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Rona Therapeutics Presents Phase 1 Data for RN0361

Rona Therapeutics, a prominent player in RNAi therapeutics, recently presented compelling Phase 1 clinical data for their innovative product RN0361 during the American Heart Association's 2025 Scientific Sessions. This novel long-acting siRNA formulation targets Apolipoprotein C3 (ApoC3), revealing significant reductions in both ApoC3 and triglycerides (TG) after a single dose.

The study demonstrated noteworthy efficacy, showcasing that RN0361 can achieve substantial and durable decreases in ApoC3 and triglycerides, lasting for a minimum of six months. The first-in-human trial indicated that a single administration not only led to impressive reductions—up to 93% in ApoC3 and 69% in triglycerides—but also did so with a favorable safety profile. The results were presented by Dr. Alex DePaoli, CMO of Rona Therapeutics, providing a glimpse into the potential of RN0361 as a groundbreaking solution to prevent pancreatitis in patients suffering from severe hypertriglyceridemia.

Conducted as a randomized, placebo-controlled single ascending dose (SAD) study, the trial involved volunteers who exhibited baseline triglyceride levels exceeding 80 mg/dL. Feedback concerning the tolerability of RN0361 was positive, with no serious adverse events reported. The only side effects included mild, self-limiting injection site reactions and transient elevations in ALT and AST, aligning with the expected responses associated with GalNAc-conjugated siRNAs.

The study tracked the impact of RN0361 on a variety of lipid parameters, including atherogenic lipoproteins, non-HDL cholesterol, VLDL cholesterol, and remnant cholesterol, regularly documenting dose-responsive suppression leading up to Day 180. Importantly, assessments showed no adverse changes in fasting glucose or HbA1c compared to placebo, indicating RN0361's specificity in targeting lipid metabolism without negatively affecting glucose levels, which is a significant concern in patients with comorbid diabetes.

The implications of elevated ApoC3 levels are serious, driving the accumulation of triglyceride-rich lipoproteins and vastly increasing the risk of acute pancreatitis and cardiovascular diseases, particularly among individuals with diabetes. Existing treatments often struggle to offer sustained control in extreme cases, making the long-lasting effects of RN0361 a promising prospect in lipid management.

Stella Shi, CEO of Rona Therapeutics, expressed optimism about these findings, stating, "These data confirm RN0361's long-acting potency and favorable safety profile, supporting its advancement in Phase 2 studies in patients with hypertriglyceridemia." With its ability to lower ApoC3, triglycerides, and atherogenic lipids for an extended period post-administration, RN0361 holds promise as a much-needed therapeutic intervention for patients at risk of severe HTG and cardiovascular conditions.

About Rona Therapeutics

Founded in 2021, Rona Therapeutics has rapidly emerged as a global leader in RNAi therapeutics, focusing on addressing cardiometabolic, obesity-related, and degenerative diseases through its revolutionary RNA technology platform. The company has effectively advanced four of its innovative programs into clinical trials, thanks to a proprietary delivery system utilizing multi-valency and oligo chemistry. Rona has successfully raised approximately $200 million from distinguished global healthcare investors and strategic partners, further solidifying its position within the biopharmaceutical sector.