SAN FRANCISCO,
May 9, 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 first quarter ended March 31, 2018 and provided an update on the
company's recent achievements and anticipated upcoming
milestones.
"During the first quarter of 2018 we continued to make
excellent progress across our product portfolio," stated
Matthew R. Patterson, Chief
Executive Officer of Audentes. "In particular, we are excited
by the positive preliminary results we have observed in our XLMTM
program and we look forward to reporting additional interim data
from the first dose cohort of ASPIRO patients at the upcoming ASGCT
annual meeting. We are also pleased by the preliminary data
from the first patient treated in our Crigler-Najjar program, which
demonstrated an encouraging early safety profile and initial
proof-of-concept for efficacy. We look forward to treating
our next patient at a higher dose in the coming
weeks."
Mr. Patterson continued, "Finally, we are excited by the
growing momentum behind our product candidate AT982, which we
believe offers a best-in-class approach for the treatment of Pompe
disease. Based on recent discussions with regulatory
authorities, we have expanded our clinical vision and
plan to conduct two separate Phase 1/2 clinical studies for the
infantile and late onset Pompe disease patient populations in
parallel. As such, we have expanded the scope of our
IND-enabling preclinical program, and now plan to include both
studies in the initial IND filing targeted for the fourth quarter
of 2018, which will support the initiation of the studies in the
first half of 2019."
Recent Achievements & Upcoming Key
Events
- AT132 for XLMTM:
-
- Completed dosing of three additional patients (cohort 1
expansion) in the 1x1014 vg/kg cohort of ASPIRO,
bringing the total number of patients enrolled to date to seven
(six AT132-treated and one delayed-treatment control). No
significant treatment-related safety signals have been identified
to date in cohort 1 expansion patients.
- Plan to report additional interim data during an oral
presentation at ASGCT on May 16,
2018. Presentation to include up to 24-week data for the
first four subjects enrolled and up to 4-week data in the cohort 1
expansion patients.
- Plan to report six-month biopsy data from the first three
patients dosed and announce plans with regard to dose escalation in
the third quarter of 2018
- AT342 for Crigler-Najjar Syndrome:
-
- Initial proof of concept established based on 12-week
data from first patient enrolled in VALENS study, a 12-year-old
male, at a dose of 1.5x1012 vg/kg
- AT342 has been well-tolerated with no significant
treatment-related safety signals to date
- Treatment resulted in a rapid decline in total bilirubin
levels from approximately 11 mg/dL at baseline to 4 mg/dL at week 2
post-dosing, with a gradual return to baseline by week 12. A
similar efficacy result was observed with low doses of AT342 in a
dose ranging study in the mouse model of Crigler Najjar, while higher doses demonstrated
durable bilirubin reduction.
- Plan to dose escalate to the 6 x1012 vg/kg and
enroll the next patient in VALENS in the coming weeks
- Plan to report interim 12-week data in the first patient
dosed in VALENS at the 51st Annual Congress of the
European Society for Paediatric Gastroenterology, Hepatology and
Nutrition (ESPGHAN) on May 10, 2018
and at ASGCT on May 17,
2018
- Plan to report the next interim data update
from VALENS in the second half of 2018
- 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 in the liver to reduce
immunogenicity, thereby addressing the key limitations of existing
enzyme replacement therapy for Pompe disease
- Plan to present additional data from AT982 in a Pompe
mouse model at ASGCT on May 16,
2018
- Plan to expand the preclinical program to support the
filing of an IND to study AT982 in both infantile and late onset
Pompe disease patients
- Plan to file the IND in the fourth quarter of 2018 and
initiate both Phase 1/2 clinical studies in the first half of
2019
- AT307 for CASQ2-CPVT:
-
- IND has been submitted. FDA has completed its initial
review and provided a short list of questions to be addressed prior
to the IND becoming active. We expect to submit responses in
the coming weeks.
- Received Fast Track designation from the FDA
- Continuing patient identification activities to better
characterize CASQ2-CPVT prevalence. Results from these
efforts will inform clinical plans as they relate to the timing of
a potential Phase 1/2 study.
- Key Corporate Milestones:
-
- In January 2018, Audentes
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 offering
expenses
- Announced the promotion of Natalie Holles to
President and Chief Operating Officer. In this new role, Ms.
Holles will oversee the day-to-day operations of the company,
including research, development, manufacturing, program management
and corporate development.
First Quarter 2018 Financial Results
- Cash Position: At March 31, 2018, Audentes had cash, cash
equivalents, and short-term investments of $326.1 million. 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 $19.9 million for the
first quarter of 2018 compared to $14.6
million for the same period in 2017, an increase of
$5.3 million. The increase in
research and development expenses 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 compensation
expense, and is partially offset by a decrease in the estimated
fair value of the contingent liability associated with the 2015
acquisition of Cardiogen Sciences, resulting in a $2.3 million reduction in research and
development expense during the first quarter of 2018. Research and
Development expenses included $2.1
million of non-cash stock-based compensation
expense.
- General and Administrative Expenses:
General and administrative expenses were $6.5 million for the first quarter of 2018
compared to $3.6 million for the same
period in 2017, an increase of $2.9
million. The increase in general and administrative expenses
was primarily attributable to increased headcount and related
facility costs, increased professional service fees, higher stock
compensation expense and higher costs driven by continued public
company regulatory compliance initiatives. General and
administrative expense includes $1.3
million of non-cash stock-based compensation
expense.
- Net Loss: Net loss was $25.6 million for the first quarter of 2018
compared to $18.1 million for the
same period 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 first 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 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 9789828 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
March 31,
|
|
2018
|
|
2017
|
|
Unaudited
|
|
|
|
|
Operating
expenses:
|
|
|
|
Research and
development
|
$
19,891
|
|
$
14,587
|
General and
administrative
|
6,519
|
|
3,658
|
Total operating
expenses
|
26,410
|
|
18,245
|
Loss from
operations
|
(26,410)
|
|
(18,245)
|
Interest income,
net
|
859
|
|
147
|
Other expense,
net
|
(20)
|
|
(17)
|
Net loss
|
$
(25,571)
|
|
$
(18,115)
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
(0.74)
|
|
$
(0.83)
|
Shares used in
computing net loss per share, basic and diluted
|
34,582,071
|
|
21,755,134
|
Selected Balance
Sheet Information:
|
(amounts in
thousands)
|
|
|
March 31,
|
|
December
31,
|
|
2018
|
|
2017
|
|
Unaudited
|
|
|
|
|
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
$ 326,128
|
|
$
133,605
|
Total
assets
|
$ 373,089
|
|
$
178,662
|
Total
liabilities
|
$
20,647
|
|
$
22,064
|
Total stockholders'
equity
|
$ 352,442
|
|
$
156,598
|
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.