SAN FRANCISCO, Jan. 7, 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 provided an update on recent progress and the
company's strategic priorities for 2019.
"2018 was an outstanding year for Audentes," stated Matthew R. Patterson, Chairman and Chief
Executive Officer. "We are particularly encouraged by the
progress in the ASPIRO Phase 1/2 study and the promising clinical
profile of AT132 for the treatment of X-linked Myotubular
Myopathy. We received RMAT and PRIME designations in the U.S.
and EU respectively, and recently held productive initial
interactions with both the FDA and EMA to discuss potential
registration pathways for AT132. We look forward to sharing
further details from those interactions in early 2019."
Mr. Patterson continued, "We are preparing for multiple
milestones and inflection points across our portfolio of product
candidates in the coming year, including the continuation of BLA
and commercial readiness activities in our XLMTM program. To
support this effort, we recently expanded our leadership team with
the addition of Eric Mosbrooker, a seasoned executive with a
demonstrated track-record of building global commercial
organizations and successfully launching rare disease products, to
lead the development and execution of our global commercial
strategy. In our Pompe disease program, we are encouraged by
the progress of our ongoing preclinical studies and are planning to
file an IND for AT845 in the third quarter of 2019. We are
also advancing 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. Finally, our ability to meet these important
milestones is supported by a strong balance sheet. With over
two years of cash runway, we are well-positioned to make meaningful
progress toward our goal of providing transformative therapies to
patients living with devastating rare diseases."
Mr. Patterson will provide a corporate update and 2019 outlook
in a presentation at the 37th Annual J.P. Morgan Healthcare
Conference on Tuesday, January 8,
2019, at 3:30 pm PST. To
access the live webcast of the presentation, please visit the
Events & Presentations page within the Investors & News
section of the Audentes website. Replays of the live webcast
will be available on the Audentes website for approximately 30 days
following the conference.
Key 2018 Accomplishments and 2019 Milestones
AT132 for XLMTM
- Reported promising safety, efficacy, and muscle biopsy data
from ASPIRO. The latest ASPIRO data set (presented
October 2018) demonstrated all six
treated patients in Cohort 1 (1x1014 vector genomes per
kilogram (vg/kg) and the sentinel patient in Cohort 2
(3x1014 vg/kg) showed meaningful improvements in
neuromuscular and respiratory function with results ranging from 4
to 48 weeks. Three patients achieved ventilator independence,
and all other patients showed significant reductions in ventilator
use. Additionally, week 24 muscle biopsy data for the first
four treated patients showed robust tissue transduction, protein
expression, and histological improvement.
- Completed dosing of full Cohort 2 (three treated patients and
one delayed-treatment control), and plan to dose an additional
three to five patients as part of a Cohort 2 expansion arm in early
2019. Next clinical data update planned at the 2019 Annual
Meeting of the American Society of Gene and Cell Therapy (ASGCT) in
May.
- Awarded Regenerative Medicine Advanced Therapy (RMAT) and
Priority Medicines (PRIME) designations by the FDA and the EMA,
respectively. Completed productive initial interactions with
both agencies to discuss potential registration pathways for
AT132. Plan to provide additional meeting details in Q1
2019.
AT845 for Pompe Disease
- Conducted encouraging preclinical studies of AT845 for the
treatment of Pompe Disease, and plan to file IND in Q3 2019.
AT342 for Crigler-Najjar Syndrome
- Established initial proof of concept based on 12-week data from
first patient enrolled in the VALENS Phase 1/2 study at a dose of
1.5x1012 vg/kg. Next program update planned in Q1
2019.
- Awarded Rare Pediatric Disease and Fast Track designations for
AT342 by the FDA.
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.
- Plan to disclose the therapeutic target and provide a program
overview in Q2 2019.
Manufacturing
- Commenced BLA readiness and process validation activities for
AT132. Chemistry, manufacturing, and controls (CMC) approach
is consistent with recent FDA gene therapy guidance. Since
program inception, clinical material has been manufactured
utilizing substantially the same process, scale, and facility
intended to supply the commercial market.
Financials
- Strengthened balance sheet with two follow-on financings,
resulting in aggregate net proceeds of approximately $380 million after the deduction of underwriting
discounts, commissions, and estimated offering expenses.
- On a proforma basis at September 30,
2018, Audentes had cash, cash equivalents and short-term
investments totaling nearly $450
million, which includes net proceeds of $162.5 million from its follow-on financing that
closed in October 2018. These resources are expected to fund
the company into 2021.
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,
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 nature and timing of the
release of information related to regulatory communications, 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.
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.