Ovid Therapeutics Receives Orphan Drug Designation from the U.S. FDA for OV101 for Treatment of Fragile X Syndrome
October 10 2017 - 8:00AM
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 orphan drug
designation for OV101 for the treatment of Fragile X syndrome.
OV101, a delta (d)-selective GABAA receptor agonist, is believed to
be the first investigational drug to target the disruption of tonic
inhibition, a key mechanism that allows a healthy human brain to
decipher excitatory and inhibitory neurological signals correctly
without being overloaded. OV101 has already received orphan drug
designation for the treatment of Angelman syndrome.
“We are delighted to receive orphan drug designation for OV101
for the treatment of Fragile X syndrome. The rapid action from the
FDA on this decision mirrors our own urgency for developing OV101
as a potential new therapy for people living with Fragile X
syndrome, a patient population in desperate need of new treatment
options,” said Matthew During M.D., DSc, FACP, FRACP, president and
chief scientific officer of Ovid Therapeutics. “This is an
important step in the continued advancement of our Fragile X
syndrome program and furthers our mission to develop impactful
medicines for people with rare neurological disorders.”
Orphan drug designation, which is intended to facilitate drug
development for rare diseases, provides substantial benefits to the
sponsor, including the potential for tax credits for clinical
development costs, study-design assistance, and several years of
market exclusivity for the product upon regulatory approval.
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 OV101
OV101 (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.
In September 2016, the FDA granted orphan drug designation for
OV101 for the treatment of Angelman 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 Ovid Therapeutics
Ovid 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 Statements
This press release includes certain
disclosures that contain “forward-looking statements,”
including, without limitation, statements regarding the continued
advancement of the Fragile X syndrome program and the development
of impactful medicines for people with rare neurological
diseases. 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
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