Aeglea BioTherapeutics Doses First Pediatric Patient with Arginase 1 Deficiency in its Phase 1/2 Trial with Pegzilarginase (A...
November 29 2017 - 8:00AM
Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a biotechnology company
committed to the discovery and development of engineered human
enzyme therapeutics for patients with rare genetic diseases and
cancer, today announced dosing of the first pediatric patient in
its ongoing Phase 1/2 clinical trial in patients with Arginase 1
Deficiency and provided a regulatory update on its pegzilarginase
(AEB1102) rare disease program.
Aeglea reached agreement with the FDA that available data
supports the prospect for benefit in pediatric patients and amended
its Phase 1/2 trial protocol to include pediatric dosing tiered by
age beginning with patients aged 14 to 17, and continuing with
patients aged 2 to 13.
“Dosing the first pediatric patient under the protocol amended
during our work with the FDA is a critical step forward for our
pegzilarginase program,” said Anthony Quinn, MB ChB, Ph.D., interim
chief executive officer of Aeglea. “Arginase 1 Deficiency is a
debilitating, progressive disease with significant morbidity and
early mortality. This development allows us, for the first time, to
begin generating data on the safety and activity of pegzilarginase
in pediatric patients as we continue our mission to provide a
treatment for patients living with this devastating disease. The
team at Aeglea is excited and energized by this positive
development, and we look forward to sharing updates from this
trial, with topline results anticipated in the third quarter of
2018.”
“I am pleased that we can now include pediatric patients as we
work to find an impactful treatment for this devastating disease,”
said George Diaz, M.D., Ph.D. professor and chief, Division of
Medical Genetics, Icahn School of Medicine at Mount Sinai. “Given
that modest reductions in plasma arginine levels achieved with a
highly restrictive diet appear to help some children clinically, I
am excited, given the reductions in plasma arginine levels into the
normal range achieved with single doses of pegzilarginase, to see
the clinical impact of longer term dosing in these patients.”
About the Clinical Trial
The Phase 1/2, multicenter, single arm, open label trial of
pegzilarginase will enroll approximately 10 patients, adult and
pediatric, with Arginase 1 Deficiency in the United States, Canada,
and Europe. The trial investigates both single ascending doses
(Part 1) and repeated dosing (Part 2). The primary endpoint of the
trial is safety and tolerability of intravenous administration of
pegzilarginase in patients with Arginase 1 Deficiency. The trial
will also evaluate the pharmacokinetic and pharmacodynamic effects
of repeated doses of pegzilarginase on plasma arginine levels, with
topline results anticipated in the third quarter of 2018.
Please visit www.clinicaltrials.gov for more
information.
About Pegzilarginase (AEB1102) in Arginase 1
Deficiency
Pegzilarginase (AEB1102) is an engineered human arginase 1
enzyme designed to degrade the amino acid arginine. Aeglea is
developing pegzilarginase to treat arginine excess in patients with
Arginase 1 Deficiency, a urea cycle disorder caused by a mutation
in the arginase 1 gene that results in the inability to degrade
arginine. Pegzilarginase is intended for use as enzyme replacement
therapy to restore the function of arginase 1 in patients by
returning elevated blood arginine levels to the normal
physiological range. Aeglea is currently recruiting patients for
its ongoing Phase 1/2 trial for the treatment of Arginase 1
Deficiency. Data from the Phase 1 portion of the trial demonstrated
that pegzilarginase reduced blood arginine levels, providing
initial human proof of mechanism.
About Aeglea BioTherapeutics
Aeglea is a biotechnology company committed to developing
enzyme-based therapeutics in the field of amino acid metabolism to
treat rare genetic diseases and cancer. The company’s engineered
human enzymes are designed to modulate the extremes of amino acid
metabolism in the blood to reduce toxic levels of amino acids in
inborn errors of metabolism or target tumor metabolism for cancer
treatment. Pegzilarginase (AEB1102), Aeglea’s lead product
candidate, is currently being studied in two ongoing Phase 1
clinical trials in patients with advanced solid tumors and acute
myeloid leukemia/myelodysplastic syndrome (AML/MDS). An additional
study of pegzilarginase combined with Merck’s pembrolizumab is
expected to initiate in early 2018. Additionally, Aeglea is
recruiting patients into its ongoing Phase 1/2 trial of
pegzilarginase for the treatment of patients with Arginase 1
Deficiency. The company is building a pipeline of additional
product candidates targeting key amino acids.
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 timing and success of our clinical trials, our
ability to generate data on the safety and activity of
pegzilarginase in pediatric patients, our ability to enroll
patients into our clinical trials, 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, 2017 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.
Media Contact:Kelly Boothe, Ph.D.Pure
Communications415.946.1076media@aegleabio.com
Investor Contact:Charles N. York IIChief
Financial OfficerAeglea BioTherapeuticsinvestors@aegleabio.com
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