CTI-1601 is an investigational medicinal therapy candidate for the treatment of Friedreich’s ataxia (FA). It is being developed by Chondrial Therapeutics, a biotechnology company that focuses on the treatment of rare mitochondrial diseases.

FA is a genetic neuromuscular disease caused by a genetic defect that inhibits the expression of the FXN gene. FXN encodes for a protein called frataxin, which is an essential mitochondrial protein. Mitochondria are energy-producing centers in the cell. Inadequate amounts of frataxin protein lead to reduced mitochondrial functions, including reduced energy production and increased oxidative stress.

Lack of frataxin eventually leads to muscle weakness, loss of coordination, vision impairment, and other motor function problems. The patients progressively lose the ability to walk and speak, and their heart muscles become very thick and weak, and eventually may fail. FA has no approved treatments or cure.

How CTI-1601 works

CTI-1601 uses a novel technology in which a carrier protein delivers the frataxin protein to the mitochondria of patients with FA. In the mitochondria, this carrier-bound frataxin is processed to mature frataxin, which then becomes active in mitochondrial metabolism. This way, deficient protein levels could be restored, which can resume normal mitochondrial function. This may further minimize patients’ symptoms and prevent disease progression.

Other information

On Aug. 1, 2017, Chondrial Theraprutics secured funding of up to $22.6 million to advance the development of CTI-1601 for the treatment of FA. At the same time, the company licensed the technology of CTI-1601.

On Aug. 3, 2017, the U.S. food and drug administration (FDA) granted CTI-1601 orphan drug designation for the treatment of FA. Orphan drug status makes the investigational drug eligible for certain development incentives, including tax credits for clinical testing and exemption from a prescription drug user fee. Orphan drug designation also entitles the company to a period of seven years of market exclusivity upon FDA approval of the drug.

Chondrial Therapeutics is working to complete all studies that may enable an investigational new drug  application to be filed with the FDA and, upon acceptance of the application by the FDA, begin human (Phase 1) clinical trials.

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