NON-CYSTIC FIBROSIS BRONCHIECTASIS
Phase 2 planned for Q2 2022
Non-cystic fibrosis bronchiectasis (BRE) is a disease characterized by dilated airways with mild to moderate airway obstruction and impaired mucociliary clearance that leads to a complex interplay of mucus accumulation and chronic neutrophil-predominant airway inflammation and damage. Typically, patients with BRE suffer from chronic cough, sputum production, and frequent and recurring pulmonary infections, all of which lead to decreased quality of life, clinical decline, and premature death. To date, there are no FDA-approved therapies for mucus relief for patients with BRE.
Phase 3 planned for Q2 2022
Approximately 2,500 lung transplants are performed annually and approximately 16,000 people are living with a lung transplant in the US. Despite slight improvements in survival rates, the 5-year survival rate remains dismal at 60%, which is the lowest survival rate of all solid organ transplants. There is an urgent need in lung transplant for therapies that promote clearance of pathogens from the airways and decrease pathological inflammation, while not leaving a patient susceptible to infection.
NON-TUBERCULOSIS MYCOBACTERIA LUNG INFECTIONS
Phase 2 planned for Q3 2022
Approximately 80,000 people in the US have non-tuberculosis mycobacteria (NTM) lung disease. NTM lung disease is caused by pulmonary infections with Mycobacterial species other than tuberculosis. NTM lung disease is characterized by excess mucus, chronic cough, and progressive lung damage. It is treated with cycles of long-term cycles of multiple antibiotics. Currently, there are limited treatment options for patients to clear mucus and improve their quality of life. As NTM detection and prevalence increase and rates of antibiotic resistance increase, additional therapies are needed to improve the quality of life of patients and enable mucus clearance.
CHRONIC OBSTRUCTIVE PULMONARY DISEASE
Phase 2 planned for Q4 2022
A subpopulation of patients with pre-COPD (current or former smokers with preserved lung function) and COPD experience chronic productive cough that is often defined as chronic mucus hypersecretion. Mucus plugging has been shown to be independently associated with lung function outcomes and may correlate with future exacerbations. Currently, there are no approved treatments used to relieve mucus plugging or promote mucus clearance in COPD. Reducing mucus plugging may decrease exacerbation rate and slow disease progression, while improving the quality of life of these patients.
Investigator-initiated IND, Q2 2022
The estimated number of children and adult undergoing tracheostomy in the United States was 22.1 and 34.3 tracheostomies per 100,000 persons, respectively in 2012. Tracheostomy is indicated in adults and children with both critical and chronic illness to bypass upper airway obstruction, facilitate chronic positive pressure ventilatory support, and to aid in the clearance of lower airway secretions. Among children who are discharged to home with a tracheostomy, a significant proportion experience recurrent tracheobronchial infection requiring medical care. Among adults with tracheostomy placed after respiratory failure, one-year mortality has been reported as alarmingly high (46.5%).
The presence of a tracheostomy adversely affects mucociliary clearance, impairs cough, and introduces a source of infection in the airways. Children with chronic tracheostomy exhibit evidence of increased airway inflammation and a significant number are chronically colonized with Pseudomonas aeruginosa, placing them at high risk for hospitalization after tracheostomy. There are only a few proven therapies to address the pathophysiological state of tracheostomy or associated infections. However, there are no therapies that directly address excess mucus secretion or tracheobronchitis.