Ribo Life Sciences' Hepatitis B siRNA Drug RBD1016 Receives NMPA Approval for Phase II Clinical Trial with Monotherapy Efficacy Lasting Nearly Six Months

Oct 24, 2024 16:14 CST Updated 16:14

Ribo Life Science

Small Nucleic Acid Drug Developer

|Organized by the Hunter Pharm Club Research Team

Recently, Suzhou Ribo Life Science Co., Ltd. (referred to as "Ribo Life Science") announced that the Phase 2 clinical trial of RBD1016 injection, independently developed by Ribo Life Science for the treatment of patients with chronic hepatitis B virus (HBV) infection, has received the clinical trial approval notice from the National Medical Products Administration.

AboutRBD1016：With BIC Potential，Single-agent efficacy can last nearly half a year.

RBD1016 is a product of Ribo Life Science based on its proprietary intellectual property, RIBO-GalSTAR.^TMA GalNAc-siRNA drug developed by the platform with global rights, targeting the X gene of HBV. It inhibits all four transcripts of HBV through the RNA interference mechanism and has the ability to simultaneously suppress HBV DNA replication, reduce cccDNA and integrated DNA-derived HBsAg, as well as other antigens.

RBD1016 has completed the Phase 1a clinical trial in healthy subjects and the Phase 1b clinical trial in chronic hepatitis B subjects. The study results show,RBD1016 Efficiently Reduces HBsAg in Hepatitis B Patients, Demonstrating Excellent Dose Dependency；Monotherapy can achieve significant clinical efficacy, with effects lasting nearly half a year.`, the reduction of HBsAg was further enhanced after two doses.`With potent effects in reducing both HBV RNA and HBcrAg。

Presented at the EASL 2023 (European Association for the Study of the Liver Annual Meeting)Phase 1b Data

RBD1016 demonstrated good safety and tolerability in both healthy subjects and hepatitis B patients.Clinical Research Results Show the Potential of BIC。

Ribo Life Science will continue to explore the monotherapy of RBD1016 and its combination with other anti-hepatitis B drugs of different mechanisms, hoping to further reduce HBsAg and achieve the goal of functional cure for hepatitis B.

Ribo Life Science Technology Platform and Pipeline Overview

Ribo Life Science was founded in 2007 as an innovative R&D company dedicated to developing RNA interference (RNAi) drugs. It is one of the main pioneers of small nucleic acid technology and the small nucleic acid pharmaceuticals industry in China. The company's research and development team consists of international experts with extensive experience in RNAi technology, drug discovery, and development. Headquartered in Kunshan, Jiangsu, the company has research and development centers in Beijing and Gothenburg, Sweden.

Ribo Life Science aligns with the innovative frontier of international small nucleic acid technology, committing to the iterative research and development of small nucleic acid chemical modification and drug delivery technologies. It has established a self-controlled, fully integrated small nucleic acid drug R&D platform that supports the entire lifecycle of small nucleic acid drugs from early-stage research to industrialization, featuring its self-developed GalNAc small nucleic acid drug delivery technology platform.RIBO-GalSTAR^TMHighly specific liver targeting, high efficiency, and long-acting characteristics with international competitive standards。

Ribo Life Science is committed to the continuous innovation of key technologies in small nucleic acid pharmaceuticals. Currently, the company’s liver-targeted delivery technology and second-generation modification technology have fully matured, supporting four varieties entering clinical stages. The company has conducted systematic and in-depth research on specific targeted delivery technologies for tissues and organs outside the liver, such as tumors, the central nervous system, lungs, kidneys, and muscles, achieving significant progress. In areas such as third-generation modification technology, multi-target interference technology, and innovative synthesis technology, the company has also carried out extensive work and achieved gratifying results.

Ribo Life Science focuses on rapidly advancing small nucleic acid drug products to the international market, aiming to develop the best-in-class RNAi therapeutic drugs in the industry. The company has now entered the rapid development phase of small nucleic acid drug varieties, with 8 clinical varieties in its pipeline, 5 of which are based on...RIBO-GalSTAR^TMTechnology. Ribo Life Science has now established a number of important intellectual property portfolios, including patents, technical secrets, and more related to RNAi technology and its products, forming a comprehensive intellectual property protection system.

