Galapagos NV has developed a series of novel potentiators in its cystic fibrosis program. The data will be presented at the North American Cystic Fibrosis Conference taking place from 17 - 19 October 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 Kalydeco®.
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 pre-clinical 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.
Galapagos initiated its research in CF in 2005 as part of a collaboration with the Cystic Fibrosis Foundation. In 2010 Galapagos decided to pursue CF as the first orphan disease in which the company is attempting to discover, develop and launch its own medicines.
In September 2013 Galapagos signed an agreement with AbbVie where they will work collaboratively to develop and commercialize oral drugs that address the main mutations in CF patients, including F508del and G551D.
Galapagos will present its novel potentiators at a poster session (abstract #41) at NACFC on 17 October at 11:50 am and 18 October at 4 pm.