|
x
|
QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
|
¨
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TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
|
|
Delaware
|
|
001-36257
|
|
27-4842691
|
|
|
(State or other
jurisdiction of
incorporation or
organization)
|
|
(Commission File
No.)
|
|
(I.R.S. Employer
Identification No.)
|
|
Large accelerated filer
|
þ
|
Accelerated filer
|
¨
|
Non-accelerated filer
|
¨
|
Smaller reporting company
|
¨
|
|
|
|
Page No.
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|
||
|
||
|
||
|
||
|
||
|
||
|
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|
|
||
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June 30, 2016
|
|
December 31, 2015
|
||||
Assets
|
(unaudited)
|
|
|
|
|||
Current assets:
|
|
|
|
|
|
||
Cash and cash equivalents
|
$
|
21,155
|
|
|
$
|
37,805
|
|
Marketable securities
|
200,359
|
|
|
191,799
|
|
||
Accounts receivable, net
|
16,566
|
|
|
12,458
|
|
||
Inventory, net
|
2,713
|
|
|
2,536
|
|
||
Prepaid expenses and other current assets
|
3,365
|
|
|
2,378
|
|
||
Prepaid taxes
|
8,499
|
|
|
8,107
|
|
||
Note receivable, current
|
47,500
|
|
|
46,849
|
|
||
Total current assets
|
300,157
|
|
|
301,932
|
|
||
Property and equipment, net
|
381
|
|
|
428
|
|
||
Other asset
|
1,859
|
|
|
1,859
|
|
||
Intangible assets, net
|
184,432
|
|
|
161,536
|
|
||
Goodwill
|
936
|
|
|
936
|
|
||
Note receivable, long term
|
46,206
|
|
|
45,573
|
|
||
Total assets
|
$
|
533,971
|
|
|
$
|
512,264
|
|
|
|
|
|
||||
Liabilities and Stockholders' Equity
|
|
|
|
|
|
||
Current liabilities:
|
|
|
|
|
|
||
Accounts payable
|
$
|
6,285
|
|
|
$
|
7,639
|
|
Accrued expenses
|
23,159
|
|
|
23,820
|
|
||
Guaranteed minimum royalty
|
2,000
|
|
|
2,000
|
|
||
Other current liabilities
|
1,187
|
|
|
958
|
|
||
Business combination-related contingent consideration
|
14,077
|
|
|
13,754
|
|
||
Derivative financial instruments, warrants
|
33,360
|
|
|
38,810
|
|
||
Total current liabilities
|
80,068
|
|
|
86,981
|
|
||
Convertible debt
|
44,092
|
|
|
43,766
|
|
||
Other non-current liabilities
|
2,548
|
|
|
3,066
|
|
||
Guaranteed minimum royalty, less current portion
|
8,488
|
|
|
8,885
|
|
||
Business combination-related contingent consideration, less current portion
|
72,300
|
|
|
45,267
|
|
||
Deferred income tax liability, net
|
12,103
|
|
|
24,328
|
|
||
Total liabilities
|
219,599
|
|
|
212,293
|
|
||
Stockholders' Equity:
|
|
|
|
|
|
||
Preferred stock $0.001 par value; 20,000,000 shares authorized; 0 issued and outstanding as of June 30, 2016 and December 31, 2015
|
—
|
|
|
—
|
|
||
Common stock $0.0001 par value; 100,000,000 shares authorized; 36,725,130 and 36,465,853 issued and outstanding as of June 30, 2016 and December 31, 2015, respectively
|
4
|
|
|
4
|
|
||
Additional paid-in capital
|
381,687
|
|
|
365,802
|
|
||
Accumulated deficit
|
(67,340
|
)
|
|
(65,153
|
)
|
||
Accumulated other comprehensive income (loss)
|
21
|
|
|
(682
|
)
|
||
Total stockholders' equity
|
314,372
|
|
|
299,971
|
|
||
Total liabilities and stockholders' equity
|
$
|
533,971
|
|
|
$
|
512,264
|
|
|
Three Months Ended June 30,
|
|
Six Months Ended June 30,
|
||||||||||||
|
2016
|
|
2015
|
|
2016
|
|
2015
|
||||||||
Net product sales
|
$
|
33,311
|
|
|
$
|
24,068
|
|
|
$
|
62,319
|
|
|
$
|
41,440
|
|
|
|
|
|
|
|
|
|
||||||||
Operating expenses:
|
|
|
|
|
|
|
|
|
|
||||||
Cost of goods sold
|
1,021
|
|
|
637
|
|
|
1,778
|
|
|
912
|
|
||||
Research and development
|
17,675
|
|
|
10,563
|
|
|
32,347
|
|
|
20,910
|
|
||||
Selling, general and administrative
|
23,205
|
|
|
19,692
|
|
|
42,330
|
|
|
34,547
|
|
||||
Change in valuation of contingent consideration
|
2,789
|
|
|
120
|
|
|
5,485
|
|
|
120
|
|
||||
Total operating expenses
|
44,690
|
|
|
31,012
|
|
|
81,940
|
|
|
56,489
|
|
||||
|
|
|
|
|
|
|
|
||||||||
Operating loss
|
(11,379
|
)
|
|
(6,944
|
)
|
|
(19,621
|
)
|
|
(15,049
|
)
|
||||
|
|
|
|
|
|
|
|
||||||||
Other income (expenses), net:
|
|
|
|
|
|
|
|
|
|
||||||
Other income (expenses), net
|
(206
|
)
|
|
522
|
|
|
4
|
|
|
349
|
|
||||
Interest expense, net
|
(147
|
)
|
|
(2,922
|
)
|
|
(309
|
)
|
|
(6,720
|
)
|
||||
Finance expense
|
—
|
|
|
—
|
|
|
—
|
|
|
(600
|
)
|
||||
Change in fair value of derivative instruments
|
(9,063
|
)
|
|
(29,418
|
)
|
|
5,277
|
|
|
(66,171
|
)
|
||||
Loss on extinguishment of debt
|
—
|
|
|
(2,250
|
)
|
|
—
|
|
|
(2,250
|
)
|
||||
Litigation settlement gain
|
—
|
|
|
15,500
|
|
|
—
|
|
|
15,500
|
|
||||
Bargain purchase gain
|
—
|
|
|
—
|
|
|
—
|
|
|
49,063
|
|
||||
Total other income (loss), net
|
(9,416
|
)
|
|
(18,568
|
)
|
|
4,972
|
|
|
(10,829
|
)
|
||||
|
|
|
|
|
|
|
|
||||||||
Loss before provision for income taxes
|
(20,795
|
)
|
|
(25,512
|
)
|
|
(14,649
|
)
|
|
(25,878
|
)
|
||||
|
|
|
|
|
|
|
|
||||||||
Income tax benefit (expense)
|
7,392
|
|
|
(15
|
)
|
|
12,462
|
|
|
