Ovid Therapeutics is focused on developing innovative small molecule therapies aimed at addressing neurological diseases, particularly epilepsies and brain conditions. Their approach involves creating targeted treatments that aim to reduce seizures while enhancing safety and tolerability. The company is cultivating a differentiated pipeline of compounds, including OV329, a GABA-aminotransferase inhibitor, and OV350, which targets the KCC2 transporter. These compounds are designed to restore balance in the brain by mitigating neuronal hyperexcitability, a key factor in various debilitating neurological disorders. Ovid's commitment to understanding the complexities of neurological systems allows them to translate preclinical findings into meaningful clinical applications.

In addition to their internal research and development efforts, Ovid Therapeutics actively engages in collaborations with other organizations to enhance their therapeutic offerings. Notable partnerships include a collaboration with Graviton to advance the development of OV888, a selective ROCK2 inhibitor, and an exclusive license agreement with AstraZeneca for a portfolio of KCC2-targeting compounds. These collaborations are aimed at addressing a range of neurological conditions characterized by seizures and other related symptoms. Ovid's strategic alliances with academic institutions and industry partners underscore their dedication to pioneering effective treatments and improving the lives of individuals affected by neurological disorders.

United States

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.

Ovid’s most advanced investigational medicine, OV101 (gaboxadol), is currently in phase 3 clinical development for the treatment of Angelman syndrome with data expected in the 4Q 2020 and in earlier clinical development for Fragile X syndrome. Ovid is also developing OV935 (soticlestat) in collaboration with Takeda Pharmaceutical Company Limited for the treatment of rare developmental and epileptic encephalopathies (DEEs) and expects to initiate pivotal trials for OV935 in 2021. 

Central Nervous System (CNS)

Genetic Diseases

Ovid Therapeutics

Dravet syndrome

Lennox-Gastaut syndrome

Rare diseases

Rare genetic disease

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.

Soticlestat (OV935)

Cerebral cavernous malformation

OV329 represents a potential best-in-class GABA-AT inhibitor designed to supplant vigabatrin.

OV888 (GV101)

Epilepsy

OV329 is a potential next-generation GABA-AT inhibitor being developed for the potential treatment of rare and treatment-resistant forms of epilepsy and seizures, such as seizures associated with status epilepticus, tuberous sclerosis complex, infantile spasms and conditions with focal onset seizures. Low levels of GABA, the primary inhibitory neurotransmitter in the brain, have been linked to neuronal hyperexcitability. OV329 is believed to work by reducing the activity of GABA-AT, thereby increasing levels of GABA in the brain, and potentially suppressing neuronal hyperexcitability known to cause seizures. OV329 may be a potential best-in-class GABA-AT inhibitor.

OV329 (GABA-AT inhibitor)

Dementia

Parkinson's disease

OV4071

Neurological disorder

Psychiatric disorder

OV350

Angelman Syndrome

Genetic disorder

OV101 (gaboxadol) is believed to be the only delta (δ)-selective GABAA&nbsp;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 been demonstrated in laboratory studies and animal models to selectively activate the δ-subunit of GABAA&nbsp;receptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through tonic inhibition.
Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and thereby address the core symptoms of these disorders. In both these syndromes, the underlying pathophysiology includes disruption of the tonic inhibition modulated through the δ-subunit of GABAA&nbsp;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 over 4,000 patients (over 1,000 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles. In 2018, Ovid announced the successful completion of its Phase 2 (STARS) trials of OV101 in adults and adolescents with Angelman syndrome. Ovid is conducting the pivotal Phase 3 (NEPTUNE) clinical trial in Angelman syndrome. In addition, Ovid is conducting a Phase 2 clinical trial (ROCKET) in Fragile X syndrome with results expected around year-end 2019 or early 2020.
The&nbsp;FDA&nbsp;has granted Orphan Drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome. 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.

OV101 (δ-selective GABAA receptor agonist) (gaboxadol)

Fragile X syndrome

OV101 is believed to be the only delta (δ)-selective GABAA 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 been demonstrated in laboratory studies and animal models to selectively activate the δ-subunit of GABAA 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 disorders. In both these syndromes, the underlying pathophysiology includes disruption of tonic inhibition modulated through the δ-subunit of GABAA 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).
The FDA has granted Orphan Drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome. 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.

OV101 (gaboxadol) 

Cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder

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. To Ovid’s knowledge, soticlestat is the only molecule with this mechanism of action in clinical development as an anti-epileptic drug (AED).

Ovid Therapeutics

Company Details