|
Delaware
|
|
71-0869350
|
(State or Other Jurisdiction of
|
|
(I.R.S. Employer
|
Incorporation or Organization)
|
|
Identification Number)
|
|
|
|
1 Cedar Brook Drive, Cranbury, NJ
|
|
08512
|
(Address of Principal Executive Offices)
|
|
(Zip Code)
|
|
|
|
(609) 662-2000
|
||
(Registrant's Telephone Number, Including Area Code)
|
Title of each class
|
Trading Symbol(s)
|
Name of each exchange on which registered
|
Common Stock
|
FOLD
|
NASDAQ Global Market
|
Large accelerated filer
x
|
|
Accelerated filer
o
|
Non-accelerated filer
o
|
|
Smaller reporting company
o
|
|
|
Emerging growth company
o
|
|
|
Page
|
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|
Item 1.
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Item 2.
|
|||
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Item 3.
|
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Item 4.
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|
Item 1.
|
|||
|
|
|
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|
|
Item 1A.
|
|||
|
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|
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Item 2.
|
|||
|
|
|
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|
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Item 3.
|
|||
|
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|
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Item 4.
|
|||
|
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Item 5.
|
|||
|
|
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|
|
Item 6.
|
|||
|
|
|||
|
|
|||
|
|
•
|
the progress and results of our preclinical and clinical trials of our drug candidates;
|
•
|
the cost of manufacturing drug supply for our clinical and preclinical studies, including the cost of manufacturing Pompe Enzyme Replacement Therapy ("ERT") and gene therapies;
|
•
|
the scope, progress, results and costs of preclinical development, laboratory testing and clinical trials for our product candidates including those testing the use of pharmacological chaperones co-formulated and co-administered with ERT and for the treatment of lysosomal storage disorders and gene therapies for the treatment of rare genetic metabolic diseases;
|
•
|
the future results of on-going preclinical research and subsequent clinical trials for cyclin-dependent kinase-like 5 ("CDKL5") deficiency, including our ability to obtain regulatory approvals and commercialize CDKL5 therapies and obtain market acceptance for such therapies;
|
•
|
the costs, timing and outcome of regulatory review of our product candidates;
|
•
|
the number and development requirements of other product candidates that we pursue;
|
•
|
the costs of commercialization activities, including product marketing, sales and distribution;
|
•
|
the emergence of competing technologies and other adverse market developments;
|
•
|
our ability to successfully commercialize Galafold
®
("migalastat HCl");
|
•
|
our ability to manufacture or supply sufficient clinical or commercial products;
|
•
|
our ability to obtain reimbursement for Galafold
®
;
|
•
|
our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold
®
;
|
•
|
our ability to obtain market acceptance of Galafold
®
;
|
•
|
the costs of preparing, filing and prosecuting patent applications and maintaining, enforcing and defending intellectual property-related claims;
|
•
|
the extent to which we acquire or invest in businesses, products and technologies;
|
•
|
our ability to successfully integrate our acquired products and technologies into our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected;
|
•
|
our ability to establish collaborations and obtain milestone, royalty or other payments from any such collaborators;
|
•
|
our ability to adjust to changes in European and United Kingdom markets as the United Kingdom leaves the European Union;
|
•
|
fluctuations in foreign currency exchange rates; and
|
•
|
changes in accounting standards.
|
|
March 31,
2019 |
|
December 31,
2018 |
||||
Assets
|
|
|
|
||||
Current assets:
|
|
|
|
||||
Cash and cash equivalents
|
$
|
96,349
|
|
|
$
|
79,749
|
|
Investments in marketable securities
|
341,978
|
|
|
