Aeglea BioTherapeutics, Inc., announced the U.S. Food and Drug Administration (FDA) has accepted the company’s investigational new drug (IND) application for AEB1102 for the treatment of Arginase I deficiency, a urea cycle disorder that results in elevated levels of the amino acid arginine, which can lead to a range of complications including intellectual disability and spasticity.
Aeglea intends to initiate a Phase 1 dose escalation study designed to assess the safety, tolerability, and pharmacokinetics of AEB1102 in patients with this inborn error of metabolism in the first half of 2016.
AEB1102 is an engineered human enzyme designed to degrade the amino acid arginine and in nonclinical studies has demonstrated the ability to reduce blood arginine levels. In addition to being developed as a potential treatment for Arginase I deficiency, AEB1102 is currently being investigated in a Phase 1 study to treat solid tumors.
