Study collaboration with MD Anderson Cancer Center will assess
BL-8040 as combination treatment with immunosuppressants
BioLineRx
Ltd. (NASDAQ: BLRX; TASE: BLRX), a clinical-stage biopharmaceutical
company dedicated to identifying, in-licensing and developing promising
therapeutic candidates, announced today that it has filed the regulatory
submissions required to commence a Phase 2 trial for BL-8040, in
combination with standard-of-care immunosuppressive therapy, as a
novel treatment for two bone marrow failure conditions: hypoplastic
myelodysplastic syndrome (hMDS) and aplastic anemia (AA). The trial is
expected to commence shortly after receipt of regulatory approval,
anticipated in the next few months.
The open-label Phase 2 trial will be conducted in collaboration with MD
Anderson Cancer Center in Houston and is expected to enroll up to 25
patients. The study will examine BL-8040’s ability to improve bone
marrow cellularity and peripheral blood counts in patients suffering
from these bone marrow failure conditions.
Both hMDS and AA are characterized by a T cell-driven autoimmune attack
on the bone marrow that results in depletion of hematopoietic
precursors, leading to anemia and low white blood cell counts. In this
regard, high CXCR4 expression on pathogenic T cells has been suggested
to facilitate infiltration of the bone marrow. BL-8040, a CXCR4
antagonist, is expected to inhibit migration of pathogenic T cells to
the bone marrow, thereby mitigating the severe depletion of
hematopoietic stem and progenitor cells.
In addition, BL-8040 can directly affect the number of hematopoietic
precursors. Preclinical studies in mice showed that multiple doses of
BL-8040 led to a marked increase in the number of hematopoietic
progenitor cells and hematopoietic stem cells in both the bone marrow
and peripheral blood. BL-8040 also promoted production of megakaryocytes
in the bone marrow, leading to a prolonged increased platelet
production. These direct effects of BL-8040, along with the exclusion of
the pathogenic T cells from the bone marrow, may improve bone marrow
cellularity and peripheral blood counts.
Dr. Kinneret Savitsky, CEO of BioLineRx, stated, “We are very pleased to
have filed the necessary regulatory submissions for an additional Phase
2 trial of BL-8040, our unique oncology platform, in these novel and
non-malignant indications. We are honored to collaborate yet again with
the prestigious MD Anderson Cancer Center, expanding upon our current
collaboration on the Phase 2 trial for treating relapsed/refractory
acute myeloid leukemia (AML), as well as our planned Phase 2 trial for
treating AML patients with the FLT3-ITD mutation. The hMDS/AA trial will
assess BL-8040 in combination with standard of care immunosuppressive
therapy, with interim results expected by the end of 2016. Both hMDS and
AA represent significant unmet medical needs and we are very hopeful
that BL-8040, as part of a novel treatment regimen, will significantly
improve bone marrow cellularity and peripheral blood counts in patients
suffering from these difficult bone marrow failure conditions.”
About BL-8040
BL-8040 is a clinical-stage drug candidate for
the treatment of acute myeloid leukemia, as well as other hematological
indications. 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% of
human cancers and its expression often correlates with disease severity.
In a Phase 1/2, open-label, dose escalation, safety and efficacy
clinical trial in 18 multiple myeloma patients, BL-8040, when combined
with G-CSF, 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.
Additionally, in a Phase 1 stem-cell mobilization study in healthy
volunteers, BL-8040 as a single agent was safe and well tolerated at all
doses tested and resulted in efficient stem-cell mobilization and
collection in all study participants. Importantly, the results of this
study support the use of BL-8040 as one-day, single-dose collection
regimen, which is a significant improvement upon the current standard of
care.
