Cocaine addiction medication EMB-001

描述(申请人提供)：长期目标：开发治疗可卡因依赖的EMB-001。背景：可卡因依赖和滥用困扰着美国160万人，并与个人和整个社会的巨大代价有关。目前还没有批准用于治疗可卡因依赖的药物，迫切需要安全有效的疗法来治疗这一严重疾病。位于什里夫波特的路易斯安那州立大学健康科学中心(路易斯安那州立大学S分校)和恩贝拉神经疗法公司正在开发用于治疗可卡因成瘾的EMB-001。EMB-001是一种由FDA批准的两种药物组成的联合药物产品，苯二氮卓类药物奥沙西潘和皮质醇合成抑制剂美替拉酮。这种新药的开发源于20年来由NIH资助的关于压力和下丘脑-垂体-肾上腺(HPA)轴在成瘾中的作用的研究。EMB-001已经在多种可卡因成瘾的动物模型中被证明是有效的，并在可卡因依赖患者中使用商业上可获得的单药进行的试点临床试验中被证明是有效的。这项建议旨在继续开发这一有前景的治疗药物，并将联合药物产品推向临床试验。具体目标如下：具体目标1：完成EMB-001的研制。具体目标2：完成支持IND提交所需的临床前药理学和毒理学研究。具体目标3：完成EMB-001的一期临床试验。在资助期结束时，我们将准备在可卡因依赖受试者的第二阶段安全性和有效性研究中评估EMB-001。 公共卫生相关性：这项提案的重点是开发EMB-001，这是一种治疗可卡因成瘾的新型联合药物产品。这项工作的目标是通过一期临床试验推进EMB-001。

DESCRIPTION (provided by applicant): Long Term Goal: To develop EMB-001 for the treatment of cocaine dependence. Background: Cocaine dependence and abuse afflict 1.6 million people in the U.S. and are associated with enormous costs to individuals and to the overall society. There are currently no medications approved for the treatment of cocaine dependence, and there is a compelling need for safe and effective therapeutics to treat this serious condition. The Louisiana State University Health Sciences Center in Shreveport (LSUHSC-S) and Embera NeuroTherapeutics are developing EMB-001 for the treatment of cocaine addiction. EMB-001 is a combination drug product comprising two FDA-approved medications, the benzodiazepine oxazepam and the cortisol synthesis inhibitor metyrapone. The development of this new medication emerged from 20 years of NIH-funded research into the role of stress and the hypothalamo-pituitary-adrenal (HPA) axis in addiction. EMB-001 has already been demonstrated to be effective in multiple animal models of cocaine addiction and in a pilot clinical trial carried out using the commercially available single agent drugs in cocaine-dependent patients. This proposal is aimed at continuing the development of this promising therapeutic and advancing the combination drug product into clinical testing. The Specific Aims are as follows: Specific Aim 1: To complete the development and manufacture of EMB-001. Specific Aim 2: To complete the preclinical pharmacology and toxicology studies needed to support an IND submission. Specific Aim 3: To complete a Phase 1 clinical trial of EMB-001. At the completion of the funding period, we will be prepared to evaluate EMB-001 in Phase 2 safety and efficacy studies in cocaine-dependent subjects. PUBLIC HEALTH RELEVANCE: This proposal is focused on the development of EMB-001, a novel combination drug product for the treatment of cocaine addiction. The goal of the work is to advance EMB-001 through a Phase 1 clinical trial.