GABAA receptor agonist(Hinye Pharmaceutical)

Announced signing of transaction transferring Ovid’s 50% of global rights to develop and commercialize soticlestat (TAK-935/OV935) to Takeda, under which Ovid is eligible to receive up to $856 million in payments, including an upfront payment of $196 million at closing Two pivotal Phase 3 trials of soticlestat in Dravet syndrome and Lennox-Gastaut syndrome on track to commence in the second quarter of 2021, and upon the successful closing of the transaction, to be fully funded and conducted by Takeda Cash balance, upon successful closing of the transaction, provides resources to develop and continue to build an exciting pipeline and platform of technologies NEW YORK, March 15, 2021 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID) (“Ovid”), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today reported financial results for the fourth quarter and full year ended December 31, 2020 and provided an overview of the Company’s recent progress. “This new agreement with Takeda strategically positions Ovid, upon closing, to advance our next generation neuroscience pipeline, while demonstrating the value we helped to create for soticlestat by bringing it to the initiation of two pivotal Phase 3 trials,” said Jeremy M. Levin, DPhil, MB, BChir, Chairman and Chief Executive Officer of Ovid Therapeutics. “Importantly, we believe this is a positive outcome for patients, potentially accelerating soticlestat’s clinical development with the potential to make this new treatment commercially available in 2024. We are excited for next steps both for this asset and for our pipeline, as we work to file three INDs over the next three years and in tandem pursue an active business development initiative.” Corporate Update In March 2021, Ovid announced that it entered into an agreement with Takeda Pharmaceutical Company Limited (“Takeda”) under which Takeda will secure global rights at closing from Ovid to develop and commercialize the investigational medicine soticlestat (TAK-935/OV935) for the treatment of developmental and epileptic encephalopathies (the “transaction”), including Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). On closing, Takeda will assume sole responsibility for further worldwide development and commercialization, and Ovid will no longer have any financial obligation to Takeda. Ovid will receive an upfront payment of $196 million at closing and is eligible to receive up to an additional $660 million upon achieving development, regulatory and sales milestones. In addition, if soticlestat achieves regulatory approval, Ovid will receive tiered royalties beginning in the low double-digits and up to 20 percent on net sales of soticlestat, subject to standard reductions in certain circumstances. The new agreement is expected to close by end of March 2021 subject to the satisfaction of customary closing conditions, including review by the appropriate regulatory agencies under the Hart-Scott-Rodino Act. Upon closing of the transaction with Takeda, all of Ovid’s financial obligations to Takeda and the soticlestat program will be terminated resulting in an estimated savings of at least $250 million. Ovid will not incur any additional expenditures relating to the development and commercialization of soticlestat. As a result of the transaction with Takeda, Ovid expects to reduce its cash spend significantly at closing. The Company anticipates quarterly operating expenses, excluding non-cash expenses, to be in the range of $8 million to $10 million, following the closing of the transaction with Takeda. Pipeline Update OV101 (gaboxadol): In December 2020, Ovid announced that topline results from the Phase 3 NEPTUNE clinical trial of OV101 (gaboxadol) for the treatment of Angelman syndrome did not achieve the primary endpoint. Ovid is completing a full analysis of the study along with the open label extension study (ELARA), and will discuss these results with the U.S. Food and Drug Administration (FDA). In December 2020, Ovid announced that topline results from the Phase 3 NEPTUNE clinical trial of OV101 (gaboxadol) for the treatment of Angelman syndrome did not achieve the primary endpoint. Ovid is completing a full analysis of the study along with the open label extension study (ELARA), and will discuss these results with the U.S. Food and Drug Administration (FDA). OV935/TAK935 (soticlestat): The two pivotal Phase 3 studies for both DS and LGS are currently on track to commence in the second quarter of 2021, and upon closing of the transaction will be fully operated and funded by Takeda. If successful, soticlestat could be commercially available as early as 2024. The two pivotal Phase 3 studies for both DS and LGS are currently on track to commence in the second quarter of 2021, and upon closing of the transaction will be fully operated and funded by Takeda. If successful, soticlestat could be commercially available as early as 2024. Over the past 18 months, Ovid has assembled a robust early-stage pipeline and plans to submit three IND applications over the next three years starting next year. OV329: A highly selective small molecule GABA aminotransferase (GABA-AT) inhibitor that will be developed for the treatment of seizures associated with tuberous sclerosis complex and infantile spasms. IND-enabling studies are anticipated in 2021 and the Company plans to submit an IND in the