|
☒
|
QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
|
☐
|
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
|
Delaware
|
20-0216859
|
(State or Other Jurisdiction of Incorporation or Organization)
|
(I.R.S. Employer Identification No.)
|
Title of each class
|
Trading Symbol(s)
|
Name of each exchange on which registered
|
Common Stock
|
PTLA
|
The Nasdaq Global Select Market
|
Large accelerated filer
|
☒
|
|
Accelerated filer
|
☐
|
Non-accelerated filer
|
☐
|
|
Smaller reporting company
|
☐
|
Emerging growth company
|
☐
|
|
|
|
|
|
|
|
Page
|
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
|
|
||
ITEM 1.
|
FINANCIAL STATEMENTS
|
|
September 30, 2019
|
|
December 31, 2018
|
||||
Assets
|
|
|
|
||||
Current assets:
|
|
|
|
||||
Cash and cash equivalents
|
$
|
304,265
|
|
|
$
|
138,951
|
|
Short-term investments
|
172,542
|
|
|
178,013
|
|
||
Restricted cash
|
3,201
|
|
|
1,062
|
|
||
Trade and other receivables, net
|
18,236
|
|
|
5,849
|
|
||
Unbilled - collaboration and license revenue
|
3,788
|
|
|
9,880
|
|
||
Inventories
|
2,357
|
|
|
7,873
|
|
||
Prepaid and other current assets
|
10,106
|
|
|
11,699
|
|
||
Total current assets
|
514,495
|
|
|
353,327
|
|
||
Property and equipment, net
|
4,561
|
|
|
5,236
|
|
||
Intangible assets
|
3,699
|
|
|
7,279
|
|
||
Operating lease right-of-use asset
|
11,836
|
|
|
—
|
|
||
Inventories, noncurrent portion
|
33,296
|
|
|
9,645
|
|
||
Prepaid and other long-term assets
|
9,298
|
|
|
10,932
|
|
||
Total assets
|
$
|
577,185
|
|
|
$
|
386,419
|
|
Liabilities and stockholders’ equity
|
|
|
|
||||
Current liabilities:
|
|
|
|
||||
Accounts payable
|
$
|
13,760
|
|
|
$
|
13,215
|
|
Accrued research and development
|
15,629
|
|
|
19,831
|
|
||
Accrued and other liabilities
|
32,852
|
|
|
22,310
|
|
||
Deferred revenue, current portion
|
1,061
|
|
|
1,847
|
|
||
Current portion of notes payable and long-term royalty-based debt
|
24,189
|
|
|
11,802
|
|
||
Total current liabilities
|
87,491
|
|
|
69,005
|
|
||
Notes payable, less current portion
|
41,928
|
|
|
48,298
|
|
||
Long term royalty-based debt, less current portion
|
157,182
|
|
|
155,256
|
|
||
Long term debt
|
57,269
|
|
|
—
|
|
||
Long term obligation to collaborator, less current portion
|
5,124
|
|
|
6,881
|
|
||
Deferred revenue, long-term
|
4,399
|
|
|
4,488
|
|
||
Long-term portion of lease liability
|
9,464
|
|
|
—
|
|
||
Other long-term liabilities
|
1,440
|
|
|
11,924
|
|
||
Total liabilities
|
364,297
|
|
|
295,852
|
|
||
Stockholders’ equity:
|
|
|
|
||||
Preferred stock, $0.001 par value, 5,000 shares authorized; no shares issued and outstanding
|
—
|
|
|
—
|
|
||
Common stock, $0.001 par value, 150,000 shares authorized at September 30, 2019 and December 31, 2018; 77,784 shares and 66,618 shares issued and outstanding at September 30, 2019 and December 31, 2018, respectively
|
79
|
|
|
68
|
|
||
Additional paid-in capital
|
1,932,428
|
|
|
1,614,320
|
|
||
Accumulated deficit
|
(1,719,691
|
)
|
|
(1,525,704
|
)
|
||
Accumulated other comprehensive income (loss)
|
72
|
|
|
(283
|
)
|
||
Total Portola stockholders’ equity
|
212,888
|
|
|
88,401
|
|
||
Noncontrolling interest
|
—
|
|
|
2,166
|
|
||
Total stockholders’ equity
|
212,888
|
|
|
90,567
|
|
||
Total liabilities and stockholders’ equity
|
$
|
577,185
|
|
|
$
|
386,419
|
|
|
Three Months Ended September 30,
|
|
Nine Months Ended September 30,
|
||||||||||||
|
2019
|
|
2018
|
|
2019
|
|
2018
|
||||||||
Revenues:
|
|
|
|
|
|
|
|
||||||||
Product revenue, net
|
$
|
35,743
|
|
|
$
|
7,176
|
|
|
$
|
83,269
|
|
|
$
|
10,047
|
|
Collaboration and license revenue
|
1,056
|
|
|
7,001
|
|
|
4,123
|
|
|
14,785
|
|
||||
Total revenues
|
36,799
|
|
|
14,177
|
|
|
87,392
|
|
|
24,832
|
|
||||
Operating expenses:
|
|
|
|
|
|
|
|
||||||||
Cost of sales
|
2,684
|
|
|
4,292
|
|
|
14,825
|
|
|
5,680
|
|
||||
Research and development
|
25,647
|
|
|
40,237
|
|
|
94,769
|
|
|
166,744
|
|
||||
Selling, general and administrative
|
52,050
|
|
|
38,792
|
|
|
158,939
|
|
|
110,547
|
|
||||
Total operating expenses
|
80,381
|
|
|
83,321
|
|
|
268,533
|
|
|
282,971
|
|
||||
Loss from operations
|
(43,582
|
)
|
|
(69,144
|
)
|
|
(181,141
|
)
|
|
(258,139
|
)
|
||||
Interest and other income, net
|
1,953
|
|
|
3,924
|
|
|
7,958
|
|
|
9,123
|
|
||||
Interest expense
|
(7,998
|
)
|
|
(5,957
|
)
|
|
(23,017
|
)
|
|
(12,642
|
)
|
||||
Net loss
|
(49,627
|
)
|
|
(71,177
|
)
|
|
(196,200
|
)
|
|
(261,658
|
)
|
||||
Net (income) loss attributable to noncontrolling interest
|
—
|
|
|
(126
|
)
|
|
2,213
|
|
|
(17
|
)
|
||||
Net loss attributable to Portola
|
$
|
(49,627
|
)
|
|
$
|
(71,303
|
)
|
|
$
|
(193,987
|
)
|
|
$
|
(261,675
|
)
|
Net loss per share attributable to Portola common stockholders:
|
|
|
|
|
|
|
|
||||||||
Basic and diluted
|
$
|
(0.68
|
)
|
|
$
|
(1.08
|
)
|
|
$
|
(2.79
|
)
|
|
$
|
(3.97
|
)
|
Shares used to compute net loss per share attributable to Portola common stockholders:
|
|
|
|
|
|
|
|
||||||||
Basic and diluted
|
73,017,609
|
|
|
66,165,104
|
|
|
69,427,124
|
|
|
65,855,672
|
|
|
Three Months Ended September 30,
|
|
Nine Months Ended September 30,
|
||||||||||||
|
2019
|
|
2018
|
|
2019
|
|
2018
|
||||||||
Net loss
|
$
|
(49,627
|
)
|
|
$
|
(71,177
|
)
|
|
$
|
(196,200
|
)
|
|
$
|
(261,658
|
)
|
Other comprehensive income (loss):
|
|
|
|
|
|
|
|
||||||||
Unrealized gain (loss) on available-for-sale securities, net of tax
|
(6
|
)
|
|
237
|
|
|
355
|
|
|
(28
|
)
|
||||
Foreign currency translation adjustment
|
117
|
|
|
—
|
|
|
75
|
|
|
—
|
|
||||
Comprehensive loss
|
(49,516
|
)
|
|
(70,940
|
)
|
|
(195,770
|
)
|
|
(261,686
|
)
|
||||
Comprehensive (income) loss attributable to noncontrolling interest
|
—
|
|
|
(126
|
)
|
|
2,213
|
|
|
(17
|
)
|
||||
Total comprehensive loss attributable to Portola
|
$
|
(49,516
|
)
|
|
$
|
(71,066
|
)
|
|
$
|
(193,557
|
)
|
|
$
|
(261,703
|
)
|
|
Common Stock
|
|
Additional
Paid-In
Capital
|
|
Accumulated
Deficit
|
|
Accumulated
Other
Comprehensive
Income (Loss)
|
|
Noncontrolling
Interest
|
|
Total
Stockholders’
Equity
|
|||||||||||||||
|
Shares
|
|
Amount
|
|
|
|
|
|
||||||||||||||||||
Balance at December 31, 2018
|
66,618
|
|
|
$
|
68
|
|
|
$
|
1,614,320
|
|
|
$
|
