SAN FRANCISCO, Aug. 6, 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 second quarter ended
June 30, 2019 and provided an update
on the company's recent achievements and anticipated upcoming
milestones.
"It is a very busy time at Audentes, and we are excited by the
significant progress we have made across our portfolio," stated
Matthew R. Patterson, Chairman and
Chief Executive Officer. "Following collaborative
interactions with FDA and EMA, we have initiated enrollment of
eight additional XLMTM patients into an ASPIRO pivotal expansion
cohort, which is designed to confirm the safety and efficacy
profile of AT132 at a dose of 3x1014 vg/kg. We are
optimistic that these data will support the submission of a BLA for
AT132 in mid-2020."
Mr. Patterson continued, "We remain on track for a third quarter
IND submission of AT845 for the treatment of Pompe disease.
And importantly, we've made substantial progress advancing our DMD
program. We expect patient dosing with NCH's AT702 construct
to commence as planned in the fourth quarter of 2019 and to submit
an IND in the first quarter of 2020 for the Audentes AT702
construct, which is designed to serve as the platform for our rapid
expansion into additional DMD genotypes next year. Together with
our work in myotonic dystrophy, we believe this rich development
pipeline positions us well for continued growth and industry
leadership in the field of AAV-based genetic medicines for
neuromuscular disease."
Recent Achievements & Upcoming Key Events
AT132 for XLMTM:
- Positive data update from ASPIRO, the Phase 1/2 clinical trial
of AT132 for the treatment of XLMTM, presented at the 22nd
Annual Meeting of the American Society of Gene and Cell Therapy
(ASGCT) in May 2019.
- 3x1014 vg/kg selected as optimal dose.
- Following collaborative interactions with the FDA and EMA,
initiated enrollment of eight additional XLMTM patients into the
ASPIRO pivotal expansion cohort, designed to confirm the
safety and efficacy profile of AT132 at a dose of 3x1014
vg/kg.
- Enrollment in the pivotal expansion cohort is expected to be
complete in the fall of 2019.
- BLA submission for AT132 planned in mid-2020; MAA submission
planned for the second half of 2020.
- Next clinical data presentation planned at the 24th
International Annual Congress of the World Muscle Society (WMS) in
Copenhagen, Denmark, October 1-5, 2019.
Additional information on the ASPIRO Pivotal Expansion
Cohort:
- The pivotal expansion cohort is enrolling eight patients,
consisting of four age-matched pairs (+/- 6 months), with one
patient from each pair randomized to receive a single dose of AT132
(3x1014 vg/kg) or serve as a delayed treatment
control. Delayed treatment control patients will be
administered AT132 following the collection of 24-week data from
the full pivotal cohort.
- Patients will be followed for safety and efficacy for
five years, with the primary analysis occurring 24 weeks
post treatment.
- Key inclusion criteria: less than 5 years of age or have been
enrolled in INCEPTUS, on invasive ventilator support for 20-24
hours per day, unable to sit without assistance for at least 30
seconds.
- The primary efficacy endpoint is defined as change from
baseline in hours of ventilatory support over time through week
24. As of the April 2019 ASPIRO
data analysis, all nine patients treated achieved sustained and
meaningful reductions in ventilatory support, with four patients
successfully completely weaned off of mechanical ventilation.
AT845 for Pompe Disease:
- Completed IND-enabling dose ranging and toxicology
studies.
- On track to submit IND in the third quarter of 2019.
AT702/AT751/AT753 for DMD:
- Plan to commence patient dosing in the fourth quarter of 2019
with AT702 produced by NCH. AT702 is designed to induce exon 2
skipping to treat DMD caused by duplications of exon 2 (Dup2) and
mutations in exons 1-5 of the dystrophin gene.
- Dose ranging and toxicology studies underway to support a first
quarter 2020 IND submission for the Audentes AT702 construct and
transition the balance of the AT702 DMD program into a Phase 1/2
clinical study utilizing this product candidate.
- The construct backbone of the Audentes AT702 product is
designed to serve as a vectorized exon skipping platform for rapid
expansion into additional DMD genotypes.
- Preclinical work is underway to advance AT751 and AT753 to
treat DMD patients with genotypes amenable to exons 51 and 53
skipping.
- In combination, AT702, AT751 and AT753 have the potential to
address more than 25% of DMD patients; plan to leverage our
vectorized exon skipping platform to develop additional product
candidates with the potential to address up to 80% of DMD patients
over time.
AT466 for DM1:
- Preclinical vector screening studies underway.
- Plan to submit IND in 2020.
Manufacturing:
- Advanced chemistry, manufacturing, and controls (CMC) BLA and
MAA-readiness efforts for AT132.
- State-of-the-art, internal, large-scale cGMP manufacturing
facility provides sufficient capacity for AT132 global
commercialization as well as the near-term clinical development of
all pipeline programs.
