Document


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): February 28, 2019
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AMICUS THERAPEUTICS, INC.
(Exact Name of Registrant as Specified in Its Charter)

Delaware
(State or Other Jurisdiction of
Incorporation)

   001-33497
  71-0869350
 (Commission File Number)
(IRS Employer Identification No.)
 
 
1 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))

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company o

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. o






Item 2.02 Results of Operations and Financial Condition

On February 28, 2019, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal year ended December 31, 2018. A copy of this press release is attached hereto as Exhibit 99.1. The Company will host a conference call and webcast on February 28, 2019 to discuss its full year results of operations. A copy of the conference call presentation materials is attached hereto as Exhibit 99.2. Both exhibits are incorporated herein by reference.

In accordance with General Instruction B.2. of Form 8-K, the information in this Current Report on Form 8-K and the Exhibits shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liability of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing. 

Item 9.01 Financial Statements and Exhibits
(d) Exhibits:

Exhibit No.
Description









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: February 28, 2019
By: /s/ Ellen S. Rosenberg
 
Name: Ellen S. Rosenberg
 
Title: General Counsel and Corporate Secretary



Exhibit
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Amicus Therapeutics Announces Full-Year 2018 Financial Results and 2019 Corporate Updates


Continued Strong Galafold Launch Trends in Early 2019 - On Track to Achieve FY19 Revenue Guidance of $160M-$180M

FY18 Galafold Revenue of $91.2M Reflects 650+ Fabry Patients Treated at end of 2018 - 1,000+ Patients Expected by end of 2019

Clinical Data Out to Month 24 and Breakthrough Therapy Designation in Late Onset Pompe Disease Continue to Support AT-GAA as Next Potential Standard of Care

Amicus Global Research and Gene Therapy Center of Excellence in Philadelphia to Further Strengthen Leadership in Gene Therapy for Rare Diseases

Strong Balance Sheet with $500M+ Cash

Conference Call and Webcast Today at 8:30 a.m. ET

CRANBURY, NJ, February 28, 2019Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, today announced financial results for the full year ended December 31, 2018. The Company also summarized recent program updates and reiterated its full-year 2019 guidance.

Corporate Highlights for Full-Year 2018 and Year-to-Date 2019
More than doubled global revenue for Galafold (migalastat) in 2018. As previously announced, revenue grew from $36.9 million in full-year 2017 to $91.2 million in full-year 2018, exceeding the high end of the full-year 2018 guidance range of $80 million to $90 million.
Strong Launch Trends Continue in Early 2019. On track to achieve full-year 2019 revenue guidance of $160M-$180M and 1,000+ patients on Galafold by year-end.
Updated AT-GAA clinical data in Pompe disease presented at WORLDSymposium™ in February 2019. As previously announced, consistent and durable responses continued across key measures of safety, functional outcomes and biomarkers for both ERT-naïve and ERT-switch patients treated with AT-GAA for up to 24 months in the ongoing Phase 1/2 clinical study.
AT-GAA received Breakthrough Therapy Designation (BTD) in late onset Pompe disease. The BTD was based on clinical efficacy results from the ongoing ATB200-02 Phase 1/2 clinical study, and further strengthens the Company’s conviction in the potential for AT-GAA to become the next standard of care.
Enrollment momentum in ongoing pivotal PROPEL study in Pompe disease and Phase 1/2 study in CLN3 Batten disease. The PROPEL study is on track to achieve full enrollment by year-end 2019. The first patient remains in the CLN3 Batten disease study with no serious adverse events after more than two months following a single administration of AAV9-CLN3 gene therapy. Further patient dosing is expected in the coming months.
Robust gene therapy pipeline continues to advance toward important data milestones. Additional two-year data from Phase 1/2 study in CLN6 Batten disease expected mid-year. Preclinical proof-of-concept for Fabry and Pompe gene therapies anticipated throughout 2019.
New Global Research and Gene Therapy Center of Excellence in Philadelphia. This new headquarters for the global Amicus science organization and the gene therapy leadership team advances the Company’s commitment to world-class science.
Strong financial position to continue executing the Galafold launch and advance development programs. The current cash position of approximately $504.2 million at December 31, 2018 is expected to fund ongoing operations into at least mid-2021.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. stated, “Over the course of last year and into early 2019 we have made great strides in our continued evolution as a leading global rare disease biotechnology company. We are on track and well-capitalized to achieve all of our 2019 key strategic priorities including our global Fabry launch, Pompe late-stage development program, and gene therapy pipeline. With a very successful, now global, commercial precision medicine product in Fabry disease, a late stage program with BTD in late onset Pompe disease and 14 Gene Therapy programs for rare diseases in development, including two in the clinic, I believe that we are now, more than ever before, strongly positioned to achieve our vision. We endeavor to deliver groundbreaking new medicines and hopefully one day cures for people living with rare metabolic diseases and to be one of a handful of leading companies in the world in this field. Achieving this vision will create enormous value for people with these life-threatening conditions as well as our shareholders.”

