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
United States Food and Drug Administration (FDA) has granted orphan
drug designation to TAK-935/OV935 for the treatment of
Lennox-Gastaut syndrome, a type of developmental and epileptic
encephalopathy. Takeda Pharmaceutical Company Limited and Ovid
formed a global collaboration in January 2017 to develop and
commercialize TAK-935/OV935 for the treatment of developmental and
epileptic encephalopathies, a group of rare epilepsies that cause
significant morbidities and can worsen over time.
Lennox-Gastaut syndrome is a rare disorder that is often
diagnosed between three and five years of age. Patients diagnosed
with Lennox-Gastaut syndrome experience multiple seizure types that
are difficult to manage and have many of the same symptoms as other
rare pediatric epilepsies.
“The FDA’s decision to grant orphan drug designation to
TAK-935/OV935 for the treatment of Lennox-Gastaut syndrome reflects
the Agency’s recognition of the urgent need for additional
treatments for this severe type of epilepsy,” said Matthew During,
M.D., DSc, FACP, FRACP, president and chief scientific officer of
Ovid Therapeutics. “This orphan drug designation is the fourth one
we have received in two years for our two lead investigational
products, underscoring our commitment to develop impactful
medicines for people living with rare neurological disorders and
our ability to rapidly advance our programs.”
TAK-935/OV935 is a potent, highly-selective, first-in-class
inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H). It is
believed that CH24H is involved in over-activation of the
glutamatergic pathway, which has been shown to play a role in the
initiation and spread of seizure activity. To Ovid and Takeda’s
knowledge, TAK-935/OV935 is the only molecule with this mechanism
of action in clinical development.
“TAK-935/OV935 has a novel mechanism of action and could be a
potentially transformative therapy for individuals diagnosed with
rare epilepsies, such as Lennox-Gastaut syndrome,” said Dr.
Emiliangelo Ratti, head of Takeda’s Neuroscience Therapeutic Area
Unit. “The granting of orphan drug designation is an important
milestone for the clinical development program for TAK-935/OV935,
which we are currently evaluating in a Phase 1b/2a clinical trial
with data anticipated in 2018.”
Orphan drug designation is intended to facilitate and expedite
drug development for rare diseases for which there are no current
treatments available. It also 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 Lennox-Gastaut
SyndromeLennox-Gastaut syndrome is one of several
disorders that together are designated as developmental and
epileptic encephalopathies. Studies estimate that Lennox–Gastaut
syndrome affects approximately 14,500 to 18,500 children under the
age of 18 and over 30,000 children and adults in the United States.
It is also estimated that between 1 percent and 4 percent of
childhood epilepsies are a result of Lennox–Gastaut syndrome. Only
10 percent of these patients have seizures that are fully
controlled by existing therapies.
Developmental and epileptic encephalopathies include epilepsy
syndromes associated with severe cognitive and behavioral
disturbances. The International League Against Epilepsy (ILAE)
defines an epileptic encephalopathy as a condition in
which “the epileptiform EEG abnormalities themselves are believed
to contribute to a progressive disturbance in cerebral
function.”
These epilepsies cause significant morbidities for patients
beyond what might be expected from the known underlying pathology
alone and can worsen over time. Developmental and epileptic
encephalopathies typically present early in life and are often
associated with severe cognitive and developmental impairment in
addition to frequent treatment-resistant seizures throughout the
person’s lifetime. These disorders vary in age of onset,
developmental outcomes, etiologies, neuropsychological deficits,
electroencephalographic (EEG) patterns, seizure types and
prognosis.
About TAK-935/OV935TAK-935/OV935, which is
being studied in developmental and epileptic encephalopathies, is a
potent, highly-selective, first-in-class inhibitor of the enzyme
cholesterol 24-hydroxylase (CH24H). CH24H is predominantly
expressed in the brain, where it plays a central role in
cholesterol homeostasis. CH24H converts cholesterol to
24S-hydroxycholesterol (24HC), which then exits the brain into the
blood plasma circulation. 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 24HC is involved in over-activation of the glutamatergic
pathway through modulation of the NMDA channel, implying its
potential role in central nervous system diseases such as epilepsy.
To Ovid and Takeda’s knowledge, TAK-935/OV935 is the only molecule
with this mechanism of action in clinical development.
TAK-935/OV935 has been tested in preclinical models to provide
data to support the advancement of the drug into human clinical
studies in patients suffering from rare epilepsy syndromes. A novel
proprietary PET ligand, developed by Takeda and Molecular
Neuroimaging, LLC (MNI), has been used to determine target enzyme
occupancy of TAK-935/OV935 in the brain. In addition, the effect of
TAK-935/OV935 on CH24H enzyme activity in the brain has been
assessed by following measurable reductions in the plasma
concentration of 24HC.
TAK-935/OV935 has completed four Phase 1 clinical studies, which
have assessed tolerability and target engagement at doses believed
to be therapeutically relevant. The FDA has granted orphan drug
designation for TAK-935/OV935 for the treatment of both Dravet
syndrome and Lennox-Gastaut syndrome. TAK-935/OV935 is being
co-developed by Ovid and Takeda Pharmaceutical Company Limited.
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 initiated the Phase
2 STARS trial of OV101 in people with Angelman syndrome in 2017 and
completed 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
epileptic encephalopathies and in August 2017 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 the progress, timing, scope and results of
clinical trials for Ovid’s product candidates, the reporting of
clinical data regarding Ovid’s product candidates, 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 September 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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