BONE MARROW ENGRAFTMENT ENHANCER
Our Bone Marrow Engraftment Enhancer (BMEE) program is developing a first-line product, TBX-1400, to enhance the engraftment of hematopoietic stem and progenitor cells during an allogeneic hematopoietic stem cell transplant (HSCT). For several decades, HSCT has been used as a form of treatment for patients with hematological cancers or inherited blood disorders to establish a healthy and functioning immune system. Current HSCT methods, however, do not have a high rate of success and can lead to a delay or failure of engraftment. Transplant recipients often must undergo multiple transplants in order for the transplant to engraft. When a transplant is successful, it can take upwards of 12-16 months for the immune system to fully reconstitute. During this time and for patients whose engraftment is unsuccessful or delayed, the patient is left in an immunocompromised state and is susceptible to bacterial, fungal or viral infections. The persistence of these infections represents the bulk of the clinical costs and complications associated with HSCT. There are many improvements that could be made to the HSCT procedure to decrease the risk of complications and to increase the success rate of HSCT for the recipient.
Our BMEE goal is to increase the change of long-term survival of a HSCT recipient by:
increasing the probability of a successful engraftment;
increasing the rate of reconstitution of mature and functional lymphocytes; and
managing the complications associated with HSCT.
Our lead product candidate, TBX-1400, is composed of T- and B-cell depleted hematopoietic stem cells treated ex vivo with our proprietary fusion protein prior to HSCT. We have shown in preclinical models that TBX-1400 increases the rate of engraftment and immune reconstitution. We are now moving into human clinical trials to determine the safety and efficacy of TBX-1400 in patients receiving a HSCT.
TBX-1400 has received Orphan Drug Designation from the U.S. Food and Drug Administration and the European Medicines Agency.
Phase I Clincial Trial:
Safety and Early Efficacy Study of TBX-1400 in Patients with Severe Combined Immunodeficiency. Clinicaltrials.gov Identifier: NCT02860559
About Severe Combined Immunodeficiency
Severe combined immunodeficiency (SCID) is a life-threatening primary immunodeficiency disease caused by defects compromising the quantity and function of specialized immune cells called T-cells and B-cells. Infants with SCID suffer from repeated life-threatening infections that usually lead to a diagnosis by the age of three to six months. Left untreated, children continue to experience severe infections; this usually leads to death by 2 years of age. Children with SCID are currently treated using bone marrow transplantation to reconstitute a normal immune system. Outcomes of these transplants are best for the youngest infants, who are transplanted before they have suffered from a severe infection (most of these infants are either siblings of a proband or are identified by neonatal screening programs), and for patients with a matching family donor. In contrast, the results of transplants performed on older infants and those performed with cells from alternative donors are less favorable.