Rona Therapeutics presents phase 1 data for RN0361, a long-acting APOC3-Targeting siRNA, at the American Heart Association 2025 Scientific Sessions

RN0361 achieved potent, durable reductions in ApoC3 and triglycerides (TG) lasting at least six months in a single dose first-in-human trial, establishing a potential best-in-class profile to prevent pancreatitis in severe hypertriglyceridemia (HTG) and reduce cardiovascular risk (CVD) from atherogenic lipoproteins. The data were presented today at the American Heart Association 2025 Scientific Sessions by Dr.Alex DePaoli, MD, CMO.

The randomized, placebo-controlled Single Ascending Dose (SAD) study in volunteers with baseline TG >80 mg/dl showed favorable tolerability, no serious adverse events, with mild, self-limited injection-site reactions (ISRs) and transient ALT and AST elevations typical of GalNAc-conjugated siRNAs. RN0361 demonstrated dose-responsive suppression of both ApoC3 and TG through Day 180, with a maximum 93% ApoC3 reduction and 69% triglyceride lowering. RN0361 also robustly reduced atherogenic lipoproteins, non-HDL-c, VLDL cholesterol and remnant cholesterol. No changes in fasting glucose or HbA1c were observed versus placebo.

Elevated ApoC3 drives triglyceride-rich lipoprotein accumulation, increasing risks of acute pancreatitis and cardiovascular disease, particularly in diabetes. Current therapies often fail to achieve sustained control in severe cases. RN0361's profound, six-month efficacy from a single dose may address this unmet need and marks a significant advance in lipid management.

Stella Shi, CEO said

These data confirm RN0361's long-acting potency and favorable safety profile, supporting its advancement in Phase 2 studies in patients with hypertriglyceridemia. By profoundly lowering ApoC3, TG and atherogenic lipids for at least six months after a single dose, RN0361 is an important potential therapeutic for the critical unmet need in severe HTG and CVD risk reduction.