HOW DOES A DRUG GET DEVELOPED?

DISCONTINUED: Stages of Development for Exenatide

The drug development process can be thought of as a series of stages, and drugs must successfully pass through each stage to become available to patients. This treatment has been evaluated, and the program has been discontinued. Thus, it is not in the pipeline.

Incretins are gut hormones that control blood sugar levels. Stable hormone analogs have been developed in recent years to treat diabetes. These incretin mimetics, such as GLP-1 analogues, are currently used for the treatment and management of type 2 diabetes. Unexpectedly, it was observed that these drugs induce frataxin expression in pancreatic ß-cells.

2014: FARA awarded the Phillip Bennett and Kyle Bryant Translational Research Award to Drs. Miriam Cnop, Mariana Igoillo-Esteve and Massimo Pandolfo at the Universite Libre de Bruxelles, Brussels, Belgium. This award allowed the team to further evaluate the potential of incretin analogs to induce frataxin and prevent or improve cell dysfunction. The team tested this in neuronal cells generated from induced pluripotent stem cells from FA patients.

A small pilot trial of incretin analogs in a few individuals with FA was conducted at the University hospital in Belgium.

A small increase in frataxin levels was observed in platelets derived from patients treated for 5 weeks with exenatide. However, no improvement on neurological functions as assessed by the SARA score or Activities of Daily Living scales, was detectable.