Rona Therapeutics' First Internally Developed siRNA Drug RN0191 Receives IND Approval in China

December 29, 2023Rona Therapeutics' First Self-Developed RN0191 Injection Successfully Obtains Implied Permission for Clinical Trial Application from China's National Medical Products Administration. As the third PCSK9 siRNA to enter synchronized clinical development in China and overseas, this product can be used to treat hypercholesterolemia, mixed hyperlipidemia, and atherosclerotic cardiovascular disease, reducing the risk of cardiovascular events.

Preclinical studies have shown that RN0191 exhibits potent and sustained pharmacological activity and excellent safety in both rodents and non-human primates, with the potential to significantly and continuously reduce serum levels with only two injections per year.Low-Density Lipoprotein Cholesterol(LDL-C) levels. In the cynomolgus monkey trials, compared with the reference product, RN0191 not only reduced low-density lipoprotein cholesterol but also demonstrated a potential advantage in significantly lowering another lipid metabolism indicator closely related to atherosclerosis—lipoprotein(a), which is expected to further reduce cardiovascular risks in patients.Residual Risk, becoming the best-in-class.

In June 2023, RN0191 received ethical approval in Australia to conduct its first human trial. The trial is currently proceeding smoothly, with existing data indicating good tolerability and safety of RN0191, while demonstrating dose-dependent pharmacological effects and efficacy. Additionally, significant reductions in several key lipid metabolism indicators, including lipoprotein(a), further confirm its potential dual advantage in significantly lowering both low-density lipoprotein and lipoprotein(a). This outcome provides strong support for the subsequent clinical trials of this differentiated product.

AboutRN0191

RN0191 is a product derived from Rona Therapeutics' innovative GAIA liver-targeting technology platform.PCSK9A double-stranded small interfering RNA (siRNA) targeting a specific pathogenic gene is a novel nucleic acid drug. This product...With independent intellectual property rights and global patent protection.

About Atherosclerotic Cardiovascular Disease

Atherosclerotic Cardiovascular Disease (ASCVD) is the leading cause of death worldwide and in China. Taking China's data as an example, in 2016, about 2.4 million people died from ASCVD, accounting for 61% of total cardiovascular disease deaths and 25% of all-cause deaths. A large amount of clinical data has confirmed that hypercholesterolemia, mainly characterized by elevated low-density lipoprotein cholesterol (LDL-C), is a Class I risk factor for ASCVD, and reducing LDL-C can effectively prevent ASCVD.

Currently, statins have become the standard treatment for lowering LDL-C, but the LDL-C target achievement rate among Chinese ASCVD patients is only 6.8%, with poor tolerance to statins being one of the reasons. Therefore, there is an urgent clinical need to...Drugs that are more effective and better tolerated for lowering LDL-C.

About Rona Therapeutics

Rona Therapeutics is a leading nucleic acid drug platform company dedicated to developing modular and programmable nucleic acid drugs to benefit patients worldwide. The company was founded by industry veterans with deep expertise in nucleic acid drugs and world-leading scientific advisors with profound biological insights.