Celtaxsys Inc. reported promising results today from an early stage clinical study of a potential anti-inflammatory drug for people with cystic fibrosis. Scientists believe that reducing excessive inflammation in people with CF will prevent lung damage and slow progression of the disease.
Although this is an early stage, Phase 2b study, the results suggest the potential for people with CF to benefit from the drug, acebilustat. The drug is intended to reduce production of a molecule that leads to inflammation and is known to be elevated in people with CF. According to Celtaxsys, when compared to participants on placebo, those who took acebilustat had a:
- 19 percent reduction in pulmonary exacerbations
- 22 percent reduced risk of having their first pulmonary exacerbation
Notably, patients with less severely impaired lung function (those with an FEV₁ greater than 75) experienced the largest benefit from acebilustat, achieving a 34 percent reduction in pulmonary exacerbations after 48 weeks of treatment.
Reducing exacerbations in CF is important because they can cause permanent lung damage and decrease lung function, increasing the risk of dying. People with CF experience this sudden worsening of symptoms because they have an exaggerated immune response to infections. This causes excessive inflammation that not only kills the germs causing the infection, but also healthy lung tissue.
Researchers are trying to discover ways to dampen this exaggerated immune response without affecting the body's natural defenses against infection. During the study, participants who took the drug did not experience an increased risk of infection or significant side effects from acebilustat.
Based on these encouraging results, Celtaxsys plans to have a larger Phase 3 trial to continue to test the drug's effect on inflammation in cystic fibrosis.
Because of the detrimental role inflammation plays in CF, the Cystic Fibrosis Foundation is committed to supporting the development of new anti-inflammatories and new anti-infectives -- therapies that can help all people with CF regardless of their type of mutation. The Foundation has provided $8 million in funding to Celtaxsys to support the development of this drug.
For more information, see the Celtaxsys press release.