Full-Year 2018 Financial Results
Total revenue in the full-year 2018 was $91.2 million, an increase from total revenue of $36.9 million in the full-year 2017.
Cash, cash equivalents, and marketable securities totaled $504.2 million at December 31, 2018 compared to $358.6 million at December 31, 2017.
Total operating expenses decreased to $405.6 million for the full-year 2018 compared to $472.7 million in the full-year 2017. Operating expenses reflecting increased investments in the Galafold launch, Pompe program, and gene therapy pipeline.
Net cash spend was $189.3 million for the full-year 2018, which was below full-year 2018 net cash spend guidance of $200 million to $225 million and reflects careful expense management.
Net loss was $349.0 million, or $1.88 per share, compared to a net loss of $284.0 million, or $1.85 per share, for the full-year 2017.

2019 Key Strategic Priorities
Nearly double again, annual revenue for Galafold (FY19 guidance of $160M-$180M in worldwide revenue) with 1,000+ Fabry patients on Galafold by year end.
Complete enrollment in pivotal study in Pompe disease and report additional Phase 2 data.
Report additional two-year results from Phase 1/2 clinical study in CLN6 Batten disease and complete enrollment in ongoing CLN3 Batten disease Phase 1/2 study.
Establish preclinical proof of concept for Fabry and Pompe gene therapies.
Maintain a strong financial position.

2019 Financial Guidance
Amicus recorded $91.2 million in full-year 2018 revenue from commercial sales and reimbursed expanded access programs for Galafold. For the full-year 2019 the Company anticipates total Galafold revenue of $160 million to $180 million. Growth in 2018 was largely driven by EU and other countries outside the U.S. and Japan. Growth in 2019 is expected to be driven by continued growth in EU markets, further geographic expansion, and further success from the first full year of launch in the U.S. and Japan.

Cash, cash equivalents, and marketable securities totaled $504.2 million at December 31, 2018. The Company expects to end 2019 with approximately $300 million in cash on hand. The current cash position is anticipated to fund ongoing operations into at least mid- 2021.

Anticipated 2019 Milestones by Program
Amicus previously announced full-year 2018 program updates as well as anticipated 2019 program milestones in early January 2019. All anticipated milestones remain on track as follows:
Galafold (migalastat) Oral Precision Medicine for Fabry Disease
On track to meet full-year 2019 revenue guidance range of $160 million to $180 million.

AT-GAA for Pompe Disease
Initial 6-month data in additional ERT-switch patients (Cohort 4) in Phase 1/2 ATB200-02 clinical study.
Retrospective natural history study data in approximately 100 ERT-treated Pompe patients.
Additional supportive studies, including an open-label study in pediatric patients.
Full enrollment in Phase 3 PROPEL study.
Advance agreed upon CMC requirements to support BLA.

Gene Therapy Pipeline:
Additional two-year data from CLN6 Batten disease Phase 1/2 study.
Full enrollment of ongoing CLN3 Batten disease Phase 1/2 study.
Preclinical data for next-generation gene therapies for Fabry, Pompe and CDD.
Preclinical work across additional neurologic LSDs.


Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, February 28, 2019 at 8:30 a.m. ET to discuss the full year 2018 financial results and corporate updates. Interested participants and investors may access the conference call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international), conference ID: 6983148.

A live audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/, and will be archived for 30 days. Web participants are encouraged to register on the website 15 minutes prior to the start of the call. A replay of the call will be available for seven days beginning at 11:30 a.m. ET on February 28, 2019. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); conference ID: 6983148.