40,006
|
|
||||
|
|
|
|
|
|
|
|
||||||||
Net income (loss)
|
$
|
(13,403
|
)
|
|
$
|
(25,527
|
)
|
|
$
|
(2,187
|
)
|
|
$
|
14,128
|
|
|
|
|
|
|
|
|
|
||||||||
Net earnings (loss) per common share, basic
|
$
|
(0.37
|
)
|
|
$
|
(0.73
|
)
|
|
$
|
(0.06
|
)
|
|
$
|
0.45
|
|
Net earnings (loss) per common share, diluted
|
$
|
(0.37
|
)
|
|
$
|
(0.73
|
)
|
|
$
|
(0.20
|
)
|
|
$
|
0.44
|
|
Weighted average common shares outstanding, basic
|
36,683,429
|
|
|
34,957,134
|
|
|
36,601,807
|
|
|
31,079,053
|
|
||||
Weighted average common shares outstanding, diluted
|
36,683,429
|
|
|
34,957,134
|
|
|
38,063,285
|
|
|
34,827,405
|
|
||||
|
|
|
|
|
|
|
|
||||||||
Comprehensive income (loss):
|
|
|
|
|
|
|
|
|
|
||||||
Net income (loss)
|
$
|
(13,403
|
)
|
|
$
|
(25,527
|
)
|
|
$
|
(2,187
|
)
|
|
$
|
14,128
|
|
Foreign currency translation
|
71
|
|
|
(30
|
)
|
|
2
|
|
|
(7
|
)
|
||||
Unrealized loss on marketable securities
|
(157
|
)
|
|
(298
|
)
|
|
(706
|
)
|
|
(3,519
|
)
|
||||
Comprehensive income (loss)
|
$
|
(13,489
|
)
|
|
$
|
(25,855
|
)
|
|
$
|
(2,891
|
)
|
|
$
|
10,602
|
|
|
For the Six Months Ended June 30,
|
||||||
|
2016
|
|
2015
|
||||
Cash Flows From Operating Activities:
|
|
|
|
||||
Net income (loss)
|
$
|
(2,187
|
)
|
|
$
|
14,128
|
|
Adjustments to reconcile net income to net cash used in operating activities:
|
|
|
|
|
|
||
Depreciation and amortization
|
7,858
|
|
|
5,536
|
|
||
Realized gain on marketable securities
|
—
|
|
|
(107
|
)
|
||
Gain upon divestiture of assets to Turing Pharmaceuticals
|
—
|
|
|
(914
|
)
|
||
Deferred income tax benefit
|
(12,286
|
)
|
|
(40,021
|
)
|
||
Interest income from notes receivable
|
(1,285
|
)
|
|
—
|
|
||
Non-cash interest expense
|
1,179
|
|
|
1,154
|
|
||
Amortization of premiums on marketable securities
|
666
|
|
|
—
|
|
||
Amortization of debt discount and deferred financing costs
|
326
|
|
|
1,010
|
|
||
Lease liability
|
—
|
|
|
(125
|
)
|
||
Legal accrual reversal
|
(2,967
|
)
|
|
—
|
|
||
Bargain purchase gain
|
—
|
|
|
(49,063
|
)
|
||
Share based compensation
|
14,286
|
|
|
10,567
|
|
||
Derivative financial instruments, warrants, issued, recorded in interest expense
|
—
|
|
|
1,050
|
|
||
Change in estimated fair value of liability classified warrants
|
(5,277
|
)
|
|
66,171
|
|
||
Change in estimated fair value of contingent consideration
|
5,485
|
|
|
113
|
|
||
Payments related to change in fair value of contingent consideration
|
(550
|
)
|
|
(90
|
)
|
||
Changes in operating assets and liabilities, net of business acquisitions:
|
|
|
|
|
|
||
Accounts receivable
|
(4,104
|
)
|
|
(3,508
|
)
|
||
Inventory
|
(174
|
)
|
|
(640
|
)
|
||
Prepaid expenses and other current assets
|
(1,197
|
)
|
|
(420
|
)
|
||
Accounts payable and accrued expenses
|
650
|
|
|
(3,417
|
)
|
||
Net cash provided by operating activities
|
423
|
|
|
1,424
|
|
||
|
|
|
|
||||
Cash Flows From Investing Activities:
|
|
|
|
|
|
||
Purchase of fixed assets
|
(24
|
)
|
|
(29
|
)
|
||
Cash paid for intangible assets
|
(4,881
|
)
|
|
(2,497
|
)
|
||
Proceeds from the sale/maturity of marketable securities
|
59,874
|
|
|
282
|
|
||
Purchase of marketable securities
|
(68,517
|
)
|
|
—
|
|
||
Cash received upon divestiture of asset
|
—
|
|
|
3,311
|
|
||
Cash paid upon acquisition, net of cash acquired
|
(500
|
)
|
|
(33,430
|
)
|
||
Net cash used in investing activities
|
(14,048
|
)
|
|
(32,363
|
)
|
||
|
|
|
|
||||
Cash Flows From Financing Activities:
|
|
|
|
|
|
||
Payment of acquisition-related contingent consideration-Manchester
|
(2,125
|
)
|
|
(1,228
|
)
|
||
Payment of acquisition-related contingent consideration-Asklepion
|
(644
|
)
|
|
—
|
|
||
Payment of guaranteed minimum royalty
|
(1,000
|
)
|
|
(1,000
|
)
|
||
Proceeds from exercise of warrants
|
90
|
|
|
586
|
|
||
Payment of other liability
|
(500
|
)
|
|
(500
|
)
|
||
Proceeds from exercise of stock options
|
1,572
|
|
|
2,669
|
|
||
Excess benefit related to stock compensation
|
(236
|
)
|
|
—
|
|
||
Proceeds received from issuance of common stock
|
—
|
|
|
149,454
|
|
||
Financing costs from issuance of common stock
|
—
|
|
|
(9,500
|
)
|
||
Net cash used in (provided by) financing activities
|
(2,843
|
)
|
|
140,481
|
|
||
|
|
|
|
||||
Effect of exchange rate changes on cash
|
(182
|
)
|
|
—
|
|
||
Net change in cash
|
(16,650
|
)
|
|
109,542
|
|
||
Cash, beginning of year
|
37,805
|
|
|
18,204
|
|
||
|
|
|
|
||||
Cash, end of period
|
$
|
21,155
|
|
|
$
|
127,746
|
|
•
|
Chenodal
®
(chenodeoxycholic acid) is approved in the United States for the treatment of patients suffering from gallstones in whom surgery poses an unacceptable health risk due to disease or advanced age. Chenodal has also been the standard of care for cerebrotendinous xanthomatosis (“CTX”) patients for more than three decades and the Company is currently pursuing adding this indication to the label.
