UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): January 7, 2013
AMICUS THERAPEUTICS, INC.
(Exact name of registrant as specified in its charter)
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Delaware |
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001-33497 |
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71-0869350 |
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(State or other Jurisdiction of |
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(Commission File Number) |
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(IRS Employer Identification No.) |
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1 Cedar Brook Drive, Cranbury, NJ |
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08512 |
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(Address of Principal Executive Offices) |
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(Zip Code) |
Registrant’s telephone number, including area code: (609) 662-2000
(Former name or former address if
changed since last report.)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
o Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
o Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
o Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
o Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Item 8.01. Other Events.
The senior management of Amicus Therapeutics, Inc. (the “Company”) is using the presentation attached as Exhibit 99.1 to this Current Report in its current meetings with investors and analysts. In addition, on January 7, 2013, the Company filed a press release, a copy of which is attached to this Current Report as Exhibit 99.2.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits: The Exhibit Index annexed hereto is incorporated herein by reference.
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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AMICUS THERAPEUTICS, INC. | |
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Date: January 7, 2013 |
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By: |
/s/ PETER M. MACALUSO |
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Name: |
Peter M. Macaluso |
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Title: |
Secretary |
EXHIBIT INDEX
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Exhibit No. |
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Description |
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99.1 |
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Presentation Materials |
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99.2 |
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Press Release dated January 7, 2013 |
Exhibit 99.1
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At the Forefront of Therapies for Rare and Orphan Diseases™ January 2013 |
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Safe Harbor This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 relating to business, operations and financial conditions of Amicus including but not limited to preclinical and clinical development of Amicus’ candidate drug products, the timing and reporting of results from clinical trials evaluating Amicus’ candidate drug products. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” “plan,” “would,” “should” and “could,” and similar expressions or words, identify forward-looking statements. Although Amicus believes the expectations reflected in such forward-looking statements are based upon reasonable assumptions, there can be no assurance that its expectations will be realized. Actual results could differ materially from those projected in Amicus’ forward-looking statements due to numerous known and unknown risks and uncertainties, including the “Risk Factors” described in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2012. All forward-looking statements are qualified in their entirety by this cautionary statement, and Amicus undertakes no obligation to revise or update this presentation to reflect events or circumstances after the date hereof. Slide 2 |
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Company Mission Amicus Therapeutics is a biopharmaceutical company at the forefront of developing next-generation medicines to treat a range of rare and orphan diseases, with a focus on improved therapies for Lysosomal Storage Disorders Slide 3 |
