AUSTIN, Texas, Dec. 1, 2020 /PRNewswire/ -- Aeglea
BioTherapeutics, Inc. (NASDAQ: AGLE), 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
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 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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SOURCE Aeglea BioTherapeutics, Inc.