT) analysis of 139 patients was performed to evaluate the efficacy endpoints, which includes any patient who enrolled in the study and received at least one dose of study drug. Patients in the study were allowed to be on one to four concomitant anti-epileptic drugs (AEDs), with the majority of patients concomitantly treated with at least three AEDs. The most common AEDs taken by the patients were valproate, clobazam, levetiracetam and topiramate.

 

Phase 2 ELEKTRA Topline Efficacy Results

The study achieved its primary endpoint, demonstrating a 27.8% median reduction from baseline in convulsive seizure (DS) and drop seizure (LGS) frequency compared to a 3.1% median increase in patients on placebo during the 12-week maintenance period (median placebo-adjusted reduction=30.5%; p=0.0007, based on the efficacy analysis set of 120 patients with seizure data in the maintenance period). During the full 20-week treatment period of the mITT DS patient population, the median percent change from baseline was a 33.8% decrease in seizure frequency compared to a 7.0% increase in seizure frequency for patients receiving placebo (median placebo-adjusted reduction=46.0%; p=0.0007). During the full treatment period of the mITT LGS patient population, the median percent change from baseline was a 20.6% decrease in seizure frequency compared to a 6.0% decrease in patients receiving placebo (median placebo-adjusted reduction=14.8%; p=0.1279).

 

Phase 2 ELEKTRA Topline Safety Results

Soticlestat was well tolerated in this study. These findings were consistent with previous studies and no new safety signals were identified. The incidence of treatment emergent adverse events was similar in both the

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treatment and placebo groups with 57 (80.3%) of soticlestat patients experiencing at least one treatment emergent adverse event compared to 52 (74.3%) placebo patients. The most frequent treatment emergent adverse events reported in soticlestat-treated patients with ≥5% difference from placebo were lethargy and constipation. The incidence of serious adverse events was similar in both soticlestat and placebo groups, with 11 (15.5%) in soticlestat experiencing at least one treatment emergent serious adverse event compared to 13 (18.6%) in placebo. There were no deaths reported.

 

ENDYMION Open-Label Extension Study Update

All patients who completed the ELEKTRA trial elected to roll over into the ENDYMION open-label extension study. The primary objective of ENDYMION is to assess the long-term safety and tolerability of soticlestat over four years of treatment in patients with rare epilepsies and, secondarily, to evaluate the effect of soticlestat on seizure frequency over time.

 

Data from the ELEKTRA patients who have rolled over into the ENDYMION study are supportive of results in the core study. The data indicate maintenance of effect over 6 months in those patients originally randomized to soticlestat, and similarly reduced seizure frequency as compared to baseline in those patients previously assigned to the placebo arm. No new safety signals were identified in ENDYMION. 

 

About Soticlestat (TAK-935/OV935)

Soticlestat is a potent, highly selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H), with the potential to reduce seizure susceptibility and improve seizure control. CH24H is predominantly expressed in the brain, where it converts cholesterol into 24S-hydroxycholesterol (24HC) to adjust the homeostatic balance of brain cholesterol. 24HC is a positive allosteric modulator of the NMDA receptor and modulates glutamatergic signaling associated with epilepsy. Glutamate is one of the main neurotransmitters in the brain and has been shown to play a role in the initiation and spread of seizure activity. Recent literature indicates that CH24H is involved in over-activation of the glutamatergic pathway through modulation of the NMDA channel and that increased expression of CH24H can disrupt the reuptake of glutamate by astrocytes, resulting in epileptogenesis and neurotoxicity. Inhibition of CH24H by soticlestat reduces the neuronal levels of 24HC and may improve excitatory/inhibitory balance of NMDA channel activity.

 

Takeda and Ovid are sharing in the development and commercialization costs of soticlestat on a 50/50 basis and, if successful, the companies will share in the profits on a 50/50 basis. Takeda will be responsible for commercialization in Japan and has the option to be responsible for commercialization in other countries in Asia and other selected countries. Ovid will be responsible for clinical development activities and commercialization of soticlestat in the United States, Europe, Canada and Israel. Under the terms of the agreement, Takeda received equity in Ovid and may be eligible to receive certain milestone payments based on the advancement of soticlestat.

 

 

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About Dravet Syndrome and Lennox-Gastaut Syndrome

Dravet syndrome and Lennox-Gastaut syndrome are types of developmental and epileptic encephalopathies (DEEs), a heterogeneous group of rare epilepsy syndromes. Dravet and Lennox-Gastaut syndrome typically become apparent during infancy or early childhood and are highly refractory to many antiseizure medications.

