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CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($)
$ in Thousands
6 Months Ended
Jun. 30, 2018
Jun. 30, 2017
Operating activities:    
Net loss $ (91,673) $ (84,214)
Adjustments to reconcile net loss to net cash (used in) provided by operating activities:    
Depreciation 54 56
Amortization of licenses 70 60
Amortization of premium on investments, net 208 139
Non-cash sublicensing expense 0 33,394
Non-cash interest expense for line of credit with Ionis Pharmaceuticals, Inc. 0 1,507
Stock-based compensation expense 18,509 7,122
Changes in operating assets and liabilities:    
Contracts receivable 2,118 0
Other current and long-term assets (2,275) (1,226)
Accounts payable 325 (799)
Payable to Ionis Pharmaceuticals, Inc. 12,772 (13,111)
Accrued compensation 465 (1,253)
Deferred rent 518 4
Accrued liabilities 17,458 1,136
Income taxes payable (720) 0
Deferred revenue (29,141) 63,193
Net cash (used in) provided by operating activities (71,312) 6,008
Investing activities:    
Purchases of short-term investments (22,197) (61,209)
Proceeds from sale of short-term investments 94,736 17,820
Purchase of property, plant and equipment (5) 0
Net cash provided by (used in) investing activities 72,534 (43,389)
Financing activities:    
Proceeds from exercise of common stock options and employee stock purchase plan issuances 3,281 0
Proceeds from line of credit from Ionis Pharmaceuticals, Inc. 0 106,000
Proceeds from issuance of common stock to Ionis in TTR transaction 156,306 0
Offering costs paid 0 (1,031)
Net cash provided by financing activities 159,587 104,969
Effect of exchange rates on cash 48 0
Net increase in cash and cash equivalents 160,857 67,588
Cash, cash equivalents and restricted cash at beginning of period 58,367 7,857
Cash, cash equivalents and restricted cash at end of period 219,224 75,445
Supplemental disclosures of non-cash investing and financing activities:    
Purchase of property, plant and equipment included in accrued liability 491 0
Purchase of property, plant and equipment included in long-term deferred rent liability 1,004 0
Acquisition of research and development licenses 563 0
Unpaid deferred offering costs 450 473
Cash, cash equivalents and restricted cash reported within the condensed consolidated balance sheets:    
Cash and cash equivalents 216,840 75,445
Restricted cash included in deposits and other assets 2,384 0
Cash, cash equivalents and restricted cash at end of period $ 219,224 $ 75,445