Audentes Therapeutics to Provide Update on New Positive Interim Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-linked Myotubular Myopathy, from the 23rd International Annual Congress of the World Muscle Society

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
[email protected] 

Media Contact:
Katie Hogan
415.951.3398
[email protected]

SOURCE Audentes Therapeutics, Inc.

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