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.
