Why Is Gene Therapy-Focused Intellia Therapeutics Stock Trading Lower On Thursday?
Why Is Gene Therapy-Focused Intellia Therapeutics Stock Trading Lower On Thursday?
A single dose of NTLA-2002 showed mean monthly attack rates relative to placebo were reduced by 75% and 77% for the 25 mg and 50 mg arms during weeks 1–16 and by 80% and 81% during weeks 5–16, respectively.On Thursday, Intellia Therapeutics, Inc. (NASDAQ:NTLA) released Phase 2 data from the ongoing Phase 1/2 study of NTLA-2002 in patients with hereditary angioedema (HAE).
周四，纳斯达克上的intellia therapeutics公司公布了NTLA-2002正在进行的遗传性血管性水肿（HAE）患者的1/2期研究的2期数据。
NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a one-time treatment for HAE, a rare genetic condition that results in recurrent attacks of severe swelling.
NTLA-2002是一种正在开发中的体内CRISPR基因编辑疗法，旨在作为HAE的一次性治疗，HAE是一种罕见的遗传性疾病，导致反复发作严重肿胀。
The data cut-off date for the analysis was April 4, when the 25th patient completed the 16-week primary observation period.
分析的数据截止日期为4月4日，当时第25位患者完成了为期16周的初级观察期。
A single dose of NTLA-2002 showed mean monthly attack rates relative to placebo were reduced by 75% and 77% for the 25 mg and 50 mg arms during weeks 1–16 and by 80% and 81% during weeks 5–16, respectively.
NTLA-2002单剂量显示，与安慰剂相比，25毫克和50毫克组在第1-16周分别减少了75%和77%的月均发作率，在第5-16周分别减少了80%和81%；
In the 50 mg arm, eight of 11 patients experienced a complete response after a single dose of NTLA-2002, with no attacks at all during the 16-week primary observation period; these eight patients continued to be attack-free through the latest follow-up (median of eight months), and no subsequent treatment has been required.
在50毫克组中，11名患者中有8名患者在NTLA-2002单剂后经历了完全缓解，在为期16周的初级观察期内没有发作；这8名患者在最新的随访中仍未发作（中位数为8个月），也无需后续治疗。
Four of the ten patients in the 25 mg arm experienced a complete response, and zero patients in the placebo arm.
在25毫克组中，十名患者中有四名经历了完全缓解，安慰剂组没有患者经历完全缓解。
Patients who received the 50 mg dose achieved a greater kallikrein protein reduction, with an 86% mean reduction from baseline compared to 55% in the 25 mg arm at week 16.
接受50毫克剂量的患者在第16周的基础上，与25毫克组的55%相比，kallikrein蛋白的减少更明显，平均减少了86％。
At both dose levels, NTLA-2002 was well tolerated.
在两个剂量水平上，NTLA-2002耐受性良好。
Based on these results, Intellia selected 50 mg for evaluation in the global, pivotal Phase 3 HAELO study, actively screening patients.
根据这些结果，intellia therapeutics选择了50毫克进行全球关键的3期HAELo研究评估，积极筛选患者。
The Phase 3 study is expected to conclude by September 2027.
第3期研究预计将于2027年9月结束。
When in June, Intellia Therapeutics released data from the Phase 1 portion of the ongoing Phase 1/2 study, a single dose of NTLA-2002 led to a 98% mean reduction in monthly HAE attack rate, with an average follow-up of over 20 months across all patients.
当6月份，intellia therapeutics发布了进行中第1/2期研究的第1部分的数据时，NTLA-2002的单剂导致月度HAE发作率平均减少了98％，所有患者的平均随访时间超过20个月。
The trial, which administered a single dose of NTLA-2002 to 10 patients, tracked a 90% reduction in attacks from weeks 1 to 16, which increased to 92% for weeks 5-16.
该试验向10名患者单次投予NTLA-2002，从第1至第16周攻击减少了90％，从第5至第16周增加至92％。
Price Action: NTLA stock is down 18.3% at $16.28 at last check Thursday.
股价走势：NTLA股价上涨18.3%，至16.28美元，在上周四最后检查时。
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Image created using Dall-E.
使用Dall-E创建的图像。
