Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Treatment for Pompe Disease
Amicus Therapeutics today announced that its global Phase 1/2 study of ATB200/AT2221 has yielded positive initial results.
Amicus Therapeutics (NASDAQ:FOLD) today announced that its global Phase 1/2 study of ATB200/AT2221 has yielded positive initial results.
According to the press release:
“ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone.”
CEO John F. Crowley said the following:
“We set out on this journey in Pompe disease at Amicus with the intent to develop a new treatment paradigm. These preliminary results, while still early, give us greater confidence that we are developing a drug regimen that may be highly differentiated from any other approach. Even at this interim analysis, it is very encouraging to see no infusion-associated reactions in these initial patients, as well as the desired PK profile and improvements in key muscle damage biomarkers in two of four patients.”