Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Al­most two years af­ter Take­da wa­gered $300 mil­lion cash to part­ner with Ar­row­head on an RNAi ther­a­py for a rare dis­ease, the com­pa­nies are spelling out Phase II da­ta that they be­lieve put them one step clos­er to their big dreams.

In a small, open la­bel study in­volv­ing on­ly 16 pa­tients who had liv­er dis­ease as­so­ci­at­ed with al­pha-1 an­tit­rypsin de­fi­cien­cy (AATD), Ar­row­head’s can­di­date — fazir­sir­an, pre­vi­ous­ly ARO-AAT — spurred sub­stan­tial re­duc­tions in ac­cu­mu­lat­ed mu­tant AAT pro­tein in the liv­er, a hall­mark of the con­di­tion. In­ves­ti­ga­tors al­so tracked im­prove­ments in symp­toms, with sev­en out of 12 who re­ceived the high, 200 mg dose see­ing re­gres­sion of liv­er fi­bro­sis.

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