Rona Therapeutics announced breakthrough preclinical data for its RNA interference therapy RN026 at the National Lipid Association Annual Scientific Sessions, demonstrating unprecedented efficacy in targeting lipoprotein(a) with potential for annual dosing. The Shanghai-based company reported that a single injection achieved 99% reduction in Lp(a) levels in non-human primate studies, positioning the therapy as a potential game-changer for cardiovascular disease treatment.
Sustained Efficacy with Extended Dosing Intervals
The preclinical studies revealed remarkable durability of RN026's therapeutic effect across multiple animal models. In transgenic mouse models, a single 2mg/kg subcutaneous injection resulted in 98% reduction in serum Apo(a) protein, which lasted for at least 42 days. The non-human primate study demonstrated even more compelling results, with a single 2mg/kg subcutaneous injection achieving maximum 99% reduction of serum Lp(a).
The durability data suggests potential for annual dosing in humans, with 95% Lp(a) reduction maintained at 98 days post-dose and approximately 80% serum Lp(a) decreasing effect sustained through 154 days. This extended duration of action could significantly improve patient compliance and reduce treatment burden compared to existing therapies requiring more frequent administration.
Precision Targeting and Safety Profile
RN026 employs a precision siRNA design that targets multiple sites inside and outside of the KIV-2 CNV region to maximize knock-down efficiency while maintaining very low hybridization-related off-target risk. The therapy demonstrated dual cardiovascular benefits, accompanied by a 25% reduction in LDL-C levels in the non-human primate study.
Safety assessments showed favorable results, with repeated dose toxicity studies in SD rats confirming no adverse findings. This safety profile, combined with the therapy's potent efficacy, supports its potential for clinical development.
Addressing Significant Unmet Medical Need
Lipoprotein(a) represents an independent risk factor for cardiovascular diseases, affecting approximately 10-20% of the world population with elevated serum levels. RN026 demonstrates potential as a breakthrough therapy for a range of cardiovascular diseases, including atherosclerotic cardiovascular disease (ASCVD), aortic stenosis, and peripheral arterial disease.
The therapy's differentiated long-lasting efficacy, potent Lp(a) reduction, and excellent safety profile position it to bring new therapeutic hope to cardiovascular patients globally. Rona Therapeutics, described as a global leader in nucleic acid innovative drug platform development, specializes in treating cardiometabolic diseases, obesity, and neurological diseases with proprietary oligonucleotide chemistry and delivery platforms.
