AUSTIN, Texas and STOCKHOLM, March 22,
2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc.
(NASDAQ:AGLE), a clinical-stage biotechnology company developing a
new generation of human enzyme therapeutics as innovative solutions
for rare metabolic diseases, and Immedica Pharma AB (Immedica)
today announced the license and supply agreement for
pegzilarginase, a novel, recombinant human arginase 1 enzyme that
has been shown to lower toxic levels of the amino acid arginine in
patients with Arginase 1 Deficiency (ARG1-D). People living with
ARG1-D, a rare, progressive disease characterized by high levels of
arginine, may experience severe spasticity-related mobility
limitations, seizures, developmental delay, intellectual disability
and early mortality.
"With a proven track record of bringing important products to
the rare disease community in Europe and the Middle East, Immedica represents an ideal
partner as we plan for the potential approval and commercialization
of pegzilarginase in key markets beyond the U.S.,"
said Anthony G. Quinn, M.B. Ch.B., Ph.D., president and chief
executive officer of Aeglea. "There is an urgent need to deliver
new therapies for Arginase 1 Deficiency due to its progressive and
devastating nature. We are pleased to have a partner who believes
in the potential of this program and shares our excitement for
bringing pegzilarginase to market to further our mission of
improving the lives of families affected by Arginase 1
Deficiency."
"We are excited to enter this partnership with Aeglea and expand
our growing portfolio to include pegzilarginase, which fits
strategically very well within our existing rare disease and urea
cycle disorder portfolio and commercial infrastructure," says
Anders Edvell, CEO of Immedica. "This agreement strengthens our
position as a partner of choice for rare disease
collaborations."
Under the terms of the agreement, Immedica will make an upfront
payment of $21.5 million to Aeglea.
Additionally, Aeglea will be eligible to receive up to
approximately $130 million in
regulatory and commercial milestones as well as mid-twenties
percentage royalties on net sales. Immedica receives
commercialization rights in Europe
and several Middle East countries.
Aeglea will continue to be responsible for certain clinical
development activities and the manufacturing of pegzilarginase, and
retains commercialization rights in the U.S. and rest of the
world.
Pegzilarginase is currently being investigated as a treatment
for ARG1-D in the PEACE study, a pivotal Phase 3 clinical trial
with topline data expected in the fourth quarter of 2021. In Phase
1/2 and ongoing open-label extension studies, pegzilarginase has
been shown to lower toxic levels of arginine in patients with
ARG1-D. After a 56 week treatment period, all 13 patients achieved
plasma arginine levels within the target range (<200µM) and 11
patients (85%) were considered clinical responders based on
improvements in mobility assessments. Pegzilarginase was also shown
to have a favorable safety profile with the most common
treatment-related serious adverse events being hypersensitivity and
hyperammonemia, both of which were infrequent, expected and managed
with standard of care. Most treatment-related adverse events were
mild and decreased in frequency over time.
About Pegzilarginase in Arginase 1 Deficiency
Pegzilarginase is a novel recombinant human enzyme, which has
been shown to rapidly and sustainably lower levels of the amino
acid arginine in plasma. Aeglea is developing pegzilarginase for
the treatment of patients with Arginase 1 Deficiency (ARG1-D), a
rare debilitating and progressive disease characterized by the
accumulation of arginine. ARG1-D presents in early childhood and
patients experience spasticity, seizures, developmental delay,
intellectual disability and early mortality. Aeglea's Phase 1/2 and
Phase 2 open-label extension (OLE) data for pegzilarginase in
patients with ARG1-D demonstrated clinical improvements and
sustained lowering of plasma arginine. The Company's ongoing
single, global pivotal Phase 3 PEACE trial is designed to assess
the effects of treatment with pegzilarginase versus placebo over 24
weeks with a primary endpoint of plasma arginine reduction.
Pegzilarginase has received multiple regulatory designations,
including Rare Pediatric Disease, Breakthrough, Fast Track and
Orphan Drug Designations from the U.S. Food and Drug Administration
as well as Orphan Drug Designation from the European Medicines
Agency.
About Aeglea BioTherapeutics
Aeglea BioTherapeutics is a clinical-stage biotechnology company
redefining the potential of human enzyme therapeutics to benefit
people with rare and devastating metabolic diseases with limited
treatment options. 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. In the second quarter of 2020,
the Company initiated a Phase 1/2 clinical trial of AGLE-177 for
the treatment of Homocystinuria. AGLE-177 has also been granted
Rare Pediatric Disease Designation. Aeglea has an active discovery
platform focused on engineering small changes in human enzymes to
have a big impact on the lives of patients and their families. For
more information, please visit http://aeglea.com.
About Immedica
Immedica is a fast-growing private European niche pharma group.
Its headquarters is based in Stockholm,
Sweden, and it has pan-European and Middle East commercial coverage. In addition,
Immedica provides some of its products to other parts of the world
via a network of regional distributors.
Immedica provides significant know-how and experience from
commercialization of niche/specialty care products across
Europe and the Middle East, and the company's management team
has an outstanding track record of operating niche pharma products
internationally. Immedica has capabilities to provide optimal
access of specialty care medicines to patients with significant
medical needs, including key areas such as regulatory affairs,
pharmacovigilance, medical affairs, pricing & reimbursement,
and product distribution.
Aeglea 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 ability to obtain regulatory approval for, and
commercialize, pegzilarginase, recognize milestone and royalty
payments from our agreement with Immedica, 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 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.