Dimebutic acid(Dimebutic acid) - 药物靶点：β-globin_专利_临床

概要

基本信息

药物类型

小分子化药

别名

HQK 1001、HQK-1001(Sodium)、ST-20

靶点

β-globin

作用机制

β-globin刺激剂(血红蛋白β链刺激剂)

治疗领域

遗传病与畸形血液及淋巴系统疾病其他疾病

在研适应症-

非在研适应症

镰状细胞血症β地中海贫血地中海贫血

原研机构

Boston University

在研机构-

非在研机构

HemaQuest Pharmaceuticals, Inc.

Boston University

最高研发阶段终止临床2期

首次获批日期-

最高研发阶段(中国)-

特殊审评-

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结构/序列

分子式C6H12O2

InChIKeyVUAXHMVRKOTJKP-UHFFFAOYSA-N

CAS号595-37-9

关联

6

项与 Dimebutic acid 相关的临床试验

NCT01601340

/ Terminated临床2期

A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease

The purpose of this study is to evaluate the effects of HQK-1001 on Hb F in subjects with sickle cell disease.

开始日期2012-07-01

申办/合作机构

HemaQuest Pharmaceuticals, Inc.

NCT01642758

/ Completed临床2期IIT

An Open-Label Phase 2 Study of HQK-1001 in Subjects With Beta Thalassemia Intermedia

Beta thalassemia intermedia syndromes are genetic anemias caused by mutations which reduce production of beta globin, a major component of adult hemoglobin A, the protein which delivers oxygen throughout the body. Patients suffer from poor growth, fatigue, heart failure, endocrine deficiencies, and eventually, many require chronic blood transfusions. There is no approved therapeutic for the deficiency of beta globin chains in beta thalassemia.
This trial will study an oral therapeutic which stimulates production of fetal globin, an alternate type which is produced by all humans, but is normally switched off in infancy. This type of globin can compensate for the missing protein in beta thalassemia.

开始日期2012-05-01

申办/合作机构

Boston University

[+1]

NCT01322269

/ Completed临床2期

A Randomized, Open-Label, Multi-Dose Study of HQK-1001 in Subjects With Sickle Cell Disease

The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.

开始日期2011-04-01

申办/合作机构

HemaQuest Pharmaceuticals, Inc.

100 项与 Dimebutic acid 相关的临床结果

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100 项与 Dimebutic acid 相关的转化医学

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100 项与 Dimebutic acid 相关的专利（医药）

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328

项与 Dimebutic acid 相关的文献（医药）

2024-12-01·EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Design, synthesis, and preclinical evaluation of 11C/18F-labeled inhibitors for RIPK1 PET imaging

Article

作者: Wu, Rui ; Yin, Honghai ; Bai, Ping ; Luo, Tianwen ; Meng, Hui ; Wu, Xiaoai ; Wang, Yanli ; Kang, Leyi ; Xie, Weiyao ; Wang, Yongle ; Wang, Changning ; Zhou, Yanting ; Zhao, Chenyao

Receptor-interacting serine/threonine-protein kinase 1 (RIPK1) is a promising target for the diagnosis and treatment of various diseases, especially neurodegenerative disorders. Developing PET imaging probes targeting RIPK1 is beneficial for visualizing the connections between RIPK1 and diseases, as well as for related drug development. In this study, we report the design and synthesis of a series of novel RIPK1 inhibitors. Three potent inhibitors, 7i, 7k, and 8a, with good cell anti-necroptosis potency and physicochemical properties, were identified and selected for PET imaging probe development. Subsequently, three PET imaging radioligands ([¹¹C]7k, [¹⁸F]7i, and [¹⁸F]8a) were successfully synthesized. In mouse PET imaging studies, all three radioligands showed good brain uptake. Among them, probe [¹⁸F]8a exhibited good binding specificity in both in vitro autoradiography and in vivo PET imaging studies. Additionally, [¹⁸F]8a demonstrated good in vivo metabolic stability. This work highlights the potential of probe [¹⁸F]8a for imaging brain RIPK1 in live animals, laying the groundwork for the future development of RIPK1 PET radioligands.

2024-10-25·ORGANIC LETTERS

Ligand-Enabled Nickel(II)-Catalyzed β-C(sp³)–H Thiolation of Ketones

Article

作者: Liu, Bin ; Liao, Gang ; Chen, Lili

We present the first example of nickel(II)-catalyzed β-C(sp³)-H thiolation of ketones, employing 2-hydrazinopyridine as an efficient directing group. This approach enables the thiolation of a diverse array of ketones at the β-position. The straightforward installation and subsequent removal of the directing group significantly enhance the synthetic versatility and practicality of this transformation.

