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 second quarter 2020 financial results,
and provided recent corporate and program highlights.
“Despite the operating challenges posed by the global pandemic,
we continued to advance our pegzilarginase program in the first
half of the year. The presentation of long-term data showing
sustained lowering of arginine levels and durable clinical response
with pegzilarginase treatment, as well as progress in our patient
identification efforts, reinforce our belief in its potential as a
life-changing therapy for those with Arginase 1 Deficiency and lay
a strong foundation for the commercial launch of pegzilarginase,”
said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive
officer of Aeglea. “Additionally, we initiated our Phase 1/2
clinical trial of ACN00177 for Homocystinuria in the second
quarter. We are continuing our patient identification activities
and look forward to dosing the first patient once the clinical
sites are able to begin screening patients.”
Recent Highlights and UpdatesPegzilarginase in
Arginase 1 Deficiency
- In May, Aeglea presented results from its 56 week analysis from
the Company’s completed Phase 1/2 clinical trial and ongoing
open-label extension study during a late-breaking oral presentation
at the 6th Congress of the European Academy of Neurology. Key
results include:
- Treatment with pegzilarginase resulted in a significant
reduction in plasma arginine from baseline with all 13 patients
achieving plasma arginine levels within the target range (<200
µM).
- 85% (11 of 13) of patients were clinical responders based on
mobility improvements evaluated using three assessments: 6MWT (6
Minute Walk Test), GMFM (Gross Motor Function) Part D (standing)
and Part E (walking, running, and jumping).
- Pegzilarginase was shown to have a favorable safety profile
with more than 750 doses administered.
- To date, Aeglea has identified more than 240 Arginase 1
Deficiency patients. The number of identified patients represents
more than 50% and 30% of the estimated genetic prevalence patient
population in the U.S. and key European markets (France, Germany,
Spain, Italy and the United Kingdom), respectively.
ACN00177 in Homocystinuria
- Aeglea initiated its Phase 1/2 clinical trial for ACN00177, a
novel engineered human enzyme therapy designed to treat
Homocystinuria, a serious metabolic disorder characterized by
elevated plasma homocysteine which leads to a wide range of
life-altering complications and reduced life expectancy.
Corporate
- Eric Bradford, M.D., M.Sc., M.B.A. has been promoted to Chief
Development Officer. Dr. Bradford will oversee the clinical
programs for pegzilarginase and ACN00177 as well as shape the
clinical development strategy for future programs from the
Company’s platform of novel human enzymes.
- Chief Medical Officer Ravi M. Rao, M.B BChir, Ph.D., will
depart the company to pursue other opportunities. Dr. Rao will
continue to support Aeglea in a medical advisor role through a
transitional period.
“Ravi has been a valued and impactful member of the Aeglea team.
While we are disappointed by his planned departure, we wish him the
best as he returns to his roots in immunology research and
development,” said Dr. Quinn. “I look forward to working more
closely with Eric as we continue to strengthen our capabilities and
advance pegzilarginase towards potential approval and launch.”
Upcoming EventsAeglea will be attending the
following virtual investor conferences in the coming quarter.
- Wells Fargo Securities Healthcare Conference, September
9-10
- H.C. Wainwright Healthcare Conference, September
13-15
- Cantor Fitzgerald Global Healthcare Conference, September
15-17
Further, Aeglea’s leadership looks forward to participating in
dialogue about the Company’s enzyme therapeutics platform during
the following industry events, with additional details to be
announced.
- World Orphan Drug Congress USA 2020, August
24-26
- Child Neurology Society Annual Meeting-International Child
Neurology Congress 2020, October 19-23
Second Quarter 2020 Financial Results
As of June 30, 2020, Aeglea had available cash, cash
equivalents, marketable securities and restricted cash of $159.2
million. Based on Aeglea’s current operating plans, management
believes it has sufficient capital resources to fund anticipated
operations through 2022.
Research and development expenses totaled $16.9 million for the
second quarter of 2020 and $14.8 million for the second quarter of
2019. The increase was primarily associated with investing in
manufacturing and pre-commercial activities for Aeglea’s lead
product candidate, pegzilarginase; ramp-up in manufacturing
activities for ACN00177 in Homocystinuria; and personnel-related
expenses offset by decreasing clinical development expenses as a
result of completing a Phase 1/2 clinical trial in patients with
Arginase 1 Deficiency and closing out cancer trials.
General and administrative expenses totaled $4.7 million for the
second quarter of 2020 and $3.8 million for the second quarter of
2019. This increase was primarily due to additional employee
headcount, ramping up commercial capabilities, and additional
facilities to support company growth.
Net loss totaled $21.4 million and $18.0 million for the second
quarter of 2020 and 2019, respectively, with non-cash stock
compensation expense of $1.6 million and $1.2 million for the
second 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.
About Aeglea BioTherapeuticsAeglea
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.
