Novartis Heart Disease Drug Fails Phase 3 Trial, Amgen's Similar Drug Drops 7%
nashnova research
Novartis's blockbuster hopeful pelacarsen missed its primary endpoint in a Phase III trial — lowering Lp(a) did not reduce cardiovascular events. Amgen, developing a similar Lp(a)-targeting drug, fell nearly 7% after hours, putting the entire Lp(a) thesis in doubt.
Where exactly did the drug fail?
Pelacarsen did lower lipoprotein(a) — Lp(a), a largely genetic risk factor for cardiovascular disease that affects roughly one in five people — but the reduction did not translate into fewer heart attacks, strokes, or cardiovascular deaths versus placebo.
In plain terms = the biomarker moved, yet patients were no better off — pharmacological "activity" and clinical "benefit" turned out to be two different things.
This means → lowering Lp(a) alone may not be enough to cut cardiovascular risk, calling into question the therapeutic logic behind the entire Lp(a) target class.
Why did the market react so sharply?
Novartis U.S.-listed shares fell more than 7% after hours; Monday pre-market they traded 4.2% below Friday's Zurich close.
Amgen dropped nearly 7% after hours — Amgen is developing a mechanistically similar Lp(a)-targeting cardiac drug, and Novartis's failure undercuts the core assumption behind Amgen's own pipeline.
This reflects a market that is not pricing the failure of one drug but repricing the viability risk of the entire Lp(a) track.
What do analysts say?
Jefferies analyst Michael Leuchten said the failure "raises questions about whether any Lp(a)-lowering therapy can ultimately demonstrate cardiovascular benefit."
He added that the sell-off may be overdone — even a successful trial would have faced questions about which patients benefit and how competitive the drug could be.
This means → the failure is real, but the stock drop likely includes a repricing of uncertainty that already existed around the bull case.
What else went wrong for Novartis this week?
This is Novartis's second major pipeline setback in a single week: days earlier, the company paused its experimental autoimmune cell therapy rap-cel after three patient deaths.
Novartis had positioned both pelacarsen and rap-cel as "paradigm changers" — one targeting a previously unaddressable genetic cardiovascular risk, the other bringing CAR-T cell therapy — a technique that reengineers a patient's own immune cells to fight disease — into autoimmune disorders.
In plain terms = Novartis bet on two "game-changing" cards, and both hit trouble in the same week.
What comes next?
Novartis has not disclosed how far pelacarsen fell short, nor whether any patient subgroup showed benefit; full data will be presented at a future medical conference.
The next key catalyst is del-desiran — an injectable drug targeting the genetic cause of myotonic dystrophy type 1 (DM1) and the centrepiece of Novartis's $12 billion Avidity acquisition.
This means → del-desiran's readout becomes the market's pivotal test for re-rating Novartis's pipeline; CEO Vas Narasimhan has tied the company's forecast of 5%–6% annual sales growth through 2030 directly to late-stage trial outcomes.
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