SAN FRANCISCO, Aug. 10, 2017 /PRNewswire/ -- Audentes
Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company
focused on developing and commercializing gene therapy products for
patients living with serious, life-threatening rare diseases, today
reported its financial results for the quarter ended June 30, 2017, and provided an update on the
company's recent achievements and anticipated upcoming
milestones.
"In the second quarter we continued to make excellent progress
advancing our entire multi-product pipeline towards upcoming key
milestones, highlighted by final preparations to initiate ASPIRO
and VALENS, the Phase 1/2 clinical studies of AT132 for the
treatment of XLMTM and AT342 for the treatment of Crigler-Najjar
Syndrome," stated Matthew R.
Patterson, President and Chief Executive Officer. "In
each program, INDs are active, drug product has been internally
manufactured and the Phase 1/2 run-in and clinical assessment
studies are ongoing. We plan to announce the first dosing of
patients in ASPIRO and VALENS in the third quarter of 2017 and to
report preliminary data from these studies by the end of the
year."
Mr. Patterson continued, "We are also excited about our
continued progress toward IND filings for AT307 for the treatment
of CASQ2-CPVT and AT982 for the treatment of Pompe disease.
We maintain our guidance toward an AT307 IND filing by year-end and
an AT982 IND filing in the first half of 2018."
Recent Key Achievements
- AT132 for X-Linked Myotubular Myopathy: Audentes is initiating
clinical study sites for ASPIRO, the Phase 1/2 study of AT132, and
plans to enroll the first patient in ASPIRO in the third quarter of
2017. Patients are expected to enroll in ASPIRO from
INCEPTUS, the clinical assessment and Phase 1/2 run-in study that
is currently ongoing at investigational sites in the United States and Europe.
- AT342 for Crigler-Najjar Syndrome: Audentes is initiating
clinical study sites for VALENS, the Phase 1/2 study of AT342, and
plans to enroll the first patient in VALENS in the third quarter of
2017. Patients are expected to enroll in VALENS from LUSTRO,
the clinical assessment and Phase 1/2 run-in study that is
currently ongoing at investigational sites in the United
States.
- Presented data at the American Society of Gene and Cell Therapy
20th Annual Meeting:
-
- In a non-human primate (NHP) study, intravenous administration
of AT132 produced by the company's proprietary manufacturing
process at 500L scale demonstrated an encouraging safety profile
and was potent. Specifically, a dose of 8 x
1014 vg/kg resulted in no significant treatment
related adverse events or safety findings, and produced MTM1
protein expression at levels 8x to 20x of endogenous levels in
target skeletal muscle.
- In a murine model of Crigler-Najjar Syndrome, doses of ≥
1.5x1012 vg/kg rapidly and durably reduced total
bilirubin to normal levels, establishing the dosing range for the
upcoming VALENS study.
- Follow-on financing: Strengthened balance sheet with
completion of a follow-on financing in April 2017. Audentes
issued 5,955,151 million shares of common stock at an offering
price of $14.50 per share, resulting
in net proceeds of $80.6 million
after the deduction of underwriting discounts, commissions and
offering expenses.
- Appointed Jennifer Jarrett to
the Board of Directors: Ms. Jarrett serves as the Chief Financial
and Business Officer of Arcus Biosciences, Inc., and
previously served as Chief Financial Officer of Medivation,
Inc. until the company's purchase by Pfizer, Inc. in
2016. Prior to Medivation, Ms. Jarrett served as Managing
Director at Citigroup, where she was responsible for building
and managing Citigroup's West Coast life sciences
investment banking practice.
Planned Upcoming Events
- AT132 for X-Linked Myotubular Myopathy:
-
- Commence enrollment in ASPIRO, a Phase 1/2 multicenter,
multinational, open-label study in 12 XLMTM patients less than five
years of age, in the third quarter of 2017
- Report preliminary data from INCEPTUS, a clinical assessment
and Phase 1/2 run-in study, in September
2017
- Report preliminary clinical data from ASPIRO by the end of
2017
- AT342 for Crigler-Najjar Syndrome:
-
- Commence enrollment in VALENS, the Phase 1/2 multicenter,
multinational, open-label study in approximately 12 patients
greater than one year of age, in the third quarter of 2017
- Report preliminary data from LUSTRO, the Phase 1/2 run-in study
of AT342, in October 2017
- Report preliminary clinical data from VALENS by the end of
2017
- AT307 for CASQ2-CPVT:
-
- Complete IND-enabling preclinical studies
- File IND by the end of 2017
- AT982 for Pompe Disease:
-
- Conduct additional preclinical studies in support of a planned
IND filing in the first half of 2018 to evaluate systemic
administration of AT982 in Pompe disease
Second Quarter 2017 Financial Results
- Cash Position: As of June 30, 2017, Audentes had
cash, cash equivalents and short-term investments of $145.0 million.
- R&D Expenses: Research and development
expenses were $18.8 million for the second quarter of
2017 compared to $11.7
million for the same period in 2016, an increase of $7.1
million. The increase was primarily due to increased R&D
expenses for our AT132 and AT307 programs, increases in our R&D
headcount and higher facility costs as we made additional
investments in our manufacturing and research facilities. For the
six months ended June 30, 2017,
research and development expenses were $33.4
million compared to $19.6
million for the same period in 2016.
