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GALNS Improves Walk Distance

Mon, 11/12/2012 - 2:42pm

BioMarin Pharmaceutical Inc. announced that the pivotal Phase 3 study of GALNS met the primary endpoint of change in six-minute walk distance compared with placebo at 24 weeks in subjects receiving weekly infusions of GALNS at the dose of 2 mg/kg (p=0.0174). MOR-004 was a randomized, double-blind, placebo-controlled study evaluating two doses of GALNS (BMN-110, N- acetylgalactosamine-6-sulfatase) for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome. Patients dosed with GALNS at 2 mg/kg every other week did not show a meaningful or statistically significant change from baseline compared to placebo. The company also announced preliminary data from the MOR-005 extension study which suggests that clinical benefits continue to improve with further dosing with GALNS. Only a limited number of patients have reached the 36 or 48 week points of total time on treatment in the extension study, and the results will be updated when the study is completed. The company confirmed that based on the results of MOR-004, and following planned discussions with regulatory authorities, it expects to submit marketing applications starting in the first quarter of 2013.

The primary endpoint of the study, change in six-minute walk distance at 24 weeks, was statistically significant in patients dosed with GALNS at 2 mg/kg every week with a mean increase of 22.5 meters (p=0.0174) over placebo. In MOR-004, patients dosed at 2 mg/kg every week showed an improvement in six-minute walk distance at week 12 compared to baseline and showed continued improvement at week 24. Preliminary analysis of a subset of the patients in the MOR-005 extension study who have reached the 36 week and 48 week timepoints in the study also showed further improvement at weeks 36 and 48.

On the secondary endpoint of three-minute stair climb, patients dosed with GALNS at 2 mg/kg every week showed a trend toward improvement at
24 weeks of 1.1 additional stairs per minute over placebo. In MOR-004, patients dosed with GALNS at 2 mg/kg every week showed an improvement in three-minute stair climb performance at week 12 compared to baseline and showed continued improvement at week 24. Preliminary analysis of a subset of patients in the extension study (MOR-005) who have reached the 36 week and 48 week timepoints in the study also showed further improvement in three-minute stair climb performance.

In the other secondary endpoint, urinary keratan sulfate (KS) levels, patients dosed with GALNS at 2 mg/kg every week showed consistent and robust reduction in urinary KS with a mean difference from baseline as compared to placebo of 40.7 percent (p less than 0.0001). Preliminary analysis of a subset of patients in the extension study (MOR-005) who have reached the 36 week and 48 week timepoints in the study showed this level of reduction was maintained.

Pulmonary function, as defined by maximum voluntary ventilation (MVV) was measured at 24 weeks. In MOR-004, patients dosed with GALNS at 2 mg/kg every week showed a trend toward improvement from baseline of
10.3 percent over placebo. Preliminary analysis of the subset of patients who reached the 48 week timepoint showed a reduction in the improvement, though an increase over baseline was maintained.

Pulmonary function, as defined by forced vital capacity (FVC) was measured at 24 weeks. In MOR-004, patients dosed with GALNS at 2 mg/kg every week showed a trend toward improvement from baseline of 3.3 percent over placebo. Preliminary analysis of the subset of patients who reached the 48 week timepoint showed continued improvement.

Date: November 5, 2012
Source: BioMarin Pharmaceutical Inc.

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