Development of NH101: A Simple and Efficient Outpatient Dry Powder Amphotericin B-DryNeb(dry powder nebulizer) for Treatment of Susceptible and Multidrug Resistant Aspergillus/ Fungal Infections

项目总结

Project Summary The overall objective of this project is to build on the successes of our Phase I SBIR studies to progress NH101: a pure (neat) dry powder of amphotericin B (AMB) combined with the DryNeb™, a novel dry powder nebulizer to “clinic ready” status. The results of this program will enable a Phase I clinical study (AUS), engagement with the FDA on the NH101 505(b)(2) regulatory approval pathway, and ultimately, an Investigational New Drug (IND) application and Phase II clinical proof of concept (POC) efficacy testing in Allergic Bronchopulmonary Aspergillosis (ABPA). NH101 (AMB-DryNeb™) satisfies multiple unmet medical needs by targeting common and triazole (antifungal classes, e.g., itraconazole) multidrug resistant (MDR) strains of aspergillus at the site of lung infection. NH101 will initially focus on cystic fibrosis (CF) and asthma-associated ABPA, a rare, orphan and severe disease of unmet medical need commonly associated with an estimated 2.5% of asthmatics (~5 million) and 15% (~20,000) of CF patients. This project will achieve three primary objectives: 1) refine, test, and produce the DryNeb™ to clinical-grade scale, 2) scale up and formulate AMB at Kg scale, and 3) perform a first in human-enabling repeat dose GLP toxicology study.