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Balance Sheets (USD $)
Dec. 31, 2012
Dec. 31, 2011
Current assets:    
Cash and cash equivalents $ 79,311,000 $ 34,577,000
Restricted cash, current portion 7,657,000 7,403,000
Deferred licensor payment   352,000
Prepaid clinical trial expenses   938,000
Prepaid expenses and other current assets 621,000 1,058,000
Total current assets 87,589,000 44,328,000
Property and equipment, net 275,000 720,000
Restricted cash-net of current portion 8,321,000  
Other assets 37,000 31,000
Total assets 96,222,000 45,079,000
Current liabilities:    
Accounts payable 2,152,000 962,000
Accrued personnel costs 2,173,000 1,500,000
Accrued costs for services 4,862,000 3,544,000
Accrued interest 22,000  
Note payable, current portion 1,160,000  
Payable to licensor   1,560,000
Deferred development funds, current portion 5,853,000 4,951,000
Deferred revenue   3,847,000
Total current liabilities 16,222,000 16,364,000
Deferred rent 9,000 7,000
Deferred development funds - net of current portion 8,321,000  
Note payable - net of current portion 2,764,000  
Redeemable convertible preferred stock warrants   2,145,000
Total liabilities 27,316,000 18,516,000
Commitments and contingencies      
Stockholders' equity (deficit):    
Common stock, $0.00001 par value, 300,000,000 and 45,000,000 shares authorized, 18,325,000 and 1,399,000 shares issued and outstanding at December 31, 2012 and 2011, respectively      
Additional paid in capital 189,212,000 2,483,000
Accumulated deficit (120,306,000) (83,507,000)
Total stockholders' equity (deficit) 68,906,000 (81,024,000)
Total liabilities, redeemable convertible preferred stock and stockholders' equity (deficit) 96,222,000 45,079,000
Series A redeemable convertible preferred stock
   
Redeemable convertible preferred stock, $0.0001 par value per share:    
Redeemable convertible preferred stock   902,000
Stockholders' equity (deficit):    
Total stockholders' equity (deficit)   902,000
Series B redeemable convertible preferred stock
   
Redeemable convertible preferred stock, $0.0001 par value per share:    
Redeemable convertible preferred stock   30,193,000
Stockholders' equity (deficit):    
Total stockholders' equity (deficit)   30,193,000
Series C redeemable convertible preferred stock
   
Redeemable convertible preferred stock, $0.0001 par value per share:    
Redeemable convertible preferred stock   40,205,000
Stockholders' equity (deficit):    
Total stockholders' equity (deficit)   40,205,000
Series D redeemable convertible preferred stock
   
Redeemable convertible preferred stock, $0.0001 par value per share:    
Redeemable convertible preferred stock   36,287,000
Stockholders' equity (deficit):    
Total stockholders' equity (deficit)   $ 36,287,000