- General and Administrative: General and administrative
expenses were $4.1 million for the second quarter of 2017
compared to $2.5 million for the same period in 2016, an
increase of $1.6 million. The increase was
primarily due to increased G&A headcount and increases in other
G&A expenses related to regulatory compliance and operations as
a public company. For the six months ended June 30, 2017, general and administrative
expenses were $7.7 million compared
to $5.1 million for the same
period in 2016.
- Net Loss: Net loss was $22.7 million for the
second quarter of 2017 compared to a net loss
of $14.2 million for the same period in 2016. For
the six months ended June 30, 2017,
our net loss was $40.9 million as
compared to $24.6 million for the
same period in 2016.
About Audentes Therapeutics, Inc.
Audentes
Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on
developing and commercializing gene therapy products for patients
living with serious, life-threatening rare diseases. We have
four product candidates in development, AT132 for the treatment of
X-Linked Myotubular Myopathy (XLMTM), AT342 for the treatment of
Crigler-Najjar Syndrome, AT982 for the treatment of Pompe disease,
and AT307 for the treatment of the CASQ2 subtype of
Catecholaminergic Polymorphic Ventricular Tachycardia
(CASQ2-CPVT). We are a focused, experienced and passionate
team committed to forging strong, global relationships with the
patient, research and medical communities.
For more information regarding Audentes, please visit
www.audentestx.com.
Forward Looking Statements
This press release
contains forward-looking statements within the meaning of the "safe
harbor" provisions of the Private Securities Litigation Reform Act
of 1995, including, but not limited to: the timing of key program
milestones, including the timing of patient enrollment and
reporting of the preliminary Phase 1/2 data from ASPIRO and VALENS,
the timing of reporting preliminary results from the ongoing
INCEPTUS and LUSTRO studies, and the timing of the conduct of IND
enabling pre-clinical studies and the filing of INDs for the CPVT
and Pompe programs. All statements other than statements of
historical fact are statements that could be deemed forward-looking
statements. Although the company believes that the
expectations reflected in such forward-looking statements are
reasonable, the company cannot guarantee future events, results,
actions, levels of activity, performance or achievements, and the
timing and results of biotechnology development and potential
regulatory approval is inherently uncertain. Forward-looking
statements are subject to risks and uncertainties that may cause
the company's actual activities or results to differ significantly
from those expressed in any forward-looking statement, including
risks and uncertainties related to the company's ability to advance
its product candidates, obtain regulatory approval of and
ultimately commercial its product candidates, the timing and
results of preclinical and clinical trials, the company's ability
to fund development activities and achieve development goals, the
company's ability to establish and scale-up manufacturing processes
that comply with regulatory requirements, the company's ability to
protect intellectual property and other risks and
uncertainties described under the heading "Risk Factors" in
documents the company files from time to time with
the Securities and Exchange Commission. These forward-looking
statements speak only as of the date of this press release, and the
company undertakes no obligation to revise or update any
forward-looking statements to reflect events or circumstances after
the date hereof.
Selected Financial Information
Operating
Results:
|
(amounts in thousands
except share and per share data)
|
|
|
Three months ended
June 30,
|
|
Six months ended June
30,
|
|
2017
|
|
2016
|
|
2017
|
|
2016
|
|
Unaudited
|
|
Unaudited
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
Research and
development
|
$
18,776
|
|
$
11,734
|
|
$
33,363
|
|
$
19,640
|
General and
administrative
|
4,065
|
|
2,469
|
|
7,723
|
|
5,101
|
Total operating
expenses
|
22,841
|
|
14,203
|
|
41,086
|
|
24,741
|
Loss from
operations
|
(22,841)
|
|
(14,203)
|
|
(41,086)
|
|
(24,741)
|
Interest income,
net
|
115
|
|
92
|
|
262
|
|
189
|
Other expense,
net
|
(13)
|
|
(48)
|
|
(30)
|
|
(71)
|
Net loss
|
$
(22,739)
|
|
$
(14,159)
|
|
$
(40,854)
|
|
$
(24,623)
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
(0.87)
|
|
$
(6.43)
|
|
$
(1.70)
|
|
$
(11.30)
|
Shares used in
computing net loss per share, basic and diluted
|
26,212,614
|
|
2,200,406
|
|
23,996,187
|
|
2,179,735
|
|
|
|
|
|
|
|
|
Selected Balance
Sheet Information:
|
(amounts in
thousands)
|
|
|
June 30,
|
|
December
31,
|
|
2017
|
|
2016
|
|
Unaudited
|
|
|
|
|
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
$
145,008
|
|
$
104,883
|
Total
assets
|
$
188,592
|
|
$
142,057
|
Total
liabilities
|
$
26,538
|
|
$
22,686
|
Total stockholders'
equity
|
$
162,054
|
|
$
119,371
|
|
|
|
|
Audentes Contacts:
Investor Contact:
Thomas Soloway, CFO
415.818.1040
ir@audentestx.com
Media Contact:
Paul Laland
415.519.6610
media@audentestx.com
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SOURCE Audentes Therapeutics, Inc.