(1,525,704
|
)
|
|
$
|
(283
|
)
|
|
$
|
2,166
|
|
|
$
|
90,567
|
|
Issuance of common stock pursuant to equity award plans
|
1,359
|
|
|
2
|
|
|
25,660
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
25,662
|
|
||||||
Stock-based compensation expense
|
—
|
|
|
—
|
|
|
12,312
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
12,312
|
|
||||||
Other comprehensive income
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
203
|
|
|
—
|
|
|
203
|
|
||||||
Net (loss) income
|
—
|
|
|
—
|
|
|
—
|
|
|
(78,156
|
)
|
|
—
|
|
|
60
|
|
|
(78,096
|
)
|
||||||
Change in noncontrolling interest
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
2
|
|
|
2
|
|
||||||
Balance at March 31, 2019
|
67,977
|
|
|
$
|
70
|
|
|
$
|
1,652,292
|
|
|
$
|
(1,603,860
|
)
|
|
$
|
(80
|
)
|
|
$
|
2,228
|
|
|
$
|
50,650
|
|
Issuance of common stock pursuant to equity award plans
|
258
|
|
|
—
|
|
|
5,966
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
5,966
|
|
||||||
Stock-based compensation expense
|
—
|
|
|
—
|
|
|
12,369
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
12,369
|
|
||||||
Other comprehensive income
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
41
|
|
|
—
|
|
|
41
|
|
||||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
(66,204
|
)
|
|
—
|
|
|
(2,273
|
)
|
|
(68,477
|
)
|
||||||
Change in noncontrolling interest
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
45
|
|
|
45
|
|
||||||
Balance at June 30, 2019
|
68,235
|
|
|
$
|
70
|
|
|
$
|
1,670,627
|
|
|
$
|
(1,670,064
|
)
|
|
$
|
(39
|
)
|
|
$
|
—
|
|
|
$
|
594
|
|
Issuance of common stock pursuant to equity award plans
|
308
|
|
|
—
|
|
|
6,479
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
6,479
|
|
||||||
Issuance of common stock pursuant to public offering, net
|
9,241
|
|
|
9
|
|
|
243,706
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
243,715
|
|
||||||
Stock-based compensation expense
|
—
|
|
|
—
|
|
|
11,616
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
11,616
|
|
||||||
Other comprehensive income
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
111
|
|
|
—
|
|
|
111
|
|
||||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
(49,627
|
)
|
|
—
|
|
|
—
|
|
|
(49,627
|
)
|
||||||
Balance at September 30, 2019
|
77,784
|
|
|
$
|
79
|
|
|
$
|
1,932,428
|
|
|
$
|
(1,719,691
|
)
|
|
$
|
72
|
|
|
$
|
—
|
|
|
$
|
212,888
|
|
|
Common Stock
|
|
Additional
Paid-In
Capital
|
|
Accumulated
Deficit
|
|
Accumulated
Other
Comprehensive
Income (Loss)
|
|
Noncontrolling
Interest
|
|
Total
Stockholders’
Equity
|
|||||||||||||||
|
Shares
|
|
Amount
|
|
|
|
|
|
||||||||||||||||||
Balance at December 31, 2017
|
65,297
|
|
|
$
|
66
|
|
|
$
|
1,551,728
|
|
|
$
|
(1,204,519
|
)
|
|
$
|
(409
|
)
|
|
$
|
2,627
|
|
|
$
|
349,493
|
|
Adjustment to accumulated deficit due to adoption of ASC 606
|
—
|
|
|
—
|
|
|
—
|
|
|
29,037
|
|
|
—
|
|
|
—
|
|
|
29,037
|
|
||||||
Issuance of common stock pursuant to equity award plans
|
514
|
|
|
1
|
|
|
5,678
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
5,679
|
|
||||||
Stock-based compensation expense
|
—
|
|
|
—
|
|
|
10,980
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
10,980
|
|
||||||
Other comprehensive loss
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(392
|
)
|
|
—
|
|
|
(392
|
)
|
||||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
(84,178
|
)
|
|
—
|
|
|
(332
|
)
|
|
(84,510
|
)
|
||||||
Change in noncontrolling interest
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(119
|
)
|
|
(119
|
)
|
||||||
Balance at March 31, 2018
|
65,811
|
|
|
$
|
67
|
|
|
$
|
1,568,386
|
|
|
$
|
(1,259,660
|
)
|
|
$
|
(801
|
)
|
|
$
|
2,176
|
|
|
$
|
310,168
|
|
Issuance of common stock pursuant to equity award plans
|
143
|
|
|
—
|
|
|
2,543
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
2,543
|
|
||||||
Stock-based compensation expense
|
—
|
|
|
—
|
|
|
13,214
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
13,214
|
|
||||||
Other comprehensive income
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
127
|
|
|
—
|
|
|
127
|
|
||||||
Net (loss) income
|
—
|
|
|
—
|
|
|
—
|
|
|
(106,194
|
)
|
|
—
|
|
|
223
|
|
|
(105,971
|
)
|
||||||
Change in noncontrolling interest
|
—
|
|
|
—
|
|
|
—
|
|
|
|
|
—
|
|
|
(20
|
)
|
|
(20
|
)
|
|||||||
Balance at June 30, 2018
|
65,954
|
|
|
$
|
67
|
|
|
$
|
1,584,143
|
|
|
$
|
(1,365,854
|
)
|
|
$
|
(674
|
)
|
|
$
|
2,379
|
|
|
$
|
220,061
|
|
Issuance of common stock pursuant to equity award plans
|
480
|
|
|
—
|
|
|
6,413
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
6,413
|
|
||||||
Stock-based compensation expense
|
—
|
|
|
—
|
|
|
11,394
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
11,394
|
|
||||||
Other comprehensive income
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
237
|
|
|
—
|
|
|
237
|
|
||||||
Net (loss) income
|
—
|
|
|
—
|
|
|
—
|
|
|
(71,303
|
)
|
|
—
|
|
|
126
|
|
|
(71,177
|
)
|
||||||
Balance at September 30, 2018
|
66,434
|
|
|
$
|
67
|
|
|
$
|
1,601,950
|
|
|
$
|
(1,437,157
|
)
|
|
$
|
(437
|
)
|
|
$
|
2,505
|
|
|
$
|
166,928
|
|
|
Nine Months Ended September 30,
|
||||||
|
2019
|
|
2018
|
||||
Operating activities
|
|
|
|
||||
Net loss
|
$
|
(196,200
|
)
|
|
$
|
(261,658
|
)
|
Adjustments to reconcile net loss to cash used in operating activities:
|
|
|
|
||||
Depreciation and amortization
|
2,337
|
|
|
2,303
|
|
||
Amortization of right-of-use asset
|
2,056
|
|
|
—
|
|
||
Accretion of discount on investment securities
|
(946
|
)
|
|
(1,519
|
)
|
||
Non-cash interest expense
|
21,216
|
|
|
12,642
|
|
||
Stock-based compensation expense, net of capitalized labor
|
41,193
|
|
|
35,587
|
|
||
Remeasurement gain on embedded derivatives liabilities
|
(3,447
|
)
|
|
(3,668
|
)
|
||
Provision for excess and obsolete inventories
|
4,131
|
|
|
2,246
|
|
||
Loss on impairment of intangibles
|
3,151
|
|
|
—
|
|
||
Others
|
67
|
|
|
14
|
|
||
Changes in operating assets and liabilities:
|
|
|
|
||||
Inventories
|
2,312
|
|
|
(15,597
|
)
|
||
Trade and other receivables, net
|
(12,387
|
)
|
|
(694
|
)
|
||
Unbilled - collaboration and license revenue
|
6,092
|
|
|
(3,178
|
)
|
||
Prepaid expenses and other current assets
|
1,656
|
|
|
(3,253
