CTI-1601

CTI-1601 for Friedreich’s ataxia

Discovered by R. Mark Payne, MD, at Indiana University School of Medicine, CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with Friedreich’s ataxia (FA). Based solely on the results of our nonclinical development program, we believe CTI-1601 is processed to mature frataxin and becomes active in mitochondrial metabolism. Due to a genetic abnormality, patients with FA are unable to produce enough of this essential protein. Currently in a Phase 1 trial, CTI-1601 has been granted Rare Pediatric Disease designation, Fast Track designation and orphan drug status by the U.S. Food and Drug Administration (FDA).

About Friedreich’s Ataxia (FA)

Friedreich’s ataxia (FA) is a rare, progressive, multi-symptom genetic disease that typically presents in mid-childhood and affects the functioning of multiple organs and systems. The most common inherited ataxia, FA is a debilitating neurodegenerative disease resulting in multiple symptoms including progressive neurologic and cardiac dysfunction – poor coordination of legs and arms, progressive loss of the ability to walk, generalized weakness, loss of sensation, scoliosis, diabetes and cardiomyopathy as well as impaired vision, hearing and speech. FA affects an estimated 4,000-5,000 individuals living in the United States and between 18,000 and 20,000 patients in the European Union. FA results from a deficiency of the mitochondrial protein, frataxin (FXN), which is found in cells throughout the body. To date, there are no medical treatment options approved for patients with FA.

We are currently studying CTI-1601, a recombinant fusion protein intended to deliver human frataxin to patients with Friedreich’s ataxia in a Phase 1 trial. For more information, click here.

Patient Resources

FA affects an estimated 4,000-5,000 individuals living in the United States and between 18,000 and 20,000 patients in the European Union. There are currently no approved treatments and no cure for FA.