Novartis Excited About Iptacopan But Downcast After Huntington’s Blow

Novartis AG's pipeline update during its fourth-quarter results presentations was a mixed bag, with the company's enthusiasm for iptacopan tempered by the disappointing news that it has terminated development of branaplam for Huntington’s disease.

Hopes were high a couple of years ago when Novartis announced that after working on branaplam for over five years for type 1 spinal muscular atrophy (SMA), it was switching its development efforts for the once-weekly, orally administered, small-molecule RNA splicing modulator to Huntington's. The strategy shift was made in part as a result of the options that had become available for SMA, namely Biogen, Inc