Financial Highlights for Full-Year Ended December 31, 2012
2013 Financial Guidance
- Cash, cash equivalents, and marketable securities totaled $99.1 million at December 31, 2012 compared to $55.7 million at December 31, 2011.
- Total revenue was $18.4 million compared to $21.4 million for the full-year 2011. The year-over-year decrease is attributed to a change in revenue recognition accounting under the expanded GlaxoSmithKline (GSK) collaboration.
- Total operating expenses were $71.3 million compared to $72.3 million in the full-year 2011 due to lower research and development expenses as well as a decrease in personnel costs.
- Cash operating expenses net of cash reimbursements received under the GSK collaboration were $40.7 million, within the full-year 2012 guidance range of $37-43 million.
- Net loss was $48.8 million, or $1.07 per share, compared to a net loss of $44.4 million, or $1.28 per share, for the full-year 2011.
As previously announced, Amicus expects full-year 2013 operating expenses to total between $52 million and $58 million, net of cash reimbursements received from GSK. Amicus and GSK are responsible for 40% and 60% of global development costs for migalastat HCl, respectively, in 2013 and beyond. The Company continues to project that the current cash position and anticipated Fabry program reimbursements from GSK are sufficient to fund operations into the second half of 2014.
Migalastat HCl for Fabry Disease
with GSK is developing the investigational pharmacological chaperone migalastat HCl for the treatment of Fabry disease. Amicus has commercial rights to all Fabry products in the United States and GSK has commercial rights to all of these products in the rest of world.
Migalastat HCl Monotherapy
Migalastat HCl monotherapy (150 mg, every-other-day) is being investigated in two ongoing randomized Phase 3 studies for Fabry Disease (
) in patients with genetic mutations identified as amenable to this pharmacological chaperone in a cell-based assay.
Migalastat HCl in Combination with ERT
- Study 011 is comparing migalastat HCl to placebo to potentially support a U.S. marketing application as well as global registration. Results were reported from the 6-month double-blind treatment period (Stage 1) and data from the 6-month open-label follow up period (Stage 2) are anticipated in the third quarter of 2013. The FDA has indicated that it will consider the entirety of the efficacy and safety data from Stage 1 and Stage 2 of Study 011. Following the 12-month results, a meeting is anticipated with the FDA to discuss a U.S. approval pathway for migalastat HCl monotherapy.
- Study 012 is comparing open-label migalastat HCl to current standard of care ERTs (Fabrazyme and Replagal) to support global registration. A total of 60 patients were randomized 1.5:1 to switch from ERT to migalastat HCl or remain on ERT. Data is anticipated in the second half of 2014 on the primary outcome measure, which is renal function assessed by iohexol Glomerular Filtration Rate (GFR) at 18 months.
In combination with ERT, migalastat HCl is designed to bind to and stabilize infused enzyme in any patient receiving ERT for Fabry disease. Amicus and GSK completed an open-label Phase 2 study (
) to investigate 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.
Based on the
from this study, the next chaperone-ERT combination study for Fabry disease is being designed to investigate intravenous treatment of migalastat HCl co-formulated with JCR Pharmaceutical Co. Ltd's proprietary recombinant human alpha-Gal A enzyme (JR-051). Amicus and GSK, in collaboration with JCR, are conducting IND-enabling studies of this chaperone-ERT co-formulated product. An IND submission for this chaperone-ERT co-formulated product is planned by year-end 2013 for entry into the clinic in early 2014.
CHART Programs for Pompe Disease
Outside the collaboration agreement with GSK, Amicus owns exclusive rights to the rest of its pipeline and applications of its CHART platform technology. In chaperone-advanced replacement therapy programs for Pompe disease, the pharmacological chaperone AT2220 is designed to bind to and stabilize human recombinant GAA (rhGAA) enzyme. Amicus is developing AT2220 co-administered with currently marketed ERTs (rhGAA, Myozyme/Lumizyme) in parallel with the development of a next-generation ERT (AT2220 co-formulated with a proprietary rhGAA enzyme). These investigational chaperone-advanced replacement therapies have the potential to increase enzyme activity in muscle and other disease-relevant tissues, improve glycogen reduction, and mitigate immunogenicity compared to Myozyme/Lumizyme alone.
About Amicus Therapeutics
- AT2220-IV Co-Administered with Marketed ERTs: Based on positive results from a Phase 2 co-administration study (Study 010) in Pompe patients, Amicus plans to initiate a repeat-dose clinical study in the third quarter of 2013 to evaluate a novel intravenous formulation of AT2220 (AT2220-IV) co-administered with Myozyme/Lumizyme. The upcoming clinical study will investigate multiple doses of AT2220-IV co-administered with Myozyme/Lumizyme every 2 weeks in treatment-naïve and ERT-experienced Pompe patients to characterize safety, PK, and anti-rhGAA antibody titers.
- Next-Generation ERT (AT2220 Co-Formulated with a Proprietary Amicus ERT): Amicus entered into a contract with Laureate Pharmaceuticals for the manufacture of a proprietary rhGAA enzyme, which is being co-formulated with AT2220 as a next-generation ERT for Pompe disease. Through this investigational chaperone-advanced replacement therapy, Amicus believes it has the potential to improve the properties of the rhGAA enzyme itself while incorporating AT2220 as a small molecule stabilizer to increase exposure and tissue uptake, and reduce immunogenicity relative to currently marketed ERTs. Successful development of a more stable ERT may also enable novel routes of delivery such as subcutaneous administration.
(Nasdaq:FOLD) is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The Company is developing small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of human genetic diseases. Amicus' late-stage programs for lysosomal storage disorders include migalastat HCl monotherapy in Phase 3 for Fabry disease; migalastat HCl co-administered with enzyme replacement therapy (ERT) in Phase 2 for Fabry disease; and AT2220 co-administered with ERT in Phase 2 for Pompe disease.
Chaperone-Advanced Replacement Therapy (CHART)
The Chaperone-Advanced Replacement Therapy (CHART) platform combines unique pharmacological chaperones with enzyme replacement therapies (ERTs) for lysosomal storage diseases (LSDs). Amicus is leveraging the CHART platform to improve currently marketed ERTs through co-administration of a pharmacological chaperone prior to ERT infusion, and to develop next-generation ERTs that consist of a proprietary lysosomal enzyme therapy co-formulated with a pharmacological chaperone. In a chaperone-advanced replacement therapy, a unique pharmacological chaperone binds to a specific therapeutic enzyme, stabilizing the enzyme in its properly folded and active form. This proposed CHART mechanism may allow for enhanced tissue uptake, greater lysosomal activity, more reduction of substrate, and lower immunogenicity compared to ERT alone. Improvements in enzyme stability may also enable more convenient delivery of next-generation ERTs.