Medeor Therapeutics Receives FDA Clearance of IND Application and Special Protocol Assessment for Pivotal Clinical Study of MDR-101

Print
| Source: Medeor Therapeutics

Personalized cellular immunotherapy designed to prevent transplant kidney rejection and eliminate the need for chronic anti-rejection drugs

Single Phase 3 study will evaluate functional immune tolerance in HLA-matched, living donor kidney transplant recipients

Company anticipates initiating the Phase 3 study in the second half of 2017

SAN MATEO, Calif., April 28, 2017 (GLOBE NEWSWIRE) -- Medeor Therapeutics, Inc., a private, clinical-stage biotechnology company dedicated to the discovery, development and commercialization of personalized cellular immunotherapies, today announced it has received clearance from the U.S. Food and Drug Administration (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 3 clinical study of MDR-101 in recipients of human leukocyte antigen (HLA)-matched, living donor kidney transplants.

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., MD, 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 3 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 3 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, MD, 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 3 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.”

About the Special Protocol Assessment
A Special Protocol Assessment (SPA) is a process in which a sponsor and the U.S. Food and Drug Administration (FDA) reach an official agreement on the protocol design, endpoints and analysis of a Phase 3 clinical study prior to initiation to determine if they adequately address scientific and regulatory requirements for FDA approval. Final marketing approval is contingent on the efficacy results, safety profile and an evaluation of the treatment risks and benefits as demonstrated in the Phase 3 study, among other requirements. For additional information on the SPA process, please visit the FDA website at www.fda.gov.

About MDR-101
Medeor’s series of cellular immunotherapy products for kidney transplant recipients, known collectively as MDR-10X, are paradigms of personalized and precision medicine. These products are manufactured from transplant kidney donor-derived stem cells and are engineered using patent protected methods and processes to create a unique formulation of multiple different types of hematopoietic (i.e., blood or bone marrow derived) cells. The doses of CD34+ hematopoietic stem and progenitor cells (HSC) and the CD3+ T cells are critical to the action of Medeor’s products. Cell doses are guided by the degree of immunologic disparity between the recipient and donor (i.e., degree of HLA match or mismatch). A single dose of MDR-101, for example, 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.

About Medeor Therapeutics, Inc.
Medeor Therapeutics is a private, clinical-stage biotechnology company dedicated to the discovery, development and commercialization of transformative and personalized cellular immunotherapies to improve outcomes in organ transplant recipients. The company’s therapies leverage a patented cell manufacturing process and are based on breakthrough technologies discovered at Stanford University, for which Medeor holds exclusive worldwide licenses. The company’s lead product candidate, MDR-101, will be evaluated in a single Phase 3 clinical study in HLA-matched, living donor kidney transplant recipients. Medeor was founded in 2014 and is based in San Mateo, Calif. For more information, visit www.medeortherapeutics.com.   

Sources

  1. Global Observatory on Donation and Transplantation, 2014 Total Kidney Transplants Globally: http://www.transplant-observatory.org/data-charts-and-tables/chart/, April 21, 2017.
  2. Hart A et al. OPTN/SRTR 2015 Annual Data Report: Kidney. Am J Transplant 2017;17 (Suppl 1):21-116.

Media Contact

Tony Plohoros
6 Degrees
tplohoros@6degreespr.com 
(908) 591-2839