|
)
|
||
Inventories, noncurrent portion
|
(23,651
|
)
|
|
(8,327
|
)
|
||
Prepaid and other long-term assets
|
1,636
|
|
|
5,475
|
|
||
Accounts payable
|
(2,967
|
)
|
|
4,749
|
|
||
Accrued research and development
|
(4,202
|
)
|
|
(28,298
|
)
|
||
Accrued and other liabilities
|
3,114
|
|
|
1,715
|
|
||
Deferred revenue
|
(875
|
)
|
|
(180
|
)
|
||
Notes payable, long term royalty-based debt and long-term obligation to collaborator
|
(8,871
|
)
|
|
(316
|
)
|
||
Other long-term liabilities
|
—
|
|
|
(720
|
)
|
||
Net cash used in operating activities
|
(164,585
|
)
|
|
(262,677
|
)
|
||
Investing activities
|
|
|
|
||||
Capital expenditures, net
|
(1,085
|
)
|
|
(1,577
|
)
|
||
Purchases of investments
|
(214,534
|
)
|
|
(218,330
|
)
|
||
Proceeds from maturities of investments
|
221,231
|
|
|
338,320
|
|
||
Net cash provided by investing activities
|
5,612
|
|
|
118,413
|
|
||
Financing activities
|
|
|
|
||||
Proceeds from debt issuance, net
|
59,203
|
|
|
95,000
|
|
||
Proceeds from issuance of common stock from public offering, net
|
244,065
|
|
|
—
|
|
||
Proceeds from issuance of common stock pursuant to equity award plans
|
23,082
|
|
|
14,635
|
|
||
Other
|
—
|
|
|
(140
|
)
|
||
Net cash provided by financing activities
|
326,350
|
|
|
109,495
|
|
||
Effect of exchange rate changes on cash, cash equivalents and restricted cash
|
76
|
|
|
—
|
|
||
Net increase (decrease) in cash, cash equivalents and restricted cash
|
167,453
|
|
|
(34,769
|
)
|
||
Cash, cash equivalents and restricted cash at beginning of period
|
140,013
|
|
|
181,741
|
|
||
Cash, cash equivalents and restricted cash at end of period
|
$
|
307,466
|
|
|
$
|
146,972
|
|
|
September 30, 2019
|
|
December 31, 2018
|
|
September 30, 2018
|
|
December 31, 2017
|
||||||||
Cash and cash equivalents
|
$
|
304,265
|
|
|
$
|
138,951
|
|
|
$
|
146,700
|
|
|
$
|
181,568
|
|
Restricted cash (SRX Cardio)
|
—
|
|
|
30
|
|
|
30
|
|
|
173
|
|
||||
Restricted cash for royalty payments to HealthCare Royalty Partners and its affiliates ("HCR")
|
3,155
|
|
|
1,032
|
|
|
242
|
|
|
—
|
|
||||
Restricted cash (lease)
|
46
|
|
|
—
|
|
|
—
|
|
|
—
|
|
||||
Total cash balance in condensed consolidated statements of cash flows
|
$
|
307,466
|
|
|
$
|
140,013
|
|
|
$
|
146,972
|
|
|
$
|
181,741
|
|
•
|
Transition Elections. We elected the package of practical expedients that permits us to not reassess under the new standard our prior conclusions about lease identification, lease classification, and initial direct costs. We also elected the practical expedient to not separate lease and non-lease components for facility lease classes of underlying assets to new or modified leases beginning on or after the adoption date. That is, we will account for each separate lease component of a contract and its associated non-lease components as a single lease component.
|
•
|
Ongoing Accounting Policy Elections. We elected the short-term lease recognition exemption whereby ROU assets and lease liabilities will not be recognized for leasing arrangements with terms less than one year.
|
|
Three Months Ended September 30, 2019
|
|
Nine Months Ended September 30, 2019
|
||||||||||||||||||||
|
Product Revenue, net
|
|
Collaboration and License Revenue
|
|
Total
|
|
Product Revenue, net
|
|
Collaboration and License Revenue
|
|
Total
|
||||||||||||
Timing of revenue recognition:
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||||
Transferred at a point in time
|
$
|
35,743
|
|
|
$
|
—
|
|
|
$
|
35,743
|
|
|
$
|
83,269
|
|
|
$
|
—
|
|
|
$
|
83,269
|
|
Transferred over time
|
—
|
|
|
1,056
|
|
|
1,056
|
|
|
—
|
|
|
4,123
|
|
|
4,123
|
|
||||||
Total
|
$
|
35,743
|
|
|
$
|
1,056
|
|
|
$
|
36,799
|
|
|
$
|
83,269
|
|
|
$
|
4,123
|
|
|
$
|
87,392
|
|
|
Three Months Ended September 30, 2018
|
|
Nine Months Ended September 30, 2018
|
||||||||||||||||||||
|
Product Revenue, net
|
|
Collaboration and License Revenue
|
|
Total
|
|
Product Revenue, net
|
|
Collaboration and License Revenue
|
|
Total
|
||||||||||||
Timing of revenue recognition:
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||||
Transferred at a point in time
|
$
|
7,176
|
|
|
$
|
—
|
|
|
$
|
7,176
|
|
|
$
|
10,047
|
|
|
$
|
—
|
|
|
$
|
10,047
|
|
Transferred over time
|
—
|
|
|
7,001
|
|
|
7,001
|
|
|
—
|
|
|
14,785
|
|
|
14,785
|
|
||||||
Total
|
$
|
7,176
|
|
|
$
|
7,001
|
|
|
$
|
14,177
|
|
|
$
|
10,047
|
|
|
$
|
14,785
|
|
|
$
|
24,832
|
|
|
Three Months Ended September 30,
|
|
Nine Months Ended September 30,
|
||||||||||||
|
2019
|
|
2018
|
|
2019
|
|
2018
|
||||||||
United States
|
$
|
32,968
|
|
|
$
|
7,176
|
|
|
$
|
80,494
|
|
|
$
|
10,047
|
|
Europe
|
2,775
|
|
|
—
|
|
|
2,775
|
|
|
—
|
|
||||
Total revenues
|
$
|
35,743
|
|
|
$
|
7,176
|
|
|
$
|
83,269
|
|
|
$
|
10,047
|
|
|
Balance at
Beginning of
Period
|
|
Addition
|
|
Deduction
|
|
Balance at End
of Period
|
||||||||
Contract assets:
|
|
|
|
|
|
|
|
||||||||
Unbilled - collaboration and license revenue
|
$
|
9,880
|
|
|
$
|
3,461
|
|
|
$
|
(9,553
|
)
|
|
$
|
3,788
|
|
Total contract assets
|
$
|
9,880
|
|
|
$
|
3,461
|
|
|
$
|
(9,553
|
)
|
|
$
|
3,788
|
|
|
|
|
|
|
|
|
|
||||||||
Contract liabilities:
|
|
|
|
|
|
|
|
||||||||
Deferred revenue
|
$
|
6,335
|
|
|
$
|
1,402
|
|
|
$
|
(2,277
|
)
|
|
$
|
5,460
|
|
Total contract liabilities
|
$
|
6,335
|
|
|
$
|
1,402
|
|
|
$
|
(2,277
|
)
|
|
$
|
5,460
|
|
|
Three Months
Ended as of September 30, 2019 |
|
Nine Months Ended September 30, 2019
|
||||
Revenue recognized according to the current period performance that was included in the contract liability at the beginning of the period
|
$
|
47
|
|
|
$
|
662
|
|
Collaborator
|
Transaction Price
Allocated to the Remaining Performance Obligation as of September 30, 2019 |
|
Expected Year
By Which Revenue
Recognition Will
Be Completed
|
|
Percentage of
Revenue
Recognized
|
|||
BMS and Pfizer - 2016 agreement
|
$
|
450
|
|
|
2021
|
|
96
|
%
|
Daiichi Sankyo - 2014 agreement
|
772
|
|
|
2020
|
|
98
|
%
|
|
Daiichi Sankyo - 2016 agreement
|
2,485
|
|
|
2023
|
|
84
|
%
|
|
Bayer - 2016 agreement
|
2,018
|
|
|
2023
|
|
87
|
%
|
|
Total
|
$
|
5,725
|
|
|
|
|
|
Level 1 -Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date.
