Amicus Therapeutics
AMICUS THERAPEUTICS INC (Form: 8-K, Received: 05/09/2017 08:03:08)

 

 

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): May 9 , 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 May 9, 2017, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the first quarter ended March 31, 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 May 9, 2017 to discuss its first 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.

 

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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: May 9, 2017

By:

/s/ ELLEN S. ROSENBERG

 

 

Ellen S. Rosenberg

 

 

General Counsel and Corporate Secretary

 

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EXHIBIT INDEX

 

Exhibit No.

 

Description

99.1

 

Press Release dated May 9, 2017

99.2

 

May 9, 2017 Conference Call Presentation Materials

 

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Exhibit 99.1

 

 

Amicus Therapeutics Announces First Quarter 2017

Financial Results and Corporate Updates

 

100+ Fabry Disease Patients Now on Reimbursed Galafold (migalastat) in the EU

 

On Target to Reach 300 Patients on Galafold by Year-End 2017

 

Additional Important Phase 1/2 Pompe Clinical Data Expected in 2Q17 and 3Q17

 

Phase 3 EB Topline Data on Track for 3Q17

 

CRANBURY, NJ, May 9, 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 first quarter ended March 31, 2017. The Company also provided program updates and reiterated full-year 2017 financial guidance.

 

“During the first quarter we continued to advance the international commercial launch of our oral precision medicine Galafold for Fabry disease, as we brought Galafold to more Fabry patients in Germany and other European markets with both new ERT switch patients as well as a notable proportion of ERT-naïve patients,” stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “We are extremely pleased with the progress of our Galafold launch in terms of patient and physician adoption, as well as pricing and reimbursement. With launch now just initiating this month in the UK, Italy, France, and additional markets, we are confident in achieving our target of 300 patients on Galafold by the end of this year. In addition to the launch, we have also made significant progress on our other four key strategic priorities for 2017: 1) targeting a Japanese new drug application (J-NDA) submission for migalastat in the second quarter, 2) establishing definitive clinical proof of concept for our novel Pompe treatment paradigm ATB200/AT2221, 3) completing our Phase 3 clinical study in patients with epidermolysis bullosa, and 4) maintaining our financial strength. We are particuarly looking forward to sharing additional, important Pompe clinical data this quarter. We continue to believe that our Pompe program will be a crucial driver toward our vision of becoming one of the world’s leading global rare disease biotechnology companies.”

 

First Quarter 2017 Financial Results

 

·                   Total product revenue in the first quarter 2017 was approximately $4.2 million, a sequential increase of 50% over total product revenue of $2.8 million in the fourth quarter 2016. Total product revenue for both periods 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 $279.8 million at March 31, 2017 compared to $330.4 million at December 31, 2016.

·                   Total operating expenses increased to $55.4 million compared to $43.0 million for the first quarter 2016 primarily due to increases in commercial costs of the Fabry monotherapy program and manufacturing scale-up on the Pompe program.

·                   Net cash spend was $50.5 million.

·                   Net loss was $55.0 million, or $0.39 per share, compared to a net loss of $43.7 million, or $0.35 per share, for the first quarter 2016.

 

2017 Financial Guidance

 

Cash, cash equivalents, and marketable securities totaled $279.8 million at March 31, 2017. The Company 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 is anticipated to fund ongoing operations into the second half of 2018.

 

Program Highlights

 

Migalastat for Fabry Disease

 

Migalastat is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic mutations. As previously announced, the European Commission (EC) has 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 . The Company has also defined a U.S. pathway to support full approval.

 

International Launch and Expanded Access Programs (EAP) Updates:

 

·                   101 patients (naïve and ERT-switch) on reimbursed Galafold as of April 30, 2017

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

·                   Reimbursement dossiers submitted and pricing discussions are now underway in 12 countries

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

 

Regulatory Updates:

 

·                   One additional approval secured outside EU (Switzerland)

·                   Regulatory submissions completed in six additional territories outside EU

 

Anticipated Upcoming Fabry Disease Program Milestones:

 

·                   EU commercial launch in additional countries and EAP in additional territories

·                   Additional regulatory submissions including a Japanese regulatory submission (J-NDA) (2Q17)

·                   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 preliminary data were reported from a global Phase 1/2 clinical study (ATB200-02) to evaluate safety, tolerability, PK, and pharmacodynamics (PD) of ATB200/AT2221. The study is fully enrolled with 3 cohorts of patients, 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:

 

·                   Additional ATB200-02 study data (2Q17 and 3Q17)

·                   Meetings with US and EU regulators

 

SD-101 for Epidermolysis Bullosa (EB)

 

SD-101 is a novel, late-stage, proprietary topical treatment and potential first-to-market 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.

