Amicus Therapeutics
AMICUS THERAPEUTICS INC (Form: 8-K, Received: 08/07/2017 07:25:49)

 

 

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): August 7 , 2017

 

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 August 7, 2017, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the second quarter ended June 30, 2017. A copy of this press release is attached hereto as Exhibit 99.1. The Company will also host a conference call and webcast on August 7, 2017 to discuss its second quarter results of operations. A copy of the conference call presentation materials is also attached hereto as Exhibit 99.2.

 

In accordance with General Instruction B.2. of Form 8-K, the information in this Current Report on Form 8-K and the Exhibit 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 a filing.

 

Item 9.01. Financial Statements and Exhibits.

 

(d) Exhibits:  The Exhibit Index annexed hereto is incorporated herein by reference.

 

2



 

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: August 7, 2017

By:

/s/ ELLEN S. ROSENBERG

 

 

Ellen S. Rosenberg

 

 

General Counsel and Corporate Secretary

 

3



 

EXHIBIT INDEX

 

Exhibit No.

 

Description

99.1

 

Press Release dated August 7, 2017

99.2

 

August 7, 2017 Conference Call Presentation Materials

 

4


Exhibit 99.1

 

 

Amicus Therapeutics Announces Second Quarter 2017

Financial Results and Corporate Updates

 

179 Fabry Disease Patients on Reimbursed Galafold (migalastat) as of July 31 — On Target to Reach
300 Patients by Year-End 2017

 

Migalastat NDA Submission Targeted for 4Q17

 

Phase 3 EB Topline Data and Complete Phase 1/2 Pompe Data on Track for Late 3Q17

 

CRANBURY, NJ, August 7, 2017 — Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company at the forefront of therapies for rare and orphan diseases, today announced financial results for the second quarter ended June 30, 2017. The Company also provided near-term program updates and reiterated full-year 2017 financial guidance.

 

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, stated, “During the second quarter and into the third quarter we have continued to successfully execute across all five of our strategic priorities which include the international launch for our oral precision medicine Galafold (migalastat) for Fabry disease, the advancement of our lead clinical programs in Epidermolysis Bullosa and Pompe disease, and further strengthening of our balance sheet. Our progress during the first half of this year has been driven by our focused execution and groundbreaking science which is tremendous for patients as well as our shareholders. We have created a solid foundation to continue building on this momentum as we approach several important milestones in the second half of this year. I believe that today, more than ever before, Amicus is well-positioned to become a leading global biotechnology company with the potential to deliver significant benefits to people living with rare devastating diseases.”

 

Second Quarter 2017 Financial Results

 

·                   Total revenue in the second quarter 2017 was approximately $7.2 million, a sequential increase of 71.4% from total revenue of $4.2 million in the first quarter 2017. Total revenue represents commercial sales of Galafold (migalastat) which commenced in May 2016, as well as reimbursed Expanded Access Programs (EAPs).

·                   Cash, cash equivalents, and marketable securities totaled $227.2 million at June 30, 2017 compared to $330.4 million at December 31, 2016.

·                   Total operating expenses increased to $53.2 million compared to $48.5 million for the second quarter 2016 primarily due to increases in manufacturing scale-up investments on the Pompe program.

·                   Net cash spend was $103.5 million for the six months ending June 30, 2017.

·                   Net loss was $48.1 million, or $0.34 per share, compared to a net loss of $51.1 million, or $0.40 per share, for the second quarter 2016.

 

2017 Financial Guidance

 

Cash, cash equivalents, and marketable securities totaled $227.2 million at June 30, 2017 compared to $330.4 million at December 31, 2016. The Company added to the quarter-ending cash position with $243.2 million in net proceeds from a follow on public offering in July 2017.

 

Amicus continues to expect full-year 2017 net operating cash spend of between $175 million to $200 million and full-year 2017 total net cash spend (including third-party milestone payments and capital expenditures) of between $200 million and $225 million. The current cash position, in addition to the net proceeds of $243.2 million from the follow-on public offering, is anticipated to fund ongoing operations into at least the second half of 2019.

 

Program Highlights

 

Migalastat for Fabry Disease

 

Migalastat is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic mutations. The European Commission (EC), in addition to regulatory authorities in Switzerland and Israel, have granted full approval for migalastat under the trade name Galafold. The EC approval may serve as the basis for regulatory approvals in more

 



 

than two-thirds of the global Fabry market that is outside the U.S . In the U.S., as previously announced, the FDA has confirmed that Amicus may submit a new drug application (NDA) for migalastat.

