RN-0191

Rona Therapeutics unveiled promising early-stage findings for its PCSK9-targeted siRNA therapy, dubbed RN0191, at the American Heart Association's (AHA) annual scientific sessions on Sunday, demonstrating notable reductions in PCSK9 and low-density lipoprotein cholesterol (LDL-C) after a single dose. The Shanghai-based biotech gained the asset securing exclusive global rights to Sanofi’s siRNA platform in 2022. Touting 'best-in-class' prospects, Rona CEO Stella Shi, said the “results highlight RN0191's potential as a transformative siRNA therapy for global patients with elevated LDL-C…either as single agent therapy or as combinatory backbone to improve cardiovascular outcome."The Phase I study randomised 32 healthy participants aged 18 to 60, to receive a single dose of RN0191 in the range of 60 mg to 600 mg or placebo. Results showed that the RN0191 group achieved up to 95% maximum reduction in PCSK9 and a 74% drop in LDL-C, with average reductions of 87% and 56%, respectively, compared with placebo. Moreover, RN0191 maintained up to 42% LDL-C reduction at six months, indicating suitability for bi-annual dosing. Additionally, RN0191 demonstrated significant reductions in ApoB, Lipoprotein(a), non-high-density lipoprotein cholesterol and total cholesterol. In terms of safety, the therapy had no serious adverse events, with only mild and short-term side effects reported at all doses.Following a $33-million Series A round in 2022, Rona secured another $35 million this year to advance RN0191 and another lipid-lowering siRNA against APOC3, named RN0361, into mid-stage testing. Besides dyslipidaemia, the company’s pipeline features a range of liver-directed siRNA therapies being developed for obesity, metabolic dysfunction-associated steatohepatitis, atherosclerotic cardiovascular disease, IgA nephropathy and hypertension.

Single dose of RN0191 achieved up to 95% individual maximum and 87% mean maximum PCSK9 reduction, and up to 74% individual maximum and 56% mean maximal LDL-C lowering Robust and sustainable treatment effects support at least bi-annual dosing regimen Best-in-class potential of novel PCSK9 siRNA RN0191 supports further development as single agent or combination to further reduce atherosclerosis cardiovascular risk SHANGHAI, Nov. 18, 2024 /PRNewswire/ -- Rona Therapeutics, a leader in innovative RNA-based therapies, today presented positive results from the Phase 1 clinical trial of RN0191 at the American Heart Association's (AHA) Annual Scientific Sessions in Chicago, IL, November 16th, 2024. RN0191 is a proprietary GalNAc conjugated PCSK9 siRNA designed to significantly lower low-density lipoprotein cholesterol (LDL-C) and other lipid parameters. The Ph1 study was a randomized, single-dose ascending, placebo-controlled study to evaluate 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 Oct. 14, 2024. A total of 32 subjects were randomized and treated with RN0191 from 60mg to 600mg, respectively. The baseline LDL-C mean level was ranging from 110-130 mg/dL. After a single-dose subcutaneous injection, RN0191 showed a favorable 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 (see table below). Mean maximum reduction of PCSK9 >85% and LDL-C >55% have been achieved. Significant and durable LDL-C reduction is achieved up to 42% through Day180, supporting a bi-annual dosing regimen for future development Significant lowering of ApoB, Lp(a), non-HDL-C and total cholesterol are also noted Remarkable treatment effects with reduction of PCSK9, LDL-C and the other lipid parameters maintained to 6 months. Stella Shi, CEO of Rona Therapeutics, shared, "We are thrilled to report that RN0191 has demonstrated best-in-class PCSK9 siRNA potential. These results highlight RN0191's potential as a transformative siRNA therapy for global patients with elevated LDL-C, a major risk factor for atherosclerotic cardiovascular disease either as single agent therapy or as combinatory backbone to improve cardiovascular outcome. " About Rona Therapeutics Rona Therapeutics is a global leader in nucleic acid innovative drug platform company, specializing in the treatment of metabolic diseases and neurological diseases. Rona Therapeutics is always 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. In addition, Rona Therapeutics is unlocking potential of extra-hepatic delivery for neurological disorders and adipose/muscle delivery or various metabolic syndromes. For Further information, Please visit: or contact: [email protected] WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED