Intravesical Liposome Treatment for Interstitial Cystitis

描述（由申请人提供）：间质性膀胱炎（IC）是一种慢性、严重的膀胱衰弱性疾病。它可以被描述为膀胱壁的慢性炎症，特征是尿频和尿急，以及严重的耻骨上和/或盆腔疼痛。目前，对IC的药物治疗有限，新的有效治疗是一个主要的未满足的医疗需求。Lipella Pharmaceuticals Inc.打算成为专门使用膀胱内脂质体技术治疗IC的领先公司。我们已经开发出一种用于膀胱内灌注的高特异性脂质体配方方法。这项技术是由dr。匹兹堡大学的Leaf Huang和Michael Chancellor说。根据他们的研究，脂质体本身可以对发炎的膀胱提供缓解作用（RO1 DK068556; Fraser et al. 2003）， Lipella制药公司希望膀胱内脂质体配方可以改善IC患者的活性。在提交该ii期提案之前，i期资金主要用于探索LP-08的临床前安全性考虑，LP-08是一种用于大鼠模型的新型膀胱内脂质体治疗IC。该ii期建议包括进一步的安全性研究，特别是测量预期重复给药的后果，以及制定后续人体试验所需的生产规模扩大程序。这里提出的努力将在一定程度上有助于在提交FDA新药研究（IND）申请之前收集临床前数据，这是在给人使用LPA-08之前的监管要求。

DESCRIPTION (provided by applicant): Interstitial cystitis (IC) is a chronic, severely debilitating disease of the urinary bladder. It can be described as a chronic inflammatory condition of the bladder wall, characterized by urinary frequency and urgency, and severe suprapubic and/or pelvic pain. Presently, there is limited medical therapy for IC and new and effective treatment is a major unmet medical need. Lipella Pharmaceuticals Inc. intends to be the leading company specializing in the use of intravesical liposome technology to treat IC. We have developed a method of formulating highly specific liposomes for intravesical instillation. The technology is licensed from inventions and research by Drs. Leaf Huang and Michael Chancellor at the University of Pittsburgh. Based on their research that a liposome itself can provides a soothing effect to an irritated bladder (RO1 DK068556; Fraser et al. 2003), Lipella Pharmaceuticals expects that intravesical liposome formulations can present an improved activity in patients with IC. Prior to submitting this phase-II proposal, phase-I funding was used primarily to explore preclinical safety considerations of LP-08, a novel intravesical liposome therapy for IC, in a rat model. This phase-II proposal includes further safety studies, specifically measuring consequences of expected repeat dosing, as well as developing manufacturing scale-up procedures necessary for subsequent human testing. The effort proposed here would, in part, contribute to pre-clinical data collection prior to filing an FDA Investigational New Drug (IND) application, a regulatory requirement prior to administering LPA-08 in humans. PUBLIC HEALTH RELEVANCE We propose key preclinical experiments in an animal model to validate follow-on investment for clinical development of a novel treatment for painful bladder syndrome, an incurable chronic illness involving pain, urinary urgency, loss of sleep, depression and anxiety in approximately 500,000 women domestically.