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JPMorgan Healthcare Conference 2013 Key Messages Phase 3 Fabry monotherapy study (Study 011) ongoing: Top-line 6-month (Stage 1) results encouraging and consistent with Phase 2 experience FDA to consider “entirety of data” from both 6 and 12 months Pompe Chaperone-ERT co-administration repeat-dose clinical study to begin 3Q13 Fabry Chaperone-ERT co-formulated product advancing towards clinic Proprietary Pompe next-generation ERT in preclinical development ~$100M cash at 12/31/12 Slide 4 |
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Core Technology and Focus Potential to Transform LSD Treatments Oral Monotherapy Orally administered Binds to and stabilizes patient’s own natural enzyme Replaces need for ERT for patients with amenable genetic mutations Slide 5 Oral Co-Administration Binds to and stabilizes currently marketed ERTs Increases tissue uptake Potentially reduces immunogenicity of ERT Chaperone Co-Formulation Proprietary next-generation ERTs All benefits of co-administration…plus stability from infusion bag to target tissue Small Molecule Pharmacological Chaperones Chaperone Chaperone ERT Next Gen ERTs |
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Fabry – Migalastat HCl – Study 013 Pompe – AT2220 – Study 010 Fabry JR051+Migalastat Fabry – Migalastat HCl – Study 011 Fabry – Migalastat HCl – Study 012 Parkinson’s AT3375 Advanced Product Pipeline One Technology, Three Novel Applications Slide 6 Chaperone-ERT Co-Administered Therapy Preclinical Phase 1 Phase 2 Phase 3 Marketing Application Other LSDS Gaucher AT2101/AT3375 Chaperone-ERT Co-Formulated Therapy Next-Gen ERTs Pompe ERT Chaperone Monotherapy |
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2013 Investment Highlights Slide 7 Migalastat HCl monotherapy: encouraging Phase 3 (Stage 1) and Phase 2 extension study results First-in-man Phase 2 results in Fabry and Pompe Chaperone-ERT Co-Administration Products Next-Generation ERTs in development Multiple potential therapeutic enhancements, including novel routes of delivery Platform Technology ~$99.1M cash (12/31/12) GSK responsible for 60% of all Fabry development costs Projected cash runway 18-24 months at current burn rates Multiple catalysts next 12-24 months Financial Strength “Amicus & GSK are committed to advancing Migalastat HCl monotherapy in Fabry” >$137M invested capital in Amicus & Migalastat HCl Fabry Chaperone-ERT co-formulation (Amicus, GSK and JCR) Pompe biologics CMO Partnerships |
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Slide 8 PHARMACOLOGICAL CHAPERONES MONOTHERAPY DEVELOPMENT IN FABRY DISEASE |
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Fabry Disease Overview Kidney GL-3 Slide 9 Progressive, multi-system lysosomal storage disease Caused by inherited GLA mutations X-linked disease Mortality due to renal failure, cardiac failure, stroke 5 – 10K patients diagnosed WW (51% female/49% male*) Significantly under-diagnosed *Fabry Registry 2011 |
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Migalastat HCl Monotherapy for Fabry Disease Development History Slide 10 Jan 2013 Highlights 102 Fabry patients WW on Migalastat HCl monotherapy as only therapy for Fabry >220 patient-years of data 57 of 59 pts completed 12 months of Study 011 and elected to continue in long-term extension studies 2006 2007 2008 2009 2010 2011 2012 Ph1 Safety Studies Complete Preliminary Ph2 Data Ph2 Extension Study – Dose Ranging* Ph3 Discussions with FDA and EMEA Ph2 Extension Data at LDN World Phase 3 Study 011* LPI Study 011 Ph3 Study 011 Results (Stage 1) LPI Study 012 Phase 3 Study 012* *Open-label extension studies ongoing in patients who completed Ph2 extension study and Ph3 studies |
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Amenable Mutations (n=5): 78% median decrease Non-Amenable Mutations (n=3): 114% median increase -71% -34% -91% -97% -78% +127% +114% -27% Week 54 (M) Weeks of treatment duration in Study 205 in Males (M) and Females (F)* Week 54 (M) Week 63 (M) Week 60 (F) Week 90 (F) Median 78% Decrease in Kidney GL-3 Observed in Study Patients with Amenable Mutations Migalastat HCl Monotherapy for Fabry Disease Phase 2 Extension Study Update – Presented at ASN (Nov. 2012) GL-3 (inclusions/PTC) Slide 11 *All patients in Study 205 completed initial Phase 2 studies of migalastat HCl monotherapy |
