Galapagos Cystic Fibrosis Drug Enters Phase I, Triggering €8.5M Payout

Galapagos will receive a $10M (€8.5M) milestone payment from its collaborator, AbbVie, after their cystic fibrosis candidate started a Phase I trial.

Galapagos is a clinical-stage biotech developing small molecule medicines for a range of indications. It partnered up with the global pharma company, AbbVie, back in 2013, to develop a number of cystic fibrosis (CF) candidates for a combination therapy. Galapagos has pocketed $10M (€8.5M) from AbbVie on the back of the news that its CF corrector, GLPG3221, will enter Phase I to test its safety, tolerability and pharmacokinetics in healthy patients.

Around 70,000 people are living with CF worldwide, a progressive disease that sees patients suffer from persistent lung infections and breathing difficulties. The disease is caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that leads to the buildup of thick, sticky mucus in the lungs, pancreas and other organs.

GLPG3221 is one of a number of candidates that Galapagos and AbbVie are looking at to form a triple combination therapy. This particular candidate is a corrector drug, meaning it helps to restore the regular function of the CFTR channel. It is a C2 corrector so part of the second generation of these drugs, which are chemically distinct from their predecessors.

Cystic fibrosis is caused by a mutation in the CFTR gene found on chromosome 7. The effects of the disease are not only felt in the lungs, but also in other organs including the pancreas.

Abbvie walked away from a massive deal with Galapagos in 2015 but decided to stay on board for the cystic fibrosis project. The biotech-pharma power couple seems to have got over its initial squabbles, with this being its second Phase I trial of the year. Galapagos CSO, Piet Wigerinck, told us why the company can attract powerful partners like AbbVie and Gilead: “We just have a very attractive model for pharma.”

The global cystic fibrosis therapy market was estimated to be worth $3.5B (€3B) in 2016, but this is expected to rise to almost $14B (€12B) by 2025 due to increasing prevalence and rising treatment rate. Like Galapagos’ candidate, Vertex’s combination drug could help CF-patients to live ‘normal’ lives again. Despite its financial trouble, ProQR has completed a Phase Ib trial with its lead CF candidate, while Antabio received €7.6M to develop a new antibiotic against Pseudomonas infections.

Galapagos and AbbVie appear to be progressing nicely towards their goal of a triple combination CF treatment, with candidates trickling through to the clinic. Galapagos has received $67M (€57M) in milestone payments from the partnership so far, which has no doubt played a big part in giving its CF candidates, and the rest of its pipeline, a big boost.