|
Level 2 -Inputs (other than quoted market prices included in Level 1) are either directly or indirectly observable for the asset or liability through correlation with market data at the measurement date and for the duration of the instrument’s anticipated life.
|
Level 3 -Inputs reflect management’s best estimate of what market participants would use in pricing the asset or liability at the measurement date. Consideration is given to the risk inherent in the valuation technique and the risk inherent in the inputs to the model.
|
|
|
|
September 30, 2019
|
|
December 31, 2018
|
||||||||||||||||||||||||||||
|
Fair Value
Hierarchy
|
|
Amortized
Cost
|
|
Unrealized
Gain
|
|
Unrealized
(Loss)
|
|
Estimated
Fair
Value
|
|
Amortized
Cost
|
|
Unrealized
Gain
|
|
Unrealized
(Loss)
|
|
Estimated
Fair
Value
|
||||||||||||||||
Money market funds
|
Level 1
|
|
$
|
88,619
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
88,619
|
|
|
$
|
19,500
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
19,500
|
|
Corporate notes and commercial paper
|
Level 2
|
|
228,354
|
|
|
2
|
|
|
(8
|
)
|
|
228,348
|
|
|
166,363
|
|
|
1
|
|
|
(205
|
)
|
|
166,159
|
|
||||||||
U.S. Treasury bills and government agency securities
|
Level 2
|
|
98,432
|
|
|
10
|
|
|
(7
|
)
|
|
98,435
|
|
|
110,270
|
|
|
1
|
|
|
(81
|
)
|
|
110,190
|
|
||||||||
|
|
|
$
|
415,405
|
|
|
$
|
12
|
|
|
$
|
(15
|
)
|
|
$
|
415,402
|
|
|
$
|
296,133
|
|
|
$
|
2
|
|
|
$
|
(286
|
)
|
|
$
|
295,849
|
|
Classified as:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||||||||
Cash equivalents
|
|
|
|
|
|
|
|
|
$
|
242,860
|
|
|
|
|
|
|
|
|
$
|
117,836
|
|
||||||||||||
Short-term investments
|
|
|
|
|
|
|
|
|
172,542
|
|
|
|
|
|
|
|
|
178,013
|
|
||||||||||||||
Total cash equivalents and investments
|
|
|
|
|
|
|
|
|
$
|
415,402
|
|
|
|
|
|
|
|
|
$
|
295,849
|
|
|
Embedded derivative liabilities
|
|
Lonza award
|
|
Total
|
||||||
Balance as of December 31, 2018
|
$
|
2,497
|
|
|
$
|
9,201
|
|
|
$
|
11,698
|
|
Net change in the fair value
|
(3,447
|
)
|
|
5,824
|
|
|
2,377
|
|
|||
Addition of derivative related to 2019 Secured Term Loan
|
2,372
|
|
|
—
|
|
|
2,372
|
|
|||
Settlement of Lonza award
|
—
|
|
|
(15,025
|
)
|
|
(15,025
|
)
|
|||
Balance as of September 30, 2019
|
$
|
1,422
|
|
|
$
|
—
|
|
|
$
|
1,422
|
|
|
September 30, 2019
|
|
December 31, 2018
|
||||
Raw materials
|
$
|
13,716
|
|
|
$
|
279
|
|
Work in process
|
21,118
|
|
|
14,395
|
|
||
Finished goods
|
819
|
|
|
2,844
|
|
||
Total inventories
|
$
|
35,653
|
|
|
$
|
17,518
|
|
|
|
|
|
||||
Balance Sheet Classification
|
|
|
|
||||
Inventories
|
$
|
2,357
|
|
|
$
|
7,873
|
|
Inventories, noncurrent portion
|
33,296
|
|
|
9,645
|
|
||
Total inventories
|
$
|
35,653
|
|
|
$
|
17,518
|
|
|
September 30, 2019
|
|
December 31, 2018
|
||||
Manufacturing related
|
$
|
6,349
|
|
|
$
|
5,465
|
|
Compensation and employee benefits
|
13,480
|
|
|
10,794
|
|
||
Current portion of lease liability
|
3,566
|
|
|
—
|
|
||
Others
|
9,457
|
|
|
6,051
|
|
||
Total accrued and other liabilities
|
$
|
32,852
|
|
|
$
|
22,310
|
|
|
September 30, 2019
|
|
December 31, 2018
|
||||
Total repayment obligations
|
$
|
62,500
|
|
|
$
|
62,500
|
|
Less: interests to be accreted in future periods
|
(6,191
|
)
|
|
(8,643
|
)
|
||
Less: payments made
|
(3,564
|
)
|
|
(497
|
)
|
||
Carrying value of notes payable
|
52,745
|
|
|
53,360
|
|
||
Less: current portion of royalties
|
(10,817
|
)
|
|
(5,062
|
)
|
||
Non-current portion of notes payable
|
$
|
41,928
|
|
|
$
|
48,298
|
|
|
September 30, 2019
|
|
December 31, 2018
|
||||
Total repayment obligations
|
$
|
290,550
|
|
|
$
|
290,550
|
|
Less: interests to be accreted in future periods
|
(109,060
|
)
|
|
(125,851
|
)
|
||
Less: payments made
|
(6,006
|
)
|
|
(816
|
)
|
||
Carrying value of long term royalty-based debt
|
175,484
|
|
|
163,883
|
|
||
Less: current portion of royalties
|
(18,302
|
)
|
|
(8,627
|
)
|
||
Non-current portion of long term royalty-based debt
|
$
|
157,182
|
|
|
$
|
155,256
|
|
Year ended December 31,
|
|
||
2022
|
$
|
9,615
|
|
2023
|
9,615
|
|
|
2024
|
9,615
|
|
|
Thereafter
|
33,655
|
|
|
Total
|
$
|
62,500
|
|
|
Shares
Subject to
Outstanding
Options
|
|
Weighted-
Average Exercise
Price Per Share
|
|||
Balance at December 31, 2018
|
7,507,690
|
|
|
$
|
33.25
|
|
Options granted
|
1,962,744
|
|
|
27.65
|
|
|
Options exercised
|
(821,489
|
)
|
|
21.68
|
|
|
Options canceled
|
(996,552
|
)
|
|
38.69
|
|
|
Balance at September 30, 2019
|
7,652,393
|
|
|
$
|
32.35
|
|
|
Shares
Subject to
Outstanding
PSOs
|
|
Weighted-
Average Exercise
Price Per Share
|
|||
Balance at December 31, 2018
|
143,335
|
|
|
$
|
23.76
|
|
Options granted
|
490,986
|
|
|
33.29
|
|
|
Options exercised
|
(31,667
|
)
|
|
23.76
|
|
|
Options canceled
|
(24,187
|
)
|
|
33.29
|
|
|
Balance at September 30, 2019
|
578,467
|
|
|
$
|
31.45
|
|
|
Shares
Subject to
Outstanding
RSUs
|
|
Weighted-
Average Grant Date
Fair Value Per Share
|
|||
Balance at December 31, 2018
|
979,278
|
|
|
$
|
34.00
|
|
RSUs granted
|
767,143
|
|
|
27.75
|
|
|
RSUs released
|
(359,726
|
)
|
|
35.24
|
|
|
RSUs canceled
|
(133,114
|
)
|
|
34.88
|
|
|
Balance at September 30, 2019
|
1,253,581
|
|
|
$
|
29.73
|
|
|
Subject to
Outstanding
PSUs
|
|
Weighted-
Average Grant Date
Fair Value Per Share
|
|||
Balance at December 31, 2018
|
153,503
|
|
|
$
|
29.85
|
|
PSUs granted
|
—
|
|
|
—
|
|
|
PSUs released
|
(52,670
|
)
|
|
28.29
|
|
|
PSUs canceled
|
(87,708
|
)
|
|
30.36
|
|
|
Balance at September 30, 2019
|
13,125
|
|
|
$
|
32.66
|
|
|
Three Months Ended September 30,
|
|
Nine Months Ended September 30,
|
||||||||||||
|
2019
|
|
2018
|
|
2019
|
|
2018
|
||||||||
Research and development
|
$
|
3,731
|
|
|
$
|
4,379
|
|
|
$
|
17,437
|
|
|
$
|
14,151
|
|
Selling, general and administrative
|
7,315
|
|
|
7,014
|
|
|
23,756
|
|
|
21,436
|
|
||||
Stock-based compensation expense included in total expenses