first half of 2022. OV882: This asset explores short hairpin RNA and its ability to interfere with UBE3A antisense that blocks UBE3A gene expression in neurons. In partnership with the University of Connecticut, Ovid is exploring the use of OV882 as a noncoding RNA construct that reduces expression of UBE3a-antisense and restores UBE3A expression via the paternal gene. Based on encouraging discovery data to date, the Company expects to select and name a development candidate by the end of 2021 and enter IND-enabling studies. OV815: Currently in target validation, OV815 is a gene-modulation approach focused on the kinesin family of proteins initially targeting KIF1A. This asset is part of a collaboration with the Genomics Center at Columbia University. Fourth Quarter and Year Ended December 31, 2020 Financial Results As of December 31, 2020, cash, cash equivalents and short-term investments totaled $72.0 million, including the receipt of a $5.0 million milestone payment from Angelini Pharma Rare Diseases AG (“Angelini”) during the fourth quarter, for the completion of the technology transfer and compound delivery of OV101 in Angelman syndrome in accordance with Ovid’s license agreement with Angelini. Revenue of $5.7 million was recognized during the fourth quarter of 2020, as compared to zero for the same period in 2020. Revenue of $12.6 million was recognized for the full year 2020, as compared to zero for the same period in 2020. Revenue recognized during 2020 was related to Ovid’s license agreement with Angelini. Research and development expenses were $16.9 million and $63.4 million for the fourth quarter and year ended December 31, 2020, respectively, as compared to $12.1 million and $42.2 million for the same periods in 2019. The increase for the year ended December 31, 2020, was primarily due to an increase in development activities related to our OV101 and OV935 development programs. General and administrative expenses were $10.4 million and $30.6 million for the fourth quarter and year ended December 31, 2020, respectively, as compared to $5.2 million and $19.3 million for the same periods in 2019. The difference was primarily due to increases in legal fees, compliance and pre-commercialization expenses and professional fees. The Company reported a net loss of $22.0 million, or basic and diluted net loss per share attributable to common stockholders of $0.34, for the fourth quarter of 2020, as compared to a net loss of $17.0 million, or basic and diluted net loss per share attributable to common stockholders of $0.35, for the same period in 2019. The Company reported a net loss of $81.0 million, or basic and diluted net loss per share attributable to common stockholders of $1.39 for the year ended December 31, 2020, compared to a net loss of $60.5 million, or basic and diluted net loss per share attributable to common stockholders of $1.54, for the year ended December 31, 2019. 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. We believe these disorders represent an attractive area for drug development as the understanding of the underlying biology has grown meaningfully over the last few years and today represents a substantial opportunity medically and commercially. Based on recent scientific advances in genetics and the biological pathways of the brain, we aim to identify, discover and acquire novel compounds for the treatment of rare neurological disorders. We have built a deep knowledge of such disorders, how to treat them and how to develop the clinically meaningful endpoints required for development of a compound in these disorders. As a result of this knowledge, Ovid has developed a robust pipeline of first-in-class compounds and programs. We continue to execute on our strategy to build this pipeline by discovering, in-licensing and collaborating with leading biopharmaceutical companies and academic institutions. These pipeline programs include programs targeting rare epilepsies, Angelman syndrome and Fragile X syndrome, as well as early-stage programs into other monogenetic disorders. Ovid’s most advanced pipeline programs include OV935 (soticlestat) in collaboration with Takeda and OV101 a δ-selective GABA A receptor agonist. Ovid’s emerging pipeline programs include OV329, a small molecule GABA aminotransferase inhibitor for seizures associated with Tuberous Sclerosis Complex and Infantile Spasms; OV882, a short hairpin RNA therapy approach for Angelman syndrome; OV815, a genetic therapy approach for KIF1A associated neurological disorder; and other non-disclosed researched targets. For more information on Ovid, please visit . 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 and Ovid’s other programs and potential programs, the closing of the Royalty, License and Termination Agreement and the potential value, benefits, and outcome of the collaboration with Takeda. 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, 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, and the ability to commercialize soticlestat. 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. Condensed Consolidated Statements of Operations (Unaudited) Selected Condensed Balance Sheet Data (Unaudited) 1Working capital defined as current assets less current liabilities. Contacts Ovid Investors and Media Ovid Therapeutics Inc. Investor Relations & Public Relations irpr@ovidrx.com Or Ovid Investors: Argot Partners Maeve Conneighton/Dawn Schottlandt 212-600-1902 Ovid@argotpartners.com Ovid Media Dan Budwick 1AB dan@1abmedia.com

