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Condensed Statements of Cash Flows (Unaudited) (USD $)
 9 Months Ended
Sep. 30, 2014
Sep. 30, 2013
Net loss $ (55,743,000) $ (41,240,000)
Adjustments to reconcile net loss to net cash used for operating activities:    
Depreciation and amortization 389,000 237,000
Stock-based compensation expense 5,809,000 8,333,000
Amortization of debt discount 425,000 396,000
Changes in operating assets and liabilities:    
Prepaid expenses and other assets (2,392,000) (202,000)
Accounts payable 211,000 514,000
Accrued clinical liabilities 2,757,000 302,000
Accrued payroll and employee-related liabilities 35,000 454,000
Other accrued expenses 1,084,000 70,000
Net cash used for operating activities (47,425,000) (31,136,000)
Investing activities:    
Purchases of property and equipment (417,000) (1,518,000)
Net cash used for investing activities (417,000) (1,518,000)
Financing activities:    
Proceeds from warrant exercises   600,000
Proceeds from purchases under the Employee Stock Purchase Plan 19,000 25,000
Proceeds from stock option exercises 2,832,000 1,120,000
Net proceeds from sale of common stock and pre-funded warrants 58,916,000  
Net cash provided by financing activities 61,767,000 1,745,000
Net increase (decrease) in cash 13,925,000 (30,909,000)
Cash at beginning of period 72,287,000 53,506,000
Cash at end of period $ 86,212,000 $ 22,597,000