BioLineRx, a clinical-stage biopharmaceutical company dedicated to identifying, in-licensing and developing promising therapeutic candidates, has received notice from the FDA confirming an Orphan Drug Designation of BL-8040 as a treatment for stem cell mobilization, in addition to the Orphan Drug Designation previously granted to BL-8040 as a treatment for Acute Myeloid Leukemia (AML).

Orphan Drug Designation is granted to therapeutics intended to treat rare diseases that affect not more than 200,000 people in the U.S. Orphan Drug Designation entitles the sponsor to a seven-year marketing exclusivity period, clinical protocol assistance with the FDA, as well as federal grants and tax credits.

The Orphan Drug Designation was granted for use of BL-8040, in combination with granulocyte colony-stimulating factor (G-CSF), to mobilize human stem cells from the bone marrow to the peripheral blood for collection for autologous or allogeneic (donor-based) transplantation.

High-dose chemotherapy followed by stem cell transplantation has become an established treatment modality for a variety of hematologic malignancies, including multiple myeloma, as well as various forms of lymphoma and leukemia. Stem cells are mobilized from the bone marrow using granulocyte colony-stimulating factor (G-CSF), harvested from the peripheral blood by apheresis, and infused to the patient after chemotherapy. This type of treatment often replaces the use of traditional bone marrow transplantation, because the stem cells are easier to collect and the treatment allows for a quicker recovery time and fewer complications.

BL-8040 is a clinical-stage drug candidate for the treatment of acute myeloid leukemia, as well as other hematological indications. It is a short peptide that functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis (growth of new blood vessels in the tumor), metastasis (spread of the disease to other organs or organ parts) and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. BL-8040 mobilizes cancer cells from the bone marrow and may therefore sensitize these cells to chemo- and bio-based anti-cancer therapy. Importantly, BL-8040 also has demonstrated a direct anti-cancer effect by inducing apoptosis (cell death). Preclinical studies show that BL-8040 is efficient, both alone and in combination with the anti-cancer drug Rituximab, in reducing bone marrow metastasis of lymphoma cells and stimulating lymphoma cell death.

BL-8040 also mobilizes stem cells from the bone marrow to the peripheral blood, enabling their collection for subsequent autologous or allogeneic transplantation in cancer patients. In a Phase 1/2, open-label, dose escalation, safety and efficacy clinical trial in 18 multiple myeloma patients, BL-8040 demonstrated an excellent safety profile at all doses tested and was highly effective in combination with G-CSF in the mobilization of hematopoietic stem cells and white blood cells from the bone marrow to the peripheral blood. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

Dr. Kinneret Savitsky, CEO of BioLineRx, said, "This is in line with our 2014 development plan for BL-8040, which includes clinical studies for both AML and stem cell mobilization. BL-8040 already was shown to be highly effective in mobilizing stem cells in combination with G-CSF in a phase I/II study in multiple myeloma patients, and initial results of BL-8040's phase II study for AML patients indicate that BL-8040 on a stand-alone basis triggers substantial mobilization of cancer cells from the bone marrow to the peripheral blood. We are eagerly looking forward to the results of the clinical trials in both indications expected in the second half of this year."