Form 8-K
 
 

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 11, 2011

AMICUS THERAPEUTICS, INC.
(Exact name of registrant as specified in its charter)

         
Delaware   001-33497   71-0869350
(State or other Jurisdiction of Incorporation)   (Commission File Number)   (IRS Employer Identification No.)
     
6 Cedar Brook Drive, Cranbury, NJ
  08512
(Address of Principal Executive Offices)   (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))

 
 

 

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Item 8.01. Other Events.

On January 11, 2011, John F. Crowley, President and Chief Executive Officer of Amicus Therapeutics, Inc. (the “Company”), participated in the 29th Annual J.P. Morgan Healthcare Conference (the “Conference”). A copy of the presentation given by Mr. Crowley at the Conference is attached to this Current Report as Exhibit 99.1. On the same date, 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.

 

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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.

Amicus Therapeutics, Inc.

Date: January 11, 2011

By: /s/ Geoffrey P. Gilmore                                     
Geoffrey P. Gilmore
Senior Vice President and General Counsel

 

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EXHIBIT INDEX

     
Exhibit No.   Description
99.1
  Presentation Materials
99.2
  Press Release dated January 11, 2011

 

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Exhibit 99.1
Exhibit 99.1
JP Morgan Healthcare Conference Jan 11, 2011 John F. Crowley, Chairman and CEO Nasdaq: FOLD www.amicustherapeutics.com


 

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 preclinical studies and clinical trials evaluating Amicus' candidate drug products, the projected cash position for the Company, and business development and other transactional activities. 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 Annual Report on Form 10-K for the year ended December 31, 2009. 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.


 

Industry Momentum in Rare Diseases "In addition to our existing discovery effort, alternative opportunities need to be explored to make treatments available for rare diseases.... this new unit has the potential to deliver multiple therapies responding to high medical needs of underserved populations of patients." - - Marc Dunoyer, Global Head, GSK Rare Diseases


 

Amicus: Building Shareholder Value in 2011 At the Forefront of Therapies for Rare Diseases(tm) Pathway to More Prevalent Disorders through Rare Diseases Strong Partnership with GSK Rare Diseases Advanced Product Pipeline Novel Pharmacological Chaperone Platform Technologies


 

Next Generation Therapy: Replacing ERT Protein Folding & Pharmacogenetics Substrate Accumulation Endoplasmic Reticulum Golgi Apparatus Lysosome Enhanced Trafficking Reduced ER Retention Decreased Substrate Pharmacological Chaperone (Oral) Misfolded/Unstable Protein Active Site Mutation Stabilized Protein Replacing ERTs in Lysosomal Storage Disorders Pharmacological Chaperone Monotherapy ~ 50% of Fabry patients


 

Improving ERTs in Lysosomal Storage Disorders Pharmacological Chaperone Co-Administration Next Generation Therapy: Enhancing ERT ~ 50% of Fabry patients Uncleared Substrate Circulation (Blood) Endosomal System Lysosome Increased Tissue Exposure Slower ERT Clearance From Circulation Additional Substrate Reduction Pharmacological Chaperone (Oral) Unstable ERT in bag/blood Stabilized ERT


 

AT2220 Alzheimer's Disease Advanced Product Pipeline Building Significant Rare Disease Franchise Preclinical Phase 1 Phase 2 Phase 3 AmigalTM Fabry Disease Pompe Disease ERT Co-admin Therapy ERT Co-admin Therapy Monotherapy PliceraTM Gaucher Disease ERT Co-admin Therapy AT3375 Parkinson's Disease


 

Amigal for Fabry Disease Disease Overview Lysosomal Storage Disease 5,000 - 10,000 patients worldwide Fabrazyme(r) and Replagal(r) ERTs current standard of care Current market: $800MM (est.) Males and Females GL-3 substrate accumulation Kidney, Heart and Brain Fatal


 

Amigal for Fabry Disease Clinical History of Amigal First in man: 2005 80+ patient-years of data 17 patients remain in Phase 2 extension study 5 patients more than 4 years 12 patients more than 3 years Phase 3 study ongoing Encouraging safety profile to date No drug-related serious adverse events No adverse event trends


 

Amigal for Fabry Disease Phase 2 Data Encouraging - Surrogate Endpoints Kidney Interstitial Capillary GL-3 Urine GL-3 Primary Surrogate Secondary Surrogate GL-3 Substrate Reduced


