pipeline
 

Recombinant Human Erythropoietin (rHuEPO) – Multiple Myeloma

Recombinant Human Erythropoietin (“rHuEPO”), a known agent for anemia, is being developed to prolong the survival of patients with advanced Multiple Myeloma (“MM”). Our rHuEPO for the treatment of multiple myeloma blood cancer was granted an orphan drug designation from the FDA in 2011.

About Erythropoietin (EPO)

Erythropoietin (EPO) is a glycoprotein hormone produced mainly by the kidney. It is the major growth regulator of the erythroid lineage. EPO stimulates erythropoiesis, the production of red blood cells, by binding to its receptor (EPO-R) on the surface of erythroid progenitor cells, promoting their proliferation and differentiation and maintaining their viability.
Erythropoietin (EPO) is a glycoprotein hormone produced mainly by the kidney. It is the major growth regulator of the erythroid lineage. EPO stimulates erythropoiesis, the production of red blood cells, by binding to its receptor (EPO-R) on the surface of erythroid progenitor cells, promoting their proliferation and differentiation and maintaining their viability.

 

Preclinical and Clinical Information

Over the last decade, several reports have indicated that the action of EPO is not restricted to the erythroid compartment, but may have additional biological, and consequently potential therapeutic, properties, broadly beyond erythropoiesis.
A clinical observation made by Professor Moshe Mittelman and colleagues confirmed the high success rate of rHuEPO in treating the anemia in patients with MM. Six patients continued treatment with rHuEPO beyond the initial designed 12 week period with very poor prognostic features of MM, whose expected survival was less than 6 months, and surprisingly, they lived for 45–133 months cumulatively with the MM diagnosis and 38–94 months with rHuEPO (with a good quality of life).
This clinical observation was further supported by pre-clinical animal studies and extensive lab work. This not only confirmed the anti-myeloma effect of rHuEPO but also detected a new unrecognized hitherto immune-mediated effect to rHuEPO, probably mediated via T cells, and to some extent, also dendritic cells and macrophages.. EPO has been found to affect both the cellular as well as the humoral components of the immune system, probably associated with the improved survival of mice and human with MM. In mice the protein profile is also changed by rHuEPO. The effect on proteins is being investigated in humans as well.

 

About Multiple Myeloma (“MM”)

Currently incurable, MM is a severe plasma cell malignancy characterized by the accumulation and proliferation of clonal plasma cells in the marrow, leading to the gradual replacement of normal hematopoiesis. The course of the disease is progressive, and various complications occur, until death. This devastating disease affects the bone marrow, bones, kidneys, central nervous system and other vital organs.
It is characterized by bone pain with or without fractures, recurrent infections, anemia and additional complications. In the course of the disease, many patients become gradually disabled and bed-ridden. In the first months, after the diagnosis, 15% of the patients die. When no treatment is given, MM has a progressive course with a median survival of 6-10 months. The median overall survival duration today with chemotherapy and other novel treatments is approximately five years, with perhaps 20% of the patients living for more than ten years. These treatments cause severe side effects, including the suppression of the immune system, susceptibility to infections, nausea, vomiting and bleeding disorders.
It is characterized by bone pain with or without fractures, recurrent infections, anemia and additional complications. In the course of the disease, many patients become gradually disabled and bed-ridden. In the first months, after the diagnosis, 15% of the patients die. When no treatment is given, MM has a progressive course with a median survival of 6-10 months. The median overall survival duration today with chemotherapy and other novel treatments is approximately five years, with perhaps 20% of the patients living for more than ten years. These treatments cause severe side effects, including the suppression of the immune system, susceptibility to infections, nausea, vomiting and bleeding disorders.

XTL plans on performing a prospective, multi-center, double blind, placebo controlled phase 2 study intended to demonstrate the effects eHuEPO has on survival, biological markers related to the disease, immune improvements and quality of life. The Company has already begun the regulatory processes necessary to begin the phase 2 clinical trial.

List of EPO scientific publications (Part I) – Professor Moshe Mittelman

List of EPO scientific publications (Part II) – Others