Following clinical hold lift, Astellas posts early Pompe gene therapy data
Fresh out of a clinical hold, Astellas is spotlighting data on its gene therapy for Pompe disease.
In four patients who got an infusion of the gene therapy, called AT845, three discontinued enzyme replacement therapy. As of the Sept. 15 cutoff, those three patients have been off enzyme replacement therapy for 19, 44 and 51 weeks, respectively.
Astellas did not bring any data on the primary efficacy endpoint — enzyme expression and activity — though it did present secondary efficacy data. The three participants who were taken off ERT maintained similar results in forced vital capacity, a measure of lung capacity, after stopping biweekly treatments. In addition, two of the three had consistent results on a six-minute walk test after being taken off ERT. The third saw a decrease in distance walked on the test after they developed a case of peripheral neuropathy.
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