BioMarin Announces IND for BMN-111 for Achondroplasia is Active
NOVATO, Calif., Jan. 3, 2012 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the Investigational New Drug (IND) application for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for achondroplasia is active. The company expects to initiate the Phase 1 trial in the first quarter of 2012.
"BMN-111 has demonstrated benefits in moderately and severely affected animal models and has been shown to promote bone growth even in normal animals," stated Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "BMN-111 will mark our seventh program in the clinic, the most ever in the history of the company. We look forward to many potentially value-creating clinical milestones in the coming year."
The primary objective of the Phase 1 trial is to assess the safety and tolerability of single and multiple doses of BMN-111 in normal adult healthy volunteers up to the maximum tolerated dose. The company expects to start the Phase 2 study in pediatric subjects in the fourth quarter of 2012 or the first quarter of 2013.
About AchondroplasiaAchondroplasia is caused by an autosomal dominant activating mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, a negative regulator of bone growth. Eighty percent of cases are the result of a spontaneous mutation, and ninety-eight percent of those cases have a G380R mutation. Achondroplasia is the most common form of human dwarfism and is characterized by failure of normal conversion of cartilage into bone. Clinical manifestations of the disease include short stature, craniomedullary compression, apnea, bowed legs, frontal bossing and midface hypoplasia, permanent sway of the lower back, spinal stenosis, recurrent ear infections and obesity.
The rate of incidence of achondroplasia is one in 15,000 to one in 40,000 live births, with approximately 18,000 to