Mechelen, Belgium; 20 December 2016 – Galapagos NV (Euronext & NASDAQ: GLPG) reports topline results from its SAPHIRA 1 Phase 2 study in cystic fibrosis patients with potentiator GLPG1837.
The SAPHIRA 1 trial included 26 patients with the G551D mutation in CFTR each receiving three sequential doses of GLPG1837. Of these, 25 patients were on stable Kalydeco treatment at screening and agreed to a one week washout prior to the start of dosing GLPG1837. One patient was naïve to Kalydeco. All subjects received GLPG1837 125 mg bid (twice-daily) for 7 days, immediately followed by 250 mg bid for 7 days and subsequently by 500 mg bid for 14 days.
A statistically significant dose dependent decrease in sweat chloride concentration was observed. At the 500 mg bid dose, sweat chloride decreased from a mean value of 98 mmol/L at baseline to 66 mmol/L (p <0.0001). For those patients exceeding the predicted target concentration, sweat chloride changed from a mean value of 94 mmol/L at baseline to 52 mmol/L.
25 patients were on stable treatment with Kalydeco prior to this study. For these patients, mean percent predicted FEV1 (ppFEV1) levels were 74% at screening (prior to Kalydeco washout). The one week wash-out resulted in a 5.4% mean decrease in absolute ppFEV1. At the end of treatment with GLPG1837, the ppFEV1 levels returned to the Kalydeco pre-washout levels.
Overall GLPG1837 was well tolerated, with observed treatment emergent adverse events being predominantly mild or moderate, and typical for a CF patient population. One patient dropped out of the study due to an increase in non-cardiac creatine phosphokinase.
“The success of this trial is an important milestone in two regards; firstly, GLPG1837 has shown safety and significant efficacy as a novel CFTR potentiator. Secondly, it demonstrates that the CF community is committed to the further development of CFTR modulators despite the complexities related to evolving standards of care,” commented Prof Jane Davies of the Royal Brompton & Harefield NHS Trust in London and principal investigator for SAPHIRA 1.
“The SAPHIRA 1 results show this is the first new potentiator since Kalydeco to demonstrate competitive results in patients harboring the G551D mutation. Galapagos has a suite of potentiators in development. Galapagos and AbbVie will further study the data before deciding which potentiator will be included in the triple combination,” said Dr Piet Wigerinck, CSO of Galapagos. “The clinical validation of our in vitro systems reinforces our belief in our approach to get to a triple combination therapy.”
Galapagos (Euronext & NASDAQ: GLPG) is a clinical-stage biotechnology company specialized in the discovery and development of small molecule medicines with novel modes of action. Our pipeline comprises a pipeline of Phase 3, Phase 2, Phase 1, pre-clinical, and discovery programs in cystic fibrosis, inflammation, fibrosis, osteoarthritis and other indications. We have discovered and developed filgotinib: in collaboration with Gilead we aim to bring this JAK1-selective inhibitor for inflammatory indications to patients all over the world. Galapagos is focused on the development and commercialization of novel medicines that will improve people’s lives. The Galapagos group, including fee-for-service subsidiary Fidelta, has approximately 480 employees, operating from its Mechelen, Belgium headquarters and facilities in The Netherlands, France, and Croatia. More information at www.glpg.com.