BioLineRx announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead oncology candidate, BL-8040, for the treatment of pancreatic cancer.
"Orphan Drug Designation in pancreatic cancer is a very important milestone in the development plan of BL-8040, and joins previously approved orphan designations by the FDA for BL-8040 in AML and stem-cell mobilization," said Philip Serlin, Chief Executive Officer of BioLineRx. "Despite advances in the treatment of various cancers with immune checkpoint inhibitors, pancreatic cancer is refractory to these treatment options, and remains an area of significant unmet medical need. We have previously reported encouraging clinical data supporting the potential of BL-8040 as part of an immunotherapy combination treatment in pancreatic cancer, and we look forward to top-line results from our ongoing pancreatic clinical studies later this year."
BL-8040 is currently being investigated in clinical studies for the treatment of pancreatic cancer under two separate immuno-oncology collaborations – one with Merck and a second collaboration with Genentech, a member of the Roche Group.
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Orphan Drug Designation by the FDA entitles BioLineRx to seven years of market exclusivity for the use of BL-8040 for the treatment of pancreatic cancer, if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.
BL-8040 is a short synthetic peptide for the treatment of hematological malignancies, solid tumors, and stem cell mobilization. It functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in many human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells and immune-cells, sensitization of cancer cells to chemo- and bio-based anti-cancer therapies, and direct anti-cancer effect by inducing programmed cell death (apoptosis). BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.