Pluristem Released PLX-R18 Hematology Phase I Study Results; Data Show Increase in All Blood Lines and Reduction in Blood Transfusions Said PLX-R18 Reduced Mortality From 29% to 18% and Was Well-tolerated With a Favorable Safety Profile

Author: Charles Gross | March 23, 2022 07:32am
Pluristem Therapeutics Inc. (NASDAQ:PSTI) (TASE: PSTI), a leading biotechnology company developing novel cell therapies, today announced positive final results from its innovative hematology Phase I study to evaluate the safety and exploratory efficacy of intramuscular injections of PLX-R18 in subjects with incomplete hematopoietic recovery following HCT. Incomplete hematopoietic recovery, or poor graft function (PGF), is a life-threatening complication for patients undergoing HCT. Current standard-of-care treatments do not result in satisfactory blood counts in some or all blood cell lineages. Consequently, patients are vulnerable to bleeding andrecurrent infections, and require repeated costly transfusions of blood products, which only provide short-term benefits. Data collated 12 months post-treatment with PLX-R18 demonstrate that: PLX-R18 was well-tolerated with a favorable safety profile. Patients treated with PLX-R18 showed an increase in all three blood cell types compared to baseline with platelets (p<0.001), hemoglobin (p=0.01) and neutrophils (p=0.15) levels increasing as early as 1 month following PLX-R18 administration and enduring up to 12 months following treatment. Following PLX-R18 treatment, the number of transfused units decreased from a mean monthly number of 5.09 for platelets and 2.91 for red blood cells at baseline to 0.55 for platelets (p=0.045) and 0 for red blood cells (p=0.0005) at 12 months. The observed annual mortality rate following PLX-R18 administration was 18% compared to 29% in a cohort of allogeneic HCT recipients with incomplete hematopoietic recovery, obtained from the Center for International Blood and Marrow Transplant Research (CIBMTR) registry, representing a similar patient population.1 PLX-R18 cell therapy was previously granted an orphan drug designation by the FDA for the treatment of graft failure and incomplete hematopoietic recovery following HCT and for ARS. The final results of the Phase I study reinforce the preclinical results of studies conducted under the FDA's Animal Rule, in collaboration with the U.S. National Institutes of Health (NIH) and the U.S. Department of Defense (DOD), in which PLX-R18 was found to be effective in supporting the recovery of bone marrow failure resulting from ARS. In addition, the FDA previously cleared Pluristem's IND application for PLX-R18 in the treatment of ARS. The IND allows Pluristem to treat victims who may have been acutely exposed to high dose radiation due to a nuclear attack or accident. "PLX-R18 aims to improve the standard of care by stimulating the regenerative potential of the bone marrow, and we are gratified by results that I believe show we are on track to do so," said Pluristem Chief Executive Officer and President Yaky Yanay. "A quick and effective way to improve the hematological profile of sick patients—resulting in faster recovery and decreased transfusions—would be a game-changer for the field, and we will continue to explore its clinical applications. The FDA's recognition of PLX-R18's potential to treat ARS is also significant. This treatment potentially carries great significance for current events in Ukraine and Europe, providing hope for treatment in the case of a nuclear event. We are proud of this product line, which might hold the key to promoting wellbeing, improving the standard of care, and possibly saving lives."

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