AUSTIN, Texas, March 18, 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, today reported
its fourth quarter and full year 2020 financial results, and
provided recent corporate and program highlights.
"2020 was a challenging year advancing our clinical trials and
at the same time prioritizing the health and well-being of the rare
disease patients we serve, who often have additional difficulties
and vulnerabilities," said Anthony
Quinn, M.B Ch.B, Ph.D., president and chief executive
officer of Aeglea. "We have been focused on mitigating the impact
of COVID-19 on our clinical timelines and I'm proud of the work the
team has done to put patient needs first while also advancing a
broad range of other impactful activities, as seen with the
regulatory designations received for AGLE-177, our continuous gains
in Arginase 1 Deficiency patient identification and the buildout of
our commercial organization."
Dr. Quinn continued, "With enrollment completion expected this
month and data expected in the fourth quarter for our pivotal Phase
3 PEACE study of pegzilarginase in Arginase 1 Deficiency, and the
potential for first-in-human data from our AGLE-177 Phase 1/2
clinical trial in Homocystinuria, 2021 is shaping up to be a
transformational year and we are well positioned to evolve into a
successful commercial-stage company."
Recent Highlights and Updates
Pegzilarginase in Arginase 1 Deficiency
- As of mid-March, 24 patients have been enrolled and randomized
in the pivotal Phase 3 PEACE clinical trial. An additional nine
patients are in active screening or scheduled to begin screening in
the next two weeks. Trial enrollment is expected to close in March,
with completion of screening and all patients randomized by the end
of April. Topline data is expected in the fourth quarter of
2021.
- As of January, Aeglea has identified over 300 Arginase 1
Deficiency patients in addressable markets. The number of currently
identified patients represents approximately 65% and 30% of the
estimated genetic prevalence populations in the U.S. and EU4 plus
the UK, respectively.
- At the 2021 JP Morgan Healthcare Conference, Aeglea provided an
update on the safety profile of pegzilarginase. As of September 2020, more than 1,350 doses had been
administered in the Phase 1/2 clinical trial and Phase 2 open-label
extension study.
AGLE-177 in Homocystinuria
- In December 2020, Aeglea
announced the U.S. Food and Drug Administration (FDA) granted Rare
Pediatric Disease Designation to AGLE-177 for the treatment of
Homocystinuria. If AGLE-177 is approved by the FDA, the company
will be eligible to receive a Priority Review Voucher.
- In January, Aeglea announced it will be adding clinical trial
sites in Australia for its Phase
1/2 clinical trial.
- In January, the Company presented the results of an analysis
which determined the Classical Homocystinuria (CBS deficiency)
population in global addressable markets may exceed 30,000
patients, with an estimated 15,000-18,000 of those patients to be
non-responsive to the existing standard of care.
Corporate
- Aeglea expanded the Company's Board of Directors to include two
new members. Alison Lawton, who
previously served as president and CEO of Kaleido Biosciences, was
appointed in December 2020.
Sara Brownstein, a principal at
Baker Bros. Advisors LP, was appointed in February 2021.
Upcoming Events
Aeglea will be attending the following virtual investor
conferences:
- Wells Fargo Annual Biotech Corporate Access Day, April 6
- 20th Annual Needham Healthcare Conference, April 12-13
Fourth Quarter and Full Year 2020 Financial Results
As of December 31, 2020, Aeglea
had available cash, cash equivalents, marketable securities and
restricted cash of $148.1 million.
Based on Aeglea's current operating plan, management believes it
has sufficient capital resources to fund anticipated operations
into 2023.
Research and development expenses totaled $15.8 million for the fourth quarter of 2020 and
$17.6 million for the fourth quarter
of 2019. The decrease was primarily associated with completing
certain manufacturing and pre-commercial activities for Aeglea's
lead product candidate, pegzilarginase.
Research and development expenses totaled $59.6 million for the year ended December 31, 2020 and $64.6 million for the year ended December 31, 2019. The decrease was primarily
associated with completing a Phase 1/2 clinical trial in patients
with Arginase 1 Deficiency and closing cancer related trials,
completing certain manufacturing and pre-commercial activities for
Aeglea's lead product candidate, pegzilarginase, along with
supporting toxicology studies for the Homocystinuria
program.
General and administrative expenses totaled $7.0 million for the fourth quarter of 2020 and
$4.3 million for the fourth quarter
of 2019. This increase was primarily due to ramping-up commercial
capabilities and infrastructure.
General and administrative expenses totaled $21.8 million for the year ended December 31, 2020 and $15.7 million for the year ended December 31, 2019. This increase was primarily
due to ramping-up commercial capabilities and infrastructure along
with additional office space to support company growth.
Net loss totaled $22.7 million and
$21.5 million for the fourth quarter
of 2020 and 2019, respectively, with non-cash stock compensation
expense of $1.6 million and
$1.2 million for the fourth quarter
of 2020 and 2019, respectively. Net loss totaled $80.9 million and $78.3
million for the years ended December
31, 2020 and 2019, respectively, with non-cash stock
compensation expense of $6.3 million
and $4.9 million for the years ended
December 31, 2020 and 2019,
respectively.
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 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 FDA as well as Orphan Drug Designation from the European
Medicines Agency.
