Clinical Development a Therapeutic Agent for COPD

粘液分泌过多进入呼吸道是几种肺部疾病的主要因素，包括慢性肺病（COPD）、哮喘、囊性纤维化和支气管扩张。尽管具有明显的医学重要性，但目前还没有有效的治疗方法来控制这些疾病的过量粘液分泌，并且很少有潜在的治疗靶点。我们发现一种叫做MARCKS的特殊蛋白质是粘液分泌途径中的关键分子。基于这一发现，生物标记开发了一种新的肽，可以抑制培养和啮齿动物气道上皮细胞粘液的高分泌

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.