FDL169, a drug being developed by Flatley Discovery Lab, is targeted for cystic fibrosis (CF) patients who carry the F508del mutation, the most common mutation in this disease. The drug is designed to work as a corrector for the cystic fibrosis transmembrane regulator (CFTR), restoring its function. Clinical studies of this drug are still in an early stage, but preclinical work supports the treatment’s potential.

History of FDL169

FDL169 was announced as a drug candidate to treat CF patients in October 2013, shortly after the company revealed that preclinical data, including studies in rodents and dogs, showed that FDL169 significantly increased chloride transport and appeared to have a good safety profile. In these studies, it also showed a differentiated profile in vivo from others in this class of drugs (CFTR regulators).

Three Phase 1 studies of FDL169 have been completed, according to the clinical trials.gov website. These studies aimed at evaluating the oral dose, pharmacokinetics, and bioavailability of FDL169 in healthy volunteers.

FDL169 is also mentioned in a review study as an example of a CFTR corrector for the transport of the chloride ion through the CFTR protein.

How FDL169 works

CFTR proteins serve as channels, allowing the passage of charged ions, such as chloride. The passage of these ions have an important role in the movement of water in the tissues, allowing for the production of a thin mucus that protects and lubricates the reproductive system, organs such as the lungs and pancreas, and tissues.

Defective CFTR proteins are produced when a mutation (e.g., F508del) occurs in the gene that encodes this protein. The mutation causes the proteins to misfold, compromising the working of the channel and the passage of water through the cell membrane. As a consequence, mucus that is thick and sticky is produced.

Like other drugs of this family (e.g., Tezacaftor), FDL169 works to restore CFTR function, increasing the amount of the protein at the cell surface and its ability to work as a chloride channel. FDL169 can be used as a monotherapy, or in combination with other drugs that work to restore CFTR, the company reported.

Next steps for FDL169

According to the Cystic Fibrosis Foundation, Phase 2 studies are expected to begin soon. These studies will evaluate FDL169’s safety and effectiveness in CF patients with two copies of the F508del mutation.

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