Novartis reported that pelacarsen failed its final-stage trial on the outcomes that matter most. The treatment did cut levels of lipoprotein(a) - a largely inherited cardiovascular risk factor that affects about one in five people - but that shift did not translate into fewer deaths, heart attacks, or strokes compared with placebo. It is a sharp blow to the idea that lowering Lp(a) alone would prevent major cardiovascular events.
Shares of Novartis fell as much as 3.9% at the open in Zurich, the steepest drop in more than four months, though the stock remains up 14% year to date. Amgen Inc., developing a similar therapy, dropped nearly 7% in after-hours trading on Friday. "This questions whether any Lp(a)-lowering therapies can ultimately show a cardiovascular benefit," said Michael Leuchten of Jefferies, who also noted any selloff could prove overdone since even a win would have left open questions about patient selection and competitiveness.
Pelacarsen had been seen as a big swing, with peak sales projections stretching from about $1.5 billion at UBS up to $5.4 billion at Jefferies, and the bank estimated just a 30% chance of success. Novartis had characterized the total market potential as exceeding $5 billion. The company is leaning on new launches to offset upcoming patent expirations, and Chief Executive Officer Vas Narasimhan has said that a series of strong late-stage results could lead the company to lift its forecast to sales growth of 5% to 6% per year through 2030.
The setback landed only days after Novartis put studies of a cell therapy for autoimmune diseases on hold, a move that followed the deaths of three patients. Pelacarsen and rapcabtagene autoleucel (rap-cel) had each been touted as breakthrough contenders - the former aimed at a previously untreatable inherited cardiovascular risk, the latter at bringing CAR-T into autoimmune treatment. It was not all bad news last week: Novartis's multiple sclerosis pill remibrutinib cleared two late-stage trials, lifting the shares by more than 6% on Tuesday.
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Novartis shared few specifics on how close pelacarsen came to hitting its endpoints or whether particular groups did better, and said full data will come at an upcoming medical meeting. Next up is del-desiran, an investigational injection aimed at the genetic root of DM1, the muscle-wasting disease, which anchors Novartis's $12 billion Avidity deal. For your wallet, the bigger takeaway is that big pipelines mean bumpy newsflow - when one program stumbles, another can still climb the wall of worry.
