Pluristem Therapeutics Recaps Key Opinion Leader Call Reviewing Hematological Programs
17 September 2019 - 2:20AM
Pluristem Therapeutics Inc.
(Nasdaq:PSTI)
(TASE:PSTI), a leading regenerative medicine company
developing novel placenta-based cell therapy products, today
provided a recap of the Key Opinion Leader call hosted by the
company this morning to review unmet medical needs in acute
radiation syndrome (ARS) and hematological deficiencies, and to
provide an update on the development of its novel cell therapy
treatment, PLX-R18. PLX cell therapy was granted an Orphan Drug
Designation by the U.S. Food and Drug Administration (FDA) for the
treatment of ARS and incomplete hematopoietic recovery following
hematopoietic cell transplantation (HCT).
An overview of HCT was provided by Dr. Jacob M.
Rowe, MD, Chief of the Department of Hematology and The Ann and
Pinky Sohn Chair in Hemato-Oncology at the Shaare Zedek Medical
Center in Jerusalem and Emeritus Professor at the Technion, Israel
Institute of Technology in Haifa, a member in the company’s ongoing
hematology program steering committee. Dr. Rowe reviewed the
medical condition of HCT and the unmet need for the significant
proportion of patients that do not respond to current treatments.
Dr. Rowe then discussed Pluristem’s ongoing Phase I study of
PLX-R18 and data from the first two cohorts:
- The study is comprised of 24 subjects in the U.S. and Israel.
Cohort 3 (4 million cells/kg) is currently enrolling
- Data from 2 first cohorts, after a follow-up period of 6
months, have shown a median increase of platelets in cohort 1 (1
million cells/kg) and cohort 2 (2 million cells/kg) of 130% and
170%, respectively
- Median increase in hemoglobin levels in cohort 1 and cohort 2
was 34% and 36%, respectively
- A 65% reduction in platelet transfusion, and a 68% reduction in
red blood cell transfusion, were observed compared to the last
month before treatment in both cohorts
- A modest increase in neutrophil levels was seen in both
cohorts
“Results from the first two cohorts in this
study demonstrate that PLX-R18 is safe and well tolerated, with no
unexpected toxicities,” said Dr. Rowe. “While the study’s primary
endpoint was safety, we can’t disregard the potential benefits of
this cell therapy drug, which even in the lower doses appears to
regenerate damaged bone marrow, leading to increased production of
platelets and red blood cells, ultimately reducing the number of
required transfusions, although caution is appropriate in
discussing early data with a small number of patients. Importantly,
we see these positive benefits sustained at the nine months
follow-up period, and we continue to monitor these patients. We
look forward to results from Cohort 3, in which patients will be
administered four million cells per kilogram, as we work to
efficiently advance PLX-R18 as a candidate in additional bone
marrow deficiency syndromes with similar unmet needs.”
The next presentation was delivered by Bert W.
Maidment, Ph.D., who served as Assistant Director, Radiation
Nuclear Countermeasures Program at the National Institutes of
Health, National Institute of Allergy and Infectious Diseases,
Division of Allergy, Immunology, and Transplantation
(NIH/NIAID/DAIT) until retiring in 2016. Dr. Maidment discussed the
health impact of radiation exposure and the need for more effective
countermeasures. “There is a broad range of devastating health
consequences resulting from radiation exposure, with hematopoietic,
gastrointestinal, cardiovascular and central nervous systems all
potentially impacted,” noted Dr. Maidment. “Notwithstanding several
FDA-approved countermeasures, including Neupogen®, Neulasta® and
Leukine®, significant treatment gaps remain, causing widespread
manifestations across vital organ systems. I believe that a more
efficacious countermeasure that can be administered pre- and
post-exposure, particularly one that is easily stored and
administered and potentially support multiple organ injuries, would
have significant utility across a number of civilian and military
applications where radiation exposure is a potential risk.”
Pluristem management provided an overview of the
company’s acute radiation syndrome (ARS) projects that are running
with several governments and agencies, including the U.S. National
Institutes of Health (NIH), testing PLX-R18 as a potential
treatment for ARS, the U.S. Department of Defense (DOD) testing the
product as a potential prophylactic countermeasure against ARS and
Fukushima University in Japan examining PLX-R18 cells for the
treatment of hematological ARS and gastrointestinal (GI). Arik
Eisenkraft, Director of Homeland Defense Projects at Pluristem and
former Head of Chemical, Biological, Radiological and Nuclear
Defense (CBRN) Protection Division in the Israeli Ministry of
Defense, summarized PLX-R18’s potential to stimulate the
regeneration of damaged bone marrow to produce blood cells while
also increasing survival and benefiting additional body systems,
suggesting that PLX-R18 has the ability to treat ARS as a
multi-organ therapy.
