US-based Flatley Discovery Lab plans to develop FDL169, an orally available small molecule, as a clinical candidate for the treatment of cystic fibrosis (CF).
The company has also started investigational new drug (IND -enabling studies and the secured data from these studies will be presented at the 27th Annual North American Cystic Fibrosis Conference to be held on 17 October in Salt Lake City, Utah.
Flatley Discovery Lab CEO Richard Fitzpatrick said FDL169 is a very potent and efficacious cystic fibrosis transmembrane conductance regulator (CFTR) corrector.
"FDL169 has a differentiated in vivo profile from other molecules in this class of drugs which we believe will produce a superior clinical profile," Fitzpatrick said.
"We believe that a combination of FDL169 and a CFTR potentiator will have a positive impact on the lives of CF patients with the ?F508 mutation."
According to the company, FDL169 has shown to significantly increase chloride transport in ex vivo human bronchial models of CF lung disease and is expected to have a good safety profile as per the preliminary studies of the molecule in rodents and dogs.
CF caused by a mutation in the CFTR protein is an autosomal recessive genetic disorder characterised by thick, sticky secretions in the lung, pancreas, liver and skin, as well as chronic pulmonary infections.
CFTR protein plays a major role in maintaining ion balance across membranes and the most common mutation in CF is ?F508, which causes the protein to misfold and not conduct chloride properly.
When used alone or in combination with a drug that potentiates CFTR, FDL169 helps in restoring CFTR dependent chloride transport.