BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage
biopharmaceutical company dedicated to identifying, in-licensing and
developing promising therapeutic candidates, announced today successful
top-line results from the Phase 1 safety and efficacy study of its lead
clinical candidate, BL-8040, as a novel approach for mobilization and
collection of bone-marrow stem cells from the peripheral blood
circulation. All safety and efficacy endpoints were met, showing that
treatment with BL-8040 as a single agent was safe and well tolerated at
all doses and resulted in efficient stem cell mobilization and
collection in all study participants. Importantly, the results support
BL-8040 as one-day, single-dose collection regimen, which is a
significant improvement upon the current standard of care.
Robust stem cell mobilization was evident in all treated participants,
across the different doses tested, supporting a novel approach to stem
cell collection. After a single administration, BL-8040 enabled
collection of a yield of stem cells that exceeds the number required to
support a transplant in all treated participants, following only one
collection procedure. The Company intends to present the full set of
study results at the European Hematology Association (EHA) Conference
taking place in Vienna in June 2015.
Dr. John DiPersio, Chief of the Division of Oncology at Washington
University School of Medicine in St. Louis, stated, “I am very impressed
and encouraged by the activity of BL-8040 in promoting stem cell
mobilization and collection as a single agent. The robust effect seen in
all participants substantially differentiates this compound from the
currently approved mobilization regimens, which require four to five
days of treatment and one to three full-day apheresis sessions, and can
be associated with side effects including bone pain. Therefore, a novel
agent with the capacity to rapidly mobilize substantial amounts of stem
cells, while providing a shorter and better tolerated drug
administration and cell collection regimen, will be of great value both
medically and in terms of patient comfort.”
“We are very enthusiastic about the study results showing the
outstanding activity of BL-8040 in promoting stem cell mobilization. The
results exceeded our expectations, and validate BL-8040 as a highly
differentiated stand-alone treatment for stem-cell collection. We plan
to meet with the FDA as soon as practicable, in order to discuss the
results of this study and obtain more clarity on the next steps in the
clinical development program for this indication,” commented Dr.
Kinneret Savitsky, CEO of BioLineRx. “In addition to stem-cell
mobilization, our BL-8040 platform is also undergoing a Phase 2 study
for treating relapsed and refractory acute myeloid leukemia patients,
results of which are expected in the second half of 2015. In addition,
as recently reported, we expect to commence clinical trials for three
additional indications for BL-8040 in the second quarter of 2015.”
The Phase 1 safety and efficacy study consisted of two parts. The first
part was a randomized, double-blind, placebo-controlled, dose escalation
study exploring the safety and tolerability of escalating repeated doses
of BL-8040 in three cohorts of eight healthy volunteers. Based on data
from the first part, an optimal safe and efficacious dose of BL-8040 was
selected, which was used as a stand-alone therapy in a single cohort of
eight healthy volunteers in the second open-label part of the study.
This part of the study was designed to assess BL-8040’s stem cell
mobilization capacity, as well as the yield of cells collected by
apheresis. Secondary efficacy endpoints of the study included the
pharmacodynamic and pharmacokinetic profile of the drug, as well as an
evaluation of the viability and biological activity of cells mobilized
by BL-8040 and collected by apheresis.
About Stem Cell Mobilization
High-dose chemotherapy followed by stem cell transplantation has become
an established treatment modality for a variety of hematologic
malignancies, including multiple myeloma, as well as various forms of
lymphoma and leukemia. Modern peripheral stem-cell harvesting often
replaces the use of traditional surgical bone marrow stem-cell
harvesting. In the modern method, stem cells are mobilized from the bone
marrow using granulocyte colony-stimulating factor (G-CSF), often with
the addition of a mobilizing agent such as Plerixafor (Mozobil),
harvested from the donor’s peripheral blood by apheresis, and infused to
the patient after chemotherapy ablation treatment. This treatment is
highly effective, the peripheral stem cells are easier to collect, and
the treatment allows for a quicker recovery time and fewer complications.
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.
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 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 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-1040 for prevention of pathological
cardiac remodeling following a myocardial infarction, which has been
out-licensed to Bellerophon BCM (f/k/a Ikaria) and is in the midst of a
pivotal CE-Mark registration trial scheduled for completion in mid-2015;
BL-8040, a cancer therapy platform, which is in the midst of a Phase 2
study for acute myeloid leukemia (AML), and has just completed a Phase 1
study in stem cell mobilization; and BL-7010 for celiac disease, which
has successfully completed a Phase 1/2 study.
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.
Copyright Business Wire 2015