BioMarin lays out its PhIII strategy for its closely-watched gene therapy for hemophilia A
When it comes to gene therapy studies, anything other than a perfect score always raises a red flag about their future in late-stage testing. And nobody gets a perfect score.
BioMarin was reminded of that lesson again today as it carefully laid out the latest promising early-stage data from a handful of hemophilia A patients who received their gene therapy BMN 270.
Their key data were good to great. Excluding one of the patients in the high dose group, leaving 6 for analysis, the mean annualized bleeding rate was reduced by 97% — from 16.3 to 0.5. “The median ABR for those same patients was reduced from 16.5 to zero. The mean annualized Factor VIII infusions were reduced by 94% from 136.7 to 8.5. The median annualized Factor VIII infusions were reduced from 138.5 to zero.” And the lower, mid-range dose also demonstrated an excellent score on bleeding and infusion rates, with a drop to zero.
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