BioLineRx In-Licenses BL-8040 for the Treatment of AML

BioLineRx Ltd. has announced that it has signed an exclusive, worldwide license agreement with Biokine Therapeutics Ltd., a Clal Biotechnology Industries portfolio company, for the development and commercialization of BL-8040 (formerly BKT-140), a Phase II ready drug candidate for the treatment of acute myeloid leukemia (AML), as well as other types of hematological cancer.

BL-8040 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.

In a Phase I/II, open-label, dose escalation, safety and efficacy clinical trial in 16 multiple myeloma patients, BL-8040 demonstrated an excellent safety profile and was well tolerated at all doses tested.

On the basis of data obtained from this study, the FDA has approved an IND application.

BL-8040 has been shown to induce the mobilization of healthy hematopoietic stem cells from the bone marrow into the peripheral blood.

BL-8040 also mobilizes cancer cells from the bone marrow and other sites and may therefore expose these cells to chemo- and bio-based anti-cancer therapy and induce apoptosis (cell death).

Pre-clinical 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.

Dr. Kinneret Savitsky, CEO of BioLineRx, commented, "BioLineRx has made a strategic decision to enter the field of oncology, where there is clearly an urgent need for next generation anti-cancer therapies utilizing novel biological pathways. We are therefore extremely pleased to in-license this promising Phase II ready drug, which we will initially develop for the treatment of acute myeloid leukemia, a true unmet medical need with very low survival rates. AML is a recognized orphan indication both in the U.S. and the EU; therefore, we plan to seek orphan designation status from the regulatory authorities in order to accelerate its development plan. In addition, based on BL-8040’s promising pre-clinical data, as well its mechanism of action, we believe it can be utilized for several other related oncology indications and we intend to explore these possibilities as well. We look forward to the upcoming Phase II clinical study for evaluating BL-8040’s efficacy on AML patients, which is expected to commence in the first half of 2013."

"We are very happy that BioLineRx will further develop this promising anti-cancer agent, and are confident that BioLineRx’s experience and expertise will advance BL-8040 through the clinical development stages in the most efficient manner,” said Professor Amnon Peled, from the Gene Therapy Institute, Hadassah Medical Center - Jerusalem, founder and CEO of Biokine Therapeutics.

“CXCR4 is one of the most important cancer targets discovered in recent years. It is essential for multiple aspects of cancer progression in over 70% of all cancers, including leukemia, breast, lung, colon, and prostate cancer. BL-8040, as a CXCR4 antagonist, therefore has the potential to target and kill various cancer cells, and studies in animal models of the disease have shown that this agent may stimulate hematological cancer cell death. In addition, for many blood cancers, the bone marrow provides protection for malignant cells from chemotherapeutic agents. Therefore, by inducing mobilization of these cells into the peripheral blood, CXCR4 antagonists literally ‘flush out’ the malignant cells from their hiding places.

“We have demonstrated in pre-clinical studies that BL-8040 is very effective in mobilizing cells out of the bone marrow, thus sensitizing chemo-resistant cells and improving treatment with other anti-cancer drugs," concluded Professor Peled.