AUSTIN, Texas, Nov. 5, 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 reported its third quarter 2020 financial results,
and provided recent corporate and program highlights.
"Although it's been a challenging environment for biotech with
the global pandemic, we have continued to be productive in our
Arginase 1 Deficiency patient identification and engagement
efforts. I am pleased with the progress we are making overall as
well as specifically at our PEACE trial sites," said Anthony Quinn, M.B Ch.B, Ph.D., president and
chief executive officer of Aeglea. "I am also excited by the
momentum we are building in our Homocystinuria program with the
recent granting of U.S. Orphan Drug Designation and a positive
opinion for EU Orphan Drug Designation for ACN00177."
Recent Highlights and Updates
Pegzilarginase in Arginase 1 Deficiency
- In September, Aeglea announced the PEACE Phase 3 pivotal trial
was more than 50% enrolled with nearly twice the number of patients
needed to complete enrollment identified at active trial sites.
Enrollment for the trial is anticipated to be completed in
January 2021.
- As of September, Aeglea has identified over 250 Arginase 1
Deficiency patients in addressable markets, a 25% increase relative
to the prior year. The number of currently identified patients
represents more than 50% and 30% of the estimated genetic
prevalence populations in the U.S. and EU5, respectively.
ACN00177 in Homocystinuria
- In October, Aeglea announced the U.S. Food and Drug
Administration granted Orphan Drug Designation to ACN00177 for the
treatment of Homocystinuria.
- Additionally, the European Medicines Agency Committee for
Orphan Medicinal Products issued a positive opinion recommending
Orphan Drug Designation for ACN00177 for the treatment of
Homocystinuria in the European Union.
Corporate
- In October, the Company strengthened its financial position
with proceeds from shares of common stock sold under its ATM
program, resulting in gross proceeds of $25
million, extending its cash runway into 2023.
Upcoming Events
Aeglea will be attending the following virtual investor
conferences in the fourth quarter:
- Piper Sandler 32nd
Annual Virtual Healthcare Conference, December 1-3
- 3rd Annual Evercore ISI HealthCONx Conference,
December 1-3
Third Quarter 2020 Financial Results
As of September 30, 2020, Aeglea
had available cash, cash equivalents, marketable securities and
restricted cash of $141.5 million. In
addition, in October 2020 the Company
raised approximately $24.6 million in
net proceeds from shares of common stock sold under its ATM
program. Based on Aeglea's current operating plan, and taking
into account the net offering proceeds, management believes it has
sufficient capital resources to fund anticipated operations into
2023.
Research and development expenses totaled $12.5 million for the third quarter of 2020 and
$17.8 million for the third quarter
of 2019. The decrease was primarily associated with completing
certain manufacturing and pre-commercial activities for Aeglea's
lead product candidate, pegzilarginase, completing a Phase 1/2
clinical trial in patients with Arginase 1 Deficiency and closing
cancer trials; offset by a ramp-up in manufacturing for ACN00177 in
Homocystinuria and higher personnel-related expenses.
General and administrative expenses totaled $5.7 million for the third quarter of 2020 and
$4.3 million for the third quarter of
2019. This increase was primarily due to ramping-up commercial
capabilities and additional facilities to support company
growth.
Net loss totaled $18.0 million and
$21.6 million for the third quarter
of 2020 and 2019, respectively, with non-cash stock compensation
expense of $1.7 million and
$1.4 million for the third quarter of
2020 and 2019, respectively.
About Pegzilarginase in Arginase 1 Deficiency
Pegzilarginase is an enhanced human arginase that enzymatically
lowers levels of the amino acid arginine. Aeglea is developing
pegzilarginase for the treatment of patients with Arginase 1
Deficiency (ARG1-D), a rare debilitating, progressive disease
presenting in childhood with persistent hyperargininemia,
spasticity, developmental delay, intellectual disability, seizures
and early mortality. Pegzilarginase is intended for use as an
enzyme therapy to reduce elevated blood arginine levels in patients
with ARG1-D. 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 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.
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. The timing of
first patient dosing in this Phase 1/2 trial will depend on
determinations by individual sites as they adjust to impacts from
COVID-19. ACN00177 has been granted Orphan Drug Designation by the
U.S. FDA and received a positive opinion on 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 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 Designations. 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
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.
Financials
|
Aeglea
BioTherapeutics, Inc.
