BioMarin has completed patient enrollment for the phase I/II clinical trial of BMN-110 or N-acetylgalactosamine 6-sulfatase (GALNS). The BMN-110 trial is intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA) or Morquio A Syndrome.
The phase I/II study is an open-label, within-patient dose escalation trial in 20 patients, followed by a treatment continuation period. 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 study is to evaluate safety, pharmacokinetics, pharmacodynamics and to identify the optimal dose of GALNS for future studies.
Henry Fuchs, CMO of BioMarin, said: The efficient enrollment of twenty patients is a critical milestone for the MPS IVA program and demonstrates both our commitment to this program and the support and enthusiasm of the MPS IVA patient community. Data generated from this study will be valuable in demonstrating safety and could be instrumental in designing a successful Phase III trial.”
“Assessments from the Phase I/II study such as plasma and urine keratan sulfate levels, pulmonary function and walk tests will be helpful in determining optimal Phase III endpoints. We appreciate the collaboration of the Morquio community in this important effort, and we hope to develop this new treatment as expeditiously as possible,” he added.