ACG-701 (Cystic Fibrosis)


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.

Cystic Fibrosis

Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and declining lung function over time. There are over 70,000 cystic fibrosis patients globally with approximately 30,000 patients in the United States. The driving force of lung function decline are “pulmonary exacerbations”, episodes of acute worsening of symptoms that result in decreased lung function. Staphylococcus aureus infections affect as high as 70% of all cystic fibrosis patients leading to repeated pulmonary exacerbations. Fusidic acid is one of the most potent S. aureus (both methicillin-sensitive and methicillin-resistant) antibiotics.  It is also anti-inflammatory and has mucin-inhibitory properties. As a result, Fusidic acid has already been included in ex-US cystic fibrosis treatment guidelines.