Fac­tor VI­II lev­els con­tin­ue to drop in Bio­Marin’s 3-year up­date on he­mo­phil­ia A gene ther­a­py — but wait...

Bio­Marin dropped their 3-year Phase II up­date for their he­mo­phil­ia A gene ther­a­py val­rox Tues­day morn­ing, beat­ing their ex­pect­ed dead­line in ear­ly June and draw­ing some rapt at­ten­tion from an­a­lysts ea­ger­ly await­ing a new set of da­ta on dura­bil­i­ty. And then, just as the da­ta were sink­ing in, the biotech threw in a curve ball, of­fer­ing a snap­shot of Phase III da­ta they will be tak­ing to the FDA soon in search of an ac­cel­er­at­ed ap­proval.

It’s com­pli­cat­ed and more than a bit cloudy. But let’s start at the top and work our way down.

As ex­pect­ed, Bio­Marin con­cen­trat­ed pri­mar­i­ly on the big over­all drop in bleed rates over the 3-year span, while Wall Street paid more at­ten­tion to the ther­a­py’s po­ten­tial for dura­bil­i­ty, which is all about fac­tor VI­II. And those lev­els con­tin­ued to fall.

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