Rona Therapeutics, a China-based company involved in developing next-generation RNAi therapeutics, announced on Sunday its phase one clinical data for RN0361, a long-acting Apolipoprotein C3 (ApoC3) siRNA, at the American Heart Association 2025 Scientific Sessions by Dr Alex DePaoli, MD, CMO.

The company says that RN0361 achieved potent, durable reductions in ApoC3 and triglycerides (TG) lasting around 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.

According to Rona, the randomised, placebo-controlled Single Ascending Dose (SAD) study in volunteers with baseline TG >80 mg/dl indicated favourable tolerability, no serious adverse events, with mild, self-limited injection-site reactions (ISRs) and transient ALT and AST elevations typical of GalNAc-conjugated siRNAs. It demonstrated dose-responsive suppression of both ApoC3 and TG to Day 180, with a maximum 93 percent ApoC3 reduction and 69 percent triglyceride lowering. It also robustly educed atherogenic lipoproteins, non-HDL-c, VLDL cholesterol and remnant cholesterol, while no changes in fasting glucose or HbA1c were seen compared to the placebo.

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."