A New Topical Antiandrogen Benefits Acne Treatment

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。虽然整体健康不受损害，痤疮可以产生皮肤和情感的疤痕，持续一生。毫不奇怪，人们对痤疮治疗的支付意愿很高，预计市场将增长到120亿美元。目前，可用的药物主要用于治疗症状，并且具有严重的缺点，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表达其生物学反应，在痤疮的发生中起重要作用，即，疾病的起因从理论上讲，雄激素受体抑制剂的使用应引起所有类型痤疮的症状缓解，更重要的是，干预从粉刺到更严重的瘢痕炎性病变的进展。目前，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，已证明一种获得专利的雄激素受体降解增强剂ASC-J 9在两种不同的人类痤疮动物模型中诱导雄激素刺激的seboretin消退方面是有效和安全的。ASC-J 9是一种新的化学特性，可能上级目前可用的痤疮治疗药物。AndroScience公司已承诺通过必要的监管审批程序开发ASC-J 9作为抗痤疮药物进行评估的大量资源。在本II期申请中，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，我们请求支持与原料药规模扩大、制剂处方和生产/质量控制以及临床前生物安全性和生物有效性检测相关的所需监管研究。所获得的信息将支持AndroSciences努力请求美国食品药品监督管理局（FDA）批准开始人体临床试验（第3阶段），并帮助确保后续资金或与已建立的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-J 9外用乳膏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而有益于公众健康。ASC-J 9外用乳膏商业化可以取代目前使用的一些不受欢迎的抗痤疮药物。痤疮是雄激素依赖性的。更有效，更安全，更好的治疗痤疮的耐受性缺乏。该项目旨在开发并最终商业化ASC-J 9外用乳膏，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。

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.