Preclinical Development of a Therapeutic Agent for COPD

描述（由申请人提供）：粘液分泌过多进入呼吸道，导致阻塞和发病率和死亡率增加，是几种肺部疾病的主要因素，包括慢性阻塞性肺疾病（COPD）、哮喘和囊性纤维化。目前还没有有效的治疗方法来控制这些疾病的粘液分泌过多。在之前发表的研究中，我们发现一种名为MARCKS的特定蛋白质是粘液分泌途径的关键分子。在此基础上，我们发现了一种新的抑制人气道上皮细胞粘液分泌的肽。

DESCRIPTION (provided by applicant): Hypersecretion of mucus into the respiratory airways, leading to obstruction and increased morbidity and mortality, is a major factor in several lung diseases, including chronic obstructive pulmonary disease (COPD), asthma, and cystic fibrosis. There are presently no effective therapies to control excessive mucus secretion in these diseases. In previously published studies, we discovered that a specific protein called MARCKS protein is a key molecule in the mucus secretory pathway. Based on this finding, a novel peptide was discovered, which inhibited mucus hypersecretion in human airway epithelial cells in culture. With financial support from SBIR Phase I grant, BioMarck Pharmaceuticals has extended these studies and confirmed that this novel peptide is an effective inhibitor of mucus hypersecretion in three different animal models when instilled intratracheally. This discovery can potentially lead to a novel and effective therapy to inhibit excessive mucus secretion in a variety of pulmonary diseases. In the present SBIR Phase II proposal, we wish to further develop this peptide in order to support an IND (Investigational New Drug) application to the FDA for possible clinical studies in human volunteers. In this proposal, the Company wishes to accomplish two major objectives. Namely, a) to develop an optimum formulation of the peptide for nebulizer-based targeted lung delivery, and b) to establish the safety of the peptide when delivered by nebulizer to laboratory animals. The series of proposed safety studies are prerequisites for approval of an IND for subsequent human clinical studies. BioMarck's goal is to develop and commercialized a novel, effective, and safe therapeutic agent to satisfy the unmet medical needs for the treatment of mucus hypersecretory diseases like COPD, the 4th leading cause of death in the United States and worldwide.