Intellia强调一次性剂量实验药物的临床改善效果-动脉网

Intellia Highlights Clinical Improvements With One-Time Dose Of Experimental Drug

benzinga 等信源发布 2025-09-25 13:57

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Intellia Therapeutics Inc.

Intellia治疗公司

(NASDAQ:

（纳斯达克：

NTLA

) on Thursday released longer-term follow-up data from the ongoing Phase 1 study of investigational nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with

周四发布了正在进行的nexiguran ziclumeran（nex-z）1期研究的长期随访数据，用于治疗遗传性ATTR淀粉样变性伴随症状。

polyneuropathy (ATTRv-PN)

多发性神经病（ATTRv-PN）

。

Results were presented at the International ATTR Amyloidosis Annual Meeting for Patients and Doctors. The results were simultaneously

结果在国际ATTR淀粉样变性年度患者与医生会议上公布，结果同时

published

已发布

in the New England Journal of Medicine.

在《新英格兰医学杂志》上。

Data

数据

Deep, durable and consistent TTR reductions continue to be observed. Across patients who received a one-time dose of 0.3 mg/kg or higher (n=33), the mean serum TTR reduction at 24 months was 92% (corresponding mean absolute

持续观察到深度、持久且一致的TTR降低。在所有接受单次剂量0.3 mg/kg或更高剂量的患者中（n=33），24个月时平均血清TTR降低率为92%（相应的平均绝对值）。

serum TTR level of 17.3 g/mL

血清TTR水平为17.3 g/mL

。

Among the 12 patients who had reached 36 months of follow-up, the mean serum TTR reduction was 90% (corresponding mean absolute serum TTR level of 20 g/mL.

在达到36个月随访的12名患者中，血清TTR平均降低90%（相应的平均绝对血清TTR水平为20 g/mL）。

Also Read:

另请阅读：

What’s Going On With Sarepta, Capricor And Other Gene Therapy Stocks On Wednesday?

周三，Sarepta、Capricor 和其他基因治疗股票发生了什么？

Favorable trends indicating stability or improvement were observed in most patients with ATTRv-PN after a single dose of nex-z.

大多数ATTRv-PN患者在单次剂量的nex-z后观察到稳定或改善的有利趋势。

Stability or improvement was based on evaluation of multiple clinical and biomarker measures, including Neuropathy Impairment Score (NIS), modified Neuropathy Impairment Score +7 (mNIS+7), modified body mass index (mBMI), Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) questionnaire, neurofilament light chain (NfL), and polyneuropathy disability (PND) score..

稳定性或改善基于对多项临床和生物标志物指标的评估，包括神经病变损伤评分（NIS）、改良神经病变损伤评分+7（mNIS+7）、改良体重指数（mBMI）、诺福克生活质量-糖尿病神经病变（QoL-DN）问卷、神经丝轻链（NfL）和多发性神经病残疾（PND）评分。

Among the 18 patients in whom a 24-month mNIS+7 assessment was completed by the data cutoff date (April 11, 2025), 13 (72%) showed improvements of a clinically meaningful threshold of ≥4 points, including most of the patients in the cohort who had progressed on Alnylam Pharmaceuticals, Inc.’s (NASDAQ: .

在数据截止日期（2025年4月11日）前完成24个月mNIS+7评估的18名患者中，有13名（72%）表现出具有临床意义的改善，改善阈值为≥4分，其中包括大多数在Alnylam Pharmaceuticals, Inc.（纳斯达克：）治疗中取得进展的患者。

ALNY

阿勒尼

) Onpattro (patisiran).

）Onpattro（patisiran）。

Among all 36 patients enrolled in the Phase 1 trial, mean values of the secondary endpoints mBMI, QoL-DN and NfL all trended toward disease improvement, and 89% of patients showed improvement or stability in PND scores through 24 months compared to baseline.

在参加第一阶段试验的所有36名患者中，次要终点指标mBMI、QoL-DN和NfL的平均值均显示出疾病改善的趋势，并且89%的患者在24个月内相比基线PND评分有所改善或保持稳定。

Nex-z has been generally well tolerated as of the data cutoff date across all patients and at all dose levels tested.

截至数据截止日期，Nex-z 在所有患者和所有测试剂量水平上普遍耐受良好。

The most commonly reported treatment-related adverse events were infusion-related reactions, which were mild or moderate and did not result in any discontinuations. Intellia began dosing patients in the Phase 3 MAGNITUDE-2 trial in April 2025.

最常见的治疗相关不良事件是输液相关反应，这些反应为轻度或中度，并未导致任何患者停止治疗。Intellia于2025年4月开始在3期MAGNITUDE-2试验中对患者进行给药。

What’s Next?

接下来是什么？

Patient screening is advancing rapidly, and enrollment completion is expected in the first half of 2026.

患者筛查正在迅速推进，预计将在2026年上半年完成入组。

Intellia anticipates submitting a biologics license application (BLA) for ATTRv-PN by 2028.

Intellia 预计将在 2028 年前提交针对 ATTRv-PN 的生物制品许可申请 (BLA)。

Price Action:

价格行为:

NTLA stock is down 4.42% at $16.98 at the last check on Thursday.

NTLA股票在上周四最后一次检查时下跌了4.42%，价格为16.98美元。

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