Developed as a GalNAc-conjugated siRNA drug (GalNAc-siRNA) for treatment of hepatitis B, RBD1016 covers patient population with hepatitis B virus genotypes A-E who represent the vast majority of hepatitis B patients in China, Europe, and the United States. 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. Seroconversion has been observed in model animals in multiple dosages of SR016，which, if confirmed in a clinical trial, may suggest a potential of Best-In-Class and a major step towards functional cure of HBV. A Phase Ia clinical study has been completed in Australia. A Phase Ib clinical study is currently ongoing in Hong Kong. Phase II studies are in preparation.
As an innovative GalNAc-siRNA drug product independently developed by Ribo, RBD1016 has demonstrated well tolerated safety profile and pharmacokinetic characteristics expected for GalNAc-siRNA in the first human clinical study, which validated Ribo's GalNAc-siRNA liver-targeting delivery technology and oligonucleotide therapeutic development platform. Ribo expects more GalNAc-siRNA drug products entering clinical stage.
SR060 is an aptamer targeting vWF and being developed for treatment of Hemophilia A. The aptamer binds specifically to vWF, which prevents the clearance of FVIII carried by vWF, resulting in prolongation of t1/2 as well as elevation of activity of FVIII. Our global partner has successfully completed Phase I and Phase IIa studies, which demonstrated good safety and efficacy of SR060. Ribo has the rights for development, manufacture and commercialization of the product in the Greater China Territory, and is planning clinical studies for Hemophilia A.
SR043 is a GalNAc-conjugated siRNA against PCSK9 (proprotein convertase subtilisin/kexin type 9) for the treatment of hyperlipidemia by lowering LDL-C (low-density lipoprotein cholesterol). The drug reduces LDL-R (low-density lipoprotein receptor) lysosomal degradation and increases the number of LDL-R on the surface of liver cells, thus reducing LDL-C levels in the blood. IND-enabling studies have been completed, and an IND application is under preparation for submission.
SR044 is a GalNAc-conjugated siRNA against APOC3 (Apolipoprotein C-III) for treatment of triglycerides (TGs)-type hypertriglyceridemia. The drug can increase degradation of TGs by elevating levels of lipoprotein lipase. The IND-enabling studies of SR044 and the preparation for IND application are currently under way.
Designed as a siRNA, SR061 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. SR061 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/IIa 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 for a confirmatory Phase III study in China Mainland 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.
SR062 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 drugs for the same target are commercially available. SR062 can achieve glucose-lowering effect through two mechanisms, 1) reducing hepatic glucose production and 2) increasing GLP-1 to protect the pancreas. SR062 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 just been successfully completed.