Next-Generation ERT (AT2220 Co-Formulated with a Proprietary Amicus ERT)
Preclinical studies of AT2220 co-formulated with rhGAA enzyme (Myozyme/Lumizyme) were presented for the first time in a
at LDN WORLD. This chaperone-ERT co-formulation resulted in up to 2.5-fold greater enzyme uptake in multiple disease-relevant tissues and led to greater glycogen reduction compared to rhGAA alone in GAA knock-out mice. Collectively these data suggest that AT2220 directly binds to and stabilizes rhGAA, potentially leading to a larger amount of properly folded, active enzyme available for uptake into tissue. AT2220 co-formulated with ERT may also mitigate Pompe ERT-related immunogenicity since properly-folded proteins are less prone to aggregation and less immunogenic.
Following the completion of these preclinical studies, Amicus entered into a contract with Laureate Pharmaceuticals for the manufacture of a proprietary rhGAA enzyme. Amicus is developing AT2220 co-formulated with this proprietary enzyme as a next-generation ERT for Pompe disease. Through this approach Amicus believes it has the potential to improve the properties of the rhGAA enzyme itself while incorporating AT2220 as a small molecule stabilizer to increase exposure and tissue uptake, and reduce immunogenicity relative to currently marketed ERTs. Successful development of a more stable ERT may also enable novel routes of delivery such as subcutaneous administration.
Conference Call and Webcast
John F. Crowley, Chairman and Chief Executive Officer, and members of the Amicus executive team will host a conference call and live audio/visual webcast on Friday, February 15, 2013 at 11:30am ET to discuss data from several programs presented at LDN WORLD. Interested participants and investors may access the conference call at 11:30 a.m. ET by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international). A live audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at
, and will be archived for 30 days. The slide presentation for the conference call/webcast will also be available at
. Web participants are encouraged to go to the web site 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 2:30 p.m. ET on February 15, 2013. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); participant code 97505816.
About Amicus Therapeutics
(Nasdaq:FOLD) is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The Company is developing orally-administered, small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of human genetic diseases. Amicus' late-stage programs for lysosomal storage disorders include migalastat HCl monotherapy in Phase 3 for Fabry disease; migalastat HCl co-administered with enzyme replacement therapy (ERT) in Phase 2 for Fabry disease; and AT2220 co-administered with ERT in Phase 2 for Pompe disease.
About AT2220 for Pompe Disease
AT2220 is an investigational, orally-administered pharmacological chaperone owned exclusively by Amicus. In published preclinical studies, AT2220-ERT co-administration resulted in significant increases in muscle rhGAA levels and decreases in glycogen levels in a mouse model of Pompe disease. Preclinical results to date also suggest that AT2220-ERT co-administration may mitigate ERT-induced immunogenicity by stabilizing the enzyme in its properly folded and active form. Initial
studies using T cells derived from blood from 50 healthy donors demonstrated that the addition of AT2220 may significantly reduce the immunogenicity of Myozyme and Lumizyme.
Pompe disease is a lysosomal storage disease characterized by progressive skeletal muscle weakness and respiratory insufficiency. It is caused by a deficiency in GAA activity, which leads to accumulation of glycogen in tissues affected by the disease (primarily muscle). Pompe disease affects an estimated 5,000 to 10,000 individuals worldwide and is clinically heterogeneous in the age of onset, the extent of organ involvement, and the rate of progression.