Aeglea BioTherapeutics Provides Update on AEB1102 Clinical Program in Rare Diseases and Cancer

--EMA Issued a Positive Opinion for Orphan Drug Designation for AEB1102--

--Initiated Two Phase 1 Clinical Trials of AEB1102 for Treatment of Hyperargininaemia and Hematological Malignancies--

AUSTIN, Texas, July 18, 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 provided a regulatory and clinical update for its AEB1102 program. AEB1102, the company’s lead investigational molecule, is a recombinant human enzyme designed to degrade the amino acid arginine and is being developed to treat two extremes of arginine metabolism.

“We have developed a clinical program for AEB1102 with three ongoing clinical trials. The momentum for this program continues to build with AEB1102 receiving Fast Track designation from the FDA for hyperargininaemia in May and also receiving a positive opinion for Orphan Drug Designation from the European Medicines Agency this month,” said David G. Lowe, Ph.D., co-founder, president and chief executive officer at Aeglea. “We believe this molecule has great potential for patients with rare diseases and cancer, and we look forward to gathering critical data as we continue to advance our programs.”

AEB1102 for the Treatment of Rare Diseases

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending Orphan Drug Designation for AEB1102 for the treatment of hyperargininaemia in the European Union.

The EMA’s Orphan Drug Designation provides benefits, such as protocol assistance, fee reductions and ten years of market exclusivity upon regulatory approval, to companies working to develop treatments for life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU and where no satisfactory treatments are available.

Additionally, Aeglea’s Phase 1 open-label, dose escalation study of AEB1102 for the treatment of patients with Arginase I deficiency has been initiated. The single arm study will enroll up to six patients to assess the safety, tolerability and pharmacokinetics of AEB1102. Enrollment is anticipated to be completed in 2016 with topline data expected in the first half of 2017.

Hyperargininaemia, or excessively high levels of arginine, is the result of a hereditary deficiency of arginase I. Arginase I deficiency is a urea cycle disorder caused by a mutation in the arginase I gene that leads to the inability to degrade arginine, the last step of the urea cycle. AEB1102 is intended to replace the function of arginase I in patients by returning elevated blood arginine levels to the normal physiological range.

AEB1102 for the Treatment of Arginine-Dependent Tumors

A Phase 1 open-label, dose escalation study of AEB1102 in advanced solid tumors, has currently enrolled six cohorts. To date, the maximum tolerated dose (MTD) has not yet been determined. Enrollment, which was initiated in October 2015, is expected to be completed by the end of 2016. Topline data are expected in 2017.

Following the determination of the MTD, additional cohorts of approximately 25 patients each with a specific solid tumor subtype will be enrolled and treated with AEB1102 at the MTD. The solid tumor subtype expansion arms are expected to initiate in 2017.

Additionally, Aeglea has initiated a Phase 1 open-label, dose escalation study of AEB1102 in patients with relapsed refractory acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The objectives of the trial will be to determine the MTD and recommended Phase 2 dose as well as to assess the safety profile of AEB1102. Based off of data from the Phase 1 trial of AEB1102 for the treatment of advanced solid tumors, the company has recently amended the trial protocol to increase the starting dose in patients with AML and MDS. Enrollment is expected to be completed in 2017.

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.

“We are pleased to have initiated our Arginase I deficiency and hematological malignancies trials, advancing our programs to bring AEB1102 one step closer to patients,” said Sandra Rojas-Caro, M.D., chief medical officer at Aeglea. “There remain significant unmet medical needs for both Arginase I deficiency and cancer. Fulfilling this need and bringing effective treatments to patients are at the center of what drives us at Aeglea.”

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 solid tumor cancer patients 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 Aeglea BioTherapeutics

Aeglea is a biotechnology company committed to developing engineered human enzymes for the treatment of rare diseases and cancers associated with abnormal amino acid metabolism. 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 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 ongoing Phase 1 clinical trials, studying AEB1102 for the treatment of patients with Arginase I deficiency as well as cancer 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.

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, the benefits of orphan drug designation, success in our collaborations 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 March 31, 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.

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

Media Contact: Kelly France, Ph.D. BrewLife 415.946.1076 media@aegleabio.com Investor Contact: Charles N. York II Aeglea BioTherapeutics investors@aegleabio.com

Source:Aeglea Biotherapeutics

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