Nucleic acid therapeutics company Rona Therapeutics announced on Monday, its positive outcome from the phase one clinical trial of RN0191, at the American Heart Association's (AHA) Annual Scientific Sessions in Chicago, Illinois on November 16.

RN0191, a proprietary GalNAc conjugated PCSK9 siRNA, is designed to reduce low-density lipoprotein cholesterol (LDL-C) and other lipid parameters.

The Ph1 study, a randomised, single-dose ascending, placebo-controlled study, is intended to assess safety, tolerability, pharmacokinetics and pharmacodynamics in healthy subjects with elevated LDL-C. The demographics included adults aged 18 to 60 years, spanning a BMI range of 19-30 kg/m2. All results are based on data in the database as of 14 October 142024. The study randomised a total of 32 subjects and treated them with RN0191 from 60mg to 600mg, respectively. In the study, the baseline LDL-C mean level was ranging from 110-130 mg/dL.

After a single-dose subcutaneous injection, RN0191 showed a favourable safety profile with no serious adverse events and only mild, transient adverse events reported across all dose- levels. Dose-dependent, significant and durable changes in PCSK9, LDL-C and other lipid parameters were observed.