Al­ny­lam's up­dat­ed lumasir­an da­ta points to PhI­II short track; Genen­tech snatch­es fourth ap­proval for Rit­ux­an

→ Al­ny­lam $AL­NY came out with some up­dat­ed da­ta from a Phase I/II study Fri­day, show­ing the com­pa­ny’s rare dis­ease drug lumasir­an is on the short track for a Phase III tri­al. The tri­al — test­ing the drug in 20 pa­tients with the ul­tra-rare pri­ma­ry hy­per­ox­aluria type 1 (PH1) — has a pri­ma­ry end­point of re­duc­ing the uri­nary ox­alate at six months. Pre­lim­i­nary re­sults, an­nounced last year, sug­gest­ed that lumasir­an led to a mean max­i­mal re­duc­tion in uri­nary ox­alate of 66% in an un­blind­ed group of four pa­tients. In this Part B study, lumasir­an demon­strat­ed a mean max­i­mal re­duc­tion in uri­nary ox­alate of 64% in pa­tients. “Based up­on our re­cent dis­cus­sions with the FDA, we are on track to ad­vance this pro­gram in­to Phase III de­vel­op­ment at mid-year, with the goal of bring­ing lumasir­an to pa­tients around the world as rapid­ly as pos­si­ble,” said Al­ny­lam’s VP and GM of the lumasir­an pro­gram Pritesh Gand­hi in a state­ment.

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