Shanghai, China — Rona Therapeutics, a global leading RNA therapy company, announced preclinical data for its self-developed RNA interference (RNAi) therapy RN026 at the National Lipid Association (NLA) Annual Scientific Sessions on May 29 (local time). Preclinical studies revealed that the therapy, targeting lipoprotein(a) (Lp(a))—an independent risk factor for cardiovascular diseases—achieved a 99% reduction in Lp(a) with potential annual dosing regimen in non-human primate (NHP) study.

Key Highlights

• Unparalleled Efficacy and Durability
  • 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
  • In NHP study, a single 2mg/kg subcutaneous injection achieved maximum 99% reduction of serum Lp(a); 95% Lp(a) reduction was maintained at 98 days post-dose with ~80% serum Lp(a) decreasing effect sustained till 154 days, suggesting annual dosing potential in human
• Precision siRNA Design
  • Targets multiple sites inside and outside of the KIV-2 CNV region to maximize knock-down efficiency
  • Very low hybridization related off-target risk
• Dual Benefits
  • Accompanied by a 25% reduction in LDL-C levels in NHP study
  • Repeated dose tox studies in SD rats confirmed no adverse findings

About RN026

RN026 demonstrates tremendous potential as a breakthrough therapy in a range of cardiovascular diseases, i.e. ASCVD, aortic stenosis, peripheral arterial disease, with differentiated long-lasting efficacy, potent Lp(a) reduction, and excellent safety profile. There is approximately 10-20% world population that has elevated serum Lp(a) level. RN026 is poised to bring new therapeutic hope to cardiovascular patients globally.

About Rona Therapeutics

Rona Therapeutics is a global leader in nucleic acid innovative drug platform company, specializing in the treatment of cardiometabolic diseases, obesity and neurological diseases with proprietary oligo chemistry and delivery platform. Rona Therapeutics is committed to developing the best and first-in-class siRNA drugs with differentiation and innovation to address unmet needs and improve outcome in cardiovascular diseases, obesity, and MASH.