BioMarin Pharmaceutical has begun treating patients in the first phase of a clinical study of BMN 195, a small molecule utrophin upregulator for the treatment of Duchenne muscular dystrophy (DMD).
BioMarin chief executive officer Jean-Jacques Bienaime said Duchenne muscular dystrophy represents a serious unmet medical need, affecting approximately 40,000 patients in the developed world.
"BMN 195 has been shown to upregulate utrophin levels in human muscle cells, as a means of augmenting muscle function. In mice 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," Bienaime said.
The single-centre, double-blind, placebo-controlled, single-dose escalation study will be 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.
Initial top-line results are expected in the third quarter of 2010.