 

SD-101 was granted FDA Breakthrough Therapy designation in 2013 based on results from a Phase 2a study for the treatment of lesions in patients suffering with EB. SD-101 is the first-ever treatment in clinical studies to show improvements in wound closure across all major EB types.

 

EB Phase 3 ESSENCE Study Highlights:

 

·                   Enrollment complete and target exceeded with more than 160 patients

·                   More than 95% of patients completing the primary treatment period have elected to continue in the open-label extension study

 

Anticipated EB Program Milestones:

 

·                   Agreement on final statistical analysis plan (SAP) with U.S. Food and Drug Administration (FDA)

·                   Top-line Phase 3 data (3Q17)

 



 

Conference Call and Webcast

 

Amicus Therapeutics will host a conference call and audio webcast today, May 9, 2017 at 8:30 a.m. ET to discuss first 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).

 

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 16795077.

 

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

 

1



 

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. 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

Senior Director, Investor Relations

spellegrino@amicusrx.com

(609) 662-5044

 

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TABLE 1

Amicus Therapeutics, Inc.

Consolidated Statements of Operations

(in thousands, except share and per share amounts)

 

 

 

Three Months Ended March 31,

 

 

 

2017

 

2016

 

Revenue:

 

 

 

 

 

Net product sales

 

$

4,169

 

$

 

Cost of goods sold

 

775

 

 

Gross Profit

 

3,394

 

 

Operating Expenses:

 

 

 

 

 

Research and development

 

30,876

 

23,425

 

Selling, general and administrative

 

19,132

 

15,701

 

Changes in fair value of contingent consideration payable

 

4,578

 

3,152

 

Restructuring charges

 

 

50

 

Depreciation

 

823

 

673

 

Total operating expenses

 

55,409

 

43,001

 

Loss from operations

 

(52,015

)

(43,001

)

Other income (expenses):

 

 

 

 

 

Interest income

 

759

 

307

 

Interest expense

 

(4,290

)

(945

)

Other income (expense)

 

610

 

(52

)

Loss before income tax benefit

 

(54,936

)

(43,691

)

Income tax expense

 

(56

)

 

Net loss attributable to common stockholders

 

$

(54,992

)

$

(43,691

)

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

 

$

(0.39

)

$

(0.35

)

Weighted-average common shares outstanding — basic and diluted

 

142,770,629

 

125,178,517

 

 



 

TABLE 2

 

Amicus Therapeutics, Inc.

Consolidated Balance Sheets

(in thousands, except share and per share amounts)

 

 

 

March 31,

 

December 31,

 

 

 

2017

 

2016

 

Assets

 

 

 

 

 

Current assets:

 

 

 

 

 

Cash and cash equivalents

 

$

44,755

 

$

187,026

 

Investments in marketable securities

 

235,087

 

143,325

 

Accounts receivable

 

1,875

 

1,304

 

Inventories

 

3,698

 

3,416

 

Prepaid expenses and other current assets

 

10,792

 

4,993

 

Total current assets

 

296,207

 

340,064

 

Property and equipment, less accumulated depreciation of $13,316 and $12,495 at March 31, 2017 and December 31, 2016, respectively

 

9,745

 

9,816

 

In-process research & development

 

486,700

 

486,700

 

Goodwill

 

197,797

 

197,797

 

Other non-current assets

 

2,932

 

2,468

 

Total Assets

 

$

993,381

 

$

1,036,845

 

 

 

 

 

 

 

Liabilities and Stockholders’ Equity

 

 

 

 

 

Current liabilities:

 

 

 

 

 

Accounts payable, accrued expenses, and other current liabilities

 

$

39,645

 

$

41,008

 

Deferred reimbursements, current portion

 

13,850

 

13,850

 

Contingent consideration payable, current portion

 

56,101

 

56,101

 

Total current liabilities

 

109,596

 

110,959

 

Deferred reimbursements

 

21,906

 

21,906

 

Convertible notes

 

156,859

 

154,464

 

Contingent consideration payable

 

218,199

 

213,621

 

Deferred income taxes

 

173,820

 

173,771

 

Other non-current liability

 

2,223

 

1,973

 

Commitments and contingencies

 

 

 

 

 

Stockholders’ equity:

 

 

 

 

 

Common stock, $0.01 par value, 250,000,000 shares authorized, 142,829,530 and 142,691,986 shares issued and outstanding at March 31, 2017 and December 31, 2016, respectively

 

1,482

 

1,480

 

Additional paid-in capital

 

1,126,148

 

1,120,156

 

Accumulated other comprehensive loss:

 

 

 

 

 

