International Pompe Association

You are here: Home News Amicus Therapeutics Announces Full-Year 2013 Financial Results and Corporate Updates

Amicus Therapeutics Announces Full-Year 2013 Financial Results and Corporate Updates - Executing 3-in-3 Strategy to Advance 3 Next-Generation ERTs into Clinic in Next 3 Years - Reiterating FY14 Cash Spend Guidance of $54-$59 Million

Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced financial results for the full-year ended December 31, 2013. The Company also provided program updates and reiterated full-year 2014 operating expense guidance.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, "During 2013 we focused on strengthening our biologics business strategy to develop next-generation ERTs for patients with lysosomal storage diseases. Through our purchase of Callidus Biopharma, we have acquired a proprietary Pompe ERT as well as a peptide tagging technology that is complementary to our CHART platform. We believe that these technologies together provide a unique tool set to enhance enzyme activity, increase enzyme uptake into tissues, and potentially address the tolerability and immunogenicity associated with current ERTs. During 2014 we are strongly positioned and well capitalized to execute our 3-in-3 strategy to advance three next-generation ERTs into the clinical in the next three years, with lead programs in Fabry, Pompe and MPS I." ...

Next-Generation ERT for Pompe Disease

Amicus is advancing a recombinant human acid alpha-glucosidase (rhGAA) for Pompe disease into late preclinical development. The Company's acquisition of Callidus Biopharma, brings a differentiated Pompe ERT, designated AT-B200, with a unique carbohydrate structure. In preclinical studies AT-B200 has shown superior tissue uptake and activity when compared to current standard of care. This ERT may be further optimized through co-formulation with Amicus' pharmacological chaperone AT2220 to improve enzyme stability and tolerability, and by applying the Company's peptide tagging technology for better targeting...

Read more

 


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.