|
$
|
11,046
|
|
|
$
|
11,393
|
|
|
$
|
41,193
|
|
|
$
|
35,587
|
|
|
|
|
|
|
|
|
|
||||||||
Capitalized stock-based compensation costs
|
$
|
(569
|
)
|
|
$
|
—
|
|
|
$
|
(927
|
)
|
|
$
|
—
|
|
|
Nine Months Ended September 30,
|
||||
|
2019
|
|
2018
|
||
Stock options to purchase common stock
|
7,652,393
|
|
|
7,200,427
|
|
Performance stock options
|
578,467
|
|
|
147,335
|
|
Common stock warrants
|
1,500
|
|
|
1,500
|
|
Restricted stock units
|
1,253,581
|
|
|
809,014
|
|
Performance stock units
|
13,125
|
|
|
160,378
|
|
Employee stock purchase plan
|
18,754
|
|
|
19,062
|
|
|
Three Months Ended September 30, 2019
|
|
Nine Months Ended September 30, 2019
|
||||
Operating lease costs
|
$
|
975
|
|
|
$
|
2,056
|
|
Short-term lease cost
|
89
|
|
|
267
|
|
||
Total
|
$
|
1,064
|
|
|
$
|
2,323
|
|
|
Nine Months Ended September 30, 2019
|
||
Cash paid for amounts included in the measurement of lease liabilities:
|
|
||
Operating cash flows from operating leases
|
$
|
2,093
|
|
Supplemental non-cash information:
|
|
||
Right-of-use asset obtained in exchange for lease obligation due to remeasurement
|
$
|
11,103
|
|
Right-of-use asset obtained in exchange for a new operating lease liability
|
$
|
270
|
|
|
Classification
|
|
As of September 30, 2019
|
||
Operating lease
|
|
|
|
||
Lease right-of-use assets
|
|
|
|
||
Non-current
|
Operating lease right-of-use asset
|
|
$
|
11,836
|
|
Lease liabilities
|
|
|
|
||
Current
|
Accrued and other liabilities
|
|
$
|
3,566
|
|
Non-current
|
Long-term portion of lease liability
|
|
$
|
9,464
|
|
|
|
|
|
||
Weighted Average Remaining Lease Term
|
|
|
|
||
Operating leases
|
|
|
3.5 years
|
||
|
|
|
|
||
Weighted Average Discount Rate
|
|
|
|
||
Operating leases
|
|
|
6.60%
|
Year ending December 31,
|
Operating Lease
|
||
Remainder of 2019
|
$
|
733
|
|
2020
|
4,173
|
|
|
2021
|
4,628
|
|
|
2022
|
4,713
|
|
|
2023
|
1,188
|
|
|
Total lease payments
|
15,435
|
|
|
Less imputed interests
|
(2,405
|
)
|
|
Total lease liabilities
|
$
|
13,030
|
|
ITEM 2.
|
MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
|
|
Description
|
|
Approved or Investigational Indication
|
|
Stage
|
|
Commercial rights
|
Andexxa
|
Reversal agent for certain Factor Xa (fXa) inhibitors
|
|
Patients treated with rivaroxaban or apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding
|
|
U.S. Approval
EU Approval
|
|
Worldwide excluding Japan
|
Bevyxxa
|
Oral fXa inhibitor
|
|
Extended duration VTE prophylaxis in acute medically ill patients in-hospital and post discharge for 35-42 days
|
|
U.S. Approval
|
|
Worldwide
|
Cerdulatinib
|
Oral, dual SYK and JAK inhibitor
|
|
Relapsed/refractory B- and T-cell malignancies
|
|
Phase 2a
|
|
Worldwide excluding topical formulation in non-oncology indications
|
•
|
A high rate of hemostatic efficacy (79%) consistent with that of the full ANNEXA-4 trial across patients with all types of bleeds (82%)
|
•
|
Of the patients that achieved excellent or good hemostatic efficacy within one hour post Andexxa, 98% (n=55/56) maintained excellent or good hemostatic control 12 hours following Andexxa administration.
|
•
|
The majority of thrombotic events occurred in patients who delayed or did not re-start anticoagulation therapy with a Factor Xa inhibitor during the follow-up period.
|
•
|
Importantly, no thrombotic events were observed among the 18 patients who re-started oral anticoagulation therapy within 30 days.
|
|
Three Months Ended September 30,
|
|
|
|
|
|
Nine Months Ended September 30,
|
|
|
|
|
||||||||||||||||
|
2019
|
|
2018
|
|
Change
|
|
% Change
|
|
2019
|
|
2018
|
|
Change
|
|
% Change
|
||||||||||||
|
(in thousands, except percentages)
|
||||||||||||||||||||||||||
Andexxa
|
$
|
32,951
|
|
|
$
|
7,728
|
|
|
$
|
25,223
|
|
|
326%
|
|
$
|
80,326
|
|
|
$
|
9,960
|
|
|
$
|
70,366
|
|
|
706%
|
Ondexxya
|
2,775
|
|
|
—
|
|
|
2,775
|
|
|
*
|
|
2,775
|
|
|
—
|
|
|
2,775
|
|
|
*
|
||||||
Bevyxxa
|
17
|
|
|
(552
|
)
|
|
569
|
|
|
103%
|
|
168
|
|
|
87
|
|
|
81
|
|
|
93%
|
||||||
Total product revenue, net
|
35,743
|
|
|
7,176
|
|
|
28,567
|
|
|
398%
|
|
83,269
|
|
|
10,047
|
|
|
73,222
|
|
|
729%
|
||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||||
Total collaboration and license revenue
|
1,056
|
|
|
7,001
|
|
|
(5,945
|
)
|
|
(85%)
|
|
4,123
|
|
|
14,785
|
|
|
(10,662
|
)
|
|
(72%)
|
||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||||
Total revenues
|
$
|
36,799
|
|
|
$
|
14,177
|
|
|
$
|
22,622
|
|
|
160%
|
|
$
|
87,392
|
|
|
$
|
24,832
|
|
|
$
|
62,560
|
|
|
252%
|
•
|
commercial product revenue earned from U.S. net sales of Andexxa, which we began shipping to customers in May 2018 and more broadly in January 2019 following the FDA approval of our Gen 2 manufacturing process, and commercial product revenue earned from the EU starting from the third quarter of 2019; offset by
|
•
|
a decrease in Phase 3 collaboration revenue from certain collaboration partners as related performance obligations have been mostly fulfilled by the first quarter of 2019.
|
|
Three Months Ended September 30,
|
|
|
|
|
|
Nine Months Ended September 30,
|
|
|
|
|
||||||||||||||||||
|
2019
|
|
2018
|
|
Change
|
|
% Change
|
|
2019
|
|
2018
|
|
Change
|
|
% Change
|
||||||||||||||
|
(in thousands, except percentages)
|
||||||||||||||||||||||||||||
Cost of sales
|
$
|
2,684
|
|
|
$
|
4,292
|
|
|
$
|
(1,608
|
)
|
|
(37
|
)%
|
|
$
|
14,825
|
|
|
$
|
5,680
|
|
|
$
|
9,145
|
|
|
161
|
%
|
•
|
Bevyxxa manufacturing related penalty for anticipated shortfall in production of $1.2 million recorded in the three months ended September 2018; and
|
•
|
Provision for excess and obsolete inventories of $1.7 million recorded in the three months ended September 2018; offset by
|
•
|
Cost of sales related to increased Andexxa sales during the three months ended September 30, 2019 compared to the three months ended September 30, 2018.
