SanegeneBio's C3-Targeting siRNA Drug SGB-9768 Receives FDA Orphan Drug Designation for C3 Glomerulopathy

SANEGENEBIO Was Incubated from Biotop Maker Space to BioBAY in 2022Continue to DevelopSANEGENEBIO is a clinical-stage global biotechnology company focused on the development of RNAi therapies. It has established a multi-pipeline portfolio of innovative drugs targeting obesity, cardiometabolic diseases, and immune-mediated conditions. Through its proprietary LEAD™ (Ligand-Enhanced And Delivery) technology platform, SANEGENEBIO overcomes the challenges of multi-tissue delivery for RNAi drugs, achieving liver/fat/muscle/immune cell targeting, long-lasting gene silencing with a single dose, and demonstrating high efficacy and safety validated by preclinical and clinical studies.

SGB-9768 Granted FDA Orphan Drug Designation for the Treatment of Rare Disease C3G

As a novel RNAi therapy, SGB-9768 has the potential to transform the treatment landscape for C3G, offering patients a new therapeutic option.

Orphan Drug Designation is a policy established by the FDA under the Orphan Drug Act to encourage the development of drugs for rare diseases or conditions. Under this policy, companies that receive orphan drug designation can enjoy various incentives, including tax benefits, FDA research and development guidance, partial registration fee exemptions, and up to 7 years of market exclusivity after the drug's approval. The recent designation of SGB-9768 marks a significant step forward in its research and development efforts in the field of rare disease treatment, which will accelerate its R&D and market launch process, ultimately benefiting patients with C3G who urgently need effective treatment options.

C3G is a rare kidney disease with an incidence rate of 1-2 cases per million people worldwide each year, predominantly affecting young adults. Approximately 50% of patients progress to end-stage renal disease within 10 years, requiring dialysis or kidney transplantation; post-transplant, 50%-70% of patients experience recurrence. Thus, although C3G is rare, it is a progressive disease that severely threatens patient survival and quality of life. The main cause of the disease is overactivation of the complement system; however, previous supportive and immunosuppressive therapies have shown limited efficacy.RNAi Therapy, as an Emerging Technology Capable of Achieving Pathogenic Gene Silencing, Offers a Transformative New Option for the Treatment of C3G with Its Mechanism of Action Intervening at the Source。

SGB-9768 is the first domestically produced innovative small interfering RNA (siRNA) drug independently developed by SANEGENEBIO in China, targeting complement C3. It utilizes the company's proprietary GalNAc liver-targeting delivery technology and specifically inhibits C3 expression through the RNAi mechanism, thereby suppressing the overactivation of the complement system at its source. Phase I clinical trial data show that after a single subcutaneous injection, SGB-9768 demonstrated good safety and tolerability, with a dose-dependent, significant, and sustained reduction in C3 protein levels and complement pathway activity. Compared with other siRNA products targeting the same site, SGB-9768 achieves higher target protein knockdown and maintains the knockdown level for a longer duration at the same dose, showcasing potential "best-in-class" advantages.

In addition to being developed for the treatment of C3G, SGB-9768 also has broad application potential. Its indications can be expanded to other complement-mediated diseases, including the larger patient populations with primary IgA nephropathy (IgAN) and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN). Currently, SGB-9768 is undergoing a multicenter Phase II clinical trial.

Dr. Wang Weimin, founder and CEO of SANEGENEBIO, said:“SGB-9768 Receives FDA Orphan Drug Designation: A Significant Recognition of Its Innovative Mechanism and Therapeutic PotentialWe believe SGB-9768 has the potential to address the urgent medical needs of patients with C3G. In response to the limited long-term treatment options available for these patients, we are committed to leveraging our cutting-edge RNAi technology platform to develop potentially transformative therapies. Moving forward, we will work closely with global regulatory agencies to advance the clinical development of SGB-9768, with the goal of bringing new hope to patients as soon as possible.”