A New Topical Antiandrogen Benefits Acne Treatment

描述(申请人提供)：痤疮是第二昂贵的皮肤病，每年导致22.3亿美元的医疗支出。虽然整体健康不会受到损害，但痤疮会造成皮肤和情感上的伤痕，会持续一生。人们为痤疮治疗付费的意愿很高，这并不令人意外，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，存在严重的缺点，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学反应，在痤疮的发生发展过程中起着重要的作用。从理论上讲，雄激素受体抑制剂的使用应该会缓解所有类型的痤疮的症状，更重要的是，干预从粉刺到更严重的、导致疤痕的炎性病变的进展。目前，还没有可用的局部雄激素受体抑制剂。在这项SBIR研究的第一阶段，一种获得专利的雄激素受体降解增强剂ASC-J9已被证明在两种不同的人类痤疮动物模型中诱导雄激素刺激的脂溢症消退是有效和安全的。ASC-J9是一种新的化学标识，可能优于目前治疗痤疮的现有药物。Android Science Corporation已投入大量资源，通过必要的监管批准程序开发ASC-J9，以评估其作为抗痤疮药物的作用。在此第二阶段申请中，我们请求支持完成新药(IND)提交调查所需的关键开发步骤；确切地说，我们要求支持与药物物质放大、药物产品配方和制造/质量控制以及临床前生物安全性和生物有效性测试相关的必要监管研究。获得的信息将支持安卓科学公司申请食品和药物管理局(FDA)批准开始人体临床试验(第三阶段)的努力，并有助于获得后续资金或与老牌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局部霜的商业化将以更安全、更有效和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局部霜商业化可能会取代目前使用的一些不受欢迎的抗痘药物。粉刺是雄激素依赖性的。目前尚缺乏更有效、更安全、耐受性更好的痤疮治疗方法。该项目旨在开发ASC-J9外用乳膏并最终将其商业化，作为一流的雄激素抑制剂，以实现缓解临床症状的目标，并实现患者和医生更高的满意度。

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.