Ovid Therapeutics and NeuroPointDX Announce Collaboration to Identify Biomarkers for Angelman Syndrome

NEW YORK & MADISON, Wis.–(BUSINESS WIRE)– Ovid Therapeutics, a privately held biopharmaceutical company committed
to developing medicines that transform the lives of people with rare
neurological disorders, and NeuroPointDX, a privately held biotech
company based in Madison, Wisconsin and Cambridge, Massachusetts, today
announced that they have entered into a collaboration to identify novel
biomarkers of Angelman syndrome by analyzing metabolomic profile data as
part of Ovid’s ongoing randomized, double-blind, placebo-controlled
Phase 2 clinical trial (STARS).

Angelman syndrome is a rare genetic disorder that is characterized by a
variety of signs and symptoms, including delayed development,
intellectual disability, severe speech impairment, problems with
movement and balance, seizures, sleep disorders and anxiety. Like many
other neurological disorders, symptoms and response to treatments vary
widely from person to person.

“With the initiation of the STARS trial and this collaboration, we are
making important progress in better understanding Angelman syndrome and
developing much needed treatment options. The metabolic profile of this
syndrome is not well understood, and we believe this biomarker study
will produce critical data to fill this gap and inform us about the
impact of OV101 as a potential treatment option,” said Matthew During,
M.D., DSc, FACP, FRACP, president and chief scientific officer of Ovid.
“This collaboration is the first step in Ovid’s broader rare
neurological disorder biomarker strategy to identify molecular markers
of treatment responders and guide enrollment of participants in our
clinical trials.”

“We are excited to partner with Ovid Therapeutics on this biomarker
collaboration, particularly as it furthers our mission to improve the
lives of people impacted by neurological disorders by identifying
biomarkers that can improve diagnosis and inform more precise treatment
strategies,” said Elizabeth Donley, chief executive officer of
NeuroPointDX.

NeuroPointDX uses its metabolomics platform technology to identify
differences in children with autism spectrum disorders compared to
typically-developing children and between subgroups of children on the
spectrum for earlier diagnosis and more precise treatment. The
collaboration will leverage NeuroPointDX’s expertise in metabolomics in
an effort to identify biomarkers associated with Angelman syndrome.
Metabolomics is a process that allows comprehensive exploration of
changes in small molecules present in the metabolism of patients to
provide insight into the physiology of a disease and the response to
treatment. In the STARS trial, this analysis is designed to provide
molecular insights into disease mechanism and assess the potential
response to OV101 to help understand the physiological impact of the
compound in people with Angelman syndrome. This study may help identify
the individuals that are most likely respond to treatment.

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
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 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 GABA_A 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

OV101 (gaboxadol) is a delta (δ)-selective GABA_A receptor
agonist and is believed to be the first investigational drug to target
the disruption of tonic inhibition, a key mechanism that allows a
healthy human brain to decipher excitatory and inhibitory neurological
signals correctly without being overloaded. Loss of tonic inhibition is
implicated in a host of rare neurological disorders and is established
in genetic models. In preclinical models, OV101 has been able to
selectively activate the δ-subunit of GABA_A receptors, which
are found in the extrasynaptic space (outside of the synapse), and
helped regulate neuronal activity through tonic inhibition.

Ovid is developing OV101 for use in both 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.

In September 2016, the United States Food and Drug Administration
granted orphan drug designation for OV101 for the treatment of Angelman
syndrome. The United States Patent and Trademark Office has granted Ovid
two patents directed to methods of treating Angelman syndrome using THIP
(OV101). The issued patents expire in 2035, without regulatory
extensions.

About Ovid Therapeutics

Ovid Therapeutics is a privately held, 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
symptoms of Angelman syndrome and Fragile X syndrome. Ovid is also
developing OV935 in collaboration with Takeda Pharmaceutical Company
Limited for the treatment of rare epileptic encephalopathies. Ovid has
initiated a Phase 2 STARS trial of OV101 in adults with Angelman
syndrome and Ovid intends to commence a Phase 1 trial in adolescents
with Angelman syndrome or Fragile X syndrome. OV935 is expected to
commence a Phase 1b/2a trial in rare epileptic encephalopathies in 2017.

For more information, visit http://www.ovidrx.com/.

About NeuroPointDX

NeuroPointDX is the diagnostics division of Stemina Biomarker Discovery.
Stemina is a biotechnology company based in Madison, WI and Cambridge,
MA that has developed a robust and reproducible proprietary platform for
identifying changes in metabolism (“biomarkers”) utilizing highly
sensitive analytical equipment and its proprietary platform technology.
The Company’s diagnostics division, NeuroPointDX, identifies biomarkers
of neurological disorders in cellular models of disease and human
samples. These biomarkers provide greater insight into the pathways that
are disrupted in patients with neurological disorders. This information
can be translated into clinical diagnostics, individualized treatment
recommendations, and new potential therapies.

The first area in which the Company is conducting clinical studies is in
patients with autism and other neuro-developmental disorders. In
September 2015, Stemina launched its 1,500 patient study the Children’s
Autism Metabolome Project (CAMP). The study is funded by a $2.7 million
grant from the National Institutes of Mental Health and an investment by
the Nancy Lurie Marks Family Foundation. The CAMP study is the most
comprehensive study of the metabolism of children with autism, and other
neuro-developmental disorders ever conducted. This study may result in a
panel of validated blood tests for autism that will more accurately
diagnose and inform individualized treatment decisions based on the
child’s own metabolism.

For more information about NeuroPointDX and Stemina, visit www.neuropointdx.com
and www.stemina.com.

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements. Forward-looking
statements contained in this press release include, without limitation,
statements regarding the collaboration with NeuroPointDX to identify
novel biomarkers of Angelman syndrome and Ovid’s progress and
expectations regarding the clinical development of OV101. Words such as
“may,” “believe,” “will,” “expect,” “plan,” “anticipate,” “estimate,”
“intend” and similar expressions (as well as other words or expressions
referencing future events, conditions or circumstances) are intended to
identify forward-looking statements. These forward-looking statements
are not guarantees of future performance and involve a number of unknown
risks, assumptions, uncertainties and factors that are beyond Ovid’s
control. All forward-looking statements are based on Ovid’s expectations
and assumptions as of the date of this press release. Actual results may
differ materially from these forward-looking statements. Except as
required by law, Ovid expressly disclaims any responsibility to update
any forward-looking statement contained herein, whether as a result of
new information, future events or otherwise.

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