The investigational drug AT132 has been awarded Regenerative Medicine Advanced Therapy designation for the treatment of X-linked Myotubular Myopathy by the United States FDA. You can read the source press release here, at Audentes Therapeutics’ website.
About X-Linked Myotubular Myopathy
X-Linked Myotubular Myopathy (XLMTM) is a genetic condition characterised by muscle weakness and reduced muscle tone. According to the NIH, it is caused by an alteration to the MTM1 gene and mainly affects males. This gene is involved in creating the enzyme myotubularin, which plays an important role in the development and maintenance of muscle cells. Alterations to the MTM1 gene may, therefore, affect the functioning of this enzyme, leading to symptoms of XLMTM.
About AT132
AT132 is being developed as an investigational treatment for XLMTM. AT132 is an AAV8 vector that carries a functional copy of the MTM1 gene that underlies XLMTM.
AT132 have previously been awarded Rare Paediatric Disease, Orphan Drug, and Fast Track designations by the FDA, as well as Priority Medicines and Orphan Drug statuses by EMA. Now, it has also been awarded Regenerative Medicine Advanced Therapy designation by the FDA.
Research into AT132
AT132 is currently being studied for XLMTM in an on-going Phase 1/2 clinical trial called Aspiro, which has recently produced positive interim data.
So far, the data indicates that patients given AT132 show improvements in their neuromuscular function (based on the CHOP-INTEND scale), as well as improved respiratory function (as shown through reduced ventilator dependence and increases in maximal inspiratory pressure). In addition, muscle biopsies from three patients at the 24-week point show effective tissue transduction (the introduction of foreign DNA). The safety profile of the experimental drug also appears to be acceptable.
For more details, you can view the original press release here.
