Rick Franklin, M.D., Ph.D., Chief Executive Officer of Tarix, commented, “We are extremely pleased that TXA127 has been granted orphan drug status for this indication by the FDA. This is an important development for Tarix and is part of our strategy to build a portfolio of products using TXA127 and our synthetic peptide PanCyte to treat a variety of serious pulmonary diseases.”

Tarix is developing TXA127 for multiple therapeutic indications, including enhancement of engraftment following peripheral blood and cord blood stem cell transplantation and acute respiratory distress syndrome (ARDS).  In 2010, TXA127 was granted Orphan Drug designation 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).

About Pulmonary Arterial Hypertension

Pulmonary arterial hypertension is a rare disease that is very difficult to diagnose in its early stages as many of the initial symptoms can be nonspecific, resulting from insufficient cardiac output.  Prognosis for the disease is extremely poor.  Untreated patients have a median survival of only two and half years, with the most common cause of death being sudden death caused by right ventricular failure.  Not all patients respond to current treatments, which include calcium channel blockers and prostacyclin analogs.  For the majority of patients that are treated with these drugs, five year survival is only 54%.  Lung transplantation offers the only hope of cure but it is reserved for patients with end-stage disease that have failed all other therapies and it is associated with high morbidity because of rejection, infection, and bronchiolitis obliterans.

About Orphan Drug Status

Orphan drug designation qualifies a company for several benefits under the Orphan Drug Act of 1983 (ODA), as amended. These benefits include a 7-year period of orphan drug exclusivity upon product approval, a tax credit for certain clinical testing expenses for the orphan drug, written guidance on the non-clinical and clinical studies needed to obtain marketing approval of an orphan drug, and orphan drug grants.

About Tarix Pharmaceuticals

Tarix Pharmaceuticals is a clinical stage biopharmaceutical company developing peptide drugs to address significant unmet medical needs. The Company’s lead product, TXA127, is a pharmaceutical formulation of the naturally occurring peptide, Angiotensin (1-7). TXA127 is currently in a Phase II clinical trial for peripheral blood stem cell engraftment, and in earlier-stage trials for cord blood transplantation and myelodysplastic syndrome. TXA127 is also being developed, under grants from the US government, as a treatment for radiation exposure.

Tarix’s second product, PanCyte, is a synthetic, long-acting analogue of Angiotensin (1-7). PanCyte is in pre-clinical development focused on several disease areas with serious unmet medical need.

# # #

The Ruth Group
Joshua Drumm (investors) / Victoria Aguiar (media)
(646) 536-7006 / (646) 536-7013
jdrumm@theruthgroup.com / vaguiar@theruthgroup.com

Rick Franklin

Chief Executive Officer

Rick.franklin@tarixpharma.com

Umbilical cord blood-derived stem cells offer increased flexibility in the degree of human leukocyte antigen (HLA) matching required for transplantation and are a critical source of donor cells for patients for whom a source of immunologically compatible donor cells are not readily available. Following transplantation, the rate at which the infused stem cells are able to repopulate patients’ blood with platelets and other mature blood cells (engraftment) determines the risk of developing serious complications such as infection and bleeding. There are currently no drugs available that speed up the engraftment process.

“Initiation of our Phase 1 clinical program in DCBT marks another significant milestone in the development of TXA127 for enhancing engraftment following stem cell transplantation,” stated Rick Franklin, CEO of Tarix Pharmaceuticals. “This program complements our ongoing placebo-controlled Phase 2 clinical study in adults receiving autologous peripheral blood stem cell transplant. We are excited to advance these programs in order to address the unmet needs of the full spectrum of patients undergoing hematopoietic stem cell transplants.”e;

About TXA127
TXA127 is unique because it directly stimulates transplanted stem cells to replenish circulating platelets, white blood cells and red blood cells, potentially reducing 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.

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).

About the Phase 1 Program
Tarix expects to conduct multiple open-label Phase 1 clinical studies of TXA127 designed to evaluate the safety and efficacy of TXA127 in the acceleration of engraftment following double cord blood stem cell transplant, involving adult and pediatric patients. The first Phase 1 study described above is expected to enroll approximately 10 adult patients with acute myelogenous leukemia (AML), myelodysplastic syndrome (MDS), acute lymphocytic leukemia (ALL), chronic myelogenous leukemia (CML), chronic lymphocytic leukemia (CLL), Hodgkin’s lymphoma (HL), or non-Hodgkin’s lymphoma (NHL) at The MD Anderson Cancer Center. For more information, please visit http://clinicaltrials.gov/ct2/show/NCT01300611.

For more information about the ongoing Phase 2 double-blind, placebo-controlled multi-center study evaluating the safety and efficacy of TXA127 in the acceleration of engraftment following autologous peripheral blood stem cell transplant, please visit http://clinicaltrials.gov/ct2/show/NCT01121120?term=tarix&rank=2.

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.

“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).

About the Phase 2 Program
The double-blind, placebo-controlled multi-center Phase 2 study will evaluate the safety and efficacy of TXA127 in the acceleration of engraftment following autologous peripheral blood 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. For more information, please visit http://clinicaltrials.gov/ct2/show/NCT01121120?term=tarix&rank=2.

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.

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.

About the Phase II Stem Cell Engraftment Trial

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 reinfusion of CD34+ cells. The 74 patient, double-blind, placebo-controlled trial is being conducted at eight leading medical centers across the United States.

TXA127 will also be evaluated in two clinical trials in cord blood transplantation. One 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 grant was awarded under Project Bioshield Authority, legislation that enables NIAID within the Department of Health and Human Services (HHS) to use a rapid award process to help stimulate research on medical countermeasures against chemical, biological, radiological or nuclear (CBRN) threats. Under the initial grant, an appropriate dose was determined. The new grant will fund the completion of these studies in preparation for further government funding taking the product through to approval.

“We are very pleased with this vote of confidence, and look forward to continuing our close relationship with NIAID and other agencies to bring this product to completion”, said Dr Richard Franklin, CEO of Tarix Pharmaceuticals.

The principle investigator is Dr. Kathleen Rodgers, a Professor at Keck-USC School of Medicine.

The primary endpoint will be the time to platelet engraftment.

The principal endpoint of the trial will be the severity and incidence of thrombocytopenia. Click here for trial details.

The primary outcome measures include viral load and CD4 counts. Click here for trial details.

The grant was awarded under Project Bioshield Authority, legislation that enables NIAID within the Department of Health and Human Services (HHS) to use a rapid award process to help stimulate research on medical countermeasures against chemical, biological, radiological or nuclear (CBRN) threats. This grant will focus on the practical development of TXA127 as a therapeutic drug to reduce radiation-induced thrombocytopenia after untoward radiation exposure as may occur to a civilian population. The principle investigator is Dr. Kathleen Rodgers, a Professor at Keck-USC School of Medicine.

The principal endpoint of the trial will be the severity and incidence of thrombocytopenia. Click here for trial details.