Non-Invasive Treatment for Uveitis

描述（由申请人提供）：本创新提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，中间和后葡萄膜炎的主要治疗方法是口服药物（有明显的全身副作用）或侵入性局部方法，如玻璃体内注射（IVT）、眼周注射或在眼内植入缓释药物输送装置。因此，在安全性、患者接受度和疗效方面，迫切需要开发一种优越的给药系统来治疗葡萄膜炎。同样的产品可用于治疗其他适应症，如糖尿病性黄斑水肿，这是一个涉及400多万美国人的庞大市场。Aciont已经完成了所有必要的GLP临床前安全性、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系统的生产，以满足FDA的要求，在人体中测试我们提议的治疗。我们的研究计划有三个具体目标：“aim1 - I期临床研究（治疗方式的安全性）。证明我们的新给药涂布器和DSP配方在人体中是安全且耐受性良好的。”aim2 - II期临床研究（对葡萄膜炎的疗效）。证明我们的无创DSP Visulex治疗葡萄膜炎既安全又有效。”Visulex-P系统的设计与部件和配方的可制造性。潜在地，我们的方法可以导致一个非侵入性的药物输送系统，允许一个非常简单的治疗，可以由护士或辅助专业人员在医生的办公室在5- 10分钟内完成。这样的系统有潜力减少严重葡萄膜炎的急性视力威胁条件的一个单一的治疗显著部分。一个长期的目标可能是训练病人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.