NOVATO, Calif., July 28, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced an update on the GALNS Phase 1/2 extension study (MOR-100) in which patients have continued treatment on an ongoing basis.
Patients originally enrolled in the initial Phase 1/2 study of GALNS (MOR-002) were continued on therapy in a new extension study (MOR-100). In the MOR-100 study, patients were treated at a 2.0 mg/kg/week dose and have been followed for an additional 24 weeks. Previously, these patients were dosed at 0.1 mg/kg/week, 1.0 mg/kg/week and 2.0 mg/kg/week for 12 weeks each in the Phase 1/2 study (MOR-002). Following the dose escalation phase, patients were then dosed at 1.0 mg/kg/week for an additional 36 to 48 weeks as part of the MOR-002 extension, before continuing on to MOR-100. Taken together, these patients have received GALNS for approximately two years.
"We now have data from MPS IVA patients that have been on GALNS for approximately 2 years and the data continues to look promising," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "Looking at the Phase 1/2 data in totality, we now see that the endurance and pulmonary function improvements are durable and clinically meaningful compared to baseline, and similar in magnitude to what we have observed during the development of Naglazyme and Aldurazyme. These data are encouraging in regard to the ongoing Phase 3 study in that the benefits of GALNS appear consistently sustained, and no new risks have emerged. Enrollment in the Phase 3 study is accelerating and its execution is BioMarin's highest product development priority."
Preliminary highlights, including information on patients treated for 24 additional weeks at 2.0 mg/kg/week are provided below. Data from the extension study will be submitted for presentation at the World
|SOURCE BioMarin Pharmaceutical Inc.|
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