-- First scientific presentation of OV-935 (TAK-935) safety and tolerability 
data in developmental and epileptic encephalopathies from the Phase 1b/2a 
clinical trial 

-- Study met primary safety and tolerability endpoints and showed initial 
evidence of potential efficacy in seizure reduction

-- Data encouraging for plasma 24HC as a potential biomarker for assessing 
treatment outcome and disease management

Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to 
developing medicines that transform the lives of people with rare neurological 
diseases, will present data on safety, tolerability, exploratory and secondary 
outcomes from the Phase 1b/2a clinical trial of OV935/TAK-935 in adult patients 
with developmental and epileptic encephalopathies (DEE). The data will be 
presented as a poster at the 12th International Epilepsy Colloquium (IEC), 
taking place May 26-28 in Lyon, France. 

Logo - https://mma.prnewswire.com/media/892228/Ovid_Therapeutics_Inc_Logo.jpg 

"DEE refers to a group of rare conditions characterized by cognitive impairment 
and severe epilepsy.1 Each of these conditions can worsen over time and cause 
significant morbidities for patients and their families so there is an urgent 
need for new medicines in this area," said Amit Rakhit, MD, MBA, Chief Medical 
Officer and Head of Research & Development at Ovid. "Together with Takeda, we 
believe that OV935 may have the potential to treat rare epilepsies and are 
committed to making a difference for patients living with these diseases."

The randomized, double blind, placebo-controlled Phase 1b/2a clinical trial 
achieved its primary endpoint of safety and tolerability and showed OV935 was 
generally well tolerated. During the open-label period, 11 patients (68.8%) 
experienced a treatment-emergent adverse event (TEAE), of which the majority 
were mild. There were five serious TEAEs, all seizure cluster related. Overall, 
the data are consistent with a favorable safety and tolerability profile and 
support the continued clinical development of OV935.

Ovid is committed to grounding innovative research by first understanding 
biological pathways and their central role in rare neurological disorders in 
order to develop clinically-relevant endpoints that measure the efficacy of 
investigational treatments and deliver tangible patient benefits. Exploratory 
endpoints in the Phase 1b/2a trial included changes in seizure frequency and 
plasma 24S-hydroxycholesterol (24HC) levels.  OV935 showed initial evidence of 
potential efficacy with a reduction in seizure frequency and a reduction in 
mean 24HC levels. This preliminary evidence suggests that plasma 24HC may be a 
promising biomarker for assessing treatment outcome and disease management.

Details of the presentation are listed below.

Title: A phase 1b/2a study of TAK-935 (OV935) as adjunctive therapy in patients 
with developmental and epileptic encephalopathies (DEE)
Poster Number: 45 
Presentation Date and Time: Sunday, May 26, 2019 at 1:30 p.m. - 3:30 p.m. CEST

Previously announced by Ovid in its December 17, 2018 press release, these 
results were also presented as an invited speakership at the 15th Antiepileptic 
Drug and Device Trials Conference, May 22-24, Miami, Fl. The IEC presentation 
and the invited speakership demonstrate a burgeoning interest in rare 
epilepsies and the hope that OV935 might be a potential treatment option. 

About Developmental and Epileptic Encephalopathies
The term developmental and epileptic encephalopathy includes a group of 
epilepsy syndromes associated with severe cognitive and behavioral 
disturbances. The International League Against Epilepsy (ILAE) defines an 
epileptic encephalopathy as a condition in which "the epileptiform EEG 
abnormalities themselves are believed to contribute to a progressive 
disturbance in cerebral function."

These epilepsies cause significant morbidities for patients beyond what might 
be expected from the known underlying pathology alone and can worsen over time. 
Developmental and epileptic encephalopathies typically present early in life 
and are often associated with severe cognitive and developmental impairment in 
addition to frequent treatment-resistant seizures throughout the person's 
lifetime. These disorders vary in age of onset, developmental outcomes, 
etiologies, neuropsychological deficits, electroencephalographic (EEG) 
patterns, seizure types, and prognosis.  

