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On Monday,
周一,
Arrowhead Pharmaceuticals, Inc.
箭头制药公司
ARWR
ARWR
released topline results from Part 2 of a Phase 1/2 study of ARO-C3, the company’s investigational RNA interference (RNAi) therapeutic designed to reduce liver production of complement component 3 (C3) as a potential therapy for various
公布了ARO-C3的1/2期研究第二部分的顶线结果,该公司的研究性RNA干扰(RNAi)治疗药物旨在减少肝脏补体成分3(C3)的生成,作为各种疾病的潜在疗法。
complement-mediated diseases
补体介导的疾病
.
。
ARO-C3 is designed to reduce hepatocyte production of complement C3 as a potential treatment for various complement-mediated renal diseases.
ARO-C3旨在减少肝细胞产生补体C3,作为治疗各种补体介导的肾脏疾病的潜在方法。
The dysregulated activity of the complement system can contribute to tissue injury and the progression of the disease. By silencing C3, investigational ARO-C3 has the potential to treat complement-mediated renal diseases by modulating the activation of the complement cascade.
补体系统的失调活动可能导致组织损伤和疾病进展。通过沉默C3,研究中的ARO-C3有可能通过调节补体级联的激活来治疗补体介导的肾脏疾病。
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Veren因与Whitecap的合并交易而飙升:详情
The company plans to present additional results at a
公司计划在会议上展示更多结果,
medical meeting in 2025
2025年的医学会议
.
。
Select Phase 1/2 Study Results
选择第一/二期研究结果
Pharmacodynamic effects: Maximum mean reduction in C3 of 89% and mean sustained reduction of greater than 87% from baseline through week 24.
药效学效应:C3最大平均降低89%,从基线到第24周平均持续降低超过87%。
Maximum mean reduction in serum AH50 (alternative pathway hemolytic assay) of 85% and mean sustained reduction greater than 76%.
血清AH50(替代途径溶血试验)最大平均降低85%,平均持续降低大于76%。
Maximum mean reduction in Wieslab AP (alternative pathway) of 100% and mean sustained reduction greater than 89%.
Wieslab AP(替代途径)最大平均减少100%,平均持续减少大于89%。
Duration of effect supportive of once every three months or less frequent subcutaneous dosing in later-stage studies
在后期研究中,支持每三个月或更少频率的皮下给药的效果持续时间
Effects on proteinuria (too much protein in the urine): The mean reduction in spot UPCR was 41%, and the maximum individual reduction was 89%.
对蛋白尿(尿液中蛋白质过多)的影响:斑点尿蛋白肌酐比值 (UPCR) 的平均降幅为 41%,最大个体降幅为 89%。
In January, the U.S. Food and Drug Administration accepted Arrowhead Pharmaceuticals’ New Drug Application for investigational plozasiran for familial chylomicronemia syndrome, a severe and rare genetic disease. The FDA provided a Prescription Drug User Fee Act (PDUFA) action date of November 18, 2025, and indicated it is not currently planning to hold an .
今年 1 月,美国食品和药物管理局接受了 Arrowhead Pharmaceuticals 针对家族性乳糜微粒血症综合征(一种严重且罕见的遗传病)研究性药物 plozasiran 的新药申请。FDA 提供了 2025 年 11 月 18 日作为《处方药使用者费用法案》(PDUFA)的行动日期,并表示目前不打算召开 。
advisory committee meeting
顾问委员会会议
.
。
In February, Arrowhead Pharmaceuticals closed a global licensing and collaboration agreement with
今年 2 月,Arrowhead Pharmaceuticals 与
Sarepta Therapeutics Inc
萨雷普塔治疗公司
. Arrowhead receives a $500 million upfront payment and $325 million
Arrowhead获得5亿美元的预付款和3.25亿美元
through Sarepta stock
通过萨雷普塔股票
.
。
Price Action:
价格行为:
ARWR stock is down 8.72% at $15.49 at the last check Monday.
ARWR股票在周一最后一次检查时下跌了8.72%,至15.49美元。
Read Next:
接下来阅读:
Pfizer’s COVID-19 Shot Partner BioNTech Reports Q4 Profit, But 2025 Guidance Falls Short On Expectations
辉瑞新冠疫苗合作伙伴BioNTech公布第四季度盈利,但2025年预期指引未达预期
Photo via Shutterstock.
通过Shutterstock拍摄的照片。
ARWR
ARWR
Arrowhead Pharmaceuticals Inc
箭头制药公司
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Sarepta Therapeutics Inc
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