Chondrial Therapeutics Licenses Friedreich’s Ataxia Therapy, Plans Further Development

Chondrial Therapeutics has licensed the investigational compound CTI-1601 for treating  Friedreich’s ataxia (FA) from Indiana University Research and Technology Corporation (IURTC) and Wake Forest University Health Sciences.

The company said it has also secured $22.6 million in financing to advance the treatment.

“The $22.6 million Series A financing, combined with the licensing of CTI-1601, is a watershed moment for Chondrial,” Carole Ben-Maimon, MD, the company’s new president and chief executive officer, said in a press release.  “I believe the company is in prime position to advance a potentially groundbreaking therapeutic for patients with Friedreich’s Ataxia and capitalize on a broader vision of targeting and treating rare, debilitating mitochondrial diseases.

“We are very appreciative of Deerfield Management, which led the financing and will be providing continuing strategic and operational support,” she said. “Additionally, we look forward to working with the scientific community, the Friedreich’s Ataxia Research Alliance, and the FDA to advance this important therapeutic.”

CTI-1601 was developed by R. Mark Payne, MD, professor of pediatric cardiology at Indiana University School of Medicine and director of the Indiana Clinical Translational Sciences Institute’s Translational Research Training Program.

Friedreich’s ataxia is a progressive neurodegenerative disorder caused by mutations of the frataxin gene. A normal gene oversees the production of a certain amount of frataxin protein. Mutations lead to much less production.

Initial FA symptoms include poor coordination, including walking problems. It can also lead to scoliosis, heart disease and diabetes.

People with FA have low levels of frataxin, which helps to move iron in the body and is involved in the formation of iron-sulfur clusters. These are necessary for the correct function of the mitochondria in cells that produce energy.

CTI-1601 uses a carrier protein to deliver frataxin to mitochondria. Once it’s there, researchers believe, it is processed into mature frataxin, a transformation that prompts it to become active in mitochondrial metabolism.

Increasing the level of the protein could lead to normal function of mitochondria and a decrease in patients’ symptoms, researchers believe.

“We have worked for the past 15 years to develop this investigational  treatment for Friedreich’s Ataxia patients, which I believe holds considerable promise,” Payne said. “This work has been supported by the Friedreich’s Ataxia Research Alliance, along with multiple national funding agencies. The investment from Deerfield represents a key validation of CTI-1601 and Chondrial’s plan to bring the product into the clinic.”

Chondrial plans to file an Investigational New Drug application for CTI-1601 with the U.S. Food and Drug Administration. When the application is approved, it will start a Phase 1 study of CTI-1601 as a treatment for FA.

“The progress of CTI-1601 from discovery to its licensing to Chondrial is an important milestone in the collective effort to advance this novel and promising therapeutic approach to address the high unmet need for treatment for individuals with Friedreich’s Ataxia.  We congratulate Chondrial in this important step as a company and are excited by the prospects of advancing this technology toward the clinic and look forward to continued partnership and support for the program,” said Jennifer Farmer, executive director of the Friedreich’s Ataxia Research Alliance.