Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical
company committed to developing medicines that transform the lives
of people with rare neurological diseases, and the University of
Connecticut School of Medicine (“UConn”), today announced a
research collaboration and license agreement to accelerate the
development of a next-generation short hairpin RNA (shRNA)-based
therapeutic for Angelman syndrome and potentially other
indications. 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. An shRNA-based therapeutic
may address this underlying genetic cause of Angelman syndrome by
reducing the expression of UBE3A-antisense, potentially restoring
the function of UBE3A. This genetic approach may be used in
combination with OV101 (gaboxadol), Ovid’s novel, small-molecule
delta (δ)-selective GABAA receptor agonist, to restore tonic
inhibition and address the underlying symptomology of individuals
with Angelman syndrome. OV101 is currently being evaluated in the
pivotal Phase 3 NEPTUNE trial in Angelman syndrome, with topline
results expected in the fourth quarter of 2020.
Under the terms of the research collaboration, Ovid will work
closely with UConn’s Stormy J. Chamberlain, Ph.D., and gain
exclusive access to identified genetic sequences for a potential
shRNA-based therapeutic. Ovid plans to validate select sequences
and leverage its translational medicine capabilities and drug
development expertise in Angelman syndrome to advance an
shRNA-based therapeutic into clinical studies. Dr. Chamberlain is a
recognized leader in the field of Angelman syndrome and UBE3A
research and currently serves as the John and Donna Krenicki
Associate Professor of Genomics and Personalized Healthcare in
UConn’s Genetics and Genome Sciences Department. In addition, Dr.
Chamberlain chairs the Angelman Syndrome Foundation (ASF)
Scientific Advisory Committee and is a member of the Dup15q
Alliance Scientific Advisory Board. Ovid will also work closely
with UConn’s Noelle Germain, Ph.D., Assistant Professor of Genetics
and Genome Sciences on these efforts.
“Ovid is deeply committed to the Angelman syndrome community. We
have made great progress and are excited to see the topline data
from our Phase 3 NEPTUNE trial with OV101 expected in Q4 2020,”
said Amit Rakhit, M.D., MBA, President and Chief Medical Officer of
Ovid Therapeutics. “We believe OV101 has the potential to serve as
a core therapy for this disorder and are now focused on building a
comprehensive and strategic Angelman syndrome longer term pipeline.
If successful, OV101 may be used in combination with genetic
approaches in the future to address the needs of Angelman syndrome.
This collaboration with Drs. Chamberlain and Germain, both
accomplished scientific leaders in the field of Angelman syndrome,
will enable us to accelerate and share in their mission to identify
and develop next-generation genetic therapies. Together with our
early-stage microRNA approach, this research collaboration now
provides us with additional targets against this disorder, greater
strategic optionality, and underpins our broad capability to bring
new therapies to individuals living with Angelman syndrome both
near-term and into the future.”
“Our lab shares in Ovid’s demonstrated commitment to advance
innovative therapeutic options for Angelman syndrome,” stated Dr.
Chamberlain. “An shRNA therapeutic can target the genetic cause of
Angelman syndrome at its source and may offer potential advantages
to other next-generation approaches, including antisense
oligonucleotide therapy, via a lower rate of degradation and
turnover and plasmid delivery allowing for a less-frequent dosing
profile. Ovid is uniquely positioned to accelerate an shRNA
therapeutic through late preclinical and clinical development, and
our lab looks forward to working with the team at Ovid towards our
common objective of impacting the lives of individuals living with
Angelman syndrome and their families.”
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 U.S. Food and Drug
Administration (FDA), European Medicines Agency 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.
Ovid is developing OV101 for the treatment of Angelman syndrome
to potentially restore tonic inhibition and thereby address several
core symptoms of Angelman syndrome. Ovid is conducting a pivotal
Phase 3 clinical trial with OV101 in Angelman syndrome (NEPTUNE),
with topline results expected in the fourth quarter of 2020. In
addition, Ovid is also exploring OV881, a microRNA approach that
may reduce the expression of UBE3A-antisense and potentially
restore UBE3A expression.
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 www.ovidrx.com.
About UConnUConn Health is Connecticut’s only
public academic medical center. Based on a 206-acre campus in
Farmington, UConn Health has a three-part mission: research,
teaching and patient care. Home to the UConn School of Medicine,
School of Dental Medicine and UConn John Dempsey Hospital with over
5,000 employees supporting nearly 1,000 students, over 800,000
annual patient visits, and innovative scientific research
contributing to the advancement of medicine. For more information,
visit health.uconn.edu.
Ovid Forward-Looking StatementsThis press
release includes certain disclosures that contain “forward-looking
statements,” including, without limitation, statements regarding:
development of short hairpin RNA based therapeutics, development of
combination therapies, potential benefits of OV101, anticipated
reporting schedule of clinical data for OV101 and the potential
benefits and value of this collaboration. 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.
Ovid Therapeutics 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
UConn ContactJessica McBride,
PhDjessica.mcbride@uconn.edu 860-878-5058
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