Being developed as the first GalNAc-siRNA drug based on Ribo's proprietary liver-targeting delivery technology RIBO-GalSTAR® for the treatment of hepatitis B, RBD1016 covers patient population with hepatitis B virus genotypes A-E and I, which represent the vast majority of hepatitis B patients in Europe, the United States and Asia. It has shown a highly efficient long-acting effect of reducing the HBsAg in serum and liver tissues in non-clinical studies. After a single treatment, inhibition of HBsAg lasted nearly 6 months. A Phase Ia clinical study has been completed in Australia. A Phase Ib clinical study is currently ongoing in Hong Kong. Phase II clinical studies are in preparation.
RBD1016 has demonstrated well tolerated safety profile and pharmacokinetic characteristics expected for GalNAc-siRNA in the clinical study.
RBD5044, a GalNAc-siRNA drug based on Ribo's proprietary liver-targeting delivery technology RIBO-GalSTAR®, is being developed for treatment of triglycerides (TGs)-type hypertriglyceridemia. RBD5044 inhibits the expression of APOC3 (Apolipoprotein C-III) to reduce the TGs level in blood by increasing the uptake of lipoprotein lipase and hepatocyte receptor-mediated residual particle and elevating the hydrolysis of triglycerides (TGs) on triglyceride-rich lipoproteins (TRLs). RBD5044 has entered the First-In-Human clinical study in Australia.
RBD4059, a GalNAc-siRNA drug based on Ribo's proprietary liver-targeting delivery technology RIBO-GalSTAR®, is being developed for treatment of thrombotic diseases. RBD4059 inhibits the expression of FXI in hepatocytes to reduce the activation of endogenous coagulation pathways, thus resulting in anticoagulant/antithrombotic effects. As the First-In-Class siRNA drug targeting FXI, RBD4059 has entered the First-In-Human clinical trial in Australia and holds the potential to become a new safe and effective antithrombotic medication.
RBD7022, a GalNAc-siRNA drug based on Ribo's proprietary liver-targeting delivery technology RIBO-GalSTAR®, is being developed for the treatment of hyperlipidemia. RBD7022 inhibits the expression of PCSK9 (proprotein convertase subtilisin/kexin type 9), to lower to reduce LDL-R (low-density lipoprotein receptor) lysosomal degradation and to increase the number of LDL-R on the surface of liver cells, thus reducing LDL-C levels in the blood. RBD7022 has received approval of clinical trial application for the First-In-Human clinical study in China.
Designed as a siRNA, RBD1007 inhibits the expression of the Caspases 2 gene via RNAi mechanism by stopping retinal ganglion cell (RGC) apoptosis and secondary axonal degeneration, thereby preventing further deterioration of vision and visual field in the diseases and achieving therapeutic effect of vision protection. RBD1007 holds the potential of becoming the First-In-Class neuroprotective agent being developed to treat non-arteritic anterior ischemic optic neuropathy (NAION), for which there is no standard therapy at present. One Phase I clinical study and one Phase II/III international multi-center clinical study (including 34 Chinese subjects) have been completed. The clinical data analysis supports further development of the drug for treatment of NAION targeting a specific subpopulation with greatest unmet medical need. An IND application is in preparation. Additional clinical development will follow for study of glaucoma and other ophthalmological diseases associated with optic neuropathy where optic nerve protection is needed.
RBD4988 is an ASO drug for treatment of type 2 diabetes and expected to become the First-In-Class drug to act on the glucagon receptor (GCGR). No drug products for the same target are commercially available. RBD4988 can achieve glucose-lowering effect through two mechanisms, which are reducing hepatic glucose production and increasing GLP-1 to protect the pancreas simultaneously. RBD4988 is expected to address unmet medical need in patients where intervention in the insulin pathway alone is not sufficient. One Phase I clinical study and three Phase II clinical studies have been completed worldwide. Two Phase II clinical studies in China have been completed successfully.