Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Treatment for Pompe Disease

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.”

Read the full press release here.