424,403
|
|
||
Accounts receivable
|
22,960
|
|
|
21,962
|
|
||
Inventories
|
8,167
|
|
|
8,390
|
|
||
Prepaid expenses and other current assets
|
13,592
|
|
|
16,592
|
|
||
Total current assets
|
483,046
|
|
|
551,096
|
|
||
Operating and finance lease right-of-use assets, less accumulated amortization of $2,111 and $0 at March 31, 2019 and December 31, 2018, respectively
|
36,308
|
|
|
—
|
|
||
Property and equipment, less accumulated depreciation of $15,794 and $15,671 at March 31, 2019 and December 31, 2018, respectively
|
13,286
|
|
|
11,375
|
|
||
In-process research & development
|
23,000
|
|
|
23,000
|
|
||
Goodwill
|
197,797
|
|
|
197,797
|
|
||
Other non-current assets
|
11,265
|
|
|
6,683
|
|
||
Total Assets
|
$
|
764,702
|
|
|
$
|
789,951
|
|
Liabilities and Stockholders’ Equity
|
|
|
|
||||
Current liabilities:
|
|
|
|
||||
Accounts payable, accrued expenses, and other current liabilities
|
$
|
60,596
|
|
|
$
|
80,625
|
|
Deferred reimbursements
|
2,750
|
|
|
5,500
|
|
||
Operating and finance lease liabilities
|
2,523
|
|
|
—
|
|
||
Total current liabilities
|
65,869
|
|
|
86,125
|
|
||
Deferred reimbursements
|
11,406
|
|
|
10,156
|
|
||
Convertible notes
|
22,052
|
|
|
175,006
|
|
||
Senior secured term loan
|
146,766
|
|
|
146,734
|
|
||
Contingent consideration payable
|
20,767
|
|
|
19,700
|
|
||
Deferred income taxes
|
6,465
|
|
|
6,465
|
|
||
Operating and finance lease liabilities
|
36,100
|
|
|
—
|
|
||
Other non-current liabilities
|
3,609
|
|
|
2,853
|
|
||
Total liabilities
|
313,034
|
|
|
447,039
|
|
||
Commitments and contingencies
|
|
|
|
|
|
||
Stockholders’ equity:
|
|
|
|
||||
Common stock, $0.01 par value, 500,000,000 shares authorized, 230,180,714 and 189,383,924 shares issued and outstanding at March 31, 2019 and December 31, 2018, respectively
|
2,347
|
|
|
1,942
|
|
||
Additional paid-in capital
|
1,970,607
|
|
|
1,740,061
|
|
||
Accumulated other comprehensive loss:
|
|
|
|
||||
Foreign currency translation adjustment
|
(1,309
|
)
|
|
495
|
|
||
Unrealized gain (loss) on available-for-sale securities
|
157
|
|
|
(427
|
)
|
||
Warrants
|
12,387
|
|
|
13,063
|
|
||
Accumulated deficit
|
(1,532,521
|
)
|
|
(1,412,222
|
)
|
||
Total stockholders’ equity
|
451,668
|
|
|
342,912
|
|
||
Total Liabilities and Stockholders’ Equity
|
$
|
764,702
|
|
|
$
|
789,951
|
|
|
Three Months Ended March 31,
|
||||||
|
2019
|
|
2018
|
||||
Revenue:
|
|
|
|
||||
Net product sales
|
$
|
34,046
|
|
|
$
|
16,696
|
|
Cost of goods sold
|
4,055
|
|
|
2,615
|
|
||
Gross profit
|
29,991
|
|
|
14,081
|
|
||
Operating expenses:
|
|
|
|
||||
Research and development
|
64,593
|
|
|
40,798
|
|
||
Selling, general and administrative
|
44,303
|
|
|
27,396
|
|
||
Changes in fair value of contingent consideration payable
|
1,383
|
|
|
1,100
|
|
||
Depreciation and amortization
|
991
|
|
|
969
|
|
||
Total operating expenses
|
111,270
|
|
|
70,263
|
|
||
Loss from operations
|
(81,279
|
)
|
|
(56,182
|
)
|
||
Other income (expense):
|
|
|
|
||||
Interest income
|
2,639
|
|
|
1,737
|
|
||
Interest expense
|
(6,454
|
)
|
|
(4,488
|
)
|
||
Loss on exchange of convertible notes
|
(36,123
|
)
|
|
—
|
|
||
Change in fair value of derivatives
|
—
|
|
|
4,861
|
|
||
Other income
|
1,086
|
|
|
2,764
|
|
||
Loss before income tax
|
(120,131
|
)
|
|
(51,308
|
)
|
||
Income tax (expense) benefit
|
(168
|
)
|
|
1,392
|
|
||
Net loss attributable to common stockholders
|
$
|
(120,299
|
)
|
|
$
|
(49,916
|
)
|
Net loss attributable to common stockholders per common share — basic and diluted
|
$
|
(0.56
|
)
|
|
$
|
(0.28
|
)
|
Weighted-average common shares outstanding — basic and diluted
|
213,519,287
|
|
|
175,977,700
|
|
Three Months Ended March 31,
|
||||||
|
2019
|
|
2018
|
||||
Net loss
|
$
|
(120,299
|
)
|
|
$
|
(49,916
|
)
|
Other comprehensive (loss) gain:
|
|
|
|
||||
Foreign currency translation adjustment gain (loss), net of tax impact of $0 and $0, respectively
|
(1,804
|
)
|
|
(1,805
|
)
|
||
Unrealized gain (loss) on available-for-sale securities
|
584
|
|
|
(441
|
)
|
||
Other comprehensive income (loss)
|
$
|
(1,220
|
)
|
|
$
|
(2,246
|
)
|
Comprehensive loss
|
$
|
(121,519
|
)
