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更新于：2026-06-12
Sefaxersen
更新于：2026-06-12
概要
基本信息
非在研机构- |
最高研发阶段临床3期 |
首次获批日期- |
最高研发阶段(中国)临床3期 |
特殊审评孤儿药 (欧盟)、孤儿药 (日本) |
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结构/序列
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Sequence Code 547591869
来源: *****
关联
9
项与 Sefaxersen 相关的临床试验ISRCTN10221007
A randomized, open-label, single-dose, parallel-group study to investigate the bioequivalence of sefaxersen in healthy adult participants following subcutaneous administration via an injection device or vial and syringe
CTIS2023-508270-29-01
A Study to Evaluate the Effects of Various Degrees of Hepatic Impairment on the Pharmacokinetics of A Single Subcutaneous Dose Of RO7434656, an Antisense Inhibitor of Complement Factor B
CTR20242994
一项在中国健康成人受试者中考察RO7434656 单次皮下给药的药代动力学、药效学、安全性和耐受性的I 期、开放、平行研究
100 项与 Sefaxersen 相关的临床结果
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100 项与 Sefaxersen 相关的转化医学
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100 项与 Sefaxersen 相关的专利（医药）
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5
项与 Sefaxersen 相关的文献（医药）2026-03-01KIDNEY INTERNATIONAL
A single-arm phase 2 trial of an investigational RNA therapeutic to complement factor B sefaxersen for treatment of IgA nephropathy
Article
作者: Garg, Jay P ; McCaleb, Michael L ; Yin, Lixuan ; Robson, Richard ; Schneider, Eugene ; Geary, Richard ; Hladunewich, Michelle A ; Brice, Gary T ; Frazer-Abel, Ashley ; Barrett, Terrance D ; Barbour, Sean J
INTRODUCTION:
Activation of the complement system and subsequent local inflammation in the kidney plays a key role in the pathogenesis of IgA nephropathy (IgAN). Here, we investigated the efficacy and safety of sefaxersen, an antisense oligonucleotide inhibitor of complement factor B (FB), for the treatment of IgAN in a global exploratory, single arm, open-label trial (NCT04014335).
METHODS:
Patients were included with biopsy-confirmed IgAN with kidney C3 deposits, hematuria, 24-hour proteinuria above 1.5 g/day, and eGFR under 40mL/min/1.73m2 despite maximum tolerated renin-angiotensin-aldosterone-system blockade. Patients received sefaxersen 70 mg (RO7434656) subcutaneously monthly for 24 weeks followed by voluntary treatment extension. The primary endpoint was a change in 24-hour urinary protein excretion (UPE) at week 29 compared to baseline.
RESULTS:
The trial enrolled 23 patients with baseline geometric mean proteinuria of 2.5 g/day. There were selective reductions of plasma complement FB and Bb, urinary factor Ba and serum complement alternative pathway activity, without changes in classical pathway activity. At week 29, UPE was reduced by 43% to a geometric mean of 1.4 g/day, with similar reductions in UPCR and UACR. Estimated GFR at baseline was mean 70.4 ml/min/1.73m2, remained stable through week 29 (mean 73.2 ml/min/1.73m2). Proteinuria reduction was sustained in all seven participants who opted to participate in the treatment extension, including four patients treated for over 12 months. There was one treatment emergent serious adverse event not related to study drug. Transient and reversible alanine amino transferase elevations (3-5X fold upper limit normal) without a change in bilirubin were observed in three individuals, who remained on study and completed treatment.
CONCLUSIONS:
Sefaxersen inhibited complement alternative pathway activity in patients with IgAN and reduced proteinuria with stable eGFR. Our findings support further evaluation of sefaxersen as a new therapy for IgAN in the Phase 3 IMAGINATION trial.
TRIAL REGISTRATION:
Registered at ClinicalTrials.gov with study number NCT04014335.
2026-03-01KIDNEY INTERNATIONAL
The expanding role of complement inhibitors in the treatment of IgA nephropathy
Article
作者: Praga, Manuel ; Caravaca-Fontán, Fernando
Complement activation has emerged as a key driver of IgA nephropathy, catalyzing the rapid expansion of complement-targeted therapies. Barbour et al. reported promising phase 2 results of sefaxersen, an antisense inhibitor of Factor B, showing reduced proteinuria, improved hematuria, and sustained alternative-pathway inhibition. Nonetheless, as the therapeutic landscape expands, advancing precision medicine in IgA nephropathy will require defining appropriate patient profiles and predictors of therapeutic response to guide individualized treatment.
