Cerecor Announces FDA Fast Track Designation for CERC-802 in MPI-CDG

On August 26, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that biopharmaceutical company Cerelco announced that its CERC-802 had been granted FDA Fast Track designation for the treatment of mannose phosphate isomerase deficiency (MPI-CDG).

MPI-CDG is a type of congenital disorder of glycosylation (CDG). CDG is a rare inherited metabolic disease that can cause multiple coagulopathies, liver disease, myopathy, hypoglycemia, and protein-losing enteropathy in patients. Among the patient population with this disease, infants have higher incidence and mortality rates. In CDG patients, misplacement or absence of sugar subunits hinders the body's utilization of monosaccharides, resulting in dysfunctional glycoproteins.

Clinical studies have shown that monosaccharide formulations can alleviate the clinical symptoms of patients with congenital disorders of glycosylation (CDG). This substrate replacement therapy increases the metabolic intermediates required for glycoprotein synthesis, thereby exerting a therapeutic effect on patients.

Mutations in the MPI gene in patients with MPI-CDG lead to mannose phosphate isomerase (MPI) deficiency. Currently, the total number of MPI-CDG patients worldwide is fewer than 50.

CERC-802 is a purified D-mannose formulation. By utilizing this drug as an alternative substrate to promote D-mannose synthesis via the salvage pathway, it can restore the ability of patients with MPI-CDG to produce mannose derivatives. D-mannose is a natural monosaccharide commonly found in animals, microorganisms, and plants; the human body obtains substrates for protein glycosylation through the consumption of such monosaccharides. In addition to Fast Track designation, CERC-802 has been granted Orphan Drug Designation by the FDA, which confers priority review eligibility upon submission of a New Drug Application.

Cerecor, founded in 2011 and headquartered in Baltimore, Maryland, USA, is a leading biopharmaceutical company. The company focuses on the fields of rare diseases, neurology, and pediatric healthcare, developing and commercializing prescription drugs. Cerecor went public on the NASDAQ Stock Market in the United States in October 2015, with the stock ticker symbol CERC.

Cerecor has proposed the CERC-800 program to explore innovative therapies for inborn errors of metabolism and has developed drug candidates including CERC-801, CERC-802, and CERC-803. The FDA has granted orphan drug designation to all three agents, and Cerecor plans to accelerate their development and regulatory submission.

Cerecor is also developing a pipeline of drugs for other indications, such as CERC-913 for the treatment of mitochondrial DNA depletion syndrome (MDS); CERC-301, an NMDA receptor antagonist, for the treatment of neurogenic orthostatic hypotension (nOH) associated with neurodegenerative diseases; and CERC-406, a catechol-O-methyltransferase (COMT) inhibitor for Parkinson’s disease.

Cerecor has launched the dietary supplements Poly-Vi-Flor and Tri-Vi-Flor for infants and children to prevent or treat deficiencies in essential vitamins and fluoride. Additionally, the company markets prescription medications for pediatric conditions, including Millipred, Karbinal ER, AcipHex Sprinkle, and Cefaclor for Oral Suspension.

Dr. Simon Pedder, Executive Chairman of Cerecor’s Board of Directors, stated, “The granting of Fast Track designation for CERC-802 is a critical step in the development of a potential therapy for MPI-CDG, an ultra-rare disorder. We will continue to work closely with the FDA to expedite the development of this drug. Currently, Cerecor’s R&D team is collecting data through clinical trials to support the New Drug Application.”

(Compiled by Xu Xiaoxue)