SAN FRANCISCO, Feb. 27, 2019 /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 fourth
quarter and full year ended December 31,
2018 and provided an update on the company's recent
achievements and anticipated upcoming milestones.
"2018 was a transformative year marked by significant progress
in advancing our mission to bring innovative gene therapy products
to patients living with severe, life-threatening rare diseases,"
stated Matthew R. Patterson,
Chairman and Chief Executive Officer. "Importantly, we
established a compelling clinical profile for AT132, our product
candidate being developed to treat XLMTM, and are in ongoing
discussions with the FDA and EMA to facilitate agreement on license
application pathways for AT132."
Mr. Patterson continued, "We enter 2019 with over two years of
cash runway, a position of strength that will ensure our research
and development efforts continue to advance across our promising
portfolio. To that end, we remain highly encouraged by
preclinical work in our Pompe disease program and continue to guide
to a third quarter IND submission for AT845. We are also
continuing to advance our new product candidate, AT720, a novel
AAV-based therapeutic being developed to treat a large
neuromuscular disease with significant unmet medical need, and plan
to disclose the therapeutic target and provide a program overview
in the second quarter of 2019."
Recent Achievements & Upcoming Key Events
AT132 for X-Linked Myotubular Myopathy (XLMTM):
- Completed productive initial interactions with the U.S. Food
and Drug Administration (FDA) and European Medicines Agency (EMA)
to discuss potential BLA and MAA submission pathways for AT132
under the Regenerative Medicine Advanced Therapy (RMAT) and
Priority Medicines (PRIME) designations, respectively.
- Proceeding with previously announced plan to enroll an
additional 3-5 patients in Cohort 2 (3x1014 vector
genomes per kilogram) of ASPIRO, the Phase 1/2 study of AT132.
- On track to select optimal dose of AT132 in the second quarter
of 2019. Optimal dose selection will be determined after an
evaluation of the six-month biopsy results from the first three
patients dosed in Cohort 2 of ASPIRO.
- Subsequent to the determination of the optimal dose, Audentes
plans to provide an updated data package to FDA and EMA to
facilitate final agreement on license application pathways in the
third quarter of 2019.
- Continuing process and facility validation efforts based on
preliminary FDA feedback on our chemistry, manufacturing, and
controls (CMC) plans, to support AT132 registration efforts.
- Since program inception, AT132 clinical material has been
manufactured utilizing the same process, facility, and scale
intended to supply the commercial market.
- Next clinical data presentation planned at the 2019 Annual
Meeting of the American Society of Gene and Cell Therapy (ASGCT) in
Washington, D.C. from April 29th to May 2nd.
AT845 for Pompe Disease:
- Encouraged by continued progress in preclinical studies.
- On track to file IND in the third quarter of 2019.
AT342 for Crigler-Najjar Syndrome:
- VALENS Phase 1/2 study ongoing.
- Next program update planned in the second quarter of 2019.
Pipeline Expansion:
- Advancing our new product candidate, AT720, a novel AAV-based
therapeutic being developed to treat a large neuromuscular disease
with significant unmet medical need.
- On track to disclose the therapeutic target and provide a
program overview in the second quarter of 2019.
Manufacturing:
- State-of-the-art, internal, large-scale cGMP manufacturing
facility provides sufficient capacity for AT132 global
commercialization as well as continued clinical development of
pipeline programs.
- Currently operating in mammalian, serum-free suspension culture
production process at 1,000-liter scale with the ability to add up
to an additional 5,000 liters of capacity.
- Internal analytical development, fill-finish, and QC testing
capabilities provide a fully-integrated platform to design,
manufacture, fill, and release our product candidates for clinical
and eventually, commercial use.
Corporate:
- Expanded our leadership team with the addition of Eric
Mosbrooker, Senior Vice President and Chief Commercial Officer, to
lead the development and execution of our global commercial
strategy.
