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 the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases. Applying our proprietary, highly-differentiated and innovative platform technologies, we are able to target a broad range of diseases and disorders through distinct RNA-targeted mechanisms of action. DMD Focus We are primarily focused on rapidly advancing the development of our potentially disease-modifying pipeline of exon-skipping drug candidates targeting DMD. DMD is a rare genetic disorder affecting children (primarily males) that characterized by progressive muscle deterioration and weakness. It is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The absence of dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement.  ... More ...  

Per Share Overview

Date 12-mos Rolling EPS Dividend P/E Ratio
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
03/2016-2.63NAN/E
12/2015-5.20NAN/E

Key Financial Ratios and Statistics

FYE: 12/31

Profitability 2016 Leverage 2016
Net Inc/Comm Equity-0.18Total Liab/Total Assets0.21
Net Inc/Total Assets-0.63Total Liab/Inv Cap0.26
Net Inc/Inv Cap-0.79Total Liab/Comm Equity0.06
Pretax Inc/Net Sales-49.30Interest Coverage RatioNA
Net Inc/Net Sales-49.30Curr Debt/Equity0.03
Cash Flow/Net Sales-45.35LTD/Equity0.02
SG&A/NetSales15.45Total Debt/Equity0.05
Asset Utilization   Liquidity  
Net Receivables Turnover1.18Quick Ratio4.78
Inventory Turnover0.02Current Ratio4.95
Inventory Day Sales0.00Net Rec/Curr Assets0.01
Net Sales/Work Cap0.02Inv/Curr Assets0.03
Net Sales/PP&E0.14  

Income Statement (Millions)

  9/30/2017 6/30/2017 3/31/2017 12/31/2016
Total Revenues(Net Sales) 45.95 35.01 16.34 5.42
Cost of Goods Sold 3.02 0.53 0.25 0.13
Selling & Admin Exps 28.18 36.07 26.22 22.94
Operating Income -45.91 -63.34 -39.25 -88.40
Interest Exp NA NA NA NA
Pretax Income -45.72 -63.16 86.09 -88.45
Other Income 0.00 0.00 125.00 0.00
Net Income Bef Extraordinary ... NA NA NA NA
Net Income -47.73 -63.05 84.09 -88.45

Balance Sheet (Millions)

Assets 9/30/2017 6/30/2017 3/31/2017 12/31/2016
Cash & Short Term Investments 617.63 300.95 390.31 328.54
Receivables - Total 24.75 17.79 12.28 5.23
Inventories - Total 64.69 41.75 30.45 12.81
Total Current Assets 735.61 390.59 457.31 373.48
Net Property, Plant & Equipment 38.87 39.39 38.41 37.80
Total Assets 800.28 450.14 511.53 424.10
Liabilities        
Accounts Payable 61.07 50.10 58.60 60.71
Debt in Current Liabilities 4.73 11.22 11.35 10.11
Total Current Liabilities 70.47 65.96 74.63 75.42
Long-Term Debt 26.55 0.00 2.34 6.04
Total Liabilities 103.13 72.37 82.39 87.41
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 -1,193.01 -1,145.28 -1,082.23 -1,166.32
Treasury Stock NA NA NA NA
Total Stockholders' Equity 697.16 377.77 429.14 336.69
Total Liabilities and Stockholders' Equity 800.28 450.14 511.53 424.10

Cash Flow Summary (Millions)

Categories 9/30/2017 6/30/2017 3/31/2017 12/31/2016
Net Cash Provided by Operating Activities -57.90 -85.15 -57.89 -81.23
Net Cash Provided by Investing Activities 124.95 -20.05 209.99 -198.77
Net Cash Provided by Financing Activities 382.24 -1.17 0.21 7.29

Annual Summary Data (Millions)

Year Sales Net Income EPS
12/201237.33-121.29-5.14
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
Growth Rates-38.27----

Stock Ownership

Type Date(Q,M) No. Owners Shares Held (000s) % Own
Institutional09/30/1725553,43982.68




Report Date : 12/4/2017

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