Initial Results in for BioLineRx AML Trial

BioLineRx has posted positive initial results for BL-8040, a drug candidate in a Phase II clinical trial for patients with relapsed or refractory acute myeloid leukemia (AML). The early results show that BL-8040, as a stand-alone therapy and in combination with high-dose Cytarabine (Ara-C), is safe at all doses tested to date, and triggers substantial mobilization of cancer cells from the bone marrow to the peripheral blood, thereby increasing the vulnerability of the cells to chemotherapy treatment. In addition, signs of robust apoptosis (cell death) of cancer cells were observed following administration of the higher doses tested to date. The study has not yet reached the highest planned doses, suggesting that a strengthening of BL-8040’s effects may be observed in future dosing cohorts.

The Phase II trial is a multicenter, open-label study under an IND, and is designed to evaluate the safety and efficacy of repeated escalating doses of BL-8040 in adult patients with relapsed or refractory AML. The primary endpoints of the study are to assess the safety and tolerability of BL-8040. Secondary endpoints include the pharmacokinetic profile of the drug and an efficacy evaluation, indicated by the extent of mobilization of cancer cells from the bone marrow to the peripheral blood, the level of cancer cell death (apoptosis) and clinical responses.

Eight patients have already been enrolled in the study, out of a total expected enrollment of as many as 50 patients at eight clinical sites in the U.S. and Israel. The study is comprised of two parts, the current dose escalation phase and a subsequent expansion phase at the highest tolerated dose found during the escalation phase. During the dose escalation phase, trial participants are recruited in cohorts of three patients at a time, and the dose is increased for each subsequent cohort depending on the safety and tolerability results of the previous cohort. The BL-8040 dosing level of the current study cohort is 1 mg/kg, with the highest planned study dose being 1.5 mg/kg.

BL-8040 is a short peptide that functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in retention of cancer cells in the bone marrow, 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.