Eli Lilly’s lepodisiran, an experimental drug targeting high levels of lipoprotein(a), or Lp(a), has shown remarkable results in reducing this cholesterol component that can increase the risk of cardiovascular disease. The Phase 2 trial data published over the weekend found a 94% drop in Lp(a) levels among participants with a genetic predisposition to high Lp(a).
Lp(a) is a type of LDL cholesterol that is largely determined by genetics and not lifestyle, affecting approximately one in five people worldwide. Currently, there are no existing treatments to reduce Lp(a). Lepodisiran works by inhibiting the liver’s production of apolipoprotein(a), a key component of Lp(a).
The Phase 2 ALPACA trial involved 320 participants with high Lp(a) who received varying doses of lepodisiran. The results showed that everyone receiving lepodisiran experienced a reduction in Lp(a) levels compared to the placebo group. Those on the highest dose saw a significant 94% drop in Lp(a), while those given two injections had a 95% reduction.
The findings indicate that lepodisiran could become the first treatment for this common genetic risk factor. Although Phase 2 trials are primarily used to assess safety and optimal dosing, the results have sparked excitement among experts. Eli Lilly has already enrolled volunteers for its Phase 3 trial of lepodisiran, which could lead to a new breakthrough in siRNA-based therapies.
Outside experts praise the drug’s potential to nearly eliminate Lp(a), a significant risk factor for cardiovascular disease. The approval of Leqvio in 2021 as an LDL-lowering treatment for genetic conditions or poor atherosclerosis is also notable.
Source: https://gizmodo.com/millions-share-a-deadly-genetic-heart-risk-this-experimental-drug-could-be-a-game-changer-2000582742