 

International Launch and Expanded Access Programs (EAP) Updates:

 

·                   179 patients (naïve and ERT-switch) on reimbursed Galafold as of July 31, 2017

·                   12 countries with reimbursement (commercial or EAP) including the top four largest EU markets

·                   Reimbursement dossiers submitted and pricing discussions are now underway in 13 countries

·                   Target of 300 patients treated with reimbursed Galafold by year-end 2017

 

Global Regulatory Updates:

 

·                   Two additional approvals secured outside the EU (Switzerland and Israel)

·                   Regulatory submissions completed in seven additional territories outside the EU, including Japan, Canada and Australia

·                   U.S. FDA confirmed NDA submission may be based on existing data (no additional gastrointestinal symptoms study required)

 

Anticipated Upcoming Fabry Disease Program Milestones:

 

·                   International commercial launch and EAPs in additional countries

·                   Additional regulatory submissions including a U.S. NDA (4Q17)

·                   Regulatory decision in Japan (1H18)

·                   Fabry ERT cell line development and optimization

 

ATB200/AT2221 for Pompe Disease

 

ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. Positive functional data in initial patients were reported during the second quarter 2017 from an ongoing global Phase 1/2 clinical study (ATB200-02) to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ATB200/AT2221. The study enrolled a total of 20 patients across three cohorts, including ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-naïve patients (Cohort 3).

 

Anticipated Upcoming Pompe Disease Program Milestones:

 

·                   Full ATB200-02 study data, including 6-month functional data in all patients (late 3Q17)

·                   Meetings with US and EU regulators

 

SD-101 for Epidermolysis Bullosa (EB)

 

SD-101 is a novel, late-stage, proprietary topical treatment with the potential to be the first approved therapy for EB. SD-101 is currently being investigated in a registration-directed Phase 3 study (ESSENCE, also known as SD-005) to support global regulatory submissions. As previously announced, Amicus has completed the analysis plan for the primary endpoints in the blinded ongoing ESSENCE study, and top-line Phase 3 data are on track for late in the third quarter of 2017. More than 95% of patients completing the primary treatment period have elected to continue in the open-label extension study

 

SD-101 was the first investigational treatment to show improvements in wound closure across all major EB types in completed Phase 2 clinical studies. SD-101 has been granted FDA Breakthrough Therapy designation, rare pediatric disease designation, and orphan drug designation.

 

Anticipated EB Program Milestones:

 

·                   Top-line Phase 3 data (late 3Q17)

 



 

Conference Call and Webcast

 

Amicus Therapeutics will host a conference call and audio webcast today, August 7, 2017 at 8:30 a.m. ET to discuss second quarter 2017 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); participant code 60127813.

 

An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/, and will be archived for 30 days. Web participants are encouraged to go to the web site 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 11:30 a.m. ET today. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); participant code 60127813.

 

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 m(2)). 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 biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.

 

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, financing plans, and the projected 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 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; and the potential that we will need

 



 

additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results for any of our product candidates. 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 previous filings with the SEC and in our Annual Report on Form 10-K for the year ended December 31, 2016 and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2017. 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

Senior Director, Investor Relations

spellegrino@amicusrx.com

(609) 662-5044

 

Media :

W2O Group

Brian Reid

breid@w2ogroup.com

(212) 257-6725

 



 

TABLE 1

 

Amicus Therapeutics, Inc.

Consolidated Statements of Operations

(Unaudited)

(in thousands, except share and per share amounts)

 

 

 

Three Months Ended June 30,

 

Six Months Ended June 30,

 

 

 

2017

 

2016

 

2017

 

2016

 

Revenue:

 

 

 

 

 

 

 

 

 

Net product sales

 

$

7,158

 

$

 

$

11,327

 

$

 

Cost of goods sold

 

1,061

 

 

1,836

 

 

Gross Profit

 

6,097

 

 

9,491

 

 

Operating Expenses:

 

 

 

 

 

 

 

 

 

Research and development

 

31,985

 

18,281

 

62,861

 

41,706

 

Selling, general and administrative

 

19,311

 

19,300

 

38,443

 

35,001

 

Changes in fair value of contingent consideration payable

 

1,050

 

10,186

 

5,628

 

13,338

 

Restructuring charges

 

 

8

 

 

58

 

Depreciation

 

812

 

767

 

1,636

 

1,440

 

Total operating expenses

 

53,158

 

48,542

 

108,568

 

