A Novartis AG drug billed as a potential blockbuster failed to reduce the risk of heart attacks and strokes in a final-stage trial, marking a major setback for a new approach to prevent cardiovascular disease.
The drug, called pelacarsen, did lower levels of lipoprotein(a), a largely genetic risk factor for cardiovascular disease that affects roughly one in five people worldwide. However, that reduction didn't translate into fewer cardiovascular deaths, heart attacks and strokes compared with placebo, the Swiss drugmaker said Friday.
“These are not the results we hoped for, but they provide important evidence that advances scientific understanding of the relationship between Lp(a) lowering and cardiovascular outcomes and may help inform future approaches to cardiovascular risk management,” Novartis Chief Medical Officer Shreeram Aradhye said in the statement.
The results surprised Wall Street, with Novartis' US-listed stock falling more than 7% shortly after the end of regular trading. The American Depositary Receipts had risen 16% this year through the close. Shares in Amgen Inc., which is also developing an Lp(a) drug, fell almost 7% in postmarket trading.
Lowering Lp(a) was considered a new frontier in battling heart disease. Millions of people have elevated levels of the cholesterol-carrying particle. The condition is also inherited, meaning it's resistant to changes in diet or exercise.
However, it's not been clear whether lowering Lp(a) will lead to a reduction in major cardiovascular events. The Novartis trial was viewed as a bellwether for the drug industry, where several other companies are trying similar approaches.
Amgen and Eli Lilly & Co. are testing their own drugs to lower Lp(a), though they're further behind in development.
Citi analyst Geoffrey Meacham said Novartis' failure “increases uncertainty across the Lp(a) field.” However, he “would not declare the mechanism dead.” Full data from the trial will be essential to determine whether the failure is a result of how the drug works, the trial's design, or a true challenge to the Lp(a) hypothesis, he said.
At one point, Novartis said the market for Lp(a) drugs could be worth more than $5 billion. Analysts at UBS had projected peak annual sales of about $1.5 billion for Novartis' drug, due to competition. The company licensed the medicine from Ionis Pharmaceuticals Inc. in 2019.
Novartis tested the drug on more than 8,000 adults to find whether targeting Lp(a) could have an effect on the risk of strokes, heart attacks and cardiovascular death, known by the acronym MACE. Data from the trial will be presented at an upcoming medical meeting, the company said.
Gissette Reyes-Soffer, an associate professor at the Columbia University Irving Medical Center, cautioned against interpreting the failure as a verdict on the broader field, noting that other studies are testing different drugs and patient populations.
“I remain optimistic,” Reyes-Soffer said.
(This story has not been edited by NDTV staff and is auto-generated from a syndicated feed.)
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