ACG-701 (Melioidosis)


Aceragen is engaged in the clinical development of ACG-701, a proprietary oral dosing regimen of sodium fusidate. The active component of ACG-701 is fusidic acid, which has been in clinical use outside the United States for over 50 years and has an excellent safety profile supported by decades of clinical use. Our plans include evaluating efficacy and safety of ACG-701 for the treatment of melioidosis and cystic fibrosis exacerbations.


Melioidosis is a life-threatening infectious disease caused by the facultative intracellular gram-negative saprophyte B. pseudomallei, endemic throughout Southeast Asia and occurring sporadically in other regions of the world. Transmission occurs either via percutaneous inoculation or inhalation, with peak cases occurring during the monsoon season in endemic areas, likely due to aerosolization of soilborne pathogens. Most patients present with acute illness complicated by bacteremia and septic shock. Dissemination can result in metastatic disease which can lead to kidney, pulmonary, liver, and splenic abscesses. Despite conventional antibiotic therapy, mortality can be as high as 40%. One of the difficulties in treating this pathogen is that it can live within host cells and do so at a very low, acidic pH. Fusidic acid has been shown to penetrate into this difficult to reach environment and still remain quite active at a low pH against B. pseudomallei, distinct from most antimicrobials.