Being developed as the first GalNAc-siRNA drug based on Ribo's proprietary liver-targeting delivery technology RIBO-GalSTARTM 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. RBD1016 is the only anti-hepatitis B siRNA drug globally that demonstrated serological conversion in animal models, indicative of its BIC potential. RBD1016 has demonstrated well tolerated safety profile and pharmacokinetic characteristics expected for GalNAc-siRNA in clinical studies. Pharmacodynamic data on hepatitis B patients indicate that RBD1016 exhibits a relatively consistent and long-lasting inhibitory effect on HBsAg, HBV DNA, HBV RNA, and HBcrAg.
A Phase 1a study in Australia and as Phase 1b study in Hong Kong have been completed. A global multicenter Phase 2 clinical study targeting Hepatitis B is in progress. A clinical trial application for a phase 2a clinical study in Hepatitis D patients has been submitted to EMA.
RBD5044, a GalNAc-siRNA drug based on Ribo's proprietary liver-targeting delivery technology RIBO-GalSTARTM, 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). Currently, RBD5044 is undergoing a phase I clinical trial in Australia, and the preliminary results showed good safety profile. A clinical trial application for Phase 2a study in Europe is under preparation.
RBD4059, a GalNAc-siRNA drug based on Ribo's proprietary liver-targeting delivery technology RIBO-GalSTARTM, 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 is undergoing a phase 1 clinical trial in Australia, and the preliminary results showed good safety profile, long-acting and strong inhibitory effectiveness. A clinical trial application for Phase 2a clinical study in Europe will be submitted in early 2024.
RBD7022, a GalNAc-siRNA drug based on Ribo's proprietary liver-targeting delivery technology RIBO-GalSTARTM, 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. Currently, a phase I clinical trial for RBD7022 is being conducted in China. The rights for development, manufacture and commercialization of RBD7022 in the China region has been licensed out to Qilu Pharmaceutical.
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. Additional clinical development may be considered for glaucoma and other ophthalmological diseases associated with optic neuropathy where optic nerve protection is needed.
RBD7007, a GalNAc-siRNA drug based on Ribo's proprietary liver-targeting delivery technology RIBO-GalSTARTM, is being developed for treatment of Complement-related diseases. RBD7007 inhibits the expression of C5 in hepatocytes to reduce the overactivation of the complement system, achieving the effect of treating complement-related diseases. RBD7007 has demonstrated robust and long-duration pharmacological effects in cynomolgus monkeys, holding the potential to be Best-In-Class. A clinical trial application for a first-in-human study is being prepared for submission to EMA.
RBD8088，the first anti-tumor oligonucleotide drug conjugate developed based on Ribo's proprietary tumor-targeting delivery technology RIBO-OncoSTARTM, is able to penetrate the blood-brain barrier and specifically target glioma cells, thereby exerting therapeutic effects. As the First-In-Class oligonucleotide therapeutic globally, RBD8088 is currently in the preclinical development stage, aiming to initiate a first-in-human clinical trial in 2025. RBD8088 holds the potential to be a novel, safe and efficacious therapeutic drug for glioma treatment.