Io­n­is un­veils pos­i­tive PhII da­ta for in-house rare dis­ease drug, paving way to block­buster ri­val­ry with Take­da

One of the mid-stage an­ti­sense drugs for rare, non-neu­ro­log­i­cal dis­eases that Brett Mo­nia boast­ed about at JP Mor­gan ear­li­er this year has de­liv­ered a sol­id slate of pos­i­tive re­sults, boost­ing the CEO’s promise to turn Io­n­is in­to one of the biggest RNA biotechs with its whol­ly-owned pipeline.

With this par­tic­u­lar can­di­date, he may even be set­ting the com­pa­ny up for a ri­val­ry with Take­da.

The tri­al, which tests IO­N­IS-PKK-LRx in hered­i­tary an­gioede­ma, is small with 20 pa­tients. But the re­sults were un­equiv­o­cal: When you com­pare the 14 adults who re­ceived an 80mg month­ly dose of the drug to the six who got place­bo, you see a mean re­duc­tion of 90% in the num­ber of month­ly HAE at­tacks through weeks 1 to 17 in the study, meet­ing the pri­ma­ry end­point (p <0.001). From weeks five through 17 (one of the sec­ondary end­points), the mean re­duc­tion in month­ly at­tacks rose to 97% (p=0.003).

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