Amicus Offers FY13 Guidance, Update on Strategic Outlook
Amicus Therapeutics (Nasdaq: FOLD), a biopharmaceutical company at the forefront of developing therapies for rare and orphan diseases, today provided its full-year 2013 strategic outlook and financial guidance. John F. Crowley, Chairman and CEO of Amicus, will discuss Amicus' corporate objectives and key milestones in a presentation at the 31st Annual J.P. Morgan Healthcare Conference on Wednesday, January 9, 2013 at 3 p.m. PT (6 p.m. ET). A live webcast of the presentation can be accessed through the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicustherapeutics.com/events.cfm, and will be archived for 90 days.
Mr. Crowley stated, "We have a great 2013 ahead of us. The Stage 1 data from our Fabry monotherapy Study 011 are certainly encouraging. We look forward to presenting additional detail about this 6-month data from Study 011 at the WORLD Symposium in February. We also look forward to receiving and analyzing the 12-month efficacy and safety data from Stage 2 of Study 011 in the second quarter of this year. With these data, we expect to engage in constructive discussions with the FDA regarding a U.S. approval pathway for migalastat HCl as the first orally available pharmacological chaperone monotherapy for Fabry disease. Additionally, our Pompe program continues to provide excellent data demonstrating positive effects of AT2220-ERT co-administration, especially regarding the potential to mitigate the immune response to ERT as shown in preclinical studies. Finally, we continue to advance our chaperone-ERT co-formulated products. We believe that these next-generation ERTs have the potential to transform the treatment paradigm for many lysosomal storage diseases."
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. Amicus expects full-year 2013 net cash spend between $52 million and $58 million. The current cash position and anticipated Fabry program reimbursements from GSK are projected to fund operations into the second half of 2014.
Amicus and GSK are co-developing all formulations of migalastat HCl under a global Fabry collaboration. 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. Amicus and GSK are responsible for 40% and 60% of global development costs, respectively, in 2013 and beyond. Outside the GSK collaboration, Amicus owns exclusive rights to the rest of its pipeline and applications of its platform technology.
Amicus is leveraging its pharmacological chaperone technology platform to develop next-generation medicines for a range of rare and orphan diseases, with a focus on improved therapies for lysosomal storage disorders. During 2013, Amicus and GSK are committed to advancing migalastat HCl for Fabry disease. Amicus will also continue to advance its pharmacologic chaperone technology platform to develop next-generation therapies (ERTs) for Pompe and additional lysosomal storage disorders. These programs include novel small molecules co-administered with existing enzyme replacement products, as well as proprietary next-generation enzyme replacement therapies that are co-formulated with pharmacologic chaperones.
Migalastat HCl Monotherapy for Fabry Disease
Migalastat HCl monotherapy (150 mg, every-other-day (QOD)) is in two randomized ongoing Phase 3 studies for Fabry Disease (Study 011 and Study 012) in patients with genetic mutations identified as amenable to this pharmacologic chaperone in a cell-based assay.
* Study 011 is comparing migalastat HCl to placebo to potentially support a U.S. marketing application. In December 2012, Amicus and GSK announced encouraging top-line Stage 1 results from the 6-month double-blind treatment period in Study 011 (Stage 1). Additional 6-month data will be presented at the Lysosomal Disease Network WORLD Symposium (LDN WORLD), to be held February 12-15, 2013, in Orlando, Florida. Data from the 6-month open-label follow up period in Study 011 (Stage 2) in which all patients received migalastat HCl are anticipated in the first half of 2013. These results will include 12 months of data for the migalastat HCl group and 6 months of data for the group that crossed over from placebo to migalastat HCl. 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. Amicus and GSK expect to meet with the FDA in 2013 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. In December 2012, this study achieved full enrollment of 60 patients, who 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.
AT2220 Co-Administered with Marketed ERTs for Pompe Disease
In January 2013, Amicus announced positive results from all four patient cohorts in a Phase 2 study (Study 010) to evaluate the safety and pharmacokinetic (PK) effects of the pharmacological chaperone AT2220 co-administered with the standard of care ERTs for Pompe disease (Myozyme^®/Lumizyme^®, or recombinant GAA enzymes). Results from this study established human proof-of-concept that AT2220-ERT co-administration increases GAA enzyme activity in muscle, particularly at the fourth and highest dose cohort of AT2220 (600 mg). Based on these results, Amicus plans to conduct a repeat-dose clinical study to investigate the effect of AT2220-ERT co-administration on ERT stability and activity, ERT-related immunogenicity, and other clinical measures. This study is expected to begin in the third quarter of 2013.
By stabilizing the folded and active form of the infused GAA enzyme, AT2220 may also mitigate ERT-induced immunogenicity since unfolded and aggregated proteins are generally more antigenic than properly folded proteins. Initial ex vivo studies using T cells derived from blood from 50 healthy donors demonstrated that the addition of AT2220 may significantly reduce the immunogenicity of Myozyme and Lumizyme. The studies utilized Antitope Ltd.'s EpiScreen™ assay and are being repeated in samples from the Pompe patients in Study 010. Results from Study 010 will be presented during LDN WORLD. Additional details regarding the Amicus Pompe program will be provided during the presentation and live webcast at the JPMorgan conference.
Migalastat HCl Co-Administered with Marketed ERTs for Fabry Disease
When co-administered with ERT, migalastat HCl is designed to bind to and stabilize infused enzyme in the circulation in any patient receiving ERT for Fabry disease. Amicus and GSK have completed an open-label Phase 2 study (Study 013) 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 23 males with Fabry disease. Positive preliminary results were announced during 2012 in patients who received migalastat HCl 150 mg co-administered with ERT. Results for migalastat HCl 450 mg co-administered with ERT will be presented at LDN WORLD.
Migalastat HCl Co-Formulated with Proprietary ERT for Fabry Disease
Migalastat HCl co-formulated with JCR Pharmaceutical Co. Ltd's proprietary investigational ERT (JR-051, recombinant human alpha-Gal A enzyme) is in preclinical development. Amicus and GSK, in collaboration with JCR, are currently conducting preclinical formulation and IND-enabling studies of this chaperone-ERT co-formulated product, which has the potential to enter the clinic in late-2013 or early 2014.
AT2220 Co-Formulated with Amicus Proprietary Next-Generation ERT
Amicus is combining its core pharmacological chaperone technology with advanced biologics capabilities to create a next-generation Pompe ERT. The Company is designing this co-formulated chaperone-ERT product with the goal of increasing exposure and tissue uptake and reducing immunogenicity of current ERTs. The co-formulation with AT2220 may also allow the ERT to be administered through novel routes. Amicus has entered into a contract with Laureate Pharmaceuticals for the contract manufacture of this next-generation ERT. Additional details regarding this aspect of the Amicus Pompe program will also be provided during the presentation and live webcast at the JPMorgan conference.
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