AEB5100 is an Engineered Human Enzyme with Novel Cystine Degrading Activity
Presentation Today at 5:54 p.m. PT at the 2018 American Society of Nephrology (ASN) Annual Meeting
AUSTIN, Texas, Oct. 26, 2018 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ: AGLE), a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer, announced that it will present preclinical data highlighting the discovery and activity of AEB5100 today in an oral presentation titled “Enzymatic Degradation of Cystine Decreases Nephrolithiasis in a Mouse Model of Cystinuria.” The Company will present today at 5:54 p.m. PT in Location 33C at the San Diego Convention Center at the 2018 American Society of Nephrology (ASN) Annual Meeting in San Diego, CA.
Cystinuria is a rare, debilitating genetic disorder characterized by frequent and recurrent kidney stone formation, which may require multiple surgical interventions with an increased risk of chronic kidney disease. Patients with cystinuria have mutations in amino acid transporters that result in markedly increased urine cystine concentration leading to kidney stone formation. Medical need remains high as current therapies for cystinuria demonstrate limited effectiveness and are associated with an increased risk of serious complications.
“Current disease management for cystinuria presents significant challenges for patients and clinicians, and approved therapies are associated with side effects and limited efficacy,” said James Wooldridge, M.D., chief medical officer of Aeglea. “This novel approach demonstrates substantial decreases in the number and size of kidney stones in a preclinical cystinuria model, which supports advancement of this program into IND enabling studies.”
The presentation describes the engineering of the cystathionine gamma-lyase (CGL) enzyme scaffold into a novel molecule with a unique specificity for degrading both cystine and cysteine. AEB5100 decreases cystine and cysteine in the plasma of mouse models of cystinuria. Reductions in plasma cystine and cysteine levels with a single dose of AEB5100 resulted in significant lowering of urine cystine levels and a decrease in urine cystine crystals. Furthermore, repeat dosing of AEB5100 reduced both the volume and number of kidney stones.
About AEB5100 (Cystinase) in Cystinuria
AEB5100 is a novel recombinant human enzyme that degrades cystine and the parent amino acid cysteine. Aeglea is developing AEB5100 for the treatment of patients with cystinuria, a rare genetic disease characterized by frequent and recurrent kidney stone formation requiring multiple procedural interventions, and by an increased risk of chronic kidney disease. Cystinuria occurs due to genetic mutations in amino acid transporters that lead to increased amounts of cystine in the urine. This results in high cystine concentrations in the urine and formation of kidney stones. Preclinical data demonstrated that AEB5100 lowered blood levels of cystine and cysteine, decreased the amount of cystine in the urine and reduced kidney stone formation in a mouse model of cystinuria.
About Aeglea BioTherapeutics
Aeglea is a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer. The Company is developing pegzilarginase, its lead investigational therapy, for the treatment of Arginase 1 Deficiency, as monotherapy in arginine-dependent cancers and in combination with an immune checkpoint inhibitor for small cell lung cancer. In addition, Aeglea has an active research pipeline of other human enzyme-based approaches in both therapeutic areas. 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. 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, 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 June 30, 2018 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.
Sharon Merrill Associates
Director, Finance & Investor Relations