预约演示
更新于：2025-02-14
Lonvoguran
更新于：2025-02-14
概要
基本信息
非在研机构- |
最高研发阶段临床3期 |
首次获批日期- |
最高研发阶段(中国)- |
特殊审评孤儿药 (美国)、孤儿药 (欧盟)、优先药物（PRIME） (欧盟)、再生医学先进疗法 (美国) |
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结构/序列
Sequence Code 1116750154
来源: *****
关联
3
项与 Lonvoguran 相关的临床试验NCT06634420
HAELO: a Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2002 in Participants with Hereditary Angioedema (HAE)
CTIS2023-507956-56-00
Long-Term Follow-Up of Subjects Treated With NTLA-2002 - ITL-2002-CL-999
NCT05120830
Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)
100 项与 Lonvoguran 相关的临床结果
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100 项与 Lonvoguran 相关的转化医学
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100 项与 Lonvoguran 相关的专利（医药）
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4
项与 Lonvoguran 相关的文献（医药）2025-01-30NEW ENGLAND JOURNAL OF MEDICINE
CRISPR-Based Therapy for Hereditary Angioedema
Article
作者: Aygören-Pürsün, Emel ; Lindsay, Karen ; Launay, David ; Shah, Mrinal Y ; Bouillet, Laurence ; Xu, Yuanxin ; Cohn, Danny M ; Katelaris, Constance H ; Maag, David ; Petersen, Remy S ; Butler, James S ; Longhurst, Hilary J ; Magerl, Markus ; Abdelhady, Ahmed M ; Golden, Adele ; Gurugama, Padmalal ; Lebwohl, David
BACKGROUND:
Hereditary angioedema is a rare genetic disease characterized by severe and unpredictable swelling attacks. NTLA-2002 is an in vivo gene-editing therapy that is based on clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9. NTLA-2002 targets the gene encoding kallikrein B1 (KLKB1). A single dose of NTLA-2002 may provide lifelong control of angioedema attacks.
METHODS:
In this phase 2 portion of a phase 1-2 trial, we randomly assigned adults with hereditary angioedema in a 2:2:1 ratio to receive NTLA-2002 in a single dose of 25 mg or 50 mg or placebo. The primary end point was the number of angioedema attacks per month (the monthly attack rate) from week 1 through week 16. Secondary end points included safety, pharmacokinetics, and pharmacodynamics (i.e., the change from baseline in total plasma kallikrein protein level); exploratory end points included patient-reported outcomes.
RESULTS:
Of the 27 patients who underwent randomization, 10 received 25 mg of NTLA-2002, 11 received 50 mg, and 6 received placebo. From week 1 through week 16, the estimated mean monthly attack rate was 0.70 (95% confidence interval [CI], 0.25 to 1.98) with 25 mg of NTLA-2002, 0.65 (95% CI, 0.24 to 1.76) with 50 mg, and 2.82 (95% CI, 0.80 to 9.89) with placebo; the difference in the estimated mean attack rate with NTLA-2002 as compared with placebo was -75% with 25 mg and -77% with 50 mg. Among patients who received NTLA-2002, 4 of the 10 patients who received 25 mg (40%) and 8 of the 11 who received 50 mg (73%) were attack-free with no additional treatment during the period from week 1 through week 16. The most common adverse events among patients who received NTLA-2002 were headache, fatigue, and nasopharyngitis. The mean percent change in total plasma kallikrein protein levels from baseline to week 16 was -55% with 25 mg and -86% with 50 mg; levels remained unchanged with placebo.
CONCLUSIONS:
NTLA-2002 administered in a single dose of 25 mg or 50 mg reduced angioedema attacks and led to robust and sustained reduction in total plasma kallikrein levels in patients with hereditary angioedema. These results support continued investigation in a larger phase 3 trial. (Funded by Intellia Therapeutics; ClinicalTrials.gov number, NCT05120830; EudraCT number, 2021-001693-33.).
2024-04-01The journal of allergy and clinical immunology. In practice
Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema
Article
作者: Riedl, Marc A ; Newman, Kenneth B ; Bordone, Laura ; Revenko, Alexey ; Cohn, Danny M
Hereditary angioedema (HAE) is typically caused by a deficiency of the protease inhibitor C1 inhibitor (C1INH). The absence of C1INH activity on plasma kallikrein and factor XIIa leads to overproduction of the vasoactive peptide bradykinin, with resulting angioedema. As the primary site of C1INH and prekallikrein production, the liver is recognized as an important therapeutic target in HAE, leading to the development of hepatic-focused treatment strategies such as GalNAc-conjugated antisense technology and gene modification. This report reviews currently available data on hepatic-focused interventions for HAE that have advanced into human trials. Donidalorsen is an investigational GalNAc3-conjugated antisense oligonucleotide that binds to prekallikrein mRNA in the liver and reduces the expression of prekallikrein. Phase 2 data with subcutaneous donidalorsen demonstrated a significant reduction in HAE attack rate compared with placebo. Phase 3 trials are underway. ADX-324 is a GalNAc3-conjugated short-interfering RNA being investigated in HAE. BMN 331 is an investigational AAV5-based gene therapy vector that expresses wild-type human C1INH and is targeted to hepatocytes. A single intravenous dose of BMN 331 is intended to replace the defective SERPING1 gene and enable patients to produce functional C1INH. A first-in-human phase 1/2 study is ongoing with BMN 331. NTLA-2002 is an investigational in vivo clustered regularly interspaced short palindromic repeats/Cas9-based therapy designed to knock out the prekallikrein-coding KLKB1 gene in hepatocytes; a phase 1/2 study is ongoing. Findings from these and other ongoing studies are highly anticipated with the expectation of expanding the array of treatment options in HAE.
