RNAi trail­blaz­er Al­ny­lam teas­es more pos­i­tive da­ta for a po­ten­tial third com­mer­cial drug

RNAi pi­o­neer Al­ny­lam snagged its first and sec­ond FDA ap­provals in the last two years. And on Wednes­day, the Cam­bridge, MA-based biotech an­nounced it has more pos­i­tive da­ta to back its next po­ten­tial com­mer­cial drug.

Al­ny­lam said lumasir­an per­formed well in a Phase III study, dubbed IL­LU­MI­NATE-B, in 18 chil­dren un­der 6 years old with pri­ma­ry hy­per­ox­aluria type 1 (PH1). While we’ll have to wait for vir­tu­al ASN in Oc­to­ber to get the full da­ta, the com­pa­ny said topline re­sults showed a “clin­i­cal­ly mean­ing­ful re­duc­tion” in spot uri­nary ox­alate:cre­a­ti­nine ra­tio — the pri­ma­ry end­point. Lumasir­an al­so did well across sec­ondary end­points, in­clud­ing mea­sures of uri­nary and plas­ma ox­alate, ac­cord­ing to the com­pa­ny.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.