BioMarin announces BMN-111 program for treatment of achondroplasia

BioMarin Pharmaceutical Inc. (Nasdaq: 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.  Additionally, in both mice and primates, BMN-111 was able to penetrate the growth plate and accelerate growth at hemodynamically acceptable doses.  We have a proven track record of expeditiously bringing life-altering therapeutics to patients and are determined to do the same with this program.  We look forward to updating you on advancements in this and other programs in our product pipeline."

Additional details of the achondroplasia program, along with an overview of BioMarin's product portfolio and advancements in the research and development pipeline will be provided today at BioMarin's R&D Day program in New York City.  Interested parties may access a live audio webcast of the presentation via the investor section of the BioMarin website, A replay of the presentation will be archived on the site for at least one week following the presentation.

SOURCE BioMarin Pharmaceutical Inc.


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