Preclinical Development of Tacrolimus for Radiation Cystitis

他克莫司治疗放射性膀胱炎的临床前开发

• 批准号: 8888531
• 负责人: JONATHAN H KAUFMAN
• 金额: $ 64.85万
• 依托单位: LIPELLA PHARMACEUTICALS, INC.
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-05-12 至 2017-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8888531
• 关键词: Basic Science; Biological Availability; Bladder; Canis familiaris; Caring; Cessation of life; Clinical; Clinical Research; Collaborations; Cyclophosphamide; Cystitis; Data; Development; Dinoprostone; Dose; Drug Formulations; Drug Kinetics; Dysuria; Foundations; Funding; Goals; Health; Hematuria; Hemorrhage; Histopathology; Hospitals; Immunosuppressive Agents; Incidence; Inflammatory; Intellectual Property; Interleukin-2; Intravenous; Intravesical Administration; Kidney; Kidney Function Tests; Lead; Letters; Life; Liposomes; Marketing; Medical; Modeling; Oral; Orphan; Patients; Pelvic Cancer; Pharmaceutical Preparations; Pharmacologic Substance; Phase; Property Rights; Quality of life; Radiation; Radiation therapy; Rare Diseases; Rattus; Reaction; Research; Research Proposals; Research Support; Rights; Route; Safety; Small Business Innovation Research Grant; Staining method; Stains; Tacrolimus; Testing; Therapeutic; Therapeutics for Rare and Neglected Diseases; Tissues; Toxic effect; Toxicology; United States National Institutes of Health; Universities; Urinary tract; Urine; Urothelium; Woman; Work; commercialization; effective therapy; follow-up; human WFDC2 protein; innovation; intravesical; lower urinary tract symptoms; lymph nodes; medical schools; men; novel; phase 1 study; pre-clinical; programs; research study; urologic

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.

描述（由申请人提供）：该项目名为“曲克莫司治疗放射性膀胱炎的临床前开发”，将资助关键实验以推进 LP-10 的商业开发。 LP-10 是一种脂质体他克莫司制剂，用于膀胱局部（局部）膀胱内给药。该制剂提供膀胱中的活性药物水平，并显着降低全身水平。放射性膀胱炎是一种罕见疾病，表现为下尿路症状，包括排尿困难、血尿和出血。目前尚无批准的治疗 RC 的疗法，这会严重降低患者的生活质量，需要长期随访治疗，并且在某些患者中会导致死亡。该项目为公私合作项目 Lipella Pharmaceuticals Inc.、匹兹堡大学医学院和 William Beaumont 医院之间的合作。 Lipella 拥有 LP-10 配方的知识产权以及最近获得 FDA 孤儿药资格的相关权利。 FDA 还向 Lipella 提供了 LP-10 IND 考虑的临床前要求。这些要求构成了本提案中描述的研究。工作范围包括 LP-10 的进一步药物和药理学表征（第一阶段）和 IND 支持研究（第二阶段）。这项研究是转化性的，将基础研究与临床开发联系起来。这个方向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疗法，为商业化做好准备。