2024-03-03Expert opinion on investigational drugs
Kallikrein inhibitors for angioedema: the progress of preclinical and early phase studies
Review
作者: Farkas, Henriette ; Balla, Zsuzsanna
INTRODUCTION:
Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent edema and predominantly caused by the dysregulation of the kinin-kallikrein system.
AREAS COVERED:
This manuscript presents the results of preclinical and early clinical trials of newer drugs targeting the dysregulated kinin-kallikrein system. ATN-249 is an oral drug that has shown promising results in preclinical and Phase I studies, and good tolerability in the prophylactic treatment of attacks. KVD900 is also an oral agent developed for the on-demand treatment of HAE attacks. It has shown positive results in Phase I/II studies, with rapid absorption. The third drug, IONIS-PKKRx, is an antisense oligonucleotide targeting plasma prekallikrein mRNA. It has shown a dose-dependent reduction of plasma prekallikrein levels and proenzyme activation in Phase I/II studies, and has shown promising results. STAR-0215 is a long acting anti-activated kallikrein monoclonal antibody. A Phase 1a single ascending dose trial evaluated its safety, pharmacokinetics, and pharmacodynamics. Lastly, NTLA-2002 is an investigational gene-editing therapy.
EXPERT OPINION:
The targeted treatment of the dysregulated kinin-kallikrein system with specific inhibitors is promising for the prevention of angioedema attacks. Ongoing phase III studies will provide further insight into the efficacy and long-term safety of these novel therapies, potentially expanding treatment options for HAE treatment.
129
项与 Lonvoguran 相关的新闻（医药）2025-02-05
·梅斯医学
临床结果临床3期基因疗法临床2期
2025-01-31
·医药魔方
财报临床3期生物类似药疫苗并购
2025-01-25
·药明康德
临床1期基因疗法细胞疗法免疫疗法临床3期
100 项与 Lonvoguran 相关的药物交易
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研发状态
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 遗传性血管性水肿 | 临床3期 | 美国 | 2024-10-07 |
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临床结果
临床结果
适应症
分期
评价
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临床2期 | 27 | NTLA-2002 25 mg | 壓窪獵蓋製廠遞觸膚鑰(餘簾獵遞積鑰網醖鑰衊) = 襯醖顧觸淵窪蓋繭艱夢 鹹製範積選鹹願鬱衊夢 (網膚壓觸鏇顧齋構糧廠, 0.25 ~ 1.98) 更多 | 积极 | 2024-10-24 | ||
NTLA-2002 50 mg | 壓窪獵蓋製廠遞觸膚鑰(餘簾獵遞積鑰網醖鑰衊) = 醖齋觸齋廠壓構膚鹹鏇 鹹製範積選鹹願鬱衊夢 (網膚壓觸鏇顧齋構糧廠, 0.24 ~ 1.76) 更多 | ||||||
临床1期 | 10 | NTLA-2002 (Week 1-16) | 遞選鏇觸壓網糧醖餘膚(廠齋窪繭繭願艱網顧齋) = 積鹹積窪鬱築窪網鏇觸 艱糧衊衊壓艱鏇鏇願衊 (齋艱積繭願廠繭鏇獵構 ) | 积极 | 2024-06-02 | ||
NTLA-2002 (Week 5-16) | 遞選鏇觸壓網糧醖餘膚(廠齋窪繭繭願艱網顧齋) = 衊夢鬱繭齋壓遞鑰顧艱 艱糧衊衊壓艱鏇鏇願衊 (齋艱積繭願廠繭鏇獵構 ) | ||||||
临床1期 | 10 | NTLA-2002 25 mg | 淵鬱獵夢淵製蓋廠願簾(選遞糧顧鏇觸糧淵窪糧) = 獵顧構構觸壓窪獵衊窪 壓憲齋蓋觸繭鬱顧簾齋 (製鹽鏇觸夢淵網觸齋範 ) 更多 | 积极 | 2024-02-01 | ||
NTLA-2002 50 mg | 淵鬱獵夢淵製蓋廠願簾(選遞糧顧鏇觸糧淵窪糧) = 簾鏇糧繭遞醖顧鑰夢齋 壓憲齋蓋觸繭鬱顧簾齋 (製鹽鏇觸夢淵網觸齋範 ) 更多 | ||||||
临床1/2期 | 6 | (25 mg) | 醖構觸築醖網艱網艱壓(齋鏇衊廠齋範襯觸艱衊) = NTLA-2002 was generally well-tolerated across both dose levels: all AEs were of mild ormoderate severity 鬱簾糧鹹淵齋築觸憲鹽 (簾積齋繭範鹽鏇餘廠鹽 ) 更多 | 积极 | 2022-09-16 | ||
(75 mg) |
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