BioLineRx Ltd. has announced dosing of the first patient in a Phase 1 trial for a second indication of its BL-8040 cancer therapy platform, as a novel treatment for the mobilization of stem cells from the bone marrow to the peripheral blood circulation, where they can be harvested for transplant supporting the treatment of hematological indications.
“Stem cell mobilization is used more and more as a method of collecting hematopoietic stem cells for transplantation, which is used to treat certain types of cancer such as myeloma, leukemia or lymphoma, as well as for the treatment of severe anemia or immune deficiency disorders. Traditionally, these cells were harvested from the bone marrow using a surgical procedure, which involves general anesthesia. Current treatment regimens involve daily injections for 4-6 days of G-CSF, with or without the addition of a mobilizing agent such as Plerixafor (Mozobil). BL-8040 given as a single injection was previously shown in a Phase 1/2 study in multiple myeloma patients to be highly effective in mobilizing stem cells in combination with G-CSF injections. Although it is a Phase 1 study, the current trial will provide us with efficacy data regarding the mobilization capacity of one or two injections of BL-8040 as a stand-alone therapy, which could significantly shorten and reduce the cost of treatment, as well as eliminate the painful side effects associated with G-CSF,” explained Dr. Kinneret Savitsky, Chief Executive Officer of BioLineRx.
“In addition, BL-8040 is currently in the midst of the dose-escalation part of a Phase 2 trial for the treatment of acute myeloid leukemia, which is being conducted at several world-leading cancer research centers in the US and Israel, with results expected in early 2015. We are very excited at this point to continue the clinical development of an additional indication in our BL-8040 cancer therapy platform, and we look forward to providing updates on these important clinical milestones over the next several months,” concluded Dr. Savitsky.
The stem cell mobilization Phase 1 study consists of two parts. Part 1 is a randomized, double-blind, placebo-controlled dose escalation study exploring the safety and tolerability of escalating repeated doses of BL-8040 in healthy volunteers.
Secondary objectives include assessment of the efficacy of BL-8040 in mobilizing stem cells as a stand-alone therapy, as well as determining the pharmacokinetic profile of the drug. This part will be performed in up to 4 cohorts, with 8 healthy volunteers in each cohort. Part 1 of the study will serve to select the optimal safe and efficacious dose of BL-8040 to be used as a stand-alone therapy in Part 2 of the study.
Part 2 is an open-label study designed to assess BL-8040’s stem cell mobilization capacity, as well as the yield of cells collected by apheresis. Secondary endpoints of the study include evaluation of the viability and biological activity of cells mobilized by BL-8040 and collected by apheresis. This part will be performed in a single cohort of 8 healthy volunteers who will receive the selected dose regimen of BL-8040 based on the data from Part 1.