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ARTICLE | Clinical News

BMN 270 improves Factor VIII levels in Phase I/II for hemophilia A

August 4, 2017 7:51 PM UTC

Next quarter, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) said it plans to start a pair of Phase III trials evaluating 4x1013 and 6x1013 vg/kg dose levels of BMN 270, an adeno-associated viral (AAV) vector delivering the Factor VIII gene, to treat severe hemophilia A. The company released its plans along with additional data from an open-label, U.K. Phase I/II trial showing that a single IV infusion of 4x1013 vg/kg BMN 270 led to a mean Factor VIII level of 51% at week 32 in 3 patients with severe hemophilia A.

BioMarin also said 4x1013 vg/kg BMN 270 led to a mean Factor VIII activity level of 31% at week 20 in 6 patients with severe hemophilia A who received Factor VIII prophylaxis before the study. The 4x1013 vg/kg dose of BMN 270 led to a mean annualized bleed rate (ABR) of 1 bleeding episode and a mean annualized Factor VIII infusion rate of 4.8 infusions compared to 12.2 bleeding episodes and 144.2 infusions, respectively at baseline...

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