Date: 2017-02-23
Type of information: Treatment of the first patient
phase: 1
Announcement: treatment of the first patient
Company: Galapagos (Belgium)
Product: GLPG2451 and GLPG2222
Action mechanism: CFTR potentiator. GLPG2451 is a CFTR potentiator and GLPG2222 is a CFTR corrector. CFTR potentiators repair defects in chloride transport, and CFTR correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways. Galapagos and Abbvie aim to develop a triple CFTR combination therapy to address 90% of patients with CF. In order to bring a more effective therapy to patients, the companies have developed multiple candidates and backups for each of the three components of a potential triple combination. GLPG2451 is the second potentiator and the third compound in the portfolio to enter the clinic.
Disease: cystic fibrosis
Therapeutic area: Rare diseases - Genetic diseases
Country: Belgium
Trial details: The study is a First-in-Human, Phase I, randomized, double-blind, placebo-controlled study evaluating single and multiple ascending oral doses of GLPG2451 and combined multiple doses of GLPG2451 and GLPG2222 in healthy subjects. The purpose of the study is to evaluate safety and tolerability after single ascending oral doses and of multiple doses of GLPG2451 given to healthy subjects compared to placebo as well as of multiple doses of the combination of GLPG2451/GLPG2222 compared to GLPG2451/placebo.(NCT02788721)
Latest news: • On February 23, 2017, Galapagos announced dosing of the first healthy volunteer with a combination of novel corrector GLPG2222 and novel potentiator GLPG2451 for cystic fibrosis in a Phase 1 study. After completion of the dosing of GLPG2451 as a monotherapy, in the new cohorts the safety, tolerability and PK of 14 days co-administration of GLPG2451 and GLPG2222 is being evaluated. The randomized, double-blind, placebo controlled, single center study is being conducted in at least 40 healthy volunteers in Belgium. In order to bring a more effective therapy to the majority of cystic fibrosis patients, Galapagos and AbbVie have a portfolio of candidates addressing three complementary components for a potential combination therapy. In the present study, potentiator GLPG2451 and C1 corrector GLPG2222 are evaluated as potential components of this triple combination. Triple combinations of CF compounds in the portfolio have consistently shown restoration of healthy activity levels in in vitro assays with human bronchial epithelial (HBE) cells of patients with the F508del mutation. These combinations result in an increase in chloride transport compared to Orkambi® in HBE cells with the homozygous F508del mutation.
