Chondrial Therapeutics Announces Dosing of First Patients in Phase 1 Clinical Program of CTI-1601 for Treatment of Friedreich’s Ataxia

CTI-1601 granted Rare Pediatric Disease Designation and Fast Track Designation by U.S. FDA

BALA CYNWYD, PA, USA I December 18, 2019 I Chondrial Therapeutics, a clinical-stage biotechnology company focused on developing treatments for rare diseases, with an initial focus on Friedreich’s ataxia (FA), today announced that the first patients have been dosed in a Phase 1 clinical trial to evaluate the safety and tolerability of single ascending doses of CTI-1601. CTI-1601 is a recombinant fusion protein intended to deliver human frataxin to patients with FA, who have decreased levels of frataxin.

“CTI-1601 is designed to address the root cause of Friedreich’s ataxia – low levels of frataxin. The initiation of our Phase 1 clinical program was supported by positive preclinical data and is an important step forward for patients affected by FA, a devastating and progressive disease for which there is no cure,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Chondrial Therapeutics. “We separately announced today a merger that will provide us with significant resources to advance our development of CTI-1601 and expand our efforts in other rare diseases that may benefit from a cell-penetrating peptide approach.”

The double-blind, placebo-controlled trial will enroll adult patients over age 18 with FA. It will evaluate the safety, tolerability and pharmacokinetics of single ascending doses of subcutaneously administered CTI-1601. Topline data from the Phase 1 clinical program are expected by the end of 2020.  

“On behalf of FARA and the FA community, we are grateful to the Chondrial team for their hard work and dedication in advancing CTI-1601 to this important milestone for a debilitating, progressive disease that lacks adequate treatment options and is currently only treated symptomatically,” said Jennifer Farmer, Executive Director of the Friedreich’s Ataxia Research Alliance (FARA). “FARA is committed to assisting Chondrial with recruitment for this trial by reaching out to individuals with FA enrolled in the Friedreich's Ataxia Global Patient Registry and other awareness efforts.”

Chondrial also announced that the U.S. Food and Drug Administration (FDA) has granted CTI-1601 Rare Pediatric Disease (RPD) Designation and Fast Track Designation. Fast Track Designation is intended to expedite review and facilitate development of drugs which are being developed to treat a serious or life-threatening condition and demonstrate the potential to fill an unmet medical need. With RPD Designation, Chondrial may also qualify for a voucher at the time of a potential future approval of CTI-1601 that can be redeemed to receive a priority review of a subsequent marketing application.

Additional information on the trial can be found on using the identifier NCT04176991.

About Friedreich’s Ataxia
Friedreich’s ataxia (FA) is a debilitating, life-shortening, progressive rare genetic disease that affects multiple body systems, particularly the brain and heart. It is the most common ataxia and is caused by a deficiency of a key protein, frataxin, which is normally present in the mitochondria of all tissues. Children and young adults progressively lose the ability to walk and speak, and their hearts become very thick and weak, and can eventually fail. Although rare, FA is the most common form of hereditary ataxia, affecting about 1 in every 50,000 people.1 There are currently no approved treatments and no cure for FA.

About CTI-1601
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), where researchers believe it 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. CTI-1601 is the only frataxin replacement therapy in clinical development. The U.S. Food and Drug Administration (FDA) granted orphan drug status to CTI-1601, which is currently in a Phase 1 trial.

About Chondrial Therapeutics
Chondrial Therapeutics is a clinical-stage biotechnology company focused on the treatment of complex rare diseases. The company’s lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program as a potential treatment for Friedreich’s ataxia, a rare and progressive genetic disease. Chondrial Therapeutics also plans to use its protein replacement therapy platform to design other fusion proteins to target additional orphan diseases characterized by deficiencies in intracellular bioactive compounds. Learn more at

SOURCE: Chondrial Therapeutics

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