Novartis, Ionis' experimental heart drug misses key goal in closely watched trial

Citi analyst Geoff Meacham said full trial data are needed to determine whether the miss reflects pelacarsen's mechanism of action, inadequate Lp(a) reduction, trial design, or doubts that lowering Lp(a) reduces heart disease risk.

There are currently no approved treatments specifically designed to lower Lp(a), which affects 20% of people worldwide.

Other experimental Lp(a)-lowering drugs in development include Amgen's olpasiran and Eli Lilly's lepodisiran, both of which are in late-stage development. Amgen shares also fell 5% in aftermarket trading.

Ionis discovered and conducted the early development of pelacarsen. In 2019, Novartis exercised an option to license the rights to develop and commercialize pelacarsen for cardiovascular therapy.

The companies said full trial data will be presented at an upcoming medical congress.