91,543

 

Loss from operations

 

(47,061

)

(48,542

)

(99,077

)

(91,543

)

Other income (expenses):

 

 

 

 

 

 

 

 

 

Interest income

 

753

 

331

 

1,512

 

638

 

Interest expense

 

(4,179

)

(1,055

)

(8,469

)

(2,000

)

Other income (expense)

 

2,400

 

(2,237

)

3,010

 

(2,289

)

Loss before income tax (expense)/benefit

 

(48,087

)

(51,503

)

(103,024

)

(95,194

)

Income tax (expense)/ benefit

 

(49

)

453

 

(105

)

453

 

Net loss attributable to common stockholders

 

$

(48,136

)

$

(51,050

)

$

(103,129

)

(94,741

)

Net loss attributable to common stockholders per common share — basic and diluted

 

$

(0.34

)

$

(0.40

)

$

(0.72

)

(0.75

)

Weighted-average common shares outstanding — basic and diluted

 

143,000,718

 

129,122,175

 

142,886,614

 

127,160,943

 

 



 

TABLE 2

 

Amicus Therapeutics, Inc.

Consolidated Balance Sheets

(Unaudited)

(in thousands, except share and per share amounts)

 

 

 

June 30,

 

December 31,

 

 

 

2017

 

2016

 

Assets

 

 

 

 

 

Current assets:

 

 

 

 

 

Cash and cash equivalents

 

$

37,394

 

$

187,026

 

Investments in marketable securities

 

189,838

 

143,325

 

Accounts receivable

 

3,786

 

1,304

 

Inventories

 

3,948

 

3,416

 

Prepaid expenses and other current assets

 

6,023

 

4,993

 

Total current assets

 

240,989

 

340,064

 

Property and equipment, less accumulated depreciation of $ 13,951 and $12,495 at June 30, 2017 and December 31, 2016, respectively

 

10,471

 

9,816

 

In-process research & development

 

486,700

 

486,700

 

Goodwill

 

197,797

 

197,797

 

Other non-current assets

 

3,009

 

2,468

 

Total Assets

 

$

938,966

 

$

1,036,845

 

 

 

 

 

 

 

Liabilities and Stockholders’ Equity

 

 

 

 

 

Current liabilities:

 

 

 

 

 

Accounts payable, accrued expenses, and other current liabilities

 

$

35,645

 

$

41,008

 

Deferred reimbursements, current portion

 

18,850

 

13,850

 

Contingent consideration payable, current portion

 

46,188

 

56,101

 

Total current liabilities

 

100,683

 

110,959

 

Deferred reimbursements

 

16,906

 

21,906

 

Convertible notes

 

159,171

 

154,464

 

Contingent consideration payable

 

219,162

 

213,621

 

Deferred income taxes

 

173,869

 

173,771

 

Other non-current liability

 

2,283

 

1,973

 

Commitments and contingencies

 

 

 

 

 

Stockholders’ equity:

 

 

 

 

 

Common stock, $0.01 par value, 250,000,000 shares authorized, 143,371,243 and 142,691,986 shares issued and outstanding at June 30, 2017 and December 31, 2016, respectively

 

1,485

 

1,480

 

Additional paid-in capital

 

1,132,229

 

1,120,156

 

Accumulated other comprehensive loss:

 

 

 

 

 

Foreign currency translation adjustment, less tax expense of $ 1,293 at June 30, 2017 and December 31, 2016

 

(192

)

1,945

 

Unrealized gain on available-for securities

 

31

 

102

 

Warrants

 

16,076

 

16,076

 

Accumulated deficit

 

(882,737

)

(779,608

)

Total stockholders’ equity

 

266,892

 

360,151

 

Total Liabilities and Stockholders’ Equity

 

$

938,966

 

$

1,036,845

 

 

FOLD—G

 


Exhibit 99.2

2Q17 Financial Results Conference Call & Webcast August 7, 2017

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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 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, financing plans, and the projected 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 presentation 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 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; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results for any of our product candidates. 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 previous filings with the SEC and in our Annual Report on Form 10-K for the year ended December 31, 2016 and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2017. 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 presentation to reflect events or circumstances after the date hereof. Introduction

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2017 Key Strategic Priorities Introduction We Remain Sharply Focused on FIVE Key Strategic Priorities as We Continue to Build a Top Global Biotechnology Company Focused on Rare Devastating Diseases Advance International Galafold Launch Submit Japanese and U.S. New Drug Applications for Migalastat Establish Definitive Proof of Concept for ATB200/AT2221 with Clear Path to Registration for Pompe Disease Successfully Complete Phase 3 EB Study Maintain Financial Strength

