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Rona Therapeutics Presented Positive Phase 1 Results of RN0191, a Novel siRNA Therapy Targeting PCSK9, at the 2024 American Heart Association (AHA) Scientific Sessions

Small Cap Bulls Editorial Team by Small Cap Bulls Editorial Team
May 10, 2025
Reading Time: 7 mins read
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  • 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 maximalย LDL-C lowering
  • Robust and sustainable treatment effects support at leastย bi-annual dosing regimen
  • Best-in-class potential of novel PCSK9ย siRNA RN0191 supports further development as single agent or combination to further reduce atherosclerosis cardiovascular risk

SHANGHAI,ย Nov. 17, 2024ย /CNW/ — Rona Therapeutics, a leader in innovative RNA-based therapies, todayย presentedย positive results from the Phase 1 clinical trial of RN0191ย atย the American Heart Association’s (AHA) Annual Scientific Sessions inย Chicago, IL, Novemberย 16th, 2024. RN0191 is aย proprietary GalNAc conjugatedย PCSK9 siRNA designed to significantly lower low-density lipoprotein cholesterol (LDL-C) and other lipid parameters.

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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. Allย resultsย are based on data in the database as ofย 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).

  • ย 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 cholesterolย are also noted
  • ย Remarkable treatment effects with reduction of PCSK9, LDL-C and the other lipid parameters maintained to 6 months.

Table.ย  Percentage change from baseline of PCSK9 and LDL-C level after single dose of RN0191

Placebo

ย  60mg

200mg

400mg

600mg

PCSK9

Mean Maximum Change (%)

-10ย %

-64ย %

-79ย %

-86ย %

-87ย %

(Days post injection)

(8)

(15)

(29)

(22)

(57)

Mean Change (%), Day 85

-4ย %

-33ย %

-69ย %

-79ย %

-83ย %

Individual Maximum Change (%)

-50ย %

-81ย %

-87ย %

-91ย %

-95ย %

LDL-C

Mean Maximum Change (%)

-6ย %

-23ย %

-48ย %

-56ย %

-51ย %

(Days post injection)

(85)

(57)

(29)

(71)

(22)

Mean Change (%), Day 85

-6ย %

-20ย %

-36ย %

-47ย %

-45ย %

Individual Maximum Change (%)

-20ย %

-63ย %

-61ย %

-71ย %

-74ย %

Stellaย 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:ย www.ronatherapeutics.comย or contact:ย BD@ronatherapeutics.com

SOURCE Rona Therapeutics

Organization Profile

Rona Therapeutics

Also from this source

  • Rona Therapeutics Raises $35 Million Series A+ Financing to Advance Innovative Metabolic siRNA Pipeline in Clinic and Next Generation RNA Platforms

Tags: BiotechHealth
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