Non-Invasive Treatment for Uveitis

描述(由申请人提供)：这一创新方案的目的是将一种治疗葡萄膜炎的非侵入性局部产品商业化，该产品安全且易于管理。葡萄膜炎是美国第三大致盲原因。到目前为止，对中间和后葡萄膜炎的主要治疗方法要么是口服药物(有显著的全身副作用)，要么是侵入性的局部方法，如玻璃体内注射(IVT)、眼周注射或眼内植入缓释药物释放装置。因此，在安全性、患者接受性和治疗葡萄膜炎的有效性方面开发一种更好的药物释放系统是非常必要的。同样的产品也可以用于治疗其他适应症，如糖尿病黄斑水肿，这是一个涉及400多万美国人的重要市场。Aciont已经完成了所有必要的临床前GLP安全性、葡萄膜炎的概念验证工作，以及我们的地塞米松磷酸钠(“DSP”)Visulex系统的制造，以满足FDA的要求，以测试我们建议的治疗方法。我们的研究计划有3个具体目标：“Aim1-I期临床研究(治疗方式的安全性)。证明我们的新型药物输送贴剂和DSP制剂在人体内是安全和耐受性良好的。”AIM2-第二阶段临床研究(对葡萄膜炎的疗效)。证明我们的非侵入性DSP Visulex治疗葡萄膜炎是安全有效的。AIM3-优化Visulex-P系统设计和组件和配方的可制造性。我们的方法可能导致一种非侵入性药物输送系统，允许护士或准专业人员在5-10分钟内在医生办公室进行非常简单的治疗。这样的系统有可能在一次治疗中减少严重葡萄膜炎的严重视力威胁的很大一部分。长期目标可能是培训患者自己管理我们的治疗系统。

DESCRIPTION (provided by applicant): The aim of this innovative proposal is to commercialize a non-invasive topical product that treats uveitis which is safe and easy to administer. Uveitis is the third leading cause of blindness in the United States. Heretofore, the primary treatment of intermediate and posterior uveitis has been either oral medications (with significant systemic side effects) or invasive local methods, such as an intravitreal injection (IVT), periocular injection or implantation of a sustained release drug delivery device into the eye. Thus, there is a great need to develop a superior drug delivery system in terms of safety, patient acceptance and efficacy for uveitis. The same product could be used to treat other indications such as diabetic macular edema, a substantial market involving more than 4 million Americans. Aciont has completed all necessary preclinical GLP safety, proof-of-concept work in uveitis and manufacturing of our dexamethasone sodium phosphate ("DSP") Visulex system needed to satisfy the FDA's requirements to test our proposed treatment in man. There are 3 specific aims for our research plan: " Aim1-Phase I Clinical Study (safety of treatment modality). Demonstrate that our new drug delivery applicator and DSP formulations are safe and well tolerated in man. " Aim2-Phase II Clinical Study (efficacy in uveitis). Demonstrate that our non-invasive DSP Visulex treatment is both safe and effective in treating uveitis. " Aim3-Optimize Visulex-P System Design and Manufacturability of Components and Formulations. Potentially, our approach can lead to a non-invasive drug delivery system that allows for a very simple treatment which can be performed by a nurse or paraprofessional in the doctor's office within a range of 5- 10 minutes. Such a system has the potential to curtail a significant portion of the acute sight threatening condition of severe uveitis in a single treatment. A long term goal could be to train patients to administer our treatment system by themselves.