SAN FRANCISCO, Nov. 6, 2018 /PRNewswire/ -- Audentes
Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company
focused on developing and commercializing innovative gene therapy
products for patients living with serious, life-threatening rare
diseases, today reported its financial results for the third
quarter ended September 30, 2018 and
provided an update on the company's recent achievements and
upcoming milestones.
"In the third quarter, we continued to see exciting progress in
the clinical development of AT132," stated Matthew R. Patterson, Chairman and Chief
Executive Officer. "We recently presented new ASPIRO data at
the 23rd International Annual Congress of the World Muscle Society
demonstrating significant and durable improvements in neuromuscular
and respiratory function in all treated patients. We are
particularly pleased that three of our Cohort 1 patients have
achieved ventilator independence, an unprecedented result for
children with congenital myopathies that have been ventilated since
birth. In addition, we have recently completed enrollment of
the Cohort 2 dose of 3x1014 vg/kg, including three
treated patients and one untreated control patient. We
look forward to engaging with the FDA and EMA in the coming weeks
to discuss development plans and the potential registration pathway
for AT132, and to advance our goal of making this important
treatment available to patients living with XLMTM as rapidly as
possible."
Mr. Patterson continued, "Additionally, our recent follow-on
financing positions Audentes to deliver on many important
milestones, including the initiation of our commercial planning
activities for AT132, progression of our Pompe program toward IND,
and expansion of our pipeline of innovative AAV-based product
candidates targeting neuromuscular diseases with significant unmet
medical need."
Recent Achievements & Upcoming Key Events
- AT132 for XLMTM:
-
- Presented additional positive interim data from ASPIRO during
an oral presentation at the 23rd International Annual Congress of
the World Muscle Society (WMS)
- The ASPIRO WMS data set included safety and efficacy results
ranging from 4 to 48 weeks for eight patients enrolled in ASPIRO,
including the seven patients enrolled in Cohort 1
(1x1014 vg/kg; six treated and one untreated control)
and the sentinel patient in Cohort 2 (3x1014 vg/kg), as
well as week 24 muscle biopsy data for the first four treated
patients. All treated patients showed meaningful improvements
in neuromuscular and respiratory function. Three patients
achieved ventilator independence, and all other patients showed
significant reductions in ventilator dependence.
Additionally, the growing body of week 24 muscle biopsy data
continued to show robust tissue transduction, protein expression,
and histological improvement with AT132 treatment at the initial
1x1014 vg/kg dose.
- Completed enrollment of Cohort 2 of ASPIRO, including three
patients treated at a dose of 3x1014 vg/kg and one
enrolled as the Cohort 2 untreated control
- Awarded Regenerative Medicine Advanced Therapy (RMAT)
designation by the FDA
- Plan to engage with FDA and EMA in Q4 2018 to discuss
development plans and the potential registration pathway for AT132
in both the United States and
Europe
- Next program update planned in Q1 2019
- AT982 for Pompe Disease:
-
- Completed analysis of nonclinical studies and determined
mechanism of the dose-dependent safety signal observed in the NHP
toxicology study
- Additional IND-enabling studies underway in the Pompe mouse
model
- Plan to file IND in 2019
- AT342 for Crigler-Najjar Syndrome:
-
- Enrollment in VALENS Phase 1/2 study ongoing
- Next program update planned in Q1 2019
- Pipeline expansion:
-
- Continuing to advance our research initiative focused on the
design and development of a novel AAV-based therapeutic targeting a
large neuromuscular disease with significant unmet medical
need
- Manufacturing:
-
- Continuing BLA preparation and validation efforts for our
internal cGMP manufacturing facility, AT132 process and analytical
methods
- Since program inception, clinical material has been
manufactured utilizing substantially the same process, scale, and
facility intended to supply the commercial market
- Current facility, scale and process yield provide sufficient
capacity for the expected global commercial needs of XLMTM and
Crigler-Najjar markets, and ongoing clinical supply for current and
planned pipeline programs
Third Quarter 2018 Financial Results
- Cash Position: As of September
30, 2018, we had cash, cash equivalents, marketable
securities and restricted cash of $285.5
million, which includes approximately $3.6 million of restricted cash and approximately
$1.3 million of long-term
investments. In October 2018,
we further strengthened our balance sheet with the completion of a
follow-on financing, issuing 5,980,000 shares of common stock
(inclusive of 780,000 shares of common stock pursuant to the
underwriters' option to purchase additional shares) at an offering
price of $29.00 per share, resulting
in net proceeds of approximately $162.8
million after the deduction of underwriting discounts,
commissions and estimated offering expenses. Our current
cash, cash equivalents, and marketable securities are expected to
fund operations into 2021.
