Chaperone-Enzyme Replacement Therapy (ERT) Combination Platform Advancing
Expects to End FY 2012 with at Least
Key Highlights and Upcoming Milestones
Second Quarter 2012 Financial Highlights (3 Months Ended
Cash, cash equivalents, and marketable securities totaled
Amicus expects full-year 2012 operating expenses within the higher end of the previously disclosed guidance range of
Chaperone Monotherapy and Chaperone-ERT Combinations for Fabry Disease
Migalastat HCl Monotherapy
Migalastat HCl monotherapy is in Phase 3 development for Fabry disease in patients with genetic mutations that are amenable to chaperone monotherapy. Amicus and GSK are currently conducting Phase 3 global registration studies (Study 011 and Study 012) of migalastat HCl monotherapy.
Study 011 is a randomized, placebo-controlled study with a six-month, double-blind primary treatment period and a six-month, open-label follow-up period. The primary endpoint is interstitial capillary globotriaosylceramide (GL-3) as measured in kidney biopsy. The six-month primary treatment period was completed in a total of 63 patients during the second quarter 2012. These patients received kidney biopsies at baseline and month six. All 63 of these patients are continuing in the six-month follow-up period, and all of these patients are expected to have 12-month kidney biopsies by year-end 2012.
Amicus and GSK have recently engaged in encouraging interactions with the
Study 012 is a randomized, open-label, Phase 3 study targeting approximately 50 total patients (30 to switch to migalastat HCl and 20 to remain on ERT). Final enrollment continues to be expected by year-end 2012.
Patients also continue to receive migalastat HCl monotherapy in Phase 2 and Phase 3 extension studies. As of
Migalastat HCl Co-Administered with ERT
When co-administered with ERT, migalastat HCl is designed to bind to and stabilize the infused enzyme, independent of alpha-Gal A mutation type. An open-label Phase 2 study (Study 013) is currently underway to investigate the effects of a single oral dose of migalastat HCl (150 mg or 450 mg) co-administered prior to ERT (Fabrazyme or Replagal) in males with Fabry disease. Results are expected to be presented at a Fall 2012 scientific congress.
Migalastat HCl Co-Formulated with Preclinical Proprietary ERT
Under the expanded Fabry collaboration, Amicus and GSK in collaboration with
Chaperone-ERT Combinations Programs for Additional Lysosomal Storage Diseases
Amicus and GSK are co-developing all formulations of migalastat HCl for Fabry disease. Outside the GSK collaboration, Amicus owns exclusive rights to the rest of its pipeline and applications of its platform technology.
Preclinical chaperone-ERT co-administration studies in animal models of Fabry, Pompe and Gaucher have shown that a pharmacological chaperone can selectively bind to and stabilize the enzyme, prevent deactivation in the circulation, and increase uptake of active enzyme into key tissues of disease. In published studies in Fabry1 and Pompe2 animal models, chaperone-ERT co-administration has also led to greater substrate reduction compared to ERT alone.
During the second quarter the Company announced positive preliminary results from the two lowest dose cohorts in a Phase 2 open-label study (Study 010) to investigate four ascending dose cohorts of the pharmacological chaperone AT2220 co-administered with ERT for Pompe disease. Additional results are anticipated at a Fall 2012 scientific congress.
In parallel with Study 010, Amicus is conducting in vitro studies using
Gaucher Disease: Preclinical Chaperone-ERT Combinations
In Gaucher disease, Amicus is continuing preclinical studies to evaluate two pharmacological chaperones, AT2101 (afegostat tartrate) and AT3375, in combination with ERT (beta-glucosidase). Both of these chaperones target the glucocerobrosidase (GCase) enzyme. Inherited genetic mutations in the GBA1 gene, which encodes for the GCase enzyme, are the cause of Gaucher disease.
Parkinson's Disease in Gaucher Carriers: Preclinical Chaperone Monotherapy
Over the last decade, GBA1 mutations have been identified as the most common genetic risk factor for Parkinson's. By targeting GCase in the brain, AT3375 could potentially treat Gaucher, Parkinson's disease in Gaucher carriers, and possibly the general Parkinson's population. By year-end 2012, Amicus expects to complete additional preclinical and IND-enabling studies of AT3375, which are supported in part by a grant from the
Conference Call and Webcast
An audio/visual webcast can also be accessed via the Investors section of the
The slide presentation for today's conference call and webcast is also available in the Investors section of the
A telephonic replay of the call will be available for seven days beginning at
About Fabry Disease
Fabry disease is an inherited lysosomal storage disease that is currently estimated to affect approximately 5,000 to 10,000 people worldwide. Fabry Disease is caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A). The role of alpha-Gal A within the body is to break down a complex lipid called globotriaosylceramide (GL-3). Reduced or absent levels of alpha-Gal A activity leads to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys, and skin. This accumulation of GL-3 is believed to cause the various symptoms of Fabry disease, including pain, kidney failure, and increased risk of heart disorders and stroke.
About Pompe Disease
Pompe disease is a lysosomal storage disease characterized by progressive skeletal muscle weakness and respiratory insufficiency. It is caused by a deficiency in lysosomal alpha-glucosidase (GAA) activity, which leads to accumulation of glycogen in tissues affected by the disease (primarily muscle). Pompe disease affects an estimated 5,000 to 10,000 individuals worldwide and is clinically heterogeneous in the age of onset, the extent of organ involvement, and the rate of progression.
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, and the projected cash position for the Company. 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 goals, progress, timing and outcomes of discussions with regulatory authorities and the potential goals, progress, timing and results of preclinical studies and 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 that results of clinical or pre-clinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities may not grant or may delay approval for our product candidates; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we will need additional funding to complete all of our studies and, our dependence on third parties in the conduct of our clinical studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding projections of the Company's cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2011. 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.
|(a development stage company)|
|Consolidated Statements of Operations|
|(In thousands, except share and per share amounts)|
|Three Months||Six Months||to|
|Research revenue||$ 2,380||$ 5,477||$ 6,686||$ 11,591||$ 57,493|
|Collaboration and milestone revenue||1,660||5,160||3,320||6,820||64,382|
|Research and development||11,618||13,723||22,743||27,727||293,347|
|General and administrative||6,720||5,819||11,122||9,914||123,163|
|Impairment of leasehold improvements||--||--||--||--||1,030|
|Depreciation and amortization||426||442||864||862||10,925|
|In-process research and development||--||--||--||--||418|
|Total operating expenses||18,764||19,984||34,729||38,503||430,405|
|Loss from operations||(14,724)||(9,347)||(24,723)||(20,092)||(308,530)|
|Other income (expenses):|
|Change in fair value of warrant liability||2,078||(118)||(1,354)||(2,494)||(1,594)|
|Loss before tax benefit||(12,641)||(9,343)||(25,991)||(22,480)||(298,047)|
|Benefit from income taxes||--||--||--||--||5,463|
|Preferred stock accretion||--||--||--||--||(802)|
|Net loss attributable to common stockholders||
||$ (25,991)||$ (22,480)||$ (312,810)|
|Net loss attributable to common stockholders per common share — basic and diluted||$ (0.37)||$ (0.20)||$ (0.75)||$ (0.53)|
|Weighted-average common shares outstanding — basic and diluted||34,530,693||46,870,067||34,514,947||42,103,642|
Sara Pellegrinospellegrino@amicusrx.com (609) 662-5044
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