Despite the availability of medicines for epilepsy, few treatment options are 
available for epileptic encephalopathies, and novel therapies are needed.

About Investigational OV935/TAK-935
OV935/TAK-935 is a potent, highly-selective, first-in-class inhibitor of the 
enzyme cholesterol 24-hydroxylase (CH24H) being investigated as an 
anti-epileptic drug (AED). CH24H is predominantly expressed in the brain, where 
it plays a central role in cholesterol homeostasis. CH24H converts cholesterol 
to 24-hydroxycholesterol (24HC), which then exits the brain into the peripheral 
circulation. 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 CH24H is involved in over-activation of the 
glutamatergic pathway through modulation of the NMDA channel, implying its 
potential role in central nervous system diseases such as epilepsy. In 
addition, preclinical data suggest OV935 has an anti-inflammatory and glial 
modulatory function with resultant anti-epileptogenic not simply a direct 
seizure suppressive activity. Ovid and Takeda believe that the novel mechanism 
of action of OV935 may potentially treat rare epilepsies by modifying disease 
pathology over time and might ultimately be considered an early treatment 
option in view of this mechanism. To Ovid and Takeda's knowledge, OV935 is the 
only molecule with this mechanism of action in clinical development. OV935 is 
an investigational drug, not approved for commercial use.

The United States Food and Drug Administration (FDA) has granted orphan drug 
designation to OV935 for the treatment of both Dravet syndrome and LGS.

About the Ovid/Takeda Collaboration
Ovid and Takeda entered into a global development and commercialization 
collaboration in January 2017 to evaluate OV935/TAK-935 across a range of rare 
epilepsy syndromes. Under the terms of the agreement, the companies share in 
the development and commercialization costs on a 50/50 basis and, if 
successful, the companies will share in the profits on a 50/50 basis. Takeda 
will lead commercialization in Japan and has the option to lead in Asia and 
other selected geographies. Ovid leads clinical development activities and 
commercialization in the United States, Europe, Canada and Israel.

About Ovid Therapeutics
Ovid Therapeutics (NASDAQ: OVID) is a New York-based biopharmaceutical company 
using its BoldMedicine(TM) 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, is currently in clinical development for the 
treatment of Angelman syndrome and Fragile X syndrome. Ovid is also developing 
OV935/TAK-935 in collaboration with Takeda Pharmaceutical Company Limited for 
the potential treatment of rare developmental and epileptic encephalopathies 
(DEE).

[1] Source: I.E. Scheffer, S. Berkovic, G. Capovilla, et al. ILAE 
classification of the epilepsies: Position paper of the ILAE Commission for 
Classification and Terminology. Epilepsia; 58 (2017): 512-521. 
https://onlinelibrary.wiley.com/doi/full/10.1111/epi.13709

For more information on Ovid, please visit http://www.ovidrx.com/. 

Forward-Looking Statements 
This press release includes certain disclosures that contain "forward-looking 
statements," including, without limitation, statements regarding advancing 
Ovid's product candidates, progress, timing, scope and results of clinical 
trials for Ovid's product candidates, and the reporting of clinical data 
regarding Ovid's product candidates. You can identify forward-looking 
statements because they contain words such as "will," "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 are set forth in Ovid's 
filings with the Securities and Exchange Commission under the caption "Risk 
Factors". Ovid assumes no obligation to update any forward-looking statements 
contained herein to reflect any change in expectations, even as new information 
becomes available.

SOURCE: Ovid Therapeutics Inc.

CONTACT: Investors: Investor Relations & Public Relations, Ovid Therapeutics 
Inc., irpr@ovidrx.com; Media: Investor Relations & Public Relations, Ovid 
Therapeutics Inc., irpr@ovidrx.com; Jenn Gordon, Spectrum Science 
Communications, jgordon@spectrumscience.com, +1 (202) 587-2580