About Galafold 
Galafold® (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable GLA variants. In these patients, Galafold works by stabilizing the body’s own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of Fabry patients may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in Australia, Canada, European Union, Israel, Japan, South Korea, Switzerland and the U.S.
U. S. INDICATIONS AND USAGE
Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.
This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
U.S. IMPORTANT SAFETY INFORMATION
ADVERSE REACTIONS
The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFIC POPULATIONS
There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus.
It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.
Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.
The safety and effectiveness of Galafold have not been established in pediatric patients.
To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf.


EU Important Safety Information
Treatment with Galafold should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. Galafold is not recommended for use in patients with a nonamenable mutation.
Galafold is not intended for concomitant use with enzyme replacement therapy.
Galafold is not recommended for use in patients with Fabry disease who have severe renal impairment (<30 mL/min/1.73 m2). The safety and efficacy of Galafold in children 0–15 years of age have not yet been established.
No dosage adjustments are required in patients with hepatic impairment or in the elderly population.
There is very limited experience with the use of this medicine in pregnant women. If you are pregnant, think you may be pregnant, or are planning to have a baby, do not take this medicine until you have checked with your doctor, pharmacist, or nurse.
While taking Galafold, effective birth control should be used. It is not known whether Galafold is excreted in human milk.
Contraindications to Galafold include hypersensitivity to the active substance or to any of the excipients listed in the PRESCRIBING INFORMATION.
It is advised to periodically monitor renal function, echocardiographic parameters and biochemical markers (every 6 months) in patients initiated on Galafold or switched to Galafold.
OVERDOSE: General medical care is recommended in the case of Galafold overdose.
The most common adverse reaction reported was headache, which was experienced by approximately 10% of patients who received Galafold. For a complete list of adverse reactions, please review the SUMMARY OF PRODUCT CHARACTERISTICS.
Call your doctor for medical advice about side effects.   

For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.

About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on Twitter and LinkedIn.


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 our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, manufacturing and supply plans, financing plans, and the projected revenues and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we 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 in particular 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 our business, including, without limitation: the potential that results of clinical or preclinical 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, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe, Japan, the US and other geographies or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we may not be able to manufacture or supply sufficient clinical or commercial products; and the potential that we will need additional funding to complete all of our studies and manufacturing. 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 revenue and 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, 2018 filed today. 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 we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.
 
CONTACTS:

Investors/Media:
Amicus Therapeutics
Sara Pellegrino, IRC
Vice President, Investor Relations and Corporate Communications
spellegrino@amicusrx.com
(609) 662-5044

Media:

Amicus Therapeutics
Marco Winkler
Director, Corporate Communications

mwinkler@amicusrx.com
(609) 662-2798

FOLD–G




TABLE 1

Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
 
Years Ended December 31,
 
2018
 
2017
 
2016
Revenue:
 
 
 
 
 
Net product sales
$
91,245

 
 
$
36,930

 
 
$
4,958

 
Cost of goods sold
14,404
 
 
 
6,236
 
 
 
833
 
 
Gross profit
76,841
 
 
 
30,694
 
 
 
4,125
 
 
Operating expenses:
 
 
 
 
 
Research and development
270,902
 
 
 
149,310
 
 
 
104,793
 
 
Selling, general and administrative
127,200
 
 
 
88,671
 
 
 
71,151
 
 
Changes in fair value of contingent consideration payable
3,300
 
 
 
(234,322
 
)
 
6,760
 
 
Loss on impairment of assets
 
 
 
465,427
 
 
 
 
 
Restructuring charges
 
 
 
 
 
 
69
 
 
Depreciation
4,216
 
 
 
3,593
 
 
 
3,242
 
 
Total operating expenses
405,618
 
 
 
472,679
 
 
 
186,015
 
 
Loss from operations
(328,777
 
)
 
(441,985
 
)
 
(181,890
 
)
Other income (expenses):
 
 
 
 
 
Interest income
10,461
 
 
 
4,096
 
 
 
1,602
 
 
Interest expense
(22,402
 
)
 
(17,240
 
)
 
(5,398
 
)
Change in fair value of derivatives
(2,739
 
)
 
 
 
 
 
 
Loss on extinguishment of debt
 
 
 
 
 
 
(13,302
 
)
Other income (expense)
(5,632
 
)
 
6,008
 
 
 
(4,793
 
)
Loss before income tax
(349,089
 
)
 
