BioLineRx receives Orphan Drug Designation for AML treatment
JERUSALEMóBioLineRx, a biopharmaceutical development company, announced today that the U.S. Food and Drug Administration (FDA) has granted an Orphan Drug Designation to BL-8040 as a therapeutic for the treatment of 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 United States. Orphan Drug Designation entitles the sponsor to a seven-year market exclusivity period, clinical protocol assistance with the FDA, as well as federal grants and tax credits.
"We are very pleased to have received Orphan Drug Designation for BL-8040, which will facilitate the development process of one of our key clinical-stage assets," stated Dr. Kinneret Savitsky, CEO of BioLineRx. "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 preclinical 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 II study expected towards the end of 2013."
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 (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 percent 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 18 multiple myeloma patients, BL-8040 demonstrated an excellent safety profile at all doses tested and was highly effective in the mobilization of hematopoietic stem cells and white blood cells from the bone marrow to the peripheral blood.
BL-8040 also mobilizes cancer cells from the bone marrow and may therefore sensitize these cells to chemo- and bio-based anti-cancer therapy. Importantly, BL-8040 has also demonstrated a direct anti-cancer effect by inducing apoptosis (cell death). Preclinical indicate 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 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.
SOURCE: BioLineRx news release