 

Preliminary Data Mean (SD) annualized change in GFR or eGFR (ml/min/1.73m2/yr) Proteinuria eGFR Amigal for Fabry Disease Phase 2 Data Encouraging - Clinical Endpoints Renal Function


 

Amigal for Fabry Disease Phase 3 Study 011 Study Overview 60 patients, 6 month trial Primary Endpoint: Amigal vs Placebo ^50% reduction in kidney interstitial capillary GL-3 Enriched patient population Males and females Status Update 36 sites initiated globally Majority of patients enrolled as of January Enrollment completion expected in 1H11 Top-line results expected in 2H11


 

Chaperone-ERT Co-administration Therapy


 

Improving ERTs for Lysosomal Storage Disorders The Problem Enzyme Replacement Therapies Denature Rapidly in Blood 125 100 75 0 25 50 0 4 8 12 16 20 24 % Initial Enzyme Activity Time (hours) Myozyme + AT2220 Cerezyme + AT2101 Fabrazyme + Amigal Myozyme Alone Cerezyme Alone Fabrazyme Alone Loss of Activity of ERTs at pH=7.4


 

Improving ERT for Fabry Disease Amigal + Fabrazyme Co-Administration In pre-clinical studies, Amigal significantly increases Fabrazyme tissue uptake and markedly reduces GL-3 levels in kidney Fabrazyme Kidney Tissue Uptake GL-3 Levels in Kidney


 

Improving ERT in Pompe Disease AT2220 + Myozyme Co-Administration Amicus plans to perform a Phase 2 trial of AT2220 co-administered with ERT based on encouraging preclinical data Myozyme Diaphragm Uptake Glycogen Levels in Diaphragm Myozyme AT2220 Glycogen (?g/mg protein) ^ + + ^ ^ + Myozyme AT2220 GAA Activity (nmol/mg protein/hr) ^ + + ^ ^ +


 

Partnership with GSK Rare Diseases and Financial Outlook


 

Strong Partnership with GSK Rare Diseases Deal Announced in October 2010 "Amicus' scientific and clinical expertise in human genetic diseases is among the best in the industry, and we are pleased to be collaborators and investors in this exceptional company." - - Marc Dunoyer, Global Head, GSK Rare Diseases "GSK has extremely impressive global clinical, regulatory and commercial expertise and a strong commitment to the development of treatments for rare diseases. " - - John Crowley, Chairman and Chief Executive Officer, Amicus Therapeutics


 

Strong Partnership with GSK Rare Diseases Exclusive Worldwide Rights for Amigal Validation of potential for technology and Fabry program GSK clinical, regulatory, commercial and manufacturing expertise Financial strength and flexibility for Amicus $30MM upfront license $31MM equity investment $170MM development + sales milestones Cost sharing on global development 50/50 in 2011 75 GSK/25 Amicus in 2012+ Tiered double digit royalties Rationale Deal Terms


 

Strong Partnership with GSK Rare Diseases Amicus Financial Strength Expected cash balance YE2010: ~ $100MM 2011 Projected Net Spend: $45-55MM Current cash along with anticipated GSK collaboration payments expected to be sufficient through anticipated Amigal U.S. commercial launch The GSK partnership allows Amicus to fully invest in Amigal and advance its pipeline while maintaining cash reserves


 

Further Opportunities in 2011 Building Shareholder Value Through Business Development R&D collaborations with pharmacological chaperone platform technology Partnerships and other opportunities to advance our neurodegenerative and chaperone-ERT co-administration programs Licensing transactions Strategic alliances Amicus is actively exploring a range of business development opportunities with multiple potential partners


 

Neurodegenerative Genetic Diseases


 

Pathway to More Prevalent Disorders Parkinson's and Gaucher: An Established Link Gaucher carriers have an estimated 5- fold increased risk for Parkinson's1 Disease, and Gaucher patients have a 20-fold risk2 Multiple independent studies in different populations Accumulation of alpha-synuclein is a leading target for new disease modifying therapies Mutations in the gene (GBA) now considered most common genetic risk factor for Parkinson's Disease 1Sidransky, New Engl J Med, 2009 Oct 22; 361(17): 1651-61 2Bultron, Journal of Inherited Metabolic Disease, 2010, 33(2):167-173


