Key Developments For Amicus Therapeutics, Inc.
Amicus Therapeutics, Inc. (FOLD.O) (Consolidated Issue listed on NASDAQ Global Market)
Amicus Therapeutics, Inc. Announces Resignation Of James E. Dentzer As CFO-Form 8-K
Amicus Therapeutics, Inc. reported in its Form 8-K that in connection with the reduction in workforce, the Company's Chief Financial Officer (CFO), James E. Dentzer, is leaving the Company effective as of October 30, 2009.
Amicus Therapeutics, Inc. Announces Preliminary Results of Phase 2 Study with Plicera for Gaucher Disease
Amicus Therapeutics, Inc. announced preliminary results from its Phase 2 randomized, open-label study to assess the safety, tolerability and preliminary efficacy of its investigational drug, Plicera (afegostat tartrate), in treatment-naive adult patients with type 1 Gaucher disease. Two dose regimens of Plicera (225 mg three days on/four days off and seven days on/seven days off) were studied during this six month trial. While all patients enrolled experienced an increase in the level of the target enzyme (GCase) as measured in white blood cells, clinically meaningful improvements in key measures of disease were observed in just one of the eighteen patients who completed the study. The preliminary results suggest that treatment with Plicera was generally well tolerated, with no serious adverse events (SAEs) reported. Nineteen subjects were enrolled and 18 subjects completed the study. One subject discontinued treatment because of an adverse event (conjunctivitis-related symptoms). Once the data are final, the Company plans to further analyze and evaluate the results in collaboration with its partner, Shire Human Genetic Therapies, Inc. (Shire HGT), and, based on this work, will determine the appropriate next steps for the Plicera program. However, based on these preliminary results, the Company does not expect to advance Plicera into Phase 3 development at this time.
Amicus Therapeutics, Inc. Announces Plan To Initiate Phase 1 Study Of AT2220 For Pompe Disease
Amicus Therapeutics, Inc. announced it plans to initiate a Phase 1 study of AT2220 (1-deoxynojirimycin HCl), its investigational drug in development for the treatment of Pompe Disease. The primary objective of this study is to evaluate the pharmacokinetics of AT2220 in muscle tissue in healthy adult subjects. The U.S. Food and Drug Administration (FDA) has agreed to Amicus' proposal for the Phase 1 study and subsequently converted the clinical hold of AT2220 to a partial hold to allow the conduct of this study. announced it plans to initiate a Phase 1 study of AT2220 (1-deoxynojirimycin HCl), its investigational drug in development for the treatment of Pompe Disease. The primary objective of this study is to evaluate the pharmacokinetics of AT2220 in muscle tissue in healthy adult subjects. The U.S. Food and Drug Administration (FDA) has agreed to Amicus' proposal for the Phase 1 study and subsequently converted the clinical hold of AT2220 to a partial hold to allow the conduct of this study. One of the recently completed preclinical studies evaluated the effects of various doses and regimens of AT2220 on glycogen reduction in an appropriate transgenic mouse model of Pompe disease. Preliminary results of this study demonstrate consistent glycogen reduction across a range of different tissues, including heart, diaphragm and multiple skeletal muscles. The Company expects to present the results of this and other preclinical studies at appropriate scientific conferences in 2010.
Amicus Therapeutics, Inc. Commences Phase III Trial Evaluating Amigal For Treatment Of Fabry Disease
Amicus Therapeutics, Inc. announced that it has commenced the U.S. registration Phase III trial with its investigational drug, Amigal (migalastat hydrochloride) for the treatment of Fabry disease. The Company has reached agreement with the U.S. Food and Drug Administration (FDA) on the key protocol design elements of the pivotal trial, including the use of the surrogate primary endpoint of the change in the amount of kidney interstitial capillary GL-3, the substrate that accumulates in the cells of Fabry patients. In addition, the FDA is in agreement that the Company is eligible to seek Accelerated Approval for Amigal according to Subpart H regulations. The Company has begun submitting the Phase III protocol to investigational sites worldwide and expects to begin the dosing of subjects in the second half of this year.
Amicus Therapeutics, Inc. Files $100 Million Mixed Securities Shelf- DJ
Dow Jones reported that Amicus Therapeutics, Inc. filed with the Securities and Exchange Commission to sell $100 million in mixed securities from time to time. The mixed securities include common and preferred stock, warrants and debt securities. The Company said it will use proceeds from the sale for general corporate purposes, which may include working capital, capital expenditures, research-and-development expenditures, clinical trial expenditures, commercial expenditures, acquisitions of new technologies or businesses, and investments. The Company will not receive any proceeds from the sale of any securities offered by any selling stockholder.
