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BioMarin Announces Third Quarter 2010 Financial Results
Date:10/28/2010

>BMN-701 for Pompe Disease: BioMarin expects to initiate a Phase I/II trial in Pompe patients in December 2010 or January 2011.  The company believes that BMN-701, a novel fusion of  insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) has the potential to be more efficacious and with a more favorable safety profile as compared to the currently approved product.
  • BMN-111 for Achondroplasia: BioMarin expects to file an IND by the end of 2011 and initiate a Phase 1 trial by the first quarter of 2012.  BMN-111 is an analog of C-type Natriuretic Peptide (CNP) for achondroplasia, a small cyclic peptide that is a positive regulator of bone growth.  Achondroplasia is the most common form of dwarfism.  There are approximately between 18,000 and 24,000 patients in the U.S. and Europe, 25 percent of which is the estimated addressable market.
  • Other early stage programs: BioMarin is working on multiple early development opportunities.

  • Non-GAAP Financial Information and ReconciliationThe above results for the three and nine months ended September 30, 2010 and September 30, 2009 and financial guidance for the year ending December 31, 2010 are presented both as determined in accordance with GAAP and on a non-GAAP basis.  As used in this release, non-GAAP income is calculated in accordance with GAAP, but excludes non-cash stock compensation expense, certain nonrecurring material items and the tax effect of the adjustments.  The following tables detail the reconciliation of non-GAAP to GAAP financial metrics:

    Reconciliation of GAAP Net Income (Loss) to Non-GAAP Net Income(In millions)(Unaudited)Three Months Ended September 30,Nine Months Ended September 30,Year Ended December 31,Year Ending December 31,Notes:2009201020092010 20092010(actuals)(forecast)GAAP Net Income (Loss)$

    6.6217.3$

    (5.2)$

    218.0$

    (0.5)$

    213.0 to 218.0Stock-based comp
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    SOURCE BioMarin Pharmaceutical Inc.
    Copyright©2010 PR Newswire.
    All rights reserved

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