Ovid Therapeutics Announces Positive Preclinical OV101 Data Demonstrating Behavioral Improvements in Fragile X Syndrome Model...
October 16 2017 - 8:00AM
Ovid Therapeutics Inc. (NASDAQ:OVID), a biopharmaceutical company
committed to developing medicines for patients with rare
neurological diseases, today announced new positive preclinical
data on OV101 that shows normalization of behavioral abnormalities
that resemble those seen in people with Fragile X syndrome. The
improvements seen with OV101, a novel agonist of the extrasynaptic
GABA receptor, were consistent across multiple behavioral measures
in a model of Fragile X syndrome. The data were presented at the
18th International Fragile X and Related Neurodevelopmental
Disorders Workshop.
“Treatment with OV101 results in significant behavioral
improvements that are consistent across all behavioral endpoints
tested,” said Matthew During, M.D., DSc, FACP, FRACP, president and
chief scientific officer of Ovid Therapeutics. “We designed this
study to deepen our understanding of the mechanisms of action and
potential benefit of OV101 to treat patients with Fragile X
syndrome.”
In the study, researchers demonstrated that acute administration
of 0.5 mg/kg of OV101 to Fmr1 knockout mice fully normalized
behavioral abnormalities relevant to Fragile X syndrome
(hyperactivity, anxiety, irritability and aggression, and
restricted and repetitive behaviors). All effects were highly
statistically significant (p < 0.001). It is believed that
symptoms of Fragile X syndrome are a result of disrupted tonic
inhibition, the key to the brain's ability to discriminate signal
from noise. The results presented indicate that by specifically
targeting the delta (δ)-subset of GABA receptors, OV101 may be able
to alleviate symptoms of Fragile X syndrome by modulating the GABA
pathway and restoring tonic inhibition.
“We have built a strong foundation in scientific analysis,
translational medicine, drug development and regulatory
capabilities. This study builds upon this foundation and
underscores the potential role of OV101 in modulating tonic
inhibition, an important underlying mechanism in certain
neurodevelopmental disorders,” said Amit Rakhit M.D., MBA, chief
medical and portfolio officer of Ovid Therapeutics. “Together with
the recent FDA orphan drug designation for OV101 for the treatment
of Fragile X syndrome, this data is another important step in our
disciplined strategy to develop OV101 as a potential first in class
compound for Fragile X syndrome.”
About OV101OV101 (gaboxadol) 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 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 tonic inhibition.
Ovid is developing OV101 for the treatment of Angelman syndrome
and Fragile X syndrome to potentially restore tonic inhibition and
relieve several of the symptoms of these disorders. In preclinical
studies, it was observed that OV101 improved symptoms of Angelman
syndrome and Fragile X syndrome. To date, gaboxadol has been tested
in over 4,000 patients (approximately 950 patient-years of
exposure) and was observed to have favorable safety and
bioavailability profiles.
The FDA granted orphan drug designation for OV101 for the
treatment of both Angelman syndrome and Fragile X syndrome. The
United States Patent and Trademark Office has granted Ovid two
patents directed to methods of treating Angelman syndrome using
OV101. The issued patents expire in 2035, without regulatory
extensions.
About Fragile X Syndrome Fragile X syndrome is
the most common inherited form of intellectual disability and
autism, with a prevalence of 1 in 3,600 to 4,000 males and 1 in
4,000 to 6,000 females in the United States. Individuals with
Fragile X syndrome often have a range of behavioral challenges,
such as cognitive impairment, anxiety, mood swings, hyperactivity,
attention deficit, poor sleep, self-injury and heightened
sensitivity to various stimuli, such as sound. Additionally,
individuals with Fragile X syndrome are prone to comorbid medical
issues including seizures and sleep disturbance. Fragile X syndrome
results from mutations in the FMR1 gene, which blocks expression of
the Fragile X Mental Retardation Protein (FMRP), an important
protein in GABA synthesis. There are no FDA-approved therapies for
Fragile X syndrome, and treatment primarily consists of behavioral
interventions and pharmacologic management of symptoms.
In studies of individuals with Fragile X syndrome and in
experimental models, extrasynaptic GABA levels are abnormally
reduced, and there is also dysregulation of GABA receptors. This
ultimately contributes to a decrease in tonic inhibition, causing
the brain to become inundated with signals and lose the ability to
separate background noise from critical information.
About Ovid TherapeuticsOvid Therapeutics
(NASDAQ:OVID) is a New York-based biopharmaceutical company using
its BoldMedicine™ approach to develop therapies that transform the
lives of patients with rare neurological disorders. Ovid’s drug
candidate, OV101, is currently in development for the treatment of
Angelman syndrome and Fragile X syndrome. Ovid has initiated the
Phase 2 STARS trial of OV101 in adults with Angelman syndrome and a
Phase 1 trial in adolescents with Angelman syndrome or Fragile X
syndrome. Ovid is also developing OV935 in collaboration with
Takeda Pharmaceutical Company Limited for the treatment of rare
epileptic encephalopathies and has initiated a Phase 1b/2a trial of
OV935.
For more information on Ovid, please visit
http://www.ovidrx.com/.
Forward-Looking StatementsThis press release
includes certain disclosures that contain “forward-looking
statements,” including, without limitation, statements regarding
progress, timing, scope and results of clinical trials for Ovid’s
product candidates, the reporting of clinical data regarding Ovid’s
product candidates, the development of OV101 as a potential first
in class compound for Fragile X syndrome and the potential use of
TAK-935/OV935 to treat rare epilepsies. 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, including its Quarterly Report on Form 10-Q for the
quarter ended June 30, 2017, 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.
ContactsInvestors: Burns McClellan Steve Klass, 212-213-0006
Sklass@burnsmc.com
Media: Pure Communications, Inc. Katie Engleman, 910-509-3977
katie@purecommunicationsinc.com
Ovid Therapeutics (NASDAQ:OVID)
Historical Stock Chart
From Mar 2024 to Apr 2024
Ovid Therapeutics (NASDAQ:OVID)
Historical Stock Chart
From Apr 2023 to Apr 2024