Condensed
Consolidated Balance Sheets
(Unaudited)
|
|
(In thousands,
except share and per share amounts)
|
|
|
|
September
30,
|
|
|
December
31,
|
|
|
|
2020
|
|
|
2019
|
|
ASSETS
|
|
|
|
|
|
|
|
|
CURRENT
ASSETS
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
54,223
|
|
|
$
|
19,253
|
|
Marketable
securities
|
|
|
85,753
|
|
|
|
52,696
|
|
Prepaid expenses and
other current assets
|
|
|
4,423
|
|
|
|
2,556
|
|
Total current
assets
|
|
|
144,399
|
|
|
|
74,505
|
|
Restricted
cash
|
|
|
1,500
|
|
|
|
1,500
|
|
Property and
equipment, net
|
|
|
5,216
|
|
|
|
2,385
|
|
Operating lease
right-of-use assets
|
|
|
4,394
|
|
|
|
4,726
|
|
Other non-current
assets
|
|
|
122
|
|
|
|
67
|
|
TOTAL
ASSETS
|
|
$
|
155,631
|
|
|
$
|
83,183
|
|
|
|
|
|
|
|
|
|
|
LIABILITIES AND
STOCKHOLDERS' EQUITY
|
|
|
|
|
|
|
|
|
CURRENT
LIABILITIES
|
|
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
2,295
|
|
|
$
|
3,154
|
|
Operating lease
liabilities
|
|
|
341
|
|
|
|
351
|
|
Accrued and other
current liabilities
|
|
|
11,713
|
|
|
|
14,854
|
|
Total current
liabilities
|
|
|
14,349
|
|
|
|
18,359
|
|
Non-current operating
lease liabilities
|
|
|
4,947
|
|
|
|
4,712
|
|
Other non-current
liabilities
|
|
|
61
|
|
|
|
31
|
|
TOTAL
LIABILITIES
|
|
|
19,357
|
|
|
|
23,102
|
|
|
|
|
|
|
|
|
|
|
STOCKHOLDERS' EQUITY
|
|
|
|
|
|
|
|
|
Preferred stock,
$0.0001 par value; 10,000,000 shares
authorized as of September 30,
2020 and December 31, 2019; no shares issued
and outstanding as of September
30, 2020 and December 31, 2019
|
|
|
—
|
|
|
|
—
|
|
Common stock, $0.0001
par value; 500,000,000 shares authorized as of September 30, 2020 and December 31, 2019;
44,692,030 shares and 29,084,437 shares issued and outstanding as of
September 30, 2020 and
December 31, 2019, respectively
|
|
|
5
|
|
|
|
3
|
|
Additional paid-in
capital
|
|
|
389,543
|
|
|
|
255,142
|
|
Accumulated other
comprehensive income
|
|
|
20
|
|
|
|
51
|
|
Accumulated
deficit
|
|
|
(253,294)
|
|
|
|
(195,115)
|
|
TOTAL
STOCKHOLDERS' EQUITY
|
|
|
136,274
|
|
|
|
60,081
|
|
TOTAL LIABILITIES AND
STOCKHOLDERS' EQUITY
|
|
$
|
155,631
|
|
|
$
|
83,183
|
|
Aeglea
BioTherapeutics, Inc.
Condensed
Consolidated Statements of Operations
(Unaudited)
|
|
(In
thousands, except share and per share amounts)
|
|
|
|
Three Months
Ended
September
30,
|
|
|
Nine Months
Ended
September
30,
|
|
|
|
2020
|
|
|
2019
|
|
|
2020
|
|
|
2019
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
|
12,451
|
|
|
|
17,839
|
|
|
|
43,882
|
|
|
|
47,034
|
|
General and
administrative
|
|
|
5,672
|
|
|
|
4,307
|
|
|
|
14,823
|
|
|
|
11,391
|
|
Total operating
expenses
|
|
|
18,123
|
|
|
|
22,146
|
|
|
|
58,705
|
|
|
|
58,425
|
|
Loss from
operations
|
|
|
(18,123)
|
|
|
|
(22,146)
|
|
|
|
(58,705)
|
|
|
|
(58,425)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest
income
|
|
|
88
|
|
|
|
585
|
|
|
|
549
|
|
|
|
1,711
|
|
Other income
(expense), net
|
|
|
2
|
|
|
|
(12)
|
|
|
|
(23)
|
|
|
|
(45)
|
|
Total other
income
|
|
|
90
|
|
|
|
573
|
|
|
|
526
|
|
|
|
1,666
|
|
Net loss
|
|
$
|
(18,033)
|
|
|
$
|
(21,573)
|
|
|
$
|
(58,179)
|
|
|
$
|
(56,759)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.29)
|
|
|
$
|
(0.66)
|
|
|
$
|
(1.18)
|
|
|
$
|
(1.80)
|
|
Weighted-average
common shares outstanding, basic and
diluted
|
|
|
62,240,412
|
|
|
|
32,894,205
|
|
|
|
49,473,350
|
|
|
|
31,596,321
|
|
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SOURCE Aeglea BioTherapeutics, Inc.