About AGLE-177 in Homocystinuria
AGLE-177 is a novel recombinant human enzyme, which degrades the
amino acid homocysteine and its related homocystine dimer. Aeglea
is developing AGLE-177 for the treatment of patients with
cystathionine beta synthase (CBS) deficiency, also known as
Classical Homocystinuria, a rare inherited disorder of methionine
metabolism that results in elevated levels of homocysteine and
homocystine. 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. Preclinical data
demonstrated that AGLE-177 improved important disease-related
abnormalities and survival in a mouse model of Homocystinuria.
AGLE-177 has received U.S. Rare Pediatric Disease and Orphan Drug
Designations as well as 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 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.
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 Annual Report on Form 10-K for the year ended
December 31, 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.
Financials
Aeglea
BioTherapeutics, Inc.
|
Consolidated
Balance Sheets
|
|
(In thousands,
except share and per share amounts)
|
|
|
|
|
|
|
December
31,
|
|
|
|
2020
|
|
|
2019
|
|
ASSETS
|
|
|
|
|
|
|
|
|
CURRENT
ASSETS
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
90,095
|
|
|
$
|
19,253
|
|
Marketable
securities
|
|
|
56,178
|
|
|
|
52,696
|
|
Prepaid expenses and
other current assets
|
|
|
3,516
|
|
|
|
2,556
|
|
Total current
assets
|
|
|
149,789
|
|
|
|
74,505
|
|
Restricted
cash
|
|
|
1,842
|
|
|
|
1,500
|
|
Property and
equipment, net
|
|
|
5,642
|
|
|
|
2,385
|
|
Operating lease
right-of-use assets
|
|
|
4,230
|
|
|
|
4,726
|
|
Other non-current
assets
|
|
|
115
|
|
|
|
67
|
|
TOTAL
ASSETS
|
|
$
|
161,618
|
|
|
$
|
83,183
|
|
|
|
|
|
|
|
|
|
|
LIABILITIES AND
STOCKHOLDERS' EQUITY
|
|
|
|
|
|
|
|
|
CURRENT
LIABILITIES
|
|
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
2,254
|
|
|
$
|
3,154
|
|
Operating lease
liabilities
|
|
|
319
|
|
|
|
351
|
|
Accrued and other
current liabilities
|
|
|
13,870
|
|
|
|
14,854
|
|
Total current
liabilities
|
|
|
16,443
|
|
|
|
18,359
|
|
Non-current operating
lease liabilities
|
|
|
5,129
|
|
|
|
4,712
|
|
Other non-current
liabilities
|
|
|
214
|
|
|
|
31
|
|
TOTAL
LIABILITIES
|
|
|
21,786
|
|
|
|
23,102
|
|
|
|
|
|
|
|
|
|
|
STOCKHOLDERS' EQUITY
|
|
|
|
|
|
|
|
|
Preferred stock,
$0.0001 par value; 10,000,000 shares authorized
as of December 31, 2020 and 2019; no
shares issued and outstanding
as of December 31, 2020 and 2019
|
|
|
—
|
|
|
|
—
|
|
Common stock, $0.0001
par value; 500,000,000 shares authorized as of December 31, 2020 and 2019; 47,959,086 shares
and
29,084,437 shares issued and outstanding as of December 31,
2020 and 2019,
respectively
|
|
|
5
|
|
|
|
3
|
|
Additional paid-in
capital
|
|
|
415,824
|
|
|
|
255,142
|
|
Accumulated other
comprehensive income
|
|
|
11
|
|
|
|
51
|
|
Accumulated
deficit
|
|
|
(276,008)
|
|
|
|
(195,115)
|
|
TOTAL
STOCKHOLDERS' EQUITY
|
|
|
139,832
|
|
|
|
60,081
|
|
TOTAL LIABILITIES AND
STOCKHOLDERS' EQUITY
|
|
$
|
161,618
|
|
|
$
|
83,183
|
|
Aeglea
BioTherapeutics, Inc.
|
Consolidated
Statements of Operations
|
|
(In
thousands, except share and per share amounts)
|
|
|
|
Year Ended
December 31,
|
|
|
|
|
2020
|
|
|
2019
|
|
|
2018
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Grant
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
3,888
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
|
59,638
|
|
|
|
64,600
|
|
|
|
36,719
|
|
|
General and
administrative
|
|
|
21,843
|
|
|
|
15,734
|
|
|
|
12,632
|
|
|
Total operating
expenses
|
|
|
81,481
|
|
|
|
80,334
|
|
|
|
49,351
|
|
|
Loss from
operations
|
|
|
(81,481)
|
|
|
|
(80,334)
|
|
|
|
(45,463)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest
income
|
|
|
593
|
|
|
|
2,143
|
|
|
|
1,172
|
|
|
Other expense,
net
|
|
|
(5)
|
|
|
|
(63)
|
|
|
|
(57)
|
|
|
Total other
income
|
|
|
588
|
|
|
|
2,080
|
|
|
|
1,115
|
|
|
Net loss
|
|
$
|
(80,893)
|
|
|
$
|
(78,254)
|
|
|
$
|
(44,348)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(1.52)
|
|
|
$
|
(2.45)
|
|
|
$
|
(2.13)
|
|
|
Weighted-average
common shares outstanding, basic and diluted
|
|
|
53,371,730
|
|
|
|
31,949,633
|
|
|
|
20,822,560
|
|
|
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SOURCE Aeglea BioTherapeutics, Inc.