Prime Highlights:
- Eli Lilly’s new experimental drug lepodisiran lowered lipoprotein(a) (Lp(a)) by 93.9% at six months in a mid-stage trial.
- No significant side effects, so it could be a game-changer for inherited risk of heart disease.
Key Facts:
- 141 patients received 400 mg of lepodisiran and 69 patients received a placebo treatment.
- Results were published at the American College of Cardiology meeting and in the New England Journal of Medicine.
Key Background:
Lipoprotein(a) or Lp(a) is an inherited risk factor that exists in approximately 1.4 billion people around the globe, 64 million of whom live within the United States. High Lp(a) is distinct from cholesterol, which may be controlled through diet and statins. There are no medications for high Lp(a). Elevated Lp(a) has been found to increase a person’s risk for heart attacks, strokes, and aortic valve disease.
Eli Lilly’s lepodisiran has also been a rising treatment option in the same class. A mid-phase trial revealed a 93.9% reduction of Lp(a) following once-dose 400 mg at six months. Notably, no significant adverse effect was observed, contributing to its safety profile. It has been dubbed a breakthrough in light of continued lack of targeted treatment in the scenario of Lp(a)-mediated cardiovascular risk.
The findings were reported at the American College of Cardiology meeting and in the New England Journal of Medicine. Dr. Steven Nissen, lead author and Cleveland Clinic staff member, noted lepodisiran is poised to meet this unmet need with low frequency dosing.
Lilly does have lepodisiran now in late-stage clinical trials to determine if it can prevent cardiovascular events. Although the drug does definitely lower Lp(a) levels significantly, only larger trials will reveal whether that indeed translates into fewer strokes and heart attacks.
Other pharma competitors are also in the pipeline for Lp(a)-targeting therapies. Competitors include Silence Therapeutics’ zerlasiran, Amgen’s olpasiran, and Novartis’ pelacarsen. Lilly also has muvalaplin in clinical development and is the sole oral drug in development for this class.
Manufacturing of such drug such as lepodisiran is most relevant to those who are at high genetic risk, especially in individuals of greater frequency for Lp(a) such as individuals of African origin.
