CF is caused by mutations in the CFTR gene that lead to an abnormal transport of chloride across cell membranes, resulting in defective hydration of the epithelial surfaces of the body and consequent mucous build up on the outside of the cells. The respiratory and digestive systems are the most affected by the disease. CF patients require lifelong treatment with multiple daily medications and ultimately may need a lung transplant, which is not curative but extends life expectancy.
Galapagos and AbbVie began a partnership in 2013 to discover and commercialize new CF therapies, and plan on developing a triple CFTR combination therapy that might address 90 percent of all CF cases. This combination therapy will include molecules that restore two of the required CFTR biomolecular activities: the correctors, which will increase CFTR levels at the cell surface, and the potentiators, which will induce the correct opening of the CFTR channel.
According to a press release, Galapagos is starting a randomized, double-blind placebo-controlled Phase 1 trial to study the company’s second potentiator compound, GLPG2451. The study will test a range of doses in healthy volunteers from the Netherlands and Belgium, with results expected in the fourth quarter of 2016. It comes with a $10 million milestone payment from AbbVie.
GLPG2451 is the third compound in the Galapagos’ portfolio to enter the clinic. The first potentiator, GLPG1837, is being tested in patients with G551D and S1251N mutations under the SAPHIRA exploratory Phase 2 program, with results expected in the second half of 2016. Its first early binding corrector, GLPG2222, entered a Phase 1 trial in January, and topline results are likely by mid-2016.
Other preclinical candidates, the early binding GLPG2851 and the late-binding correctors GLPG2665 and GLPG2737, have been selected for Phase 1 clinical trials.
Combined, these compounds are designed to allow chloride ion transport and significantly improve hydration of the lung and pancreatic ducts surface, ameliorating the symptoms of CF patients.
CF is a rare, life-threatening genetic disease that affects approximately 80,000 patients worldwide.
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