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Rona Therapeutics Unveils Phase 1 Data for RN0361

On November 10, 2025, Rona Therapeutics, a pioneering name in RNA interference (RNAi) therapeutics, made headlines at the American Heart Association 2025 Scientific Sessions by presenting critical Phase 1 clinical data for their long-acting Apolipoprotein C3 (ApoC3) targeting siRNA, known as RN0361. This innovative treatment is crucial for managing severe hypertriglyceridemia (HTG) and reducing cardiovascular risk associated with atherogenic lipoproteins.

Groundbreaking Efficacy Findings

The Phase 1 trial involved a randomized, placebo-controlled study focused on volunteers suffering from elevated triglyceride levels (TG) above 80 mg/dl. RN0361 demonstrated impressive results, boasting sustained reductions in ApoC3 and triglycerides over a six-month period after just a single dose. Participants experienced a maximum reduction of 93% in ApoC3 levels and a 69% decrease in triglyceride levels.

Dr. Alex DePaoli, the Chief Medical Officer of Rona Therapeutics, highlighted the study's findings, stating that RN0361 presents a potential best-in-class solution capable of providing lasting benefits in lipid management. Importantly, the treatment was well-tolerated by participants, with no serious adverse effects but some mild and transient injection-site reactions. Moreover, there were no significant changes in fasting glucose or glycosylated hemoglobin (HbA1c) when compared to placebo, indicating RN0361's favorable safety profile.

Unmet Needs Addressed

High levels of ApoC3 are known to lead to the accumulation of triglyceride-rich lipoproteins, significantly increasing the risk of acute pancreatitis and cardiovascular diseases, especially in diabetic patients. Traditional therapies often fall short in effectively controlling these conditions, which emphasizes the importance of RN0361's unique therapeutic approach. By enabling sustained efficacy post a single administration, RN0361 could fill a critical gap in treatment options for individuals suffering from severe hypertriglyceridemia and its associated complications.

In a statement from Stella Shi, the CEO of Rona Therapeutics, she remarked, "These data confirm RN0361's long-acting potency and favorable safety profile, supporting its advancement in Phase 2 studies in patients with hypertriglyceridemia." The emphasis on a sustained reduction in ApoC3 and TG levels underscores RN0361's potential as a transformative solution for managing lipid profiles effectively.

Future Directions

The clinical success of RN0361 represents a pivotal moment for Rona Therapeutics, reinforcing their commitment to pioneering therapeutic solutions addressing cardiometabolic disorders. Following the promising results from Phase 1 trials, the company plans to advance into Phase 2 studies, which will further evaluate RN0361's efficacy and safety in a larger cohort of patients affected by hypertriglyceridemia.

Rona Therapeutics has pioneered the utilization of next-generation RNA technology since its establishment in 2021. With four programs already transitioned into clinical phases and approximately $200 million raised from prominent healthcare investors, the company is poised for continued innovation.

In conclusion, the unveiling of Phase 1 results for RN0361 at the AHA 2025 Scientific Sessions anticipates a new era of treatment for severe HTG and cardiovascular risk management. By targeting ApoC3, RN0361 holds promise as an effective and long-lasting therapeutic option for patients facing these challenging health issues.