Clinical Development a Therapeutic Agent for COPD

Hypersecretion of mucus into the respiratory airways is a major contributing factor in several lung diseases, including chronic pulmonary disease (COPD), asthma, cystic fibrosis, and bronchiectasis. Despite the obvious medical importance, there presently are no effective therapies to control excess mucus secretion in these diseases, and very few potential therapeutic targets. We discovered that a specific protein called MARCKS is a key molecule in the mucus secretary pathway. Based on this finding BioMarck developed a novel peptide, which inhibited mucus hypersecretion in human airway epithelial cells in culture and in a rodent model of asthma. With funding from SBIR Phase I and Phase II grants, BioMarck continued work on this project, has accomplished GMP manufacturing of a peptide (BIO-11006) that inhibits MARCKS, and successfully finished the nondinical safety studies of BIO-11006 in order to support an Investigational New Drug (IND) application to the FDA. This SBIR Phase II Competing Continuation grant application, if funded, will allow BioMarck to file an IND with FDA and conduct a dose-escalating, placebo-controlled, double-blind Phase 1 clinical trial in 56 healthy volunteers designed to evaluate the safety and tolerability of BIO-11006. This will be followed by testing BIO-11006 efficacy and pharmacodynamics in 184 COPD patients in a multi-center double-blind, placebo-controlled Phase 2a clinical trial. This project, if successful will lead to commercialization of BIO-11006 by providing data required for further expanded clinical trials in collaboration with a large pharmaceutical company. Worldwide, the sodal and economic burden of COPD is enormous. COPD, being the fourth leading cause of death, accounts for over 3 million deaths each year worldwide. The respiratory market is desperate for effective drugs for the treatment of asthma and COPD.