Fourth Quarter and Full-Year 2010 Financials Summary
Amicus announced net loss attributable to common stockholders of
"We have started 2011 on a very positive note and intend to build on our momentum through the achievement of multiple key milestones across all of our programs during the year," said
2011 Key Milestones
In 2011 Amicus expects to achieve multiple key milestones across its three areas of focus: Amigal (migalastat HCl) for the treatment of Fabry Disease, the evaluation of pharmacological chaperones co-administered with enzyme replacement therapy (ERT), and the investigation of pharmacological chaperones for the treatment of diseases of neurodegeneration. Among the milestones expected this year are results from the following studies:
Amigal™ (migalastat HCl) for the Treatment of Fabry Disease
The Phase 3 study (Study 011) of migalastat HCl remains the Company's number one priority. Study 011 is ongoing and patients are being enrolled at 36 investigational sites worldwide. A majority of the planned 60 patients have been enrolled in the study. The Company expects to complete enrollment in the first half of 2011 and to report preliminary results from this study in the second half of the year.
Amicus and GSK intend to commence an additional Phase 3 study (Study 012) in the first half of 2011. Study 012 will be an 18-month, randomized, open-label study comparing migalastat HCl to enzyme replacement therapy (ERT) in approximately 60 subjects. The primary outcome of efficacy will be renal function as measured by glomerular filtration rate (GFR).
Seventeen subjects continue to receive treatment in the ongoing Phase 2 long-term extension study. Amicus plans to provide a data update from this study at the Lysosomal Disease Network WORLD Symposium in
Chaperone-ERT Combination Therapy
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. Preclinical proof of concept has been established for Fabry disease and Pompe disease. Amicus plans to present a review of historical and new data from these studies at the Lysosomal Disease Network WORLD Symposium in
Amicus and its partner GSK are sponsoring an ongoing Phase 2 study evaluating the co-administration of migalastat HCl with ERT for Fabry disease. Results from this study are expected in the second half of 2011.
Additionally, the Company expects to initiate a Phase 2 study with its pharmacological chaperone AT2220 co-administered with ERT for Pompe disease in the first half of 2011 and expects results from this study to be available in the second half of the year. The Company intends to seek U.S.
Diseases of Neurodegeneration
Amicus is investigating the potential use of pharmacological chaperones for the treatment of genetically defined sub-populations of patients with Parkinson's Disease and Alzheimer's Disease. Amicus previously reported encouraging results from preclinical studies evaluating the use of a pharmacological chaperone for the treatment of Parkinson's Disease. In 2011 it expects to complete late-stage preclinical proof of concept studies, including IND-enabling activities, for its molecule AT3375, which is in development for the treatment of Parkinson's Disease. The Amicus Parkinson's Disease program is funded in part by a grant from
Additionally, Amicus continues to advance its preclinical program evaluating a pharmacological chaperone approach for the treatment of Alzheimer's disease. The Company expects to continue preclinical proof of concept studies during 2011. The Amicus Alzheimer's Disease program is funded in part by a grant from the Alzheimer's
Amicus plans to present new information from both its Parkinson's Disease and Alzheimer's Disease programs at the Lysosomal Disease Network WORLD Symposium in
2011 Financial Guidance
As previously reported, the Company expects to spend a total of
In 2011, the Company will continue to evaluate additional business development opportunities to further build shareholder value. The Company is actively exploring a range of opportunities with multiple potential partners.
Additional Financial Results & Notes
Net loss attributable to common stockholders for the three months ended
Upon the signing the GSK collaboration agreement, Amicus received an upfront payment of
Conference Call and Webcast
An audio webcast and archive can also be accessed via the investor section of the Amicus Therapeutics Web site at http://www.amicustherapeutics.com under Investors: Events and Presentations. 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. After the live webcast, a webcast replay will remain available in the Investors section of the Amicus Therapeutics Web site for 30 days.
A telephonic replay of the call will be available for seven days beginning at
Amicus' press releases are available at www.amicustherapeutics.com.
This press release contains, and the accompanying conference call will contain, "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of Amicus' candidate drug products, the timing and reporting of results from preclinical studies and clinical trials evaluating Amicus' candidate drug products, the projected cash position for the Company, including achievement of development and commercialization milestone payments and sales royalties under our collaboration with
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Statements of Operations
(In thousands, except share and per share amounts)
February 4, 2002
Ended December 31,
Ended December 31,
Research and development
General and administrative
Impairment of leasehold improvements
Depreciation and amortization
In-process research and development
Total operating expenses
Income/(loss) from operations
Other income (expenses):
Change in fair value of warrant liability
Income/(loss) before tax benefit
Benefit from income taxes
Preferred stock accretion
Net Income/(loss) attributable to common stockholders
Net Income/(loss) attributable to common stockholders per
common share — basic
Weighted-average common shares
outstanding — basic
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