Ionis, Novartis' key experimental heart drug fails in late-stage trial

Sept 4 — Ionis Pharmaceuticals and partner Novartis' experimental drug to lower high levels of a ​cholesterol-carrying particle in heart patients failed in a late-stage trial, the companies said on Friday, sending Ionis' shares down 12% in aftermarket trading.

The ‌drug, pelacarsen, did not meet the main goal of reducing the risk of events such as ⁠death from heart disease, heart attack, ​stroke, or needing an urgent procedure ⁠to restore blood flow to the heart, when compared with placebo.

Consistent with previous ‌studies, pelacarsen lowered ‌levels of the cholesterol-carrying particle lipoprotein(a) in patients, the companies said.

Lipoprotein(a) ⁠is a type of lipid, or fat, in ⁠the body that contains and is similar in structure to low-density lipoprotein (LDL) cholesterol, often thought of as "bad cholesterol." Increased Lp(a) levels have been linked to a higher risk for cardiovascular disease.

"Although pelacarsen substantially lowered lipoprotein(a) levels consistent with previous studies, we are disappointed that this did not ‌translate to cardiovascular risk reduction," said Ionis CEO ​Brett Monia.

There are currently no approved treatments specifically designed to lower lipoprotein(a). 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 fell 5% in aftermarket trading after the trial results. Last month J.P.Morgan analysts noted that a trial win for pelacarsen would have a positive read through for ​Amgen's olpasiran.

Investors have been on the lookout for data from Novartis' three experimental drugs - ‌pelacarsen, multiple sclerosis drug ‌remibrutinib ⁠and genetic therapy del-desiran - which together represent more than $10 billion in potential peak annual sales and are seen as drivers of the Swiss drugmaker's long-term growth.

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.

(Reporting by Christy Santhosh in Bengaluru; Editing by ​Shailesh Kuber)

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