Aeglea Announces Publication Of Nonclinical Data On Lead Molecule, AEB1102, In Human Molecular Genetics

AUSTIN, Texas--(BUSINESS WIRE)--Aeglea BioTherapeutics, Inc., a biopharmaceutical company committed to developing enzyme-based therapeutics in the field of amino acid metabolism to treat inborn errors of metabolism and cancer, today announced publication of nonclinical data on its lead molecule, AEB1102, in Human Molecular Genetics. AEB1102 is an engineered human enzyme designed to degrade arginine, an amino acid, and is being developed to treat cancer and the rare disease arginase I deficiency, which is an inborn error of metabolism leading to toxic levels of arginine in the blood. Study results showed that AEB1102 reduced plasma levels of arginine in a dose-dependent manner in both a neonatal and an adult animal model of arginase I deficiency and that this reduction was sustained with repeated dosing.

“These nonclinical findings further demonstrate the potential of AEB1102 to reduce blood arginine levels and supports the continued development of AEB1102 to treat this rare disease, arginase I deficiency”

“Arginase I deficiency is a serious condition that often causes seizures and intellectual disability, resulting in a significant impact on the quality of life for patients and their caregivers,” said Lindsay C. Burrage, M.D, Ph.D., Assistant Professor, Department of Molecular and Human Genetics, Baylor College of Medicine and lead author of the study. “Elevated arginine levels are thought to contribute to the neurologic features of this disease. However, reducing plasma arginine levels to normal or near-normal levels in patients with arginase I deficiency is challenging with current therapies and thus, new treatment options for these patients are needed.”

“These nonclinical findings further demonstrate the potential of AEB1102 to reduce blood arginine levels and supports the continued development of AEB1102 to treat this rare disease, arginase I deficiency,” said David G. Lowe, Ph.D., co-founder, president and chief executive officer of Aeglea. “Our engineered human enzymes, such as AEB1102, are designed to target specific amino acids in the blood in order to potentially reduce toxic levels of amino acids in inborn errors of metabolism or to exploit the dependence of tumors dependent on amino acids by reducing levels below the normal range.”

The publication, titled “Human Recombinant Arginase Enzyme Reduces Plasma Arginine in Mouse Models of Arginase Deficiency” is available at http://hmg.oxfordjournals.org/content/early/2015/09/12/hmg.ddv352.

About Arginase I Deficiency

Arginase I deficiency, an inborn error of metabolism, is a genetic disorder caused by a deficiency of arginase I (ARG1), a urea cycle enzyme that converts arginine to ornithine and urea. Individuals with arginase I deficiency have elevated plasma arginine levels (hyperarginimenia). Patients display slowing of growth at age one to three years, followed by development of spasticity, slowing of normal cognitive development, and the inability to meet developmental milestones. If untreated, arginase deficiency usually progresses to severe spasticity, loss of ambulation and severe intellectual disability, with seizures common.¹

About Aeglea BioTherapeutics

Aeglea is a biopharmaceutical company committed to developing enzyme-based therapeutics in the field of amino acid metabolism to treat inborn errors of metabolism and cancer. The company’s engineered human enzymes are designed to degrade specific amino acids in the blood in order to reduce toxic levels of amino acids in inborn errors of metabolism or to exploit the dependence of certain cancers on specific amino acids. Aeglea’s lead product candidate, AEB1102, is engineered to degrade the amino acid arginine and is expected to enter clinical trials in 2015 in cancer and 2016 in arginase I deficiency. The company is building a pipeline of additional product candidates targeting key amino acids, including AEB4104, which degrades homocysteine, a target for an inborn error of metabolism, as well as two potential treatments for cancer, AEB3103, which degrades cysteine/cystine, and AEB2109, which degrades methionine.

For more information, visit http://aegleabio.com.

¹ http://www.ncbi.nlm.nih.gov/books/NBK1159/. Accessed August 27, 2015.