CRANBURY, N.J., March 01, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD)... provided program updates. John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. states that "establishing our novel Pompe treatment paradigm ATB200/AT2221 as a highly differentiated therapy" is one of five key strategic priorities...
Program Update: ATB200/AT2221 for Pompe Disease
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. Positive preliminary data were reported in the fourth quarter of 2016 and during the 13th Annual WORLDSymposium™ in San Diego, CA in February 2017 from a global clinical study (ATB200-02) to evaluate safety, tolerability, PK, and pharmacodynamics (PD) of ATB200/AT2221. The study is enrolling 3 cohorts of patients, including ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-naïve patients (Cohort 3).
Key Preliminary Data Highlights from ATB200-02 Study in Initial ERT-Switch and ERT-Naïve Patients:
- No infusion-associated reactions following 150+ infusions in initial patients treated for a maximum of 36 weeks (n=13)
- Available PK and PD (muscle and glycogen biomarkers) data through week 18 in eight initial ERT-switch patients and two ERT-naïve showed:
- The desired PK profile
- Improvements in key muscle damage biomarkers (creatine kinase (CK) enzyme, alanine aminotransferase (ALT), and aspartate aminotransferase (AST)) in a majority of ERT-switch patients and both ERT-naïve patients
- Reductions in a biomarker of glycogen substrate urine hexose tetrasaccharide (Hex4) in all patients
- Target enrollment achieved across all patient cohorts
Anticipated Upcoming Pompe Disease Program Milestones:
- ATB200-02 study data in additional naïve and non-ambulatory patients, as well as extension-phase data on all patient cohorts, in the second and third quarter of 2017
- Meetings with US and EU regulators
- Press release (external link)
- Press release (PDF, 42 kB)
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