Cerecor Announces Positive Phase I Safety Data for CERC-801 in Healthy Volunteers
ROCKVILLE, Md., April 29, 2019 (GLOBE NEWSWIRE) -- 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.
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.
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.
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.
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.
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.
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.
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.
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.
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Released April 29, 2019