Novel Cystic Fibrosis Treatment Reduces Frequency of Exacerbations in Phase 2 Study


Celtaxsys’ acebilustat is the first novel anti-inflammatory molecule with the potential to both reduce pulmonary exacerbations and prolong time to first exacerbation.

Acebilustat, a novel cystic fibrosis (CF) treatment, showed clinically meaningful improvement in pulmonary exacerbations in patients with CF, according to newly-released data from a phase 2 trial.

Based on results from the phase 2 EMPIRE-CF trial, acebilustat is the first novel anti-inflammatory molecule with the potential to both reduce the frequency of pulmonary exacerbations (PEx) and prolong time to first exacerbation.

The trial evaluated acebilustat for the treatment of CF in 200 patients, irrespective of causative genotype, over 48 weeks of therapy.

On a per protocol assessment, patients treated with acebilustat demonstrated a 19% reduction in PEx and a 22% decreased risk in progressing to first PEx versus placebo. More than 40% of patients treated with acebilustat completed the study without experiencing a PEx, an increase of 32% compared with patients treated with placebo, according to the data. Additionally, the benefits of acebilustat on pulmonary exacerbations were apparent as early as 4 months after the start of treatment and persisted through the 48-week period of the study.

“Pulmonary exacerbations, which are a clinical marker of unbridled lung inflammation, are significant events leading to acute decompensation and chronic decline of lung function and are strongly related to reduced survival,” Stuart Elborn, MD, professor of respiratory medicine at Imperial College, director of the Adult CF Centre, Royal Brompton Hospital London, and professor of respiratory medicine at Queen’s University Belfast, said in a press release. “Given this, acebilustat has the potential to help protect patients from the progressive and irreversible damage that is associated with CF.”

The researchers observed no difference in lung function, as measured by the primary endpoint of FEV1 percent predicted (FEV1pp) compared with placebo-treated patients over the time of treatment. FEV1pp response did not correlate with PEx rates.

Overall, patients with less severe impairment of lung function (FEV1pp >75) showed the largest benefit from acebilustat treatment, achieving a 34% reduction in PEx rate, a 43% reduction in risk of experiencing their first exacerbation, and a 96% increased likelihood of being exacerbation free after 48 weeks of treatment. Additionally, patients concomitantly treated with CFTR modulator therapy exhibited a clinically meaningful 20% reduction in PEx, a 29% increased time to first exacerbation, and a 47% higher likelihood of no exacerbations compared with patients treated with CFTR modulators and a placebo.

“Patients in key prospectively-identified sub-groups, including those with mild obstruction at baseline or taking CFTR modulator therapy, derived the most benefit in pulmonary exacerbations,” Steven M. Rowe, MD, MSPH, professor of medicine and director of the Gregory Fleming James Cystic Fibrosis Research Center at the University of Alabama at Birmingham, said in a press release. “That benefit, when used in combination with a CFTR modulator, is an important consideration given the likelihood of an increase in the number of CF patients who are eligible to be treated with new CFTR modulators over the coming years.”

In the trial, acebilustat was well tolerated with no increased risk of infection. The most common adverse effects associated with acebilustat were infective pulmonary exacerbation, cough, nasopharyngitis, nasal congestion, headache, sputum increased, hemoptysis, and fatigue.

Full trial results will be presented this fall at the North American Cystic Fibrosis Foundation annual meeting.


Celtaxsys Announces Results of Phase 2 Trial Showing Clinically Meaningful Improvement in Pulmonary Exacerbations in Cystic Fibrosis Patients [news release]. Celtaxsys’s website. Accessed August 2, 2018.

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