Galapagos NV has announced the first dosing in its Phase 2 exploratory program of GLPG1837 in
patients with cystic fibrosis (CF). GLPG1837 is a candidate CFTR potentiator drug in clinical development for the treatment of Class III mutations in cystic fibrosis.
The SAPHIRA Phase 2 program will explore the safety, tolerability and efficacy properties of GLP1837 in CF patients with a G551D (SAPHIRA 1) or S1251N (SAPHIRA 2) Class III mutation. Topline results from the SAPHIRA Phase 2 program are expected in Q4 2016.
“Today’s announcement is a landmark achievement in our CF program, with the first CF patient
being treated with a Galapagos potentiator,” said Onno van de Stolpe, CEO of Galapagos.
“Recruitment for the SAPHIRA program is rapid, and we look forward to seeing to what extent our
promising in vitro data translates into clinical results. We aim to start and report a number of
clinical studies with additional compounds in the CF portfolio throughout 2016.”
SAPHIRA 2, an open-label study of two doses of GLPG1837 in at least six CF patients with the
S1251N mutation, was first dosed in a patient last week. SAPHIRA 1, an open-label study of three
doses of GLPG1837 in at least 12 patients with the G551D mutation, is expected to begin dosing
soon. The SAPHIRA Phase 2 program will explore the safety, tolerability, efficacy, and medicinelike
properties of GLPG1837 in patients in six EU countries and Australia.
Primary objectives are to evaluate the safety and tolerability; secondary objectives are to assess changes in sweat chloride from baseline as the biomarker of cystic fibrosis transmembrane conductance regulator (CFTR) ion channel function and to explore the changes in pulmonary function (forced expiratory volume in 1 second [FEV1]) from baseline.
Both studies will include subjects treated with Kalydeco®1 as well as those who are naïve to this drug. In each study, different doses of GLPG1837 tablets will be administered twice daily for a total duration of four weeks.