2024-10-11·ORGANIC LETTERS

Visible-Light-Promoted Iron(II)/Lewis Base Catalysis for the Alkylation of Morita–Baylis–Hillman Acetates Using Carboxylic Acids

Article

作者: Thapa, Satyam ; Sai Naik, Mudavath Bhargav ; Akondi, Srirama Murthy ; Golagani, Durga ; Prakash, Kota Krishna

A novel photoinduced Fe(OTf)₂/2,4,6-collidine-catalyzed alkylation of Morita-Baylis-Hillman (MBH) acetates using carboxylic acids in a regio- and stereoselective manner is reported. This method demonstrates a broad scope, encompassing various carboxylic acids and MBH acetates, including drugs and bioactive molecules, to synthesize densely functionalized cinnamates and acrylates. The reactions are performed in the green solvent dimethyl carbonate under oxidant-free conditions. Based on control experiments, a plausible mechanism involving an Fe(II)-Fe(III)-Fe(II) cycle is proposed.

1

项与 Dimebutic acid 相关的新闻（医药）

2008-12-09

·BioSpace

HemaQuest Announces Clinical Progress on New Sickle Cell Anemia Drug

SAN FRANCISCO, CA--(Marketwire - December 09, 2008) - HemaQuest Pharmaceuticals announced today the successful completion of Phase 1 clinical trials of HQK-1001, an orally-administered therapeutic which the company is developing to treat hemoglobin disorders, including sickle cell anemia and beta thalassemia. The company presented the results at the annual meeting of the American Society of Hematology in San Francisco.

100 项与 Dimebutic acid 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
β地中海贫血	临床2期	黎巴嫩	HemaQuest Pharmaceuticals, Inc.	2012-05-01
镰状细胞血症	临床2期	美国	HemaQuest Pharmaceuticals, Inc.	2011-04-01
镰状细胞血症	临床2期	加拿大	HemaQuest Pharmaceuticals, Inc.	2011-04-01
镰状细胞血症	临床2期	埃及	HemaQuest Pharmaceuticals, Inc.	2011-04-01
镰状细胞血症	临床2期	牙买加	HemaQuest Pharmaceuticals, Inc.	2011-04-01
镰状细胞血症	临床2期	黎巴嫩	HemaQuest Pharmaceuticals, Inc.	2011-04-01
地中海贫血	临床2期	-	Boston University	-

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT01601340 (Pubmed \| Am J Hematol)	临床2期	镰状细胞血症	76	HQK-1001	憲鏇築鬱顧獵網鬱範製(構願鹽鹹衊壓遞遞繭鑰) = The most common adverse events in the HQK-1001 group, usually graded as mild or moderate, consisted of abdominal pain 蓋鹹觸廠顧鑰鹹築築廠 (鹽餘艱構鑰窪膚觸鑰選 ) 更多	不佳	2014-07-01
				Placebo
NCT01322269 (Pubmed \| Am J Hematol)	临床2期	镰状细胞血症	52	HQK-1001 30 mg/kg	艱簾鏇廠觸窪膚蓋憲膚(衊艱願鏇製觸壓壓範廠) = 鏇簾衊衊餘觸獵遞壓願襯觸鑰獵夢衊獵觸範憲 (網鹹願製壓醖衊鑰顧鬱, 0.8 ~ 3.2) 更多	-	2013-11-01
				HQK-1001 40 mg/kg	艱簾鏇廠觸窪膚蓋憲膚(衊艱願鏇製觸壓壓範廠) = 壓顧廠構鹽夢製壓膚壓襯觸鑰獵夢衊獵觸範憲 (網鹹願製壓醖衊鑰顧鬱, 1.7 ~ 3.8)
NCT00842088 (Pubmed \| Am J Hematol)	临床1/2期	镰状细胞血症	24	HQK-1001 10 mg/kg	鹹簾網壓餘鏇鹽構築遞(窪淵鏇鏇鬱壓製壓鬱夢) = 膚襯鑰網膚糧積觸獵衊觸糧範廠窪醖蓋選積顧 (積齋餘選獵廠顧獵積憲 ) 更多	-	2012-11-01
				HQK-1001 20 mg/kg	鹹簾網壓餘鏇鹽構築遞(窪淵鏇鏇鬱壓製壓鬱夢) = 範鏇積壓窪鏇襯獵繭積觸糧範廠窪醖蓋選積顧 (積齋餘選獵廠顧獵積憲 ) 更多