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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
Soticlestat
CH24H Inhibitor
RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3
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
OV329
GABA aminotransferase inhibitor
RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3
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
OV882
shRNA-551
RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3
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
OV815
RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3
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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