(449,121
 
)
 
(203,781
 
)
Income tax benefit
94
 
 
 
165,119
 
 
 
3,739
 
 
Net loss attributable to common stockholders
$
(348,995

)
 
$
(284,002

)
 
$
(200,042

)
Net loss attributable to common stockholders per common share — basic and diluted
$
(1.88

)
 
$
(1.85

)
 
$
(1.49

)
Weighted-average common shares outstanding — basic and diluted
185,790,021
 
 
 
153,355,144
 
 
 
134,401,588
 
 


TABLE 2         
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
 
December 31,
 
2018
 
2017
Assets
 
 
 
Current assets:
 
 
 
Cash and cash equivalents
$
79,749

 
 
$
49,060

 
Investments in marketable securities
424,403
 
 
 
309,502
 
 
Accounts receivable
21,962
 
 
 
9,464
 
 
Inventories
8,390
 
 
 
4,623
 
 
Prepaid expenses and other current assets
16,592
 
 
 
19,316
 
 
Total current assets
551,096
 
 
 
391,965
 
 
Property and equipment, less accumulated depreciation of $15,671 and $12,515 at December 31, 2018 and 2017, respectively
11,375
 
 
 
9,062
 
 
In-process research & development
23,000
 
 
 
23,000
 
 
Goodwill
197,797
 
 
 
197,797
 
 
Other non-current assets
6,683
 
 
 
5,200
 
 
Total Assets
$
789,951

 
 
$
627,024

 
Liabilities and Stockholders' Equity
 
 
 
Current liabilities:
 
 
 
Accounts payable, accrued expenses, and other current liabilities
$
80,625

 
 
$
53,890

 
Deferred reimbursements
5,500
 
 
 
7,750
 
 
Contingent consideration payable
 
 
 
8,400
 
 
Total current liabilities
86,125
 
 
 
70,040
 
 
Deferred reimbursements
10,156
 
 
 
14,156
 
 
Convertible notes
175,006
 
 
 
164,167
 
 
Senior secured term loan
146,734
 
 
 
 
 
Contingent consideration payable
19,700
 
 
 
17,000
 
 
Deferred income taxes
6,465
 
 
 
6,465
 
 
Other non-current liabilities
2,853
 
 
 
2,346
 
 
Total Liabilities
447,039
 
 
 
274,174
 
 
Commitments and contingencies
 
 
 
Stockholders' equity:
 
 
 
Common stock, $.01 par value, 500,000,000 shares authorized, 189,383,924 shares issued and outstanding at December 31, 2018 Common stock, $.01 par value, 250,000,000 shares authorized, 166,989,790 shares issued and outstanding at December 31, 2017
1,942
 
 
 
1,721
 
 
Additional paid-in capital
1,740,061
 
 
 
1,400,758
 
 
Accumulated other comprehensive loss:
 
 
 
Foreign currency translation adjustment
495
 
 
 
(1,659
 
)
Unrealized loss on available-for securities
(427
 
)
 
(436
 
)
Warrants
13,063
 
 
 
16,076
 
 
Accumulated deficit
(1,412,222
 
)
 
(1,063,610
 
)
Total stockholders' equity
342,912
 
 
 
352,850
 
 
Total Liabilities and Stockholders' Equity
$
789,951

 
 
$
627,024

 


exhibit992
FY18 Financial Results Conference Call & Webcast February 28, 2019


 
2 Forward-Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, manufacturing and supply plans, financing plans, and the projected revenues and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we 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 in particular 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 our business, including, without limitation: the potential that results of clinical or preclinical 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, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe, Japan, the US and other geographies or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we may not be able to manufacture or supply sufficient clinical or commercial products; and the potential that we will need additional funding to complete all of our studies and manufacturing. 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 revenue and 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, 2018 filed today. 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 we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.


