PHILADELPHIA--(BUSINESS WIRE)--Medeor Therapeutics today announced positive interim data from its multicenter, international Phase III clinical trial. Kidney transplant recipients who received a living donor kidney from an HLA-matched relative achieved functional tolerance and were free from the regimen of immunosuppressive drugs. Typically, these drugs are required to prevent rejection and failure of the kidney transplant. Study results will be presented during a late-breaking oral presentation at the American Society of Nephrology (ASN) Kidney Week 2023 Annual Meeting.
Following a kidney transplant, patients today are required to take daily immunosuppression medications for the rest of their lives to prevent rejection of the transplanted kidney. Stopping or skipping medication may cause a rejection to occur. Kidney rejection is hard to diagnose in its early stages and is often not reversible once it starts and can result in the loss of the transplanted kidney. However, a lifetime of immunosuppression drugs increases the risk of cancer, diabetes, infection and other medical problems. The litany of side effects and toxicities ultimately impact graft and patient survival such that 30% to 50% of all kidney transplants fail by 10 years post-transplant1. Last year saw a record number of kidney transplants with more than 25,000 patients undergoing the procedure, a 3.4% increase from 2021, according to the United Network for Organ Sharing.
Medeor’s lead product candidate, MDR-101, is a single-dose cellular therapy derived from a living kidney donor’s blood. The therapy is designed to establish mixed chimerism, which occurs when a low level of donor blood cells remains in the blood of the kidney recipient after infusion of donor stem cells. The study was designed to see whether kidney transplant recipients who received a living donor kidney from an HLA-matched relative with the same transplant genes, could taper immunosuppression to tacrolimus monotherapy by six weeks, followed by complete withdrawal by one year and remain off them for at least 2 years after withdrawal without graft loss, death, acute kidney rejection or graft versus host disease.
The study protocol was originally designed by the late Dr. Samuel Strober, M.D., professor of immunology and rheumatology at Stanford University and one of the founders of Medeor, who was dedicated to finding a way to free transplant recipients from the burden of immunosuppressant drugs. Twenty patients received MDR-101 and were compared to 10 similar patients who had standard of care. Results include:
- The study’s primary efficacy endpoint was met and exceeded. To date, 12 patients, or 63% of the study participants (compared to the protocol anticipated success rate of 48%), completed the trial and have been off immunosuppression therapy for two years.
- 4 additional patients have less than six months to complete the trial and currently remain free of immunosuppression therapy.
- 3 patients resumed immunosuppression therapy during the trial and one patient withdrew from the study at six months.
Moreover, the Kidney Disease Quality of Life (KDQOL-36) survey demonstrated statistically significant improvements in the Treated group vs Control group at two- and three-years post-transplant. These include Burden of Kidney Disease, which is defined by interference with daily life, or the patient feeling like a burden, and Mental Health, which includes depression, anxiety, etc.
“The results of this study have the potential to truly change the trajectory of the treatment for kidney transplants,” said Giovanni Ferrara, President and CEO, at Medeor Therapeutics. “By combining donor and recipient cells to create mixed chimerism and immune tolerance within the transplant recipient, we are working to alleviate the stress and burden of daily immunosuppressants, thereby improving quality of life and prolonged graft survival. We are buoyed by the study results presented today and look forward to the completion of our study and making MDR-101 the new treatment standard for donor matched kidney transplants.”
"The results from this study are extremely promising. This innovative trial provides direction toward reducing the need for life-long anti-rejection medications in transplant recipients,” said Dr. Dixon Kaufman, Medical Director, UW Health Transplant Center at the University of Wisconsin. “With the achievement of mixed chimerism in this trial, we are on course to provide a safer and more effective treatment approach for many patients in need of a kidney transplant.”
Regulatory Status
MDR-101 is being studied under an IND in the U.S. and a CTA in Canada and has been granted Orphan Drug Designation in both the U.S. and EU. MDR-101 is also designated as a Regenerative Medicine Advance Therapy (RMAT) by the FDA. The clinical trial is being conducted under an FDA Special Protocol Assessment (SPA).
About MDR-101
MDR-101 is a cellular therapy manufactured from a living kidney donor’s blood and peripheral stem cells. MDR-101 is intended to induce donor specific immune tolerance in order to avert transplant kidney rejection, eliminate the cumulative and serious side effects associated with immunosuppressive drugs and thereby preserve transplant kidney function and survival. The study was supported by the California Institute of Regenerative Medicine (CIRM). For more information on Medeor’s phase 3 trial, please visit www.clinicaltrials.gov, (NCT03363945).
About Medeor Therapeutics
Medeor Therapeutics is working to improve the lives of transplant patients by eliminating or reducing the need for a life-long regimen of immunosuppressant medications and their potential life-threatening side effects. Medeor’s Phase 3 clinical study demonstrates the significant opportunities of this one-time therapy. For more information, visit www.medeortx.com.
1 Poggio ED, Augustine JJ, Arrigain S, Brennan DC, Schold JD. Long-term kidney transplant graft survival-Making progress when most needed. Am J Transplant. 2021 Aug;21(8):2824-2832. doi: 10.1111/ajt.16463. Epub 2021 Feb 8. PMID: 33346917.