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BioMarin Provides Update on GALNS Phase 1/2 Extension Study (MOR-100)
Date:7/28/2011

Lysosomal Storage Disease conference in February 2012.  

Highlights of MOR-100 Extension Study:

  • No new or consistent patterns of clinically meaningful or treatment limiting adverse events were reported.  
  • Measures of 6-minute walk distance, 3-minute stair climb and pulmonary function all generally improved and were sustained for at least 2 years.
  • For the group of patients with baseline 6-minute walk distance <325 meters, mean walk distance remains consistently improved for the entire period of observation.  Specifically, 24 weeks after resuming 2.0 mg/kg/week dosing in MOR-100, the mean 6-minute walk distance was 38 meters.  This is roughly equivalent to that observed after the first 24 weeks of the original Phase 1/2 trial (MOR-002).  The ongoing Phase 3 study is using mean improvement in 6-minute walk distance in patients with baseline walk distance <325 meters as the primary endpoint.  
  • 6-minute walk distance improvements were greater in the subgroup of patients with baseline walk distance <325 meters.  However, variability of this test increases with longer-term follow-up, likely as a result of issues related to the progressive nature of Morquio syndrome. Several measures to reduce variability have already been incorporated in the ongoing Phase 3 trial: large sample size, selection of patients not expected to require surgical correction of skeletal deformity in the subsequent 24 weeks, duplicate measures of 6-minute walk test, conclusion of the trial at 24 weeks, extensive oversight of the performance of the walk test according to ATS criteria and inclusion of placebo for comparative purposes.
  • 3-minute stair climb and pulmonary function have consistently improved over the entire duration of the study.  24 weeks after returning to 2.0 mg/kg, patients can now climb a mean of 13.6 stairs per minute and FVC has improved a mean of 15.9%, compared to 6.9 stairs per m
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  • SOURCE BioMarin Pharmaceutical Inc.
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