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

 

1,487

 

1,945

 

Unrealized gain/ (loss) on available-for securities

 

185

 

102

 

Warrants

 

16,076

 

16,076

 

Accumulated deficit

 

(834,600

)

(779,608

)

Total stockholders’ equity

 

310,778

 

360,151

 

Total Liabilities and Stockholders’ Equity

 

$

993,381

 

$

1,036,845

 

 


Exhibit 99.2

1Q17 Financial Results & Corporate Updates Conference Call & Webcast May 9, 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. 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

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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 New Drug Application (J-NDA) 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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Galafold™ (Migalastat) Precision Medicine for Fabry Disease Continue Launch Execution and Geographic Expansion

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Successful International Launch Underway (as of 4/30/17) Initial Launch Success Driven by Germany with ERT-Switch & Naïve Patients, Reimbursement Now Available in 12 Countries Including Four of Top EU5* Galafold: Precision Medicine for Fabry Disease Patients (Switch & Naïve) on reimbursed Galafold (4/30/17) 101 Countries with pricing discussions ongoing 12 Countries with available reimbursement* 11 Countries with Amicus footprint 27 *Commercial and Expanded Access Programs (EAPs) Target Number of Patients on Reimbursed Galafold by YE17 300

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German Launch Update (as of 4/30/17) Galafold: Precision Medicine for Fabry Disease Germany is an Important Indicator for EU Launch Success *Market share assumptions based on estimated number of ERT-treated patients and naïve patients with amenable mutations in Germany as of April 2017 IMPORTANT EARLY INDICATORS IN GERMANY Majority switch patients, but growing naïve segment ~45% share of amenable patients (switch and naïve)* Switches from both Fabrazyme & Replagal™ commensurate with market share Male / female mix Most major centers prescribing Final price to be effective in 2Q17 Galafold ~45% ERT ~55% Current Approximate Market Share*

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UK Market Dynamics Galafold: Precision Medicine for Fabry Disease Galafold Positioned for Success Following Positive Final NICE Publication and more than a Decade of Clinical Experience Among Largest Treatment Centers * Estimates based on detailed market mapping and physician chart reviews **Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16 - www.nice.org.uk/guidance/hst4 MARKET DYNAMICS IN THE UK Funding effective May 23, 2017 Highly concentrated at major centers Clinical experience at multiple sites ~450 ERT-treated patients 50%+ amenability rate projected* “Migalastat has a lower total cost than ERT, and potentially provides greater health benefits than ERT.” -NICE Highly Specialised Technologies Guidance [HST4]**

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EU Launch Strategy INITIAL FOCUS ON TOP 5 COUNTRIES Launched in Germany, UK, Italy and France Spain reimbursement discussions underway ~2,000 Fabry patients treated ~70-75% of EU market value ~25% of global Fabry market Focus on EU Top 5 Plus Key Mid-Sized EU Markets in 2017 Galafold: Precision Medicine for Fabry Disease INVEST IN KEY MID-SIZED EU COUNTRIES AND SELECT EAP OPPORTUNITIES Austria, Nordics, Netherlands, Belgium, etc. ~10% of EU market value Selectively invest in key EAP markets

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Global Regulatory Strategy to Reach More Patients Galafold: Precision Medicine for Fabry Disease EU Approval and Launch (May 30, 2016) Active Expanded Access Programs (EAP) in 2* Territories with More Initiated EU Approval is Gateway to ~75% of Global ERT Market 6 Additional Regulatory Submissions Complete, Process Initiated in Other Key Geographies U.S. Full Approval Pathway Defined by Amicus Japanese NDA Targeted 2Q17 1 Additional Approval (Switzerland) * Two EAPs converted to commercial reimbursement

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ATB200 Novel ERT for Pompe Disease Establishing Human Proof of Concept and Validating Biologics Platform in 2017

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Novel Pompe Treatment Paradigm with Three Key Differentiators Novel ERT for Pompe Disease – ATB200 + Chaperone Chaperone addition Optimized mixture of glycans ATB200 + Chaperone: A Highly Differentiated Approach ATB200 (Novel ERT) High levels of M6P and bis M6P *Artist rendering, not actual product image

