Second Component of Promising Investigational Cystic Fibrosis Combo Treatment Selected

Second Component of Promising Investigational Cystic Fibrosis Combo Treatment Selected

shutterstock_214333159Galapagos is a company specializing in novel mechanisms of small-molecule and antibody programs for cystic fibrosis.  In a recent press release, the company revealed that before the end of 2015, they are going to conduct a Phase 1 clinical trial of a new corrector called GLPG2222 in conjunction with a potentiator called GLPG1837 as treatment for patients with Cystic Fibrosis. Additionally, Galapagos said that another trial of a final triple component therapy will be chosen in 2015. Pre-clinical studies with the Galapagos combination have shown higher efficacy than other therapies for cystic fibrosis.

Cystic fibrosis (CF) is a heritable condition causing symptoms such as lung infections, sinus infections, stunted growth and diarrhea. Evidence has established that CF is caused by a mutation in the gene that encodes cystic fibrosis transmembrane conductance regulator (CFTR), a protein that regulates the mechanisms of sweat mucus and digestive juices. CF has no cure and therapies usually target the mutation in the delF508 that affects nearly 70% of CF patients. This therapy involves a combination of a corrector to repair the mutation plus a potentiator to allow the opening of the CF channel.

Galapagos and AbbVie are developing and commercializing molecules to address mutations in the CFTR gene found in Cystic Fibrosis. The potentiator GLPG1837 is in Phase 1, and the corrector GLPG2222 is in pre-clinical stage. The companies have developed various novel correctors that when triple combined with GLPG1837 revealed an encouraging >40% restoration of healthy activity in cells with the delF508 mutation derived from CF patients.

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Furthermore, the companies’ corrector series composites have shown promising features as therapy agents supporting additional pre-clinical development. From this composite series, Galapagos nominated the first corrector building block that in combination with the potentiator GLPG1837 forms a basis for their triple combination clinical trial.

In the press release, Dr. Piet Wigerinck Chief Scientific Officer of Galapagos, said “Galapagos and AbbVie have five different chemical series with several excellent compounds from which to choose its novel CF correctors. The novel corrector nominated today forms the second of three ingredients needed for building our triple combination therapy. This selection brings us a big step closer to realizing our ambition of developing novel and best-in-class therapies for CF. The Galapagos-AbbVie team is working on selecting a second corrector candidate from our series to augment further the CFTR delF508 levels obtained by the dual GLPG1837/corrector combination.”

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