|
Product candidate
|
Phase of
Development
|
|
Three Months Ended September 30,
|
|
|
|
|
|
Nine Months Ended September 30,
|
|
|
|
|
||||||||||||||||||
|
2019
|
|
2018
|
|
Change
|
|
% Change
|
|
2019
|
|
2018
|
|
Change
|
|
% Change
|
||||||||||||||||
Andexanet alfa
|
Phase 2/3/4
|
|
$
|
14,995
|
|
|
$
|
25,727
|
|
|
$
|
(10,732
|
)
|
|
(42
|
%)
|
|
$
|
58,109
|
|
|
$
|
122,757
|
|
|
$
|
(64,648
|
)
|
|
(53
|
%)
|
Betrixaban
|
Phase 1/3
|
|
1,200
|
|
|
6,799
|
|
|
(5,599
|
)
|
|
(82
|
%)
|
|
2,581
|
|
|
20,235
|
|
|
(17,654
|
)
|
|
(87
|
%)
|
||||||
Cerdulatinib
|
Phase 1/2a
|
|
7,304
|
|
|
6,433
|
|
|
871
|
|
|
14
|
%
|
|
23,501
|
|
|
17,826
|
|
|
5,675
|
|
|
32
|
%
|
||||||
Other research and development expenses(1)
|
|
|
2,148
|
|
|
1,278
|
|
|
870
|
|
|
68
|
%
|
|
10,578
|
|
|
5,926
|
|
|
4,652
|
|
|
79
|
%
|
||||||
Total research and development expenses
|
|
|
$
|
25,647
|
|
|
$
|
40,237
|
|
|
$
|
(14,590
|
)
|
|
(36
|
%)
|
|
$
|
94,769
|
|
|
$
|
166,744
|
|
|
$
|
(71,975
|
)
|
|
(43
|
%)
|
(1)
|
Amounts in all periods include costs for other potential product candidates.
|
•
|
decreased program costs related to Andexxa, which was largely the result of capitalizing manufacturing expenses that were recorded as research and development expenses in 2018; and
|
•
|
decreased program costs related to Bevyxxa, which was the result of overall decreased spending; offset by
|
•
|
increased program costs related to 1) cerdulatinib, primarily due to increased manufacturing expenses and 2) an impairment charge of $3.2 million due to the abandonment of the SRX Cardio program during the second quarter of 2019.
|
|
Three Months Ended September 30,
|
|
|
|
|
|
Nine Months Ended September 30,
|
|
|
|
|
||||||||||||||||||
|
2019
|
|
2018
|
|
Change
|
|
% Change
|
|
2019
|
|
2018
|
|
Change
|
|
% Change
|
||||||||||||||
|
(in thousands, except percentages)
|
||||||||||||||||||||||||||||
Selling, general and administrative expenses
|
$
|
52,050
|
|
|
$
|
38,792
|
|
|
$
|
13,258
|
|
|
34
|
%
|
|
$
|
158,939
|
|
|
$
|
110,547
|
|
|
$
|
48,392
|
|
|
44
|
%
|
•
|
increased headcount-related costs of $9.4 million resulting from the hiring of our sales force and supporting commercial functions; and
|
•
|
increased external costs of $4.8 million associated with commercial and marketing initiatives to support the launch of Andexxa.
|
•
|
increased headcount-related costs of $32.3 million resulting from the hiring of our sales force and supporting commercial functions; and
|
•
|
increased external costs of $15.0 million associated with commercial and marketing initiatives to support the launch of Andexxa.
|
|
Three Months Ended September 30,
|
|
|
|
|
|
Nine Months Ended September 30,
|
|
|
|
|
||||||||||||||||||
|
2019
|
|
2018
|
|
Change
|
|
% Change
|
|
2019
|
|
2018
|
|
Change
|
|
% Change
|
||||||||||||||
|
(in thousands, except percentages)
|
||||||||||||||||||||||||||||
Interest expense
|
$
|
7,998
|
|
|
$
|
5,957
|
|
|
$
|
2,041
|
|
|
34
|
%
|
|
$
|
23,017
|
|
|
$
|
12,642
|
|
|
$
|
10,375
|
|
|
82
|
%
|
•
|
An additional $95.0 million of funding received in May 2018 under the Andexxa Royalty Sales Agreement with HCR; and
|
•
|
An additional $62.5 million of funding received in March 2019 under the Credit Agreement with HCR and Athyrium.
|
•
|
the timing, receipt and amount of sales, profit sharing or royalties, if any, from our current and potential products;
|
•
|
the cost of manufacturing our current products and product candidates, including process improvements in order to manufacture product candidates at commercial scale, and establishing commercial supplies of our product candidates;
|
•
|
the cost and timing of establishing sales, marketing and distribution capabilities in the United States and abroad;
|
•
|
the terms and timing of any other collaborative, licensing and other arrangements that we may establish;
|
•
|
the receipt of any collaboration payments;
|
•
|
the number and characteristics of product candidates that we pursue;
|
•
|
the cost, timing and outcomes of regulatory approvals;
|
•
|
the scope, rate of progress, results and cost of our clinical studies, preclinical testing and other related activities;
|
•
|
the cost of preparing, filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights;
|
•
|
the extent to which we acquire or invest in businesses, products or technologies, although we currently have no commitments or agreements relating to any of these types of transactions; and
|
•
|
partnerships and other strategic options for our products and product candidates.
|
|
Nine Months Ended September 30,
|
||||||
|
2019
|
|
2018
|
||||
|
(in thousands)
|
||||||
Cash used in operating activities
|
$
|
(164,585
|
)
|
|
$
|
(262,677
|
)
|
Cash provided by investing activities
|
5,612
|
|
|
118,413
|
|
||
Cash provided by financing activities
|
326,350
|
|
|
109,495
|
|
||
Effect of exchange rate changes on cash, cash equivalents and restricted cash
|
76
|
|
|
—
|
|
||
Net increase (decrease) in cash, cash equivalents and restricted cash
|
$
|
167,453
|
|
|
$
|
(34,769
|
)
|
ITEM 3:
|
QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK
|
ITEM 4:
|
CONTROLS AND PROCEDURES
|
ITEM 1.
|
LEGAL PROCEEDINGS
|
Item 1A.
|
RISK FACTORS.
|
•
|
establish and scale-up manufacturing capabilities and a sales, marketing and distribution infrastructure to commercialize our products in the U.S. and abroad;
|
•
|
initiate or continue clinical studies, including a post-marketing randomized controlled trial of Andexxa;
|
•
|
continue the research and development of our product candidates;
|
•
|
seek to discover or in-license additional product candidates;
|
•
|
seek regulatory approvals for our product candidates that successfully complete clinical studies; and
|
•
|
enhance operational, compliance, financial, quality and information management systems.
|
•
|
the level of demand and market acceptance;
|
•
|
the results of our clinical trials;
|
•
|
our ability to obtain and maintain desired regulatory approvals in the U.S., EU and other foreign jurisdictions;
|
•
|
the extent to which coverage and reimbursement is available from government and health administration authorities, private health insurers, managed care programs and other third-party payors;
|
•
|
rebates, discount, other pricing concessions and fees that we may provide to integrated delivery networks, group purchasing organizations, other purchasers and pharmacy benefits managers and other third-party payors;
|
•
|
the timing, cost and level of investment in our marketing efforts to support sales;
|
•
|
the timing, cost and level of investment in our research and development activities involving approved products and product candidates;
|
•
|
the cost of manufacturing, distribution and the amount of legally mandated discounts to government entities, other discounts and rebates, product returns and other gross-to-net deductions;
|
•
|
the risk/benefit profile, cost and reimbursement of existing and potential future drugs which compete with approved products;
|
•
|
the timing and amount of non-cash items such as stock compensation expenses, reserves, cost of goods sold and non-recurring charges such as inventory write-offs; and
|
•
|
expenditures that we will or may incur to acquire or develop additional product candidates and technologies.
|
•
|
product sales of Andexxa and Ondexxya, and if approved for commercial marketing, our product candidates;
|
•
|
the costs of commercialization activities, including product sales, marketing, manufacturing and distribution and general corporate and commercial infrastructure;
|
•
|
the costs and timing of international expansion;
|
•
|
the timing of, and costs involved in, seeking and obtaining approvals from the FDA and other regulatory authorities;
|
•
|
the possible development of additional product candidates, including through in-licensing and acquisitions;
|
•
|
the degree and rate of market acceptance of any products launched by us or partners;
|
•
|
our ability to enter into additional collaboration, licensing, commercialization or other financing arrangements and the terms and timing of such arrangements;
|
•
|
the rate of progress and cost of our clinical studies; and
|
•
|
the emergence of competing technologies or other adverse market developments.