|
|
$
|
(52,162
|
)
|
|
Common Stock
|
|
Additional
Paid-In Capital |
|
Warrants
|
|
Other
Comprehensive Gain (Loss) |
|
Accumulated
Deficit |
|
Total
Stockholders' Equity |
|||||||||||||||
|
Shares
|
|
Amount
|
|||||||||||||||||||||||
Balance at December 31, 2018
|
189,383,924
|
|
|
$
|
1,942
|
|
|
$
|
1,740,061
|
|
|
$
|
13,063
|
|
|
$
|
68
|
|
|
$
|
(1,412,222
|
)
|
|
$
|
342,912
|
|
Stock issued from exercise of stock options, net
|
578,451
|
|
|
6
|
|
|
3,947
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
3,953
|
|
||||||
Restricted stock tax vesting
|
301,058
|
|
|
—
|
|
|
(1,940
|
)
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(1,940
|
)
|
||||||
Stock issued for contingent consideration
|
771,804
|
|
|
8
|
|
|
9,308
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
9,316
|
|
||||||
Stock-based compensation
|
—
|
|
|
—
|
|
|
12,744
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
12,744
|
|
||||||
Warrants exercised
|
101,787
|
|
|
1
|
|
|
1,487
|
|
|
(676
|
)
|
|
—
|
|
|
—
|
|
|
812
|
|
||||||
Equity component of the convertible notes
|
39,043,690
|
|
|
390
|
|
|
190,368
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
190,758
|
|
||||||
Termination of capped call confirmations
|
—
|
|
|
—
|
|
|
14,632
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
14,632
|
|
||||||
Unrealized holding gain on available-for-sale securities
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
584
|
|
|
—
|
|
|
584
|
|
||||||
Foreign currency translation adjustment
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(1,804
|
)
|
|
—
|
|
|
(1,804
|
)
|
||||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(120,299
|
)
|
|
(120,299
|
)
|
||||||
Balance at March 31, 2019
|
230,180,714
|
|
|
$
|
2,347
|
|
|
$
|
1,970,607
|
|
|
$
|
12,387
|
|
|
$
|
(1,152
|
)
|
|
$
|
(1,532,521
|
)
|
|
$
|
451,668
|
|
|
Common Stock
|
|
Additional
Paid-In Capital |
|
Warrants
|
|
Other
Comprehensive Gain (Loss) |
|
Accumulated
Deficit |
|
Total
Stockholders' Equity |
|||||||||||||||
|
Shares
|
|
Amount
|
|||||||||||||||||||||||
Balance at December 31, 2017
|
166,989,790
|
|
|
$
|
1,721
|
|
|
$
|
1,400,758
|
|
|
$
|
16,076
|
|
|
$
|
(2,095
|
)
|
|
$
|
(1,063,610
|
)
|
|
$
|
352,850
|
|
Stock issued from exercise of options, net
|
560,721
|
|
|
6
|
|
|
3,972
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
3,978
|
|
||||||
Stock issued from equity financing
|
20,239,839
|
|
|
202
|
|
|
294,382
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
294,584
|
|
||||||
Restricted stock tax vesting
|
181,868
|
|
|
—
|
|
|
(1,912
|
)
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(1,912
|
)
|
||||||
Stock issued for contingent consideration
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
||||||
Stock-based compensation
|
—
|
|
|
—
|
|
|
7,478
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
7,478
|
|
||||||
Reclassification upon ASU 2018-02 adoption
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(383
|
)
|
|
383
|
|
|
—
|
|
||||||
Change in fair value of derivatives
|
—
|
|
|
—
|
|
|
(83,199
|
)
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(83,199
|
)
|
||||||
Unrealized holding gain (loss) on available-for-sale securities
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(441
|
)
|
|
—
|
|
|
(441
|
)
|
||||||
Foreign currency translation adjustment
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(1,805
|
)
|
|
—
|
|
|
(1,805
|
)
|
||||||
Change in fair value of derivatives
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
||||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(49,916
|
)
|
|
(49,916
|
)
|
||||||
Balance at March 31, 2018
|
187,972,218
|
|
|
$
|
1,929
|
|
|
$
|
1,621,479
|
|
|
$
|
16,076
|
|
|
$
|
(4,724
|
)
|
|
$
|
(1,113,143
|
)
|
|
$
|
521,617
|
|
|
Three Months Ended March 31,
|
||||||
|
2019
|
|
2018
|
||||
Operating activities
|
|
|
|
||||
Net loss
|
$
|
(120,299
|
)
|
|
$
|
(49,916
|
)
|
Adjustments to reconcile net loss to net cash used in operating activities:
|
|
|
|
||||
Amortization of debt discount and deferred financing
|
1,649
|
|
|
2,601
|
|
||
Depreciation and amortization
|
991
|
|
|
969
|
|
||
