The development of lung-directed therapeutics is limited by poor translational fidelity between preclinical models and early-phase clinical trials. We report a first-in-human Phase 0 intratarget microdosing study demonstrating the feasibility of intrapulmonary delivery and pharmacological interrogation of a novel inflammasome inhibitor. A 100 μg microdose of ADS032, a dual NLRP1/NLRP3...
Introduction High attrition in drug development remains a significant challenge for research and development organisations worldwide. Most novel drug compounds fail at an early stage, often without knowledge of target engagement or molecular efficacy at diseased tissue level. Phase 0 trials provide an opportunity to provide mechanistic data in a small number of patients at the earliest stage of...
Abstract The COVID-19 pandemic has highlighted the importance of efficient drug discovery in respiratory disease. The traditional set up of clinical trials is expensive and allows for significant attrition of new drugs, many of which undergo extensive safety testing before being abandoned for lack of efficacy. Phase 0 trials, named as they sit between pre-clinical research and phase I, allow...