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Aeglea BioTherapeutics Doses First Pediatric Patient with Arginase 1 Deficiency in its Phase 1/2 Trial with Pegzilarginase (AEB1102)
Aeglea reached agreement with the
“Dosing the first pediatric patient under the protocol amended during our work with the
“I am pleased that we can now include pediatric patients as we work to find an impactful treatment for this devastating disease,” said
About the Clinical Trial
The Phase 1/2, multicenter, single arm, open label trial of pegzilarginase will enroll approximately 10 patients, adult and pediatric, with Arginase 1 Deficiency in
Please visit www.clinicaltrials.gov for more information.
About Pegzilarginase (AEB1102) in Arginase 1 Deficiency
Pegzilarginase (AEB1102) is an engineered human arginase 1 enzyme designed to degrade the amino acid arginine. Aeglea is developing pegzilarginase to treat arginine excess in patients with Arginase 1 Deficiency, a urea cycle disorder caused by a mutation in the arginase 1 gene that results in the inability to degrade arginine. Pegzilarginase is intended for use as enzyme replacement therapy to restore the function of arginase 1 in patients by returning elevated blood arginine levels to the normal physiological range. Aeglea is currently recruiting patients for its ongoing Phase 1/2 trial for the treatment of Arginase 1 Deficiency. Data from the Phase 1 portion of the trial demonstrated that pegzilarginase reduced blood arginine levels, providing initial human proof of mechanism.
Aeglea is a biotechnology company committed to developing enzyme-based therapeutics in the field of amino acid metabolism to treat rare genetic diseases and cancer. The company’s engineered human enzymes are designed to modulate the extremes of amino acid metabolism in the blood to reduce toxic levels of amino acids in inborn errors of metabolism or target tumor metabolism for cancer treatment. Pegzilarginase (AEB1102), Aeglea’s lead product candidate, is currently being studied in two ongoing Phase 1 clinical trials in patients with advanced solid tumors and acute myeloid leukemia/myelodysplastic syndrome (AML/MDS). An additional study of pegzilarginase combined with Merck’s pembrolizumab is expected to initiate in early 2018. Additionally, Aeglea is recruiting patients into its ongoing Phase 1/2 trial of pegzilarginase for the treatment of patients with Arginase 1 Deficiency. The company is building a pipeline of additional product candidates targeting key amino acids.
For more information, please visit http://aegleabio.com.
Safe Harbor / Forward Looking Statements
This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, statements we make regarding the timing and success of our clinical trials, our ability to generate data on the safety and activity of pegzilarginase in pediatric patients, our ability to enroll patients into our clinical trials, and the potential therapeutic benefits and economic value of our lead product candidate or other product candidates. Further information on potential risk factors that could affect our business and its financial results are detailed in our most recent Quarterly Report on Form 10-Q for the quarter ended September 30, 2017 filed with the Securities and Exchange Commission (
Source: Aeglea BioTherapeutics, Inc.