|
•
|
Cholbam
®
(cholic acid) is approved in the United States for the treatment of bile acid synthesis disorders due to single enzyme defects and is further indicated for adjunctive treatment of patients with peroxisomal disorders.
|
•
|
Thiola
®
(tiopronin) is approved in the United States for the prevention of cysteine (kidney) stone formation in patients with cystinuria.
|
Cash paid upon consummation
|
$
|
500
|
|
Present value of contingent consideration
|
25,000
|
|
|
Total purchase price
|
$
|
25,500
|
|
|
|
||
Fair Value of Assets Acquired and Liabilities Assumed
|
|
||
Acquired product rights: L-UDCA (intangible asset)
|
$
|
25,500
|
|
Total purchase price
|
$
|
25,500
|
|
Cash paid upon consummation
|
$
|
33,430
|
|
Present value of contingent consideration and service fees
|
42,010
|
|
|
Fair Value of 661,279 shares issued to Asklepion
|
15,844
|
|
|
Total Purchase Price
|
$
|
91,284
|
|
|
|
||
Fair Value of Assets Acquired and Liabilities Assumed
|
|
||
Acquired product rights: Cholbam (Intangible Asset)
|
$
|
83,200
|
|
Pediatric Priority Review Voucher
|
96,250
|
|
|
Inventory
|
777
|
|
|
Deferred tax liability
|
(39,880
|
)
|
|
Total Allocation of Purchase Price
|
$
|
140,347
|
|
Bargain Purchase Gain
|
(49,063
|
)
|
|
Total Purchase Price
|
$
|
91,284
|
|
|
June 30, 2016
|
|
December 31, 2015
|
||||
Marketable other than equity securities:
|
|
|
|
||||
Commercial paper
|
$
|
40,823
|
|
|
$
|
31,864
|
|
Corporate debt securities
|
130,023
|
|
|
125,547
|
|
||
Securities of government sponsored entities
|
29,513
|
|
|
34,388
|
|
||
Total Marketable Securities:
|
$
|
200,359
|
|
|
$
|
191,799
|
|
|
Remaining Contractual Maturity
(in years) |
|
Amortized Cost
|
|
Unrealized Gains
|
|
Unrealized Losses
|
|
Aggregate Estimated Fair Value
|
||||||||
Marketable other than equity securities:
|
|
|
|
|
|
|
|
|
|
||||||||
Commercial paper
|
Less than 1
|
|
$
|
40,841
|
|
|
$
|
2
|
|
|
$
|
(20
|
)
|
|
$
|
40,823
|
|
Corporate debt securities
|
Less than 1
|
|
92,226
|
|
|
26
|
|
|
(25
|
)
|
|
92,227
|
|
||||
Securities of government-sponsored entities
|
Less than 1
|
|
5,000
|
|
|
—
|
|
|
—
|
|
|
5,000
|
|
||||
Total maturity less than 1 year
|
|
|
138,067
|
|
|
28
|
|
|
(45
|
)
|
|
138,050
|
|
||||
Corporate debt securities
|
1 to 2
|
|
37,718
|
|
|
103
|
|
|
(25
|
)
|
|
37,796
|
|
||||
Securities of government-sponsored entities
|
1 to 2
|
|
24,511
|
|
|
3
|
|
|
(1
|
)
|
|
24,513
|
|
||||
Total maturity 1 to 2 years
|
|
|
62,229
|
|
|
106
|
|
|
(26
|
)
|
|
62,309
|
|
||||
Total available-for-sale securities
|
|
|
$
|
200,296
|
|
|
$
|
134
|
|
|
$
|
(71
|
)
|
|
$
|
200,359
|
|
|
Remaining Contractual Maturity
(in years)
|
|
Amortized Cost
|
|
Unrealized Gains
|
|
Unrealized Losses
|
|
Aggregate Estimated Fair Value
|
||||||||
Marketable Other than Equity Securities:
|
|
|
|
|
|
|
|
|
|
||||||||
Commercial paper
|
Less than 1
|
|
$
|
31,899
|
|
|
$
|
6
|
|
|
$
|
(41
|
)
|
|
$
|
31,864
|
|
Corporate debt securities
|
Less than 1
|
|
69,859
|
|
|
—
|
|
|
(164
|
)
|
|
69,695
|
|
||||
Total maturity less than 1 year
|
|
|
101,758
|
|
|
6
|
|
|
(205
|
)
|
|
101,559
|
|
||||
Corporate debt securities
|
1 to 2
|
|
56,162
|
|
|
—
|
|
|
(310
|
)
|
|
55,852
|
|
||||
Securities of government-sponsored entities
|
1 to 2
|
|
34,522
|
|
|
2
|
|
|
(136
|
)
|
|
34,388
|
|
||||
Total maturity 1 to 2 years
|
|
|
90,684
|
|
|
2
|
|
|
(446
|
)
|
|
90,240
|
|
||||
Total available-for-sale securities
|
|
|
$
|
192,442
|
|
|
$
|
8
|
|
|
$
|
(651
|
)
|
|
$
|
191,799
|
|
|
June 30, 2016
|
|
December 31, 2015
|
||||
Fair value of common stock
|
$
|
17.81
|
|
|
$
|
19.29
|
|
Remaining life of the warrants (in years)
|
1.6 - 3.5 years
|
|
|
2.1 - 4.0 years
|
|
||
Risk-free interest rate
|
.53-.79%
|
|
|
1.11-1.59%
|
|
||
Expected volatility
|
65-85%
|
|
|
75-85%
|
|
||
Dividend yield
|
—
|
%
|
|
—
|
%
|
|
As of June 30, 2016
|
||||||||||||||
|
Total carrying and estimated fair value
|
|
Quoted prices in active markets
(Level 1) |
|
Significant other observable inputs (Level 2)
|
|
Significant unobservable inputs (Level 3)
|
||||||||
Asset:
|
|
|
|
|
|
|
|
||||||||
Marketable securities, available-for-sale
|
$
|
200,359
|
|
|
$
|
—
|
|
|
$
|
200,359
|
|
|
$
|
—
|
|
|
|
|
|
|
|
|
|
||||||||
Liabilities:
|
|
|
|
|
|
|
|
||||||||
Derivative liability related to warrants
|
$
|
33,360
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
33,360
|
|
Business combination-related contingent consideration
|
$
|
86,377
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
86,377
|
|
|
As of December 31, 2015
|
||||||||||||||
|
Total carrying and estimated fair value
|
|
Quoted prices in active markets
(Level 1)
|
|
Significant other observable inputs (Level 2)
|
|
Significant unobservable inputs (Level 3)
|
||||||||
Asset:
|
|
|
|
|
|
|
|
||||||||
Marketable securities, available-for-sale
|
$
|
191,799
|
|
|
$
|
—
|
|
|
$
|
191,799
|
|
|
$
|
—
|
|
|
|
|
|
|
|
|
|
||||||||
Liabilities:
|
|
|
|
|
|
|
|
||||||||
Derivative liability related to warrants
|
$
|
38,810
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
38,810
|
|
