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our pipeline

Our unique understanding of the complexities of neurologic systems allows us to turn clinical potential into clinical meaning. The Ovid pipeline includes several candidates that would potentially be the first to make a meaningful impact in the lives of individuals with certain rare neurological conditions, and all address significant markets.

product candidate
δ-selective GABAA receptor agonist
R P P1 P2 P3
Angelman syndrome

OV101 (gaboxadol) in Angelman syndrome

OV101 (gaboxadol) is a delta (δ)-selective GABAA receptor agonist. These receptors are thought to have a central role in tonic inhibition, a key physiological process of the brain believed to be a core pathophysiology underlying certain neurodevelopmental disorders.

Tonic inhibition is crucial as it modulates the ability of neurons to distinguish a real signal from extraneous noise—the imbalance in tonic inhibition leads to the many of the symptoms we see in AS.

About angelman syndrome

Angelman syndrome (AS) is a genetic disorder in which the still-developing brains of young children are overloaded with excitatory neuronal signals, leading to a range of severe neurological symptoms affecting behavior, sleep, learning, motor skills and communication, and which are present throughout life. Many people with AS also suffer from epilepsy. The key deficits are in:

Behavior: Heightened hyperactivity and anxiety leading to maladaptive behaviors such as biting and pinching

Sleep: Increased nighttime awakenings and diminished total sleep time

Learning: Impaired cognitive performance and memory loss

Motor: Uncoordinated movements, tremors, and abnormal gait limit mobility and independence with walking

Communication: Significantly delayed development with little or no verbal speech, often leading to frustration and aggression

Epilepsy: Those with epilepsy most frequently experience atypical absences, generalized tonic-clonic, atonic or myoclonic seizures. Angelman syndrome (AS) occurs when a mutated gene, UBE3A, creates a nonfunctional protein of the same name. Without functional UBE3A proteins, the levels of GABA, one of the most important neurotransmitters in the brain, are absent or low in the extrasynaptic space, which is a key area surrounding the space between nerves and where each nerve passes signals from one to the other. The absence of GABA in this area greatly reduces tonic inhibition, a critical process in neurons (brain cells) across the brain.

AS was first described in 1965—yet in over 60 years not a single treatment option has been approved. There is an urgent need for effective, disease management treatment options. The neurological dysregulation in AS results in significant impairments that can be as difficult for individuals with AS to manage as it is for their caregivers to continuously manage:

Living with AS: Individuals with AS require around-the-clock care and are unlikely to ever live independently

Caring for someone with AS: Caregivers constantly supervise their loved one with AS, often experiencing their own stress, anxiety and exhaustion in co-managing symptoms.

Fragile X syndrome

OV101 (gaboxadol) in Fragile X syndrome

OV101 is a first-in-class, oral, highly selective extrasynaptic GABAA receptor (GABAAR) agonist. It passes the blood-brain barrier and binds tightly to the unique extrasynaptic GABAA receptors on neurons throughout the brain, restoring the levels of tonic inhibition that exist when intrinsic GABAA levels are low.

OV101 is the only drug of its kind in clinical development for Fragile X syndrome. More about the unmet needs of Fragile X syndrome »

Studies of OV101 in animal models of Fragile X syndrome show that it normalizes:

⦁ Anxiety
⦁ Irritability
⦁ Repetitive behaviors
⦁ Hyperactivity

The Phase 2 study of OV101, known as the ROCKET study, has been completed and positive topline results have been released. OV101 met the study’s primary objective of safety and tolerability and produced statistically significant reductions in behavioral and functional symptoms in individuals with Fragile X syndrome. See press release >>

Reference: Cogram P, Deacon RMJ, Warner-Schmidt JL, et al. Gaboxadol normalizes behavioral abnormalities in a mouse model of Fragile X syndrome. Front Behav Neurosci 2019;13:141. DOI: 10.3389/fnbeh.2019.00141.

About Fragile X Syndrome

Fragile X syndrome (FXS) is an inherited disorder characterized by an overload of excitatory signals in the brain, leading to significant intellectual impairments that can be difficult to manage for both individuals and caregivers. The key disturbances are:

Behavior: Hyperactivity, anxiety and increased sensitivity to auditory stimuli

Sleep: Disrupted sleep patterns

Learning: Cognitive dysfunction

Motor: Reduced fine motor skills

Communication: Delayed speech development and impaired use of language (exacerbated by concomitant autism) 

Fragile X syndrome (FXS) is caused when the presence of a mutation on the FMR1 gene (located on the X chromosome) disrupts signaling pathways in nerve cells across the brain. This signaling shutdown causes the production of GABA, one of the most important neurotransmitters in the brain, to be reduced and plummet in key areas surrounding the space between nerves and where nerves pass signals from one to the other. The absence of GABA in this area greatly reduces a critical process in each cell and across all of the brain, called tonic inhibition. Tonic inhibition is crucial as it modulates the ability of cells in the brain to distinguish a real signal from noise—and without it leads to the severe symptoms of Fragile X.

