Buoyed by he­mo­phil­ia A gene ther­a­py up­date, Bio­Marin switch­es to head-to-head study -- but shares slide on dura­bil­i­ty con­cerns

With its he­mo­phil­ia A gene ther­a­py poised to go through a piv­otal pro­gram by the end of next year, Bio­Marin $BM­RN set out to boost hopes for its suc­cess with a new batch of Phase I/II da­ta for its val­rox pro­gram (val­oc­toco­gene rox­a­parvovec/BMN 270). But the biotech’s shares turned wob­bly and start­ed to slide on con­cerns that the new num­bers may have re­vealed some longterm weak­ness on ef­fi­ca­cy.

Re­searchers at a con­fer­ence in Glas­gow are point­ing to one of the dosage arms where they tracked a 97% cut in an­nu­al­ized bleed­ing rate. And the biotech now plans to boost the num­ber of pa­tients in a reg­is­tra­tion study so they can pro­vide clear ev­i­dence of the gene ther­a­py’s abil­i­ty to beat the cur­rent stan­dard of care us­ing fac­tor re­place­ment ther­a­py.

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