RBD5044

As a GalNAc-conjugated siRNA drug developed based on Ribo Life Science's RIBO-GalSTARTM liver-targeted delivery technology for the treatment of hypertriglyceridemia, RBD5044 can inhibit hepatic APOC3 (apolipoprotein C3) expression, upregulate lipoprotein lipase and receptor-mediated remnant particle uptake in hepatocytes, and promote the hydrolysis of triglycerides (TGs) on triglyceride-rich lipoproteins (TRLs), thereby achieving the goal of reducing blood TG levels. Currently, RBD5044 is undergoing Phase I clinical trials in Australia, with preliminary results showing good safety. The application for Phase 2a clinical trials in Europe is being prepared.

RBD4059

As a GalNAc-conjugated siRNA drug developed based on Ribo Life Science's RIBO-GalSTARTM liver-targeted delivery technology for the treatment of thrombotic diseases, RBD4059 can inhibit the expression of FXI in hepatocytes, thereby reducing the activation of the intrinsic coagulation pathway to achieve anticoagulant/antithrombotic effects. As the world’s first siRNA drug (First-In-Class) targeting the FXI pathway, RBD4059 is currently undergoing Phase 1 clinical trials in Australia. Preliminary results indicate good safety, as well as long-lasting and potent characteristics. A Phase 2a clinical trial application in Europe is expected to be submitted in early 2024.

RBD7022

As a GalNAc-conjugated siRNA drug developed based on Ribo Life Science's RIBO-GalSTARTM liver-targeted delivery technology for the treatment of hyperlipidemia, RBD7022 reduces LDL-C (low-density lipoprotein cholesterol) levels in the blood by inhibiting the expression of PCSK9 (proprotein convertase subtilisin/kexin type 9), decreasing lysosomal degradation of LDL-R (low-density lipoprotein receptor), and increasing the number of LDL-R on the surface of hepatocytes. It is currently undergoing Phase 1 clinical trials in China. The development, manufacturing, and commercialization rights of RBD7022 in China have been licensed to Qilu Pharmaceutical.

RBD1007

An siRNA drug targeting Caspases 2 for optic nerve protection, which inhibits the expression of the target gene through RNAi, prevents the apoptosis of retinal ganglion cells (RGC) and subsequent neuroaxonal degeneration, thereby halting further deterioration of vision and visual field, achieving a therapeutic effect of vision protection. The first clinical indication under development is non-arteritic anterior ischemic optic neuropathy (NAION), for which there is currently no standard clinical treatment. RBD1007 is expected to become the First-In-Class drug for optic nerve protection. Globally, one Phase I clinical study and one Phase II/III international multicenter clinical study (including 34 Chinese subjects) have been completed. Analysis of clinical trial data supports conducting a Phase III confirmatory clinical study in NAION subgroups with significant unmet clinical needs. Plans are in place to explore clinical studies for open-angle glaucoma and other ophthalmic conditions involving optic neuropathy that require optic nerve protection.

RBD7007

As a GalNAc-conjugated siRNA drug developed based on Ribo Life Science's RIBO-GalSTARTM liver-targeted delivery technology and aimed at treating complement-related diseases, RBD7007 can inhibit the expression of a complement component (target undisclosed) in hepatocytes, thereby reducing overactivation of the complement system to achieve therapeutic effects for complement-related diseases. RBD7007 has demonstrated potent and long-lasting efficacy in cynomolgus monkeys, showing Best-In-Class potential. The preparation for submission of the first-in-human clinical trial application to the EU is currently underway.

RBD8088

As the first small nucleic acid drug for glioblastoma developed based on Ribo Life Science's RIBO-OncoSTARTM tumor-targeted delivery platform, RBD8088 can cross the blood-brain barrier and target glioma cells to achieve therapeutic effects. As the world’s first (First-In-Class) small nucleic acid drug targeting glioblastoma, RBD8088 is currently undergoing preclinical research, with plans to initiate its first clinical trial in 2025. It is expected to become a novel, safe, and effective treatment for glioblastoma.

References

1. Official Website of the Company

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