 
Introduction 3 Rare Disease Day – February 28, 2019 Rare Disease Day 2019 is “Bridging Health and Social Care”


 
Introduction 4 Amicus Highlights GALAFOLD’S EXTRAORDINARY LAUNCH AT-GAA IN POMPE: POTENTIAL TO LEADING GENE THERAPY PORTFOLIO IN RARE SUCCESS BECOME STANDARD OF CARE METABOLIC DISEASES • 650+ Patients and $91.2M Global Sales in FY18 • Continued Strength of Clinical Data • Pipeline of 14 Gene Therapies • FY19 Guidance of $160M-$180M • Granted Breakthrough Therapy Designation • 2 Clinical Stage Programs • $500M Potential Sales by 2023 • Multiple Data Expected Throughout 2019 • Established Global Research and Gene Therapy • $1B+ Addressable Market Opportunity by 2028 • 100+ Pompe Patients on AT-GAA by YE19 Center of Excellence in Philadelphia • $1B-$2B+ Market Opportunity • $1B+ Peak Recurring Market Opportunity FINANCIAL STRENGTH 2023 VISION • $500M+ Cash at 12/31/18 (runway into mid-2021) • 5,000+ Lives Transformed • Growing Contribution from Galafold Revenues • $1B+ in Revenue • Leading Global Rare Disease Biotech


 
Introduction 5 2019 Key Strategic Priorities 1 Nearly double annual revenue for Galafold (guidance $160M-$180M) Complete enrollment in AT-GAA Pivotal Study (PROPEL) and 2 report additional Phase 1/2 data Report additional 2-year clinical results in CLN6-Batten disease and 3 complete enrollment in ongoing CLN3-Batten disease Phase 1/2 study Establish preclinical proof of concept for Fabry and Pompe gene 4 therapies 5 Maintain strong financial position


 
Galafold® (migalastat) Global Launch… …taking a leadership role in the treatment of Fabry disease “We push ideas as far and as fast as possible” - Amicus Belief Statement


 
Galafold: Precision Medicine for Fabry Disease 7 Galafold Snapshot (as of December 31, 2018) $91.2M $160-180M One of the Most FY18 Galafold FY19 Global Revenue Galafold Successful Rare Rev. Guidance Disease Launches Geographic 24 Countries with Expansion in Pricing & 2019 Reimbursement 8 Regulatory 348 Approvals: Amenable Australia, Canada, EU, Variants in U.S. Israel, Japan, S. Korea, Switzerland , U.S. Label Galafold is indicated for adults with a confirmed diagnosis of Fabry Disease and an amenable mutation/variant. The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.


 
Galafold: Precision Medicine for Fabry Disease 8 Galafold Success and FY18 Galafold Revenue Guidance On Track to Nearly DOUBLE Revenue Again and Serve 1,000+ Patients in 2019 $160M-180M Guidance $91.2M $32.6M $36.9M $20.6M $80$21.3M-90M $4.9M $16.7M FY16 FY17 FY18 FY19


 
Galafold: Precision Medicine for Fabry Disease 9 Upside Potential Total Amenable Patient Population (“TAPP”) WORLDWIDE Estimate based on 35% - 50% amenability Diagnosis grows due to newborn screening in U.S. & Japan $1B+ Addressable Market TAPP: 4,700-6,750 Peak Potential Opportunity by 2028 WORLDWIDE Diagnosis continues at current rate TAPP: 4,200-6,000 Today WORLDWIDE* (U.S. & Japan Added) 2028 TAPP: 3,800-5,500 EU & ROW Only TAPP: 2,000-3,000 2018 2017 *WORLDWIDE includes total amenable patient population in all Fabry ERT commercial markets today Estimated effect of newborn screening on adult diagnostic rate.


 
AT-GAA Novel ERT for Pompe Disease “We encourage and embrace constant innovation” - Amicus Belief Statement


 
AT-GAA for Pompe Disease 11 AT-GAA: Breakthrough Therapy Designation U.S. FDA Granted BTD to AT-GAA in Late-Onset Pompe Disease (LOPD) BTD Criteria • Intended to treat a serious or life threatening disease or condition • Preliminary clinical evidence indicates drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints AT-GAA BTD Based on Ph 1/2 Clinical Efficacy • Improvements in 6-minute walk distance • Comparison to natural history of treated patients BTD Features • Intensive guidance on an efficient drug development program • Potential Rolling BLA • Organizational commitment involving senior managers • Potential for Priority Review • All Fast Track program features


 
AT-GAA for Pompe Disease 12 AT-GAA: 2019 Objectives Advance AT-GAA for as Many Patients Worldwide as Quickly as Possible  Additional Phase 1/2 Data (up to 24 Months)  Breakthrough Therapy Designation • Enroll PROPEL study (n=100) • Present additional Phase 1/2 data (Cohort 4) • Report natural history study data • Initiate supportive studies • Advance agreed upon CMC requirements to support BLA