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Phase 1/2 ATB200-02 Study Design Pompe Phase 1/2 Study ATB200-02 Preliminary Data Phase 1/2 Clinical Study to Evaluate Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of ATB200 + Chaperone (ATB200/AT2221) 18-Week Primary Treatment Period with Long-Term Extension (n=20) ATB200 5mg/kg (wk 2) 10mg/kg (wk 4) 20mg/kg (wk 6) ATB200 20mg/kg + AT2221 (Low Dose) wks 8,10,12 ATB200 20mg/kg + AT2221 (High Dose) wk 14+ Cohort 1 (Ambulatory ERT-Switch, n=11) Cohort 2 (Non-Ambulatory ERT-Switch, n=4) & Cohort 3 (ERT-Naïve, n=5) ATB200 20mg/kg + AT2221 (High Dose) wk 2+ Plasma PK Safety/Tolerability Infusion-Associated Reactions Antibody & Cytokine Levels Pharmacodynamics Efficacy (Long-Term Extension) Assessments:

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Preliminary Clinical Data Summary (as of February 2017) Safety (N=13)* No serious adverse events (SAEs) related to ATB200/AT2221 AEs were generally mild and transient Tolerability No infusion-associated reactions following 150+ infusions in all patients enrolled to date PK (N=10)** Clinical PK profile as predicted consistent with previously reported preclinical data Biomarkers of muscle damage (CK, AST, ALT) and substrate (urine Hex4) (N=10)** Decrease or normalization of muscle injury biomarkers in a majority of patients Decreases in urine Hex4 in all patients Improvement in all biomarkers suggests positive effect of ATB200/AT2221 on muscle cells Novel ERT for Pompe Disease – ATB200 + Chaperone ATB200/AT2221 Demonstrates Promising Preliminary Results in Initial ERT-Switch and Naïve Patients at the Targeted Therapeutic Dose *N=10 from Cohort 1 (Ambulatory ERT-Switch); N=1 from Cohort 2 (Non-Ambulatory ERT-Switch); N=2 from Cohort 3 (Naïve) **N=8 from Cohort 1 & N=2 from Cohort 3

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Pompe Clinical Study ATB200-02 Data Cascade Novel ERT for Pompe Disease – ATB200 + Chaperone A Cascade of Additional Data Points on Track for 2Q17 and 3Q17 to Demonstrate Proof of Concept Pompe Milestones in 2017 Meeting with U.S. and EU regulators 18-WEEK DATA Safety / tolerability Pharmacokinetics (PK) EXTENSION DATA Motor/pulmonary function Additional extension study data (all Cohorts) Data in ERT-naïve patients (Cohort 3) Data in non-ambulatory ERT-switch patients (Cohort 2) Additional data & initial extension data in Cohort 1 Biomarkers Immunogenicity

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SD-101 for Epidermolysis Bullosa Potential First-in-Class Treatment with Phase 3 Data Anticipated 3Q17

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Phase 3 ESSENCE Study - Delivering on Our EB Vision Phase 3 Study Overenrolled (>160 Patients) with Top-Line Data On Track for 3Q17 SD-101 for EB SD-005 Study Design Optimized 95%+ participation in extension study Study overenrolled (>160 patients) Top-line data anticipated 3Q17 Status Sample size of up to 150 patients Larger baseline target wound size Time to wound closure endpoint elevated

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Financial Summary

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1Q17 Select Financial Results Financial Summary March 31, 2017 March 31, 2016 Product revenue $4.2m - R&D Expense $30.9m $23.4m SG&A Expense $19.1m $15.7m Net Loss ($55.0) ($43.7) Net Loss Per Share ($0.39) ($0.35) Continue to Focus on Revenue of $4.2M from Sales of Galafold

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Financial Summary & Guidance Financial Summary Financial Position March 31, 2017 Cash $279.8M Debt $250M FY17 Net Operating Cash Flow Guidance $175-$200M FY17 Net Cash Spend Guidance* $200-$225M Cash Runway 2H18 Capitalization March 31, 2017 Shares Outstanding 142,829,530 Cash Position Provides Runway Under Current Operating Plan into mid-2017 Strong Balance Sheet with $279.8M Cash at 3/31/17 and Cash Runway Into 2H18 *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 2H18 Phase 3 top-line data 3Q17 300 patients on reimbursed Galafold by YE17* Japan NDA submission in 2Q17 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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Building a Top Global Biotech in Devastating Rare Diseases FIRST ORAL PRECISION MEDICINE FOR FABRY DISEASE WORLD CLASS SCIENCE & DRUG DEVELOPMENT ATB200/AT2221 NOVEL TREATMENT PARADIGM FOR POMPE IN PHASE 1/2 ~$280M CASH BALANCE Two Phase 3 PROGRAMS (FABRY & EB) 1 BREAKTHROUGH THERAPY DESIGNATION TREATING PATIENTS IN 24 COUNTRIES 3 PROGRAMS IN CLINIC IN 3 RARE DISEASES $3B+ MARKET OPPORTUNITY FOR CURRENT PIPELINE PROTEIN ENGINEERING & GLYCOBIOLOGY

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Thank You

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