Stock-based compensation
|
12,744
|
|
|
7,478
|
|
||
Loss on exchange of convertible debt
|
36,123
|
|
|
—
|
|
||
Change in fair value of derivatives
|
—
|
|
|
(4,861
|
)
|
||
Non-cash changes in the fair value of contingent consideration payable
|
1,383
|
|
|
1,100
|
|
||
Foreign currency remeasurement (gain) loss
|
325
|
|
|
(2,957
|
)
|
||
Changes in operating assets and liabilities:
|
|
|
|
||||
Accounts receivable
|
(1,001
|
)
|
|
(1,047
|
)
|
||
Inventories
|
345
|
|
|
(3,253
|
)
|
||
Prepaid expenses and other current assets
|
3,059
|
|
|
10,875
|
|
||
Accounts payable and accrued expenses
|
(8,858
|
)
|
|
(10,347
|
)
|
||
Other non-current assets and liabilities
|
(3,507
|
)
|
|
(163
|
)
|
||
Deferred reimbursements
|
(1,500
|
)
|
|
—
|
|
||
Net cash used in operating activities
|
$
|
(78,546
|
)
|
|
$
|
(49,521
|
)
|
Investing activities
|
|
|
|
||||
Sale and redemption of marketable securities
|
135,187
|
|
|
121,226
|
|
||
Purchases of marketable securities
|
(52,178
|
)
|
|
(303,010
|
)
|
||
Capital expenditures
|
(2,944
|
)
|
|
(819
|
)
|
||
Net cash used in investing activities
|
$
|
80,065
|
|
|
$
|
(182,603
|
)
|
Financing activities
|
|
|
|
||||
Proceeds from issuance of common stock, net of issuance costs
|
—
|
|
|
294,584
|
|
||
Payment of finance leases
|
(75
|
)
|
|
(71
|
)
|
||
Purchase of vested restricted stock units
|
(1,938
|
)
|
|
(1,912
|
)
|
||
Proceeds from termination of capped call confirmations
|
14,632
|
|
|
—
|
|
||
Proceeds from exercise of stock options
|
3,953
|
|
|
3,978
|
|
||
Proceeds of exercise of warrants
|
812
|
|
|
—
|
|
||
Net cash provided by financing activities
|
$
|
17,384
|
|
|
$
|
296,579
|
|
Effect of exchange rate changes on cash, cash equivalents and restricted cash
|
$
|
(2,350
|
)
|
|
$
|
860
|
|
Net decrease in cash, cash equivalents and restricted cash
|
16,553
|
|
|
65,315
|
|
||
Cash, cash equivalents and restricted cash at beginning of period
|
$
|
82,375
|
|
|
$
|
51,237
|
|
Cash, cash equivalents and restricted cash at end of period
|
$
|
98,928
|
|
|
$
|
116,552
|
|
Supplemental disclosures of cash flow information
|
|
|
|
||||
Cash paid during the period for interest
|
$
|
4,846
|
|
|
$
|
12
|
|
Capital expenditures, unpaid
|
$
|
—
|
|
|
$
|
142
|
|
Payment of contingent consideration in shares
|
$
|
9,316
|
|
|
$
|
—
|
|
|
Three Months Ended March 31,
|
||||||
(in thousands)
|
2019
|
|
2018
|
||||
U.S.
|
$
|
9,068
|
|
|
$
|
—
|
|
Ex-U.S.
|
24,978
|
|
|
16,696
|
|
||
Total net product sales
|
$
|
34,046
|
|
|
$
|
16,696
|
|
|
As of March 31, 2019
|
||||||||||||||
(in thousands)
|
Cost
|
|
Gross
unrealized
Gain
|
|
Gross
unrealized
Loss
|
|
Fair
Value
|
||||||||
Cash and cash equivalents
|
$
|
96,349
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
96,349
|
|
Corporate debt securities, current portion
|
138,194
|
|
|
96
|
|
|
(6
|
)
|
|
138,284
|
|
||||
Commercial paper
|
134,948
|
|
|
52
|
|
|
(7
|
)
|
|
134,993
|
|
||||
Asset-backed securities
|
68,278
|
|
|
30
|
|
|
(8
|
)
|
|
68,300
|
|
||||
Money market
|
350
|
|
|
—
|
|
|
—
|
|
|
350
|
|
||||
Certificates of deposit
|
51
|
|
|
—
|
|
|
—
|
|
|
51
|
|
||||
|
$
|
438,170
|
|
|
$
|
178
|
|
|
$
|
(21
|
)
|
|
$
|
438,327
|
|
Included in cash and cash equivalents
|
$
|
96,349
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
96,349
|
|
Included in marketable securities, current and non-current
|
341,821
|
|
|
178
|
|
|
(21
|
)
|
|
341,978
|
|
||||
Total cash, cash equivalents and marketable securities
|
$
|
438,170
|
|
|
$
|
178
|
|
|
$
|
(21
|
)
|
|
$
|
438,327
|
|
|
As of December 31, 2018
|
||||||||||||||
(in thousands)
|
Cost
|
|
Gross
unrealized
Gain
|
|
Gross
unrealized
Loss
|
|
Fair
Value
|
||||||||
Cash and cash equivalents
|
$
|
79,749
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
79,749
|
|
Corporate debt securities, current portion
|
240,969
|
|
|
7
|
|
|
(250
|
)
|
|
240,726
|
|
||||
Commercial paper
|
115,245
|
|
|
—
|
|
|
(104
|
)
|
|
115,141
|
|
||||
Asset-backed securities
|
68,215
|
|
|
4
|
|
|
(84
|
)
|
|
68,135
|
|
||||
Money market
|
350
|
|
|
—
|
|
|
—
|
|
|
350
|
|
||||
Certificates of deposit
|
51
|
|
|
—
|
|
|
—
|
|
|
51
|
