An Improved Method of Delivering a Sclerosing Agent for the Treatment of a Malignant Pleural Effusion.

Project Summary / Abstract. We are developing an improved method to treat patients with malignant pleural effusions (MPE), a serious condition that occurs in 175,000 patients in the US each year, affecting between 7 to 15% of all cancer patients. MPE results in an excessive accumulation of fluid in the pleural cavity, frequently leaving the patient to suffer from shortness of breath and overall poor quality of life. Management of MPE is palliative and the main problem with the current techniques such as pleurodesis is its clinical failure rate due to the inefficient and non-uniform distribution of the sclerosing agent. Currently, there is no standardization or consensus within the medical community on the optimal delivery technique for a sclerosing agent because of the tradeoffs and variability in patient satisfaction, efficacy, costs, and complexities. Our focus is on the development of an improved clinical approach to administering a sclerosing agent, which will overcome the problems with standard of care. In Phase I feasibility studies, we developed and evaluated several foam formulations and have selected a lead candidate. We fully characterized the lead, demonstrating its desired adhesiveness, gelation and reverse thermo-sensitive properties as well as its efficacy in an animal model. These results enabled us to optimize the lead formulation and in parallel select the final design of our proprietary delivery system with important end user input during our active Phase II project. The fully functional prototype will be evaluated for safety and efficacy during biocompatibility studies conducted under GLP conditions, in order to prepare a data package for an IND application to the Food and Drug Administration. Once successfully completed, these Phase II results will prepare us to initiate clinical trials where we will test the combination product in collaboration with several prestigious clinical partners who are an important part of our team. The proposed CRP aligns with the corporate strategy and timeline and would enable us to develop a clear regulatory path and help us accelerate the process of obtaining IND approval. Our multidisciplinary team lead by an experienced cardiothoracic surgeon and medical product developer, has the demonstrated requisite expertise ranging from innovating in chemical and mechanical engineering, navigating the FDA regulatory pathway, successfully commercializing pharmaceutical and medical device products, to successfully exiting companies. Working together with these experts and an experienced regulatory firm, we intend to accomplish an important milestone toward commercialization. The funding sought through the SBIR CRP program will further enable us to leverage our network for securing third party investors, many whom are already expressing interest. Our novel approach is expected to significantly impact the current clinical standard of care and result in markedly improved clinical patient outcomes with a simple, minimally invasive procedure.Project Narrative. We have developed a prototype combination product that improves the outcome of pleurodesis, a procedure used in the management of malignant pleural effusion (MPE), a complication of late stage cancer affecting 7 to 15% of all cancer patients. To accelerate our novel delivery system for administering the sclerosing agent, we will develop a regulatory strategy and pursue an Investigational New Drug application with the Food and Drug Administration. Our approach is expected to significantly impact the current clinical standard of care and markedly improve the clinical outcomes with a simple, minimally invasive procedure.