BioMarin Pharmaceutical has discontinued development of BMN 195 for the treatment of Duchenne muscular dystrophy (DMD) based on results from its Phase I clinical study.

In the dose-escalation trial, administration of up to 400mg/kg of BMN 195 did not achieve the plasma concentrations needed to increase utrophin expression.

The study also found that plasma concentrations of BMN 195, a small molecule utrophin up-regulator, were even lower on repeat administration.

DMD is a fatal neuromuscular disorder caused by a genetic defect that results in DMD patients lacking an important protein called dystrophin, which is crucial in maintaining muscle integrity and function.