Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on
becoming a leader in development and commercialization of
treatments for orphan diseases and neurology, announced today that
it has achieved significant milestones in its clinical development
programs. The first patient has been enrolled in a Phase I
Proof-of-Concept Trial investigating the safety, tolerability and
effects on blood pressure in patients with orthostatic hypotension
associated with diabetes (“DOH”). In addition, in October
2019, the Company completed dosing healthy volunteers in a Phase I
Safety Study of CERC-802, an ultra-pure, oral, crystalline
formulation of D-mannose currently in development for the treatment
of MPI Deficiency (“MPI-CDG”).
Dr. Simon Pedder, Executive Chairman of the Board, commented,
“We are very enthusiastic about our continued progress from our
research and development team. It’s very exciting to see the
advancement and expansion of the CERC-301 development program into
a substantially broader patient population namely the millions of
diabetic patients suffering from the untoward effects of
Orthostatic Hypotension.”
“Likewise, the completion of the CERC-802 Phase I Safety Study
allows us to advance our second asset for the treatment of
Congenital Disorders of Glycosylation (CDGs) getting us another
step closer to helping the hundreds of patients and families
world-wide. We continue to stay on track against our R&D
milestones which could deliver our first product approval as early
as 2021 with an associated Priority Review Voucher.”
About the CERC-301 Proof-of-Concept Study in
DOH The purpose of this study is to assess the single dose
effects of CERC-301 in patients with symptomatic Orthostatic
Hypotension (“OH”) associated with diabetes. This study is a
randomized, double-blind, placebo-controlled, two-way cross-over
trial, over two, 24-hour, in-clinic visits. At each visit, patients
will receive a single 20 mg dose of CERC-301 or placebo and then
undergo a series of orthostatic challenge tests over the 24-hour,
in-clinic period. Patients will also complete an OH symptomatic
assessment following each orthostatic challenge. Safety,
tolerability and pharmacokinetic (“PK”) data will also be
collected. As part of the routine laboratory tests, particular
interest will be paid to the patient’s plasma glucose levels over
the course of the study.
Clin301-101 was a study in patients with nOH associated with
Parkinson’s disease. In the Clin301-101 study, a single 20 mg dose
of CERC-301 achieved clinically meaningful improvements over
baseline and placebo with a maximum improvement of 29.1 mmHg upon
standing throughout the 6-hour study period. All doses tested were
safe and well tolerated with no serious adverse event
reported.
Of note, in previous clinical studies conducted by Cerecor, 20
mg doses of CERC-301 were safe and well tolerated for up to 28
days.
About CERC-301CERC-301 is an orally available,
NR2B-specific, NMDA receptor antagonist being developed for the
treatment of symptomatic OH.
About Orthostatic Hypotension (“OH”)Orthostatic
hypotension is a sudden fall in blood pressure that occurs when a
person assumes a standing position. It can be due to a lesion of
the baroreflex loop, which senses a change in blood pressure and
adjusts heart rate and activates sympathetic nerve system fibers to
cause the blood vessels to narrow and correct blood pressure. It
may also be caused by hypovolemia (a decreased amount of blood in
the body), resulting from the excessive use of diuretics,
vasodilators, or other types of drugs, dehydration, or prolonged
bed rest. The disorder may be associated with Addison's Disease,
diabetes, spinal cord injuries, dialysis, advanced age and certain
neurological disorders including Multiple System Atrophy with
Orthostatic Hypotension (formerly known as Shy-Drager syndrome),
autonomic system neuropathies, and other dysautonomias. Symptoms,
which generally occur after sudden standing, include dizziness,
lightheadedness, blurred vision, and syncope (temporary loss of
consciousness).
Current treatment options for OH target symptom burden
reductions to increase quality of life such as correcting
aggravating factors (i.e. discontinuation of hypotension drugs and
correction of anemia and vitamin deficiencies); nonpharmacologic
measures such as intravascular volume expansion, increased physical
activity, reduction of meal size, compression stocking/abdominal
binder, and sleeping arrangement; and drug therapies (i.e.
droxidopa, midrodrine).
