Galapagos has reported topline results from its Saphira 1 Phase 2 study in cystic fibrosis patients with potentiator GLPG1837.
The company said that 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.”