Rona Therapeutics, a global developer of next-generation RNAi therapeutics, presented Phase 1 clinical data for RN0361, its long-acting Apolipoprotein C3 (ApoC3) siRNA, at the American Heart Association (AHA) 2025 Scientific Sessions. The results highlight RN0361’s potent and durable reductions in ApoC3 and triglycerides (TG) lasting at least six months following a single dose, supporting its potential as a best-in-class therapy for severe hypertriglyceridemia (HTG) and cardiovascular disease (CVD) risk reduction.
Phase 1 Study Overview
- Design: Randomized, placebo-controlled Single Ascending Dose (SAD) trial in volunteers with baseline triglycerides >80 mg/dL.
- Tolerability: RN0361 was well tolerated with no serious adverse events. Mild, transient injection-site reactions (ISRs) and short-lived ALT/AST elevations were consistent with known effects of GalNAc-conjugated siRNAs.
- Efficacy:
- Up to 93% reduction in ApoC3
- Up to 69% reduction in triglycerides
- Sustained effect through Day 180
- Significant lowering of non-HDL-C, VLDL-C, and remnant cholesterol
- No change in fasting glucose or HbA1c compared with placebo
Clinical Significance
Elevated ApoC3 contributes to the buildup of triglyceride-rich lipoproteins, increasing the risk of acute pancreatitis and CVD, especially in patients with diabetes. Current therapies often fail to deliver sustained triglyceride control in severe cases. RN0361’s single-dose, six-month efficacy could represent a major advancement in lipid management and prevention of triglyceride-driven complications.
Executive Commentary
“These data confirm RN0361’s long-acting potency and favorable safety profile, supporting its advancement into Phase 2 studies,” said Stella Shi, CEO of Rona Therapeutics. “By profoundly lowering ApoC3, triglycerides, and atherogenic lipids for at least six months after a single dose, RN0361 addresses a critical unmet need in severe HTG and cardiovascular risk reduction.”
About Rona Therapeutics
Founded in 2021, Rona Therapeutics is a global RNAi therapeutics company focused on cardiometabolic, obesity, and degenerative diseases. Leveraging proprietary innovations in RNA delivery, multi-valency, and oligo chemistry, the company has advanced four clinical programs and raised approximately $200 million from leading healthcare investors and strategic partners.
