Amicus Therapeutics Announces Successful Completion of End of Phase 2 Meeting With FDA for Amigal in Fabry Disease
Agency Supports Amigal Move to Phase 3; Eligible for Accelerated Approval; Final Global Regulatory Plan Expected by Year End
CRANBURY, N.J., August 04, 2008 /PRNewswire-FirstCall/ -- Amicus Therapeutics , a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases, announced today that it has successfully completed an End of Phase 2 meeting for Amigal(TM) with the U.S. Food and Drug Administration (FDA). The FDA indicated that the data from the completed Phase 2 clinical studies of Amigal support the start of Phase 3 development and agreed that Amigal meets the criteria to be considered for accelerated approval. The Agency further indicated that it is not opposed to the use of a surrogate primary endpoint, pending further discussion and final agreement on the Phase 3 trial design.
Amicus, along with its partner Shire Human Genetic Therapies, Inc. ("Shire"), is engaged in ongoing discussions with the FDA and the European Medicines Agency (EMEA) regarding plans for a global Phase 3 clinical development program for Amigal. In line with previous guidance, Amicus expects to complete these interactions in the second half of 2008, and subject to the outcome of the discussions, the Company plans to initiate Phase 3 development of Amigal in the first half of 2009.
"We are very pleased with the outcome of the End of Phase 2 meeting with the FDA," said John F. Crowley, President and CEO of Amicus Therapeutics. "We look forward to continuing our work with the FDA and EMEA to design a global Phase 3 program for Amigal in Fabry disease."
The meeting followed the successful conduct of Phase 2 clinical studies in patients with Fabry disease that showed that Amigal was generally safe and well-tolerated at all doses evaluated. In a majority of patients, treatment with Amigal resulted in increased activity of the enzyme deficient in Fabry patients (alpha-GAL) and a reduction of kidney GL-3 as measured in urine.
In parallel with the regulatory process, 23 of the original 26 patients will continue to be treated with Amigal in a voluntary Phase 2 extension study to monitor long term safety and efficacy and to evaluate additional doses and dose regimens. Data from this extension study are expected to be available by Q1 2009. In addition, Amicus expects to conduct clinical pharmacology studies to support the Phase 3 program.
Amicus is developing Amigal as part of a strategic collaboration with Shire Human Genetic Therapies, Inc., a wholly-owned subsidiary of Shire Limited, to develop and commercialize Amicus' three lead pharmacological chaperone compounds for lysosomal storage disorders. Under the agreement, Shire received commercial rights outside of the United States. Amicus retains all U.S. rights.
Amigal (migalastat hydrochloride) is an experimental, oral therapy for the treatment of Fabry disease, a lysosomal storage disorder, which can cause damage to specific areas of the body, including the kidneys, heart, nervous system, and skin. Amigal, a pharmacological chaperone, acts by selectively binding to the misfolded enzyme responsible for Fabry disease, alpha-GAL. After binding to the enzyme, Amigal promotes the proper folding, processing, and trafficking of the enzyme from the endoplasmic reticulum to its final destination, the lysosome, the area of the cell where the enzyme does its work. Once it reaches the lysosome, the pharmacological chaperone is displaced, and the enzyme can perform its normal, biological function, which is the breakdown of its natural substrate, GL-3.
About Amicus Therapeutics
Amicus Therapeutics is a biopharmaceutical company developing novel, oral therapeutics known as pharmacological chaperones for the treatment of a range of human genetic diseases. Pharmacological chaperone technology involves the use of small molecules that selectively bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. Amicus has completed Phase 2 clinical trials of Amigal for the treatment of Fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for the treatment of Gaucher disease and AT2220 for the treatment of Pompe disease.
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "targets," "likely," "will," "would," "should" and "could," and similar expressions or words identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. The inclusion of forward-looking statements should not be regarded as a representation by Amicus that any of its plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong. They can be affected by inaccurate assumptions Amicus might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the progress, timing and outcomes of ongoing discussions with regulatory authorities and the potential progress, timing and results of clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in the business of Amicus, including, without limitation: the potential inability to reach final agreement with regulatory agencies on the use of a surrogate endpoint and phase 3 trial design for Amigal, the potential that results of clinical or pre-clinical studies indicate that the product candidates are unsafe or ineffective; and, our dependence on third parties in the conduct of our clinical studies; further, the results of earlier clinical trials may not be predictive of future results; and other risks detailed in our annual Report on Form 10-K for the year ended December 31, 2007, and our other public filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and Amicus undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
CONTACT: John Quirk of Porter Novelli Life Sciences, +1-212-601-8296, forAmicus Therapeutics
Web site: http://www.amicustherapeutics.com/
Ticker Symbol: (NASDAQ-NMS:FOLD)
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Posted: August 2008