Chondrial Announces FDA Orphan Drug Designation for CTI-1601, a Novel Investigational Technology for the Treatment of Friedreich’s Ataxia

 

BALA CYNWYD, Pa.Aug. 3, 2017 — Chondrial Therapeutics, Inc., an emerging biotechnology company focused on the treatment of rare mitochondrial diseases, announced today that the US Food and Drug Administration (FDA) has granted orphan drug designation to its lead investigational drug candidate, CTI-1601, being developed for the treatment of Friedreich’s Ataxia.

 

Orphan drug designation is granted by the FDA Office of Orphan Products Development to drugs being developed for safe and effective treatment of diseases that affect fewer than 200,000 people in the U.S.  For a drug to qualify for orphan drug designation, FDA must determine there is a medically plausible basis for the use of the drug for the rare disease or condition.

 

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 may entitle the company to a period of seven years of market exclusivity upon FDA approval of the drug.

 

Carole Ben-Maimon, MD, president and CEO of Chondrial Therapeutics, commented, “We are very pleased to receive orphan drug designation for CTI-1601.  Our team is working to complete all Investigational New Drug (IND) enabling studies so that we can file an IND and begin human clinical trials.”

 

About Friedreich’s Ataxia

 

Friedreich’s Ataxia (FA) is a progressive disease that affects multiple body systems, particularly the brain and heart. FA is the most common ataxia in man 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. There is currently no cure for FA.

 

About Chondrial Therapeutics

 

Chondrial Therapeutics is an emerging biotechnology company focused on the treatment of rare mitochondrial diseases. Chondrial’s lead compound is CTI-1601, which the Company is developing as a potential treatment for Friedreich’s Ataxia. CTI-1601 is designed to deliver the frataxin protein to the mitochondria of patients with Friedreich’s Ataxia, who, due to a genetic abnormality, are unable to produce this essential protein.

 

Chondrial Therapeutics Secures Up to $22.6 Million in Series A Financing and Licenses Novel Technology for the Treatment of Friedreich’s Ataxia

Company Plans to Advance Pipeline of Product Candidates Focused on the Treatment of Rare Mitochondrial Diseases

 

Carole Ben-Maimon, MD, Named President & CEO

 

Bala Cynwyd, PA – January 31, 2017 – Chondrial Therapeutics, Inc., an emerging biotechnology company focused on the treatment of rare mitochondrial diseases, announced today that it has secured up to $22.6 million in Series A financing led by Deerfield Management and has appointed Carole Ben-Maimon, MD, as the company’s president and chief executive officer. Concurrently, Chondrial announced the licensing of a novel investigational technology, CTI-1601, for the treatment of Friedreich’s Ataxia from Indiana University Research and Technology Corp. (IURTC) and Wake Forest University Health Sciences. CTI-1601 was invented by R. Mark Payne, MD, professor of Pediatric Cardiology at Indiana University School of Medicine and director of the Translational Research Training Program of Indiana Clinical Translational Sciences Institute.

 

Chondrial plans to use the proceeds from its Series A financing to advance the development of CTI-1601 and to enhance its executive and management team as the company seeks to build a pipeline of product candidates focused on the treatment of rare mitochondrial diseases. The clinical development of CTI-1601, as well as Chondrial’s ongoing research and development targeting rare mitochondrial diseases, will be conducted at the company’s newly established laboratory at the Science Center in Philadelphia. Chondrial expects to file an Investigational New Drug (IND) application for CTI-1601 with the U.S. Food and Drug Administration (FDA) and, upon acceptance of the IND by the FDA, initiate Phase 1 clinical trials of the compound.

 

In conjunction with the financing, Chondrial has named Carole Ben-Maimon, MD, president and chief executive officer. Dr. Ben-Maimon brings to Chondrial more than 25 years of experience in research and development and biopharma management. Dr. Ben-Maimon joins Chondrial after serving as an advisor with Deerfield Management where she assisted in the evaluation of business and drug development plans in the brand and generic industry. Chondrial Therapeutics’ co-founders, Dr. Payne and Steven Plump, both of whom were part of the Indianapolis team that conducted proof of concept research on CTI-1601, will remain with Chondrial, with Dr. Payne retaining the positions of chief scientific officer and board member, and Steven Plump serving in an executive advisory capacity. Additionally, Thomas Hamilton, a member of the Board of Directors at the Friedreich’s Ataxia Research Alliance (FARA) and initial sole investor in Chondrial, has agreed to continue his role on Chondrial’s Board.

 

Carole Ben-Maimon, MD, president and CEO of Chondrial Therapeutics, commented, “The $22.6 million Series A financing, combined with the licensing of CTI-1601, is a watershed moment for Chondrial. 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. Additionally, we look forward to working with the scientific community, the Friedreich’s Ataxia Research Alliance, and the FDA to advance this important therapeutic.”

