AT2220 (1-deoxynojirimycin HCl) for the treatment of Pompe Disease
Amicus continued to make progress throughout the fourth quarter with its ongoing Phase 2 clinical trial of AT2220 in adult Pompe patients. The trial includes an 11-week treatment period with an optional extension study. The objectives of the trial include the evaluation of the safety and pharmacodynamics of multiple doses and regimens of AT2220. The results of this study are expected to be available in the second half of 2009.
In addition, Amicus is continuing to conduct preclinical animal studies to evaluate the effects of administering AT2220 in combination with ERT. Results announced at the American Society of Human Genetics conference in November 2008 indicate that AT2220 in combination with ERT has the potential to increase the stability and tissue uptake of ERT. Amicus is conducting additional preclinical proof-of-concept studies to determine the feasibility of a combination that may be appropriate for Pompe patients who are not amenable to chaperone monotherapy. Amicus expects to discuss additional results at relevant scientific conferences during 2009.
Preclinical Chaperone Programs
Amicus continues to invest in research and development to assess the potential for applying its versatile chaperone technology platform to the treatment of a broader range of human genetic diseases. As part of this effort, Amicus continues to conduct preclinical studies in Parkinson's disease and is investing in new research aimed at evaluating disease targets for other neurodegenerative and genetic disorders.
2009 Financial Guidance
In 2009, Amicus expects to report approximately $70 million in operating expenses, which it expects to be offset by approximately $50 million in program cost-sharing reimbursements and clinical mil
|SOURCE Amicus Therapeutics|
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