Business combination-related contingent consideration
|
$
|
59,021
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
59,021
|
|
|
Fair Value Measurements of Common Stock Warrants Using Significant Unobservable Inputs (Level 3)
|
||
Balance at January 1, 2016
|
$
|
38,810
|
|
Reclassification of derivative liability to equity upon exercise of warrants
|
(173
|
)
|
|
Change in estimated fair value of liability classified warrants
|
(5,277
|
)
|
|
Balance at June 30, 2016
|
$
|
33,360
|
|
|
Fair Value Measurements
of Acquisition-Related Contingent Consideration (Level 3) |
||
Balance at January 1, 2016
|
$
|
59,021
|
|
Increase from revaluation of contingent consideration
|
5,485
|
|
|
Acquisition of L-UDCA
|
25,000
|
|
|
Contractual payments
|
(1,510
|
)
|
|
Contractual payments accrued at June 30, 2016
|
(1,677
|
)
|
|
Foreign currency impact
|
58
|
|
|
Balance at June 30, 2016
|
$
|
86,377
|
|
|
June 30, 2016
|
|
December 31, 2015
|
||||
Finite-lived intangible assets
|
$
|
209,787
|
|
|
$
|
179,096
|
|
Less: accumulated amortization
|
(25,355
|
)
|
|
(17,560
|
)
|
||
Net carrying value
|
$
|
184,432
|
|
|
$
|
161,536
|
|
|
Three Months Ended June 30,
|
|
Six Months Ended June 30,
|
||||||||||||
|
2016
|
|
2015
|
|
2016
|
|
2015
|
||||||||
Research and development
|
$
|
81
|
|
|
$
|
193
|
|
|
$
|
163
|
|
|
$
|
414
|
|
Selling, general and administrative
|
3,829
|
|
|
3,614
|
|
|
7,632
|
|
|
5,128
|
|
||||
Total amortization expense
|
$
|
3,910
|
|
|
$
|
3,807
|
|
|
$
|
7,795
|
|
|
$
|
5,542
|
|
|
June 30, 2016
|
|
December 31, 2015
|
||||
Government rebates payable
|
$
|
3,826
|
|
|
$
|
3,158
|
|
Compensation related costs
|
4,765
|
|
|
7,143
|
|
||
Accrued royalties and contingent consideration
|
5,069
|
|
|
4,688
|
|
||
Research and development
|
5,725
|
|
|
4,281
|
|
||
Selling, general and administrative
|
1,619
|
|
|
2,704
|
|
||
Miscellaneous accrued
|
2,155
|
|
|
1,846
|
|
||
|
$
|
23,159
|
|
|
$
|
23,820
|
|
|
Three Months Ended
|
||||||||||||||||||||
|
June 30, 2016
|
|
June 30, 2015
|
||||||||||||||||||
|
Shares
|
|
Net Income
|
|
EPS
|
|
Shares
|
|
Net Income
|
|
EPS
|
||||||||||
Basic Earnings per Share
|
36,683,429
|
|
|
$
|
(13,403
|
)
|
|
$
|
(0.37
|
)
|
|
34,957,134
|
|
|
$
|
(25,527
|
)
|
|
$
|
(0.73
|
)
|
Dilutive earnings per share
|
36,683,429
|
|
|
$
|
(13,403
|
)
|
|
$
|
(0.37
|
)
|
|
34,957,134
|
|
|
$
|
(25,527
|
)
|
|
$
|
(0.73
|
)
|
|
Six Months Ended
|
||||||||||||||||||||
|
June 30, 2016
|
|
June 30, 2015
|
||||||||||||||||||
|
Shares
|
|
Net Income
|
|
EPS
|
|
Shares
|
|
Net Income
|
|
EPS
|
||||||||||
Basic net earnings (loss) per share
|
36,601,807
|
|
|
$
|
(2,187
|
)
|
|
$
|
(0.06
|
)
|
|
31,079,053
|
|
|
$
|
14,128
|
|
|
$
|
0.45
|
|
Dilutive shares related to derivative warrants
|
1,461,478
|
|
|
—
|
|
|
|
|
|
—
|
|
|
—
|
|
|
|
|
||||
Change in fair value of derivative instruments
|
—
|
|
|
(5,277
|
)
|
|
|
|
|
—
|
|
|
—
|
|
|
|
|
||||
Convertible debt
|
—
|
|
|
—
|
|
|
|
|
|
2,642,160
|
|
|
1,035
|
|
|
|
|
||||
Restricted stock
|
—
|
|
|
—
|
|
|
|
|
|
292,027
|
|
|
—
|
|
|
|
|
||||
Stock options
|
—
|
|
|
—
|
|
|
|
|
|
814,165
|
|
|
—
|
|
|
|
|||||
Dilutive net earnings (loss) per share
|
38,063,285
|
|
|
$
|
(7,464
|
)
|
|
$
|
(0.20
|
)
|
|
34,827,405
|
|
|
$
|
15,163
|
|
|
$
|
0.44
|
|
|
Three Months Ended
|
|
Six Months Ended
|
||||||||
|
June 30, 2016
|
|
June 30, 2015
|
|
June 30, 2016
|
|
June 30, 2015
|
||||
Restricted stock units
|
0.3
|
|
|
—
|
|
|
0.3
|
|
|
—
|
|
Convertible debt
|
2.6
|
|
|
2.6
|
|
|
2.6
|
|
|
—
|
|
Options
|
6.1
|
|
|
2.6
|
|
|
5.9
|
|
|
1.2
|
|
Warrants
|
2.7
|
|
|
—
|
|
|
—
|
|
|
3.3
|
|
Total anti-dilutive shares
|
11.7
|
|
|
5.2
|
|
|
8.8
|
|
|
4.5
|
|
|
Total
|
|
Less than 1 year
|
|
1-3 years
|
|
3-5 years
|
|
More than 5 years
|
||||||||||
Operating leases
|
$
|
2,140
|
|
|
$
|
1,054
|
|
|
$
|
1,086
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Note payable, including contractual interest
|
52,037
|
|
|
2,070
|
|
|
49,967
|
|
|
—
|
|
|
—
|
|
|||||
Sales support services
|
3,262
|
|
|
416
|
|
|
833
|
|
|
2,013
|
|
|
—
|
|
|||||
Product supply contracts
|
1,515
|
|
|
1,265
|
|
|
250
|
|
|
—
|
|
|
—
|
|
|||||
Purchase order commitments
|
444
|
|
|
219
|
|
|
225
|
|
|
—
|
|
|
—
|
|
|||||
|
$
|
59,398
|
|
|
$
|
5,024
|
|
|
$
|
52,361
|
|
|
$
|
2,013
|
|
|
$
|
—
|
|
|
Number of
Restricted Stock Units
|
|
Weighted
Average
Grant Date Fair
Value
|
|||
Outstanding December 31, 2015
|
429,666
|
|
|
$
|
20.38
|
|
Granted
|
130,000
|
|
|
16.01
|
|
|
Vested
|
(77,666
|
)
|
|
16.63
|
|
|
Forfeited/canceled
|
(34,335
|
)
|
|
12.07
|
|
|
Outstanding June 30, 2016
|
447,665
|
|
|
$
|
20.40
|
|
|
Shares
Underlying
Options
|
|
Weighted
Average
Exercise
Price
|
|
Weighted Average Remaining Contractual Life (years)
|
|
Aggregate Intrinsic Value (in thousands)
|
|||||
Outstanding at December 31, 2015
|
5,665,584
|
|
|
$
|
17.05
|
|
|
8.75
|
|
$
|
31,542
|
|
Granted
|
1,381,750
|
|
|
16.09
|
|
|
|
|
|
|||
Exercised
|
(166,611
|
)
|
|
9.43
|
|
|
|
|
|
|||
Forfeited/canceled
|
(157,741
|
)
|
|
20.58
|
|
|
|
|
|
|||
Outstanding at June 30, 2016
|
6,722,982
|
|
|
$
|
16.96