 

Dravet syndrome is most commonly caused by a genetic mutation in the SCN1A gene and affects approximately 1 in 15,000 to 1 in 21,000 people in the United States. Dravet syndrome is characterized by prolonged focal seizures that can evolve to convulsive tonic-clonic seizures. Children with Dravet syndrome experience developmental disabilities as seizures increase. Other common symptoms include changes in appetite, difficulty balancing and a crouched gait when walking.  

 

Lennox-Gastaut syndrome is estimated to affect approximately 1 in 11,000 people in the United States. Lennox-Gastaut syndrome is a heterogeneous condition and characterized by several different types of seizures, most commonly atonic (drop), tonic and atypical absence seizures. Children with Lennox-Gastaut syndrome may also develop cognitive dysfunction, delays in reaching developmental milestones and behavioral problems. Lennox-Gastaut syndrome can be caused by a variety of underlying conditions, but in some cases no cause can be identified.

 

Ovid Conference Call and Webcast Information

Ovid Therapeutics will host a live conference call and webcast today at 8:00 a.m. Eastern Time. The live webcast can be accessed by visiting the Investors section of the Company’s website at investors.ovidrx.com. Alternatively, please call 866-830-1640 (U.S.) or 210-874-7820 (international) to listen to the live conference call. The conference ID number for the live call is 7926217. A replay of the webcast will be available on the Company’s website following the live conference call.

 

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries.

For more information, visit https://www.takeda.com.

 

Important Notice

For the purposes of this notice, “press release” means this document, any oral presentation, any question and

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answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws.

The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, “Takeda” is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words “we”, “us” and “our” are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.

 

Takeda Forward-Looking Statements

This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takeda’s future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as “targets”, “plans”, “believes”, “hopes”, “continues”, “expects”, “aims”, “intends”, “ensures”, “will”, “may”, “should”, “would”, “could” “anticipates”, “estimates”, “projects” or similar expressions or the negative thereof. These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takeda’s global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takeda’s operations and the timing of any such divestment(s); and other factors identified in Takeda’s most recent Annual Report on Form 20-F and Takeda’s other reports filed with the U.S. Securities and Exchange Commission, available on Takeda’s website at: https://www.takeda.com/investors/reports/sec-filings/ or at www.sec.gov. Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an

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estimate, forecast, guarantee or projection of Takeda’s future results.

 

About Ovid Therapeutics

Ovid Therapeutics Inc. is a New York-based biopharmaceutical company using its BoldMedicine® approach to develop medicines that transform the lives of patients with rare neurological disorders. Ovid has a broad pipeline of potential first-in-class medicines. The Company’s most advanced investigational medicine, OV101 (gaboxadol), is currently in clinical development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid is also developing OV935 (soticlestat) in collaboration with Takeda Pharmaceutical Company Limited for the potential treatment of rare developmental and epileptic encephalopathies (DEE). For more information on Ovid, please visit www.ovidrx.com.

 

Ovid Forward-Looking Statements

This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding the potential benefits, clinical and regulatory development and commercialization of soticlestat, the potential value and benefits of the collaboration with Takeda, the anticipated reporting schedule of clinical data, the likelihood that data will support future development, and the association of data with treatment outcomes. You can identify forward-looking statements because they contain words such as “will,” “appears,” “believes” and “expects.” Forward-looking statements are based on Ovid’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include uncertainties in the development and regulatory approval processes, and 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 of the clinical outcomes may materially change as patient enrollment continues and/or more patient data become available. Additional risks that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovid’s filings with the Securities and Exchange Commission under the caption “Risk Factors.” Such risks may be amplified by the COVID-19 pandemic and its potential impact on Ovid’s business and the global economy. Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

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Takeda Media Contacts:

 

Japanese Media

Kazumi Kobayashi

kazumi.kobayashi@takeda.com

+81 (0) 3-3278-2095

Media outside Japan

Chris Stamm

chris.stamm@takeda.com

+1 (617) 347-7726

 

 

Ovid Investors and Media:

 

Ovid Therapeutics Inc.

Investor Relations & Public Relations

irpr@ovidrx.com

 

Or

 

 

Ovid Investors:

Ovid Media:

Steve Klass

Burns McClellan, Inc.

+1 (212) 213-0006

sklass@burnsmc.com

Dan Budwick

1AB

dan@abmedia.com

 

 

# # #

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SLIDE 0

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

SLIDE 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 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

SLIDE 2

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

SLIDE 3

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

SLIDE 4

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

SLIDE 5

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

SLIDE 6

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

SLIDE 7

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

SLIDE 8

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