On July 30, 2018 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, reported its schedule of medical conference presentations for the fall of 2018 (Press release, Aeglea BioTherapeutics, JUL 30, 2018, View Source [SID1234528358]). The three-conference schedule includes five abstract acceptances on the Company’s latest data concerning the rare genetic disease Arginase 1 Deficiency (ARG1-D), uveal and cutaneous melanoma, and human-derived enzymatic approaches to treating the metabolic disorder homocystinuria.
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"This has been an active and successful year for Aeglea," said Anthony G. Quinn, M.B Ch.B, Ph.D., Aeglea’s president and chief executive officer. "We have a busy fall conference schedule during which we will provide important data updates from our clinical and preclinical programs. The repeat dose update from our ARG1-D trials builds on the initial data presented in April 2018 that demonstrated for the first time that rapid and sustained lowering of plasma arginine levels with pegzilarginase, our lead investigational therapy, was accompanied by clinically relevant treatment effects after only eight weeks of dosing. We look forward to providing new interim data from our rare genetic disease and cancer clinical trials with pegzilarginase in the third and fourth quarters of 2018."
Conference Schedule
Event: Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM)
Date:September 4-7, 2018
Location:Athens, Greece
Title: Improvements in Arginase 1 Deficiency-related Disease Manifestations Following Plasma Arginine Reduction with Pegzilarginase
Event: American Society of Human Genetics (ASHG) Annual Meeting
Date: October 16-20, 2018
Location:San Diego, CA
Title: 1) Improvements in Arginase 1 Deficiency-related Disease Manifestations Following Plasma Arginine Reduction with Pegzilarginase (Early Phase 2 Results); 2) Clinical Features of Arginase 1 Deficiency: Review of Literature Case Series; 3) Improved Survival and Amelioration of Disease-Related Liver Pathology in a Mouse Model of Homocystinuria with a Novel Homocysteine Degrading Enzyme
Event: European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress
Date: October 19-23, 2018
Location: Munich, Germany
Title: Initial Cohort Expansion Results of Sustained Arginine Depletion with Pegzilarginase in Melanoma Patients in a Phase 1 Advanced Solid Tumor Trial
Details regarding the date and time of each abstract will be announced before each conference.
About Pegzilarginase in Arginase 1 Deficiency
Pegzilarginase is an enhanced human arginase that enzymatically degrades the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, a debilitating urea cycle disorder caused by deficiency of a key arginine metabolizing enzyme that leads to severe and progressive hyperargininemia-related neurological abnormalities, hyperammonemia and early mortality. Pegzilarginase is intended for use as an enzyme replacement therapy in patients to reduce elevated blood arginine levels. The Company’s interim Phase 1/2 data demonstrated clinically relevant treatment effects and rapid and sustained lowering of plasma arginine in Arginase 1 Deficiency patients.
About Pegzilarginase in Cancer
Pegzilarginase is an enhanced human arginase that enzymatically degrades the amino acid arginine. In some cancers, tumor cells stop producing specific amino acids and must acquire them from the blood, making the tumor cells susceptible to starvation through depletion of those amino acids. Aeglea is developing pegzilarginase to exploit vulnerabilities in some cancers that lead to an increased dependency on extracellular arginine. Pegzilarginase targets these arginine dependent cancers by depleting blood arginine levels to below the normal range. Preclinical data demonstrated that the resulting arginine starvation inhibits proliferation, induces cell death, increases turnover of cell components and promotes anti-tumor immune responses. The Company’s Phase 1 data in advanced solid tumors demonstrated that pegzilarginase was well tolerated at doses that produced marked and sustained reductions in blood arginine levels below the normal range.