There is a significant unmet medical need as there are no approved therapies that treat the underlying cause of the condition. Current treatment options are limited to physical, behavior, communication, and symptomatic therapy—as such a large, multidisciplinary team is needed to care for individuals with Fragile X, including specialists in psychiatry, psychology, neurology and genetic counselling.

In certain cases, due to the severity of the condition, caregivers provide extensive support to their loved ones with Fragile X syndrome even for basic daily activities like feeding and bathing.

product candidate
CH24H Inhibitor
R P P1 P2 P3
Dravet | LGS

Soticlestat in Dravet syndrome and Lennox-Gastaut Syndrome (LGS)

Dravet syndrome and Lennox-Gastaut syndrome are types of developmental and epileptic encephalopathies (DEEs), a heterogeneous group of rare epilepsy syndromes. Dravet syndrome is caused by a genetic mutation in the SCN1A gene. Researchers believe this mutation can lead to excess production of glutamate, an excitatory neurotransmitter, that in turn leads to seizures. LGS is a heterogeneous condition and characterized by several different types of seizures, most commonly atonic (drop), tonic and atypical absence seizures.

Soticlestat restores appropriate glutamate levels by targeting the CH24H pathway, thereby increasing the health of the central nervous system (CNS) and reducing inflammation. We believe soticlestat has the potential to reduce seizure susceptibility and improve seizure control and may provide benefit where other mechanisms of conventional anti-epileptic drugs have not.

Studies of soticlestat in animal models suggest the drug may:

⦁ Significantly reduce seizure burden
⦁ Reduce glutamate excitotoxicity
⦁ Improve cognitive function
⦁ Improve survival
⦁ Provide protection from seizure-related mortality

Ovid Therapeutics designed and conducted a robust clinical program for individuals with developmental and epileptic encephalopathies (DEE).  A Phase 1b/2a study was completed and positive results were released. Additionally, Ovid completed the Phase 2 ELEKTRA study in individuals with Lennox-Gastaut syndrome or Dravet syndrome and positive topline results have been reported.

Further development and research of soticlestat will be led by our partner Takeda, including Phase 3 studies in children and adults with Dravet syndrome and Lennox-Gastaut syndrome scheduled to begin in 2021. Additionally, the ongoing open-label ENDYMION extension study will continue under direction from our partner Takeda.

References: 1. Nishi T et al. Poster presented at: SfN 2018. 2. Hasegawa S et al. Poster presented at: SfN 2018. 3. Nishi T et al. Poster presented at: AES 2017. Abstract 2.260. 4. Hawkins NA et al. Oral presentation at: AES 2018. Abstract1.286.
product candidate
GABA aminotransferase inhibitor
R P P1 P2 P3
Seizures associated with Tuberous Sclerosis Complex and Infantile Spasms

OV329 for the treatment of seizures associated with Tuberous Sclerosis Complex and Infantile Spasms

OV329 functions by substantially reducing the activity of GABA aminotransferase (GABA-AT), a key enzyme responsible for the degradation of the brain’s major inhibitory neurotransmitter, GABA. By inhibiting the metabolism of GABA, OV329 leads to increased concentrations of GABA. Given that epilepsy is characterized by excessive neuronal excitation, the increased levels of GABA may suppress this excitatory signaling and may reduce seizures. We anticipate that if successful, OV329 could be used to treat seizures associated with Tuberous Sclerosis Complex and Infantile Spasms, and we are currently assessing the safety of this approach in the non-clinical setting.

product candidate
R P P1 P2 P3
Angelman syndrome

OV882 in Angelman syndrome

Boldly committed to the community, Ovid Therapeutics is advancing the search for therapeutic options for Angelman syndrome.

OV882 is a short hairpin RNA (shRNA-551) we are evaluating as a potential disease-modifying gene therapy for Angelman syndrome. The most common cause of Angelman syndrome is the loss of functional UBE3A protein due to a defect in the maternal copy of the UBE3A gene. Our aim is to develop a disease-modifying noncoding RNA vector that reduces expression of UBE3A antisense and restores UBE3A expression via the paternal gene copy. We are in early stages of our research.

This research program is being conducted in collaboration with the University of Connecticut School of Medicine.

product candidate
R P P1 P2 P3
KIF1A Associated Neurological Disorder (KAND)

OV815 for KIF1A Associated Neurological Disorder (KAND)

Ovid is advancing development of a potential therapeutic for KIF1A Associated Neurological Disorder (KAND). We are developing genetic therapies to target either the mutant motor protein or its RNA precursor to reduce or eliminate the impact of the non-functional KIF1A protein and restore normal axonal transport of important cargo within the neuron.

This research program is being conducted in collaboration with Columbia University Irving Medical Center.

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