BioMarin eyeing pivotal hemophilia gene therapy trial

In updated results from its Phase I/II study of gene therapy BMN 270 to treat severe hemophilia A, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) said Monday that the seven patients treated with the study's highest dose have consistently had median Factor VIII levels with the normal range from weeks 20-44 of treatment. The company said that in 3Q17, it plans to start a "potentially registration enabling" Phase IIb study of the candidate.

Among six patients who received the highest dose and were previously on prophylactic Factor VIII therapy, mean annualized bleeding rates fell 91%, and mean annualized Factor VIII infusions fell 98%. All patients have ceased using steroid therapy with no lasting effect on Factor VIII expression or alanine aminotransferase (ALT) levels...