BioMarin Pharmaceutical has initiated a Phase I/II clinical trial for BMN-110 or N-acetylgalactosamine 6-sulfatase, intended for the treatment of the lysosomal storage disorder mucopolysaccharidosis type IVA, or Morquio A syndrome.
The Phase I/II study is designed as an open-label, within-patient dose escalation trial in approximately 20 patients followed by a treatment continuation phase. All patients to be enrolled in the study have already been identified. During the dose escalation phase of the study, subjects will receive weekly intravenous infusions of BMN-110 in three consecutive 12-week dosing intervals.
The objectives of the Phase I/II study will be to evaluate safety, pharmacokinetics, pharmacodynamics and to identify the optimal dose of N-acetylgalactosamine 6-sulfatase (GALNS) for future studies. The company expects to report initial results in the first half of 2010.
Henry Fuchs, chief medical officer of BioMarin, said: GALNS has been shown in mice to reach important tissues including cartilage and different zones of the bone such as bone marrow, calcified bone and importantly, the growth plate. Our experiments have also shown that GALNS is taken up into human Morquio chondrocytes in vitro and reaches the lysosome to clear keratan sulfate.
BioMarin has developed and manufactures two FDA-approved enzyme replacement therapies, one for the treatment of MPS I and one for the treatment of MPS VI. Naglazyme (galsulfase) for MPS VI is wholly developed and commercialized by BioMarin. Aldurazyme (laronidase) for MPS I is manufactured by BioMarin and marketed by Genzyme Corporation.