On December 29, 2023, Rona Therapeutics's first independently developed RN0191 injection has successfully obtained the implicit license for clinical trial application approved by the National Medical Products Adminstration. As the third PCSK9 siRNA to enter simultaneous clinical development in China and overseas, this product can be used to treat hypercholesterolemia, mixed hyperlipidemia and atherosclerotic cardiovascular disease to reduce the risk of cardiovascular events.

About RN0191

RN0191 is a double-stranded small interfering RNA (siRNA) derived from Rona Therapeutics’s GAIA liver-targeting technology platform targeting the PCSK9 target. It is a new type of nucleic acid drug targeting specific disease-causing genes. This product has independent intellectual property rights. and global patent protection.

Preclinical studies have shown that RN0191 has potent and long-lasting pharmacological activity and excellent safety in rodents and non-human primates, and is expected to significantly and sustainably reduce serum low-density lipoprotein with just two injections per year. Cholesterol (LDL-C) levels. In the cynomolgus monkey trial, compared with the benchmark product, RN0191, in addition to reducing low-density lipoprotein cholesterol, also showed a higher reduction in lipoprotein (a), another lipid metabolism indicator closely related to atherosclerosis. The potential advantages are expected to further reduce the residual risk of patients with cardiovascular disease and become best-in-class.

In June 2023, RN0191 received ethical approval to conduct its first human trial in Australia. The experiment is currently progressing smoothly, and existing data show that RN0191 is well tolerated and safe, and shows dose-dependent efficacy and efficacy. At the same time, the decrease in multiple important lipid metabolism indicators, including lipoprotein(a), was also very significant, further confirming its potential advantage of significantly dually reducing low-density lipoprotein and lipoprotein(a). This result provides strong support for the development of subsequent clinical trials of this differentiated product.

About atherosclerotic cardiovascular disease

Atherosclerotic cardiovascular disease (ASCVD) is the first cause of death worldwide and in China. Taking Chinese data as an example, there were approximately 2.4 million ASCVD deaths in 2016, accounting for 61% of total cardiovascular disease deaths and 25% of all-cause deaths. A large amount of clinical data has confirmed that hypercholesterolemia.

About Rona Therapeutics

Rona Therapeutics is a pioneering RNA therapeutics platform company dedicated to discovery and development of modular and programmable RNA medicines to deliver to patients around the world.

Backed by blue-chip investors, Rona has developed proprietary RAZOR™ platform to discover differentiated siRNA therapeutics from next-generation chemical modification and novel biological insights. Together with partners, Rona is dedicated to advancing transformative RNA therapeutics into clinic in metabolic syndrome, glomerulonephritis.