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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