Amicus Therapeutics
Mar 1, 2017
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Amicus Therapeutics Announces Full-Year 2016 Financial Results and Corporate Updates

Growing Momentum for EU Galafold (Migalastat) Launch for Fabry Disease Tracking Toward 300 Patients by Year-End 2017

 Target Enrollment Achieved in Phase 1/2 Pompe Study - Additional Data Expected in 2Q17 and 3Q17

Phase 3 EB Program Remains on Track for Topline Data in Mid-2017

CRANBURY, N.J., March 01, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biotechnology company at the forefront of therapies for rare and orphan diseases, today announced financial results for the full year ended December 31, 2016. The Company also provided program updates and reiterated full-year 2017 financial guidance.

"Throughout 2016 we made significant progress with the international launch of our first commercial product Galafold and continued to advance and expand our robust pipeline of first- and/or best-in-class medicines for people living with devastating rare diseases," stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. "During 2017 we are laser focused on five key strategic priorities to advance our vision to develop and deliver great medicines for patients and to create significant shareholder value: 1) advancing the international launch of Galafold for Fabry disease, 2) completing our regulatory submission for migalastat in Japan (J-NDA), 3) establishing our novel Pompe treatment paradigm ATB200/AT2221 as a highly differentiated therapy, 4) successfully completing our Phase 3 clinical study in patients with epidermolysis bullosa, and 5) maintaining our financial strength. With one commercial-stage medicine and two medicines in clinical development, as well as a biologics platform for future growth, we are building a leading global biotechnology company focused on delivering meaningful benefits for patients living with devastating rare diseases."

2016 Full-Year Financial Results

2017 Financial Guidance

Cash, cash equivalents, and marketable securities totaled $330.4 million at December 31, 2016. As previously announced, the Company strengthened the balance sheet during 2016 with a $100 million at-the-market (ATM) equity financing as well as a $250 million convertible debt offering. The Company expects full-year 2017 net operating cash spend of between $175 million to $200 million and expects 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:

Regulatory Updates:

Anticipated Upcoming Fabry Disease Program Milestones:

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 in the fourth quarter of 2016  and during the 13th Annual WORLDSymposium™ in San Diego, CA in February 2017 from a global clinical study (ATB200-02) to evaluate safety, tolerability, PK, and pharmacodynamics (PD) of ATB200/AT2221. The study is enrolling 3 cohorts of patients, including ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-naïve patients (Cohort 3).

Key Preliminary Data Highlights from ATB200-02 Study in Initial ERT-Switch and ERT-Naïve Patients:

Anticipated Upcoming Pompe Disease Program Milestones:

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:

Anticipated EB Program Milestones:

Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, March 1, 2017 at 8:30 a.m. ET to discuss full-year 2016 financial results and corporate updates. Interested participants and investors may access the conference call at 8:00 a.m. ET by dialing 877-303-5859 (U.S./ Canada) or 678-224-7784 (international) participant code 77299407.

An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://www.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 77299407.

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.

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. In particular, this press release relates to the preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221. The inclusion of forward-looking statements arising from this preliminary data and study 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. The preliminary data and Phase 1/2 study discussed herein is inherently preliminary and early in the study, derived from a limited patient set, and later trial results with this patient set or others may not be consistent with these preliminary results. With respect to statements regarding projections of the Company's cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our previous filings with the SEC and in our Annual Report on Form 10-K for the year ended December 31, 2016 to be filed later 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.


TABLE 1
Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
   
  Years Ended December 31,
   2016   2015   2014 
Revenue:      
Net Product Sales $4,958  $  —  $  — 
Research revenue        1,224 
Total revenue  4,958      1,224 
Cost of goods sold   833      
       
Gross Profit  4,125      1,224 
Operating Expenses:      
Research and development  104,793   76,943   47,624 
Selling, general and administrative  71,151    47,269   20,717 
Changes in fair value of contingent consideration payable   6,760   4,377   100 
Restructuring charges  69   15   (63)
Depreciation  3,242   1,833   1,547 
Total operating expenses  186,015   130,437   69,925 
Loss from operations   (181,890)  (130,437)  (68,701)
Other income (expenses):      
Interest income  1,602   929   223 
Interest expense  (5,398)  (1,578)  (1,484)
Loss on extinguishment of debt  (13,302)  (952)   
Other expense  (4,793)   (80)  (77)
Loss before income tax benefit  (203,781)  (132,118)  (70,039)
Income tax benefit  3,739      1,113 
       
Net loss attributable to common stockholders $(200,042) $(132,118) $(68,926)
Net loss attributable to common stockholders per common share — basic and diluted $(1.49) $(1.20) $(0.93)
Weighted‑average common shares outstanding — basic and diluted  134,401,588   109,923,815   74,444,157 

                                                                                                           

TABLE 2
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)

   
  December 31,
   2016   2015 
Assets    
Current assets:    
Cash and cash equivalents  $187,026  $69,485 
Investments in marketable securities   143,325   144,548 
Accounts receivable   1,304    
Inventories   3,416    
Prepaid expenses and other current assets   4,993   2,568 
Total current assets   340,064   216,601 
Property and equipment, less accumulated depreciation of $12,495 and $13,353 at December 31, 2016 and 2015, respectively   9,816   6,178 
In-process research & development    486,700   486,700 
Goodwill   197,797   197,797 
Other non-current assets    2,468   1,108 
Total Assets  $  1,036,845  $908,384 
     
Liabilities and Stockholders' Equity    
Current liabilities:    
Accounts payable, accrued expenses, and other current liabilities  $41,008  $32,216 
Deferred reimbursements, current portion  13,850       — 
Contingent consideration payable, current portion   56,101   41,400 
Total current liabilities   110,959   73,616 
Deferred reimbursements   21,906    35,756 
Convertible notes   154,464     — 
Due to related party     41,601 
Contingent consideration payable  213,621   232,677 
Deferred income taxes  173,771   176,219 
Other non-current liability   1,973   681 
Commitments and contingencies    
Stockholders' equity:    
Common stock, $.01 par value, 250,000,000 shares authorized,
142,691,986  shares issued and outstanding at December 31, 2016
Common stock, $.01 par value, 250,000,000 shares authorized,
125,027,034  shares issued and outstanding at December 31, 2015
  1,480   1,306 
Additional paid-in capital   1,120,156   917,454 
Accumulated other comprehensive loss:    
Foreign currency translation adjustment, less tax benefit of $1,293 at December 31, 2016   1,945    
Unrealized gain/ (loss) on available-for securities  102   (115)
Warrants    16,076   8,755 
Accumulated deficit   (779,608)  (579,566)
Total stockholders' equity   360,151   347,834 
Total Liabilities and Stockholders' Equity  $1,036,845  $908,384 
         

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CONTACTS:



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Amicus Therapeutics

Sara Pellegrino

Senior Director, Investor Relations

spellegrino@amicusrx.com

(609) 662-5044



Media:

MWW PR

Sid Dinsay

sdinsay@mww.com

(646) 381-9017

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