 

Pathway to More Prevalent Disorders Collaboration with The Michael J. Fox Foundation "Currently available treatments for Parkinson's provide symptomatic relief only. Our therapy is designed to address a deficiency that is inherited in a subset of the Parkinson's population, and to actually modify the course of the disease. " - - David J. Lockhart, Ph.D., Chief Scientific Officer, Amicus Therapeutics "I am pleased to continue working with Amicus on their exciting pharmacological chaperone approach to modify the progression of Parkinson's Disease. Amicus' earlier compound partially reversed the motor deficits in our alpha-synuclein overexpression mouse model, and we are eager to test the improved compounds." - - Marie-Francoise Chesselet, M.D., Ph.D., Charles H. Markham Professor of Neurology and Chair, Department of Neurobiology, David Geffen School of Medicine at UCLA


 

Pathway to More Prevalent Disorders Significant Advancement in Parkinson's Untreated PC-Treated Vehicle 3 weeks 15 weeks 15 weeks Synuclein Synuclein/NeuN Established proof-of-concept in Parkinson's animal models; increasing Gaucher enzyme (GCase) leads to marked synuclein reduction Prevention of synuclein accumulation in the brain Improvement in behavior and motor function


 

Pathway to More Prevalent Disorders Significant Improvement in Parkinson's Animals "...Amicus' compound partially reversed the motor deficits in our alpha-synuclein overexpression mouse model..." - - Marie-Francoise Chesselet, M.D., Ph.D., Charles H. Markham Professor of Neurology and Chair, Department of Neurobiology, David Geffen School of Medicine at UCLA The Pole Test: A measurement of synuclein-dependent motor behavior


 

Pathway to More Prevalent Disorders Alzheimer's: Link to Lysosomal Storage Disorders "The sheer bulk of waste proteins that are accumulating within the neurons in Alzheimer's disease brains is enormous...." - - Ralph A. Nixon, professor of psychiatry and cell biology , Langone Medical Center and the Nathan Kline Institute


 

Pathway to More Prevalent Disorders Amicus and Alzheimer's: A Pharmacological Chaperone Approach Presenillin-1 target Missense mutations 50,000-150,000 patients in U.S. Early pre-clinical POC established Lysosomal enzyme target ~4.5MM patients in U.S. Amicus is researching novel approaches to treating two distinct targets and patient populations within Alzheimer's Disease Genetic (Familial) Alzheimer's Sporadic Alzheimer's


 

1H 2011 2H 2011 1st patient enrolled in Amigal/ERT co-admin study 1st patient enrolled in Amigal Ph 3 study 012 Complete enrollment of Amigal Ph 3 study 011 Amigal Ph 2 extension study data update Start AT2220/ERT co-admin study in Pompe Amigal/ERT co-admin study data in Fabry Complete Parkinson's preclinical, toxicology and manufacturing IND-enabling activities Amigal Ph 3 study 011 top-line results Alzheimer's further preclinical proof of concept in: Presenilin-1 (Familial) Lysosomal enzyme target (Sporadic) AT2220/ERT co-admin study data in Pompe Amicus: Building Shareholder Value in 2011 Amicus 2011 Expected Milestones Ten milestones we expect to deliver in 2011 1H 2011


 

Amicus: Building Shareholder Value in 2011 January 2011 Value Proposition Amicus is a leader in rare diseases ~ $125MM Market Cap ~ $100MM cash today Product and platform company Ten expected milestones in 2011 Evaluating additional business development opportunities


 

JP Morgan Healthcare Conference Jan 11, 2011 John F. Crowley, Chairman and CEO Nasdaq: FOLD www.amicustherapeutics.com
Exhibit 99.2
Exhibit 99.2
(AMICUS LOGO)
Amicus Therapeutics Provides 2011 Business Outlook and Expected Key Milestones
Significant progress expected to further establish Company as a leader in rare diseases
Cranbury, NJ, January 11, 2011 — Amicus Therapeutics (NASDAQ: FOLD) today will provide the Company’s business outlook and expected key milestones for 2011 at the 29th Annual J.P. Morgan Healthcare Conference.
At the conference, Amicus is providing an update on its three key areas of focus: Amigal (migalastat hydrochloride) for the treatment of Fabry Disease, the evaluation of pharmacological chaperones co-administered with ERT, and the investigation of pharmacological chaperones for the treatment of diseases of neurodegeneration. The Company intends to identify key milestones expected in 2011 across these three areas, including results from the following studies:
 
