Developing Intravesical Liposomes for Interstitial Cystitis

开发治疗间质性膀胱炎的膀胱内脂质体

• 批准号: 8137753
• 负责人: JONATHAN H KAUFMAN
• 金额: $ 23.95万
• 依托单位: LIPELLA PHARMACEUTICALS, INC.
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-21 至 2013-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8137753
• 关键词: Adverse event; Affect; Anesthesia procedures; Applications Grants; Biological Sciences; Biotechnology; Birds; Bladder; Bladder Diseases; Cauterization - action; Chairperson; Chronic Disease; Cicatrix; Clinical; Clinical Data; Clinical Trials; Conduct Clinical Trials; Cyclic GMP; Data; Databases; Development; Eating; Ensure; Etiology; Fibromyalgia; Financial Support; Food; Funding; Goals; Grant; Health; Hospitals; Human; Human Experimentation; Institutes; Institutional Review Boards; Interstitial Cystitis; Intravenous; Intravesical Instillation; Investigational Drugs; Investigational New Drug Application; Investments; Legal patent; Life; Liposomes; Measures; Mediating; Modeling; Monitor; National Institute of Diabetes and Digestive and Kidney Diseases; Neurobiology; Outcome; Patients; Pelvic Pain; Performance; Pharmaceutical Preparations; Pharmacologic Substance; Phase; Phospholipids; Physicians; Preparation; Principal Investigator; Procedures; Process; Production; Quality Control; Quality of life; Rattus; Recruitment Activity; Research; Risk; Route; Safety; Small Business Innovation Research Grant; Small Business Technology Transfer Research; Source; Sterility; Symptoms; Syndrome; Taiwan; Time; Toxicology; Tuberculosis Vaccines; Ulcer; United States; United States National Institutes of Health; Urology; cGMP production; chronic pain; clinical research site; design; egg; expectation; institutional financing; intravesical; manufacturing scale-up; meetings; novel; nutrition; operation; phase 3 study; pilot trial; pre-clinical; preclinical study; product development; programs; research and development; response

DESCRIPTION (provided by applicant): LP08 is a novel intravesical treatment for interstitial cystitis (IC), a poorly understood chronic disorder of unknown etiology consisting of irritative bladder symptoms and pelvic pain that dramatically affects quality of life. In IC/PBS, two distinct subtypes are generally recognized. Ulcerative (classic) IC and non-ulcerative IC/PBS clearly differ in terms of cystoscopic findings and symptom response to treatments. LP08 is a patent-protected investigational drug for IC comprised of pure liposomes. The purified phospholipid component of LP08 is known to be safe as a food source when eaten or delivered as a component of intravenous nutrition. There is no expectation that a novel route of bladder administration will present any safety issues. We will investigate the safety and efficacy of LP08 in an IND trial of ulcerative IC patients. Lipella Pharmaceuticals Inc. is a company formed by the inventors of LP08 in May of 2005. Lipella has received phase-I and phase-II SBIR support, as well as significant financial support from the Pittsburgh Life Sciences Greenhouse, a biotechnology investment organization, and accredited investors. The IND clinical trial will be conducted at the William Beaumont Hospital, the co-applicant of this STTR. Beaumont's urology department chairman, Kenneth Peters, is internationally recognized as a leader in the treatment and study of ulcerative IC and has a database of over 100 ulcerative IC patients. The urology department at the William Beaumont Hospital has been one of the leading centers in the world for the past 15 years on the study of IC and a top center in the NIDDK sponsored multi-institutional trials on IC including the ICDB, ICCTG and the ICCRN. This Fast-track STTR proposal advances commercial research funded by a prior phase-I NIH SBIR grant which has been completed, as well as a subsequent phase-II SBIR grant which has also been funded and is currently in progress. (The reason that the fast-track application process is chosen is so the pilot human batch produced in phase-I can produce both the regulatory data needed for the IND application and serve as the initial clinical supply without concerns for product shelf life and storage.) These prior and existing grants fund the preclinical development of a novel treatment for IC. The current STTR proposal would fund the clinical development of this product. Successful completion of this STTR would provide significant clinical data on a novel therapy for a syndrome with large unmet clinical demand, an asset highly attractive to institutional investors. In Summary, the aims of this proposal are designed to obtain significant clinical data for an experimental interstitial cystitis drug, by leveraging ongoing results of the cGMP liposome manufacturing capabilities at Lipella Pharmaceuticals and the IC-specific clinical capabilities of William Beaumont Hospital. PUBLIC HEALTH RELEVANCE: Successful completion of this STTR would provide significant clinical data on a novel therapy for interstitial cystitis, a syndrome with large unmet clinical demand. The development of an IND as well as the production of the first batch of product for human use is the focus of the phase-I portion of the current STTR proposal. IND approval from the FDA is the milestone between the phase-I and phase-II portions of this fast-track proposal. Manufacturing for and operational of the IND clinical trial is the focus of the phase-II portion of this STTR grant proposal.

