Rona Therapeutics presented topline results from its first-in-human Phase 1 study of RN0361, a long-acting small interfering RNA (siRNA) targeting apolipoprotein C3 (ApoC3), at the American Heart Association (AHA) 2025 Scientific Sessions, the company said. The trial evaluated single-dose administration in volunteers with elevated triglycerides (TG > 80 mg/dL) and found durable reductions in ApoC3 and triglycerides lasting at least six months.
According to the company, RN0361 demonstrated dose-dependent suppression of ApoC3 reaching a maximum reduction of 93 %, and triglycerides decreased by up to 69 %. The study also showed meaningful decreases in non-HDL cholesterol, VLDL-cholesterol and remnant cholesterol. The company said no drug-related serious adverse events occurred; reported adverse events were limited to mild injection-site reactions and transient ALT/AST elevations consistent with the GalNAc-siRNA class.
The company claims that the data position RN0361 as a potential best-in-class therapy for severe hypertriglyceridemia and atherogenic lipoprotein reduction, with implications for preventing acute pancreatitis and cardiovascular disease. According to the company, the long-acting durability from a single dose supports plans to initiate Phase 2 development in patients with severe hypertriglyceridemia.
The firm said that moving into Phase 2 will allow evaluation in target patient populations and assessment of clinical benefits related to pancreatitis prevention and cardiovascular-risk reduction. The presentation at AHA raises the profile of the company’s RNAi cardiovascular pipeline and reinforces investor and clinical interest in the program.
