Cholesterol: Pharmaceutical Companies Bet on ‘s Little-Known Form, Lp(a)
Pharmaceutical companies are betting on a little-known form of cholesterol, Lp(a), to develop the next blockbuster heart drugs. For years, the focus in cardiology has been on lowering LDL cholesterol levels, which is often referred to as “bad” cholesterol. But recent discoveries have shifted this perspective, highlighting Lp(a) as a significant risk factor for heart attacks.
Discovered in 1963, Lp(a) has flown under the radar for decades. Yet, studies show that people with high levels of Lp(a) face more than double the risk of heart attack compared to those without elevated levels. Alarmingly, an estimated one in five people worldwide have elevated Lp(a), making it a pressing concern for public health.
Now, major pharmaceutical players like Novartis, Amgen, and Eli Lilly are racing against time. They are currently in late-stage trials testing drugs designed to lower Lp(a). Novartis’ experimental drug, pelacarsen, aims to cut Lp(a) levels significantly. In fact, these companies have demonstrated that their drugs can slash Lp(a) by more than 80%—a remarkable achievement that could reshape treatment protocols.
Yet, there’s still uncertainty surrounding this development. Less than 1% of adults in the U.S. were tested for Lp(a) in 2024. Leading cardiology organizations recommend that every adult should undergo testing at least once in their lifetime. So why aren’t more people getting tested? The exact amount of Lp(a) that needs to be lowered to prevent heart attacks remains unclear.
Dr. Steve Nissen emphasizes the importance of keeping an open mind: “We thought raising HDL would be beneficial and that didn’t work, so I think we have to keep an open mind.” His words resonate as researchers explore new avenues in gene editing and PCSK9 inhibitors to combat cardiovascular diseases.
Jay Bradner notes the significance of this shift: “The clarity of the signal from population genetics and the encouraging signs from earlier trials render this a very smart bet.” As these trials progress, the stakes are high—not just for pharmaceutical companies but for millions at risk of heart disease.
The potential market for Lp(a)-targeted therapies is enormous; analysts predict annual sales could reach $5.6 billion by 2032. However, with Amgen’s pivotal trial results delayed, uncertainty looms over how effective these treatments will ultimately be.
Asad Haider points out the importance of Novartis’ trial: “That’s why this Novartis trial is going to be so important in how people think about the unlock.” The outcome could provide crucial insights into managing heart health moving forward—an essential goal given that heart disease continues to be a leading cause of death worldwide.
