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BioMarin Announces Positive Results From Phase 2A Clinical Study of 6R-BH4 in Sickle Cell Disease

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 function.

-- 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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