|
||||
|
$
|
504,579
|
|
|
$
|
11
|
|
|
$
|
(438
|
)
|
|
$
|
504,152
|
|
Included in cash and cash equivalents
|
$
|
79,749
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
79,749
|
|
Included in marketable securities
|
424,830
|
|
|
11
|
|
|
(438
|
)
|
|
424,403
|
|
||||
Total cash, cash equivalents and marketable securities
|
$
|
504,579
|
|
|
$
|
11
|
|
|
$
|
(438
|
)
|
|
$
|
504,152
|
|
(in thousands)
|
March 31, 2019
|
|
December 31, 2018
|
|
March 31, 2018
|
|
December 31, 2017
|
||||||||
Cash and cash equivalents
|
$
|
96,349
|
|
|
$
|
79,749
|
|
|
$
|
114,322
|
|
|
$
|
49,060
|
|
Restricted cash
|
2,579
|
|
|
2,626
|
|
|
2,230
|
|
|
2,177
|
|
||||
Cash and cash equivalents and restricted cash shown in the statement of cash flows
|
$
|
98,928
|
|
|
$
|
82,375
|
|
|
$
|
116,552
|
|
|
$
|
51,237
|
|
(in thousands)
|
|
March 31, 2019
|
|
December 31, 2018
|
||||
Raw materials
|
|
$
|
1,321
|
|
|
$
|
1,291
|
|
Work-in-process
|
|
$
|
3,041
|
|
|
3,485
|
|
|
Finished goods
|
|
3,805
|
|
|
3,614
|
|
||
Total inventories
|
|
$
|
8,167
|
|
|
$
|
8,390
|
|
Liability component (in thousands)
|
|
March 31, 2019
|
|
December 31, 2018
|
||||
Principal
|
|
180,702
|
|
|
$
|
400,000
|
|
|
Less: debt discount (1)
|
|
(11,076
|
)
|
|
(74,145
|
)
|
||
Less: deferred financing (1)
|
|
(808
|
)
|
|
(4,115
|
)
|
||
Net carrying value of the debt
|
|
$
|
168,818
|
|
|
$
|
321,740
|
|
|
|
Three Months Ended March 31,
|
||||||
Interest component (in thousands)
|
|
2019
|
|
2018
|
||||
Contractual interest expense
|
|
4,813
|
|
|
$
|
1,887
|
|
|
Amortization of debt discount
|
|
1,559
|
|
|
2,472
|
|
||
Amortization of deferred financing
|
|
83
|
|
|
129
|
|
||
Total
|
|
$
|
6,455
|
|
|
$
|
4,488
|
|
|
Three Months Ended March 31,
|
||||||
|
2019
|
|
2018
|
||||
Expected stock price volatility
|
74.2
|
%
|
|
81.2
|
%
|
||
Risk free interest rate
|
2.5
|
%
|
|
2.3
|
%
|
||
Expected life of options (years)
|
5.68
|
|
|
5.67
|
|
||
Expected annual dividend per share
|
$
|
—
|
|
|
$
|
—
|
|
|
Number of
Shares
|
|
Weighted
Average
Exercise Price
|
|
Weighted
Average
Remaining
Contractual Life
|
|
Aggregate
Intrinsic
Value
|
|||||
|
(in thousands)
|
|
|
|
|
|
(in millions)
|
|||||
Options outstanding, December 31, 2018
|
15,810
|
|
|
$
|
8.63
|
|
|
|
|
|
|
|
Granted
|
3,315
|
|
|
$
|
10.15
|
|
|
|
|
|
|
|
Exercised
|
(578
|
)
|
|
$
|
6.83
|
|
|
|
|
|
|
|
Forfeited
|
(195
|
)
|
|
$
|
10.33
|
|
|
|
|
|
|
|
Expired
|
(21
|
)
|
|
$
|
10.77
|
|
|
|
|
|
||
Options outstanding, March 31, 2019
|
18,331
|
|
|
$
|
8.94
|
|
|
6.9 years
|
|
$
|
90.2
|
|
Vested and unvested expected to vest, March 31, 2019
|
17,325
|
|
|
$
|
8.84
|
|
|
6.8 years
|
|
$
|
87.1
|
|
Exercisable at March 31, 2019
|
10,538
|
|
|
$
|
7.84
|
|
|
5.5 years
|
|
$
|
62.8
|
|
|
Number of Shares
|
|
Weighted
Average Grant Date
Fair Value
|
|
Weighted Average
Remaining Years
|
|
Aggregate Intrinsic
Value
|
|||||
|
(in thousands)
|
|
|
|
|
|
(in millions)
|
|||||
Non-vested units as of December 31, 2018
|
3,712
|
|
|
$
|
10.59
|
|
|
|
|
|
|
|
Granted
|
3,031
|
|
|
$
|
10.91
|
|
|
|
|
|
|
|
Vested
|
(600
|
)
|
|
$
|
9.07
|
|
|
|
|
|
|
|
Forfeited
|
(133
|
)
|
|
$
|
12.73
|
|
|
|
|
|
|
|
Non-vested units as of March 31, 2019
|
6,010
|
|
|
$
|
10.86
|
|
|
3.0 years
|
|
$
|
81.7
|
|
|
|
Three Months Ended March 31,
|
||||||
(in thousands)
|
|
2019
|
|
2018
|
||||
Equity compensation expense recognized in:
|
|
|
|
|
|
|
||
Research and development expense
|
|
$
|
5,032
|
|
|
$
|
3,057
|
|
Selling, general and administrative expense
|
|
7,712
|
|
|
4,421
|
|
||
Total equity compensation expense
|
|
$
|
12,744
|
|
|
$
|
7,478
|
|
(in thousands)
|
|
Level 2
|
|
Total
|
||||
Assets:
|
|
|
|
|
|
|
||
Commercial paper
|
|
$
|
134,993
|
|
|
$
|
134,993
|
|
Asset-backed securities
|
|
68,300
|
|
|
68,300
|
|
||
Corporate debt securities
|
|
138,284
|
|
|
138,284
|
|
||
Money market funds
|
|
3,963
|
|
|
3,963
|
|
||
|
|
$
|
345,540
|
|
|
$
|
345,540
|
|
|
|
Level 2
|
|
Level 3
|
|
Total
|
||||||
Liabilities:
|
|
|
|
|
|
|
|
|
|
|||
Contingent consideration payable
|
|
$
|
—
|
|
|
$
|
20,767
|
|
|
$
|
20,767
|
|
Deferred compensation plan liability
|
|
