SAN FRANCISCO, May 7, 2019 /PRNewswire/ -- Audentes
Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic
medicines company focused on developing and commercializing
innovative products for serious rare neuromuscular diseases, today
reported its financial results for the first quarter ended
March 31, 2019 and provided an update
on the company's recent achievements and anticipated upcoming
milestones.
"We are excited by the continued strong momentum across our
business," stated Matthew R.
Patterson, Chairman and Chief Executive Officer. "We
recently presented new positive data from ASPIRO, the Phase 1/2
study of AT132 for the treatment of XLMTM, at the 22nd
Annual Meeting of the American Society of Gene and Cell
Therapy. These data further support the compelling profile of
this important product candidate and help further our progress
toward the goal of making AT132 globally available to patients
living with XLMTM as rapidly as possible."
Mr. Patterson continued, "In our Pompe disease program, this
quarter we successfully completed an initial NHP safety study of
our product candidate AT845 and the encouraging results give us
added confidence as we complete GLP toxicology and dose-ranging
studies to support a third quarter IND submission. Finally,
we are thrilled to have recently announced the expansion of our
technology platform and development pipeline with the addition of
programs utilizing vectorized antisense to address Duchenne
muscular dystrophy and myotonic dystrophy, two devastating
neuromuscular diseases."
Recent Achievements & Upcoming Key Events
AT132 for X-Linked Myotubular Myopathy (XLMTM):
- Presented new positive data from ASPIRO at the 22nd Annual
Meeting of the American Society of Gene and Cell Therapy
(ASGCT).
- The ASGCT data set included safety and efficacy assessments out
to 48 weeks of follow-up for six treated patients in dose Cohort 1
(1x1014 vg/kg) and 24 weeks for three treated patients
in dose Cohort 2 (3x1014 vg/kg).
- Patients receiving AT132 achieved reductions in ventilator
dependence not previously observed in chronically ventilated
patients with neuromuscular disorders, including four patients
achieving ventilator independence and all other treated patients
demonstrating sustained and clinically meaningful reductions in
ventilator use.
- The data also demonstrated sustained improvements in
neuromuscular function in both dose cohorts with corresponding
achievement of clinically meaningful motor milestones.
Additionally, muscle biopsy data continued to show robust tissue
transduction, protein expression, and histological improvements in
24 and 48-week muscle biopsies in all patients, with evidence of
more rapid pathological improvement by week 24 in the Cohort 2
biopsy samples.
- AT132 has been generally well-tolerated and has shown a
manageable safety profile across both dose cohorts with no
clinically meaningful safety differences between doses to
date.
- Enrollment of an additional three to five patients in Cohort 2
(3x1014 vg/kg) of ASPIRO is ongoing.
- On track to select optimal dose of AT132 in the second quarter
of 2019. Plan to provide an updated data package to FDA and on
track to provide update on the license application submission plans
for AT132 in the third quarter of 2019.
- Next clinical data presentation planned at the 24th
International Annual Congress of the World Muscle Society (WMS) in
Copenhagen, Denmark, October 1-5, 2019.
AT845 for Pompe Disease:
- Successfully completed an initial non-GLP NHP study, which
showed a clean safety profile.
- Previously planned GLP toxicology and dose-ranging studies in
progress.
- On track to file IND in the third quarter of 2019.
AT702/AT751/AT753 for Duchenne Muscular Dystrophy (DMD):
- Announced exclusive license agreement with the Research
Institute at Nationwide Children's Hospital to develop AT702, an
AAV-antisense candidate designed to induce exon 2 skipping for DMD
caused by duplications of exon 2 and mutations in exons 1-5 of the
dystrophin gene. Conducting additional preclinical work of AT702
and plan to commence a Phase 1/2 study at Nationwide Children's in
the fourth quarter of 2019.
- Separate from the Nationwide Children's collaboration,
conducting preclinical work to advance AT751 and AT753, additional
vectorized exon skipping candidates designed to treat DMD patients
with genotypes amenable to exon 51 and exon 53 skipping. Both AT751
and AT753 utilize the same vector construct backbone as AT702,
enabling a potentially accelerated path into clinical
development.
- Initial programs target more than 25% of patients with DMD.
Plan to leverage vectorized exon skipping platform to develop
further product candidates to address up to 80% of DMD patients
over time.
AT466 for Myotonic Dystrophy (DM1):
- Collaboration announced with Nationwide Children's to evaluate
vectorized RNA knockdown and vectorized exon skipping for DM1.
- Preclinical studies are underway, and IND planned for
2020.
AT342 for Crigler-Najjar Syndrome and AT307 for CASQ2-CPVT:
- Completed a strategic review of our product candidates and plan
to focus on those programs that have the potential to create the
greatest long-term value for patients and shareholders.
- Plan to explore outlicensing opportunities to continue to
advance these important and promising gene therapy programs.
Manufacturing:
- Announced new internal cGMP plasmid manufacturing facility,
further establishing the company's leadership in AAV manufacturing
and enabling the rapid advancement of neuromuscular programs from
preclinical development to commercialization.
