Years af­ter a ma­jor tri­al set­back, No­var­tis switch­es gears with SMA drug. This time they're try­ing it for Hunt­ing­ton's

Four years af­ter a Phase I/II set­back in spinal mus­cu­lar at­ro­phy (SMA), No­var­tis is hop­ing its drug branaplam will find more suc­cess in a new neu­ro­log­i­cal in­di­ca­tion: Hunt­ing­ton’s dis­ease.

The de­ci­sion was an­nounced a year af­ter the head of re­search, Jay Brad­ner, said he did not see a “big op­por­tu­ni­ty” in SMA, ac­cord­ing to Reuters. No­var­tis says it has pre­clin­i­cal da­ta show­ing that branaplam re­duces lev­els of mu­tant hunt­ingtin pro­tein, and SMA da­ta show­ing pa­tients on the drug had re­duc­tions in hunt­ingtin mR­NA. The FDA gave branaplam their or­phan drug des­ig­na­tion, and No­var­tis plans to move forth with a Phase IIb tri­al next year.

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