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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