CRANBURY, N.J. and SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD)… today presented additional positive preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in a poster at the 13th Annual WORLDSymposium™ in San Diego, CA. 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.
“We are very pleased to share these additional positive preliminary clinical results here at the WORLDSymposium as we continue with the cascade of data from this important study in Pompe Disease patients,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “In addition to excellent continued safety, tolerability and PK profile, we now see evidence that this novel Amicus treatment paradigm is further reducing important biomarkers of disease in both ERT-switch patients as well as in the first ERT-treatment naïve patients. In particular, demonstrating with these data that we can further reduce these biomarkers in a switch population is very encouraging. To our knowledge, there are no published data for other Pompe ERTs where these types of reductions in biomarkers have been shown. Moreover, the fact that these biomarkers have demonstrated a sustained reduction in a majority of patients over the first 18 weeks suggests that the effects of our approach may indeed be persistent and durable. We look forward to data in more patients and over longer periods of time in the months ahead, including important measures of muscle function in these patients. We could not be more pleased with the preliminary data to date.”…
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