The leading American AAV-based genetic medicines company, Audentes Therapeutics Inc., has reportedly presented new positive data from ASPIRO, the AT132 Phase 1/2 clinical trial for the treatment of XLMTM (X-linked Myotubular Myopathy). The data has been presented at the 22^nd Annual Meeting of ASGCT (American Society of Gene & Cell Therapy) by Perry B. Shieh, Ph.D., M.D., ASPIRO’s principal investigator.

Sources stated that the newly presented data include safety & efficacy assessments for eleven patients enrolled in ASPIRO. Sources also cited that the key evaluations include neuromuscular function as assessed by achievement of motor milestones and improvements in CHOP INTEND score; respiratory function as assessed by improvements in MIP (maximal inspiratory pressure) and reduction in ventilator dependence; along with mRNA, vector copy number, protein expression as well as histological improvement as evaluated through muscle biopsy.

Dr. Shieh was quoted saying that the promising clinical profile, ascertained by AT132 in the ASPIRO Phase 1/2 study, is hugely supported by the latest data. He added that the results across the dose cohorts exhibit clinically meaningful improvements in both respiratory and neuromuscular functions, progressive achievement of motor developmental milestones and long-term improvements in muscle pathology.

According to Matthew R. Patterson, the Chairman & Chief Executive Officer at Audentes, the firm is thrilled to share the latest results and is simultaneously striving towards its goal of providing patients suffering from XLMTM worldwide with AT132 at the earliest. The company looks forward to further significant steps for the program, that include optimal dose selection and discussions with US & European regulators with respect to the probable pathways to license application, he added.

For the record, AT132 is an AAV8 vector used in the XLMTM treatment. Trusted reports cite that the preclinical development of AT132 had been conducted in close alliance with French biotech company Genethon. AT132 has been granted the RMAT (Regenerative Medicine & Advanced Therapy), Fast Track, Rare Pediatric Disease & Orphan Drug designations by the USFDA, while PRIME (Priority Medicines) & Orphan Drug designations by the EMA (European Medicines Agency).  

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