Galapagos are developing a new series of potentiators that show superior CFTR opening in comparison to Kalydeco. This superior opening was seen with class 2, 3 or 4 mutations, including F508del and G551D (see graph/image).
This new series are highly potent and have favourable PK profiles. These potentiators have a superior in vitro safety profile in comparison to Kalydeco, including a clean CYP profile (less interaction with other medications).
Galapagos plan to nominate a pre-clinical candidate by the end of 2013 and plan to start clinical trials with this potentiator by the end of 2014.
Galapagos & AbbVie Alliance
“In September 2013 Galapagos and AbbVie entered into a global alliance to discover, develop and commercialize novel therapies in cystic fibrosis (CF), an inherited chronic disease that affects 70,000 people worldwide. AbbVie and Galapagos are working collaboratively, contributing technologies and resources to develop and commercialize oral drugs that address the main mutations in CF patients, including F508del and G551D. The goal of the collaboration is to identify compounds that correct defects in expression (corrector) of and/or increase the activity (potentiator) of the main mutations in the cystic fibrosis transmembrane regulator (CFTR) protein, including the F508del mutation, which is the most common with 90 percent prevalence among patients with CF.”
This image, from the Galapagos website, shows the planned timeline.