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.

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.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER