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CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($)
$ in Thousands
12 Months Ended
Dec. 31, 2017
Dec. 31, 2016
Dec. 31, 2015
Operating activities:      
Net loss $ (121,559) $ (83,217) $ (61,422)
Adjustments to reconcile net loss to net cash used in operating activities:      
Depreciation 108 12 0
Amortization of licenses 120 119 119
Amortization of premium on investments, net 499 170 18
Non-cash interest expense for line of credit with Ionis Pharmaceuticals, Inc. 1,731 0 0
Non-cash sublicensing expense 33,394 0 0
Stock-based compensation expense 17,539 10,149 6,496
Changes in operating assets and liabilities:      
Contracts receivable (5,413) 0 0
Other current and long-term assets (1,761) 64 (286)
Accounts payable 1,905 (54) 239
Payable to Ionis Pharmaceuticals, Inc. (43,385) 15,157 9,198
Accrued compensation 1,578 1,582 923
Deferred rent (15) 20 35
Accrued liabilities 6,587 637 405
Income taxes payable 1,789 0 0
Deferred revenue 70,693 0 0
Net cash used in operating activities (36,190) (55,361) (44,275)
Investing activities:      
Purchases of short-term investments (301,377) (16,638) (35,975)
Proceeds from sale of short-term investments 98,778 51,464 960
Purchases of property, plant and equipment (9) (179) (10)
Net cash (used in) provided by investing activities (202,608) 34,647 (35,025)
Financing activities:      
Proceeds from issuance of Series A convertible preferred stock to Ionis Pharmaceuticals, Inc. 0 0 100,000
Capital contribution from Ionis Pharmaceuticals, Inc. 0 0 8,689
Proceeds from issuance of common stock, net of underwriters' discounts 135,438 0 0
Proceeds from sale of common stock to Novartis in private placement 50,000 0 0
Proceeds from line of credit from Ionis Pharmaceuticals, Inc. 106,000 0 0
Offering costs paid (2,037) (818) 0
Net cash provided by (used in) financing activities 289,401 (818) 108,689
Effect of exchange rates on cash (93) 0 0
Net increase (decrease) in cash and cash equivalents 50,510 (21,532) 29,389
Cash and cash equivalents at beginning of period 7,857 29,389 0
Cash and cash equivalents at end of period 58,367 7,857 29,389
Supplemental disclosures of non-cash financing activities:      
Unpaid deferred offering costs 0 291 0
Conversion of preferred stock to common stock upon initial public offering 100,000 0 0
Conversion of line of credit from Ionis Pharmaceuticals, Inc. into common stock $ 107,731 $ 0 $ 0