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

 

"We are very pleased to have received Orphan Drug Designation forBL-8040, which will facilitate the development process of one of our keyclinical-stage assets," stated Dr. Kinneret Savitsky, CEO of BioLineRx. "Currently, AML treatmentoptions, especially in patients with a relapse of the disease, areextremely limited. BL-8040 has the potential to be a significantaddition to the drug arsenal for this disease, especially whenconsidering its promising preclinical results, unique biologicalmechanism and ability to synergize with other drugs already approved forthis disease. Therefore, we are eagerly looking forward to the partialresults of BL-8040's Phase II study expected towards the end of 2013."

BL-8040 is a clinical-stage drug candidatefor the treatment of acute myeloid leukemia, as well as other types ofhematological cancers. It is a short peptide that functions as ahigh-affinity antagonist for CXCR4, a chemokine receptor that isdirectly involved in tumor progression, angiogenesis (growth of newblood vessels in the tumor), metastasis (spread of the disease to otherorgans or organ parts) and cell survival. CXCR4 is over-expressed inmore than 70 percent of human cancers and its expression often correlates withdisease severity. In a Phase I/II, open-label, dose escalation, safetyand efficacy clinical trial in 18 multiple myeloma patients, BL-8040demonstrated an excellent safety profile at all doses tested and washighly effective in the mobilization of hematopoietic stem cells andwhite blood cells from the bone marrow to the peripheral blood.

 

BL-8040 also mobilizes cancer cells from the bone marrow and maytherefore sensitize these cells to chemo- and bio-based anti-cancertherapy. Importantly, BL-8040 has also demonstrated a direct anti-cancereffect by inducing apoptosis (cell death). Preclinical indicatethat BL-8040 is efficient, both alone and in combination with theanti-cancer drug Rituximab, in reducing bone marrow metastasis oflymphoma cells and stimulating lymphoma cell death. BL-8040 was licensedby BioLineRx from Biokine Therapeutics and was previously developedunder the name BKT-140.