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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
Conference call and webcast
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
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
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
About Audentes Therapeutics, Inc.
For more information regarding Audentes, please visit www.audentestx.com.
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