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）：痤疮是第二昂贵的皮肤病，每年造成 22.3 亿美元的医疗支出。虽然整体健康不会受到损害，但痤疮可能会造成持续一生的皮肤和情感疤痕。痤疮治疗的支付意愿很高，这并不奇怪，市场预计将增长到 120 亿美元。目前，现有的药物主要用于治疗症状，并存在严重的缺点，例如皮肤刺激、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发挥其生物反应，在痤疮（即疾病的原因）的发展中发挥重要作用。理论上，使用雄激素受体抑制剂应该可以缓解所有类型痤疮的症状，更重要的是，可以干预从粉刺到更严重、引起疤痕的炎症病变的进展。目前，尚无可用的局部雄激素受体抑制剂。在这项 SBIR 研究的第一阶段中，一种专利雄激素受体降解增强剂 ASC-J9 已被证明能够在两种不同的人类痤疮动物模型中有效且安全地诱导雄激素刺激的皮脂溢消退。 ASC-J9 是一种新的化学成分，可能优于目前可用的痤疮治疗药物。 AndroScience Corporation 已投入大量资源来开发 ASC-J9，并通过必要的监管审批程序来评估其作为抗痤疮药物的效果。在此第 2 阶段申请中，我们请求支持以完成新药研究 (IND) 提交所需的关键开发步骤；确切地说，我们正在请求支持与原料药放大、药品配方和制造/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 请求美国食品和药物管理局 (FDA) 批准开始人体临床试验（第 3 阶段），并有助于获得后续资金或与成熟的制药公司建立合作伙伴关系，以注册必要的流程将新药推向市场。我们相信，ASC-J9外用霜的商业化将通过以更安全、更有效和更令人满意的方式减轻痤疮患者的临床症状，从而造福公众健康。 ASC-J9外用霜的商业化可以取代目前使用的一些不良抗痤疮药物。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。该项目旨在开发并最终商业化ASC-J9外用霜作为一流的雄激素抑制剂，以实现缓解临床症状并提高患者和医生满意度的目标。