Abstract: TH-PO1255
A Randomized, Placebo-Controlled Phase 2 Trial of a Long-Acting, C3-Targeting siRNA in IgA Nephropathy
Session Information
- Late-Breaking Research Posters
October 22, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Pharmacology (PharmacoKinetics, -Dynamics, -Genomics)
- 1900 Pharmacology (PharmacoKinetics, -Dynamics, -Genomics)
Authors
- Lv, Jicheng, Peking University First Hospital, Beijing, China
- Chang, Jie, The First Affiliated Hospital of Henan University of Science and Technology, Luoyang, Henan, China
- Wu, Henglan, Jiaxing First Hospital, Jiaxing, China
- Yan, Rui, Guizhou Medical University Affiliated Hospital, Guiyang, China
- Hou, Shuang, Peking University Shenzhen Hospital, Shenzhen, Guangdong, China
- Li, Guisen, Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital, Chengdu, China
- Luo, Qun, Ningbo No.2 Hospital, Ningbo, China
- Qi, Hualin, Shanghai Pudong Hospital, Shanghai, China
- Zhu, Lingling, Suzhou Rigerna Therapeutics, Ltd., Suzhou, China
- Yang, Julia, Suzhou Rigerna Therapeutics, Ltd., Suzhou, China
- Du, Zhiqiang, Suzhou Rigerna Therapeutics, Ltd., Suzhou, China
- Li, Haitao, Suzhou Rigerna Therapeutics, Ltd., Suzhou, China
- Huang, Yuanyu, Suzhou Rigerna Therapeutics, Ltd., Suzhou, China
- Zhang, Hong, Peking University First Hospital, Beijing, China
Background
Complement activation contributes to kidney injury and progression in immunoglobulin A Nephropathy (IgAN). RG002C0106 is a Complement C3 siRNA molecular for sustained complement modulation with infrequent dosing. We report clinical results from the first randomized, placebo-controlled Phase 2 trial study(NCT07305974)of a C3-targeting siRNA in IgAN.
Methods
This study enrolled 34 adults with biopsy-confirmed IgAN into a 24-week randomized, placebo-controlled period, followed by a 96-week open-label extension. Participants were randomized the ratio of 4:1 receiving RG002C0106 regimens and placebo. An independent third party conducted the interim analysis and provided the summary result.
Results
RG002C0106 demonstrated a remarkably rapid onset of action, with response evident by 2 weeks, and deepening through 12 weeks. Placebo-adjusted mean 24h uPCR reductions were 50% in the low-dose regimen and 52%–67% across three high-dose regimens. Serial first-morning-void uPCR showed a clear downward trend. Notably, significant hematuria improvements occurred at Week 12. RG002C0106 was generally well tolerated, without serious adverse events or serious infections as of the cutoff.
Conclusion
This is the 1st randomized, placebo-controlled Phase 2 Trial and show rapid proteinuria reduction by 2 weeks and a deep response reaching up to 60% at 12 weeks (results of 24 weeks will update on Oct 15th,2026) . The rapid onset, depth of response, preliminary safety profile, and infrequent dosing may differentiate RG002C0106 from existing IgAN therapies. RG002C0106 could provide a foundational complement-targeted treatment in IgAN treatment paradigm.
Funding
- Commercial Support – Rigerna Therapeutics (Beijing) Co., Ltd. Rigerna Therapeutics Co., Ltd.