AUSTIN, Texas, Oct. 26, 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 Orphan Drug 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.
Additionally, the European Medicines Agency (EMA) Committee for
Orphan Medicinal Products (COMP) has issued a positive opinion
recommending Orphan Drug Designation for ACN00177 for the treatment
of Homocystinuria in the European Union. ACN00177 is a novel
engineered human enzyme therapy designed to lower the total level
of homocysteine in the plasma.
"People with Homocystinuria are living with serious
complications, including severe vision issues, skeletal
abnormalities, intellectual disability and a high risk of
thrombosis which is a common cause of early death, yet many
patients do not have effective treatments. These designations
underscore the very high unmet need for new treatments for this
progressive disease and reaffirms our belief that ACN00177 has the
potential to change the lives of people with Homocystinuria," said
Anthony Quinn, M.B Ch.B, Ph.D.,
president and chief executive officer of Aeglea. "We look forward
to dosing the first person in our Phase 1/2 clinical trial of
ACN00177 which will move us one step closer to our goal of
providing an impactful therapy for people with Homocystinuria who
are in need of new treatment options."
The FDA grants Orphan Drug Designation to drugs and biologics
that are intended for the safe and effective treatment, diagnosis
or prevention of rare diseases or disorders that affect fewer than
200,000 people in the U.S. The designation provides certain
benefits, including the potential for up to seven years of market
exclusivity upon FDA approval, prescription drug user fee waivers
and tax credits for qualified clinical trials. The EMA's Orphan
Drug Designation provides benefits, such as protocol assistance,
fee reductions and up to ten years of market exclusivity upon
regulatory approval, to companies working to develop treatments for
life-threatening or chronically debilitating conditions that affect
no more than five in 10,000 people in the EU and where no
satisfactory treatments are available.
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. The Company 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 our cash forecasts, 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, the potential addressable markets of
the our product candidates 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
June 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.