ACG-701 (Melioidosis)

Aceragen is developing ACG-701, an investigational oral antibiotic for the treatment of melioidosis.

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 an acute illness complicated by bacteremia and septic shock. Despite conventional antibiotic therapy, mortality can be as high as 40%. One of the difficulties in treating this B. pseudomallei is that it can live within host cells at a very low acidic pH.

ACG-701 is an innovative dosing regimen of sodium fusidate. Sodium fusidate is a member of the fusidane class of antibiotics that acts against bacteria by inhibiting an important step in protein synthesis. Sodium fusidate has been shown to remain active at a low pH against B. pseudomallei, making it distinct from most other antibiotics.

We are conducting a Phase 2 clinical trial (the TERRA study) to evaluate the safety and efficacy of ACG-701 in patients with melioidosis.

For more information on the TERRA study, please refer to ClinicalTrials.gov.