Phase 3 study of Amigal for Fabry Disease in 2H11
 
 
Phase 2 study of Amigal co-administered with enzyme replacement therapy (ERT) for Fabry Disease in 2H11
 
 
Phase 2 study of AT2220 co-administered with ERT for Pompe Disease in 2H11
 
 
Late-stage preclinical proof of concept studies of AT3375 for Parkinson’s Disease, including completion of additional IND-enabling activities, in 2H11.
“2011 promises to be a transformational year for Amicus. This is an exciting time in the rare disease field and we are uniquely positioned to develop new therapies for patients and to build value for our shareholders,” said John F. Crowley, Chairman and CEO of Amicus. “This year we intend to achieve multiple milestones, led by our anticipated Phase 3 results for Amigal in Fabry Disease, which we expect to achieve in collaboration with our new partner, GSK Rare Diseases. In addition, we intend to move forward with Phase 2 studies evaluating chaperones co-administered with enzyme replacement therapy (ERT) in both Fabry and Pompe diseases. Finally, we expect important progress in our preclinical programs investigating the use of pharmacological chaperones in genetically defined sub-populations of Parkinson’s disease and Alzheimer’s disease. ”
Financial Guidance
The Company expects to begin 2011 with a cash balance of approximately $100 million and to spend between $45 and $55 million on 2011 operating expenses (net of cost sharing and milestones related to GSK collaboration). The current cash position, including anticipated payments from GSK in connection with the collaboration, is expected to be sufficient to fund the Company’s operations and capital expenditure requirements through the anticipated commercial launch of Amigal in the United States.
In 2011, the Company intends to evaluate additional business development opportunities to further build shareholder value. The Company indicates that it is actively exploring a range of opportunities with multiple potential partners.

 

 


 

Amigal (migalastat hydrochloride) for the treatment of Fabry Disease
On October 29, 2010, Amicus announced a definitive agreement with GlaxoSmithKline PLC (GSK) to develop and commercialize Amigal (migalastat HCl), currently in Phase 3 for the treatment of Fabry disease. Under the terms of the agreement, GSK received an exclusive worldwide license to develop, manufacture and commercialize migalastat HCl. Additionally, as part of the agreement, GSK and Amicus also intend to advance clinical studies exploring the co-administration of migalastat HCl with ERT 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 2011.
Amicus and GSK intend to commence an additional Phase 3 study (Study 012) in the first quarter 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).
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 and GSK intend to initiate a Phase 2 study with migalastat HCl co-administered with ERT for Fabry disease. This open-label Phase 2 study to investigate drug-drug interactions between migalastat HCl and ERT for Fabry disease is planned to commence in the first quarter of 2011 and results 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 2011. The Company intends to seek U.S. FDA approval to lift the current partial-hold on the AT2220 program as part of its development plan.
Diseases of Neurodegeneration
Amicus previously reported encouraging results from preclinical studies evaluating the use of a pharmacological chaperone for the treatment of Parkinson’s Disease. Today Amicus will announce that in 2011 it expects to complete late stage preclinical proof of concept studies, including IND-enabling activities, for its pharmacological chaperone 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 The Michael J. Fox Foundation (MJFF).
Additionally, Amicus is reporting today that it 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 Drug Discovery Foundation (ADDF).

 

 


 

About Amicus Therapeutics
Amicus Therapeutics is developing orally-administered, small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of rare diseases including lysosomal storage disorders and diseases of neurodegeneration. Amicus’ lead program is in Phase 3 for the treatment of Fabry disease.
Forward-Looking Statements
This press release contains “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 GlaxoSmithKline, and business development and other transactional activities that seek to strengthen the Company’s financial position. 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, including achievement of development and commercialization milestone payments and sales royalties under our collaboration with GlaxoSmithKline. Additionally, with respect to statements relating to potential business development opportunities and other transactions that seek to strengthen our financial position, we may not be successful in identifying suitable collaborators, establishing and implementing such collaborations or completing other transactions that could improve our financial position. 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, 2009. 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:
ir@amicustherapeutics.com
(609) 662-2000
FOLD—G