Developing Intravesical Liposomes for Interstitial Cystitis

描述（由申请人提供）：LP 08是一种新型膀胱内治疗间质性膀胱炎（IC）的药物，IC是一种病因不明的慢性疾病，由刺激性膀胱症状和盆腔疼痛组成，严重影响生活质量。在IC/PBS中，通常识别两种不同的亚型。溃疡性（经典）IC和非溃疡性IC/PBS在膀胱镜检查结果和对治疗的症状反应方面明显不同。LP 08是一种受专利保护的IC研究药物，由纯脂质体组成。已知LP 08的纯化磷脂组分在作为静脉营养成分食用或递送时作为食物来源是安全的。预期新的膀胱给药途径不会出现任何安全性问题。我们将在溃疡性IC患者的IND试验中研究LP 08的安全性和有效性。Lipella制药公司是由LP 08的发明者于2005年5月成立的公司。Lipella已经获得了SBIR第一阶段和第二阶段的支持，以及来自匹兹堡生命科学温室（一个生物技术投资组织）和认可投资者的大量财政支持。IND临床试验将在本STTR的共同申请人William博蒙医院进行。博蒙泌尿科主任肯尼思·彼得斯是国际公认的溃疡性IC治疗和研究的领导者，拥有100多名溃疡性IC患者的数据库。William博蒙医院的泌尿外科在过去15年中一直是世界上IC研究的领先中心之一，也是NIDDK申办的IC多机构试验（包括ICDB、ICCTG和ICCRN）的顶级中心。这一快速通道STTR提案推进了由先前的第一阶段NIH SBIR赠款资助的商业研究，该赠款已经完成，以及随后的第二阶段SBIR赠款也已获得资助，目前正在进行中。(The选择快速通道申请过程的原因是，I期生产的中试人体批次可以产生IND申请所需的监管数据，并作为初始临床供应，而无需考虑产品有效期和储存。）这些先前和现有的赠款资助了IC新疗法的临床前开发。目前的STTR提案将资助该产品的临床开发。该STTR的成功完成将为具有大量未满足临床需求的综合征的新疗法提供重要的临床数据，这是对机构投资者极具吸引力的资产。总之，本提案旨在通过利用Lipella Pharmaceuticals的cGMP脂质体生产能力和William博蒙医院的IC特定临床能力的持续结果，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：成功完成该STTR将为间质性膀胱炎的新型治疗提供重要的临床数据，间质性膀胱炎是一种具有大量未满足临床需求的综合征。IND的开发以及第一批人用产品的生产是当前STTR提案第一阶段的重点。FDA的IND批准是这一快速通道提案的第一阶段和第二阶段之间的里程碑。IND临床试验的生产和运营是STTR资助计划II期部分的重点。

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.