SAN FRANCISCO, Oct. 5, 2018 /PRNewswire/ --
Conference call and webcast Friday, October 5, 2018 at 8:30 am ET
Webcast may be accessed via
the Investor and Media page of the Audentes website
Call
may be accessed by dialing (833) 659-8620 (U.S.) or (409) 767-9247
(international)
and using conference ID# 8458939
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, will host a webcast and conference call from the
23rd International Annual Congress of the World Muscle Society, on
Friday, October 5, 2018 at
8:30 am ET. The call will focus on
new positive interim data from ASPIRO, the Phase 1/2 clinical trial
of AT132 for the treatment of X-linked Myotubular Myopathy
(XLMTM).
The newly reported data include follow-up assessments ranging
from 4 to 48 weeks for the eight patients enrolled in ASPIRO to
date, including the seven patients enrolled in Cohort 1
(1x1014 vg/kg; six treated and one untreated control)
and one patient enrolled to date in Cohort 2 (3x1014
vg/kg). Key assessments include neuromuscular function as
measured by CHOP INTEND; respiratory function as measured by
maximal inspiratory pressure (MIP) and ventilator dependence; and
vector transduction, transgene expression and histological
improvement as assessed via muscle biopsy. All treated
patients continue to show meaningful improvements in neuromuscular
and respiratory function, with no new treatment-related SAEs
reported since the last scientific update in May 2018.
Conference Call
At 8:30 a.m.
Eastern Time today, October 5,
2018, Audentes management will host a conference call and a
simultaneous webcast to discuss the new positive interim data from
ASPIRO that will be presented at the 23rd International Annual
Congress of the World Muscle Society. 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 (833) 659-8620 (U.S.) or (409) 767-9247
(international) and dial the conference ID# 8458939 to access the
call. A replay of the webcast will be available on the
Audentes website for approximately 30 days.
About AT132 for X-linked Myotubular Myopathy
AT132 is
the Audentes product candidate being developed to treat XLMTM, a
rare monogenic disease characterized by extreme muscle weakness,
respiratory failure and early death, with an estimated 50 percent
mortality rate by 18 months of age. XLMTM is caused by
mutations in the MTM1 gene, which encodes the protein
myotubularin. Myotubularin plays an important role in the
development, maintenance and function of skeletal muscle
cells. AT132 is comprised of an AAV8 vector containing a
functional copy of the MTM1 gene. Over the course of 2018,
Audentes has reported promising safety, efficacy and muscle biopsy
data from ASPIRO, a multicenter, ascending dose
Phase 1/2 clinical study to evaluate the safety and
preliminary efficacy of AT132 in approximately 12 XLMTM patients
less than five years of age. The preclinical development of
AT132 was conducted in collaboration with Genethon
(www.genethon.fr).
AT132 has been granted Regenerative Medicine Advanced Therapy
(RMAT), Rare Pediatric Disease, Fast Track and Orphan Drug
designations by the FDA, and Priority Medicines (PRIME) and
Orphan Drug designations by the European Medicines Agency
(EMA).
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.
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.