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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Disclaimer: The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.

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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Disclaimer: The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.

CRANBURY, N.J. and SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) --  Amicus Therapeutics (Nasdaq:FOLD)... today presented new scientific findings and preclinical data on functional outcomes in an oral presentation and 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 in muscles, co-administered with AT2221, a pharmacological chaperone designed to stabilize ERT in circulation. 

In previously reported preclinical studies, ATB200 was associated with increased tissue enzyme levels and reduced glycogen levels in muscle, which was further improved when AT2221 was co-administered with ATB200. For the first time at WORLDSymposium, Hung Do, PhD, Chief Science Officer of Amicus Therapeutics, presented results from preclinical studies that showed ATB200/AT2221 reversed cellular dysfunction and progressively increased muscle strength following every-other-week administration at 20 mg/kg over a five month period in an animal model of Pompe disease (GAA knock-out mice)...

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Disclaimer: The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.