 
Gene Therapy Pipeline “We have a duty to obsolete our own technologies” - Amicus Belief Statement


 
Amicus Pipeline 14 DISCOVERY PRECLINICAL PHASE 1/2 PHASE 3 REGULATORY COMMERCIAL Fabry Franchise Galafold®(migalastat) monotherapy Fabry Gene Therapy PENN Pompe Franchise AT-GAA (Novel ERT + Chaperone) Pompe Gene Therapy PENN Batten Franchise – Gene Therapies NCH CLN6 Batten Disease Advancing one NCH CLN3 Batten Disease of the most CLN8 Batten Disease NCH robust rare CLN1 Batten Disease NCH disease Rare CNS and Other Gene Therapies portfolios in CDKL5 Deficiency Disorder GTx / ERT PENN biotechnology Niemann-Pick Type C (NPC) NCH Tay-Sachs Disease NCH Wolman Disease NCH NCH / Other PENN


 
Amicus Gene Therapy Programs 15 Leading LSD Gene Therapy Portfolio Multiple Platforms Provide 14 Gene Therapy Programs and R&D Engine for Future Growth Gene Therapy Anticipated Milestones 2-Year CLN6 Batten Disease Phase 1/2 Data (Mid- 2019) CLN3 Batten Disease Phase 1/2 Study Enrolling CLN8 and CLN1 Batten Disease Preclinical Studies Underway Fabry and Pompe Preclinical Studies in Progress Engines for Future Growth


 
Amicus Gene Therapy Programs 16 Amicus Global Research and Gene Therapy Center of Excellence in Philadelphia Strengthens Amicus Capabilities as a Leading Global Rare Disease Biotechnology Company in Burgeoning Hub for Medical Breakthroughs » New 75,000 sqft state-of-the-art facility » Located in uCity Square Adjacent to Penn Campus » Global Amicus science organization and gene therapy leadership team headquarters » Co-led by Jeff Castelli, PhD, Chief Portfolio Officer and Head of Gene Therapy and Hung Do, PhD, CSO » Initial group occupying temporary space » Permanent space to eventually hold ~200 employees Artist rendering


 
Milestones “We have a duty to obsolete our own technologies” - Amicus Belief Statement


 
Upcoming Milestones 18 Anticipated Milestones: 2019 Well-Positioned to Create Significant Value for Shareholders and Patients in 2019 Galafold: Fabry Disease AT-GAA: Pompe Disease Gene Therapy Programs • FY19 revenue guidance $160M-  Additional Phase 1/2 data (21 • Ongoing CLN3 Batten disease $180M and 24 months) Phase 1/2 study enrollment • Growth in existing markets  Breakthrough therapy • Additional 2-year data from CLN6 designation (BTD) in LOPD Batten disease Phase 1/2 study • Expansion into new markets • PROPEL pivotal study enrollment • Preclinical proof of concept for • Diagnostic initiatives (n=100) Fabry, Pompe and CDD • Additional Phase 1/2 data • Preclinical work across additional (Cohort 4) neurologic LSDs • Natural history study data • Additional supportive studies • Advance CMC requirements to support BLA


 
A Rare Company Commercial Organizational Strength “We are business led and science driven” - Amicus Belief Statement


 
Commercial Organizational Strength 20 Global Footprint Global operations in North America EU place across the largest rare disease markets » employees supporting our global 150+ Galafold business » ~100 in Commercial » ~50 in Medical Affairs » countries with offices, including US, 11 EU5, Japan ROW APAC countries with direct presence (Amicus » 20 personnel) » 24 markets with reimbursement » markets with Amicus presence (direct or 27 distributor)


 
Commercial Organizational Strength 21 Experienced Passionate Commercial Team Proven global capabilities driving Galafold’s extraordinary launch success Diverse Experience in Background Passion for Rare Disease and Patients Drug Launch Experiences Galafold is indicated for adults with a confirmed diagnosis of Fabry Disease and an amenable mutation/variant. The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.