Yaky Yanay, Chief Executive Officer of
Pluristem, concluded the call with an update on the status of
current projects and provided an overview of next steps. “I would
like to thank Drs. Rowe and Maidment for offering their
perspectives on these difficult-to-treat hematological conditions.
We look forward to advancing our ARS program to complete the
development and registration of PLX-R18 for ARS, targeting
governmental purchase contracts.
- After reaching an understanding with the FDA on the regulatory
pathway needed and conducting additional mechanism studies with the
support of the NIH, we have progressed our discussions with the
Biomedical Advanced Research and Development Authority (BARDA) to
submit a proposal by the end of September, aiming for a study,
strategically designed to demonstrate the superiority of PLX-R18
versus current standards of care, with the goal of executing a full
contract once the study is completed.
- Following the studies conducted by the U.S. Department of
Defense, we are now progressing to further small and large animal
studies in relevant DoD models, as required by the FDA animal rule
pathway.
We remain committed to completing our ongoing
Phase I study of PLX-R18 in incomplete hematopoietic recovery
following HCT as quickly as possible and will seek to expand
PLX-R18 for use with respect to additional medical conditions with
the goal of bringing innovative, safe and effective treatments for
patients with a variety of bone marrow deficiency indications.”
About Pluristem
TherapeuticsPluristem Therapeutics Inc. is a leading
regenerative medicine company developing novel placenta-based cell
therapy product candidates. The Company has reported robust
clinical trial data in multiple indications for its patented PLX
cell product candidates and is currently conducting late stage
clinical trials in several indications. PLX cell product candidates
are believed to release a range of therapeutic proteins in response
to inflammation, ischemia, muscle trauma, hematological disorders
and radiation damage. The cells are grown using the Company's
proprietary three-dimensional expansion technology and can be
administered to patients off-the-shelf, without tissue matching.
Pluristem has a strong intellectual property position; a
Company-owned and operated GMP-certified manufacturing and research
facility; strategic relationships with major research institutions;
and a seasoned management team.
Safe Harbor StatementThis press release
contains express or implied forward-looking statements within the
Private Securities Litigation Reform Act of 1995 and other U.S.
Federal securities laws. For example, Pluristem is using
forward-looking statements when it discusses the potential benefits
of PLX-R18 as identified in the study, the potential use of PLX-R18
in civilian and military applications where radiation exposure is a
potential risk, that PLX-R18 has the ability to treat ARS as a
multi-organ therapy, the advancement of its ARS program to
ultimately target governmental purchase contracts, its
progress with respect to small and large animal studies in relevant
DoD models, as required by the FDA animal rule pathway, its aim to
submit a proposal by the end of September with respect to BARDA,
with the hopes of commencing a study designed to demonstrate the
superiority of PLX-R18 in the treatment of ARS versus current
standards of care and with the goal of executing a full contract
once the study is completed and its aim to expand the focus of
PLX-R18 to additional medical conditions with the goal of bringing
innovative, safe and effective treatments for patients with a
variety of bone marrow deficiency indications. These
forward-looking statements and their implications are based on the
current expectations of the management of Pluristem only, and are
subject to a number of factors and uncertainties that could cause
actual results to differ materially from those described in the
forward-looking statements. The following factors, among others,
could cause actual results to differ materially from those
described in the forward-looking statements: changes in technology
and market requirements; Pluristem may encounter delays or
obstacles in launching and/or successfully completing its clinical
trials; Pluristem’s products may not be approved by regulatory
agencies, Pluristem’s technology may not be validated as it
progresses further and its methods may not be accepted by the
scientific community; Pluristem may be unable to retain or attract
key employees whose knowledge is essential to the development of
its products; unforeseen scientific difficulties may develop with
Pluristem’s process; Pluristem’s products may wind up being more
expensive than it anticipates; results in the laboratory may not
translate to equally good results in real clinical settings;
results of preclinical studies may not correlate with the results
of human clinical trials; Pluristem’s patents may not be
sufficient; Pluristem’s products may harm recipients; changes in
legislation may adversely impact Pluristem; inability to timely
develop and introduce new technologies, products and applications;
loss of market share and pressure on pricing resulting from
competition, which could cause the actual results or performance of
Pluristem to differ materially from those contemplated in such
forward-looking statements. Except as otherwise required by law,
Pluristem undertakes no obligation to publicly release any
revisions to these forward-looking statements to reflect events or
circumstances after the date hereof or to reflect the occurrence of
unanticipated events. For a more detailed description of the risks
and uncertainties affecting Pluristem, reference is made to
Pluristem's reports filed from time to time with the Securities and
Exchange Commission.
Contact:Efrat Kaduri Director of Business,
Investor and Public Relations972-74-7108600efratk@pluristem.com
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