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 clinical-stage biopharmaceutical company focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultra-rare 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 current product candidate pipeline has 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
06/2017-6.75NAN/E
03/2017-6.52NAN/E
12/2016-6.21NAN/E
09/20162.01NA35.29
06/20162.61NA18.74
03/20163.24NA19.54
12/20153.96NA28.33
09/2015-3.03NAN/E

Key Financial Ratios and Statistics

FYE: 12/31

Profitability 2016 Leverage 2016
Net Inc/Comm Equity-0.24Total Liab/Total Assets0.12
Net Inc/Total Assets-0.45Total Liab/Inv Cap0.14
Net Inc/Inv Cap-0.51Total Liab/Comm Equity0.07
Pretax Inc/Net Sales-1848.41Interest Coverage RatioNA
Net Inc/Net Sales-1848.68Curr Debt/EquityNA
Cash Flow/Net Sales-1210.34LTD/EquityNA
SG&A/NetSales488.24Total Debt/EquityNA
Asset Utilization   Liquidity  
Net Receivables TurnoverNAQuick Ratio6.67
Inventory TurnoverNACurrent Ratio6.67
Inventory Day SalesNANet Rec/Curr AssetsNA
Net Sales/Work Cap0.00Inv/Curr AssetsNA
Net Sales/PP&E0.01  

Income Statement (Millions)

  6/30/2017 3/31/2017 12/31/2016 9/30/2016
Total Revenues(Net Sales) 0.00 NA 0.01 0.11
Cost of Goods Sold NA NA NA NA
Selling & Admin Exps 20.01 18.69 19.81 17.18
Operating Income -78.44 -69.95 -70.55 -65.78
Interest Exp NA NA NA NA
Pretax Income -72.88 -68.29 -71.25 -64.91
Other Income 5.56 1.66 -0.70 0.88
Net Income Bef Extraordinary ... NA NA NA NA
Net Income -72.89 -68.29 -71.29 -64.91

Balance Sheet (Millions)

Assets 6/30/2017 3/31/2017 12/31/2016 9/30/2016
Cash & Short Term Investments 400.11 425.46 381.56 397.84
Receivables - Total NA NA NA NA
Inventories - Total NA NA NA NA
Total Current Assets 419.79 444.69 401.70 416.79
Net Property, Plant & Equipment 16.41 16.52 17.06 18.23
Total Assets 497.54 545.82 540.63 515.17
Liabilities        
Accounts Payable 62.01 51.12 59.92 50.10
Debt in Current Liabilities NA NA NA NA
Total Current Liabilities 62.68 51.76 60.26 50.43
Long-Term Debt NA NA NA NA
Total Liabilities 68.09 57.34 66.65 59.28
Stockholder's Equity        
Minority Interest NA NA NA NA
Preferred Stock 0.00 0.00 0.00 0.00
Common Stock 0.04 0.04 0.04 0.04
Retained Earnings -671.71 -598.82 -530.53 -459.25
Treasury Stock NA NA NA NA
Total Stockholders' Equity 429.45 488.48 473.97 455.89
Total Liabilities and Stockholders' Equity 497.54 545.82 540.63 515.17

Cash Flow Summary (Millions)

Categories 6/30/2017 3/31/2017 12/31/2016 9/30/2016
Net Cash Provided by Operating Activities -48.82 -61.21 -47.68 -28.75
Net Cash Provided by Investing Activities -21.08 -9.40 -8.36 52.51
Net Cash Provided by Financing Activities 1.53 68.97 74.94 63.45

Annual Summary Data (Millions)

Year Sales Net Income EPS
12/20120.00-16.33--
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
Growth RatesInfinity----

Stock Ownership

Type Date(Q,M) No. Owners Shares Held (000s) % Own
Institutional09/30/1717841,54497.85




Report Date : 10/23/2017

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