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

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 maximalLDL-C lowering

Robust and sustainable treatment effects support at leastbi-annual dosing regimen

Best-in-class potential of novel PCSK9siRNA RN0191 supports further development as single agent or combination to further reduce atherosclerosis cardiovascular risk

SHANGHAI,Nov. 18,2024 -- 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. Allresultsare 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%

StellaShi,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:www.ronatherapeutics.comor contact:[email protected]