Media Contact:Kelly Boothe, Ph.D. Director,
Corporate Communications Aeglea BioTherapeutics 512.399.5458
media@aegleabio.com
Investor Contact:Joey PerroneSenior Director,
Finance & Investor RelationsAeglea
BioTherapeuticsinvestors@aegleabio.com
Financials
Aeglea BioTherapeutics,
Inc.Condensed Consolidated Balance
Sheets(Unaudited)
(In thousands, except share and per share
amounts)
|
|
June 30, |
|
|
December 31, |
|
|
|
2020 |
|
|
2019 |
|
ASSETS |
|
|
|
|
|
|
|
|
CURRENT ASSETS |
|
|
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
65,713 |
|
|
$ |
19,253 |
|
Marketable securities |
|
|
92,017 |
|
|
|
52,696 |
|
Prepaid expenses and other current assets |
|
|
4,136 |
|
|
|
2,556 |
|
Total current assets |
|
|
161,866 |
|
|
|
74,505 |
|
Restricted cash |
|
|
1,500 |
|
|
|
1,500 |
|
Property and equipment,
net |
|
|
4,896 |
|
|
|
2,385 |
|
Operating lease right-of-use
assets |
|
|
4,557 |
|
|
|
4,726 |
|
Other non-current assets |
|
|
92 |
|
|
|
67 |
|
TOTAL ASSETS |
|
$ |
172,911 |
|
|
$ |
83,183 |
|
|
|
|
|
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS’
EQUITY |
|
|
|
|
|
|
|
|
CURRENT LIABILITIES |
|
|
|
|
|
|
|
|
Accounts payable |
|
$ |
4,151 |
|
|
$ |
3,154 |
|
Operating lease liabilities |
|
|
228 |
|
|
|
351 |
|
Accrued and other current liabilities |
|
|
11,511 |
|
|
|
14,854 |
|
Total current liabilities |
|
|
15,890 |
|
|
|
18,359 |
|
Non-current operating lease
liabilities |
|
|
4,695 |
|
|
|
4,712 |
|
Other non-current
liabilities |
|
|
68 |
|
|
|
31 |
|
TOTAL LIABILITIES |
|
|
20,653 |
|
|
|
23,102 |
|
|
|
|
|
|
|
|
|
|
STOCKHOLDERS’ EQUITY |
|
|
|
|
|
|
|
|
Preferred stock, $0.0001 par value; 10,000,000 shares authorizedas
of June 30, 2020 and December 31, 2019; no shares issued
andoutstanding as of June 30, 2020 and December 31, 2019 |
|
|
— |
|
|
|
— |
|
Common stock, $0.0001 par value; 500,000,000 shares
authorized as of June 30, 2020 and December 31, 2019;
44,599,847 shares and 29,084,437 shares issued and outstanding
as of June 30, 2020 and December 31, 2019, respectively |
|
|
5 |
|
|
|
3 |
|
Additional paid-in capital |
|
|
387,475 |
|
|
|
255,142 |
|
Accumulated other comprehensive income |
|
|
39 |
|
|
|
51 |
|
Accumulated deficit |
|
|
(235,261 |
) |
|
|
(195,115 |
) |
TOTAL STOCKHOLDERS’
EQUITY |
|
|
152,258 |
|
|
|
60,081 |
|
TOTAL LIABILITIES AND
STOCKHOLDERS’ EQUITY |
|
$ |
172,911 |
|
|
$ |
83,183 |
|
Aeglea BioTherapeutics,
Inc.Condensed Consolidated Statements of
Operations(Unaudited)
(In thousands, except share and per
share amounts)
|
|
Three Months EndedJune 30, |
|
|
Six Months EndedJune 30, |
|
|
|
2020 |
|
|
2019 |
|
|
2020 |
|
|
2019 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
16,869 |
|
|
|
14,806 |
|
|
|
31,431 |
|
|
|
29,195 |
|
General and administrative |
|
|
4,691 |
|
|
|
3,816 |
|
|
|
9,151 |
|
|
|
7,084 |
|
Total operating expenses |
|
|
21,560 |
|
|
|
18,622 |
|
|
|
40,582 |
|
|
|
36,279 |
|
Loss from operations |
|
|
(21,560 |
) |
|
|
(18,622 |
) |
|
|
(40,582 |
) |
|
|
(36,279 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Other income (expense): |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income |
|
|
161 |
|
|
|
619 |
|
|
|
461 |
|
|
|
1,126 |
|
Other expense, net |
|
|
(19 |
) |
|
|
(16 |
) |
|
|
(25 |
) |
|
|
(33 |
) |
Total other income |
|
|
142 |
|
|
|
603 |
|
|
|
436 |
|
|
|
1,093 |
|
Net loss |
|
$ |
(21,418 |
) |
|
$ |
(18,019 |
) |
|
$ |
(40,146 |
) |
|
$ |
(35,186 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share, basic and
diluted |
|
$ |
(0.40 |
) |
|
$ |
(0.55 |
) |
|
$ |
(0.93 |
) |
|
$ |
(1.14 |
) |
Weighted-average common shares
outstanding, basic and diluted |
|
|
52,941,603 |
|
|
|
32,840,357 |
|
|
|
43,019,670 |
|
|
|
30,936,623 |
|
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