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Aeglea BioTherapeutics Receives FDA Rare Pediatric Disease Designation For ACN00177 For The Treatment Of Homocystinuria

Company eligible to receive Priority Review Voucher upon FDA approval of ACN00177

AUSTIN, Texas, Dec. 1, 2020 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (AGLE) - Get Aeglea BioTherapeutics Inc Report, a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high burden diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to ACN00177 for the treatment of Homocystinuria, a serious metabolic disorder characterized by elevated plasma homocysteine which leads to a wide range of life-altering complications and reduced life expectancy. ACN00177 is a novel engineered human enzyme therapy designed to lower the total level of homocysteine in the plasma. Aeglea initiated a Phase 1/2 clinical trial earlier this year to investigate ACN00177 for the treatment of Homocystinuria.

"Homocystinuria is a serious and progressive disease that significantly impacts the quality of life of patients - with a high risk of life threatening blood clots, severe vision issues, skeletal abnormalities, developmental delay and intellectual disability," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "There is a significant need for new and better therapies for Homocystinuria patients given the lack of effective treatment options for many patients. The Rare Pediatric Disease Designation speaks to the significant impact of this disease beginning in childhood and is an important incentive in promoting drug development to improve outcomes in this patient population."

The FDA grants rare pediatric disease designation for diseases with serious or life-threatening manifestations that primarily affect fewer than 200,000 people in the United States and who are less than 18 years old. If a Biologics License Application (BLA) for the treatment of Homocystinuria is approved by the FDA, Aeglea may be eligible to receive a Priority Review Voucher (PRV), which can be redeemed to obtain priority review for any subsequent marketing application or may be sold or transferred. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

About ACN00177 in Homocystinuria

Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities including severe osteoporosis, developmental delay, intellectual disability, lens dislocation and severe near-sightedness. ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. With this mechanism, ACN00177 is intended to lower the abnormally high blood levels of homocysteine in patients with Homocystinuria. Preclinical data demonstrated that ACN00177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. ACN00177 has received both U.S. and EU Orphan Drug Designation. Aeglea initiated a Phase 1/2 trial in the second quarter of 2020 and continues patient identification and administrative activities.

About Aeglea BioTherapeutics

Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare and other high burden diseases. Aeglea's lead product candidate, pegzilarginase, is in a pivotal Phase 3 trial for the treatment of Arginase 1 Deficiency and has received both Rare Pediatric Disease and Breakthrough Therapy Designation. The Company initiated a Phase 1/2 clinical trial of ACN00177 for the treatment of Homocystinuria in the second quarter of 2020. Aeglea has an active discovery platform, with the most advanced program for Cystinuria. For more information, please visit

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 potential addressable markets of our product candidates, the timing and success of our clinical trials and related data, the timing and expectations for regulatory submissions and approvals, timing and results of meetings with regulators, the timing of announcements and updates relating to our clinical trials and related data, our ability to enroll patients into our clinical trials, the expected impact of the COVID-19 pandemic on our operations and clinical trials, success in our collaborations, our cash forecasts, 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 September 30, 2020 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.

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