SAN FRANCISCO, Nov. 14, 2017 /PRNewswire/ -- Audentes
Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company
focused on developing and commercializing gene therapy products for
patients living with serious, life-threatening rare diseases, today
reported its financial results for the quarter ended September 30, 2017, and provided an update on the
company's recent achievements and anticipated upcoming
milestones.
"I am pleased with the excellent progress we have made to
advance our pipeline toward key value inflection milestones,
including the recent completion of dosing for the first cohort in
ASPIRO, the Phase 1/2 clinical study of AT132 for the treatment of
XLMTM," stated Matthew R. Patterson,
President and Chief Executive Officer. "While we are only
weeks into the study, we are encouraged by the progress to date and
look forward to sharing preliminary safety and efficacy data from
the first cohort of patients in early January 2018. This will
be the first of many catalysts over the coming months as we execute
on our ambitious goal of creating the world's leading gene therapy
company focused on developing a multi-product pipeline to treat
serious, life-threatening rare diseases with high unmet medical
need."
Recent Achievements & Upcoming Key Events
- AT132 for X-Linked Myotubular Myopathy:
-
- Completed dosing of the first cohort of patients in ASPIRO, the
Phase 1/2 clinical study of AT132. To date, AT132 has been
well-tolerated by all patients with no significant
treatment-related safety signals.
- Announced interim results from INCEPTUS, a prospective natural
history run-in study in patients with XLMTM. The primary
objectives of INCEPTUS are to characterize the clinical condition
of children with XLMTM, identify subjects for potential enrollment
in ASPIRO, and serve as a longitudinal baseline and with-in patient
control for ASPIRO. Preliminary results from INCEPTUS confirm
the significant neuromuscular and respiratory deficits experienced
by XLMTM patients, and provide insight into the relevance and
sensitivity of assessments used in the ASPIRO study.
- Received approval for the Clinical Trial Authorisation (CTA)
application for AT132 from the United
Kingdom's (U.K.) Medicines and Healthcare Products
Regulatory Agency (MHRA), and anticipate initiation of U.K.
enrollment in the first quarter of 2018 for ASPIRO.
- Plan to report preliminary clinical data from ASPIRO in early
January 2018
- AT342 for Crigler-Najjar:
-
- Plan to discuss initial results of LUSTRO, the prospective
natural history run-in study being conducted in patients with
Crigler-Najjar Syndrome, on the third quarter 2017 earnings
call
- Plan to dose first patient in VALENS, the Phase 1/2 clinical
study of AT342, in the first quarter of 2018, and to report
preliminary data in the second quarter of 2018
- Received approval for the CTA application for AT342 from the
MHRA, and anticipate initiation of U.K. enrollment in the first
quarter of 2018 for VALENS
- AT982 for Pompe Disease:
-
- Conducting a comprehensive in vivo construct selection and
dose-ranging study evaluating a range of AAV8 and AAV9 vector
constructs designed to express GAA selectively within tissues
relevant to Pompe disease, including skeletal muscle, motor neurons
and liver. Candidate vectors are being evaluated across a
broad range of neuromuscular function and biochemical endpoints at
multiple dose levels.
- Plan to select optimal construct and file IND in the first half
of 2018
- AT307 for CASQ2-CPVT:
-
- Completed successful pre-IND/CTA meetings with both the U.S.
Food and Drug Administration (FDA) and MHRA, and have incorporated
input from both agencies into IND filing plans
- Expect to file IND in the first quarter of 2018
- Appointed Fulvio Mavilio, Ph.D.
as Vice President Scientific Affairs, Europe. Dr. Mavilio is
an internationally recognized scientific leader who has made
important contributions to the field of molecular genetics and gene
therapy for rare diseases.
Third Quarter 2017 Financial Results
- Cash Position: As of September 30, 2017, Audentes
had cash, cash equivalents and short-term investments of
approximately $156.0 million.
- R&D Expenses: Research and development
expenses were $20.9 million for the third quarter of 2017
compared to $12.5 million for
the same period in 2016, an increase of $8.4 million. The
increase was primarily due to increased research and development
expenses for our AT132 and AT307 programs, increases in our
research and development headcount and higher facility costs as we
made additional investments in our manufacturing and research
facilities. For the nine months ended September 30, 2017, research and development
expenses were $54.2 million compared
to $32.2 million for the same
period in 2016.