Orthostatic Hypotension affects numerous comorbid disease
conditions with significant underserved patient populations (see
chart) in the United States and in the rest of the world.
Comorbid Disease |
% with OH |
U.S. Based Estimated Population |
Estimated # of Patients |
Advanced Age |
30%1,2 |
46,000,000 > 65 Years of Age8 |
13,800,000 |
Diabetes |
16 to 25%2,3,4 |
30,000,0009 |
6,000,000 |
Parkinson’s Disease |
15 to 58%4 |
1,200,000 Patients10 |
420,000 |
ESRD Dialysis |
15 to 50%5 |
660,000 Patients / 1,980,000 Procedures11 |
216,450 |
Spinal Cord Injury |
35 to 60%6,7 |
288,000 Patients12 |
136,800 |
Professor Christopher Mathias MBBS DPhil DSc FRCP FMedSci, a
world-renowned thought leader in Autonomic and Neurovascular
Disorders from the Imperial College and University College London
Hospitals stated, “The unique mechanism of action of CERC-301 and
its demonstrated effect on blood pressure clearly warrants further
investigation for the treatment of conditions associated with
hypotension. Its potential to be, perhaps, safer and more effective
in a much broader patient population than our current therapeutic
options provide is promising. It’s exciting to think of a
compound being developed for Orthostatic Hypotension in patients
suffering from Parkinson’s disease and diabetes that may also have
clinical utility for other conditions exacerbated by hypotension
such as advanced age, intradialytic hypotension and patients with
spinal cord injury.”
About the CERC-802 Phase I Study
(Clin802-101)The single-center, US-based safety,
tolerability and PK study was an open-label, randomized,
single-dose, 4-way crossover study in 16 healthy adult volunteers,
of which 14 completed. CERC-802 had no serious adverse
events. All CERC‑802 related adverse events were transient
and resolved with no sequalae. PK data is expected in early
2020.
“The doses were generally well tolerated by the majority of
healthy volunteers and anticipated PK data should provide a solid
foundation for the ongoing development of CERC-802,” said Dr. Perry
Calias, PhD, Chief Scientific Officer at Cerecor. “We are
also very excited about the progress being made with the CDG FIRST
Trial our retrospective study seeking to collect natural history,
efficacy and safety data from CDG patients treated with
monosaccharide substrate replacement therapy for, PGM1-CDG, MPI-CDG
and Leukocyte Adhesion Deficiency Type II (LADII) also known as
SLC35C1-CDG. We believe the information gathered through this
study will be instrumental in facilitating regulatory approval of
all three CERC-800 programs through the 505(b)(2) pathway.”
About
CERC-802 CERC-802
is an ultra-pure formulation of D-mannose, a naturally occurring
monosaccharide commonly found in animals, microorganisms, and
plants, including edible fruits and herbs. D-mannose is consumed by
the body to provide substrates for protein glycosylation, the
process by which carbohydrates are utilized to modify certain
proteins as it relates to protein structure and function. CERC-802
has been granted Orphan Drug Designation (ODD) and Rare Pediatric
Disease Designation (RPDD) by the FDA, making the Company eligible
to receive a Priority Review Voucher (PRV) upon approval of an
NDA.
About MPI-CDGCDGs are a group of rare,
inherited, metabolic disorders caused by glycosylation defects that
present as a broad range of clinical symptoms, including
coagulopathy, hepatopathy, myopathy, hypoglycemia, protein-losing
enteropathy and reduced cell counts. CDGs have high infant
morbidity and mortality with no FDA-approved treatments. CDG
patients are born with a genetic defect that hinders their ability
to utilize certain monosaccharides in the production of
glycoproteins. A deletion or misplacement of a sugar subunit
produces a dysfunctional glycoprotein, resulting in a myriad of
medical issues.
Dietary monosaccharide formulations have been shown to alleviate
several of the clinical manifestations in CDG patients. These
substrate replacement therapies work by increasing the availability
of metabolic intermediates for glycoprotein synthesis. Biallelic
pathogenic variants of the MPI gene lead to enzymatic deficiencies
of mannose-6-phosphate isomerase (MPI enzyme) associated with the
clinical syndrome MPI-CDG. The overall estimated occurrence of
MPI-CDG worldwide is less than 50 cases, although MPI-CDG is
suspected to be under-diagnosed.