- Research and Development Expense: Research and
development expense was $29.9 million
for the third quarter of 2018 compared to $20.9 million for the same period in 2017, an
increase of $9.0 million. The
increase in research and development expense was primarily
attributable to an increase in development costs related to our
AT982 program, increased headcount and related facility costs,
increased internal manufacturing costs and higher stock-based
compensation expense. Research and development expense for
the third quarter includes $2.6
million of non-cash stock-based compensation expense.
For the nine months ended September 30,
2018, research and development expense was $76.2 million compared to $54.2 million for the same period in 2017.
- General and Administrative Expense: General and
administrative expense was $7.8
million for the third quarter of 2018 compared to
$4.3 million for the same period in
2017, an increase of $3.5 million.
The increase in general and administrative expense was
primarily attributable to increases in headcount and related
facility costs, professional service fees, stock-based compensation
expense and compliance costs associated with operating as a public
company. General and administrative expense for the third
quarter includes $2.0 million of
non-cash stock-based compensation expense. For the nine
months ended September 30, 2018,
general and administrative expense was $20.6
million compared to $12.1
million for the same period in 2017.
- Net Loss: Net loss was $36.3
million for the third quarter of 2018 compared to
$25.0 million for the same period in
2017. For the nine months ended September 30, 2018, net loss was $93.2 million as compared to $65.9 million for the same period in 2017.
Conference Call
At 4:30 pm Eastern
Time today after the market closes, Audentes management will
host a conference call and a simultaneous webcast to discuss its
third quarter 2018 financial results and provide a corporate
update. To access a live webcast of the conference call,
please visit the Events & Presentations page within the
Investor + 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# 1473209 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 biotechnology company focused on
developing and commercializing innovative gene therapy products for
patients living with serious, life-threatening rare diseases.
We are currently conducting Phase 1/2 clinical studies of our lead
product candidates, AT132 for the treatment of X-linked Myotubular
Myopathy (XLMTM), and AT342 for the treatment of Crigler-Najjar
syndrome. We have two additional product candidates in
development, including 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: anticipated clinical milestones,
potential pipeline expansion, the timing and nature of clinical
development activities, the timing of the release of data from
ongoing or upcoming clinical trials, the timing of regulatory
filings, the expected safety profile of the company's product
candidates, the expected benefits of the company's product
candidates, the expected capacity of the company's internal
manufacturing facility, and the use and adequacy of cash
reserves. 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, 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
September 30,
|
|
Nine months ended
September 30,
|
|
2018
|
|
2017
|
|
2018
|
|
2017
|
|
Unaudited
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
Research and
development
|
$
29,918
|
|
$
20,868
|
|
$
76,157
|
|
$
54,231
|
General and
administrative
|
7,817
|
|
4,342
|
|
20,617
|
|
12,065
|
Total operating
expenses
|
37,735
|
|
25,210
|
|
96,774
|
|
66,296
|
Loss from
operations
|
(37,735)
|
|
(25,210)
|
|
(96,774)
|
|
(66,296)
|
Interest income,
net
|
1,509
|
|
221
|
|
3,662
|
|
483
|
Other expense,
net
|
(65)
|
|
(20)
|
|
(117)
|
|
(50)
|
Net loss
|
$
(36,291)
|
|
$
(25,009)
|
|
$
(93,229)
|
|
$
(65,863)
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
(0.97)
|
|
$
(0.88)
|
|
$
(2.57)
|
|
$
(2.59)
|
Shares used in
computing net loss per share, basic and diluted
|
37,359,877
|
|
28,388,145
|
|
36,302,803
|
|
25,476,261
|
Selected Balance
Sheet Information:
(amounts in thousands)
|
|
|
|
|
|
September
30,
|
|
December
31,
|
|
2018
|
|
2017
|
|
Unaudited
|
|
|
|
|
|
|
Cash, cash
equivalents, marketable securities and restricted cash
|
$
285,542
|
|
$
137,299
|
Total
assets
|
$
337,773
|
|
$
178,662
|
Total
liabilities
|
$
27,218
|
|
$
22,064
|
Total stockholders'
equity
|
$
310,555
|
|
$
156,598
|
Audentes Contacts:
Investor Contact:
Andrew Chang
415.818.1033
achang@audentestx.com
Media Contact:
Katie Hogan
415.951.3398
khogan@audentestx.com
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SOURCE Audentes Therapeutics, Inc.