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Migalastat HCl Monotherapy for Fabry Disease Phase 3 Study 011: Design Kidney Biopsy 1:1 Randomization Stratified by gender Baseline 6-Month Open-Label Follow-Up Period 12-Month Optional Extension Placebo QOD Migalastat HCl 150 mg Every-Other-Day (QOD) Open-Label Migalastat HCl 150 mg QOD Slide 12 6-Month Double-Blind Primary Treatment Period Month 12 Month 6 |
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Migalastat HCl Monotherapy for Fabry Disease Phase 3 Study 011: Top-Line 6-Month (Stage 1) Results Kidney Interstitial Capillary GL-3 – Surrogate Biomarker Considered Likely to Predict Clinical Benefit in Fabry Patients P=0.3 Placebo Migalastat HCl 150 mg QOD 9/32 13/32 Median % Change In Kidney Interstitial Capillary GL-3 (Baseline to Month 6) Placebo Migalastat HCl 150 mg QOD P=0.09 Slide 13 % With >50% Reduction in Kidney Interstitial Capillary GL-3 (Baseline to Month 6) |
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FDA Responsiveness and Flexibility in Rare Diseases PDUFA V Impact “FDA assesses the benefit-risk of new drugs on a case-by-case basis, considering the degree of unmet need and the severity and morbidity of the condition the drug is intended to treat. This approach has been critical to increasing patient access to new drugs for cancer and rare and other serious diseases, where existing therapies have been few and limited in their effectiveness.” Janet Woodcock, M.D. Director, CDER U.S. FDA Slide 14 Statement on FDA User Fee Agreements: Strengthening FDA and the Medical Products Industry for the Benefit of Patients (March 29, 2012) |
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Migalastat HCl Monotherapy for Fabry Disease Regulatory Guidance and Path Forward Study 011 is an Ongoing 12-Month Pivotal Study of Migalastat HCl in Patients with Fabry Disease with Amenable Mutations Slide 15 U.S. FDA Feedback 6-month analysis is Stage 1 FDA to consider Stage 1 and Stage 2 (12-month) data for NDA submission FDA will evaluate efficacy and safety based on entirety of Study 011 data (no single endpoint will be determinitive) FDA meeting anticipated in mid-2013 to discuss US approval pathway |
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Migalastat HCl Monotherapy for Fabry Disease Study 011 12-Month Analysis Plans Placebo arm Stage 1 vs. Stage 2 (migalastat HCl 6 months vs. placebo 6 months) Treatment arm Stage 1 vs. Stage 2 (migalastat HCl 12 months vs. 6 months) Treatment arm Stage 1 + placebo arm Stage 2 (pooled migalastat HCl 6 months) vs. placebo arm Stage 1 Additional safety data Slide 16 Study 011 Design (1:1 Randomization) Study Arm Stage 1: Month 0-6 Stage 2: Month 6-12* Placebo Placebo Migalastat HCl Treatment Migalastat HCl Migalastat HCl switch *Additional 12-24 month open-label extension following Stage 2 12-Month Descriptive Comparisons – Results Anticipated 1H13 |
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Migalastat HCl Monotherapy for Fabry Disease Phase 3 Study 012: Overview and Status Comparing open-label oral migalastat HCl (150 mg QOD) to ERT (Replagal and Fabrazyme) Switch from ERT to migalastat HCl or remain on ERT (1.5:1 randomization) Fabry patients with amenable mutations, no kidney biopsies Fully enrolled with 60 patients (26 males and 34 females) Clinical Outcome is renal function (Iohexol GFR) at 18 months 18-month treatment period expected to complete in 2Q14 Slide 17 |
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Migalastat HCl Monotherapy Key Anticipated Phase 3 Inflection Points Detailed Study 011 6-month data at LDN WORLD Feb. 2013 Study 011 12-month (Stage 2) top-line data 2Q13 FDA meeting to discuss U.S. approval pathway Mid-2013 Completion of Study 012 18-month treatment period 2Q14 Study 012 top-line data anticipated 2H14 Slide 18 |
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Migalastat HCl Monotherapy: Amenable Mutations Evolving Epidemiology Although ~30% of Currently Diagnosed Fabry Patients Estimated to Have Amenable Mutations, 75-100% of Patients Identified in Recent Newborn Screening Studies Have Mutations Potentially Amenable to Migalastat HCl Recent Newborn Screening Studies # Newborns Screened # Confirmed Fabry Mutations % Potentially Amenable Burton, 2012, US 8,012 7 [1: ~1100] TBD Mechtler, 2011, Austria 34,736 9 [1: ~3,800] 100% Hwu, 2009, Taiwan 171,977 75 [1: ~2300] 75% Spada, 2006, Italy 37,104 12 [1: ~3100] 86% Index Patient 3-5 :1 Index Due to X-linked nature of Fabry, patients identified by screening typically yield 3-5 affected family members (Weidemann 2010) Slide 19 Burton, LDN WORLD Symposium, 2012 Feb. Mechtler et al., The Lancet, 2011 Dec. 3. Hwu et al., Hum Mutation, 2009 Jun 4. Spada et al., Am J Human Genet., 2006 Jul |
