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BioMarin Announces Phase 1 Results For BMN-111 For Achondroplasia

NOVATO, Calif., Sept. 26, 2012 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the completion of a Phase 1 study for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for achondroplasia. The company expects to initiate a Phase 2 trial in mid-2013.

The Phase 1 study was a two-part, double-blind, placebo-controlled study in healthy adult males. Part 1 examined a series of single subcutaneous doses and Part 2 included ten days of either fixed dosing or dose escalation. The primary objective of the study was to evaluate safety, tolerability and pharmacokinetics (PK) of single and multiple doses of BMN-111 in 48 healthy adult volunteers.

BMN-111 was generally well-tolerated. Mild, transient, self-limited hypotension was observed. The majority of these cases were asymptomatic, and only observed upon assumption of an upright posture following recumbence. No dose-limiting toxicities were identified outside of these cardiovascular findings. Systemic exposure to BMN-111 was similar at these doses to what has been observed to cause growth in healthy and disease model animals. All adverse events were of mild severity. 

"We have identified a safe starting dose for treatment of achondroplastic children," stated Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "Further, we have identified the likely side effects of excessive exposure, at least in the short-term, and we do expect BMN-111 to be well-tolerated in children. We are presently evaluating options for further development in various forms of achondroplasia."

Based on the results of the Phase 1 study, the design of a Phase 2 proof-of-concept and dose finding study is underway.  The goal of the program is to assess growth velocity as well as medical complications of achondroplasia, including non-growth endpoints. To better understand the nature of the disease, a study to collect consistent baseline growth measurements in children with achondroplasia was initiated in the second quarter of 2012. Participants in this study will be considered for enrollment in the Phase 2 study. 

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