Program Update for Phase 1/2 Pompe Study by Amicus Therapeutics

Category: Archive
March 2, 2017

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

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