Suzhou Ribo Life Science Co., Ltd.，the leading innovative oligonucleotide therapeutics company in China, announced on Sept 19, 2022, that the company has received the approval from the CDE of the NMPA for the clinical trial application of its innovative PCKS9 targeting oligonucleotide drug product RBD7022 to conduct the First-In-Human clinical trial in China.
Unlike small molecule and biological lipid lowering drugs, RBD7022 is an siRNA drug targeting PCSK9 mRNA in hepatocytes via RNA interference mechanism to inhibit the expression of PCSK9 protein, which, in turn, increases the amount of LDL-R on the hepatocyte surface, further resulting in an enhanced clearance of LDL-C and achieving lipid lowering purpose.
RBD7022, independently developed by Ribo using its proprietary siRNA delivery system – RIBO-GalSTARTM, is the second GalNAc conjugated siRNA that entered clinical development following the anti-HBV drug RBD1016. As demonstrated by the preclinical study results, RBＤ7022 is characterized by the good safety profile, the highly effective and durable lipid lowering capability. Following a single subcutaneous dosing, the efficacy can be sustained for several months with the inhibition of LDL-C up to 50%.
As the aging population has been increasing over the recent years, there is a huge lipid abnormal patient population that continues to grow on annual basis. Studies have revealed that high LDL-C is the 5th and the 6th risk factor, yet still on the rise, leading to the modality globally and in China, respectively. Among the ASCVD patients and high risk ASCVD patients, only 44% and 27% patients can achieve the targeted level of LDL-C following lipid lowering treatment with standard of care. Lowering LDL-C is the important clinical treatment for cardiovascular diseases. However, there is a significant portion of Chinese patients who are resistant to statin drugs or have low compliance. PCSK9-targeted siRNA drug holds the potential to significantly improve the prevention and treatment arsenal for patients suffering cardiovascular diseases.
Dr. Liming Gan, Global R&D President at Ribo, commented “As RBD7022 entered the clinical development, it has laid a solid foundation for Ribo to achieve the goal to bring novel treatment for patients with lipid abnormalities. With the innovative oligonucleotide drug development platform and the competitive pipeline in the cardiovascular and metabolic disease areas that Ribo has built, we have high confidence in brining hopes to patients in China and rest of the world by developing novel therapeutics.
“For the last fifteen years since its establishment in 2007, Ribo, with dedicated efforts, has built a fully integrated technology platform for the research and development of innovative oligonucleotide therapeutics．RBD7022 is the 5th drug product that Ribo has brought into the clinical development, and it also marks that RIBO-GalSTARTM, the proprietary siRNA delivery system independently developed by Ribo for GalNAc conjugated siRNA, has become mature”, said Zicai Liang, Ph.D., Chairman and CEO of Ribo, “We anticipate that each year multiple new oligonucleotide products will enter clinical development. Aiming to treat diseases with unmet medical needs，Ribo will accelerate development process to bring novel therapeutics for the benefit of many patients.”