Amicus Therapeutics Announces FDA Agreement to Commence Phase 2 Study of AT2220 Co-administered with Enzyme Replacement Therapy for Pompe Disease
CRANBURY, N.J., March 8, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold for the AT2220 (1-deoxynojirimycin HCI) Investigational New Drug Application (IND). AT2220 is a pharmacological chaperone in development as a treatment for Pompe disease. Based on data provided by Amicus, the FDA agreed with the Company's proposal to resume clinical development of AT2220, starting with a new Phase 2 study designed to evaluate the use of AT2220 when co-administered with enzyme replacement therapy (ERT) in subjects with Pompe disease. The Company expects to commence this study in the first half of 2011 and to report preliminary results in the second half of 2011.
Co-administration of Pharmacological Chaperones with ERT
Amicus previously reported promising preclinical data demonstrating that the co-administration of a pharmacological chaperone with ERT has the potential to address key limitations of ERT. The addition of a pharmacological chaperone has been shown to prevent the loss of activity of ERT in the circulation, increase tissue uptake, and increase substrate reduction in multiple disease-relevant tissues. Preclinical proof of concept has been established for Fabry disease and Pompe disease.
Data from Phase 1 studies in 72 healthy volunteers demonstrated that AT2220 was generally safe and well tolerated at all doses evaluated with no drug-related serious adverse events. Based on these data and encouraging safety data from preclinical studies, Amicus initiated a Phase 2 clinical trial of AT2220 as a monotherapy treatment in adults with Pompe disease. The protocol involved initial treatment with a high dose of AT2220. Two patients enrolled in the trial experienced adverse events categorized as serious and probably related to treatment with AT2220, and as a result the IND was placed on clinical hold.
Amicus completed a thorough investigation of the events, including the completion of additional preclinical and Phase 1 studies. As a result the Company decided to continue development of AT2220 co-administered with ERT but not as a monotherapy.
About Pompe Disease
Pompe disease affects an estimated 5,000-10,000 individuals world-wide and is clinically heterogeneous in the age of onset, the extent of organ involvement, and the rate of progression. The early onset form of the disease is the most severe, progresses most rapidly, and is characterized by musculoskeletal, pulmonary, gastrointestinal, and cardiac symptoms that usually lead to death from cardio-respiratory failure between 1 and 2 years of age. The late onset form of the disease begins between childhood and adulthood and has a slower rate of progression that is characterized by musculoskeletal and pulmonary symptoms that usually lead to progressive muscle weakness and respiratory insufficiency. A high majority of patients have the late onset form of the disease. The U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation for the active ingredient in AT2220 in the United States.
About Amicus Therapeutics
Amicus Therapeutics is a biopharmaceutical company focused on developing treatments for rare 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 diseases including lysosomal storage disorders and diseases of neurodegeneration. Amicus' lead program, Amigal (migalastat HCl) is in Phase 3 development for the treatment of Fabry disease and is partnered with GSK Rare Diseases.