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Tarix Pharmaceuticals’ TXA127 Receives Orphan Drug Designation for Stem Cell Engraftment

Tarix Pharmaceuticals today announced that its drug, TXA127, has been granted Orphan Drug designation from the 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 (NYSE: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,
  • TXA127 will also be evaluated in two clinical trials in cord blood transplantation. 1 of these, being conducted at M D Anderson, will involve adult patients, while the other, to be done at Duke University, will be performed with children,
  •  The Phase II study will evaluate the safety and efficacy of TXA127 in the acceleration of platelet recovery following autologous peripheral blood stem cell transplant in patients with Hodgkin lymphoma or non-Hodgkin lymphoma or multiple myeloma undergoing limited re-infusion of CD34+ cells,
  •  The 74 patient, double-blind, placebo-controlled trial is being conducted at 8 US medical centers.

Tarix is a private company developing peptide drugs for multiple indications. TXA127 is a naturally occurring peptide and is being developed to enhance stem cell transplantation, and for the treatment of acute respiratory distress syndrome (ARDS). TXA127 is also being developed for radiation protection.