HLA-matched Living donor kidney transplant (LDKT)
Our lead product candidate is MDR-101, a cellular therapy manufactured from a living kidney donor’s blood and bone marrow cells. The therapy is designed to allow certain kidney transplant recipients — with a genetically matched, or HLA-matched, living kidney donor — to stop all immunosuppressive (anti-rejection) drug use, while preserving long-term transplant kidney function and survival.
A Phase 2 trial of the Stanford predecessor product candidate demonstrated the ability to achieve persistent mixed chimerism in the majority of patients. Approximately 74% have been off all immunosuppressive drugs for more than two years, including some with ongoing durable responses lasting five to 10 years from the time of stopping all immunosuppressive drugs. We are currently enrolling patients in a pivotal Phase 3 clinical trial. More information on this trial is available at https://clinicaltrials.gov/ct2/show/NCT03363945.
Our product candidate MDR-102 is a cellular immunotherapy for kidney transplant recipients with a genetically mismatched, or HLA-mismatched, living donor intended to reduce the risk of kidney rejection, minimize immunosuppressive (anti-rejection drug) therapy and, thereby, improve long-term transplant kidney function and survival. Like MDR-101, this therapy is also manufactured from a kidney donor’s blood and bone marrow cells. However, it uses a different proprietary composition.
In a Phase 1/2a study of the Stanford predecessor product candidate, Stanford researchers identified a CD34+ cell dose that resulted in the achievement of sustained mixed chimerism lasting 12 months or longer. Recipients with 12 months of sustained mixed chimerism were able to reduce their anti-rejection drug requirement to a single drug (monotherapy) without GvHD, acute rejection, allograft loss, or patient death We expect to open a Phase 2/3 trial of MDR-102 in the fourth quarter of 2019. More information on this clinical trial is available at https://clinicaltrials.gov/ct2/show/NCT03605654.
Delayed Tolerance LDKT
Our product candidate intended for delayed therapy in transplant patients is MDR-103. This product is designed to treat transplant recipients who received an HLA-matched living donor kidney transplant months or years before and whose donor is still living. MDR-103 is intended to allow these patients to eliminate all immunosuppressive drug use, while preserving transplant kidney function and survival.
We expect to initiate a Phase 2 proof-of-concept trial of MDR-103 in these patients. The primary efficacy endpoint is the proportion of subjects achieving persistent mixed chimerism, defined as achieving mixed chimerism for at least six months. We also intend to explore delayed therapy in HLA-mismatched kidney transplant recipients. More information on this clinical trial is available at https://clinicaltrials.gov/ct2/show/NCT03606746.
Sickle Cell Disease
This product candidate targets adult patients with hemoglobinopathies, such as sickle cell anemia or sickle-β-thalassemia major (HbSβ0). We believe the CD34+ hematopoietic stem and progenitor cells, contained in MDR-301, leads to the establishment of stable mixed chimerism and the production of red blood cells with normal hemoglobin. Lowering the relative amounts of sickle hemoglobin and replacing it with normal adult hemoglobin may reduce sickle cell disease-related symptoms, most notably debilitating pain crises from vaso-occlusive episodes.