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. Pre-clinical studies show that BL-8040
inhibits the growth of various tumor types including multiple myeloma,
non-Hodgkin's lymphoma, leukemia, non-small cell lung carcinoma,
neuroblastoma and melanoma. BL-8040 also significantly and
preferentially stimulated apoptotic cell death of malignant cells
(multiple myeloma, non-Hodgkin's lymphoma and leukemia). Significant
synergistic and/or additive tumor cell killing activity has been
observed in-vitro and in-vivo when tumor cells were treated with BL-8040
together with Rituximab, Bortezomib, Imatinib, Cytarabine and the FLT-3
inhibitor AC-220 (in NHL, MM, CML, AML, and AML-FLT3-ITD models,
respectively). In addition, the current Phase 2 clinical trial in AML
patients has demonstrated robust mobilization and apoptosis of cancer
cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and
was previously developed under the name BKT-140.
About AA and hMDS
Aplastic anemia (AA) and hypoplastic
myelodysplastic syndrome (hMDS) are hematological conditions caused by
progressive bone marrow failure, and characterized by ineffective
production of all blood cells, leading to severe anemia and cytopenias
(low blood counts). AA and hMDS result from disorders of the
hematopoietic stem cells in the bone marrow. Hematopoiesis is disrupted
and the number and quality of blood-forming cells decline irreversibly,
further impairing blood production. Treatment may include
immunosuppressive therapy, chemotherapy or hematopoietic stem cell
transplant.
About BioLineRx
BioLineRx is a publicly-traded,
clinical-stage biopharmaceutical company dedicated to identifying,
in-licensing and developing promising therapeutic candidates. The
Company in-licenses novel compounds primarily from academic institutions
and biotech companies based in Israel, develops them through
pre-clinical and/or clinical stages, and then partners with
pharmaceutical companies for advanced clinical development and/or
commercialization.
BioLineRx’s current portfolio consists of a variety of clinical and
pre-clinical projects, including: BL-8040, a cancer therapy platform,
which is in the midst of a Phase 2 study for relapsed/refractory acute
myeloid leukemia (AML), has recently initiated a Phase 2b study as an
AML consolidation treatment, and has successfully completed a Phase 1
study in stem cell mobilization; BL-7010 for celiac disease, which has
successfully completed a Phase 1/2 study; and BL-1040 for prevention of
pathological cardiac remodeling following a myocardial infarction, which
has been out-licensed to Bellerophon BCM (f/k/a Ikaria).
In December 2014, BioLineRx entered into a strategic collaboration
with Novartis for the co-development of selected Israeli-sourced novel
drug candidates. The companies intend to co-develop a number of
pre-clinical and early clinical therapeutic projects through clinical
proof-of-concept for potential future licensing by Novartis.
For more information on BioLineRx, please visit www.biolinerx.com
or download the investor relations mobile device app, which allows users
access to the Company’s SEC documents, press releases, and events.
BioLineRx’s IR app is available on the iTunes App Store as well as the
Google Play Store.
Various statements in this release concerning BioLineRx’s future
expectations, including specifically those related to the development
and commercialization of BL-8040, constitute “forward-looking
statements” within the meaning of the Private Securities Litigation
Reform Act of 1995. These statements include words such as “may,”
“expects,” “anticipates,” “believes,” and “intends,” and describe
opinions about future events. These forward-looking statements involve
known and unknown risks and uncertainties that may cause the actual
results, performance or achievements of BioLineRx to be materially
different from any future results, performance or achievements expressed
or implied by such forward-looking statements. Some of these risks are:
changes in relationships with collaborators; the impact of competitive
products and technological changes; risks relating to the development of
new products; and the ability to implement technological improvements.
These and other factors are more fully discussed in the “Risk Factors”
section of BioLineRx’s most recent annual report on Form 20-F filed with
the Securities and Exchange Commission on March 23, 2015. In addition,
any forward-looking statements represent BioLineRx’s views only as of
the date of this release and should not be relied upon as representing
its views as of any subsequent date. BioLineRx does not assume any
obligation to update any forward-looking statements unless required by
law.
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