|
|
|
8.21
|
|
$
|
26,854
|
|
|
Three Months Ended June 30,
|
|
Six Months Ended June 30,
|
||||||||||||
|
2016
|
|
2015
|
|
2016
|
|
2015
|
||||||||
Research and development
|
$
|
2,641
|
|
|
$
|
1,749
|
|
|
$
|
5,127
|
|
|
$
|
3,968
|
|
Selling, general & administrative
|
4,852
|
|
|
3,245
|
|
|
9,159
|
|
|
6,599
|
|
||||
Total
|
$
|
7,493
|
|
|
$
|
4,994
|
|
|
$
|
14,286
|
|
|
$
|
10,567
|
|
|
June 30, 2016
|
|
December 31, 2015
|
||||
Raw materials
|
$
|
232
|
|
|
$
|
289
|
|
Finished goods
|
2,481
|
|
|
2,247
|
|
||
Total inventory
|
$
|
2,713
|
|
|
$
|
2,536
|
|
•
|
Chenodal
®
(chenodeoxycholic acid)
is approved in the United States for the treatment of patients suffering from gallstones in whom surgery poses an unacceptable health risk due to disease or advanced age. Chenodal has also been the standard of care for cerebrotendinous xanthomatosis (“CTX”) patients for more than three decades and we are currently pursuing adding this indication to the label.
|
•
|
Cholbam
®
(cholic acid)
is approved in the United States for the treatment of bile acid synthesis disorders due to single enzyme defects and is further indicated for adjunctive treatment of patients with peroxisomal disorders.
|
•
|
Thiola
®
(tiopronin)
is approved in the United States for the prevention of cysteine (kidney) stone formation in patients with severe homozygous cystinuria.
|
•
|
sterol 27-hydroxylase (presenting as cerebrotendinous xanthomatosis, CTX) deficiency;
|
•
|
2- (or alpha-) methylacyl-CoA racemase (AMACR) deficiency; and
|
•
|
cholesterol 7 alpha-hydroxylase (CYP7A1) deficiency.
|
|
Three Months Ended June 30,
|
|
Six Months Ended June 30,
|
||||||||||||||||||||
|
2016
|
|
2015
|
|
Increase
|
|
2016
|
|
2015
|
|
Increase
|
||||||||||||
Net product sales
|
$
|
33,311
|
|
|
$
|
24,068
|
|
|
$
|
9,243
|
|
|
$
|
62,319
|
|
|
$
|
41,440
|
|
|
$
|
20,879
|
|
|
Three Months Ended June 30,
|
|
Six Months Ended June 30,
|
||||||||||||||||||||
|
2016
|
|
2015
|
|
Increase
|
|
2016
|
|
2015
|
|
Increase
|
||||||||||||
Cost of goods sold
|
$
|
1,021
|
|
|
$
|
637
|
|
|
$
|
384
|
|
|
$
|
1,778
|
|
|
$
|
912
|
|
|
$
|
866
|
|
Research and development
|
17,675
|
|
|
10,563
|
|
|
7,112
|
|
|
32,347
|
|
|
20,910
|
|
|
11,437
|
|
||||||
Selling, general and administrative
|
23,205
|
|
|
19,692
|
|
|
3,513
|
|
|
42,330
|
|
|
34,547
|
|
|
7,783
|
|
||||||
Change in valuation of contingent consideration
|
2,789
|
|
|
120
|
|
|
2,669
|
|
|
5,485
|
|
|
120
|
|
|
5,365
|
|
||||||
|
$
|
44,690
|
|
|
$
|
31,012
|
|
|
$
|
13,678
|
|
|
$
|
81,940
|
|
|
$
|
56,489
|
|
|
$
|
25,451
|
|
|
Three Months Ended June 30,
|
|
Six Months Ended June 30,
|
||||||||||||||||||||
|
2016
|
|
2015
|
|
Increase (decrease)
|
|
2016
|
|
2015
|
|
Increase (decrease)
|
||||||||||||
Other income, net
|
$
|
(206
|
)
|
|
$
|
522
|
|
|
$
|
(728
|
)
|
|
$
|
4
|
|
|
$
|
349
|
|
|
$
|
(345
|
)
|
Interest expense, net
|
(147
|
)
|
|
(2,922
|
)
|
|
2,775
|
|
|
(309
|
)
|
|
(6,720
|
)
|
|
6,411
|
|
||||||
Finance expense
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(600
|
)
|
|
600
|
|
||||||
Change in fair value of derivative instruments
|
(9,063
|
)
|
|
(29,418
|
)
|
|
20,355
|
|
|
5,277
|
|
|
(66,171
|
)
|
|
71,448
|
|
||||||
Loss on extinguishment of debt
|
—
|
|
|
(2,250
|
)
|
|
2,250
|
|
|
—
|
|
|
(2,250
|
)
|
|
2,250
|
|
||||||
Litigation settlement gain
|
—
|
|
|
15,500
|
|
|
(15,500
|
)
|
|
—
|
|
|
15,500
|
|
|
(15,500
|
)
|
||||||
Bargain purchase gain
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
49,063
|
|
|
(49,063
|
)
|
||||||
|
$
|
(9,416
|
)
|
|
$
|
(18,568
|
)
|
|
$
|
9,152
|
|
|
$
|
4,972
|
|
|
$
|
(10,829
|
)
|
|
$
|
15,801
|
|
|
June 30, 2016
|
|
December 31, 2015
|
||||
Cash & Cash Equivalents
|
$
|
21,155
|
|
|
$
|
37,805
|
|
Marketable securities
|
200,359
|
|
|
191,799
|
|
||
Accumulated Deficit
|
(67,340
|
)
|
|
(65,153
|
)
|
||
Stockholders' Equity
|
314,372
|
|
|
299,971
|
|
||
|
|
|
|
||||
Net Working Capital
|
$
|
220,089
|
|
|
$
|
214,951
|
|
Net Working Capital Ratio
|
3.75
|
|
|
3.47
|
|
•
|
revenue growth of our marketed products;
|
•
|
the rate of progress and cost of our clinical trials, preclinical studies and other discovery and research and development activities;
|
•
|
the timing of, and costs involved in, seeking and obtaining marketing approvals for our products, and in maintaining quality systems standards for our products;
|
•
|
our ability to manufacture sufficient quantities of our products to meet expected demand;
|
•
|
the costs of preparing, filing, prosecuting, maintaining and enforcing any patent claims and other intellectual property rights, litigation costs and the results of litigation;
|
•
|
our ability to enter into collaboration, licensing or distribution arrangements and the terms and timing of these arrangements;
|
•
|
the potential need to expand our business, resulting in additional payroll and other overhead expenses;
|
•
|
the potential acquisition or in-licensing of other products or technologies; and
|
•
|
the emergence of competing technologies or other adverse market or technological developments.
