Aeglea BioTherapeutics Doses First Patient in Phase 1 Trial of AEB1102 for the Treatment of Hematological Malignancies

AUSTIN, Texas, Aug. 29, 2016 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a biotechnology company committed to developing enzyme-based therapeutics in the field of amino acid metabolism to treat rare diseases and cancer, today announced the dosing of the first patient in a Phase 1 trial of AEB1102, a recombinant human enzyme designed to degrade the amino acid arginine, for the treatment of the hematological malignancies acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

“Cancer cells such as those found in AML patients are believed to have a metabolic dependence on arginine for growth. Deprivation of arginine by AEB1102 has the potential to impact this challenging disease,” said David G. Lowe, Ph.D., co-founder, president and chief executive officer. “We are pleased to expand the AEB1102 oncology program to include hematological malignancies in addition to our ongoing Phase 1 trial in advanced solid tumors.”

“We are rapidly executing on our strategy for AEB1102 in oncology, having now dosed our first patient with AML/MDS refractory to hypomethylating agents,” said Sandra Rojas-Caro, M.D., chief medical officer. “Current treatment options for patients who have these forms of hematological malignancies are limited. AEB1102 may have the potential to offer a significant clinical benefit for these patients in need."

About the Trial

The Phase 1, multicenter, single-arm, open-label, dose escalation trial of AEB1102 for the treatment of hematological malignancies is designed to assess the safety and tolerability of AEB1102 as a single agent. The trial will enroll patients with relapsed or refractory AML or MDS refractory to hypomethylating agents. Key objectives of the trial include determining the maximum tolerated dose, pharmacokinetics, pharmacodynamics (including reduction of circulating levels of arginine) and the recommended Phase 2 dose. Disease-specific expansion cohorts will be enrolled at the maximally tolerated or biologically relevant dose.

Please refer to for additional clinical trial details.

About AEB1102

AEB1102 is a recombinant human arginase I enzyme designed to degrade the amino acid arginine. Aeglea is developing AEB1102 to treat two extremes of arginine metabolism, including arginine excess in patients with Arginase I deficiency, as well as some cancers which have been shown to have a metabolic dependency on arginine. In patients with Arginase I deficiency, AEB1102 is intended for use as Enzyme Replacement Therapy to restore the function of arginase I in patients and return elevated blood arginine levels to the normal physiological range. Aeglea is currently conducting a Phase 1 clinical trial in patients with advanced solid tumors to evaluate the safety and tolerability of AEB1102. Data from this trial demonstrated that AEB1102 has the ability to reduce blood arginine levels, providing initial human proof of mechanism.

About Arginine Dependence in Cancer Cells

Dysregulation of amino acid metabolism has been shown to be a key event in tumor growth and development. Unlike healthy cells, these tumors cells have an abnormally high appetite for certain amino acids and are unable to create their own supply, making them vulnerable to starvation through depletion of that amino acid in the blood. AEB1102 is intended to address an unmet need for these tumor types by degrading arginine in the blood, reducing its level below the normal range to starve the tumor.

About Aeglea BioTherapeutics

Aeglea is a biotechnology company committed to developing recombinant human enzymes for the treatment of rare diseases and cancers associated with abnormal amino acid metabolism. The company’s recombinant human enzymes are designed to degrade specific amino acids in the blood in order to reduce toxic levels of amino acids in rare diseases or to starve tumors dependent on amino acids by reducing levels below the normal range. Aeglea’s clinical program for its lead product candidate, AEB1102, includes three recently initiated Phase 1 clinical trials, studying AEB1102 for the treatment of patients with Arginase I deficiency as well as patients with solid tumors or hematological malignancies. 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.

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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, objectives and success of our clinical trials and the potential therapeutic benefits and economic value of our lead product. 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 June 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. BrewLife 415.946.1076 Investor Contact: Charles N. York II Chief Financial Officer Aeglea BioTherapeutics

Source:Aeglea Biotherapeutics