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Pompe Phase 1/2 data cascade Pompe end of Phase 2 meeting Runway into 2H18 Meaningful revenue contribution from Galafold EB Phase 3 data Galafold international launch continues Japan NDA submission Fabry GI study initiation 2017 Key Anticipated Milestones in 2017 Introduction Phase 1/2 data cascade in 2Q and 3Q Meetings with U.S. and EU regulators Significant revenue contribution Cash runway into 2H19 Phase 3 top-line data 3Q17 Japan NDA submission in 2Q17 U.S. NDA submission in 4Q17 300 patients on reimbursed Galafold by YE17* Pompe Disease (ATB200/AT2221) Strong Balance Sheet Epidermolysis Bullosa (EB) (SD-101) Fabry Disease (Galafold) *Commercial and Expanded Access Programs (EAPs)

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Galafold™ (Migalastat) Precision Medicine for Fabry Disease Continue Successful Launch Execution and Geographic Expansion

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Successful International Launch Underway (as of 7/31/17) Driven by Top EU5 Countries, Galafold is Quickly Reaching ERT-Switch & Naïve Patients, Reimbursement Now Available in 12 Countries* Galafold: Precision Medicine for Fabry Disease Patients (Switch & Naïve) on reimbursed Galafold (7/31/17) 179 Countries with pricing discussions ongoing 13 Countries with available reimbursement* 12 Countries with Amicus footprint 27 Target Number of Patients on Reimbursed Galafold by YE17 300 *Commercial and Expanded Access Programs (EAPs)

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Global Regulatory Strategy to Reach More Patients Galafold: Precision Medicine for Fabry Disease EU Approval and Launch (May 30, 2016) 2 Expanded Access Programs (EAP) with More Initiated EU Approval and Planned U.S. NDA Submission Provide Pathway to Reach Global Fabry Market 7 Additional Regulatory Submissions Complete, Process Initiated in Other Key Geographies U.S. NDA Submission Targeted 4Q17 Japanese NDA Submitted 2Q17 2 Additional Approvals (Switzerland, Israel) * Two EAPs converted to commercial reimbursement

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Financial Summary

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2Q17 Select Financial Results Financial Summary ($000s) except per share data June 30, 2017 June 30, 2016 Product revenue $7,158 - R&D Expense $31,985 $18,281 SG&A Expense $19,311 $19,300 Net Loss $(48,136) $(51,050) Net Loss Per Share $(0.34) $(0.40) 2Q17 Revenue of $7.2M from Sales of Galafold

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Financial Summary & Guidance Financial Summary Financial Position June 30, 2017 Cash $227M Debt $250M FY17 Net Operating Cash Spend Guidance $175-$200M FY17 Net Cash Spend Guidance* $200-$225M Cash Runway 2H19 Capitalization July 25, 2017 Shares Outstanding 164,566,069 Cash Position Provides Runway Under Current Operating Plan into mid-2017 Strong Balance Sheet with $227M Cash at 6/30/17 and Cash Runway Into 2H19 *Includes third party milestone payments and capital expenditures

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Closing Remarks

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Pompe Phase 1/2 data cascade Pompe end of Phase 2 meeting Runway into 2H18 Meaningful revenue contribution from Galafold EB Phase 3 data Galafold international launch continues Japan NDA submission Fabry GI study initiation 2017 Key Anticipated Milestones in 2017 Conclusion Phase 1/2 data cascade in 2Q and 3Q Meetings with U.S. and EU regulators Significant revenue contribution Cash runway into 2H19 Phase 3 top-line data 3Q17 Japan NDA submission in 2Q17 U.S. NDA submission in 4Q17 300 patients on reimbursed Galafold by YE17* Pompe Disease (ATB200/AT2221) Strong Balance Sheet Epidermolysis Bullosa (EB) (SD-101) Fabry Disease (Galafold) *Commercial and Expanded Access Programs (EAPs)

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Our Vision – Maximizing Impact on Patients to Drive Shareholder Value Conclusion 13 2010 2014 2018 YE2016 2023 ~5,000 Patients* ~800 Patients* ~250 Patients* ~90 Patients ~37 Patients The Ultimate Measure of Our Success Will be the Number of Patients with Devastating Rare Diseases Treated with an Amicus Medicine = 20 patients *Clinical & Commercial

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Thank You

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