- General and Administrative: General and administrative
expenses were $4.3 million for the third quarter of 2017
compared to $2.9 million for the same period in 2016, an
increase of $1.4 million. The increase was primarily
due to increased G&A headcount and increases in other G&A
expenses related to regulatory compliance and operations as a
public company. For the nine months ended September 30, 2017, general and administrative
expenses were $12.1 million compared
to $8.0 million for the same
period in 2016.
- Net Loss: Net loss was $25.0 million for the
third quarter of 2017, compared to a net loss
of $15.4 million for the same period in 2016. For
the nine months ended September 30,
2017, our net loss was $65.9
million as compared to $40.0
million for the same period 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 third quarter 2017
financial results and provide a corporate update. To access a live
webcast of the conference call, please visit the Investor and Media
page 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 2599735 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 gene therapy products for patients
living with serious, life-threatening rare diseases. We are
currently conducting a Phase 1/2 clinical study of our lead product
candidate AT132 for the treatment of X-Linked Myotubular Myopathy
(XLMTM) and have three additional product candidates in
development, including AT342 for the treatment of Crigler-Najjar
Syndrome, 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: the timing of key program
milestones, including plans to dose patients in, and report
clinical data from, ASPIRO, the Phase 1/2 study of AT132 in XLMTM,
and VALENS, the Phase 1/2 study of AT342 in Crigler-Najjar
Syndrome; plans to develop a multi-product pipeline; the utility of
INCEPTUS data to provide insight into the relevance and sensitivity
of assessments in ASPIRO; successful completion of pre-clinical
programs and plans to file INDs in the Pompe disease and CASQ2-CPVT
programs. 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, the
company's ability to establish and scale-up manufacturing processes
that comply with regulatory requirements, the company's ability to
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,
|
|
|
2017
|
|
|
2016
|
|
|
2017
|
|
|
2016
|
|
|
Unaudited
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
$
|
20,868
|
|
|
$
|
12,531
|
|
|
$
|
54,231
|
|
|
$
|
32,171
|
|
General and
administrative
|
|
4,342
|
|
|
|
2,942
|
|
|
|
12,065
|
|
|
|
8,043
|
|
Total operating
expenses
|
|
25,210
|
|
|
|
15,473
|
|
|
|
66,296
|
|
|
|
40,214
|
|
Loss from
operations
|
|
(25,210)
|
|
|
|
(15,473)
|
|
|
|
(66,296)
|
|
|
|
(40,214)
|
|
Interest income,
net
|
|
221
|
|
|
|
116
|
|
|
|
483
|
|
|
|
305
|
|
Other expense,
net
|
|
(20)
|
|
|
|
(12)
|
|
|
|
(50)
|
|
|
|
(83)
|
|
Net loss
|
$
|
(25,009)
|
|
|
$
|
(15,369)
|
|
|
$
|
(65,863)
|
|
|
$
|
(39,992)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
|
(0.88)
|
|
|
$
|
(0.94)
|
|
|
$
|
(2.59)
|
|
|
$
|
(5.74)
|
|
Shares used in
computing net loss per share, basic and diluted
|
|
28,388,145
|
|
|
|
16,423,894
|
|
|
|
25,476,261
|
|
|
|
6,962,446
|
|
Selected Balance Sheet Information:
(amounts in thousands)
|
September
30,
|
|
|
December
31,
|
|
|
2017
|
|
|
2016
|
|
|
Unaudited
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
$
|
156,045
|
|
|
$
|
104,883
|
|
Total
assets
|
$
|
200,247
|
|
|
$
|
142,057
|
|
Total
liabilities
|
$
|
24,925
|
|
|
$
|
22,686
|
|
Total stockholders'
equity
|
$
|
175,322
|
|
|
$
|
119,371
|
|
Audentes Contacts:
Investor Contact:
Andrew Chang
Director, Investor Relations
415.818.1033
ir@audentestx.com
Media Contact:
Paul Laland
415.519.6610
media@audentestx.com
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SOURCE Audentes Therapeutics, Inc.