Twice thwarted in a week! Novartis (NVS.US) fails in phase III trial for blockbuster heart disease drug, stock plummets; Amgen (AMGN.US) dragged down as well

According to Golden Ten Data APP, Swiss pharmaceutical giant Novartis (NVS.US) has suffered another clinical trial setback—its second major candidate drug to fail in late-stage research within a week. This time, the loss concerns its potential blockbuster heart disease drug, pelacarsen.

Reportedly, the drug did lower levels of lipoprotein(a)—a key hereditary risk marker for cardiovascular disease affecting about one-fifth of people. However, Novartis announced late last Friday that despite decreased lipoprotein(a) levels, the drug did not significantly reduce cardiovascular deaths, heart attacks, or strokes compared to placebo.

This failure has dealt a heavy blow to industry hopes that the drug could open new pathways for cardiovascular disease prevention. Just days earlier, Novartis had temporarily halted a trial of an experimental cell therapy for autoimmune diseases following the deaths of three patients.

As a result of the news, Novartis' US-listed shares fell by more than 7% after regular trading on Friday. Shares of Amgen (AMGN.US), which develops similar heart medications, also dropped nearly 6% after hours last Friday.

Jefferies analyst Michael Leuchten noted, "This outcome raises market doubts about whether any therapy that lowers lipoprotein(a) can ultimately yield clinical cardiovascular benefits." However, he also stated that the market's negative reaction might be exaggerated, since even with a successful trial, there would still be questions about which patients would truly benefit and how the drug would compete with rivals.

Novartis is counting on a series of new drugs to support its growth as patents for several of its blockbuster products are set to expire. CEO Vas Narasimhan has said that if late-stage trial results are favorable, the company could raise its projected annual sales growth to 5%-6% before 2030.

Novartis' next highly anticipated research milestone is an experimental injectable drug aiming to treat DM1 (a type of muscular dystrophy) at the genetic level rather than merely alleviating symptoms. The therapy, called del-desiran, is a core asset acquired via Novartis’ $12 billion purchase of Avidity.

The significance of these two setbacks goes beyond individual drugs. Novartis had previously positioned both pelacarsen and rapcabtagene autoleucel (rap-cel for short) as potential breakthroughs set to reshape their respective treatment landscapes—the former targeting previously untreatable hereditary cardiovascular risk, and the latter expanding CAR-T cell therapy into the autoimmune disease space.

Notably, pelacarsen's setback came against a backdrop of mixed news for Novartis last week: at the same time, another of the company’s experimental therapies delivered on its promise, offering some consolation. Its oral multiple sclerosis drug remibrutinib succeeded in two late-stage trials, driving the company’s shares up by over 6% on Tuesday.

Novartis has yet to disclose detailed data on pelacarsen’s trial failure, including how close the drug came to meeting its primary endpoint and whether certain subgroups of patients benefitted more. The company stated that full results will be presented at an upcoming medical conference.