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Slide 20 PHARMACOLOGICAL CHAPERONES CO-ADMINISTERED WITH MARKETED ERTS IMPROVING CURRENT ERTS FOR LYSOSOMAL STORAGE DISORDERS |
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Photo by J Pastor/Univ of Florida Protein Instability Blood & Infusion Bag LSD Products Today Potential Issues Slide 21 Immunogenicity Dosing Limitations Duration of Infusion |
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Chaperone-ERT Combination Therapy Goals Potential to Improve Safety and Efficacy of ERT While Reducing Patient Burden Enhance ERT pharmacokinetics (greater overall exposure) Increase enzyme uptake in tissues, possibly into cell types and tissues not well-served by ERT alone (e.g., podocytes, cardiomyocytes, brain) Improve substrate reduction in disease-relevant cell types and tissues Mitigate ERT-mediated immunogenicity Reduce burden of therapy for patients (e.g., more convenient ERT) Slide 22 |
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Fabry Chaperone-ERT Co-Administration Phase 2 Study 013: Preliminary Results Oral Migalastat HCl 150 mg* Co-Administered with Fabrazyme or Replagal Led to Consistent Increases in Levels of Active Plasma Enzyme and Tissue Uptake (Skin) Plasma rhGLA Activity (Area Under Curve) Slide 23 Mean Skin GLA Activity (Day 2) +90% +160% +80% *Single oral dose 2 hours prior to ERT infusion (n=4) (n=5) (n=3) ERT + 150 mg Migalastat |
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Pompe Chaperone-ERT Co-Administration Phase 2 Study 010: Cohorts 1-4 Oral AT2220 Co-Administered with Myozyme/Lumizyme Also Leads to Consistent Increases in Plasma Enzyme Activity and Tissue Uptake (Muscle) *Cohort 1 (AT2220 50 mg) muscle GAA activity not shown; 50 mg dose did not demonstrate meaningful change in tissue uptake (muscle) Plasma AUC rhGAA Activity Muscle GAA Activity (Day 3)* Slide 24 +133% +7% +25% (n=3) (n=3) (n=2) |
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Pompe Chaperone-ERT Co-Administration ERT-Related Immunogenicity Problem “…. identification of patients at risk for developing high sustained antibody titer is critical.”1 “…. approximately 40% of the administered alglucosidase alfa was captured by circulating antibodies.”2 “…. infusion-associated reactions (IARs) [occur] in ~50% of patients receiving alglucosidase alfa infusions.”3 Slide 25 Banugaria et al., Gen. Med., 2011 Aug. de Vries et al., Mol Genet Metab., 2010 Dec. Banati et al., Muscle Nerve, 2011 Dec. |
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Pompe Chaperone-ERT Co-Administration Potential to Mitigate ERT Immunogenicity AT2220 Mitigates Human T-Cell Response Induced by Lumizyme ex vivo and May Significantly Reduce Immunogenicity of Lumizyme % Donor PBMCs with T-Cell Proliferation PBMCs from 50 Healthy Volunteers Lumizyme Lumizyme + 50uM AT2220 14/50 6/50 5/50 19/50 Lumizyme 27oC Lumizyme 27oC + 50uM AT2220 Slide 26 |
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Slide 27 Pompe Chaperone-ERT Co-Administration Repeat-Dose Clinical Study Anticipated to Begin 3Q13 Target enrollment Adolescents/adults with Pompe disease ERT naïve and ERT experienced Endpoints PK Safety Efficacy Immunogenicity Treatment duration 12-24 week primary treatment period with potential extension |
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Slide 28 PHARMACOLOGICAL CHAPERONES CO-FORMULATED WITH RECOMBINANT ERTS TOWARD THE NEXT-GENERATION OF PROPRIETARY ERTS FOR LYSOSOMAL STORAGE DISORDERS |
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Fabry Chaperone-ERT Co-Formulation Proprietary ERT JR-051* + Migalastat HCl Slide 29 JR-051 + Migalastat HCl Fabrazyme Co-Formulation Results in Significantly Greater GL-3 Reduction than Previously Observed Time (Hours) Heart GL-3 Kidney GL-3 Medium Dose JR-051 (~ Fab 1 mg/kg) *JR-051 designed to be biosimilar to Fabrazyme **0 = wild-type, 100 = untreated KO mouse % Elevated > Wild-Type** % Elevated > Wild-Type** Preliminary Results JR-051 High Dose JR-051 Medium Dose JR-051 (~ Fab 1 mg/kg) High Dose JR-051 JR-051 Alone JR-051 + migalastat Migalastat HCl Prevents Loss of Enzyme Activity in Blood JR-051 + Migalastat HCl Fabrazyme 160 140 120 80 60 40 20 100 8 6 4 2 16 24 18 0 12 10 0 14 20 22 JR-051 % Initial Activity |