 

William Slattery, a partner at Deerfield Management, stated, “Chondrial, supported by the outstanding institutions of Indiana University and Wake Forest Baptist Medical Center, combined with the collaboration with the Friedreich’s Ataxia Research Alliance, creates an opportunity to develop treatments that could have potential wide-ranging benefits for patients. We are very excited to work with this outstanding team. I congratulate Dr. Ben-Maimon on her new position at Chondrial and look forward to Deerfield’s ongoing support of Chondrial as the company advances CTI-1601 toward an IND and into the clinic.”

 

Discovered by Dr. Payne while at Wake Forest Baptist Medical Center, CTI-1601 utilizes a carrier protein to deliver frataxin, the deficient protein in Friedreich’s Ataxia, to the mitochondria where researchers believe it is processed to mature frataxin and becomes active in mitochondrial metabolism. Friedreich’s Ataxia is a debilitating, life-shortening, degenerative neuromuscular disease that occurs in about 5,000 people in the United States, and about twice that number in Europe. The disease is caused by an abnormal gene, which is inherited in an autosomal recessive fashion and is responsible for the production of frataxin. People with Friedreich’s Ataxia have very low levels of frataxin, which is active in the mitochondria and assists in energy production. Chondrial’s premise in the research program is that by replacing the deficient protein the mitochondria will resume normal function, patients’ symptoms may be minimized, and disease progression may be curtailed.

 

Dr. Payne remarked, “We have worked for the past 15 years to develop this investigational treatment for Friedreich’s Ataxia patients, which I believe holds considerable promise. 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. I look forward to working closely with the highly experienced management team at Chondrial, as well as the team at Deerfield, to advance this technology and address additional opportunities to treat rare mitochondrial diseases.”

 

The core technology is based on intellectual property owned by Wake Forest Baptist Medical Center and licensed through its commercialization arm, Wake Forest Innovations, to Chondrial. The Catalyst Fund – a $15 million technology development program of Wake Forest Innovations, that is managed by Pappas Capital in Durham, N.C., will also make an investment in the Series A round as a minority participant.

 

Jeff Brennan, vice president, technology development and commercialization, Wake Forest Innovations stated, “These licensing and funding agreements reflect our mission to improve health through collaborative innovation. We are pleased to be a part of bringing this important discovery closer to positive patient outcomes. The Deerfield investment represents precisely the type of ‘multiplier effect’ we envisioned when creating the Catalyst Fund.”

 

Katherine Moynihan, Ph.D., technology manager at IURTC stated, “Dr. Payne’s research into CTI-1601 is one example of the excellent work conducted at Indiana University, and we now welcome the opportunity to support Chondrial in the ongoing advancement of CTI-1601. Ultimately, our hope and the hope of all those involved is that this will one day lead to a treatment for Friedreich’s Ataxia.”

 

Jennifer Farmer, Executive Director, Friedreich’s Ataxia Research Alliance (FARA), concluded, “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.”

 

About Chondrial Therapeutics

Chondrial Therapeutics is an emerging biotechnology company focused on the treatment of rare mitochondrial diseases. Chondrial’s lead compound is CTI-1601, which the Company is developing as a potential treatment for Friedreich’s Ataxia. CTI-1601 is designed to deliver the frataxin protein to the mitochondria of patients with Friedreich’s Ataxia, who, due to a genetic abnormality, are unable to produce this essential protein. Chondrial expects to file an Investigational New Drug (IND) application for CTI-1601 with the U.S. Food and Drug Administration (FDA) and, upon acceptance of the IND by the FDA, initiate Phase 1 clinical trials of the compound. For more information on Chondrial, please visit Chondrial Therapeutics.

 

About Deerfield

Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. For more information, please visit Deerfield.

 

About Wake Forest Innovations

Wake Forest Innovations improves health through collaborative innovation between industry and the faculty and staff of Wake Forest Baptist Medical Center. We accomplish this through our three centers: the Center for Technology Innovation & Commercialization, which accelerates the development and commercialization of inventions; the Center for Industry Research Collaboration, which expedites access to specialized clinical and research capabilities; and the Center for Applied Learning, which promotes best clinical practices through experiential training. For more information, please visit wake forest innovations.

 

About Indiana University Research and Technology Corp.

IURTC is a not-for-profit corporation tasked with the protecting and commercializing of technology emanating from innovations by IU researchers. Since 1997, IU research has generated more than 2,700 inventions resulting in more than 4,100 global patent applications being field by IURTC. For more information, please visit http://iurtc.iu.edu/.

 

About FARA

The Friedreich’s Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich’s Ataxia. For more information, please visit CureFA.org.

 

Contacts:

Andrew Mielach, Vice President
Tiberend Strategic Advisors, New York
T: +1 212 375 2694
amielach@tiberend.com