Bioline RX Ltd (NASDAQ:BLRX) announced positive top-line results from BL-8040’s Phase 2 clinical trial in relapsed or refractory acute myeloid leukemia (r/r AML). Detailed results are planned to be presented at an upcoming scientific conference.

The BL-8040 oncology platform is a short cyclic peptide that functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. Results of the Phase 2 clinical trial showed that BL-8040, as a single agent and in combination with Cytarabine (Ara-C), was safe and well tolerated at all doses tested up to and including the highest dose level of 2.0 mg/kg, with no major adverse events (n=45). The composite complete remission rate, including both complete remission (CR) and complete remission with incomplete blood count recovery (CRi), was 38% in subjects receiving up to two cycles of BL-8040 treatment at doses of 1 mg/kg and higher (n=39). Patients included in the study were patients that had undergone a significant number of prior treatments or that were refractory to induction treatment. The data include three compassionate-use patients treated at the study sites under the identical treatment protocol.

Importantly, the data suggest, for the first time, a correlation between improved clinical response and patients with a high disease burden in the bone marrow, along with a lower peripheral circulation of AML blasts at baseline (indicative of potential CXCR4 disease dependency). This finding may serve as a biomarker for patient selection in future BL-8040 AML studies.

In addition, treatment with BL-8040 continues to show a triple effect on the leukemic cells. First, BL-8040 monotherapy triggered robust mobilization of AML cells from the bone marrow to the peripheral blood, thereby sensitizing these cells to the Ara-C chemotherapy and improving its efficacy. Second, BL-8040 monotherapy showed a direct apoptotic effect on the leukemia cells in the bone marrow. Last, BL-8040 induced leukemia progenitor cells towards differentiation, as evidenced by a decrease in the number of leukemia progenitor cells, along with a three-fold increase in differentiated granulocytes, in the bone marrow biopsy conducted on day 3 of the treatment cycle prior to the Ara-C treatment, as compared to the biopsy performed at baseline.

Dr. Jorge Cortes, Chief of the AML and CML Sections at the MD Anderson Cancer Center inHouston, stated, “The outlook for relapsed or refractory AML patients remains poor. I am therefore very encouraged by the response rate demonstrated using the combination of BL-8040 with Cytarabine in such patients, which is backed by a sound scientific rationale. The response rate is especially compelling for the identified sub-population. These impressive clinical results from the Phase 2 study support further development of the compound in the AML space.”

Dr. Kinneret Savitsky, CEO of BioLineRx, commented, “We are very enthusiastic about the positive results from the Phase 2 trial with BL-8040 for the treatment of relapsed or refractory AML. We are especially excited that for the first time, we see a direct correlation between clinical response and a specific subset of the study patient population. Given that AML is a heterogeneous disease, the ability to pre-define the population that may benefit from CXCR4 inhibition is very important for future development.”

“The results continue to demonstrate that BL-8040 not only significantly induces mobilization of leukemic cells from the protective microenvironment of the bone marrow into the peripheral blood, but also directly leads to apoptosis of leukemic progenitor cells and triggers terminal differentiation of the cells into granulocytes. Combined with the impressive remission rate reported from subjects receiving BL-8040 doses of 1 mg/kg or higher, the results strongly suggest that BL-8040 has potent anti-leukemic activity and, in combination with Cytarabine, may improve the response typically achieved in this advanced AML population. These successful results also reinforce our excitement about BL-8040’s overall potential in the AML space, including as an AML consolidation treatment that is currently being investigated in a large Phase 2b study at approximately 25 sites in Germany. Given these positive results, we now plan to meet with the regulatory authorities to discuss the next steps in the development of this promising program.”

“In order to further expand and enhance the potential of this unique oncology platform, we are continuing to perform and plan multiple additional clinical studies for BL-8040, including our recently announced immuno-oncology collaboration with Merck on a Phase 2 study to investigate BL-8040 in combination with KEYTRUDA® for the treatment of pancreatic cancer,” concluded Dr. Savitsky. (Original Source)

Shares of Biolinerx are up nearly 3% in pre-market trading. BLRX has a 1-year high of $2.80 and a 1-year low of $0.85. The stock’s 50-day moving average is $1.04 and its 200-day moving average is $1.27.

On the ratings front, Roth Capital analyst Joseph Pantginis reiterated a Buy rating on BLRX, with a price target of $7, in a report issued on March 21. The current price target implies an upside of 493.2% from current levels. According to, Pantginis has a total average return of -6.4%, a 33.3% success rate, and is ranked #3691 out of 3749 analysts.

Bioline Rx Ltd is a clinical stage biopharmaceutical development company engaged in identifying, in-licensing & developing therapeutic compounds.