Tarix Pharmaceuticals (www.tarixpharma.com) today announced that it's drug, TXA127, has been granted Orphan Drug designation from the U.S. Food and Drug Administration (FDA) as a treatment to enhance engraftment in patients receiving a stem cell transplant. TXA127 has also received Orphan Drug designation for the treatment of Myelodysplastic Syndrome (MDS).
TXA127 is a small peptide which stimulates early hematopoietic precursor cells in the bone marrow. This leads to an increase in all formed cells in the blood, including platelets, neutrophils, lymphocytes, and red blood cells. TXA127 is currently in a multi-center Phase II trial for the acceleration of engraftment in patients undergoing autologous peripheral blood stem cell transplants as well as multiple clinical trials to evaluate the effects of TXA127 on the acceleration of engraftment in patients receiving double cord blood transplants.
After receiving a stem cell transplant, it can take many weeks for the stem cells to repopulate the patient's blood. During this time the patient is at risk of serious infection or bleeding. There are no drugs available to speed up the engraftment process.
"Most attempts to enhance engraftment involve expansion of the number of stem cells by culture in the laboratory prior to transplantation in the patient. TXA127, on the other hand, in effect, expands the stem cells in the patient after the transplant," according to Rick Franklin, CEO of Tarix Pharmaceuticals.