BioMarin commences BMN 673 Phase 1 trial in hematological malignancies

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the initiation of a Phase 1 trial for BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, for the treatment of patients with advanced hematological malignancies.  A Phase 1/2 trial for BMN 673 for the treatment of patients with solid tumors was initiated in January 2011 and is ongoing.

"We are excited to be at the forefront of studying the potential benefit of PARP inhibitors in hematological malignancies," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin.  "Our approach to the development of our PARP inhibitor is to identify tumor mutations that are more susceptible to treatment with BMN 673 and to target subgroups based on the genetic basis of the disease.  We are committed to the advancement of our pipeline and look forward to many clinical milestones over the next 18 months."

This Phase 1 trial is a two-arm, open-label dose escalation study to determine the maximum tolerated dose and to assess the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of once daily, oral BMN 673 in patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic lymphocytic leukemia (CLL) or mantle cell lymphoma (MCL).  The study will enroll approximately 80 patients across multiple sites in the U.S. and UK.