NEW YORK, June 19, 2020 (GLOBE NEWSWIRE) --Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to OV101 (gaboxadol) for the treatment of Angelman syndrome. OV101 is believed to be the only delta (δ)-selective GABA A receptor agonist in development and is currently being evaluated in the Company’s pivotal Phase 3 NEPTUNE trial in Angelman syndrome, with topline results expected in the fourth quarter of 2020. The FDA has previously granted Orphan Drug and Fast Track designations for OV101 for the treatment of Angelman syndrome. Under the Creating Hope Act passed into federal law in 2012, the FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S. If a new drug application (NDA) for OV101 in Angelman syndrome is approved, Ovid may be eligible to receive a priority review voucher from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application or may be transferred and/or sold to other companies for their programs, such as has recently been done by other voucher recipients. “OV101 has the potential to become the first FDA-approved therapy for individuals living with Angelman syndrome. Receiving Rare Pediatric Disease Designation from the FDA is a significant milestone for this program and underscores the critical value of our work,” said Amit Rakhit, M.D., MBA, President and Chief Medical Officer of Ovid Therapeutics. “Importantly, with this designation, we may be eligible to receive a priority review voucher from the FDA, providing significant value as we work diligently towards the completion of our ongoing pivotal Phase 3 NEPTUNE trial. We are grateful to the FDA and Congress for having enacted this law which helps Ovid and all companies developing innovative drugs for rare pediatric conditions.” About Angelman Syndrome Angelman syndrome is a rare genetic condition that is characterized by a variety of signs and symptoms. Characteristic features of this condition include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. The most common cause of Angelman syndrome is the loss of function of the gene that codes for ubiquitin protein ligase E3A (UBE3A), which plays a critical role in nerve cell communication, resulting in impaired tonic inhibition. Individuals with Angelman syndrome typically have normal lifespans but are unable to live independently. Therefore, they require constant support from a network of specialists and caregivers. Angelman syndrome affects approximately 1 in 12,000 to 1 in 20,000 people globally. There are no approved therapies by the FDA, European Medicines Agency (EMA), or rest of world for Angelman syndrome, and treatment primarily consists of behavioral interventions and pharmacologic management of symptoms. Angelman syndrome is associated with a reduction in tonic inhibition, a function of the delta (δ)-selective GABA A receptor that allows a human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. If tonic inhibition is reduced, the brain becomes inundated with signals and loses the ability to separate background noise from critical information. About OV101 (gaboxadol) OV101 is believed to be the only delta (δ)-selective GABA A receptor agonist in development and the first investigational drug to specifically target the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders. OV101 has demonstrated in laboratory studies and animal models to selectively activate the δ-subunit of GABA A receptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through modulation of tonic inhibition. Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and thereby address several core symptoms of these conditions. In both these syndromes, the underlying pathophysiology includes disruption of tonic inhibition modulated through the δ-subunit of GABA A receptors. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. This compound has also previously been tested in more than 4,000 patients (more than 1,000 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles. Ovid is conducting a pivotal Phase 3 clinical trial with OV101 in Angelman syndrome (NEPTUNE) and has completed a Phase 2 signal-finding clinical trial with OV101 in Fragile X syndrome (ROCKET). OV101 has received Rare Pediatric Disease Designation from the FDA for the treatment of Angelman syndrome. The FDA has also granted Orphan Drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome. In addition, the European Commission (EC) has granted orphan drug designation to OV101 for the treatment of Angelman syndrome. The U.S. Patent and Trademark Office has granted Ovid patents directed to methods of treating Angelman syndrome and Fragile X syndrome using OV101. The issued patents expire in 2035 without regulatory extensions. 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 . Forward-Looking Statements This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding: advancing and commercializing Ovid’s product candidates, progress, timing, scope and the development and potential benefits of Ovid’s product candidates; and the anticipated reporting schedule of clinical data regarding Ovid’s product candidates. 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. Contacts Investors and Media: Ovid Therapeutics Inc. Investor Relations & Public Relations irpr@ovidrx.com Or Investors: Steve Klass Burns McClellan, Inc. sklass@burnsmc.com (212) 213-0006