SAN FRANCISCO, March 8, 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 fourth
quarter and full year ended December 31,
2017 and provided an update on the company's recent
achievements and anticipated upcoming milestones.
"2017 was a transformational year marked by significant
achievements across our portfolio of gene therapy product
candidates," stated Matthew R.
Patterson, President and Chief Executive Officer.
"Among our many accomplishments, we are particularly pleased to
have recently reported positive interim data from the first dose
cohort of patients in ASPIRO, the Phase 1/2 clinical study of our
product candidate AT132 to treat XLMTM. These promising
initial data add to the growing body of evidence of the utility of
AAV-based gene therapy to treat rare neuromuscular diseases, and we
are optimistic that they signal a turning point in our ability to
bring meaningful benefit to patients and families living with
XLMTM."
Mr. Patterson continued, "As we look forward to the year ahead,
we are excited by our many anticipated upcoming pipeline catalysts,
including sharing additional data from our ASPIRO study in XLMTM,
new data from our VALENS study in Crigler-Najjar, and advancing
additional product candidates into the clinic for Pompe disease and
CASQ2-CPVT. With our recently completed follow-on offering, we are
well capitalized to continue our mission to develop transformative
gene therapy products for patients living with serious,
life-threatening rare diseases."
Recent Achievements & Upcoming Key Events
- AT132 for X-Linked Myotubular Myopathy (XLMTM):
-
- Announced positive interim data in the first three patients
dosed in ASPIRO, the Phase 1/2 clinical study of AT132 to treat
XLMTM. Plan to dose three additional patients in the first cohort
of ASPIRO and to report additional interim data in the second
quarter of 2018
- Received Rare Pediatric Disease and Fast Track designations for
AT132 from FDA
- AT342 for Crigler-Najjar Syndrome:
-
- Initiated dosing in the first cohort of patients in VALENS, the
Phase 1/2 clinical study of AT342 to treat Crigler-Najjar Syndrome,
in the first quarter of 2018 and plan to report preliminary data
from VALENS in the second quarter of 2018
- Received Rare Pediatric Disease and Fast Track designations for
AT342 from FDA
- AT982 for Pompe Disease:
-
- Announced selection of the clinical development candidate,
AT982, a novel AAV8 vector designed to express GAA in tissues
relevant to Pompe disease, including skeletal muscle, the heart and
the nervous system, and to reduce immunogenicity, thereby
addressing the key limitations of existing ERT therapy for
Pompe
- Plan to file IND in mid-2018 and to initiate a Phase 1/2
clinical study in the fourth quarter of 2018
- AT307 for CASQ2-CPVT:
-
- Completed IND-enabling preclinical studies of AT307
- Plan to file IND in the first quarter of 2018 and to initiate a
Phase 1/2 clinical study in the fourth quarter of 2018
- Initiated activities to identify CASQ2-CPVT patients and
further characterize the disease burden and unmet medical need for
patients living with CPVT
- Manufacturing:
-
- Completed manufacturing campaigns of all product candidates in
our state-of-the-art, 2x500L bioreactor-scale cGMP facility,
designed to support global commercial licensure
- Continued optimization of our serum-free suspension mammalian
cell culture manufacturing process, which we expect will support
commercial production for all of our product candidates
- Built a world-class scientific team to support manufacturing
and development operations, including expertise in novel product
design, process and assay development, and AAV immunology
Fourth Quarter and Full Year 2017 Financial Results
- Cash Position: At December 31,
2017, Audentes had cash, cash equivalents, and short-term
investments of $133.6 million. In
January 2018, Audentes further
strengthened its balance sheet with the completion of a follow-on
financing, issuing 6,612,500 shares of common stock at an offering
price of $35.00 per share, resulting
in net proceeds of approximately $217.2
million after the deduction of underwriting discounts,
commissions and estimated offering expenses. Current cash, cash
equivalents and short-term investments are planned to fund
operations into the second half of 2020.
- Research and Development Expenses: Research and
development expenses were $21.7
million for the fourth quarter of 2017 and $75.9 million for the year ended December 31, 2017, compared to $16.6 million and $48.8
million, respectively, for the same periods in 2016. The
increase in research and development expenses was primarily
attributable to an increase in research and development headcount
and related facility costs, increased internal manufacturing costs,
an increase in preclinical and clinical study costs for our
development programs, and an increase in expenses related to
expanded internal research activities.
- General and Administrative Expenses: General and
administrative expenses were $5.2
million for the fourth quarter of 2017 and $17.3 million for the year ended December 31, 2017, compared to $3.2 million and $11.3
million, respectively, for the same periods in 2016. The
increase in general and administrative expenses was primarily
attributable to increased general and administrative headcount and
related facility costs, increased professional service and audit
fees, and higher insurance costs.
- Net Loss: Net loss was $24.4
million for the fourth quarter of 2017 and $90.2 million for the year ended December 31, 2017, compared to $19.7 million and $59.7
million, respectively, for the same periods in 2016.
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 2017
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 1-833-659-8620 (U.S.) or 1-409-767-9247 (international) and
dial the conference ID 5890848 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, the
timing and nature of clinical development activities, the timing of
the release of data from ongoing clinical trials, 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
Operating Results:
(amounts in thousands, except share and per share data)
|
Three months ended
December 31,
|
|
Year ended December
31,
|
|
2017
|
|
2016
|
|
2017
|
|
2016
|
|
Unaudited
|
|
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
Research and
development
|
$
21,671
|
|
$
16,599
|
|
$
75,902
|
|
$
48,770
|
General and
administrative
|
5,210
|
|
3,233
|
|
17,275
|
|
11,276
|
Total operating
expenses
|
26,881
|
|
19,832
|
|
93,177
|
|
60,046
|
Loss from
operations
|
(26,881)
|
|
(19,832)
|
|
(93,177)
|
|
(60,046)
|
Interest
income
|
284
|
|
167
|
|
767
|
|
472
|
Other income
(expense), net
|
(24)
|
|
(11)
|
|
(74)
|
|
(94)
|
Loss before income
taxes
|
(26,621)
|
|
(19,676)
|
|
(92,484)
|
|
(59,668)
|
Income tax
benefit
|
2,246
|
|
-
|
|
2,246
|
|
-
|
Net Loss
|
$
(24,375)
|
|
$
(19,676)
|
|
$
(90,238)
|
|
$
(59,668)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
(0.82)
|
|
$
(0.91)
|
|
$
(3.40)
|
|
$
(5.59)
|
Shares used in
computing net loss per share, basic and diluted
|
29,847,934
|
|
21,726,223
|
|
26,578,162
|
|
10,673,559
|
Selected Balance Sheet Information:
(amounts in thousands)
|
December
31,
|
|
December
31,
|
|
2017
|
|
2016
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
$
133,605
|
|
$
104,883
|
Total
assets
|
$
178,662
|
|
$
142,057
|
Total
liabilities
|
$
22,064
|
|
$
22,686
|
Total stockholders'
equity
|
$
156,598
|
|
$
119,371
|
Audentes Contacts:
Investor Contact:
Andrew Chang
415.818.1033
achang@audentestx.com
Media Contact:
Paul Laland
415.519.6610
plaland@audentestx.com
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SOURCE Audentes Therapeutics, Inc.