Company Information


Address: 435 DEVON PARK DRIVE,
SUITE 715 
City: WAYNE 
State: PA 
Zip Code: 19087 
Telephone: 610-254-4201 

Description of Business

Primary SIC Code: 2836
(As filed with the SEC)  

We are a clinical stage biopharmaceutical company with an emphasis on identifying the genetic drivers of disease and applying this understanding to the pursuit of differentiated novel therapies primarily for pediatric onset, life-altering diseases, including rare and orphan diseases. We look to find treatments for genetically defined diseases for which there are limited therapeutic options currently available, with a primary focus on pediatric patients. This strategy begins with identifying and genetically validating a therapeutic target and using genomics to guide product development. The strategy also involves identifying and acquiring otherwise abandoned or overlooked drug candidates and matching targets and mechanisms of action to novel genetic discoveries. We have partnered with the Center for Applied Genomics, or CAG, at The Children's Hospital of Philadelphia, or CHOP, to implement a genomic medicine driven approach to drug development.  ... More ...  

Per Share Overview

Date 12-mos Rolling EPS Dividend P/E Ratio

Key Financial Ratios and Statistics

FYE: 12/31

Profitability 2017 Leverage 2017
Net Inc/Comm Equity-0.14Total Liab/Total Assets0.12
Net Inc/Total Assets-1.00Total Liab/Inv Cap0.14
Net Inc/Inv Cap-1.13Total Liab/Comm Equity0.02
Pretax Inc/Net SalesNAInterest Coverage Ratio-845.68
Net Inc/Net SalesNACurr Debt/EquityNA
Cash Flow/Net SalesNALTD/EquityNA
SG&A/NetSalesNATotal Debt/EquityNA
Asset Utilization   Liquidity  
Net Receivables TurnoverNAQuick Ratio8.36
Inventory TurnoverNACurrent Ratio8.36
Inventory Day SalesNANet Rec/Curr AssetsNA
Net Sales/Work CapNAInv/Curr AssetsNA
Net Sales/PP&ENA  

Income Statement (Millions)

  3/31/2018 12/31/2017 9/30/2017 6/30/2017
Total Revenues(Net Sales) NA NA NA NA
Cost of Goods Sold NA NA NA NA
Selling & Admin Exps 2.17 1.90 2.27 2.37
Operating Income -8.74 -7.16 -8.57 -8.04
Interest Exp NA 0.04 NA NA
Pretax Income -8.71 -7.16 -8.61 -8.03
Other Income 0.03 NA NA NA
Net Income Bef Extraordinary ... NA NA NA NA
Net Income -8.71 -7.16 -8.61 -8.03

Balance Sheet (Millions)

Assets 3/31/2018 12/31/2017 9/30/2017 6/30/2017
Cash & Short Term Investments 26.52 33.73 14.96 21.36
Receivables - Total NA NA NA NA
Inventories - Total NA NA NA NA
Total Current Assets 27.32 34.62 15.91 22.43
Net Property, Plant & Equipment 0.07 0.09 0.10 0.30
Total Assets 27.43 34.76 16.95 22.74
Accounts Payable 4.74 4.14 6.86 4.92
Debt in Current Liabilities NA NA NA NA
Total Current Liabilities 4.74 4.14 6.86 4.92
Long-Term Debt NA NA NA NA
Total Liabilities 4.74 4.14 6.86 4.92
Stockholder's Equity        
Minority Interest NA NA NA NA
Preferred Stock NA NA NA NA
Common Stock 0.01 0.01 0.00 0.00
Retained Earnings -223.69 -214.98 -207.82 -199.21
Treasury Stock NA NA NA NA
Total Stockholders' Equity 22.70 30.62 10.09 17.82
Total Liabilities and Stockholders' Equity 27.43 34.76 16.95 22.74

Cash Flow Summary (Millions)

Categories 3/31/2018 12/31/2017 9/30/2017 6/30/2017
Net Cash Provided by Operating Activities -7.24 -8.34 -6.40 -7.85
Net Cash Provided by Investing Activities 0.00 -0.00 0.15 0.00
Net Cash Provided by Financing Activities 0.03 27.12 -0.15 0.00

Annual Summary Data (Millions)

Year Sales Net Income EPS
Growth RatesNaN----

Stock Ownership

Type Date(Q,M) No. Owners Shares Held (000s) % Own

Report Date : 7/16/2018

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