Shanghai-based nucleic acid drug developer Rona Therapeutics has announced the presentation of positive Phase I results for its investigational drug, RN0191, at the American Heart Association’s (AHA) Annual Scientific Sessions in Chicago, IL. The study’s findings highlight the potential of RN0191 in managing lipid levels and offer promising news for the treatment of cardiovascular diseases.
RN0191’s Design and Study Protocol
RN0191 is a proprietary GalNAc-conjugated PCSK9 siRNA therapy designed to significantly lower low-density lipoprotein cholesterol (LDL-C) and other lipid parameters. The Phase I study was a randomized, single-dose ascending, placebo-controlled trial that evaluated the safety, tolerability, pharmacokinetics, and pharmacodynamics of RN0191 in healthy subjects with elevated LDL-C levels.
Study Results and Efficacy
As of October 14, 2024, a total of 32 subjects were randomized and treated with RN0191 at doses ranging from 60mg to 600mg. The drug demonstrated a favorable safety profile, with no serious adverse events reported and only mild, transient adverse events observed across all dose levels. Dose-dependent, significant, and durable changes in PCSK9, LDL-C, and other lipid parameters were noted. Mean maximum reduction of PCSK9 was over 85%, and LDL-C was over 55%. Notably, significant and durable LDL-C reduction of up to 42% was maintained through Day 180, supporting a potential bi-annual dosing regimen for future development. Additionally, significant lowering of ApoB, Lp(a), non-HDL-C, and total cholesterol was observed.-Fineline Info & Tech
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