Bris­tol My­ers pulls lym­phoma in­di­ca­tion for Is­to­dax af­ter con­fir­ma­to­ry tri­al falls flat

Amid an in­dus­try­wide re­view of can­cer drugs with ac­cel­er­at­ed ap­proval, Bris­tol My­ers Squibb had to make the tough call last month to yank an ap­proval for lead­ing I/O drug Op­di­vo af­ter flop­ping a con­fir­ma­to­ry study. Now, a sec­ond Bris­tol My­ers drug is on the chop­ping block.

Bris­tol My­ers has pulled ag­ing HDAC in­hibitor Is­to­dax’s in­di­ca­tion in pe­riph­er­al T cell lym­phoma af­ter a Phase III con­fir­ma­to­ry study for the drug flopped on its pro­gres­sion-free sur­vival end­point, the drug­mak­er said Mon­day.

Is­to­dax, a Cel­gene drug first ap­proved way back in 2009, earned its ac­cel­er­at­ed ap­proval in PT­CL, a form of non-Hodgkin lym­phoma, in 2011, but it took near­ly a decade for the con­fir­ma­to­ry re­sults to ges­tate. At ASH 2020, Bris­tol My­ers rolled out da­ta show­ing Is­to­dax couldn’t ben­e­fit clin­i­cal out­comes when added to a reg­i­men of cy­clophos­phamide, dox­oru­bicin, vin­cristine, and pred­nisone (CHOP).

So late in its life, Is­to­dax is a mi­cro-earn­er for Bris­tol My­ers so pulling one of its two in­di­ca­tions won’t make much of a dif­fer­ence in terms of the drug­mak­er’s topline. But the re­sults are no­table as part of an in­dus­try­wide reck­on­ing for can­cer drugs that earn the FDA’s ac­cel­er­at­ed nod and even­tu­al­ly flop con­fir­ma­to­ry stud­ies.

Just last week, Bris­tol My­ers pulled its US in­di­ca­tion for check­point in­hibitor Op­di­vo as a monother­a­py for he­pa­to­cel­lu­lar car­ci­no­ma in pa­tients pre­vi­ous­ly treat­ed with so­rafenib af­ter an FDA On­co­log­ic Drugs Ad­vi­so­ry Com­mit­tee rec­om­mend­ed pulling the nod.

Op­di­vo won an ac­cel­er­at­ed ap­proval as a sec­ond-line treat­ment for HCC back in 2017, be­com­ing the first im­munother­a­py to get OK’d for this in­di­ca­tion. But two years lat­er, a con­fir­ma­to­ry study didn’t meet sta­tis­ti­cal sig­nif­i­cance in its pri­ma­ry end­point of over­all sur­vival. Last March, the drug land­ed an­oth­er, sep­a­rate ac­cel­er­at­ed ap­proval in com­bi­na­tion with Yer­voy for the same set­ting.

Op­di­vo in monother­a­py HCC was one of two in­di­ca­tions ODAC vot­ed against out of a six-in­di­ca­tion re­view. The oth­er in­di­ca­tion re­ceiv­ing a no-vote was Mer­ck’s Keytru­da as a third-line treat­ment for stom­ach can­cer, which Mer­ck opt­ed to with­draw ear­li­er in Ju­ly.

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