描述（由申请人提供）：LP08是一种新型膀胱内治疗方法，用于治疗间质性膀胱炎（IC），这是一种人们知之甚少、病因不明的慢性疾病，包括刺激性膀胱症状和盆腔疼痛，严重影响生活质量。在 IC/PBS 中，通常识别出两种不同的亚型。溃疡性（经典）IC 和非溃疡性 IC/PBS 在膀胱镜检查结果和治疗症状反应方面存在明显差异。 LP08是一种受专利保护的IC研究药物，由纯脂质体组成。众所周知，LP08 的纯化磷脂成分在食用或作为静脉营养成分输送时作为食物来源是安全的。预计膀胱给药的新途径不会出现任何安全问题。我们将在溃疡性 IC 患者的 IND 试验中研究 LP08 的安全性和有效性。 Lipella Pharmaceuticals Inc. 是一家由 LP08 的发明者于 2005 年 5 月成立的公司。 Lipella 获得了 SBIR 的 I 期和 II 期支持，以及生物技术投资组织匹兹堡生命科学温室 (Pittsburgh Life Sciences Greenhouse) 和认可的投资者。 IND临床试验将在本STTR的共同申请人William Beaumont医院进行。博蒙特泌尿外科主任 Kenneth Peters 是国际公认的溃疡性 IC 治疗和研究领域的领导者，拥有超过 100 名溃疡性 IC 患者的数据库。 William Beaumont 医院的泌尿外科在过去 15 年中一直是世界领先的 IC 研究中心之一，也是 NIDDK 赞助的多机构 IC 试验的顶级中心，包括 ICDB、ICCTG 和 ICCRN 。这项快速 STTR 提案推进了商业研究，该研究由先前已完成的 NIH SBIR 第一阶段拨款以及随后也已获得资助且目前正在进行的第二阶段 SBIR 拨款资助。 （选择快速申请流程的原因是，一期生产的中试人体批次既可以产生 IND 申请所需的监管数据，又可以作为初始临床供应，而无需担心产品保质期和储存。 ) 这些先前和现有的赠款为 IC 新型治疗方法的临床前开发提供资金。目前的 STTR 提案将为该产品的临床开发提供资金。该 STTR 的成功完成将为一种针对临床需求大量未满足的综合征的新疗法提供重要的临床数据，这对机构投资者来说是极具吸引力的资产。总之，该提案的目的是通过利用 Lipella Pharmaceuticals 的 cGMP 脂质体制造能力和 William Beaumont 医院的 IC 特异性临床能力的持续成果，获得实验性间质性膀胱炎药物的重要临床数据。公共卫生相关性：该 STTR 的成功完成将为间质性膀胱炎的新疗法提供重要的临床数据，间质性膀胱炎是一种临床需求尚未得到满足的综合征。 IND 的开发以及第一批人用产品的生产是当前 STTR 提案第一阶段部分的重点。 FDA 的 IND 批准是该快速通道提案的第一阶段和第二阶段部分之间的里程碑。 IND 临床试验的制造和运营是本 STTR 拨款提案第二阶段部分的重点。