BioMarin's (BMRN) Gene Therapy Enters First Phase III Study
Zacks Investment Research | Dec 19, 2017 09:48PM ET
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced initiation of the first phase III study, evaluating the 6e13 vg/kg dose of its investigational gene therapy, valoctocogene roxaparvovec, for treatment of patients with severe hemophilia A. The company plans to enroll the first patient in the second phase III study with the 4e13 vg/kg dose at the beginning of 2018 for the given indication.
The two phase III studies, GENEr8-1 and GENEr8-2, were designed to evaluate the efficacy and safety of valoctocogene roxaparvovec in patients with severe hemophilia A across the two doses mentioned above. While the primary endpoint in both the studies will be factor VIII (a blood clotting protein) activity level achieved following administration of valoctocogene roxaparvovec, the secondary endpoints will measure annualized factor VIII replacement therapy use rate and annualized bleed rate.
BioMarin’s share price has gained 9.7% in the last 12 months, comparing favorably with the Zacks Investment Research
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