AUSTIN, Texas, Jan. 17, 2017 -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a biotechnology company committed to developing enzyme-based therapeutics in the field of amino acid metabolism to treat rare genetic diseases and cancer, today announced that it will present new clinical and research data in two presentations at the 2017 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting being held at the Phoenix Convention Center from March 21-25, 2017 in Phoenix, Arizona.
Aeglea’s first presentation will include initial data from its Phase 1, open-label study of AEB1102 in two adult patients with Arginase I deficiency. The results show that AEB1102 was well tolerated and effective at reducing arginine levels in the blood of both patients. Treatment with AEB1102 resulted in a dose-proportional decrease in plasma arginine levels of between 25 and 49 percent of pre-dose levels one week after dosing.
The company’s second presentation includes research data on newborn screening protocols. The research data demonstrated that Arginase I deficiency can be screened with very high efficiency, which underscores the company’s belief that it is a potential candidate for future addition to the Recommended Uniform Screening Panel (RUSP).
Full abstracts are available online at www.acmgmeeting.net. Details of each of the two poster presentations are listed below.
Abstract Title (#809): Initial Results of a Phase I Open-Label Study of AEB1102 Enzyme Replacement Therapy in Adult Patients with Arginase I Deficiency
Date: Thursday, March 23, 2017
Presentation Time: 10 a.m. – 11:30 a.m. MT
Abstract Title (#118): Newborn Screening for Arginase Deficiency in the U.S. – Where Do We Need to Go?
Date: Friday, March 24, 2017
Presentation Time: 10:30 a.m. – Noon MT
About Aeglea BioTherapeutics
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. 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). Additionally, Aeglea is recruiting patients into its ongoing Phase 1/2 trial of AEB1102 for the treatment of patients with Arginase I deficiency. The company is building a pipeline of additional product candidates targeting key amino acids, including AEB4104, which degrades homocystine, 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, 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 safety and efficiency of our newborn screening protocols, potential for Arginase 1 deficiency to be added to the RUSP, potential data from future clinical trials, and the potential therapeutic benefits and economic value of our lead product candidate, AEB1102. 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, 2016, filed with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
Media Contact: Kelly Boothe, Ph.D. Pure Communications 415.946.1076 [email protected] Investor Contact: Charles N. York II Chief Financial Officer Aeglea BioTherapeutics [email protected]


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