On June 18, 2018 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported that new data presented at the 23rd Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper), held in Stockholm, Sweden, shows that BL-8040, combined with high dose cytarabine (HiDAC), significantly enhanced overall survival in difficult-to-treat relapsed or refractory AML (r/r AML) patients in a Phase 2a clinical trial (Press release, BioLineRx, JUN 18, 2018, View Source;p=RssLanding&cat=news&id=2354886 [SID1234527372]). In addition, an important new finding shows a statistically significant correlation between patient response and the mobilization of AML blasts. Responding patients demonstrated a clear and significant increase in the number of AML blasts in the peripheral blood following BL-8040 treatment, whereas non-responding patients were largely unaffected.

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"We are extremely pleased to see further significant improvement in overall survival for this very difficult-to-treat patient population, as data continues to accumulate from our Phase 2a proof-of-concept study in relapsed or refractory AML," stated Philip A. Serlin, Chief Executive Officer of BioLineRx. "In addition, exciting new findings indicate a clear correlation between patient response and mobilization of AML blasts, thus identifying a potential biomarker for selecting patients likely to respond to BL-8040. These encouraging results strongly support the continued development of BL-8040 in relapsed or refractory AML, giving BioLineRx broad therapeutic coverage in the AML space, with potential activity at different stages of the disease and in different patient populations. We look forward to providing additional updates on overall survival from this study, and continue to execute on our other two important AML trials currently ongoing – a large, randomized, controlled Phase 2b study in consolidation AML, and a Phase 1b/2 study in maintenance of AML under our collaboration with Genentech," added Mr. Serlin.

The Phase 2a study consisted of 42 patients in two cohorts: (i) dose-escalation (range 0.5-2.0 mg/kg) and (ii) dose-expansion at the selected dose of 1.5 mg/kg. Patients with r/r AML were treated daily with BL-8040 monotherapy for two days followed by combined administration of BL-8040 and HiDAC for 5 days, for 1-2 cycles. Efficacy endpoints included response rate (CR/CRi), overall survival, duration of response and event-free survival.

BL-8040 in combination with HiDAC was safe and well tolerated at all BL-8040 dose levels (range 0.5-2.0 mg/kg). The response rate for all dosing levels was 29% and median overall survival was 9.1 months, compared with historical data on overall survival of 6.1 months for HiDAC alone. In patients receiving the 1.5 mg/kg dose selected for expansion (n=23), the response rate was 39% and median overall survival was 10.7 months with 1-year, 2-year and 3-year survival rates of 38.1%, 23.8% and 23.8%, respectively. Furthermore, median overall survival for responding patients at the 1.5 mg/kg dose (n=9) was 21.8 months, with 1-year, 2-year and 3-year survival rates of 66.7%, 44.4% and 44.4%, respectively. Responding patients also demonstrated a statistically significant mean 6.3-fold increase (p=0.003) in the number of AML blasts in the peripheral blood following BL-8040 monotherapy treatment, whereas in non-responding patients the mean-fold increase was minor and non-significant (1.66-fold; p=0.21).

About BL-8040

BL-8040 is a short peptide for the treatment of acute myeloid leukemia, solid tumors, and stem cell mobilization. It functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of 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 from the bone marrow, thereby sensitizing cancer cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing cell death (apoptosis). In addition, BL-8040 has also demonstrated robust mobilization of other cell types, including the mobilization of hematopoietic stem and progenitor cells, T, B, NK and antigen presenting cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.