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
first patient has been randomized in the company’s single, pivotal
Phase 3 NEPTUNE clinical trial evaluating OV101 (gaboxadol), a
novel delta (δ)-selective GABAA receptor agonist, in Angelman
syndrome. The company anticipates topline data from the trial to be
available in mid-2020.
“There are currently no other therapies in clinical development
for Angelman syndrome and no approved treatments for this disorder
exist today,” said Amit Rakhit, MD, MBA, Chief Medical Officer and
Head of Research & Development at Ovid. “We believe the first
patient randomized in the pivotal Phase 3 NEPTUNE trial is a major
step toward establishing the clinical effectiveness of OV101 in
Angelman syndrome and one that we hope will result in the first
approved medicine for individuals living with this rare neurologic
condition.”
NEPTUNE is a randomized, double-blind, placebo-controlled, Phase
3 clinical trial designed to assess the effects of treatment with
OV101 (oral, once-daily dosing) versus placebo over 12 weeks. The
trial will include approximately 60 pediatric patients ages 4 to 12
years diagnosed with Angelman syndrome. The sole primary endpoint
will be change in overall score on the Clinical Global
Impression-Improvement-Angelman syndrome (CGI-I-AS) scale.
Secondary endpoints include sleep, communication, motor function,
socialization, daily living skills and behavior domains. A limited
number of children, ages 2 to 3 years, will also be enrolled in the
study to evaluate OV101’s pharmacokinetics (PK) along with safety
and tolerability in this younger age group. Participants who
complete the NEPTUNE trial will be eligible to roll over into the
ongoing open-label extension trial (ELARA) should they choose to do
so.
“In the U.S., it is estimated there are approximately 24,000
individuals with Angelman syndrome,” said Amanda Moore, CEO of the
Angelman Syndrome Foundation. “Those with Angelman syndrome require
24-hour care throughout their lives and are unable to live
independently. The Angelman syndrome community is urgently seeking
therapeutic options since none currently exist and I am excited
about the potential of OV101 to offer hope for patients and their
families.”
As previously announced, based on Ovid’s interactions with
regulatory authorities in the U.S. and Germany regarding a Phase 3
program and registration path, Ovid, the U.S. Food and Drug
Administration (FDA) and Germany’s Federal Institute for Drugs and
Medical Devices (BfArM) agree on the study design of the Phase 3
NEPTUNE trial, including the use of CGI-I-AS as the primary
efficacy outcome measure. Both the FDA and BfArM agreed that
NEPTUNE, if positive, could support the filings of a New Drug
Application (NDA) and Marketing Authorization Application (MAA),
respectively.
About Angelman Syndrome Angelman syndrome is a
rare 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 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, 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 (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
thereby address the core symptoms of these disorders. In both these
syndromes, the underlying pathophysiology includes disruption of
the 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 over
4,000 patients (over 1,000 patient-years of exposure) and was
observed to have favorable safety and bioavailability profiles. In
2018, Ovid announced the successful completion of its Phase 2
(STARS) trials of OV101 in adults and adolescents with Angelman
syndrome. Ovid is conducting the pivotal Phase 3 (NEPTUNE) clinical
trial in Angelman syndrome. In addition, Ovid is conducting a Phase
2 clinical trial (ROCKET) in Fragile X syndrome with results
expected around year-end 2019 or early 2020.
The FDA has granted Orphan Drug and Fast Track
designations for OV101 for both the treatment of Angelman syndrome
and Fragile X syndrome. 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 TherapeuticsOvid 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 Statements This press release
includes certain disclosures that contain “forward-looking
statements,” including, without limitation, statements regarding
advancing Ovid’s product candidates, progress, timing, scope and
results of clinical trials for Ovid’s product candidates, and the
reporting of clinical data regarding Ovid’s product candidates. 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 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.
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 Engleman1ABkatie@1abmedia.com(919)
333-7722
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