ACG-701 (Cystic Fibrosis)

Aceragen is developing ACG-701, a novel oral antibiotic in the United States. ACG-701 is an investigational therapy for the acute treatment of cystic fibrosis-related pulmonary exacerbations (PEx).

Cystic fibrosis (CF) is a progressive, genetic disease that causes mucus build-up in the lungs, recurrent lung infections and declining lung function over time. Pulmonary exacerbations (PEx), defined as episodes of acute worsening of symptoms, are the driving force of lung function decline. There is currently no FDA-approved therapy for the treatment of CF-related PEx.

ACG-701 is a novel 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 used outside the United States for over 50 years and is included in ex-US cystic fibrosis treatment guidelines. Sodium fusidate is a potent S. aureus antibiotic and has potential anti-inflammatory and anti-mucin properties.

We are conducting a Phase 2 clinical trial in the United States (the REPRIEVE study) to evaluate the safety and efficacy profile of ACG-701 in individuals with acute cystic fibrosis-related pulmonary exacerbations.

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