Company Information

  

Address: 215 FIRST STREET
SUITE 415 
City: CAMBRIDGE 
State: MA 
Zip Code: 02142 
Telephone: 617-274-4000 

Description of Business

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

We are a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, gene therapy and other genetic medicine approaches for the treatment of rare neuromuscular diseases. Applying our proprietary, highly-differentiated and innovative RNA-targeted platform technologies, we are able to develop candidate therapies for a broad range of diseases and disorders. Our first commercial product in the U.S., EXONDYS 51, was granted accelerated approval by the FDA on September 19, 2016. EXONDYS 51 is indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. A summary description of our product and main product candidates is as follows: * EXONDYS 51, our first product, uses our PMO chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene.  ... More ...  

Per Share Overview

Date 12-mos Rolling EPS Dividend P/E Ratio
03/2018-2.91NAN/E
12/2017-0.86NAN/E
09/2017-2.13NAN/E
06/2017-2.46NAN/E
03/2017-2.68NAN/E
12/2016-5.49NAN/E
09/2016-5.28NAN/E
06/2016-2.97NAN/E

Key Financial Ratios and Statistics

FYE: 12/31

Profitability 2017 Leverage 2017
Net Inc/Comm Equity-0.03Total Liab/Total Assets0.40
Net Inc/Total Assets-0.04Total Liab/Inv Cap0.43
Net Inc/Inv Cap-0.04Total Liab/Comm Equity0.26
Pretax Inc/Net Sales-0.31Interest Coverage RatioNA
Net Inc/Net Sales-0.33Curr Debt/Equity0.01
Cash Flow/Net Sales-1.50LTD/Equity0.54
SG&A/NetSales0.79Total Debt/Equity0.55
Asset Utilization   Liquidity  
Net Receivables Turnover8.91Quick Ratio12.96
Inventory Turnover0.15Current Ratio13.91
Inventory Day Sales0.00Net Rec/Curr Assets0.02
Net Sales/Work Cap0.14Inv/Curr Assets0.07
Net Sales/PP&E3.58  

Income Statement (Millions)

  3/31/2018 12/31/2017 9/30/2017 6/30/2017
Total Revenues(Net Sales) 64.60 57.28 45.95 35.01
Cost of Goods Sold 5.58 3.55 3.02 0.56
Selling & Admin Exps 43.34 32.22 28.18 36.07
Operating Income -30.74 -23.15 -45.91 -63.34
Interest Exp NA NA NA NA
Pretax Income -35.22 -25.84 -45.72 -63.16
Other Income 0.00 0.00 0.00 0.00
Net Income Bef Extraordinary ... NA NA NA NA
Net Income -35.36 -24.00 -47.73 -63.05

Balance Sheet (Millions)

Assets 3/31/2018 12/31/2017 9/30/2017 6/30/2017
Cash & Short Term Investments 1,048.99 1,079.06 617.63 300.95
Receivables - Total 39.85 29.47 24.75 17.79
Inventories - Total 99.38 83.61 64.69 41.75
Total Current Assets 1,219.42 1,228.64 735.61 390.59
Net Property, Plant & Equipment 53.93 43.16 38.87 39.39
Total Assets 1,300.28 1,307.96 800.28 450.14
Liabilities        
Accounts Payable 83.03 77.45 61.07 50.10
Debt in Current Liabilities 3.45 6.18 4.73 11.22
Total Current Liabilities 91.20 88.33 70.47 65.96
Long-Term Debt 427.37 424.88 26.55 0.00
Total Liabilities 523.52 518.75 103.13 72.37
Stockholder's Equity        
Minority Interest NA NA NA NA
Preferred Stock 0.00 0.00 0.00 0.00
Common Stock 0.01 0.01 0.01 0.01
Retained Earnings -1,252.37 -1,217.01 -1,193.01 -1,145.28
Treasury Stock NA NA NA NA
Total Stockholders' Equity 776.76 789.22 697.16 377.77
Total Liabilities and Stockholders' Equity 1,300.28 1,307.96 800.28 450.14

Cash Flow Summary (Millions)

Categories 3/31/2018 12/31/2017 9/30/2017 6/30/2017
Net Cash Provided by Operating Activities -35.67 -31.06 -57.90 -85.15
Net Cash Provided by Investing Activities -14.26 -493.70 124.95 -20.05
Net Cash Provided by Financing Activities 7.47 506.81 382.24 -1.17

Annual Summary Data (Millions)

Year Sales Net Income EPS
12/201314.22-111.98-3.31
12/20149.76-135.79-2.81
12/20151.25-220.03-5.20
12/20165.42-267.26-5.49
12/2017154.58-50.69-0.86
Growth Rates81.58----

Stock Ownership

Type Date(Q,M) No. Owners Shares Held (000s) % Own
Institutional03/31/1830856,87486.79




Report Date : 6/18/2018

Powered By Edgar Online © 2018EDGAR®Online, a division of Donnelley Financial Solutions. EDGAR® is a federally registered trademark of the U.S. Securities and Exchange Commission. EDGAR Online is not affiliated with or approved by the U.S. Securities and Exchange Commission.