3,613
|
|
|
—
|
|
|
3,613
|
|
|||
|
|
$
|
3,613
|
|
|
$
|
20,767
|
|
|
$
|
24,380
|
|
(in thousands)
|
Level 2
|
|
Total
|
||||
Assets:
|
|
|
|
||||
Commercial paper
|
$
|
115,141
|
|
|
$
|
115,141
|
|
Asset-backed securities
|
68,135
|
|
|
68,135
|
|
||
Corporate debt securities
|
240,726
|
|
|
240,726
|
|
||
Money market funds
|
3,082
|
|
|
3,082
|
|
||
|
$
|
427,084
|
|
|
$
|
427,084
|
|
|
Level 2
|
|
Level 3
|
|
Total
|
||||||
Liabilities:
|
|
|
|
|
|
|
|
|
|||
Contingent consideration payable
|
$
|
—
|
|
|
$
|
19,700
|
|
|
$
|
19,700
|
|
Deferred compensation plan liability
|
2,732
|
|
|
—
|
|
|
2,732
|
|
|||
|
$
|
2,732
|
|
|
$
|
19,700
|
|
|
$
|
22,432
|
|
Contingent Consideration
Liability
|
|
Fair Value as of
March 31, 2019 |
|
Valuation Technique
|
|
Unobservable Input
|
|
Range
|
||
|
|
(in thousands)
|
|
|
|
|
|
|
||
|
|
|
|
|
|
|
|
|
||
|
|
|
|
|
|
Discount rate
|
|
10.3%
|
||
|
|
|
|
|
|
|
|
|
|
|
Clinical and regulatory milestones
|
|
$
|
20,538
|
|
|
Probability weighted discounted cash flow
|
|
Probability of achievement of milestones
|
|
75%-78%
|
|
|
|
|
|
|
|
|
|
||
|
|
|
|
|
|
Projected year of payments
|
|
2021-2022
|
|
|
Three Months Ended March 31,
|
||||||
(in thousands)
|
|
2019
|
|
2018
|
||||
Balance, beginning of the period
|
|
$
|
28,700
|
|
|
$
|
25,400
|
|
Payment of contingent consideration in stock
|
|
(9,316
|
)
|
|
—
|
|
||
Changes in fair value during the period, included in Statement of Operations
|
|
1,383
|
|
|
1,100
|
|
||
Balance, end of the period
|
|
$
|
20,767
|
|
|
$
|
26,500
|
|
(in thousands, except year and discount rate amounts)
|
|
March 31, 2019
|
||
Operating lease ROU asset
|
|
$
|
36,208
|
|
|
|
|
||
Current portion of the operating lease liabilities
|
|
$
|
2,421
|
|
Non-current portion of the operating lease liabilities
|
|
35,991
|
|
|
Total operating lease liability
|
|
$
|
38,412
|
|
|
|
|
||
Weighted-average remaining lease terms (years)
|
|
14.6
|
|
|
Weighted-average discount rate
|
|
12.8
|
%
|
( in thousands)
|
|
Operating Leases
|
||
2019 (excludes the three months ended March 31, 2019)
|
|
$
|
4,494
|
|
2020
|
|
9,776
|
|
|
2021
|
|
10,516
|
|
|
2022
|
|
10,418
|
|
|
2023
|
|
10,830
|
|
|
Thereafter
|
|
174,006
|
|
|
Total lease payments
|
|
$
|
220,040
|
|
Less lease incentives
|
|
(28,939
|
)
|
|
Less imputed interest
|
|
(152,689
|
)
|
|
Total operating lease liability
|
|
$
|
38,412
|
|
( in thousands)
|
|
Operating Leases
|
||
2019
|
|
$
|
6,244
|
|
2020
|
|
4,063
|
|
|
2021
|
|
3,560
|
|
|
2022
|
|
3,371
|
|
|
2023
|
|
3,611
|
|
|
Thereafter
|
|
10,038
|
|
|
Total lease payments
|
|
$
|
30,887
|
|
|
|
Three Months Ended March 31,
|
||||||
(in thousands, except per share amounts)
|
|
2019
|
|
2018
|
||||
Numerator:
|
|
|
|
|
|
|
||
Net loss attributable to common stockholders
|
|
$
|
(120,299
|
)
|
|
$
|
(49,916
|
)
|
Denominator:
|
|
|
|
|
||||
Weighted average common shares outstanding — basic and diluted
|
|
213,519,287
|
|
|
175,977,700
|
|
|
|
As of March 31,
|
||||
(in thousands)
|
|
2019
|
|
2018
|
||
Options to purchase common stock
|
|
18,331
|
|
|
16,176
|
|
Convertible notes
|
|
5,017
|
|
|
40,850
|
|
Outstanding warrants, convertible to common stock
|
|
2,554
|
|
|
3,110
|
|
Unvested restricted stock units
|
|
6,010
|
|
|
3,541
|
|
Vested restricted stock units, unissued
|
|
189
|
|
|
90
|
|
Total number of potentially issuable shares
|
|
32,101
|
|
|
63,767
|
|
•
|
Commercial and regulatory success in Fabry disease
. During the three month ended March 31, 2019, Galafold
®
revenue totaled approximately
$34.0 million
, an increase of $17.4 million compared to the same period in the prior year. We continue to see strong momentum in new markets including in the U.S. and Japan, as well as in our more mature markets. In the countries we have been operating the longest, such as Germany and the United Kingdom, we see an increasing proportion of previously untreated patients come onto Galafold
®
. In the U.S., we continue to see a significant increase in patients from a growing and very wide prescriber base. Across all markets, we see a high rate of compliance and adherence to this oral treatment option.
