Rona Therapeutics Presented Positive Phase 1 Results Of RN0191, A Novel Sirna Therapy Targeting PCSK9, At The 2024 American Heart Association (AHA) Scientific Sessions
Rona Therapeutics Presented Positive Phase 1 Results Of RN0191, A Novel Sirna Therapy Targeting PCSK9, At The 2024 American Heart Association (AHA) Scientific Sessions . (MENAFN - PR Newswire) 
  
  
 
 
 
 
 
   
  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 ...
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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 maximal

LDL-C lowering

Robust and sustainable effects support at least

bi-annual dosing regimen Best-in-class potential of PCSK9

siRNA RN0191 supports further development as single agent or combination to further reduce atherosclerosis cardiovascular risk SHANGHAI, Nov. 17, 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.

Table.

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

Placebo

60mg 200mg 400mg 600mg PCSK9









Mean Maximum Change (%) -10

% -64

% -79

% -86

% -87

% (Days post injection) (8) (15) (29) (22) (57) Mean Change (%), Day 85 -4

% -33

% -69

% -79

% -83

% Individual Maximum Change (%) -50

% -81

% -87

% -91

% -95

% LDL-C









Mean Maximum Change (%) -6

% -23

% -48

% -56

% -51

% (Days post injection) (85) (57) (29) (71) (22) Mean Change (%), Day 85 -6

% -20

% -36

% -47

% -45

% Individual Maximum Change (%) -20

% -63

% -61

% -71

% -74

%

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,

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[email protected]

SOURCE Rona Therapeutics

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https://menafn.com/1108895170/Rona-Therapeutics-Presented-Positive-Phase-1-Results-Of-RN0191-A-Novel-Sirna-Therapy-Targeting-PCSK9-At-The-2024-American-Heart-Association-AHA-Scientific-Sessions
2024-11-17
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(MENAFN - PR Newswire) 
  
  
 
 
 
 
 
   
  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 ...
Rona Therapeutics Presented Positive Phase 1 Results Of RN0191, A Novel Sirna Therapy Targeting PCSK9, At The 2024 American Heart Association (AHA) Scientific Sessions