Ovid Therapeutics Announces OV101 Granted Fast Track Designation by U.S. FDA for Treatment of Angelman Syndrome
December 19 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 Fast Track
designation to OV101 for the treatment of Angelman
syndrome.
“This designation is an important milestone for both Ovid and
the Angelman community as it enables increased dialog with the FDA,
speeding our ability to bring this potential therapeutic option to
people living with Angelman syndrome. We believe that OV101, with
its novel mechanism of action, has the potential to be an
innovative and impactful therapy,” said Amit Rakhit, M.D., MBA,
chief medical and portfolio management officer of Ovid
Therapeutics. “In addition to the regulatory milestones of orphan
drug and Fast Track designations for Angelman syndrome, we achieved
significant clinical progress with our OV101 program. As a result
of positive Phase 1 data, we were recently able to expand our
ongoing Phase 2 STARS clinical trial to include both adults and
adolescents with Angelman syndrome. We look forward to data from
the STARS trial in the second half of 2018.”
OV101 is a delta (δ)-selective GABAA receptor agonist that
targets the disruption of tonic inhibition, a central physiological
process of the brain that is thought to be the underlying
cause of Angelman syndrome and other neurodevelopmental disorders.
Ovid is currently studying OV101 in its Phase 2 STARS clinical
trial, a randomized, double-blind, placebo-controlled study
investigating the safety and efficacy of OV101 in patients with
Angelman syndrome. Upon successful completion of a Phase 1
pharmacokinetic (PK) and safety study showing that OV101 has a
similar PK profile in adolescents as in adults, Ovid recently
amended the STARS protocol to include patients aged 13 years and
older.
The FDA’s Fast Track process is designed to expedite the
development and review of drugs used to treat serious conditions
and fill an unmet medical need. Fast Track designation enables the
company to have early and frequent communication with the FDA
throughout the drug development and review process, often leading
to faster drug approval and patient access.
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, a central physiological
process of the brain 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. Gaboxadol
has previously 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 has granted orphan drug and Fast Track
designations for OV101 for the treatment of Angelman
syndrome and orphan drug designation for the treatment of
Fragile X syndrome. The U.S. Patent and Trademark Office has
granted Ovid patents directed to methods of treating Angelman
syndrome using OV101. The issued patents expire in
2035 for Angelman syndrome.
About Angelman Syndrome Angelman syndrome
is a genetic disorder that is characterized by a variety of
signs and symptoms. Characteristic features of
this disorder 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 disruption of a gene that codes for
ubiquitin protein ligase E3A (UBE3A). Angelman syndrome
affects approximately 1 in 12,000 to 20,000 people in the
U.S. There are currently no U.S. Food and Drug
Administration (FDA)-approved therapies for the treatment of
Angelman syndrome.
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 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 progress, timing, scope and results of
clinical trials for Ovid’s product candidates, the timing of
clinical data, the development of therapies for younger patients,
the provision of access to effective therapies, and the FDA Fast
Track process leading to faster drug approval and patient
access. 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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