Medeor Therapeutics, a private, clinical-stage biotechnology company dedicated to the discovery, development and commercialization of personalized cellular immunotherapies, has received clearance from the FDA for an Investigational New Drug (IND) application for the company’s lead product candidate MDR-101. In addition, the FDA has agreed to a Special Protocol Assessment (SPA) for the design of a pivotal phase III clinical study of MDR-101 in.

MDR-101 is a first-in-class precision cellular immunotherapy designed to eliminate the need for chronic anti-rejection therapies in kidney transplant recipients with a one-time infusion. A single dose of MDR-101 has been shown to produce hematologic mixed chimerism, the co-existence of both recipient-derived and donor-derived blood and immune cells in the recipient, which supports clonal deletion and immune regulation, the biologic mechanisms underlying immune tolerance. By achieving mixed chimerism, MDR-101 is able to essentially reprogram a recipient’s immune system to accept the donor kidney, inducing donor-specific immune tolerance and thereby preventing transplant organ rejection.

“The current standard of care to prevent rejection of organ transplants is far from optimal due to unacceptably high long-term failure rates and treatment-associated complications, and new approaches are greatly needed to relieve organ transplant recipients from the litany of undesirable effects associated with anti-rejection drug regimens,” said D. Scott Batty, Jr., M.D., chief medical officer of Medeor. “MDR-101 has the potential to address the two most critical transplant patient needs: preventing organ rejection and mitigating anti-rejection treatment-associated toxicities. While our first indication is HLA-matched, living donor kidney transplants, the proprietary technology on which MDR-101 is based has the potential for use in all solid organ transplant recipients, regardless of degree of HLA match.”

The prospective, randomized, multi-center, open-label, controlled phase III clinical study is designed to evaluate the efficacy and safety of MDR-101 in HLA-matched living donor kidney transplant recipients, with the primary endpoint being functional donor-specific immune tolerance. Functional immune tolerance is defined as remaining off all immunosuppression (anti-rejection) drugs after completion of anti-rejection immunosuppressant drug therapy withdrawal, compared to control. Under the terms of the SPA, if the phase III study demonstrates safety and efficacy, this would lead to the submission of a biologics license application (BLA).

The kidney is the most commonly transplanted organ, with more than 79,000 kidney transplants performed globally in 2014.1 In the U.S. alone, more than 18,000 kidney transplants were performed in 2015.2

“I am pleased that the Medeor team has accomplished so much in a short period of time,” said Steven R. Deitcher, M.D., co-founder, president and chief executive officer of Medeor. “In addition to filing the IND and reaching an SPA agreement with the FDA for a single pivotal study of MDR-101, we successfully executed technology transfer from Stanford University, established an efficient and scalable MDR-101 manufacturing process, and were issued U.S. patents for the underlying technology. We now have a clear regulatory pathway for MDR-101 and look forward to initiating the phase III study in the fall of 2017. Concurrently, we will continue to maximize the potential of our personalized cellular immunotherapy technology by advancing our MDR-102 program in HLA-mismatched, living donor kidney recipients, MDR-103 program in persons with an existing living donor kidney, exploring opportunities in liver transplants and developing a manufacturing process appropriate for deceased donor transplants.”