SanegeneBio Announces Positive Phase I Clinical Data for SGB-9768, a First-in-Class C3-Targeting siRNA Therapeutic

December 202410DaySANEGENEBIO Presents Positive Preclinical and Partial Phase I Clinical Trial Results for SGB-9768, a Small Nucleic Acid (siRNA) Drug Targeting Complement Factor C3, at the 8th Complement Drug Development Conference in Boston, USA

SANEGENEBIO Conducts a Phase I Study in New Zealand and China: A Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SGB-9768 in Healthy Subjects. The first participant dosing was completed in New Zealand and China in May and August 2024, respectively. As of December 1, 2024, a total of 55 participants were randomly assigned to receive either SGB-9768 or placebo treatment. Trial data indicate that after a single subcutaneous injection, SGB-9768 demonstrated favorable safety and tolerability, along with dose-dependent, significant, and sustained reductions in C3 levels and complement pathway activity. Compared with other siRNA products targeting the same pathway, SGB-9768 shows efficacy at lower doses.Higher knockdown efficiency of target protein C3.

About Complement-Mediated Kidney Diseases

The complement system is an important component of innate immunity, playing a regulatory role in adaptive immune responses and performing crucial immune and physiological functions in the human body. It protects the body from infection and clears dead cells and apoptotic substances. However, once the complement system is dysregulated or excessively activated, it can induce inflammation and damage self-tissues, causing immune injury. This is closely related to the occurrence and development of some blood diseases, ophthalmic diseases, and kidney diseases. Chronic kidney disease is a major public health issue globally and in China, with a prevalence rate of 10.8% in China, meaning one in every ten adults suffers from chronic kidney disease. Many kidney diseases involve complement-mediated pathogenesis, including IgA nephropathy, C3 glomerulopathy, and immune complex-mediated membranoproliferative glomerulonephritis. Currently, treatment progress for complement-mediated kidney diseases is limited, with many complement-targeting drugs still in clinical research stages, indicating significant unmet clinical needs in this field. Complement inhibition is expected to become a new therapeutic target for treating complement-mediated kidney diseases. Complement C3 is a critical component linking upstream activation pathways and terminal pathways in the complement system. Inhibition of C3 activity has been shown to significantly suppress the complement system, making it a potent therapeutic target for such diseases. Therefore, developing safe and effective siRNA drugs targeting complement C3 holds substantial clinical application value for complement-mediated kidney diseases.

AboutSGB-9768

SGB-9768 is a siRNA drug independently developed by SANEGENEBIO that targets the Complement 3 (C3) protein. It utilizes the company's uniquely innovative next-generation LEAD™ GalNAc technology for delivery to liver cells and inhibits C3 synthesis through the RNAi mechanism, thereby suppressing complement activation. It is intended for the treatment of complement-mediated kidney diseases, including adult IgA nephropathy, C3 glomerulopathy, and immune complex-mediated membranoproliferative glomerulonephritis. Preclinical trial data shows that SGB-9768 can be administered once every 3 or 6 months, effectively and continuously reducing C3 synthesis. Compared to competing products, it demonstrates superior efficacy and good safety tolerance. With advantages such as lower treatment frequency, better patient compliance, and long-lasting effects, it has the potential to become China's first and world-leading siRNA drug targeting Complement C3.

AboutSANEGENEBIO

SANEGENEBIO, founded in early 2021, is a biopharmaceutical company dedicated to developing novel small nucleic acid drugs based on RNA interference (RNAi) technology, with research and development centers in both China and the United States. The founding team consists of senior experts in the field of nucleic acid drugs, possessing years of experience in nucleic acid drug development and cutting-edge technical capabilities within the industry. SANEGENEBIO has established a globally leading nucleic acid drug platform with proprietary intellectual property, featuring chemical modification and delivery technologies for both intrahepatic and extrahepatic applications, enabling more efficient knockdown of disease-causing genes. The company has rapidly advanced the research and development of a series of small nucleic acid products covering cardiovascular and cerebrovascular diseases, metabolic disorders, immune-related diseases, and neurological conditions. SANEGENEBIO is committed to scientific innovation, creating novel RNAi therapeutics to address unmet global medical needs and benefit patients and their families.