|
•
|
Pompe clinical program milestones
. We reported positive data from a Phase 1/2 clinical study to evaluate Pompe disease patients treated with our novel treatment paradigm AT-GAA for up to 24 months. The U.S. FDA also granted breakthrough therapy designation ("BTD") for AT-GAA for the treatment of late-onset Pompe disease. We are currently enrolling a global pivotal study of AT-GAA (ATB200-03, also known as PROPEL) which is on track to enroll approximately 100 participants with late-onset Pompe disease at up to 90 global sites.
|
•
|
Pipeline Growth:
With 14 new gene therapy programs, we have established a leading portfolio of medicines for people living with rare metabolic disorders. Through our license with NCH, we acquired worldwide development and commercial rights for ten gene therapy programs in rare, neurologic LSDs with lead programs in CLN6, CLN3, and CLN8 Batten disease. An additional four programs were added to the pipeline through the collaboration
with
|
•
|
Manufacturing
. We successfully scaled up manufacturing of our Pompe biologic to commercial scale (1,000L) for our pivotal PROPEL study and commercial supply. Our supply agreement with WuXi Biologics and current capacity are expected to produce sufficient quantities to serve the entire Pompe population as quickly as possible after receipt of applicable regulatory approvals. Through our collaborations with NCH and Penn, we also gain access to their preclinical manufacturing capabilities, clinical supply and CMO relationships for those gene therapy programs.
|
•
|
Financial strength.
Total cash, cash equivalents and marketable securities of $
438.3
million at March 31, 2019 compared to $504.2 million at December 31, 2018. The current cash position, including expected Galafold
®
revenues, is sufficient to fund ongoing Fabry, Pompe and gene therapy program operations into at least mid-2021. Potential future business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact our future capital requirements.
|
|
|
Three Months Ended March 31,
|
||||||||||
(in thousands)
|
|
2019
|
|
2018
|
|
Change
|
||||||
Net product sales
|
|
$
|
34,046
|
|
|
$
|
16,696
|
|
|
$
|
17,350
|
|
Cost of goods sold
|
|
4,055
|
|
|
2,615
|
|
|
1,440
|
|
|||
Cost of goods sold as a percentage of net product sales
|
|
11.9
|
%
|
|
15.7
|
%
|
|
(3.8
|
)%
|
|||
Operating expenses:
|
|
|
|
|
|
|
||||||
Research and development
|
|
64,593
|
|
|
40,798
|
|
|
23,795
|
|
|||
Selling, general and administrative
|
|
44,303
|
|
|
27,396
|
|
|
16,907
|
|
|||
Changes in fair value of contingent consideration payable
|
|
1,383
|
|
|
1,100
|
|
|
283
|
|
|||
Depreciation and amortization
|
|
991
|
|
|
969
|
|
|
22
|
|
|||
Other income (expense):
|
|
|
|
|
|
|
||||||
Interest income
|
|
2,639
|
|
|
1,737
|
|
|
902
|
|
|||
Interest expense
|
|
(6,454
|
)
|
|
(4,488
|
)
|
|
(1,966
|
)
|
|||
Change in fair value of derivatives
|
|
—
|
|
|
4,861
|
|
|
(4,861
|
)
|
|||
Loss on exchange of convertible notes
|
|
(36,123
|
)
|
|
—
|
|
|
(36,123
|
)
|
|||
Other income
|
|
1,086
|
|
|
2,764
|
|
|
(1,678
|
)
|
|||
Income tax (expense) benefit
|
|
(168
|
)
|
|
1,392
|
|
|
(1,560
|
)
|
|||
Net loss attributable to common stockholders
|
|
$
|
(120,299
|
)
|
|
$
|
(49,916
|
)
|
|
$
|
(70,383
|
)
|
(in thousands)
|
|
Three Months Ended March 31,
|
||||||
Projects
|
|
2019
|
|
2018
|
||||
Third party direct project expenses
|
|
|
|
|
|
|
||
Migalastat (Fabry Disease)
|
|
$
|
4,356
|
|
|
$
|
3,708
|
|
AT-GAA (Pompe Disease)
|
|
28,286
|
|
|
15,095
|
|
||
Gene therapy programs
|
|
2,192
|
|
|
—
|
|
||
Pre-clinical and other programs
|
|
460
|
|
|
658
|
|
||
Total third party direct project expenses
|
|
35,294
|
|
|
19,461
|
|
||
Other project costs
|
|
|
|
|
|
|
||
Personnel costs
|
|
19,634
|
|
|
14,949
|
|
||
Other costs
|
|
9,665
|
|
|
6,388
|
|
||
Total other project costs
|
|
29,299
|
|
|
21,337
|
|
||
Total research and development costs
|
|
$
|
64,593
|
|
|
$
|
40,798
|
|
•
|
the progress and results of our preclinical and clinical trials of our drug candidates and gene therapy candidates;
|
•
|
the cost of manufacturing drug and gene therapy supply for our clinical and preclinical studies, including the significant cost of manufacturing Pompe ERT and gene therapies;
|
•
|