Fourth Quarter and Full Year 2018 Financial Results
- Cash Position: At December 31,
2018, Audentes had cash, cash equivalents, marketable
securities and restricted cash of $418.1
million. In 2018, we strengthened the balance sheet with two
follow-on financings, resulting in aggregate net proceeds of
approximately $380 million after the
deduction of underwriting discounts, commissions, and offering
expenses. Our current cash, cash equivalents, and marketable
securities are expected to fund operations into 2021.
- Research and Development Expenses: Research and
development expenses were $28.2
million for the fourth quarter of 2018 and $104.4 million for the year ended December 31, 2018, compared to $21.7 million and $75.9
million, respectively, for the same periods in 2017. The
increase in research and development expenses was primarily
attributable to increases in our research and development headcount
and related personnel and facilities costs as we continued to make
investments in our internal research and development and
manufacturing capabilities.
- General and Administrative Expenses: General and
administrative expenses were $9.4
million for the fourth quarter of 2018 and $30.0 million for the year ended December 31, 2018, compared to $5.2 million and $17.3
million, respectively, for the same periods in 2017. The
increase in general and administrative expenses was primarily
attributable to increases in our general and administrative
headcount and related personnel and facilities costs, increases in
consulting, professional services and audit fees, and costs related
to on-going regulatory compliance activities as a public
company.
- Net Loss: Net loss was $35.6
million for the fourth quarter of 2018 and $128.8 million for the year ended December 31, 2018, compared to $24.4 million and $90.2
million, respectively, for the same periods in 2017.
Conference Call
At 4:30 p.m. Eastern
Time today, Audentes management will host a conference call
and a simultaneous webcast to discuss its fourth quarter and full
year 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 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# 1196899 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 AT845 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, the
timing and nature of clinical development activities, the timing of
the release of data from ongoing clinical trials, the timing of
regulatory interactions and the nature of potential agreements
reached with regulatory authorities, the timing of regulatory
filings, the expected benefits of the company's product candidates
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, 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
Amounts in thousands
except share and per share data
Operating Results
|
Three months ended
December 31,
|
|
Year ended December
31,
|
|
2018
|
|
2017
|
|
2018
|
|
2017
|
|
Unaudited
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
Research and
development
|
$
28,213
|
|
$
21,671
|
|
$
104,370
|
|
$
75,902
|
General and
administrative
|
9,385
|
|
5,210
|
|
30,002
|
|
17,275
|
Total operating
expenses
|
37,598
|
|
26,881
|
|
134,372
|
|
93,177
|
Loss from
operations
|
(37,598)
|
|
(26,881)
|
|
(134,372)
|
|
(93,177)
|
Interest income,
net
|
2,368
|
|
284
|
|
6,030
|
|
767
|
Other expense,
net
|
(362)
|
|
(24)
|
|
(479)
|
|
(74)
|
Loss before income
taxes
|
(35,592)
|
|
(26,621)
|
|
(128,821)
|
|
(92,484)
|
Income tax
benefit
|
-
|
|
2,246
|
|
-
|
|
2,246
|
Net loss
|
$
(35,592)
|
|
$
(24,375)
|
|
$
(128,821)
|
|
$
(90,238)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
(0.84)
|
|
$
(0.82)
|
|
$
(3.40)
|
|
$
(3.40)
|
Shares used in
computing net loss per share, basic and diluted
|
42,400,890
|
|
29,847,934
|
|
37,839,855
|
|
26,578,162
|
Selected Balance Sheet Data
|
December
31,
|
|
December
31,
|
|
2018
|
|
2017
|
|
|
|
|
Cash, cash
equivalents, marketable securities and restricted cash
|
$
418,055
|
|
$
137,299
|
Total
assets
|
$
472,555
|
|
$
178,662
|
Total
liabilities
|
$
29,801
|
|
$
22,064
|
Total stockholders'
equity
|
$
442,754
|
|
$
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.