|
•
|
lower demonstrated efficacy, safety and/or tolerability compared to other drugs;
|
•
|
prevalence and severity of adverse side-effects;
|
•
|
lack of cost-effectiveness;
|
•
|
lack of coverage and adequate reimbursement availability from third-party payers;
|
•
|
a decision to wait for the approval of other therapies in development that have significant perceived advantages over our drug;
|
•
|
convenience and ease of administration;
|
•
|
other potential advantages of alternative treatment methods; and
|
•
|
ineffective marketing and/or distribution support.
|
•
|
our preclinical tests or clinical trials may produce negative or inconclusive results, and we may decide, or regulators may require us, to conduct additional preclinical testing or clinical trials or we may abandon projects that we expect to be promising;
|
•
|
regulators may require us to conduct studies of the long-term effects associated with the use of our product candidates;
|
•
|
regulators or institutional review boards may not authorize us to commence a clinical trial or conduct a clinical trial at a prospective trial site;
|
•
|
the FDA or any non-United States regulatory authority may impose conditions on us regarding the scope or design of our clinical trials or may require us to resubmit our clinical trial protocols to institutional review boards for re-inspection due to changes in the regulatory environment;
|
•
|
the number of patients required for our clinical trials may be larger than we anticipate or participants may drop out of our clinical trials at a higher rate than we anticipate;
|
•
|
our third-party contractors or clinical investigators may fail to comply with regulatory requirements or fail to meet their contractual obligations to us in a timely manner;
|
•
|
we might have to suspend or terminate one or more of our clinical trials if we, regulators or institutional review boards determine that the participants are being exposed to unacceptable health risks;
|
•
|
regulators or institutional review boards may require that we hold, suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements;
|
•
|
the cost of our clinical trials may be greater than we anticipate;
|
•
|
the supply or quality of our product candidates or other materials necessary to conduct our clinical trials may be insufficient or inadequate or we may not be able to reach agreements on acceptable terms with prospective clinical research organizations; and
|
•
|
the effects of our product candidates may not be the desired effects or may include undesirable side effects or the product candidates may have other unexpected characteristics.
|
•
|
be delayed in obtaining, or may not be able to obtain, marketing approval for one or more of our product candidates;
|
•
|
obtain approval for indications that are not as broad as intended or entirely different than those indications for which we sought approval; and
|
•
|
have the product removed from the market after obtaining marketing approval.
|
•
|
obtaining supplies of sparsentan, RE-024 and subsequent product candidates for completion of our clinical trials on a timely basis;
|
•
|
successful completion of pre-clinical and clinical studies;
|
•
|
obtaining marketing approvals from the FDA and similar regulatory authorities outside the United States;
|
•
|
establishing commercial-scale manufacturing arrangements with third-party manufacturers whose manufacturing facilities are operated in compliance with cGMP regulations;
|
•
|
launching commercial sales of the product, whether alone or in collaboration with others;
|
•
|
acceptance of the product by patients, the medical community and third-party payers;
|
•
|
reimbursement from medical, medicaid or private payers;
|
•
|
competition from other companies;
|
•
|
successful protection of our intellectual property rights from competing products in the United States and abroad; and
|
•
|
a continued acceptable safety and efficacy profile of our product candidates following approval.
|
•
|
regulatory authorities may require the addition of restrictive labeling statements;
|
•
|
regulatory authorities may withdraw their approval of the product; and
|
•
|
we may be required to change the way the product is administered or conduct additional clinical trials.
|
•
|
the prevalence and severity of any side effects, including any limitations or warnings contained in a product’s approved labeling;
|
•
|
the efficacy and potential advantages over alternative treatments;
|
•
|
the pricing of our product candidates;
|
•
|
relative convenience and ease of administration;
|
•
|
the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies;
|
•
|
the strength of marketing and distribution support and timing of market introduction of competitive products;
|
•
|
publicity concerning our products or competing products and treatments; and
|
•
|
sufficient third-party insurance coverage or reimbursement.
|
•
|
we or our licensors were the first to make the inventions covered by each of our pending patent applications;
|
•
|
we or our licensors were the first to file patent applications for these inventions;
|
•
|
others will not independently develop similar or alternative technologies or duplicate any of our technologies;
|
•
|
any patents issued to us or our licensors that provide a basis for commercially viable products will provide us with any competitive advantages or will not be challenged by third parties;
|
•
|
we will develop additional proprietary technologies that are patentable;
|
•
|
we will file patent applications for new proprietary technologies promptly or at all;
|
•
|
the claims we make in our patents will be upheld by patent offices in the United States and elsewhere;
|
•
|
our patents will not expire prior to or shortly after commencing commercialization of a product; and
|
•
|
the patents of others will not have a negative effect on our ability to do business.
|
•
|
a covered benefit under its health plan;
|
•
|
safe, effective and medically necessary;
|
•
|
appropriate for the specific patient;
|
•
|
cost-effective; and
|
•
|
neither experimental nor investigational.
|
•
|
reliance on the third party for regulatory compliance and quality assurance;
|
•
|
limitations on supply availability resulting from capacity and scheduling constraints of the third parties;
|
•
|
impact on our reputation in the marketplace if manufacturers of our products fail to meet the demands of our customers;
|
•
|
the possible breach of the manufacturing agreement by the third party because of factors beyond our control; and
|
•
|
the possible termination or nonrenewal of the agreement by the third party, based on its own business priorities, at a time that is costly or inconvenient for us.
