Belgium-based biotechnology firm Galapagos has reported positive topline data from the Phase II ALBATROSS clinical trial of GLPG2222 in patients with heterozygous class III/F508del cystic fibrosis transmembrane conductance regulator (CFTR).
GLPG2222 is an investigational C1 corrector that was administered on top of 150mg twice-daily ivacaftor (Kalydeco).
The investigational candidate was found to be well-tolerated, with mild or moderate treatment-emergent adverse events and no serious adverse events.
A dose-dependent increase in FEV1 and a statistically significant dose-dependent decrease in sweat chloride were observed during the trial.
Galapagos CSO Dr Piet Wigerinck said: “The ALBATROSS results are the first results demonstrating that GLPG2222 is well tolerated in CF patients.
“In addition, the exposures achieved, coupled with the activity observed, support our dose selection plans for the investigational triple combination therapy.
“We are impressed with the magnitude of the effects we saw on sweat chloride and FEV1 in patients whose treatment with ivacaftor has been optimised following years of therapy.”
The randomised, double-blinded, placebo-controlled Phase II trial assessed the safety, tolerability and pharmacokinetics of once-a-day 150mg and 300mg doses of GLPG2222 in 37 subjects across Europe and Australia.
Throughout the trial, patients were given long-term stable Kalydeco treatment, and achieved targeted exposures of GLPG2222 consistent with those observed in healthy participants.
ALBATROSS principal investigator professor Scott Bell said: “The results of this trial are encouraging as they show that the addition of the novel CFTR corrector molecule GLPG2222 on top of highly efficacious CFTR modulator treatment already given for years in patients with gating mutations was well tolerated and may bring additional benefit to patients.”