Published on January 12, 2010 at 12:00 AM
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that the first subject has initiated treatment in the Phase 1 clinical study of BMN 195, a small molecule utrophin upregulator, for the treatment of Duchenne muscular dystrohpy (DMD). Initial top-line results are expected in the third quarter of 2010.
"Duchenne muscular dystrophy represents a serious unmet medical need affecting approximately 40,000 patients in the developed world, and we are excited to advance our program into the clinic in hopes of providing the first therapeutic option to treat this disease," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "BMN 195 has been shown to upregulate utrophin levels in human muscle cells, as a means of augmenting muscle function. In mice with mutations in the normal dystrophin gene, BMN 195 has been shown to improve strength. Therefore, BMN 195 may have the potential to treat the entire spectrum of DMD patients, regardless of the type of genetic abnormality."
The Phase 1 clinical trial is a single-center, double-blind, placebo-controlled, single-dose escalation study followed by a multiple-dose escalation study of BMN 195 administered orally in healthy volunteers. The primary objective is to assess the safety, tolerability and pharmacokinetics of BMN 195 in healthy volunteers, and enable subsequent studies in patients with DMD.
SOURCE BioMarin Pharmaceutical Inc.