BioLineRx Says BL-8040 Gets FDA's Orphan Drug Designation For Treatment Of AML

(RTTNews) - BioLineRx Ltd. (BLRX), an Israel-based biopharmaceutical development company, Monday announced that the U.S. Food & Drug Administration or FDA has granted an Orphan Drug Designation to BL-8040 as a therapeutic for the treatment of acute myeloid leukemia or AML.

FDA grants Orphan Drug Designation to therapeutics intended to treat rare diseases that affect not more than 200 thousand people in the United States. It entitles the sponsor to a seven-year market exclusivity period, clinical protocol assistance with the FDA, as well as federal grants and tax credits.

BioLineRx noted that BL-8040 is a clinical-stage drug candidate for the treatment of acute myeloid leukemia, as well as other types of hematological cancers. 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, metastasis and cell survival.

The company said that BL-8040 demonstrated an excellent safety profile at all doses tested in a Phase 1/2, open-label, dose escalation, safety and efficacy clinical trial in 18 multiple myeloma patients, and was highly effective in the mobilization of hematopoietic stem cells and white blood cells from the bone marrow to the peripheral blood.

Kinneret Savitsky, Chief Executive Officer, said, "Currently, AML treatment options, especially in patients with a relapse of the disease, are extremely limited. BL-8040 has the potential to be a significant addition to the drug arsenal for this disease, especially when considering its promising pre-clinical results, unique biological mechanism and ability to synergize with other drugs already approved for this disease. Therefore, we are eagerly looking forward to the partial results of BL-8040's Phase 2 study expected towards the end of 2013."