NEJM发布遗传性肝病RNAi疗法2期临床试验结果,降低致病蛋白83%

2022-06-30 王聪 “生物世界”公众号

患者肝脏中 Z-AAT 降低幅度中位数达83%,且肝脏健康相关生物标志物得到了显著且持续改善,高剂量组(200mg)的12位患者中有7人观察到肝脏纤维化消退。

α1-抗胰蛋白酶缺乏症(Alpha1-Antitrypsin Deficiency,AATD)是一种常染色体共显性遗传病,α1-抗胰蛋白酶(AAT)由肝脏细胞中的 SERPINA1 基因编码并分泌到循环系统中,通过其抗蛋白酶活性保护肺组织。SERPINA1 基因突变(单个氨基酸突变Glu342Lys)会导致错误折叠的α1-抗胰蛋白酶(Z-AAT)在肝脏中的积累,同时也让肺部缺乏正常的 AAT,前者会导致肝脏纤维化和肝硬化(20%-40%的患者会出现肝硬化,终末期只能靠肝移植治疗),后者会导致肺气肿、肺癌。

近日,国际顶尖医学期刊《新英格兰医学杂志》(NEJM)发表了题为:Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency 的临床试验论文。

这项2期临床试验旨在确定 Fazirsiran(RNAi疗法)在治疗 AAT 缺乏相关肝病患者中的安全性和有效性。Fazirsiran 是一款由 GalNAc 递送 siRNA 的特异性靶向肝脏的 RNAi 疗法,通过靶向降解 Z-AAT 的 mRNA,从而减少 Z-AAT 蛋白合成,缓解肝脏损伤。

这项2期临床试验显示,患者肝脏中 Z-AAT 降低幅度中位数达83%,且肝脏健康相关生物标志物得到了显着且持续改善,高剂量组(200mg)的12位患者中有7人观察到肝脏纤维化消退。

这项2期临床试验,共16名成年患者参与,他们均为 SERPINA1 基因的 Glu342Lys 纯合突变,即产生 Z-AAT 蛋白,且已出现肝纤维化症状。这16名患者被分为3个队列,分别是队列1(人,接受200mg剂量)、队列2(8人,接受200mg剂量)、队列1b(4人,接受100mg剂量)。他们分别在第0天和第4周皮下注射 Fazirsiran,然后每12周注射一次。主要终点是肝脏中 Z-AAT 浓度从基线到第24周时的变化(队列1和队列1b)或从基线到第48周时的变化(队列2)。

试验结果显示,3个队列的所有16名患者的肝脏中 Z-AAT 蛋白水平均显着降低,在第24周或第48周时,肝脏中 Z-AAT 降低幅度中位数为83.3%。血清中 Z-AAT 水平最多下降了约90%。此外,所有患者的肝脏中 Z-AAT 突变蛋白负担也得到了显着降低。患者肝脏炎症情况也到了显着改善。

接受高剂量(200mg)治疗的12名患者中,有7名患者的肝脏纤维化得到了消退(其中还包括2名肝硬化患者)。且所有患者均未出现导致停药的严重不良事件。

这项2期临床试验数据表明,基于 RNAi 的 Fazirsiran 能够显着改善 AAT 缺乏相关肝病患者的多个健康相关生物标志物,能够可靠且持续地沉默突变基因的表达,对患者健康产生积极影响。基于这些数据,武田公司和 Arrowhead 将启动3期临床试验。

Fazirsiran 最初由 Arrowhead Pharmaceuticals(NASDAQ: ARWR)开发,2020年10月,武田公司( TSE:4502/NYSE:TAK)与其达成合作开发协议,根据协议条款,五天支付了3亿美元预付款,Arrowhead 还有资格获得高达7.4亿美元的后续里程碑付款。双方共同开发 Fazirsiran,如果获得批准,双方将在美国市场50/50分配销售收入,在美国以外市场,Arrowhead 有资格获得销售额的20%-25%。

关于 RNAi 疗法

RNAi(RNA干扰,也叫RNA沉默)是由斯坦福大学的 Andrew Fire 教授和麻省大学的 Craig Mello 教授发现的细胞内的一种基因表达调控机制。这一发现于2006年获得了诺贝尔生理学或医学奖。

通过特异性靶向信使RNA(mRNA),实现对相应蛋白质表达的抑制,2006年,RNAi机制的发现者斯坦福大学的Andrew Fire和麻省大学的Craig Mello荣获诺贝尔生理学或医学奖。

RNAi 通过特异性靶向信使RNA(mRNA),实现对相应蛋白质表达的抑制,可用来调控与疾病相关的基因的表达,因此被认为是一种有前途的治疗工具,尤其是那些使用传统方法难以成药的基因。

2018年,FDA 批准了 Alnylam 公司的 RNAi 疗法——Patisiran。用于遗传性ATTR淀粉样变性成人患者第1阶段或第2阶段多发性神经病的治疗。这是全球第一个 RNAi 药物,标志着 RNAi 治疗时代的到来。截至目前,FDA 共批准了5款 RNAi 药物上市,均来自 Alnylam 公司(其中1款为 Alnylam 与诺华合作开发)。

 

原始出处:

Pavel Strnad, et al. Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency. NEJM, 2022.

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    2022-06-30 小元

    NEJM上果然牛,感谢梅斯更新及时

    0

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