Aeglea BioTherapeutics Doses First Patients in Pha

AUSTIN, Texas, Sept. 15, 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 genetic rare diseases and cancer, today announced the dosing of the first two patients in a Phase 1 trial of AEB1102, a recombinant human enzyme designed to degrade the amino acid arginine, for the treatment of patients with Arginase I deficiency.

“Initiating dosing of Aeglea's lead product candidate AEB1102 in two patients with this genetic rare disease is a key milestone as this is the first clinical trial of an enzyme replacement therapy for Arginase I deficiency.  We took an important step forward for this patient community and the company as we continue to build on the momentum of this program after receiving both Orphan Drug Designation from the European Commission and Fast Track designation from the FDA earlier this year,” said David G. Lowe, Ph.D., co-founder, president and chief executive officer. “We now have three ongoing clinical trials investigating AEB1102 in a genetic rare disease, advanced solid tumors and hematological malignancies and look forward to presenting data in 2017.”

“If left untreated, Arginase I deficiency can be a devastating disease for patients and their families,” said Sandra Rojas-Caro, M.D., chief medical officer. “We believe AEB1102 has the potential to address this unmet medical need by acting on the underlying cause of the disease.”

Roberto Zori, M.D., professor and chief, Division of Genetics and Metabolism at the University of Florida and an investigator in the trial said, “We do not at this moment have any specific therapeutic available for our patients with Arginase I deficiency and we are therefore happy to participate in this effort to better understand the disease and to develop treatments for this condition.” 

About the Trial

The Phase 1, multicenter, single arm, open label, dose escalation trial of AEB1102 will enroll up to six patients, 18 years of age or older, with Arginase I deficiency.  The primary endpoint of the trial is safety and tolerability of intravenous administration of AEB1102 in patients with Arginase I deficiency.  The trial will also evaluate the pharmacokinetic and pharmacodynamic effects of single ascending doses of AEB1102 including plasma arginine levels.

Please refer to www.clinicaltrials.gov for additional clinical trial details.

About Arginase I Deficiency

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.

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 are predicted 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 three Phase 1 clinical trials to evaluate the safety and tolerability of AEB1102 in patients with Arginase I deficiency, advanced solid tumors and hematological malignancies. Data from the Phase 1 trial of AEB1102 in patients with advanced solid tumors demonstrated that AEB1102 has the ability to reduce blood arginine levels, providing initial human proof of mechanism. AEB1102 has received Fast Track designation from the FDA for the treatment of hyperargininemia secondary to Arginase I deficiency and Orphan Drug Designation from the European Commission for the treatment of hyperargininemia.

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 Phase 1 clinical trials, studying AEB1102 for the treatment of patients with Arginase I deficiency, advanced solid tumors and hematological malignancies. The company is building a pipeline of additional product candidates targeting key amino acids, including AEB4104, which degrades homocystine, a target for a genetic rare disease, as well as two potential treatments for cancer, AEB3103, which degrades cysteine/cystine, and AEB2109, which degrades methionine.

For more information , 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, objectives and success of our 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 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 kboothe@brewlife.com Investor Contact: Charles N. York II Chief Financial Officer Aeglea BioTherapeutics investors@aegleabio.com