Acebilustat

An investigational drug for the treatment of cystic fibrosis and other inflammatory diseases

Acebilustat is a small molecule drug that is currently under investigation for its potential use in treating cystic fibrosis and other inflammatory diseases. It is being developed by Celtaxsys, a pharmaceutical company focused on novel treatments for inflammatory disorders.

Mechanism of Action

Acebilustat is an inhibitor of the enzyme leukotriene A4 hydrolase (LTA4H), which plays a crucial role in the production of leukotriene B4 (LTB4). LTB4 is a potent inflammatory mediator that is involved in the recruitment and activation of neutrophils, a type of white blood cell that contributes to inflammation and tissue damage in various diseases, including cystic fibrosis. By inhibiting LTA4H, acebilustat reduces the production of LTB4, thereby potentially decreasing inflammation and improving clinical outcomes in patients.

Clinical Development

Acebilustat has been evaluated in several clinical trials to assess its safety, tolerability, and efficacy in patients with cystic fibrosis. In these studies, acebilustat was generally well-tolerated, and some evidence suggested that it could reduce markers of inflammation and improve lung function in patients.

Phase 2 Trials

In a Phase 2 clinical trial, acebilustat was administered to patients with cystic fibrosis to evaluate its effects on lung function and inflammatory markers. The trial demonstrated that acebilustat was safe and showed a trend towards reducing pulmonary exacerbations, although the primary endpoint was not met.

Ongoing Research

Research is ongoing to further explore the potential benefits of acebilustat in cystic fibrosis and other inflammatory conditions. Additional studies are needed to confirm its efficacy and to determine the optimal dosing regimen.

Potential Applications

While acebilustat is primarily being developed for cystic fibrosis, its mechanism of action suggests that it may have potential applications in other diseases characterized by excessive inflammation and neutrophil activity, such as chronic obstructive pulmonary disease (COPD) and rheumatoid arthritis.

Also see

• Cystic fibrosis
• Leukotriene B4
• Neutrophil
• Chronic obstructive pulmonary disease
• Rheumatoid arthritis