Belgian biotech firm Galapagos NV (Euronext: GLPG) has developed a series of novel potentiators in its cystic fibrosis program, and says it expects to see clinical trials in late 2014. The data will be presented at the North American Cystic Fibrosis Conference taking place from October 17-19 in Salt Lake City, Utah.
In its CF research program, Galapagos developed novel potentiator series that are highly potent and that show superior opening of the defective CFTR channel in comparison to Vertex Pharmaceuticals’ (Nasdaq: VRTX) Kalydeco (ivacaftor). This was shown in assays of cells that carry a CFTR class II, III or IV mutation.
Furthermore, the superior channel opening was confirmed in primary cells from F508del and G551D patients. In addition to its efficacy, the Galapagos series show good metabolic stability and permeability and favorable PK profiles. In comparison to Kalydeco, the new potentiators have a superior in vitro safety profile, including a clean CYP profile and high selectivity. These results encourage Galapagos to nominate a preclinical candidate from this series at the end of this year, with the first clinical trials starting at the end of 2014. Galapagos has pending patents on these novel potentiators.
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