|
•
|
Incur additional debt;
|
•
|
Make certain investments and acquisitions;
|
•
|
Guarantee the indebtedness of others or our subsidiaries;
|
•
|
Create liens or encumbrances;
|
•
|
Engage in new lines of business;
|
•
|
Enter into transactions with affiliates;
|
•
|
Pay cash dividends and make distributions;
|
•
|
Redeem or repurchase capital shares;
|
•
|
Sell, lease or transfer certain parts of our business or property;
|
•
|
Prepay other indebtedness; and
|
•
|
Acquire new companies and merge or consolidate.
|
•
|
the prevalence and severity of any side effects;
|
•
|
efficacy and potential advantages compared to alternative treatments;
|
•
|
the price we charge for our products;
|
•
|
interpretations of the results of our clinical trials;
|
•
|
the willingness of physicians and healthcare organizations to change their current treatment practices;
|
•
|
the willingness of hospitals and hospital systems to include our products as treatment options;
|
•
|
convenience and ease of administration compared to alternative treatments;
|
•
|
the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies;
|
•
|
the willingness of the target patient population to pay for our products, including co-pays under their health coverage plans;
|
•
|
the strength of marketing and distribution support; and
|
•
|
the availability of third-party coverage and adequate reimbursement.
|
•
|
the number of patients required for clinical studies of our product candidates may be larger than we anticipate, enrollment in these clinical studies may be insufficient or slower than we anticipate or patients may drop out of these clinical studies at a higher rate than we anticipate;
|
•
|
clinical studies of our product candidates may produce negative or inconclusive results, and we may decide, or regulators may require us, to conduct additional clinical studies or abandon product development programs;
|
•
|
the cost of clinical studies or the manufacturing of our product candidates may be greater than we anticipate;
|
•
|
our third-party contractors may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all;
|
•
|
we might have to suspend or terminate clinical studies of our product candidates for various reasons, including unanticipated serious side effects, other unexpected characteristics or unacceptable health risks;
|
•
|
regulators may not approve our proposed clinical development plans;
|
•
|
regulators or institutional review boards may not authorize us or our investigators to commence a clinical study or conduct a clinical study at a prospective study site;
|
•
|
regulators or institutional review boards may require that we or our investigators suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements; and
|
•
|
the supply or quality of our product candidates or other materials necessary to conduct clinical studies of our product candidates may be insufficient or inadequate.
|
•
|
be delayed in obtaining marketing approval for our product candidates;
|
•
|
not obtain marketing approval at all;
|
•
|
obtain approval for indications that are not as broad as intended;
|
•
|
have the product removed from the market after obtaining marketing approval;
|
•
|
be subject to additional post-marketing testing requirements; or
|
•
|
be subject to restrictions on how the product is distributed or used.
|
•
|
mandate modifications to product labelling or promotional materials or require us to provide corrective information to healthcare practitioners;
|
•
|
suspend any regulatory approvals;
|
•
|
suspend any ongoing clinical trials;
|
•
|
refuse to approve pending applications or supplements to approved applications filed by us, our partners or our potential future partners;
|
•
|
impose restrictions on operations, including costly new manufacturing requirements; or
|
•
|
seize or detain products or require a product recall.
|
•
|
collaborators have significant discretion in determining the efforts and resources that they will apply to any such collaborations;
|
•
|
collaborators may pursue pricing which is not consistent with our global strategies;
|
•
|
collaborators may not pursue development and commercialization of our product candidates or may elect not to continue or renew development or commercialization programs based on clinical study results, changes in their strategic focus due to the acquisition of competitive products, availability of funding or other external factors, such as a business combination that diverts resources or creates competing priorities;
|
•
|
collaborators may delay clinical studies, provide insufficient funding for a clinical study program, stop a clinical study, abandon a product candidate, repeat or conduct new clinical studies or require a new formulation of a product candidate for clinical testing;
|
•
|
collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our products or product candidates;
|
•
|
a collaborator with marketing and distribution rights to one or more products may not commit sufficient resources to their marketing and distribution;
|
•
|
collaborators may not properly maintain or defend our intellectual property rights or may use our intellectual property or proprietary information in a way that gives rise to actual or threatened litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential liability;
|
•
|
disputes may arise between us and a collaborator that causes the delay or termination of the research, development or commercialization of our product candidates or that results in costly litigation or arbitration that diverts management attention and resources;
|
•
|
collaborations may be terminated and, if terminated, may result in a need for additional capital to pursue further development or commercialization of the applicable product candidates; and
|
•
|
collaborators may own or co-own intellectual property covering our products that results from our collaborating with them, and in such cases, we may not have the exclusive right to commercialize such intellectual property.
|
•
|
decreased demand for our products;
|
•
|
injury to our reputation and significant negative media attention;
|
•
|
withdrawal of patients from clinical studies or cancellation of studies;
|
•
|
significant costs to defend the related litigation;
|
•
|
substantial monetary awards to patients;
|
•
|
loss of revenue; and
|
•
|
the inability to commercialize any additional products that we may develop.
|
•
|
different regulatory requirements for drug approvals in foreign countries;
|
•
|
differing payor reimbursement regimes, governmental payors or patient self-pay systems and price control;
|
•
|
reduced protection for intellectual property rights;
|
•
|
unexpected changes in tariffs, trade barriers and regulatory requirements;
|
•
|
economic weakness, including inflation or political instability in particular foreign economies and markets;
|
•
|
compliance with tax, employment, immigration and labor laws for employees living or traveling abroad;
|
•
|
foreign taxes, including withholding of payroll taxes;
|
•
|
foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country;
|
•
|
workforce uncertainty in countries where labor unrest is more common than in the United States;
|
•
|
production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and
|
•
|
business interruptions resulting from geopolitical actions, including war and terrorism, or natural disasters including earthquakes, typhoons, floods and fires.
|
•
|
warning letters;
|
•
|
civil or criminal penalties and fines;
|
•
|
injunctions;
|
•
|
suspension or withdrawal of regulatory approval;
|
•
|
suspension of any ongoing clinical studies;
|
•
|
voluntary or mandatory product recalls and publicity requirements;
|
•
|
refusal to accept or approve applications for marketing approval of new drugs or biologics or supplements to approved applications submitted by us;
|
•
|
restrictions on operations, including costly new manufacturing requirements; or
|
•
|
seizure or detention of our products or import bans.
|
•
|
a product candidate may not be deemed safe or effective;
|
•
|
FDA officials may not find the data from preclinical studies and clinical studies sufficient;
|
•
|
the FDA may find our manufacturing data insufficient to support approval
|
•
|
the FDA might not approve our or our third-party manufacturer’s processes or facilities; or
|
•
|
the FDA may change its approval policies or adopt new regulations.
|
•
|
our ability to set a price we believe is fair for our products;
|
•
|
our ability to generate revenue and achieve or maintain profitability; and
|
•
|
the availability of capital.
