Trial results for Inclisiran (KJX839) in participants with hypercholesterolemia were posted on ClinicalTrials.gov on 2026-04-14. The study demonstrated that Inclisiran led to a -35.14% greater reduction in LDL-C levels compared to placebo (p=0.0001) and significantly more participants achieving LDL-C targets. Additionally, Inclisiran was associated with fewer muscle-related adverse events.
Background
The study, titled "Study of Efficacy, Safety, Tolerability and Quality of Life of Inclisiran (KJX839) vs Placebo, on Top of Ongoing Individually Optimized Lipid-lowering Therapy, in Participants With Hypercholesterolemia," aimed to evaluate inclisiran in individuals with hypercholesterolemia.
Trial design
The study (NCT05192941) was a Phase 4, randomized, placebo-controlled trial that enrolled 1770 participants with hypercholesterolemia. Participants were randomly assigned to receive either inclisiran sodium or placebo, on top of ongoing individually optimized lipid-lowering therapy. The study investigated the efficacy, safety, tolerability, and quality of life of inclisiran.
Key results
The trial results demonstrated significant differences between the inclisiran and placebo groups across several key outcomes:
- For the outcome "Number of Participants Achieving Individual LDL-C Target (<55 mg/dL or <70 mg/dL)":
- 752 participants in the inclisiran group achieved the target.
- 266 participants in the placebo group achieved the target.
- For the outcome "Relative Change From Baseline to Mean LDL-C Level Over the Double-blind Treatment Period":
- The inclisiran group showed a least squares mean change of -59.45%.
- The placebo group showed a least squares mean change of -24.31%.
- For the outcome "Number of Participants Experiencing at Least One Muscle-related Adverse Event From Day 1 to Day 360":
- 107 participants in the inclisiran group experienced a muscle-related adverse event.
- 167 participants in the placebo group experienced a muscle-related adverse event.
Several measurements related to "Number of Participants Experiencing Self-reported Pain" were also recorded. For these outcomes, the number of participants in the inclisiran group ranged from 45 to 189, while in the placebo group, it ranged from 71 to 177. Associated analyses included a Negative binomial model (Ratio of rates: 0.93, 95.0% CI: 0.81 to 1.06, p-value: 0.1349) and two Mixed Models Analyses (least squares mean differences of -0.11, p-values: 0.0389 and 0.0285).
What this means
The results from this Phase 4 trial indicate that inclisiran significantly improved LDL-C target achievement and reduced LDL-C levels from baseline compared to placebo in participants with hypercholesterolemia. The substantial difference in LDL-C reduction and the high odds ratio for achieving LDL-C targets suggest that inclisiran is an effective treatment for managing hypercholesterolemia. Furthermore, the lower incidence of muscle-related adverse events with inclisiran compared to placebo is a notable safety finding. These data provide important insights for clinicians managing patients with hypercholesterolemia, especially concerning lipid-lowering efficacy and muscle-related side effect profiles.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT05192941, titled "Study of Efficacy, Safety, Tolerability and Quality of Life of Inclisiran (KJX839) vs Placebo, on Top of Ongoing Individually Optimized Lipid-lowering Therapy, in Participants With Hypercholesterolemia," were posted on 2026-04-14 on clinicaltrials.gov.