|
•
|
our inability to recruit and retain adequate numbers of effective sales and marketing personnel;
|
•
|
the inability of sales personnel to obtain access to or persuade adequate numbers of physicians to prescribe our products;
|
•
|
the lack of complementary products to be offered by our sales personnel, which may put us at a competitive disadvantage against companies with broader product lines;
|
•
|
unforeseen costs associated with expanding our own sales and marketing team for new products or with entering into a partnering agreement with an independent sales and marketing organization; and
|
•
|
efforts by our competitors to commercialize competitive products.
|
•
|
complete the Phase 2 DUET trial of sparsentan for the treatment of FSGS;
|
•
|
continue our ongoing clinical development of RE-024 for the treatment of PKAN;
|
•
|
complete requirements for filing of L-UDCA;
|
•
|
continue the research and development of additional product candidates;
|
•
|
expand our sales and marketing infrastructure to commercialize Cholbam and any new products for which we may obtain regulatory approval; and
|
•
|
expand operational, financial, and management information systems and personnel, including personnel to support product development efforts and our obligations as a public company.
|
•
|
the progress and results of our pre-clinical and clinical studies of sparsentan, RE-024 and L-UDCA and other drug candidates;
|
•
|
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;
|
•
|
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; and
|
•
|
our ability to establish collaborations and obtain milestone, royalty or other payments from any such collaborators.
|
•
|
results of clinical trials of our product candidates or those of our competitors;
|
•
|
our entry into or the loss of a significant collaboration;
|
•
|
regulatory or legal developments in the United States and other countries, including changes in the health care payment systems;
|
•
|
our ability to obtain and maintain marketing approvals from the FDA or similar regulatory authorities outside the United States;
|
•
|
variations in our financial results or those of companies that are perceived to be similar to us;
|
•
|
changes in the structure of healthcare payment systems;
|
•
|
market conditions in the pharmaceutical and biotechnology sectors and issuance of new or changed securities analysts’ reports or recommendations;
|
•
|
general economic, industry and market conditions;
|
•
|
results of clinical trials conducted by others on drugs that would compete with our product candidates;
|
•
|
developments or disputes concerning patents or other proprietary rights;
|
•
|
public concern over our product candidates or any products approved in the future;
|
•
|
litigation;
|
•
|
future sales or anticipated sales of our common stock by us or our stockholders; and
|
•
|
the other factors described in this “Risk Factors” section.
|
•
|
integrating personnel, operations and systems, while maintaining focus on producing and delivering consistent, high quality products;
|
•
|
coordinating geographically dispersed organizations;
|
•
|
distracting employees from operations;
|
•
|
retaining existing customers and attracting new customers; and
|
•
|
managing inefficiencies associated with integrating the operations of the Company.
|
•
|
inability of sales personnel to obtain access to or convince adequate numbers of physicians to prescribe our products;
|
•
|
inability to recruit, retain and effectively manage adequate numbers of effective sales personnel;
|
•
|
lack of complementary products to be offered by sales personnel, which may put us at a competitive disadvantage relative to companies that have more extensive product lines; and
|
•
|
unforeseen delays, costs and expenses associated with maintaining our sales organization.
|
•
|
decreased demand for any product candidates or products that we may develop;
|
•
|
damage to our reputation;
|
•
|
regulatory investigations that could require costly recalls or product modifications;
|
•
|
withdrawal of clinical trial participants;
|
•
|
costs to defend the related litigation;
|
•
|
substantial monetary awards to trial participants or patients, including awards that substantially exceed our product liability insurance, which we would then be required to pay from other sources, if available, and would damage our ability to obtain liability insurance at reasonable costs, or at all, in the future;
|
•
|
loss of revenue;
|
•
|
the diversion of management’s attention from managing our business; and
|
•
|
the inability to commercialize any products that we may develop.
|
•
|
our failure to demonstrate to the satisfaction of the FDA or comparable regulatory authorities that a product candidate is safe and effective for a particular indication;
|
•
|
the results of clinical trials may not meet the level of statistical significance required by the FDA or comparable regulatory authorities for approval;
|
•
|
our inability to demonstrate that a product candidate’s benefits outweigh its risks;
|
•
|
our inability to demonstrate that the product candidate presents an advantage over existing therapies;
|
•
|
the FDA’s or comparable regulatory authorities’ disagreement with the manner in which we interpret the data from preclinical studies or clinical trials;
|
•
|
failure of the third-party manufacturers with which we contract for clinical or commercial supplies to satisfactorily complete an FDA pre-approval inspection of the facility or facilities at which the product is manufactured to assess compliance with the FDA’s cGMP regulations to assure that the facilities, methods and controls are adequate to preserve the drug’s identity, strength, quality and purity; and
|
•
|
a change in the approval policies or regulations of the FDA or comparable regulatory authorities or a change in the laws governing the approval process.
|
•
|
make it more difficult for us to satisfy our obligations with respect to any other debt we may incur in the future;
|
•
|
increase our vulnerability to general adverse economic and industry conditions;
|
•
|
require us to dedicate a substantial portion of our cash flow from operations to payments on our indebtedness and related interest, thereby reducing the availability of our cash flow to fund working capital, capital expenditures and other general corporate purposes;
|
•
|
limit our flexibility in planning for, or reacting to, changes in our business and the industry in which we operate;
|
•
|
increase our cost of borrowing;
|
•
|
place us at a competitive disadvantage compared to our competitors that may have less debt; and
|
•
|
limit our ability to obtain additional financing for working capital, capital expenditures, acquisitions, debt service requirements or general corporate purposes.
|
•
|
failure to pay (for more than 30 days) interest when due;
|
•
|
failure to pay principal when due;
|
•
|
failure to deliver shares of Common Stock upon conversion of a Note;
|
•
|
failure to provide notice of a fundamental change;
|
•
|
acceleration on other indebtedness of the Company in excess of $10 million (other than indebtedness that is non-recourse to the Company); or
|
•
|
certain types of bankruptcy or insolvency involving the Company.
|
3.1
|
Certificate of Incorporation of the Company (incorporated by reference to Exhibit 3.1 to Amendment No. 2 to the Company’s General Form for Registration of Securities on Form 10-12G, filed with the SEC on October 28, 2010).
|
3.2
|
Certificate of Amendment of Certificate of Incorporation of the Company (incorporated by reference to Exhibit 3.1 to the Company’s Current Report on Form 8-K, filed with the SEC on June 11, 2015).
|
3.3
|
Amended and Restated Bylaws of the Company (incorporated by reference to Exhibit 3.2 to the Company’s Current Report on Form 8-K, filed with the SEC on June 11, 2015).
