BioMarin Pharmaceutical Inc announced that patient enrolment has been completed for the phase-I/II clinical trial for BMN-110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. The company expects to report initial results in the first half of 2010.
"The efficient enrolment 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," said Henry Fuchs, chief medical officer of BioMarin. "We appreciate the collaboration of the Morquio community in this important effort, and we hope to develop this new treatment as expeditiously as possible."
Christian Hendriksz, FRCPCH, MRCP, consultant in Metabolic Disorders, Birmingham Children's Hospital, UK, added, "Morquio is a serious and debilitating disease in which accumulation of keratan sulfate results in impaired breathing and walking, recurrent infections, impaired bone and joint function, dysmorphology and an overall impaired quality of life. The rapid accrual of patients for the phase-I/II study speaks to the magnitude of the unmet need for this disease."
The phase-I/II study is designed as 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 phase-I/II study will be to evaluate safety, pharmacokinetics, pharmacodynamics and to identify the optimal dose of GALNS for future studies. BioMarin plans to provide an extension study in which all patients in the phase-I/II study will be eligible to participate.
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.
Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a disorder characterized by deficient activity of N-acetylgalactosamine 6-sulfatase (GALNS)causing excessive lysosomal storage of keratan sulfate (KS).
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises three approved products and multiple clinical and preclinical product candidates.