A New Topical Antiandrogen Benefits Acne Treatment

一种新的外用抗雄激素有益于痤疮治疗

• 批准号: 7155625
• 负责人: CHARLES C-Y SHIH
• 金额: $ 44.6万
• 依托单位: ANDROSCIENCE CORPORATION
• 依托单位国家: 美国
• 财政年份: 2004
• 资助国家: 美国
• 起止时间: 2004-07-19 至 2008-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7155625
• 关键词: androgen receptor; androgens; clinical trials; human; toxicology

DESCRIPTION (provided by applicant): Acne is the second most expensive skin disease, resulting in $2.23 billion in medical spending annually. Although the overall health is not impaired, acne can produce cutaneous and emotional scars that last a lifetime. It is not surprising that there is high willingness to pay for acne treatment, and the market is expected to grow to $12 billion. Currently, available medicines are mainly used to treat symptoms and are associated with severe drawbacks, such as skin irritation, requiring long-term treatment, teratogenesis, and acquisition of antibiotic resistance. Androgens, which act through androgen receptor to express their biological response, play an important role in the development of acne, i.e., the cause of disease. In theory, use of an androgen receptor inhibitor should induce symptom relief for all types of acne and more importantly, intervenes in the progression from comedones to the more severe, scar-causing inflammatory lesions. Currently, there is no topical androgen receptor inhibitor available. In Phase I of this SBIR study, a patented androgen receptor degradation enhancer, ASC-J9, has been demonstrated to be effective and safe in inducing regression of androgen-stimulated seborrhea in two different animal models for human acne. ASC-J9, a new chemical identity, is potentially superior to currently available medicines for acne treatment. AndroScience Corporation has committed substantial resources in developing ASC-J9 through the necessary regulatory approval process for evaluation as an anti-acne drug. In this Phase 2 application, we request support to complete critical development steps needed for Investigation of New Drug (IND) submission; precisely we are asking support for required regulatory studies related to drug substance scale-up, drug product formulation and manufacturing/quality control, as well as preclinical biosafety and bioefficacy testing. The acquired information will support AndroSciences effort to request approval from the Food and Drug Administration (FDA) to commence human clinical trials (Phase 3) and also help in securing follow-up funding or forming partnerships with established pharmaceutical companies to register the process necessary to bring a new drug to the marketplace. We believe that commercialization of an ASC-J9 topical cream will benefit public health by alleviating clinical symptoms in acne sufferers in a safer, and more effective and satisfactory fashion. ASC-J9 topical cream commercialization could replace some undesirable anti-acne drugs currently in use. Acnes are androgen-dependent. More effective, safer, and better-tolerated treatments for acnes are lacking. This project is designed to develop and eventually commercialize ASC-J9 topical cream as the first-in-class androgen inhibitor to accomplish the goal to alleviate clinical symptoms and achieve a higher degree of satisfaction in patients and doctors.

描述（由申请人提供）：痤疮是第二大最昂贵的皮肤病，每年导致22.3亿美元的医疗支出。虽然整体健康不会受损，但痤疮会产生持续一生的皮肤和情感伤疤。人们愿意为痤疮治疗付费，这并不奇怪，市场预计将增长到120亿美元。目前，可获得的药物主要用于治疗症状，并伴有严重的缺陷，如皮肤刺激、需要长期治疗、致畸和获得抗生素耐药性。雄激素通过雄激素受体表达其生物反应，在痤疮的发生，即疾病的起因中起着重要作用。理论上，使用雄激素受体抑制剂可以缓解所有类型的痤疮的症状，更重要的是，可以干预从粉刺到更严重的、引起疤痕的炎症性病变。目前，还没有可用的外用雄激素受体抑制剂。在这项SBIR研究的I期，一种获得专利的雄激素受体降解增强剂ASC-J9在两种不同的人类痤疮动物模型中被证明是有效和安全的，可诱导雄激素刺激的皮脂分泌消退。ASC-J9是一种新的化学特性，可能优于目前可用的治疗痤疮的药物。AndroScience公司已投入大量资源开发ASC-J9，通过必要的监管批准程序评估作为抗痤疮药物。在此2期申请中，我们要求支持完成新药调查（IND）提交所需的关键开发步骤；确切地说，我们要求支持与原料药放大、药品配方和生产/质量控制以及临床前生物安全和生物功效测试相关的必要监管研究。获得的信息将支持AndroSciences向美国食品和药物管理局（FDA）申请批准开始人体临床试验（三期），并帮助确保后续资金或与知名制药公司建立合作伙伴关系，以注册将新药推向市场所需的过程。我们相信，ASC-J9外用面霜的商业化将以更安全、更有效、更令人满意的方式缓解痤疮患者的临床症状，从而有利于公众健康。ASC-J9外用面霜的商业化可以取代目前使用的一些不良的抗痤疮药物。痤疮是雄激素依赖性的。目前还缺乏更有效、更安全、耐受性更好的痤疮治疗方法。本项目旨在开发并最终商业化ASC-J9外用乳膏作为一流的雄激素抑制剂，以达到缓解临床症状，提高患者和医生满意度的目的。