Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on
becoming a leader in the development and commercialization of
treatments for rare and orphan diseases, today announced a
collaboration with The Frontiers in Congenital Disorders of
Glycosylation Consortium (FCDGC) led by Eva Morava-Kozicz, M.D.
Ph.D., Principal Investigator of the trial, Professor of Medical
Genetics, Senior Associate Consultant, Department of Clinical
Genomics, Mayo Clinic and Editor in Chief of the Journal of
Inherited Metabolic Disease on a prospective pivotal trial
evaluating the safety and efficacy of CERC-801 in patients
suffering from Phosphoglucomutase-1 deficiency related congenital
disorders of glycosylation (PGM1-CDG).
Dr. H. Jeffrey Wilkins, M.D., Chief Medical Officer for Cerecor,
stated, “We are delighted to be working with the premier group of
thought leaders from The FCDGC to study CERC-801 in patients
suffering from PGM1-CDG. We intend to use the data generated from
this prospective trial to support our submission package to the FDA
for CERC-801 as the first approved product for the treatment and
management of PGM1-CDG.”
This trial will study the safety, tolerability, and efficacy of
CERC-801 in patients with PGM1-CDG using daily therapeutic doses of
CERC-801 in approximately ten patients. Outcome measures will
include evaluation of clinical symptoms and clinically meaningful
biomarkers.
Andrea Miller, JD, MHA, President & Founder of CDG CARE,
stated, “We are truly excited to see the collaboration between
FCDGC and Cerecor and this prospective trial in PGM1-CDG. CDG is an
area of high unmet need where there are no approved therapies
today. The possibility for there to be an approved therapy for
PGM1-CDG is exciting and has the potential to improve the quality
of life of patients suffering from this ultra rare form of
CDG.”
About PGM1-CDG
CDGs 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.
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.
While there are no U.S. Food and Drug Administration-approved
treatments for the treatment of CDGs, dietary monosaccharide
formulations have been shown to alleviate several of the clinical
manifestations in CDG patients. These restorative monosaccharide
therapies work by increasing the availability of metabolic
intermediates for glycoprotein synthesis. PGM1-CDG is caused by
mutation in the PGM1 gene encoding an enzyme responsible for the
interconversion of glucose-6-phosphate to glucose-1-phosphate.
Glucose-1-phosphate can be utilized to supply UDP-galactose, a
substrate that donates galactose subunits for glycoprotein
synthesis. CERC-801 uses therapeutic doses of D-galactose to
restore glycosylation in patients with PGM1 deficiency.
About FCDGC
The Frontiers in CDG Consortium leverages cross-disciplinary,
team-based clinical science to address decades of unresolved
questions, increase clinical trial readiness, advance and share
knowledge, develop treatments, and address current unmet patient
needs. The Consortium establishes a nation-wide network of ten
regional academic centers, the Sanford Burnham Presbyterian Medical
Discovery Institute and the patient advocacy group CDG CARE.
CDG CARE (Community Alliance and Resource Exchange) is the
Patient Advocacy Group representing all Congenital Disorders of
Glycosylation (CDG) and Deglycosylation (CDDG) for the FCDGC. CDG
CARE is a nonprofit 501(c)(3) organization founded by parents
seeking information and support for a group of disorders known as
CDG. Their mission is to promote greater awareness and
understanding of CDG, to provide information and support to
families affected by CDG, and to advocate for and fund scientific
research to advance the diagnosis and treatment of all CDGs.
About CERC-800s
CERC-801, CERC-802 and CERC-803 are restorative monosaccharide
therapies with known therapeutic utility for the treatment of
select CDGs. Oral administration at therapeutic doses of CERC-801,
CERC-802, and CERC-803 replenishes critical metabolic intermediates
that are reduced or absent due to genetic mutation, overcoming
single enzyme defects in respective CDGs to support glycoprotein
synthesis, maintenance and function.
About Cerecor
Cerecor is a biopharmaceutical company focused on becoming a
leader in the development and commercialization of treatments for
rare and orphan diseases. The company is advancing its
clinical-stage pipeline of innovative therapies that address unmet
patient needs within rare and orphan diseases. The company's
rare disease pipeline includes CERC-801, CERC-802 and CERC-803
("CERC-800 compounds"), which are therapies for inherited metabolic
disorders known as congenital disorders of glycosylation. The
U.S. Food and Drug Administration ("FDA") granted Rare Pediatric
Disease Designation (RPDD) and Orphan Drug Designation (ODD) to all
three CERC-800 compounds, thus potentially qualifying the Company
to receive a Priority Review Voucher (PRV) upon approval of each
new drug application (NDA). The company is also developing
CERC-002, CERC-006 and CERC-007. CERC-002 is an anti-LIGHT
monoclonal antibody being developed for the treatment of severe
pediatric-onset Crohn's disease, and is also being studied for
COVID-19 acute respiratory distress syndrome. CERC-006 is a
dual mTOR inhibitor being developed for the treatment of complex
lymphatic malformations and has been granted ODD and RPDD by the
FDA, thus potentially qualifying the company to receive a fourth
PRV upon approval of an NDA. CERC-007 is an anti-IL-18
monoclonal antibody being developed for the treatment of autoimmune
inflammatory diseases such as Still’s disease and multiple
myeloma.
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,” “might,” “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; 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, which might be slowed by the COVID-19
pandemic; regulatory risks; Cerecor's cash position and the
potential need for it to raise additional capital; general economic
and market risks and uncertainties, including those caused by the
COVID-19 pandemic; 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 HarrellInvestor RelationsChief Commercial OfficerCerecor
Inc.jharrell@cerecor.com623.439.2220 office
Cerecor (NASDAQ:CERC)
Historical Stock Chart
From Mar 2024 to Apr 2024
Cerecor (NASDAQ:CERC)
Historical Stock Chart
From Apr 2023 to Apr 2024