SGB-9768 (C3 siRNA) Receives Clinical Trial Approval in China for the Treatment of Complement-Mediated Diseases

BOSTON, SHANGHAI, and SUZHOU – June 25th, 2024, SanegeneBio announced that the clinical trial application for SGB-9768 injection, an investigational siRNA medicine for the treatment of complement-mediated kidney diseases, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), Immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN), has recently received implicit approval from the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA).

SGB-9768 is an RNAi drug targeting the complement component 3 (C3) protein and was approved for a Phase 1 clinical trial in New Zealand in February 2024. The drug utilizes SanegeneBio's proprietary GalNAc technology to deliver siRNA to liver cells and reduce C3 production through RNAi, thereby inhibiting complement activation and treating complement-mediated kidney diseases. SGB-9768 is designed for administration once every three or six months, offering the advantages of infrequent dosing, improved patient compliance, and durable efficacy. Preclinical studies have demonstrated that SGB-9768 can effectively and sustainably reduce C3 synthesis. Compared with benchmark compounds, it has shown superior efficacy along with favorable safety and tolerability profiles, supporting its potential to become a best-in-class RNAi therapeutic targeting C3.

The complement system is an important component of innate immunity that regulates adaptive immune responses and plays a critical role in maintaining normal immune and physiological functions. It protects the body against infection and facilitates the clearance of dead cells and apoptotic material.

However, dysregulation or excessive activation of the complement system can induce inflammation and damage healthy tissues, resulting in immune-mediated injury. Such abnormalities are closely associated with the development and progression of various hematologic, ophthalmic, and kidney diseases. Chronic kidney disease (CKD) is a major public health challenge in China and worldwide. In China, the prevalence of CKD among adults is approximately 10.8%^[1], meaning that nearly one in ten adults is affected by the disease. Complement activation contributes to the pathogenesis of many kidney diseases, including IgAN, C3G, and IC-MPGN. Currently, treatment options for complement-mediated kidney diseases remain limited, and many complement-targeting therapies are still under clinical investigation, highlighting substantial unmet medical needs in this field. Complement inhibition has emerged as a promising therapeutic strategy for complement-mediated kidney diseases. As a key component linking the upstream activation pathways and terminal pathways of the complement cascade, C3 represents an attractive therapeutic target. Inhibition of C3 activity has been shown to effectively suppress complement activation, making it a compelling target for the treatment of these diseases. Therefore, the development of safe and effective siRNA therapeutics targeting C3 may offer significant clinical value for patients with complement-mediated kidney diseases.

• "Currently, treatment options for complement-mediated kidney diseases remain limited, and there are significant unmet medical needs both in China and globally. A growing body of evidence suggests that SGB-9768, as an innovative siRNA therapeutic, demonstrates potent inhibition of C3 activity and has the potential to become a transformative treatment option for patients with complement-mediated kidney diseases. We also believe it may have broader applications across other complement-related diseases. SanegeneBio will continue to advance the clinical development of SGB-9768 as rapidly as possible and work toward achieving meaningful clinical progress, with the goal of bringing more and better treatment options to patients with immune-related diseases."

said Dr. Weimin Wang, Founder and Chief Executive Officer of SanegeneBio.

[1] Zhang, L.,F. Wang, L. Wang, et al (2012).”Prevalence of chronic kidney disease in China: a cross-sectional survey.” Lancet 379(9818): 815-822.

About SanegeneBio

SanegeneBio is a global, venture-backed, fully-integrated biotechnology company focused on developing RNAi-based therapeutics. Founded in 2021, SanegeneBio is led by a team of RNAi pioneers with unrivaled experience in this Nobel Prize-winning technology. With R&D operations in Boston, Shanghai and Suzhou, our vision clear – RNAi technology will power blockbuster medicines in diverse therapeutic areas, improving the quality and longevity of life for countless patients in the coming years. Our fast-growing pipeline includes experimental medicines for autoimmune nephropathies, obesity, and cardiometabolic indications. SanegeneBio has initiated clinical trials for several experimental medicines, and is committed to developing potential best-in-class and first-in-class therapeutics which leverage our industry-leading and differentiated LEAD™ tissue-selective RNAi delivery technology. For more information, please visit: www.sanegenebio.com and engage with us on LinkedIn.

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