Belgium-based Galapagos NV has started its Phase 1 clinical trial evaluating its novel C2 corrector GLPG2737 in healthy volunteers. The drug was developed as part of a triple combination therapy for the treatment of cystic fibrosis (CF).
The randomized, double-blind, placebo-controlled clinical trial will evaluate the safety, tolerability and therapeutic properties of single and multiple ascending doses of GLPG2737, administrated orally to 64 healthy volunteers in one clinical site in the Netherlands.
In the first part of the trial, researchers will evaluate the drug in single ascending doses, and in the second part, multiple ascending doses will be administered daily for 14 days.
The initiation of the study brings a $10 million milestone payment to Galapagos from AbbVie. Topline results are expected in mid-2017.
Galapagos and AbbVie have been working to develop a group of cystic fibrosis drug candidates for a triple combination therapy that corrects and restores the CFTR channel activity (which is defective in CF patients) through corrector drugs, and allows the channel to open properly through potentiator drugs.
GLPG2737 is the first of the next-generation corrector molecules developed, and the final drug necessary to complete the first triple combination therapy for CF.
Galapagos has other drugs currently being tested in the clinic, including the potentiator molecules GLPG1837 and GLPG2451, and the corrector GLPG2222.
In the lab, these triple combination therapies were able to restore normal activity levels of the CFTR channel in human bronchial epithelial (HBE) cell cultures of patients with the F508del mutation in the CFTR gene. Mutations of the CFTR gene affecting chloride ion channel function lead to cystic fibrosis and result in the dysregulation of epithelial fluid transport in the lung, pancreas and other organs.
“We are pleased to initiate a Phase 1 study with the first of our C2 correctors for cystic fibrosis,” Dr. Piet Wigerinck, chief scientific officer of Galapagos, said in a company press release. “This step brings us closer to our goal of initiating a patient evaluation of a triple combination therapy by mid-2017.”