|
•
|
the federal Anti-Kickback Statute, which prohibits, among other things, any person or entity from knowingly and willfully offering, soliciting, receiving or providing remuneration, directly or indirectly, in exchange for or to induce either the referral of an individual for, or the purchase, order or recommendation of, any good or service for which payment may be made under federal healthcare programs, such as the Medicare and Medicaid programs;
|
•
|
the federal Physician Payments Sunshine Act or Open Payments Program provisions and the implementing regulations which will require, among other things, extensive tracking of physician and teaching hospital payments, maintenance of a payments database, and public reporting of the payment data;
|
•
|
the federal civil False Claims Act, which prohibits, among other things, individuals or entities from knowingly presenting, or causing to be presented, false claims, or knowingly using false statements, to obtain payment from the federal government;
|
•
|
federal criminal laws that prohibit executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters;
|
•
|
federal and state laws governing data privacy and the EU general data privacy regulation (“GDPR”);
|
•
|
the Foreign Corrupt Practices Act and similar statutes and regulations in foreign jurisdictions, which makes it unlawful for certain classes of persons and entities to make payments to foreign government officials to assist in obtaining or retaining business;
|
•
|
the federal Health Insurance Portability and Accountability Act of 1996, as amended by the Health Information Technology for Economic and Clinical Health Act, and their implementing regulations which governs the conduct of certain electronic healthcare transactions and protects the security and privacy of protected health information;
|
•
|
the Drug Quality and Security Act which requires manufacturers and other distribution parties to create systems to trace certain prescription drugs as they are distributed in the United States;
|
•
|
state law equivalents of each of the above federal laws, such as anti-kickback and false claims laws which may apply to items or services reimbursed by any third-party payor, including commercial insurers; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government; state and local laws that require pharmaceutical companies to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures; state laws that require the reporting of information related to drug pricing; and state and local laws requiring the registration of pharmaceutical sales and medical representatives; and
|
•
|
EU member states’ laws and the industry self-regulation codes of conduct governing promotion of medicinal products, interactions with physicians, misleading and comparative advertising and unfair commercial practices.
|
•
|
the timing and amount of revenues generated from sale of our products or product candidates;
|
•
|
our ability to meet the expectations of investors related to the commercialization of our products and product candidates;
|
•
|
regulatory actions or decisions, including the timing and outcome of any potential future FDA or EMA decision, or other products or product candidates, including those of our competitors;
|
•
|
inaccurate sales or cash forecasting of our products or product candidates;
|
•
|
changes in laws or regulations;
|
•
|
announcements by us or our competitors of significant acquisitions, strategic partnerships, joint ventures or capital commitments;
|
•
|
results of clinical trials or regulatory actions with respect to our products or product candidates;
|
•
|
market conditions in the pharmaceutical and biotechnology sectors;
|
•
|
changes in the structure of healthcare payment systems;
|
•
|
actual or anticipated changes in earnings estimates or changes in stock market analyst recommendations regarding our common stock, other comparable companies or our industry generally;
|
•
|
trading volume of our common stock;
|
•
|
sales of our common stock by us or our stockholders;
|
•
|
general economic, industry and market conditions; and
|
•
|
the other risks described in this “Risk factors” section.
|
•
|
our board of directors is divided into three classes with staggered three-year terms which may delay or prevent a change of our management or a change in control;
|
•
|
our board of directors has the right to elect directors to fill a vacancy created by the expansion of the board of directors or the resignation, death or removal of a director, which prevents stockholders from being able to fill vacancies on our board of directors;
|
•
|
our stockholders may not act by written consent or call special stockholders’ meetings; as a result, a holder, or holders, controlling a majority of our capital stock would not be able to take certain actions other than at annual stockholders’ meetings or special stockholders’ meetings called by the board of directors, the chairman of the board, the chief executive officer or the president;
|
•
|
our certificate of incorporation prohibits cumulative voting in the election of directors, which limits the ability of minority stockholders to elect director candidates;
|
•
|
stockholders must provide advance notice and additional disclosures in order to nominate individuals for election to the board of directors or to propose matters that can be acted upon at a stockholders’ meeting, which may discourage or deter a potential acquirer from conducting a solicitation of proxies to elect the acquirer’s own slate of directors or otherwise attempting to obtain control of our company; and
|
•
|
our board of directors may issue, without stockholder approval, shares of undesignated preferred stock; the ability to issue undesignated preferred stock makes it possible for our board of directors to issue preferred stock with voting or other rights or preferences that could impede the success of any attempt to acquire us.
|
ITEM 2.
|
UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS
|
ITEM 3.
|
DEFAULTS UPON SENIOR SECURITIES
|
ITEM 4.
|
MINE SAFETY DISCLOSURES
|
ITEM 5.
|
OTHER INFORMATION
|
|
|
|
|
Incorporation By Reference
|
||||||
Exhibit
Number
|
|
Exhibit Description
|
|
Form
|
|
SEC File No.
|
|
Exhibit
|
|
Filing Date
|
|
|
|
|
|
|
|
|
|
|
|
3.1
|
|
|
8-K
|
|
001-35935
|
|
3.1
|
|
5/28/2013
|
|
|
|
|
|
|
|
|
|
|
|
|
3.2
|
|
|
8-K
|
|
001-35935
|
|
3.1
|
|
6/11/2018
|
|
|
|
|
|
|
|
|
|
|
|
|
3.3
|
|
|
8-K
|
|
001-35935
|
|
3.2
|
|
5/28/2013
|
|
|
|
|
|
|
|
|
|
|
|
|
4.1
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
4.2
|
|
|
S-1
|
|
333-187901
|
|
4.1
|
|
5/17/2013
|
|
|
|
|
|
|
|
|
|
|
|
|
4.5
|
|
|
10-Q
|
|
001-35935
|
|
4.7
|
|
11/6/2013
|
|
|
|
|
|
|
|
|
|
|
|
|
4.6
|
|
|
10-Q
|
|
001-35935
|
|
4.8
|
|
11/6/2013
|
|
|
|
|
|
|
|
|
|
|
|
|
4.7
|
|
|
10-Q
|
|
001-35935
|
|
4.9
|
|
11/6/2013
|
|
|
|
|
|
|
|
|
|
|
|
|
10.52*
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
31.1*
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
31.2*
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
32.1*
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
101.INS*
|
|
XBRL Instance Document – the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document.
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
101.SCH*
|
|
XBRL Taxonomy Extension Schema Document
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
101.CAL*
|
|
XBRL Taxonomy Extension Calculation Linkbase Document
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Incorporation By Reference
|
||||||
Exhibit
Number
|
|
Exhibit Description
|
|
Form
|
|
SEC File No.
|
|
Exhibit
|
|
Filing Date
|
101.DEF*
|
|
XBRL Taxonomy Extension Definition Linkbase Document
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
101.LAB*
|
|
XBRL Taxonomy Extension Label Linkbase Document
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
101.PRE*
|
|
XBRL Taxonomy Extension Presentation Linkbase Document
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
104
|
|
The cover page from the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, formatted in Inline XBRL
|
|
|
|
|
|
|
|
|
*
|
Filed herewith
|
(1)
|
This certification accompanies the Form 10-Q to which it relates, is not deemed filed with the Securities and Exchange Commission and is not to be incorporated by reference into any filing of the Registrant under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended (whether made before or after the date of the Form 10-Q), irrespective of any general incorporation language contained in such filing.
|
|
PORTOLA PHARMACEUTICALS, INC.
|
||
|
|
|
|
|
|
|
|
Date: November 5, 2019
|
By:
|
|
/s/ Mardi C. Dier
|
|
|
|
Mardi C. Dier
|
|
|
|
Chief Financial and Business Officer
|
|
|
|
(Principal Financial Officer)
|
|
|
|
|
Date: November 5, 2019
|
By:
|
|
/s/ Scott Garland
|
|
|
|
Scott Garland
|
|
|
|
President and Chief Executive Officer
|
|
|
|
(Principal Executive Officer)
|
Date: November 5, 2019
|
|
|
|
|
|
/s/ Mardi C. Dier
|
|
|
|
Mardi C. Dier
|
|
|
|
Chief Financial Officer
|
|
|
|
(Principal Financial Officer)
|
|
Date: November 5, 2019
|
|
|
|
|
|
/s/ Scott Garland
|
|
|
|
Scott Garland
|
|
|
|
President and Chief Executive Officer
|
|
|
|
(Principal Executive Officer)
|
|
1.
|
The Company’s Quarterly Report on Form 10-Q for the period ended September 30, 2019, to which this Certification is attached as Exhibit 32.1 (the “Quarterly Report”) fully complies with the requirements of Section 13(a) or Section 15(d) of the Exchange Act, and
|
2.
|
The information contained in the Quarterly Report fairly presents, in all material respects, the financial condition and results of operations of the Company.
|
|
|
|
/s/ MARDI C. DIER
|
|
/s/ SCOTT GARLAND
|
Mardi C. Dier
|
|
Scott Garland
|
Chief Financial Officer
|
|
President and Chief Executive Officer
|
(Principal Financial Officer)
|
|
(Principal Executive Officer)
|