About the CDG First Trial The CDG FIRST
(Congenital Disorders of Glycosylation Formative Retrospective
Study) trial is a multi-center, international, non-interventional,
retrospective study that follows general principles of periodic
assessment of CDG patients in routine practice. The
objectives of the study are to collect natural history and
treatment-related data of patients diagnosed with PGM1-CDG, MPI-CDG
or SLC35C1-CDG who are either treated with or without D-galactose,
D-mannose and L-fucose, respectively, as well as patients with
other CDGs who are treated with one of the sugars.
About Cerecor Cerecor is a biopharmaceutical
company focused on becoming a leader in development and
commercialization of treatments for orphan diseases and
neurological conditions. The Company is building a robust pipeline
of innovative therapies in orphan diseases and neurology. The
Company’s pediatric rare disease pipeline is led by CERC-801,
CERC-802 and CERC-803 (“CERC-800 programs”), which are therapies
for inborn errors of metabolism, specifically disorders known as
Congenital Disorders of Glycosylation. The FDA granted Rare
Pediatric Disease Designation and Orphan Drug Designation (“ODD”)
to all three CERC-800 compounds, thus qualifying the Company to
receive a Priority Review Voucher (“PRV”) upon approval of a new
drug application (“NDA”). The PRV may be sold or transferred an
unlimited number of times. The Company plans to leverage the
505(b)(2) NDA pathway for all three compounds to accelerate
development and approval. The Company is also in the process
of developing one other preclinical pediatric orphan rare disease
compound, CERC-913, for the treatment of mitochondrial DNA
Depletion Syndrome. The Company’s neurology pipeline is led
by CERC-301, a Glutamate NR2B selective, NMDA Receptor antagonist,
which Cerecor is currently exploring as a novel treatment for
orthostatic hypotension. The Company is also developing
CERC-406, a CNS-targeted COMT inhibitor for Parkinson’s
Disease. The Company also has one marketed product,
Millipred®, an oral prednisolone indicated across a wide variety of
inflammatory conditions and indications.
For more information about Cerecor, please visit
www.cerecor.com.
Forward-Looking StatementsThis press release
may include forward-looking statements made pursuant to the Private
Securities Litigation Reform Act of 1995. Forward-looking
statements are statements that are not historical facts. Such
forward-looking statements are subject to significant risks and
uncertainties that are subject to change based on various factors
(many of which are beyond Cerecor’s control), which could cause
actual results to differ from the forward-looking statements. Such
statements may include, without limitation, statements with respect
to Cerecor’s plans, objectives, projections, expectations and
intentions and other statements identified by words such as
“projects,” “may,” “will,” “could,” “would,” “should,” “continue,”
“seeks,” “aims,” “predicts,” “believes,” “expects,” “anticipates,”
“estimates,” “intends,” “plans,” “potential,” or similar
expressions (including their use in the negative), or by
discussions of future matters such as: the development of product
candidates or products; timing and success of trial results and
regulatory review; potential attributes and benefits of product
candidates; the expansion of Cerecor’s drug portfolio; and other
statements that are not historical. These statements are based upon
the current beliefs and expectations of Cerecor’s management but
are subject to significant risks and uncertainties, including: drug
development costs, timing and other risks, including reliance on
investigators and enrollment of patients in clinical trials;
regulatory risks; reliance on and the need to attract, integrate
and retain key personnel; Cerecor’s cash position and the potential
need for it to raise additional capital; and those other risks
detailed in Cerecor’s filings with the Securities and Exchange
Commission. Actual results may differ from those set forth in the
forward-looking statements. Except as required by applicable law,
Cerecor expressly disclaims any obligations or undertaking to
release publicly any updates or revisions to any forward-looking
statements contained herein to reflect any change in Cerecor’s
expectations with respect thereto or any change in events,
conditions or circumstances on which any statement is based.
For Media and Investor Inquiries
James Harrell, Chief Commercial OfficerCerecor
Inc.jharrell@cerecor.com623.439.2220 office
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