BioLineRx, Ltd., has announced the initiation of a Phase 2 trial of BL-8040 as a single agent to mobilize and collect bone marrow stem cells from peripheral blood circulation, which will be used to harvest hematopoietic stem cells to treat several hematological cancers, including lymphoma. The compound is currently in Phase 2 testing for the treatment of relapsed and refractory acute myeloid leukemia (AML) patients.
BL-8040 is a short peptide with high affinity antagonism for CXCR4, a receptor involved in tumor progression, metastasis, and cell survival. This chemokine receptor is overexpressed in more than 70 percent of human cancers, an expression that often correlates with disease severity. In a Phase 1/2 study assessing the drug’s efficacy and safety in multiple myeloma patients, BL-8040 demonstrated, in conjugation with G-CSF (Granulocyte-colony stimulating factor), a good safety profile at all doses tested. Moreover, the drug was efficient to mobilize hematopoietic stem cells and white blood cells from the bone marrow to peripheral blood.
Additionally, pre-clinical studies also showed that BL-8040 inhibited the growth of several tumors, including multiple myeloma, non-Hodgkin’s lymphoma, leukemia, non-small cell lung carcinoma, neuroblastoma and melanoma, preferentially stimulating the apoptotic death of malignant cells.
The new Phase 2 study, a collaboration with the Washington University School of Medicine, Division of Oncology and Hematology, will evaluate the drug’s ability to promote stem cell mobilization for transplantation purposes. The primary endpoints include, on the donor side, the ability of a single injection of BL-8040 to mobilize a sufficient amount of cells for transplantation and, on the recipient side, the functionality and engraftment success of the BL-8040 collected cells following transplantation. Safety, tolerability, incidence of grade 2–4 acute graft versus host disease (GvHD) in recipients, and graft durability will also be assessed.
The study will enroll up to 24 adult donor/recipient pairs.
“We have already completed a successful Phase 1 safety and efficacy study in healthy volunteers, supporting BL-8040 as one-day, single-dose collection regimen for rapidly mobilizing substantial amounts of stem cells. This represents a significant improvement upon the current standard of care. Since there are no approved drugs for stem cell mobilization to support allogeneic transplant, we are looking forward to the partial results expected by the end of 2016 and topline results expected by the end of 2017,” Dr. Kinneret Savitsky, chief executive officer of BioLineRx, said in a press release.