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)...

Read more

• Press release (external link)
• Press release (PDF, 24 kB)

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.

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

CRANBURY, N.J., Dec. 08, 2016 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced positive preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221. 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...

Read more

• Press release (external link)
• Press release (PDF, 25 kB)

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.

At the "Steps Forward in Pompe Disease" 8th European Symposium on November 11-12, 2016 in Amsterdam (NL), organised by Sanofi Genzyme, one session was dedicated to a review of 10 years of enzyme replacement therapy. A company perspective was presented by Henk Schuring, Vice President and Head of Rare Neurological Diseases (Sanofi Genzyme), a doctor's perspective was given by Ans van der Ploeg (Erasmus MC Rotterdam, The Netherlands), and insights into the patient community perspectives were given in a speech delivered by Tiffany House, President of the IPA. We want to share this speech with the Pompe Community:

Past, Present and Future

2016 has been an interesting year for the Pompe Community. It is the ten-year anniversary of the approval of a treatment for Pompe by the EMA and FDA: a milestone that should be celebrated. The majority of patients around the world now have access to treatment. The IPA recognizes that this treatment is not a cure, and its effectiveness varies for patients. But what we know without a doubt is that it is an important first step in the treatment of Pompe Disease. The natural history of Pompe disease is clear: patients will exhibit progressive muscle weakness. What is also clear after ten years of commercial treatment, and seven years of clinical trials before that, is that enzyme replacement therapy (ERT) slows or halts the disease process for most patients.

The ten-year anniversary of the approval of ERT is a chance to look back at how far we have come, and also to look towards the future and where we want to go. I think to truly understand where we are today, we need to go back even further than the last 10 years. We need to take a moment to remember the days and months and years before treatment was a reality. Before there were promising trials in the works.