Subjects Show Improvement in Endothelial Dysfunction Conference Call and Webcast to Be Held Today at 5:00 p.m. ET (22:00 CET)
NOVATO, Calif., Oct. 15 /PRNewswire-FirstCall/ -- BioMarin
Pharmaceutical Inc. (Nasdaq: BMRN) today announced results from its Phase
2a multi-center, open-label, dose-escalation clinical study of 6R-BH4 in
patients with sickle cell disease (SCD) designed to evaluate whether 6R-BH4
can improve the endothelial dysfunction observed in SCD patients. Oral
administration of 6R-BH4 was associated with an improvement in endothelial
dysfunction in sickle cell disease patients.
Key findings from the study:
-- Endothelial dysfunction, measured using the EndoPAT device to assess
peripheral arterial tonometry (PAT), was common in the sickle cell
disease patient population. At baseline, 56% of the patients in the
trial had endothelial dysfunction (PAT score less than or equal to
1.67), consistent with prior studies using other methods for measuring
-- Endothelial dysfunction in SCD patients treated with escalating doses
of 6R-BH4 showed improvement at week 8 (5 mg/kg; p=0.042), week 12
(10mg/kg; p=0.003) and week 16 (20 mg/kg; p=0.075).
-- SCD patients with an abnormal PAT scores of less than or equal to
1.67 at baseline demonstrated greater improvement at all dose
levels (2.5, 5, 10 and 20 mg/kg).
-- The mean endothelial function PAT score improved from the abnormal to
the normal range (>1.67) in SCD patients with an abnormal PAT score at
-- Improvement in endothelial dysfunction appeared to be dose-dependent.
-- 6R-BH4 was well-to
|SOURCE BioMarin Pharmaceutical Inc.|
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