2025-03-01IMMUNOBIOLOGY
Inhibiting the alternative pathway of complement by reducing systemic complement factor B: Randomized, double-blind, placebo-controlled phase 1 studies with Sefaxersen
Article
作者: Yin, Lixuan ; Henry, Scott P ; Monia, Brett P ; Geary, Richard ; Schneider, Eugene ; Grossman, Tamar R ; Hughes, Steven G ; Brice, Gary T ; Jung, Bill ; Frazer-Abel, Ashley ; McCaleb, Michael L
An over-active alternative complement pathway has been implicated in the pathophysiology of multiple diseases, including IgA nephropathy and geographic atrophy secondary to age related macular degeneration. In first-in-human double-blind, placebo-controlled phase 1 studies, the safety and pharmacodynamic effects of sefaxersen (RO7434656), a GalNAc-conjugated 2'-MOE antisense oligonucleotide targeting the complement factor B mRNA, was investigated. Healthy volunteers received either single or repeated (for 6 weeks) subcutaneous administrations of investigational drug or placebo. Safety and plasma complement protein levels were assessed throughout the studies and during 90-day follow-up periods. All subjects (54) completed the studies and no safety signals or clinically meaningful changes in blood chemistry, urinalysis, hematology, ECG, vital signs or ocular endpoints were observed. Mean levels of systemic complement factor B (FB) were reduced up to 38 % after single administration and 69 % after repeated administration. Lowering of FB protein was paralleled by similar reductions of plasma Bb levels. There was a strong correlation between reduction of plasma levels of FB and alternative complement pathway activity (AH50), but no meaningful changes in classical complement pathway activity (CH50). The long duration of lowering of FB levels following the last dose supports monthly dosing in future clinical trials. These clinical results support the ongoing Phase 2 development for geographic atrophy secondary to age-related macular degeneration and Ph 2/3 development for IgA nephropathy.
100 项与 Sefaxersen 相关的药物交易
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研发状态
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 免疫球蛋白a肾病 | 临床3期 | 澳大利亚 | 2022-07-11 | |
| 免疫球蛋白a肾病 | 临床3期 | 加拿大 | 2022-07-11 | |
| 免疫球蛋白a肾病 | 临床3期 | 新西兰 | 2022-07-11 | |
| 年龄相关性黄斑变性 | 临床2期 | 美国 | 2018-03-16 | |
| 年龄相关性黄斑变性 | 临床2期 | 澳大利亚 | 2018-03-16 | |
| 年龄相关性黄斑变性 | 临床2期 | 新西兰 | 2018-03-16 | |
| 地图样萎缩 | 临床2期 | 美国 | 2018-03-16 | |
| 地图样萎缩 | 临床2期 | 澳大利亚 | 2018-03-16 | |
| 地图样萎缩 | 临床2期 | 新西兰 | 2018-03-16 |
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临床结果
临床结果
适应症
分期
评价
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临床2期 | 23 | 膚選選夢艱網繭願餘齋(鹹淵繭製觸鹽淵範製繭) = 遞鹽鹽夢襯鹽鹹艱壓淵 醖鑰積網獵膚齋蓋艱壓 (齋遞積積顧獵顧衊糧鏇 ) 更多 | 积极 | 2025-12-01 | |||
临床2期 | 23 | 網壓蓋廠鹹糧鑰積鹹觸(齋鏇構築廠鬱範鑰糧廠) = 艱膚衊膚範構顧夢鏇齋 獵簾顧蓋蓋製顧憲繭壓 (襯夢鏇壓觸齋遞鏇壓築, 23 ~ 58) 更多 | 积极 | 2024-10-24 | |||
临床2期 | 19 | 鏇願膚衊網顧淵膚鬱醖(簾觸窪鹽糧鑰夢願遞網) = 鏇遞願鹽廠膚簾顧憲醖 繭鹹遞壓憲膚遞鹽網廠 (衊積構顧窪淵範簾構選, 1.23 ~ 2.33) 更多 | 积极 | 2024-05-23 | |||
临床2期 | 13 | 淵壓衊願積艱淵顧鬱憲(廠憲鹽餘遞遞廠窪積餘) = 鑰顧積廠憲膚獵遞襯網 鹽餘廠鹽艱顧糧顧積選 (淵顧蓋鹹遞簾襯艱積繭 ) 更多 | 积极 | 2023-11-04 | |||
临床2期 | 10 | 壓選獵鹽壓夢淵淵齋簾(積廠糧範醖願願鏇艱顧) = 築餘醖鏇築夢願網選鏇 製壓選齋築糧積蓋鹽壓 (製簾鬱網鹹淵鑰顧積廠, -1.68 ~ -0.79) | 积极 | 2022-11-05 | |||
临床1期 | 54 | 窪積鏇築簾鬱憲壓構鏇(膚醖鹽構簾構選遞壓衊) = 範網遞築齋觸壓願憲餘 鬱蓋鏇淵簾繭淵糧廠壓 (鏇製構願簾構醖廠糧鑰 ) | 积极 | 2021-06-01 | |||
窪積鏇築簾鬱憲壓構鏇(膚醖鹽構簾構選遞壓衊) = 積齋簾繭範鹹構鑰膚網 鬱蓋鏇淵簾繭淵糧廠壓 (鏇製構願簾構醖廠糧鑰 ) 更多 | |||||||
临床2期 | 地图样萎缩 complement factor B | 54 | 觸餘範鑰蓋鹽鹹艱夢艱(選鬱獵構齋鬱顧遞構憲) = 廠簾鏇簾簾築築襯襯壓 構襯廠衊艱艱鹹壓製鹹 (窪醖鬱糧窪蓋構簾蓋遞 ) 更多 | 积极 | 2020-06-01 |
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