-- Results from the Phase 1 Clinical Trial of
Adolescents with Angelman and Fragile X Syndromes Demonstrated
OV101 was Well Tolerated with a Pharmacokinetic Profile Similar to
Adults --
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 upon
successful completion of the Phase 1 clinical trial to evaluate the
pharmacokinetics (PK), safety and tolerability of OV101 in
adolescents diagnosed with Angelman syndrome or Fragile X syndrome,
the Company will now include adolescent patients aged 13 years and
older in the Phase 2 STARS trial.
The Phase 1 PK study was strategically designed to assess the PK
profile of OV101, an agonist of the extrasynaptic GABAA receptor,
in adolescent patients. While OV101 has an extensive safety
database of over 4,000 adults, the Phase 1 clinical trial
represented the first time adolescents were given OV101. In the
trial, OV101 was found to be generally safe and well tolerated and
its PK profile in adolescents was similar to previous data
generated in young adults, supporting the inclusion of adolescent
patients in the STARS clinical trial.
“This first clinical data of OV101 in adolescents in
genetically-defined disorders with GABA hypofunction provide
important information to our overall development strategy and are
an important step to enable development of OV101 in younger ages,”
said Amit Rakhit, M.D., MBA, chief medical and portfolio officer of
Ovid Therapeutics. “Angelman syndrome and Fragile X syndrome are
disorders that impact patients from birth and our goal is to
develop a therapy for a broad range of patient age groups. At Ovid,
we strive to provide access to effective therapies as early as
possible and we are now able to offer enrollment in our STARS trial
to adolescent Angelman syndrome participants. With the inclusion of
adolescents, we now anticipate data from the STARS trial to be
available in the second half of 2018.”
“We have assembled a team at Ovid that not only has deep
expertise in neurology but also significant experience developing
therapies for children. Part of our core strategy is to rapidly
develop our medicines for younger patients, which is a patient
population usually addressed late in the drug development process,”
said Jeremy Levin, D.Phil., MB Chir, chairman of the board of
directors and chief executive officer of Ovid Therapeutics. “Data
from our PK study combined with beginning enrollment of adolescent
Angelman syndrome patients is an example of how we are executing
that strategy. We will continue to execute in a disciplined manner
against our goals to develop impactful therapies, particularly for
children.”
About the Phase 1 Trial
The Phase 1 single dose, single-arm, open-label clinical trial
of OV101 enrolled seven male and five female adolescent patients,
aged 13 to 17, who had been diagnosed with either Angelman syndrome
or Fragile X syndrome. Participants received a single 5mg oral dose
of OV101. Overall results of the PK study met the objectives
and showed that PK parameters in adolescents with Angelman and
Fragile X syndrome were not significantly different from previous
data generated in young adults. Additional details on the Phase 1
clinical trial can be found at www.clinicaltrials.gov.
Ovid recently presented strong preclinical data on the impact of
OV101 on animal models of Fragile X syndrome and has also received
FDA orphan drug designation for OV101 for both Fragile X syndrome
and Angelman syndrome. Coupled with the favorable Phase 1 data,
this forms a solid foundation for the overall strategy of
developing OV101 for younger age groups in both populations.
About the Phase 2 STARS Trial
The STARS trial is a randomized, double-blind,
placebo-controlled Phase 2 clinical trial investigating the safety
and efficacy of OV101 that was designed in consultation with the
Angelman syndrome community. The trial is expected to enroll
approximately 75 patients in the United States and Israel aged 13
to 49 years with a confirmed diagnosis of Angelman syndrome. The
primary endpoint of the trial is to assess the safety and
tolerability of OV101. Additionally, the trial has several
exploratory endpoints to evaluate measures of gross and fine motor
skills, maladaptive behavior, sleep, clinical global impression and
health-related quality of life questionnaires.
Learn more about the STARS trial at www.clinicaltrials.gov
and www.angelmanstudy.com.
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 United States.
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 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, 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 designation for OV101 for the
treatment of both Angelman syndrome and Fragile X syndrome. The
United States 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 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
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 Statements
This 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 execution of Ovid goals for not only
OV101 but also OV935. 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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