Hutchmed is preparing to file for approval of a drug for the rare autoimmune disorder warm antibody autoimmune haemolytic anaemia (wAIHA) in China, after chalking up a win in a pivotal phase 2/3 trial.

和黄医药正准备在中国为其用于治疗罕见的自身免疫性疾病——温抗体型自身免疫性溶血性贫血（wAIHA）的药物申请批准，此前该药物在一项关键的2/3期试验中取得了成功。

The Hong Kong biopharma company has reported topline results from the 100-subject ESLIM-02 trial of sovleplenib, an oral Syk inhibitor, and said it hopes to file for approval as a treatment for wAIHA with China's National Medical Products Administration (NMPA) in the first half of this year.

这家香港生物制药公司报告了口服Syk抑制剂索伐普利尼（sovleplenib）的100名受试者ESLIM-02试验的初步结果，并表示希望在今年上半年向中国国家药品监督管理局（NMPA）提交该药物用于治疗wAIHA的上市申请。

In wAIHA, the immune system erroneously produces antibodies that attack and destroy the body's own healthy red blood cells. It causes symptoms like fatigue, weakness, and shortness of breath, and can be a life-threatening condition if not managed well.

在温抗体型自身免疫性溶血性贫血 (wAIHA) 中，免疫系统错误地产生攻击并破坏体内健康红细胞的抗体。它会引起疲劳、虚弱和呼吸急促等症状，如果管理不当，可能会危及生命。

wAIHA form is the most common form of autoimmune haemolytic anaemia, accounting for up to 80% of all adult cases, and is generally treated using corticosteroids, immunosuppressant medicines, and anti-CD20 antibody rituximab.

温抗体型自身免疫性溶血性贫血是最常见的自身免疫性溶血性贫血形式，占所有成人病例的80％，通常使用皮质类固醇、免疫抑制剂和抗CD20抗体利妥昔单抗进行治疗。

ESLIM-02 showed that, compared to placebo, sovleplenib was more effective at achieving a durable haemoglobin response rate in the five to 24 weeks after starting treatment.

ESLIM-02 显示，与安慰剂相比，索夫勒普利尼在开始治疗后的五到二十四周内实现持久的血红蛋白反应率方面更有效。

Results from the phase 2 part of the study, published in

已发表的该研究第二阶段的结果

The Lancet Haematology

《柳叶刀·血液学》

in January 2025 revealed an overall haemoglobin response rate of 43.8% for sovleplenib in the first eight weeks, with a zero rate for placebo, rising to 66.7% over the 24 weeks.

2025年1月公布的数据显示，sovleplenib在前八周的总体血红蛋白反应率为43.8%，而安慰剂组为零，在24周内该比率上升至66.7%。

'The positive topline results from ESLIM-02 highlight sovleplenib's potential to deliver rapid and durable haemoglobin responses in wAIHA patients who have limited options after failing standard therapies,' commented Prof Fengkui Zhang of the Chinese Academy of Medical Sciences Blood Diseases Hospital, one of the study's principal investigators..

“ESLIM-02的积极顶线结果凸显了sovleplenib在对标准治疗失败后选择有限的wAIHA患者中实现快速且持久的血红蛋白反应的潜力，”中国医学科学院血液病医院的张凤奎教授评论道，他是该研究的主要研究者之一。

'This could represent a meaningful advancement for managing this challenging condition,' he added.

“这可能代表了管理这种棘手状况的一个有意义的进步，”他补充道。

Hutchmed is also developing sovleplenib for chronic immune thrombocytopenia (ITP), another disease characterised by the destruction of blood cells – in this case, platelets – although progress in that indication has been held up by a manufacturing issue that led to a marketing application in China being withdrawn.

和黄医药还在开发索乐匹尼布用于治疗另一种以血细胞破坏为特征的疾病——慢性免疫性血小板减少症 (ITP)，但该药在这一适应症上的进展因生产问题而受阻，导致其在中国的上市申请被撤回。

The company has said it plans to resubmit the drug for approval in ITP in the second quarter of 2026..

该公司表示，计划在2026年第二季度重新提交该药物用于ITP的审批。

Another Syk inhibitor, Rigel/Grifols' Tavalisse/Tavlesse (fostamatinib), is already on the market for ITP but failed to show significant efficacy in a phase 3 wAIHA study reported in 2022.

另一种Syk抑制剂，Rigel/Grifols的Tavalisse/Tavlesse（福斯塔马替尼），已经上市用于治疗ITP，但在2022年报告的一项针对wAIHA的三期研究中未能显示出显著疗效。

At the moment, there are no therapies specifically for wAIHA, although various other candidates are in clinical development for the rare disorder, including Sanofi's BTK inhibitor Wayrilz (rilzabrutinib), Johnson & Johnson's anti-FcRn antibody Imaavy (nipocalimab), and Zenas BioPharma's CD19xFcγRIIb bispecific antibody obexelimab..

目前，虽然有其他多种候选药物正在针对这种罕见病进行临床开发，但尚无专门针对温抗体型自身免疫性溶血性贫血（wAIHA）的疗法，这些候选药物包括赛诺菲的BTK抑制剂Wayrilz（rilzabrutinib）、强生的抗FcRn抗体Imaavy（nipocalimab），以及Zenas BioPharma的CD19xFcγRIIb双特异性抗体obexelimab。