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BioMarin Provides Update on GALNS Phase 1/2 Extension Study (MOR-100)

inute and 1.5% reported at the end of the first 24 weeks of the Phase 1/2 trial.  Stair climb and pulmonary function are secondary and tertiary endpoints, respectively, in the ongoing prospective Phase 3 trial.
  • The data from the extension study to date support 6-minute walk distance as the primary endpoint and patients with baseline walk distance <325 meters as an entry criteria in the ongoing GALNS Phase 3 trial, given the large and durable effect of the drug on these measures in this population.

  • Simon Jones, Consultant in Paediatric Metabolic Medicine, St. Mary's Hospital, Manchester UK commented, "I'm pleased with the outcome of the GALNS program to date.  The longer-term data look good with sustained improvements in endurance and pulmonary function.  Patients feel stronger and function better than I expected.  All in all, we look forward to the results of the Phase 3 study as it is well designed and will, if successful, support introduction of an important product to patients in need."

    Hank Fuchs, M.D., Chief Medical Officer of BioMarin added, "It's remarkable that improvements in endurance are sustained in spite of the severe and progressive nature of Morquio syndrome.  Patients with Morquio syndrome can miss therapy, for example, due to the need for corrective surgery.  In spite of that, resumption of therapy returns patients to their original state of health or better."Table 1 – Summary of Efficacy Results Through Week 24 of MOR-100 Extension Study Statistic


    Week 24


    Week 36


    Week 0


    Week 12


    Week 24Dose

    1.0 mg/kg

    2.0 mg/kg

    1.0 mg/kg for previous 36-48 weeks, Begin 2.0 mg/kg

    2.0 mg/kg

    2.0 mg/kgENDURANCE6 Minute Walk Test – change from Baseline (meters)



    (Std. Dev.)





    SOURCE BioMarin Pharmaceutical Inc.
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