Two big heart-drug trials miss, despite strong biological rationale

Novartis's pelacarsen and a Novo Nordisk IL-6 drug both failed to cut heart attacks and strokes, even with genetics pointing the way.

Two studies presented Saturday at the American Heart Association annual meeting came up empty. Novartis's pelacarsen lowered lp(a), a blood substance that genetics suggested should reduce cardiovascular risk when pushed down, but it showed no effect on the rate of heart attacks and strokes. A large Novo Nordisk study of an anti-inflammatory drug targeting the protein IL-6 also showed no benefit.

STAT senior writer Matthew Herper uses the results to argue that new technologies have repeatedly failed to speed medicines to patients in heart disease, the field behind the largest killer in the developed world. The piece says better genetic understanding was expected to raise the odds of success and did not. Its title frames this as a reason for more skepticism about AI hype.

Why it matters for abundance: cheaper, faster drug discovery is only worth as much as the odds that a promising target works in people. These trials show a target can look right on genetic and biomarker evidence and still fail at the outcome that counts. If so, the bottleneck is large outcome trials, not just finding candidates, and tools that generate more candidates will not by themselves make treatments cheaper or quicker to reach patients.

Worth watching: STAT's related coverage says cardiologists still plan to pursue anti-inflammatory approaches despite Novo's failure, and a separate item reports Amgen's Repatha was linked to fewer heart attacks. Caveat: only the opening of the article was available to us, behind the STAT+ paywall. Its specific argument about AI, and the trial sizes and effect figures, are not visible to us, so read STAT's full piece for those.

Source: STAT