Phase 1/2 study of BMN 701 for the treatment of Pompe disease
BioMarin Pharmaceutical announced it has initiated a Phase 1/2 trial of BMN 701 for the treatment of Pompe disease. The trial is an open-label study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamic and clinical activity of BMN 701. BMN 701 will be administered as an intravenous infusion every 2 weeks at doses of 5mg/kg, 10mg/kg and 20mg/kg. The company expects to enroll approximately 30 patients between the ages of 13-65 years old with late-onset Pompe disease for a treatment period of 24 weeks.
BMN 701 is a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease, a lysosomal storage disorder. This disorder is caused by a deficiency in the lysosomal enzyme acid alpha glucosidase which leads to the accumulation of glycogen in muscle cell lysosomes and results in cell death and eventual degeneration of skeletal muscles.
For more information call (415) 506-6700 or visit www.bmrn.com.