Rona Therapeutics Announces Phase 1 Clinical Data of RN0361, a Long-Acting ApoC3-Targeting siRNA Therapy Demonstrating Sustained Lipid-Lowering for at Least Six Months

November 10,Rona Therapeutics（Rona Therapeutics) announcedAt the American Heart Association Annual MeetingOrally reported itsLong-acting Targeted ApoC3 siRNA DrugPhase 1 Clinical Study Results of RN0361.Results from the first-in-human study showed that the product achieved significant and sustained reductions in ApoC3 and triglycerides, with effects lasting at least six months.。

This is a randomized, placebo-controlled, single ascending dose study enrolling subjects with baseline triglyceride levels exceeding 80 mg/dL. The results showed,RN0361 demonstrated a significant dose-dependent inhibitory effect on ApoC3 and TG, lasting until 180 days post-administration, with a maximum reduction of 93% in ApoC3 and 69% in triglycerides. Meanwhile,RN0361Significant and sustained reductions in non-HDL cholesterol, VLDL cholesterol, and remnant cholesterol were also achieved.

Compared with the placebo group, there was no significant difference in fasting blood glucose and glycosylated hemoglobin (HbA1c) in the treatment group. The productWell tolerated, no serious adverse events observed; mild and self-limiting injection site reactions; transient elevation of ALT and AST consistent with the typical characteristics of GalNAc-conjugated siRNA drugs.

Public data shows that the elevation of ApoC3 promotes the aggregation of triglyceride-rich lipoproteins, thereby increasing the risk of acute pancreatitis and cardiovascular diseases, especially in diabetic patients. Current treatment options often struggle to achieve sustained control in critically ill patients.RN0361 Achieves Six Months of Durable Efficacy with a Single Dose, Marking a Significant Advance in the Field of Lipid Management。

References:
[1] Rona Therapeutics Presents Phase I Clinical Data of RN0361 at the 2025 American Heart Association Annual Meeting. From https://www.prnasia.com/story/511059-1.shtml

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