- Made continued progress on chemistry, manufacturing, and
controls (CMC) BLA and MAA-readiness efforts for AT132 based on FDA
and EMA feedback on the company's preliminary filing strategy.
Corporate:
- Expanded leadership team with the additions of Mark Meltz, Senior Vice President, General
Counsel, to lead the legal, compliance, and corporate governance
functions and Sarah Spencer, Vice
President, Corporate Communications, to lead internal and external
communications to build global awareness of the company, its
mission, culture, and innovative product portfolio.
First Quarter 2019 Financial Results
- Cash Position: At March 31,
2019, Audentes had cash, cash equivalents and marketable
securities of $375.0 million, which
is expected to fund operations into 2021.
- Research and Development Expenses: Research and
development expenses were $39.8
million for the first quarter of 2019 compared to
$19.9 million for the same period in
2018, an increase of $19.9 million.
The increase in research and development expense was primarily
attributable to increased R&D headcount and related facility
costs, a one-time licensing fee of $7.0
million related to the AT702 program, increased internal
manufacturing costs, increased clinical trial expenses for our
AT132 program, increased pre-clinical and clinical trial planning
costs for our AT845 program, and an increase in stock compensation
expense.
- General and Administrative Expenses: General and
administrative expenses were $12.0
million for the first quarter of 2019 compared to
$6.5 million for the same period in
2018, an increase of $5.5 million.
The increase in general and administrative expenses was primarily
attributable to increased headcount and related facility costs,
increased professional service and consulting fees, higher costs
related to public company regulatory compliance and increase in
stock compensation expense.
- Net Loss: Net loss was $49.4
million for the first quarter of 2019 compared to
$25.6 million for the same period in
2018.
Conference Call
At 4:30 p.m. Eastern
Time today, Audentes management will host a conference call
and a simultaneous webcast to discuss its first quarter 2019
financial results and provide a corporate update. To access a
live webcast of the conference call, please visit the Events &
Presentations page within the Investors + Media section of the
Audentes website at www.audentestx.com. Alternatively, please
call (833) 659-8620 (U.S.) or (409) 767-9247 (international) and
dial the conference ID# 6839198 to access the call.
A replay of the webcast will be available on the Audentes
website for approximately 30 days.
About Audentes Therapeutics, Inc.
Audentes
Therapeutics (Nasdaq: BOLD) is a leading AAV-based genetic
medicines company focused on developing and commercializing
innovative products for serious rare neuromuscular diseases.
We are leveraging our AAV gene therapy technology platform and
proprietary manufacturing expertise to develop programs across
three modalities: gene replacement, vectorized exon skipping, and
vectorized RNA knockdown. Our product candidates are showing
promising therapeutic profiles in clinical and preclinical studies
across a range of neuromuscular diseases. Audentes is a focused,
experienced and passionate team driven by the goal of improving the
lives of patients.
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 expected benefits of the
company's product candidates and technology platform, the timing
and nature of the company's research, development and manufacturing
activities, the nature of the results of the company's data, the
timing for optimal dose selection and providing an update regarding
license application submission plans for AT132, the timing of
regulatory filings for AT132, AT845 and AT466, the expected market
size of DMD and DM1 and the company's potential penetration into
those markets and the company's intention to outlicense
Crigler-Najjar and CASQ2-CPVT. 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 commercialize its product candidates, the timing and
results of preclinical and clinical trials, the company's ability
to fund development activities and achieve development goals,
establish and scale-up manufacturing processes that comply with
regulatory requirements, and 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 Amounts in thousands except share and per
share data
Operating
Results
|
|
Three months ended
March 31,
|
|
2019
|
|
2018
|
|
Unaudited
|
|
|
|
|
Operating
expenses:
|
|
|
|
Research and
development
|
$
39,837
|
|
$
19,891
|
General and
administrative
|
11,993
|
|
6,519
|
Total operating
expenses
|
51,830
|
|
26,410
|
Loss from
operations
|
(51,830)
|
|
(26,410)
|
Interest income,
net
|
2,472
|
|
859
|
Other expense,
net
|
(33)
|
|
(20)
|
Net loss
|
$
(49,391)
|
|
$
(25,571)
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
(1.13)
|
|
$
(0.74)
|
Shares used in
computing net loss per share, basic and diluted
|
43,625,316
|
|
34,582,071
|
Selected Balance
Sheet Data
|
|
|
March 31,
2019
|
|
|
December 31,
2018
|
|
Unaudited
|
|
|
Audited
|
|
|
|
|
|
Cash, cash
equivalents, marketable securities
|
|
|
|
and restricted
cash
|
$
378,769
|
|
|
$
418,055
|
Total
assets
|
$
455,176
|
|
|
$
472,555
|
Total
liabilities
|
$
53,181
|
|
|
$
29,801
|
Total stockholders'
equity
|
$
401,995
|
|
|
$
442,754
|
Audentes Contacts:
Investor Contact:
Andrew Chang
415.818.1033
achang@audentestx.com
Media Contacts:
Sarah Spencer
415.957.2020
sspencer@audentestx.com
Katie Hogan
415.951.3398
khogan@audentestx.com
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SOURCE Audentes Therapeutics, Inc.