Tides Express | SanegeneBio Announces Clinical Progress of SGB-9768 (C3 siRNA)

Study Design and Patient Baseline

The Phase I clinical trial data disclosed this time integrate the results of the SGB-9768-001 study (hereinafter referred to as "001 study") conducted in New Zealand and the SGB-9768-002 study (hereinafter referred to as "002 study") conducted in China. Both studies are randomized, double-blind, placebo-controlled, single-ascending-dose studies conducted in healthy subjects, aiming to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) profiles of SGB-9768 after subcutaneous administration.

As of September 30, 2025, a total of 74 healthy subjects (29 in New Zealand, 45 in China) were enrolled in two studies, completing the exploration of seven different doses. Study 001 included five dose cohorts (25 mg, 50 mg, 100 mg, 200 mg, 400 mg); Study 002 included six dose cohorts (50 mg, 100 mg, 200 mg, 400 mg, 600 mg, 800 mg).

The baseline characteristics of the subjects were consistent with the population characteristics of the respective study locations: Study 001 was predominantly White, and Study 002 was entirely Chinese. In both studies, the internal baseline characteristics of all cohorts were generally balanced.

After a single administration of SGB-9768, serum C3 levels showed a dose-dependent, significant, and sustained reduction, while the alternative complement pathway activity was almost completely inhibited.

• Study 001 (New Zealand): In the 400 mg dose group, serum C3 levels reached the maximum reduction at approximately Week 4, with an average maximal knockdown of 86.8%. The inhibition of the complement alternative pathway activity reached 100%, which was sustained until Week 24.

• Study 002 (China): In the 600 mg dose group, the serum C3 level also reached its maximum reduction at approximately week 4, with an average maximum knockdown of 96.3%. The alternative complement pathway activity was inhibited by 100%, which could be maintained until week 24.

• Overall, within the dose range of 50–400 mg, the reductions in C3 and alternative complement pathway activity observed in Study 002 were slightly greater than those in Study 001.

Good safety and tolerability

• In two Phase I studies, SGB-9768 demonstrated good safety and tolerability.

• No dose-limiting toxicity, no drug-related serious adverse events, or adverse events leading to withdrawal from the study occurred in the research.

• Phase I study's favorable safety data supports the further clinical development of SGB-9768.

The complement system is an important component of innate immunity, playing a crucial role in protecting the body from infections, clearing dead cells, and removing apoptotic materials. However, once the complement system is dysregulated or excessively activated, it can induce inflammation and damage self-tissues, causing immune injury, which is closely related to the occurrence and progression of various kidney diseases. Complement-mediated kidney diseases mainly include IgAN and C3G. IgAN is the most common primary glomerular disease in China, with a large patient population of about 4 million, predominantly occurring in young and middle-aged adults. This disease is also the most common cause of end-stage renal disease in China. Studies have shown that approximately half of the patients are accompanied by...Persistent ProteinuriaPatients with lgAN will progress to end-stage renal disease within 10-20 years after diagnosis and require dialysis or kidney transplantation. C3G is a rare kidney disease, with a global incidence of approximately 1-2 cases per million people annually, and it is also commonly seen in young adults. The disease progresses rapidly, with about 50% of patients progressing to end-stage renal disease within 10 years of diagnosis; the recurrence rate after kidney transplantation is as high as 50%-70%, making clinical management extremely challenging. Both lgAN and C3G are closely related to overactivation of the complement system, facing severe challenges such as rapid disease progression and limited effective treatment options, significantly affecting patients' long-term survival and quality of life.

AboutSGB-9768

SGB-9768 is the first domestically produced innovative siRNA drug independently developed by SANEGENEBIO that targets complement C3 in China. The drug utilizes the company's proprietary GalNAc liver-targeting delivery technology and specifically inhibits C3 expression through the RNA interference (RNAi) mechanism, thereby suppressing the overactivation of the complement system at its source. It provides a novel therapeutic option for various complement-mediated kidney diseases, including IgAN, C3G, and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN). Phase I clinical trial data show that after a single subcutaneous injection, SGB-9768 demonstrated good safety and tolerability, with dose-dependent, significant, and long-lasting reductions in C3 protein levels and complement pathway activity. Compared to other siRNA products targeting the same site, SGB-9768 achieves higher target protein knockdown at the same dose and maintains the knockdown effect longer, showing "best-in-class" potential. Due to its remarkable long-acting properties, SGB-9768 has the potential for dosing once every three or six months, greatly enhancing patient medication convenience. Currently, the drug is undergoing Phase II clinical trials in China and has been granted orphan drug designation by the U.S. FDA for the treatment of C3G.

AboutSANEGENEBIO

SANEGENEBIO is a globally integrated clinical-stage biotechnology company focused on the development of RNAi therapies. Leveraging its proprietary GalNAc liver delivery platform and the industry-leading LEAD™ (Ligand and Enhancer Assisted Delivery) extrahepatic delivery platform, the company is committed to addressing significant unmet clinical needs in the fields of obesity, cardiometabolic diseases, and immune-mediated disorders.

Source:

1. SANEGENEBIO