Ionis Pharmaceuticals has reported positive topline results from the ESSENCE study evaluating olezarsen in individuals with moderate hypertriglyceridemia who are at risk of atherosclerotic cardiovascular disease (ASCVD).
The study met its primary endpoint, demonstrating a statistically significant, placebo-adjusted reduction in triglyceride levels of 61% and 58% at six months with 80mg and 50mg monthly doses respectively (p < 0.0001). Olezarsen also met all key secondary endpoints, with the majority of participants achieving triglyceride levels below 150mg/dL, bringing them into the normal range. Olezarsen was well tolerated, with a favourable safety profile consistent with previous studies.
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Olezarsen is an antisense oligonucleotide (ASO) designed to selectively inhibit the expression of the APOC3 gene. By decreasing the production of apolipoprotein C-III (ApoC-III) — a protein that hinders triglyceride metabolism — it promotes the efficient breakdown and clearance of triglyceride-rich lipoproteins. This leads to a marked reduction in plasma triglyceride levels, positioning olezarsen as a promising treatment for severe hypertriglyceridemia, familial chylomicronemia syndrome (FCS) and other dyslipidemia.
Urgent need for new options to lower triglyceride levels
Key opinion leaders (KOLs) interviewed by GlobalData emphasised a significant unmet need for effective treatments targeting rare genetic lipid disorders, particularly FCS and familial hypercholesterolemia. Notably, there are currently no FDA-approved therapies for FCS, underscoring the urgency for new options that can lower triglyceride levels and mitigate associated complications. With its targeted mechanism and encouraging data, olezarsen has the potential to address this therapeutic gap. According to GlobalData, the drug is projected to achieve global sales of around $849 million by 2032.
Pivotal Phase III results from the ongoing CORE and CORE2 trials, which are evaluating olezarsen for the treatment of severe hypertriglyceridemia (sHTG), are expected in the third quarter of 2025. This data is highly anticipated and will be crucial in determining the drug’s efficacy and safety profile. Positive results could pave the way for regulatory approval and establish olezarsen as a first-in-class treatment option for patients with limited alternatives.
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