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Fabry Chaperone-ERT Co-Formulation Development Status and Anticipated Milestones Slide 30 Now manufacturing at 2,000 L scale IND-enabling studies underway Potential to enter clinic 4Q13/1Q14 Advancing JR-051 + Migalastat HCl Toward Clinic |
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Pompe Chaperone-ERT Co-Formulation Next-Generation ERT for Pompe Slide 31 Combining Core Pharmacological Chaperone Technology with Advanced Biologics Capabilities to Create a Next-Generation Pompe ERT Potential Improvements Optimized glycosylation (e.g., M6-P) De-immunization Recombinant Human GAA Next-Generation Pompe ERT P M C P C C C N H O H O H H O O H H C l . ATT2220 Small Molecule Stabilizer Increased exposure & tissue uptake Reduced immunogenicity Formulation for SQ route of administration |
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Slide 32 2013 KEY MILESTONES AND CATALYSTS |
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Slide 33 2013 Anticipated Milestones Building Shareholder Value Migalastat HCl Monotherapy for Fabry Disease Study 011 6-Month data (Stage 1) at LDN WORLD Feb. 2013 Top-line Study 011 12-month data (Stage 2) 2Q13 FDA meeting to discuss U.S. approval pathway Mid-2013 Pompe Chaperone-ERT Co-Administration IND-enabling studies and clinical supply manufacturing Ongoing Potential entry into clinic 4Q13/1Q14 Phase 2 Study 010 data at LDN WORLD (all 4 cohorts) Feb. 2013 Initiation of repeat-dose clinical study 3Q13 Fabry Chaperone-ERT Co-Formulation (Migalastat HCl + JR-051) Fabry Chaperone-ERT Co-Administration Phase 2 Study 013 data at LDN WORLD (oral migalastat HCl 450 mg + ERT) Feb. 2013 |
Exhibit 99.2
Amicus Therapeutics Provides Full-Year 2013
Financial Guidance and Strategic Outlook
6-Month (Stage 1) Results from Ongoing Phase 3 Fabry Disease Monotherapy Study at Lysosomal Storage Disease Network WORLD Symposium (LDN WORLD) in February 2013; 12-Month Results Expected in 2Q13
Pompe, Fabry and Other New Chaperone-Enzyme Replacement Therapy (ERT)
Programs Advancing in 2013
FY13 Cash Spend Guidance Range of $52-$58 Million — Current Cash Expected to Fund Operations into at Least 2H14
CRANBURY, NJ, US, January 7, 2013 — 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.
Strategic Outlook
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.
Chaperone-ERT Co-Administration
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.
Chaperone-ERT Co-Formulation
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.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of developing therapies for rare and orphan diseases. The Company is developing orally-administered, 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.
About Migalastat HCl for Fabry Disease
Amicus in collaboration with GlaxoSmithKline (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. As a monotherapy, migalastat HCl is designed to bind to and stabilize, or “chaperone” alpha-galactosidase A (alpha-Gal A) enzyme in patients with genetic mutations that are amenable to this chaperone in a cell-based assay. For patients currently receiving ERT for Fabry disease, migalastat HCl in combination with ERT may improve ERT outcomes by keeping the infused alpha-Gal A enzyme in its properly folded and active form.
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 AT2220 for Pompe Disease
AT2220 is an investigational, orally-administered pharmacological chaperone owned exclusively by Amicus. In published preclinical studies, AT2220-ERT co-administration resulted in significant increases in muscle rhGAA levels and decreases in glycogen levels in a mouse model of Pompe disease. Preclinical results to date also suggest that AT2220-ERT co-administration may mitigate ERT-induced immunogenicity by stabilizing the enzyme in its properly folded and active form.
Pompe disease is a lysosomal storage disease characterized by progressive skeletal muscle weakness and respiratory insufficiency. It is caused by a deficiency in 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.
Forward-Looking Statements
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 Quarterly Report on Form 10-Q for the quarter ended June 30, 2012. 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.
CONTACTS:
Investors/Media:
Sara Pellegrino
spellegrino@amicusrx.com
(609) 662-5044
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