the scope, progress, results and costs of preclinical development, laboratory testing and clinical trials for our product candidates including those testing the use of pharmacological chaperones co-formulated and co-administered with ERT and for the treatment of LSDs and gene therapies for the treatment of rare genetic metabolic diseases;
|
•
|
the future results of on-going preclinical research and subsequent clinical trials for CDD, including our ability to obtain regulatory approvals and commercialize CDKL5 therapies and obtain market acceptance for such therapies;
|
•
|
the costs, timing and outcome of regulatory review of our product candidates;
|
•
|
the number and development requirements of other product candidates that we pursue;
|
•
|
the costs of commercialization activities, including product marketing, sales and distribution;
|
•
|
the emergence of competing technologies and other adverse market developments;
|
•
|
our ability to successfully commercialize Galafold
®
("migalastat HCl");
|
•
|
our ability to manufacture or supply sufficient clinical or commercial products;
|
•
|
our ability to obtain reimbursement for Galafold
®
;
|
•
|
our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold
®
;
|
•
|
our ability to obtain market acceptance of Galafold
®
;
|
•
|
the costs of preparing, filing and prosecuting patent applications and maintaining, enforcing and defending intellectual property-related claims;
|
•
|
the extent to which we acquire or invest in businesses, products and technologies;
|
•
|
our ability to successfully integrate our acquired products and technologies into our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected;
|
•
|
our ability to establish collaborations and obtain milestone, royalty or other payments from any such collaborators;
|
•
|
our ability to adjust to changes in European and United Kingdom markets as the United Kingdom leaves the European Union; and
|
•
|
fluctuations in foreign currency exchange rates; and changes in accounting standards.
|
Exhibit
Number
|
|
Description
|
|
|
|
10.1
|
|
|
|
|
|
|
|
|
31.1
|
|
|
|
|
|
31.2
|
|
|
|
|
|
32.1
|
|
|
|
|
|
101
|
|
The following financial information from this Quarterly Report on Form 10-Q for the three months ended March 31, 2019, formatted in XBRL (Extensible Business Reporting Language) and filed electronically herewith: (i) the Consolidated Balance Sheets; (ii) the Consolidated Statements of Operations; (iii) the Consolidated Statements of Comprehensive Loss; (iv) the Consolidated Statements of Cash Flows; (v) and the Notes to the Consolidated Financial Statements
|
|
AMICUS THERAPEUTICS, INC.
|
|
|
|
|
Date: May 9, 2019
|
By:
|
/s/ John F. Crowley
|
|
|
John F. Crowley
|
|
|
Chairman and Chief Executive Officer
|
|
|
(Principal Executive Officer)
|
|
|
|
Date: May 9, 2019
|
By:
|
/s/ Daphne Quimi
|
|
|
Daphne Quimi
|
|
|
Chief Financial Officer
|
|
|
(Principal Financial Officer)
|
(a)
|
Counterparty:
|
(b)
|
Dealer:
|
To:
|
[●]
|
To:
|
[●]
|
To:
|
[●]
|
15.
|
Agreements and Acknowledgements Regarding Hedging.
Counterparty acknowledges and agrees that:
|
VWAPPrice
|
Cash Settlement Amount
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
$[●]
|
1.
|
I have reviewed this quarterly report on Form 10-Q of Amicus Therapeutics, Inc.;
|
2.
|
Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
|
3.
|
Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
|
4.
|
The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
|
5.
|
The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
|
Date: May 9, 2019
|
/s/ John F. Crowley
|
|
John F. Crowley
|
|
Chairman and Chief Executive Officer
|
1.
|
I have reviewed this quarterly report on Form 10-Q of Amicus Therapeutics, Inc.;
|
2.
|
Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
|
3.
|
Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
|
4.
|
The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
|
5.
|
The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions):
|
Date: May 9, 2019
|
/s/ Daphne Quimi
|
|
Daphne Quimi
|
|
Chief Financial Officer
|
Date: May 9, 2019
|
By:
|
/s/ John F. Crowley
|
|
|
John F. Crowley
|
|
|
Chairman and Chief Executive Officer
|
|
|
|
Date: May 9, 2019
|
By:
|
/s/ Daphne Quimi
|
|
|
Daphne Quimi
|
|
|
Chief Financial Officer
|