Celtaxsys has completed its Phase 2b trial assessing the safety and effectiveness of acebilustat (formerly CTX-4430) for cystic fibrosis (CF), with the company announcing the last visit of the last enrolled patient.
Details of the trial, supported by the Cystic Fibrosis Foundation, will be presented at the upcoming 41st European Cystic Fibrosis Society Conference June 6-9 in Belgrade, Serbia, in an oral presentation titled “Demographics of patients in a phase 2 trial of acebilustat in patients with cystic fibrosis (EMPIRE CF).”
The company expects to release clinical trial results in July.
“Completing this trial represents a significant milestone for the development of our lead medicine, the company and the CF community. We are excited to explore once daily acebilustat’s potential to reduce excessive lung inflammation as a complement to a CF patient’s current treatment regimen, irrespective of genotype,” Greg Duncan, president and CEO of Celtaxsys, said in a press release.
“We would like to thank all of the patients and medical professionals who helped execute this groundbreaking program and are very much looking forward to sharing the Phase 2b trial results with the cystic fibrosis community this summer,” he said.
In the EMPIRE CF trial (NCT02443688), 200 cystic fibrosis patients at sites across the U.S. and Europe were randomized to receive either a placebo or one of two oral doses of acebilustat — 50 mg or 100 mg — once a day for 48 weeks, or 11 months.
Researchers performed follow-up visits every four weeks — from week four to week 52, four weeks after treatment completion.
The study’s main objective was to determine the safety and tolerability of a once-daily oral dose of acebilustat. Safety was determined by the incidence of treatment emergent adverse events compared with controls.
Researchers assessed the therapy’s effectiveness by measuring the changes, from the beginning of the study until its completion at 52 weeks, in forced expiratory volume in one second (FEV1) percent predicted — a measure of lung function.
Acebilustat inhibits an enzyme involved in the production of the so-called leukotriene B4 (LTB4), a potent signaling molecule that promotes inflammation. Scientists believe LTB4 plays a key role in the development of diseases like cystic fibrosis that stem from inflammation. Acebilustat was developed to reduce inflammation and improve breathing.
The U.S. Food and Drug Administration and the European Medicines Agency have designated acebilustat an orphan drug, a status that covers therapies for rare diseases. The designation offers companies incentives to develop a therapy and opens the way to its accelerated regulatory approval.