 
Commercial Organizational Strength 22 An Innovative Product Novel Mechanism, Precision Medicine, Commercial Execution Product Execution . Differentiated product profile – . Clear launch focus on priority first oral treatment option patient segments . Rapid determination of . Efficient outreach to key Fabry amenability (website) disease centers . Driving science through . Strong education efforts on Publications, FollowMe Registry importance of genotype and Post Marketing Studies Galafold is indicated for adults with a confirmed diagnosis of Fabry Disease and an amenable mutation/variant. The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.


 
Commercial Organizational Strength 23 Market Access Our medicines must be fairly priced and broadly accessible.” - Amicus Founding Belief . Proven patient-centric strategy of ensuring fair price and broad accessibility . Rapid, successful reimbursement in global markets . Amicus assist™ patient support program . Amicus PROMISE to limit price increases to consumer inflation . Amicus “pledge” to reinvest until there’s a cure Galafold is indicated for adults with a confirmed diagnosis of Fabry Disease and an amenable mutation/variant. The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.


 
Financial Summary Full Year Ended December 31, 2019 “Maximizing Value for Shareholders is the Foundation for Our Future Successes” - Amicus Belief Statement


 
Financial Summary 25 FY18 Select Financial Results FY18 Revenue of $91.2M Primarily from International Galafold Sales Dec. 31, 2018 (in thousands, except per share data) Dec. 31, 2017 Product revenue 91,245 36,930 Cost of goods sold 14,404 6,236 R&D expense 270,902* 149,310 SG&A expense 127,200 88,671 Changes in fair value of contingent consideration 3,300 (234,322) Loss on impairment of assets - 465,427 Loss from operations (328,777) (441,985) Income tax benefit 94 165,119 Net loss (348,995) (284,002) Net loss per share (1.88) (1.85) *Inclusive of upfront payment of $100 million for the Celenex asset acquisition


 
Financial Summary 26 Financial Summary and Guidance Strong Balance Sheet with $500M+ Cash at 12/31/18 - Cash Runway into 2021 FINANCIAL POSITION December 31, 2018 Cash ~$504M Cash Runway Into at least mid-2021 CAPITALIZATION Shares Outstanding 189,383,924 FINANCIAL GUIDANCE Projected YE 2019 Cash Balance ~$300M Galafold Revenue Guidance $160M-$180M


 
Closing 27 Impacting Lives >350 Patients* | ~$36M Global Sales >700 Patients* | ~$91M1 Global Sales 5,000 Patients* | $1B Global Sales YE17 YE18 2023 *Clinical & commercial, all figures approximate 1Preliminary unaudited


 
Appendix


 
AT-GAA for Pompe Disease 29 Pompe Patient Experience in Phase 1/2 Clinical Study (ATB200-02) Consistent and Durable Responses Across Key Measures of Safety, Functional Outcomes and Biomarkers in both ERT-Switch and ERT-Naïve Pompe Patients out to Month 18 6-Minute Walk Test (m) Change at Month 24a,b Cohort Baseline (n=10) (n=8) Mean (SD) Cohort 1 397.2 +53.6 ERT-Switch Ambulatory (96.8) (36.4) Change at Month 21 Cohort Baseline (n=5) (n=5) Mean (SD) Cohort 3 399.5 +54.8 ERT-Naïve (83.5) (34.7) FVC (% Predicted) Change at Month 24a,b,c Cohort Baseline (n=9*) (n=7) Mean (SD) Cohort 1 52.6 -0.6 ERT-Switch Ambulatory* (14.7) (2.8) Change at Month 21 Cohort Baseline (n=5) (n=5) Mean (SD) Cohort 3 53.4 +6.1 ERT-Naïve (20.3) (9.7) aOne patient in Cohort 1 discontinued from study (withdrew consent) before Month 24. bAt the time of this interim analysis, 1 patient in Cohort 1 had not reached Month 24. cBaseline FVC missing for 1 patient in Cohort 1


 
AT-GAA for Pompe Disease 30 PROPEL (ATB200-03) Study Design 52-Week Primary Treatment Period Long-Term Extension (Double-Blind) (Open-Label) Participants with Late-Onset AT-GAA Pompe Disease Bi-Weekly ~100 Patients AT-GAA 90 Clinical Sites Worldwide Bi-weekly Standard of Care ERT-Switch ERT-Naïve Bi-Weekly Primary Endpoint: 6-Minute Walk Test at Week 52 Multiple Secondary Endpoints


 
Thank You “Our passion for making a difference unites us” -Amicus Belief Statement