- Completed construction and commissioning of state-of-the-art,
internal plasmid manufacturing facility to support production of
nonclinical and cGMP-grade plasmids for all of our development
programs, including the potential commercialization of AT132.
Corporate:
- Appointed Edward R. Conner, M.D.
as Senior Vice President and Chief Medical Officer. Ed is
responsible for leading the company's global clinical development
strategy and oversees clinical development, clinical operations,
regulatory affairs, medical affairs and patient advocacy.
- Promoted Fulvio Mavilio, Ph.D.
to Senior Vice President of Translational Science. Fulvio is
responsible for advancing the company's pipeline from discovery
through to IND-enabling preclinical development and oversees
discovery biology, pharmacology/toxicology, bioinformatics and
bioanalytics.
Second Quarter 2019 Financial Results
- Cash Position: As of June 30,
2019, cash, cash equivalents and marketable securities were
$378.6 million.
- Research and Development Expenses: Research and
development expense was $37.3 million
for the second quarter of 2019 compared to $26.3 million for the same period in 2018, an
increase of $11.0 million. The
increase was primarily attributable to higher direct program
expenses for AT132 and AT845, along with additional R&D
headcount to advance clinical and pre-clinical programs. Included
in R&D expense for the three months ended June 30, 2019 was $3.2
million of non-cash stock-based compensation expense,
compared to $2.3 million in the same
period in 2018. For the six months ended June 30, 2019, research and development expense
was $77.1 million compared to
$46.2 million for the same period in
2018.
- General and Administrative Expenses: General and
administrative expense was $9.8
million for the second quarter of 2019 compared to
$6.3 million for the same period in
2018. The increase was primarily attributable to headcount
increases and infrastructure investment to support growth. Included
in G&A expense for the three months ended June 30, 2019 was $2.9
million of non-cash stock-based compensation expense,
compared to $1.8 million in the same
period in 2018. For the six months ended June 30, 2019, general and administrative expense
was $21.8 million compared to
$12.8 million for the same period in
2018.
- Net Loss: Net loss was $44.8
million for the second quarter of 2019 compared to
$31.4 million for the same period in
2018. Basic and diluted net loss per share for the three months
ended June 30, 2019, was $1.01 compared with $0.85 for the same period in 2018. For the six
months ended June 30, 2019, net loss
was $94.2 million compared to
$56.9 million for the same period in
2018. Basic and diluted net loss per share for the six months ended
June 30, 2019, was $2.14 compared with $1.59 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 second 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# 4244738 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 timing and nature of availability and release of
the company's clinical data results, the timing of regulatory
filings for AT132, AT845, AT702, and AT466, the timing of patient
dosing for AT702, the timing for patient enrollment in the ASPIRO
pivotal expansion and the potential penetration into the DMD and
DM1 markets. 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
June 30,
|
|
Six months ended June
30,
|
|
2019
|
|
2018
|
|
2019
|
|
2018
|
|
Unaudited
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
Research and
development
|
$
37,299
|
|
$
26,348
|
|
$
77,136
|
|
$
46,239
|
General and
administrative
|
9,778
|
|
6,281
|
|
21,771
|
|
12,800
|
Total operating
expenses
|
47,077
|
|
32,629
|
|
98,907
|
|
59,039
|
Loss from
operations
|
(47,077)
|
|
(32,629)
|
|
(98,907)
|
|
(59,039)
|
Interest income,
net
|
2,353
|
|
1,294
|
|
4,825
|
|
2,153
|
Other expense,
net
|
(40)
|
|
(32)
|
|
(73)
|
|
(52)
|
Net loss
|
$
(44,764)
|
|
$
(31,367)
|
|
$
(94,155)
|
|
$
(56,938)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
(1.01)
|
|
$
(0.85)
|
|
$
(2.14)
|
|
$
(1.59)
|
Shares used in
computing net loss per share, basic and diluted
|
44,426,279
|
|
36,935,940
|
|
44,028,010
|
|
35,765,506
|
Selected Balance
Sheet Data
|
|
|
|
June 30,
2019
|
|
December 31,
2018
|
|
|
Unaudited
|
|
|
|
|
|
Cash, cash
equivalents, marketable securities and restricted cash
|
|
$
382,386
|
|
$
418,055
|
Total
assets
|
|
$
478,114
|
|
$
472,555
|
Total
liabilities
|
|
$
58,412
|
|
$
29,801
|
Total stockholders'
equity
|
|
$
419,702
|
|
$
442,754
|
Audentes Contacts:
Investor Contact:
Andrew Chang
415.818.1033
achang@audentestx.com
Media Contacts:
Sarah Spencer
415.957.2020
sspencer@audentestx.com
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SOURCE Audentes Therapeutics, Inc.