Galapagos dosed the first cystic fibrosis (CF) patient with GLPG2222, its novel CF corrector, in a Phase 2a clinical trial evaluating the drug as an add-on therapy to Kalydeco (ivacaftor) in CF patients harboring one F508del CFTR mutation and one gating mutation.
The ALBATROSS Phase 2a clinical trial was designed to evaluate the safety and tolerability of two doses of oral GLPG2222 or a placebo in up to 35 subjects with cystic fibrosis for 29 days. Study participants must be on stable treatment with Kalydeco for at least 28 days before starting the trial.
“The aim of the ALBATROSS study is to enhance our understanding of GLPG2222 in a CF patient population,” Piet Wigerinck, chief scientific officer of Galapagos, said in a press release.
Secondary objectives include assessing percent-predicted forced expiratory volume in one second (ppFEV1; a measure of lung function) and changes in sweat chloride, among others. Galapagos expects top-line results to be available in late 2017.
“With ALBATROSS we expect to learn more about our dosing modeling for the triple combination therapy; patients who participate may potentially help other CF patients still in need of a life-changing therapy,” Wigerinck said.
Galapagos and AbbVie agreed to collaborate in 2013 to develop potentiator and corrector molecules for the treatment of cystic fibrosis. The ultimate goal of the collaboration is to develop a new triple combination therapy to treat 90 percent of all CF patients.
The three drugs in the therapy have different mechanisms of action to restore the malfunction of the CFTR protein, which is defective in CF patients.
The two companies developed a number of drug candidates and backups for each of the three components of a potential triple combination. GLPG2222 is the first early binding corrector in Galapagos’ portfolio.
Galapagos also announced it has filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA), triggering a $10 million milestone payment from AbbVie.
Kalydeco was developed and commercialized by Vertex for cystic fibrosis patients. Kalydeco is a potentiator of the CFTR protein and was the first drug to address the underlying causes of CF instead of focusing only on its symptoms.
The drug was first approved by the FDA for adults with CF in December 2014. It was later approved for children ages 2 and older after new research was published in March 2015. Kalydeco is also available in Europe, Canada, Australia, and New Zealand.