Preclinical Development of Tacrolimus for Radiation Cystitis

描述(由申请人提供)：该项目名为“治疗放射性膀胱炎的曲克莫司的临床前开发”，将资助推进LP-10商业开发的关键实验。LP-10是一种脂质体他克莫司制剂，用于局部(局部)膀胱内给药。该配方提供了膀胱中的活性药物水平，并显著降低了全身水平。放射性膀胱炎是一种罕见的疾病，下尿路症状包括排尿困难、血尿和出血。目前还没有被批准的治疗RC的方法，这可能会严重降低患者的生活质量，需要长期的后续治疗，在一些患者中，还会导致死亡。这个项目是公私合营的 利佩拉制药公司、匹兹堡大学医学院和威廉·博蒙特医院之间的合作。Lipella拥有LP-10配方的知识产权，以及与其最近从FDA获得孤儿指定相关的权利。FDA还向Lipella提供了IND考虑LP-10的临床前要求。这些要求构成了本提案中所述的研究。工作范围包括LP-10的进一步药学和药理学特征(第一阶段)和支持IND的研究(第二阶段)。这项研究是翻译性的，并将基础研究与临床开发联系起来。在这个方向上的进展将直接支持我们的最终目标，即开发一种安全有效的治疗方法，为商业化做好准备。

DESCRIPTION (provided by applicant): This project, entitied "Preclinical Development of Tracrolimus for Radiation Cystitis" will fund key experiments to advance the commercial development of LP-10. LP-10 is a liposomal tacrolimus formulation for local (topical) intravesical administration to the urinary bladder. The formulation provides active drug levels in the bladder with significantly reduced systemic levels. Radiation cystitis is a rare disease defined by lower urinary tract symptoms that include dysuria, hematuria, and hemorrhage. There are currently no approved therapies to treat RC, which can severely degrade a patient's quality of life, require long-term follow-up treatment, and, in some patients, lead to death. This project is public-private collaboration between Lipella Pharmaceuticals Inc., the University of Pittsburgh School of Medicine, and William Beaumont Hospital. Lipella owns intellectual property rights to the LP-10 formulation as well as rights associated with its recent receipt of orphan designation from the FDA. The FDA has also provided Lipella with pre-clinical requirements for IND consideration of LP-10. These requirements constitute the studies described in this proposal. The scope of work includes further pharmaceutical and pharmacological characterization of LP-10 (Phase I) and IND-enabling studies (Phase II). This research is translational, and bridges basic research to clinical development. Progress in this direction will directly support our ultimate goal of developing a safe and effective therapy ready for commercialization.