Earlier today the first patient was enrolled in a Phase II clinical study of TXA127, a pharmaceutical grade formulation of Angiotensin (1-7), a naturally occurring peptide known to stimulate early hematopoietic precursor cells in the bone marrow, developed by Tarix Pharmaceuticals.

Angiotensin (1-7) is part of the renin-angiotensin-system (RAS). For many years researchers thought that it was an inactive by-product of degradation of other molecules in the RAS. In the 1990s, angiotensin (1-7) began to be studied for its action on myocardial tissue, and a specific receptor for angiotensin (1-7) was identified. This mas receptor is expressed in a variety of tissues, including bone marrow, brain, heart, and lung. The peptideis produced in-vivo by the action of the enzyme angiotensin converting enzyme 2 (ACE2), which removes the last amino acid from angiotensin II, producing the seven amino acid peptide, Angiotensin (1-7).

Bone marrow
Bone marrow, the soft, sponge-like material found inside bones, contains immature cells known as hematopoietic or blood-forming stem cells. Hematopoietic stem cells divide to form more blood-forming stem cells, or they mature into one of three types of blood cells, including white blood cells, red blood cells and platelets. Most hematopoietic stem cells are found in the bone marrow, but some cells, called peripheral blood stem cells (PBSCs), are found in the bloodstream. Blood in the umbilical cord also contains hematopoietic stem cells. Cells from any of these sources can be used in transplants.

Clinical trial
Aclinical trial (NCT01121120) is currently enrolling patients undergoing autologous peripheral hematopoietic stem cell transplant. The double-blind, placebo-controlled multi-center Phase II study will evaluate the safety and efficacy of TXA127 in the acceleration of engraftment following autologous peripheral hematopoietic stem cell transplant in patients with Hodgkin or non-Hodgkin lymphomas or multiple myeloma undergoing limited reinfusion of CD34+ cells. The study is expected to enroll a total of 74 patients at eight leading U.S. medical centers. TXA127 will also be evaluated in two clinical trials in cord blood transplantation. One conducted at M D Anderson will involve adult patients, while the other at Duke University, will enroll pediatric patients.

Increased risk of serious infection
Following a stem cell transplant, it can typically take several weeks for the stem cells to repopulate the patient’s blood with platelets and other mature blood cells. During this time the patient is at increased risk of serious infection and bleeding. There are currently no drugs available that speed up the engraftment process.

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“TXA127 has the potential to reduce the time it takes for blood cell counts to return to safe levels following stem cell transplant, which may accelerate the recovery process and reduce some of the health risks associated with stem cell transplant,” stated Rick Franklin, CEO of Tarix Pharmaceuticals. “TXA127 is unique because it directly stimulates transplanted stem cells to replenish circulating platelets, white blood cells, and red blood cells.”

TXA127 was 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).

For more information:
Acceleration of Platelet Recovery Following Autologous Peripheral Blood Stem Cell Transplant (PBSC) in Hodgkin, Non-Hodgkin Lymphoma or Multiple Myeloma Patients (TXA127-PBSC).

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