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BioMarin Pharmaceutical Inc. Message Board

  • blutribe27 blutribe27 Oct 19, 2010 8:07 AM Flag

    GREAT news on clinical data...

    BioMarin Announces Program for BMN-111 for the Treatment of AchondroplasiaLast update: 10/19/2010 8:00:00 AMNOVATO, Calif., Oct 19, 2010 /PRNewswire via COMTEX/ -- BioMarin Pharmaceutical Inc. (BMRN) today announced its program for BMN-111, a peptide therapeutic for the treatment of achondroplasia. BioMarin plans to file an IND in the fourth quarter of 2011 and to initiate a Phase 1 clinical trial by the first quarter of 2012. BMN-111, for the treatment of achondroplasia, is an analog of C-type Natriuretic Peptide (CNP), a small cyclic peptide that is a positive regulator of bone growth. It is produced and has a receptor in the growth plate, and along with the fibroblast growth factor receptor 3 (FGFR3), regulates normal bone growth. In addition to short stature, there are complications in achondroplasia that are related to bone compression (e.g. foramen magnum narrowing, spinal stenosis, upper respiratory narrowing) of nervous tissues or other tissues. "This program is a perfect fit in our growing pipeline. It is an unmet medical need in an orphan disease population and can leverage our expertise in biologic manufacturing," said Hank Fuchs, M.D., Executive Vice President and Chief Medical Officer of BioMarin. "Proof-of-concept studies demonstrate that CNP can reverse the achondroplastic phenotype in mice.

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