Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on
becoming a leader in development and commercialization of
treatments for rare and orphan diseases in pediatrics and
neurology, announced today that it has completed dosing in a Phase
l study for CERC‑801, an ultra-pure, oral, crystalline formulation
of D-galactose currently in development for the treatment of
Phosphoglucomutase 1 (PGM1) deficiency, also known as PGM1-CDG.
The single-center, US-based safety, tolerability and
pharmacokinetic study was an open-label, randomized, single-dose,
4-way crossover study in 16 healthy adult volunteers. CERC-801 was
shown to be safe and well-tolerated at the studied doses, with no
serious adverse events. CERC‑801 related adverse events were mild
and transient. Pharmacokinetic (PK) data is expected in the summer
of 2019.
“We believe the excellent safety profile and anticipated PK data
will provide a solid foundation for the ongoing development of
CERC-801,” said Dr. Perry Calias, PhD, Chief Scientific Officer at
Cerecor. “We have also initiated a 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. 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 Congenital Disorders of Glycosylation
Congenital disorders of glycosylation (CDG) are a rapidly
expanding group of rare Inborn Errors of Metabolism (IEM) due to
defects in glycosylation. Glycosylation is the process by which
carbohydrate complexes are created, modified and attached to
proteins and lipids, creating glycoconjugates that are essential
for cell structure and function in all tissues and organs. CDG is
caused by a specific inherited mutation and more than 100 CDGs have
been identified to date. CDG typically present in infancy and can
be associated with a broad spectrum of symptoms that include
severe, disabling or life-threatening cases. 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.
About CERC-801
CERC-801 is an ultra-pure, oral, crystalline formulation of
D-galactose, a naturally occurring monosaccharide found in dairy
products and fruit. D-Galactose 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-801 has been granted Orphan
Drug Designation and awarded Rare Pediatric Disease Designation by
the FDA, granting eligibility for receipt of a Priority Review
Voucher upon approval of an NDA.
About PGM1-CDG
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. Substrate replacement with CERC-801 in
patients with PGM1 Deficiency is hypothesized to increase available
UDP-galactose pools and repair galactose-deficient
glycoproteins.
About CERC-802
CERC-802 is an ultra-pure, oral, crystalline formulation of
D-mannose that serves as a substrate replacement therapy. Oral
administration of D-mannose replenishes critical metabolic
intermediates, reduced or absent due to a genetic mutation, to
support glycoprotein synthesis, maintenance, and function. CERC-802
has been granted Orphan Drug Designation and awarded Rare Pediatric
Disease Designation by the FDA, granting eligibility for receipt of
a Priority Review Voucher upon approval of an NDA.
About MPI-CDG
MPI Deficiency is caused by loss of function mutations in
the MPI gene encoding an enzyme responsible for the
conversion of fructose-6-phosphate to mannose-6-phosphate,
eventually providing GDP-mannose as a donor substrate for
glycoprotein synthesis.
About CERC-803
CERC-803 is an ultra-pure, oral, crystalline formulation of
L-fucose currently in development for the treatment of Leukocyte
Adhesion Deficiency Type II (LADII), also known as SLC35C1-CDG
(CDG-IIc). CERC-803 has been granted Orphan Drug Designation and
awarded Rare Pediatric Disease Designation by the FDA, granting
eligibility for receipt of a Priority Review Voucher upon approval
of an NDA.
About SLC35C1-CDG
Currently there is no approved product for the treatment
CDG-IIc. CDG-IIc is caused by loss-of-function mutations in
the SLC35C1 gene encoding a GDP-fucose transporter found
in the Golgi apparatus, for the primary site for protein
fucosylation. Substrate replacement with CERC-803 in patients with
CDG-IIc is expected to reverse the hypofucosylation defects found
in this patient population.
About the CDG Retrospective Study
(CLIN800-401)
Clin800-401 is a non-interventional, retrospective study of the
natural history and routine clinical management of CDG. The
objective of the study is to collect natural history and
treatment-related data of patients diagnosed with
Phosphoglucomutase 1 (PGM1)-deficiency, Mannose-Phosphate Isomerase
(MPI) Deficiency (MPI-CDG) and Leukocyte Adhesion Deficiency Type
II (LADII), also known as SLC35C1-CDG (CDG-IIc). The study is being
conducted at approximately 20 sites in the United States and in
Europe.
About Cerecor
Cerecor is a biopharmaceutical company focused on becoming a
leader in the development of orphan neurologic and pediatric
therapies that make a difference in the lives of patients. The
Company’s pipeline is led by CERC-301, which Cerecor is currently
exploring as a novel treatment for neurogenic orthostatic
hypotension. Cerecor has six additional programs in development,
including CERC-406 for Parkinson’s Disease, CERC-611 for epilepsy,
CERC-801, CERC-802, and CERC 803 for Congenital Disorders of
Glycosylation and CERC-913 for DGUOK Deficiency a mitochondrial DNA
Depletion Syndrome. The Company’s R&D efforts are supported by
revenue from its franchise of commercial medications led by
Poly-Vi-Flor® and Tri-Vi-Flor® (multivitamin and fluoride
supplement tablet, chewable and suspension/drops). In
February 2018, the Company added to its marketed product portfolio
by acquiring Karbinal™ ER, AcipHex® Sprinkle™, Cefaclor for Oral
Suspension, and Flexichamber™.
For more information about Cerecor, please visit
www.cerecor.com.
Forward-Looking Statements
This 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 (including as it may be impacted by government
shut-downs), 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; Cerecor’s cash position
and the potential need for it to raise additional capital; risks
associated with acquisitions, including the need to quickly and
successfully integrate acquired assets and personnel; 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
John Woolford Westwicke
Partnersjohn.woolford@westwicke.com443-213-0506 office410-375-3658
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