Company Information

  

Address: 60 LEVERONI COURT  
City: NOVATO 
State: CA 
Zip Code: 94949 
Telephone: 415-483-8800 

Description of Business

Primary SIC Code: 2834
(As filed with the SEC)  

We are a biopharmaceutical company focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. We target diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no currently approved therapies. Since our inception in 2010, we have in-licensed potential treatments for multiple rare genetic disorders. Our strategy, which is predicated upon time- and cost-efficient drug development, allows us to pursue multiple programs in parallel with the goal of delivering safe and effective therapies to patients with the utmost urgency. Our approved product and current product candidate pipeline have been either in-licensed from academic institutions or derived from partnerships with other pharmaceutical companies.  ... More ...  

Per Share Overview

Date 12-mos Rolling EPS Dividend P/E Ratio
03/2018-4.87NAN/E
12/2017-7.12NAN/E
09/2017-6.97NAN/E
06/2017-6.75NAN/E
03/2017-6.52NAN/E
12/2016-6.21NAN/E
09/20162.01NA35.29
06/20162.61NA18.74

Key Financial Ratios and Statistics

FYE: 12/31

Profitability 2017 Leverage 2017
Net Inc/Comm Equity-0.25Total Liab/Total Assets0.22
Net Inc/Total Assets-0.62Total Liab/Inv Cap0.26
Net Inc/Inv Cap-0.72Total Liab/Comm Equity0.09
Pretax Inc/Net Sales-121.88Interest Coverage RatioNA
Net Inc/Net Sales-115.67Curr Debt/EquityNA
Cash Flow/Net Sales-97.18LTD/EquityNA
SG&A/NetSales38.25Total Debt/EquityNA
Asset Utilization   Liquidity  
Net Receivables Turnover1.01Quick Ratio3.79
Inventory Turnover0.00Current Ratio3.80
Inventory Day Sales0.00Net Rec/Curr Assets0.02
Net Sales/Work Cap0.01Inv/Curr Assets0.00
Net Sales/PP&E0.12  

Income Statement (Millions)

  3/31/2018 12/31/2017 9/30/2017 6/30/2017
Total Revenues(Net Sales) 10.68 2.41 0.20 0.00
Cost of Goods Sold 0.23 0.00 NA 0.00
Selling & Admin Exps 31.44 37.72 23.50 20.01
Operating Income -96.49 -96.83 -83.71 -78.44
Interest Exp NA NA NA NA
Pretax Income 30.29 -97.95 -79.22 -72.88
Other Income 126.78 -1.11 4.49 5.56
Net Income Bef Extraordinary ... NA NA NA NA
Net Income 30.25 -81.73 -79.23 -72.89

Balance Sheet (Millions)

Assets 3/31/2018 12/31/2017 9/30/2017 6/30/2017
Cash & Short Term Investments 541.89 234.95 371.53 400.11
Receivables - Total 3.67 5.17 NA NA
Inventories - Total 2.07 0.76 NA NA
Total Current Assets 581.58 269.58 390.90 419.79
Net Property, Plant & Equipment 20.83 21.84 16.03 16.41
Total Assets 817.35 490.75 435.05 497.54
Liabilities        
Accounts Payable 56.86 70.31 63.57 62.01
Debt in Current Liabilities NA NA NA NA
Total Current Liabilities 57.58 71.01 64.26 62.68
Long-Term Debt NA NA NA NA
Total Liabilities 93.69 107.30 69.49 68.09
Stockholder's Equity        
Minority Interest NA NA NA NA
Preferred Stock 0.00 0.00 0.00 0.00
Common Stock 0.05 0.04 0.04 0.04
Retained Earnings -802.42 -832.67 -750.94 -671.71
Treasury Stock NA NA NA NA
Total Stockholders' Equity 723.66 383.45 365.56 429.45
Total Liabilities and Stockholders' Equity 817.35 490.75 435.05 497.54

Cash Flow Summary (Millions)

Categories 3/31/2018 12/31/2017 9/30/2017 6/30/2017
Net Cash Provided by Operating Activities -89.48 -81.82 -61.99 -48.82
Net Cash Provided by Investing Activities -71.03 57.10 29.79 -19.86
Net Cash Provided by Financing Activities 286.42 64.44 1.33 1.53

Annual Summary Data (Millions)

Year Sales Net Income EPS
12/20130.00-35.07--
12/20140.00-59.80-2.25
12/20150.00-145.623.96
12/20160.13-245.87-6.21
12/20172.61-302.14-7.12
Growth RatesInfinity----

Stock Ownership

Type Date(Q,M) No. Owners Shares Held (000s) % Own
Institutional06/30/1819050,013--




Report Date : 7/16/2018

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