Ovid Therapeutics Receives FDA Rare Pediatric Disease Designation for OV101 for the Treatment of Angelman Syndrome
June 19 2020 - 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 Rare Pediatric
Disease Designation to OV101 (gaboxadol) for the treatment of
Angelman syndrome. OV101 is believed to be the only delta
(δ)-selective GABAA receptor agonist in development and is
currently being evaluated in the Company’s pivotal Phase 3 NEPTUNE
trial in Angelman syndrome, with topline results expected in the
fourth quarter of 2020. The FDA has previously granted Orphan Drug
and Fast Track designations for OV101 for the treatment of Angelman
syndrome.
Under the Creating Hope Act passed into federal law in 2012, the
FDA grants Rare Pediatric Disease Designation for serious and
life-threatening diseases that primarily affect children ages 18
years or younger and fewer than 200,000 people in the U.S. If a new
drug application (NDA) for OV101 in Angelman syndrome is approved,
Ovid may be eligible to receive a priority review voucher from the
FDA, which can be redeemed to obtain priority review for any
subsequent marketing application or may be transferred and/or sold
to other companies for their programs, such as has recently been
done by other voucher recipients.
“OV101 has the potential to become the first FDA-approved
therapy for individuals living with Angelman syndrome. Receiving
Rare Pediatric Disease Designation from the FDA is a significant
milestone for this program and underscores the critical value of
our work,” said Amit Rakhit, M.D., MBA, President and Chief Medical
Officer of Ovid Therapeutics. “Importantly, with this designation,
we may be eligible to receive a priority review voucher from the
FDA, providing significant value as we work diligently towards the
completion of our ongoing pivotal Phase 3 NEPTUNE trial. We are
grateful to the FDA and Congress for having enacted this law which
helps Ovid and all companies developing innovative drugs for rare
pediatric conditions.”
About Angelman Syndrome Angelman syndrome
is a rare genetic condition that is characterized by a variety of
signs and symptoms. Characteristic features of this condition
include delayed development, intellectual disability, severe speech
impairment, problems with movement and balance, seizures, sleep
disorders and anxiety. The most common cause of Angelman syndrome
is the loss of function of the gene that codes for ubiquitin
protein ligase E3A (UBE3A), which plays a critical role in nerve
cell communication, resulting in impaired tonic inhibition.
Individuals with Angelman syndrome typically have normal lifespans
but are unable to live independently. Therefore, they require
constant support from a network of specialists and caregivers.
Angelman syndrome affects approximately 1 in 12,000 to 1 in 20,000
people globally.
There are no approved therapies by the FDA, European Medicines
Agency (EMA), or rest of world for Angelman syndrome, and treatment
primarily consists of behavioral interventions and pharmacologic
management of symptoms.
Angelman syndrome is associated with a reduction in tonic
inhibition, a function of the delta (δ)-selective GABAA receptor
that allows a human brain to decipher excitatory and inhibitory
neurological signals correctly without being overloaded. If tonic
inhibition is reduced, the brain becomes inundated with signals and
loses the ability to separate background noise from critical
information.
About OV101 (gaboxadol)OV101 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, a central physiological process of
the brain that is thought to be the underlying cause of certain
neurodevelopmental disorders. OV101 has 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 modulation of tonic inhibition.
Ovid is developing OV101 for the treatment of Angelman syndrome
and Fragile X syndrome to potentially restore tonic inhibition and
thereby address several core symptoms of these conditions. In both
these syndromes, the underlying pathophysiology includes disruption
of tonic inhibition modulated through the δ-subunit of GABAA
receptors. In preclinical studies, it was observed that OV101
improved symptoms of Angelman syndrome and Fragile X syndrome. This
compound has also previously been tested in more than 4,000
patients (more than 1,000 patient-years of exposure) and was
observed to have favorable safety and bioavailability profiles.
Ovid is conducting a pivotal Phase 3 clinical trial with OV101 in
Angelman syndrome (NEPTUNE) and has completed a Phase 2
signal-finding clinical trial with OV101 in Fragile X syndrome
(ROCKET).
OV101 has received Rare Pediatric Disease Designation from the
FDA for the treatment of Angelman syndrome. The FDA has also
granted Orphan Drug and Fast Track designations for OV101 for both
the treatment of Angelman syndrome and Fragile X syndrome. In
addition, the European Commission (EC) has granted orphan drug
designation to OV101 for the treatment of Angelman syndrome. The
U.S. Patent and Trademark Office has granted Ovid patents directed
to methods of treating Angelman syndrome and Fragile X syndrome
using OV101. The issued patents expire in 2035 without regulatory
extensions.
About Ovid Therapeutics Ovid Therapeutics
Inc. is a New York-based biopharmaceutical company using its
BoldMedicine® approach to develop medicines that transform the
lives of patients with rare neurological disorders. Ovid has a
broad pipeline of potential first-in-class medicines. The Company’s
most advanced investigational medicine, OV101 (gaboxadol), is
currently in clinical development for the treatment of Angelman
syndrome and Fragile X syndrome. Ovid is also developing OV935
(soticlestat) in collaboration with Takeda Pharmaceutical Company
Limited for the potential treatment of rare developmental and
epileptic encephalopathies (DEE). 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:
advancing and commercializing Ovid’s product candidates, progress,
timing, scope and the development and potential benefits of Ovid’s
product candidates; and the anticipated reporting schedule of
clinical data regarding Ovid’s product candidates. You can identify
forward-looking statements because they contain words such as
“will,” “appears,” “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 include uncertainties in the development and regulatory
approval processes, and the fact that initial data from clinical
trials may not be indicative, and are not guarantees, of the final
results of the clinical trials and are subject to the risk that one
or more of the clinical outcomes may materially change as patient
enrollment continues and/or more patient data become available.
Additional risks 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
under the caption “Risk Factors”. Such risks may be amplified by
the COVID-19 pandemic and its potential impact on Ovid’s business
and the global economy. Ovid assumes no obligation to update any
forward-looking statements contained herein to reflect any change
in expectations, even as new information becomes available.
Contacts
Investors and Media:Ovid Therapeutics
Inc.Investor Relations & Public Relationsirpr@ovidrx.com
Or
Investors: Steve KlassBurns McClellan,
Inc.sklass@burnsmc.com (212) 213-0006
Media: Katie Engleman 1AB
katie@1abmedia.com
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