|
4.1
|
Form of Warrant Certificate, dated June 30, 2014, issued to the Lenders under the Credit Agreement (incorporated by reference to Exhibit 4.1 to the Company’s Current Report on Form 8-K, filed with the SEC on July 7, 2014).
|
4.2
|
Form of Warrant issued to the purchasers in the private placement of 3,045,929 shares of common stock, dated February 14, 2013 (incorporated by reference to Exhibit 4.1 to the Company’s Current Report on Form 8-K, filed with the SEC on February 19, 2013).
|
4.3
|
Form of Common Stock Purchase Warrant, dated August 15, 2013, issued to the purchasers of securities in the private placement of the Company closed on August 15, 2013 (incorporated by reference to Exhibit 4.1 to the Company’s Current Report on Form 8-K, filed with the SEC on August 20, 2013).
|
4.4
|
Form of Note Purchase Agreement for principal senior convertible notes with an interest rate of 4.50% due 2019 (“2019 Notes”), dated May 29, 2014, by and among the Company and the investors identified therein (incorporated by reference to Exhibit 10.1 to the Company’s Current Report on Form 8-K, filed with the SEC on June 4, 2014).
|
4.5
|
Form of Indenture for 2019 Notes, dated May 30, 2014 (incorporated by reference to Exhibit 10.2 to the Company’s Current Report on Form 8-K, filed with the SEC on June 4, 2014).
|
4.6
|
Form of Note for 2019 Notes, dated May 30, 2014 (incorporated by reference to Exhibit 10.3 to the Company’s Current Report on Form 8-K, filed with the SEC on May 29, 2014).
|
4.7
|
Registration Rights Agreement, dated February 12, 2013, by and among the Company and the February 2013 Purchasers (incorporated by reference to Exhibit 10.2 to the Company’s Current Report on Form 8-K, filed with the SEC on February 19, 2013).
|
4.8
|
Registration Rights Agreement, dated August 15, 2013, by and among the Company and the August 2013 Purchasers (incorporated by reference to Exhibit 10.2 to the Company’s Current Report on Form 8-K, filed with the SEC on August 20, 2013).
|
4.9
|
First Amendment to Registration Rights Agreement, dated August 14, 2013, by and among the Company and the purchasers signatory thereto (incorporated by reference to Exhibit 10.4 to the Company’s Current Report on Form 8-K, filed with the SEC on August 20, 2013).
|
4.10
|
Form of Indenture for Senior Debt Securities (incorporated by reference to Exhibit 4.10 to the Company’s Registration Statement on Form S-8, filed with the SEC on September 9, 2014).
|
4.11
|
Form of Indenture for Subordinated Debt Securities (incorporated by reference to Exhibit 4.11 to the Company’s Registration Statement on Form S-8, filed with the SEC on September 9, 2014).
|
10.1
|
Asset Purchase Agreement dated as of June 9, 2016 between Retrophin, Inc. and Asklepion Pharmaceuticals, LLC * †
|
10.2
|
Retrophin, Inc. 2015 Equity Incentive Plan, as amended (incorporated by reference to Exhibit 99.1 to the Company's current report on Form 8-K, filed with the SEC on May 19, 2016
|
31.1
|
Chief Executive Officer's Certification pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 *
|
31.2
|
Chief Financial Officer's Certification pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 *
|
32.1
|
Chief Executive Officer’s Certification pursuant to Section 906 of the Sarbanes Oxley Act of 2002 *
|
32.2
|
Chief Financial Officer’s Certification pursuant to Section 906 of the Sarbanes Oxley Act of 2002 *
|
101.INS
|
XBRL Instance Document
|
101.SCH
|
XBRL Taxonomy Extension Schema Document
|
101.CAL
|
XBRL Taxonomy Extension Calculation Linkbase Document
|
101.DEF
|
XBRL Taxonomy Extension Definition Linkbase Document
|
101.LAB
|
XBRL Taxonomy Extension Label Linkbase Document
|
101.PRE
|
Taxonomy Extension Presentation Linkbase Document
|
*
|
Filed herewith.
|
†
|
Confidential treatment has been requested with respect to certain portions of this exhibit. Omitted parties have filed separately with the SEC.
|
Date: August 4, 2016
|
RETROPHIN, INC.
|
||
|
|
|
|
|
By:
|
/s/ Stephen Aselage
|
|
|
|
Name:
|
Stephen Aselage
|
|
|
Title:
|
Chief Executive Officer
|
|
|
|
|
|
By:
|
/s/ Laura Clague
|
|
|
|
Name:
|
Laura Clague
|
|
|
Title:
|
Chief Financial Officer
|
Annual Net Revenues of Product
|
Royalty Rate
As Percent of Net Revenues |
[…***…]
|
[…***…]
|
[…***…]
|
[…***…]
|
[…***…]
|
[…***…]
|
[…***…]
|
[…***…]
|
If to Buyer, to:
|
Retrophin, Inc.
12255 El Camino Real Suite 250 San Diego, CA 92130 Attention: General Counsel |
If to Seller, to:
|
Asklepion Pharmaceuticals, LLC
729 East Pratt St Suite 360 |
1.
|
I have reviewed this Quarterly Report on Form 10-Q of Retrophin, 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(s) 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:
|
(a)
|
Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
|
(b)
|
Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
|
(c)
|
Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
|
(d)
|
Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
|
5.
|
The registrant’s other certifying officer(s) 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):
|
(a)
|
All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and
|
(b)
|
Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
|
|
|
|
|
Date: August 4, 2016
|
|
|
|
|
|
|
|
|
|
|
/s/ Stephen Aselage
|
|
|
|
Stephen Aselage
|
|
|
|
Chief Executive Officer
|
|
|
|
(Principal Executive Officer)
|
1.
|
I have reviewed this Quarterly Report on Form 10-Q of Retrophin, 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(s) 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:
|
(a)
|
Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
|
(b)
|
Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
|
(c)
|
Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
|
(d)
|
Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
|
5.
|
The registrant’s other certifying officer(s) 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):
|
(a)
|
All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and
|
(b)
|
Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
|
|
|
|
|
Date: August 4, 2016
|
|
|
|
|
|
|
|
|
|
|
/s/ Laura Clague
|
|
|
|
Laura Clague
|
|
|
|
Chief Financial Officer
|
|
|
|
(Principle Financial Officer)
|
|
|
|
|
Date: August 4, 2016
|
|
|
|
|
|
|
|
|
|
|
/s/ Stephen Aselage
|
|
|
|
Stephen Aselage
|
|
|
|
Chief Executive Officer
|
|
|
|
(Principal Executive Officer)
|
|
|
|
|
Date: August 4, 2016
|
|
|
|
|
|
|
|
|
|
|
/s/ Laura Clague
|
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Laura Clague
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Chief Financial Officer
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(Principal Financial Officer)
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