Full Press Release Details
Throughout this update,
unless otherwise designated, the terms "we", "us", "our", "Cellect", "the
Company" and "our Company" refer to Cellect Biotechnology Ltd. and its wholly-owned subsidiaries. References
to "ordinary shares", "ADSs", "warrants" and "share capital" refer to the ordinary
shares, ADSs, warrants and share capital, respectively, of Cellect.
We are an emerging biotechnology
company that has developed a novel technology platform known as ApoGraft that functionally selects stem cells in order to improve
the safety and efficacy of regenerative medicine and cell therapies. We aim to become the standard enabling technology for the
enrichment of the stem cell population for companies developing stem cell therapies, for physicians practicing regenerative medicine
and for researchers and academia engaged in cell based medicine and research.
We believe our innovative
technology platform represents a potential breakthrough in the field of regenerative medicine by using functional selection of
stem cells. Efficient selection enables retention of most of the stem cells from various starting bulk of cells while neutralizing
harmful mature cells from this bulk of raw material. Animal models suggest that this process results in dramatic decrease of toxicity
coupled with the enrichment of the stem cell population.
Our ApoGraft technology
platform takes advantage of a functional characteristic of stem cells relating to apoptosis. Apoptosis is the process of programmed
cell death and is a vital part of physiological development and homeostasis of all organisms. Stem cells flourish in an environment
where normal cells die because their major role is reconstitution of damaged tissue. Stem cells are attracted to areas of cell
death, areas typified by very high levels of apoptotic activity and apoptotic-inducing signals.
We are currently developing
our first product based on our ApoGraft technology platform, the ApoTainer selection kit that utilizes FasL-coated magnetic beads.
The ApoTainer selection kit is intended to be an easy to use, cost effective, off the shelf stem cell selection kit. In October
2018, we announced that we optimized the beads size, coating technology, elimination of the release of FasL into the medium, all
while preserving the biological activity observed in our ongoing human clinical trial. Pre-clinical proof of concept testing of
the ApoTainer has shown that the use of FasL-coated magnetic beads significantly increases the active surface allowing a dramatic
increase of interactions between the selecting agent and the cells. Further, such testing showed that the outcome increases specific
elimination of certain (but not all) of the non-stem cells while full preservation of the number and function of the stem and progenitor
The ApoGraft technology
platform is being tested for clinical use in allogeneic (using stem cells from a donor) hematopoietic stem cell transplantation,
or HSCT for the treatment of hematological malignancies (blood cancers such as leukemia and lymphoma). HSCT, also known as bone
marrow transplantation, has for decades been curative for many patients with hematological malignancies. Clinical trials have shown
that HSCT can also be used for other non-malignant indications (such as autoimmune diseases), but is rarely used due to severe
toxicity. Application of allogeneic HSCT is limited by graft-versus-host-disease, or GvHD, a condition in which the transplanted
immune cells (populating the graft in much higher numbers then the stem cells) recognize the host cells and organs as foreign and
attack them. GvHD does not resolve by itself and is a major cause of transplant-related morbidity and mortality. Despite improvements
in the outcome of HSCT over recent years through improved supportive care, infection control and use of reduced intensity and reduced
toxicity conditioning regimens, HSCT is still associated with significant morbidity and mortality mainly due to GvHD, and as such
HSCT is restricted to patients with life threatening advanced diseases. Due to non-efficient selection of stem cells for HSCT,
the complex and expansive laboratory process performed using technologies currently available is able to reduce toxicity only at
a significant tradeoff - failure of engraftment, graft rejection, cancer reoccurrence and high costs of treatment.
We have chosen allogeneic
HSCT for the treatment of hematological malignancies as our first target indication for our ApoGraft technology platform in order
to clinically validate that our technology can efficiently select stem cells resulting in neutralizing harmful cells and their
associated medical complications. We believe that demonstrating the safety of our technology for this indication will validate
the use of our ApoGraft technology platform for the treatment of other indications (e.g., nonmalignant bone marrow failure, solid
organ transplantation and auto-immune diseases) and consequently for the adoption of our ApoGraft technology platform by stem cell
therapeutic companies, academia, researchers and others seeking to enrich their stem cell population. In that regard, we believe
that after the first reported results of our human trials, as discussed further below, we will achieve validation of our product's
safety profile, which may result in expediting further development of our technology for multiple indications, even before marketing
approval is obtained. In addition, we believe such validation of our proof of concept will provide us with the opportunity to license
our ApoGraft technology platform in the near term.
We plan to bring our
ApoTainer selection kits to market for HSCT as a combination product subject to the primary jurisdiction of the Center for Biologics
Evaluation and Research, or CBER. The term "combination product", when used to describe our ApoTainer selection kits,
refers to a product, regulated by the FDA, which is comprised of a consumable medical device (container) with a biological activity.
In September 2017, we
announced that the FDA granted orphan drug designation for ApoGraft for the prevention of acute and chronic GvHD in transplant
patients. We plan in the future to apply for fast track and breakthrough technology, which, if received, would result in a reduced
cost of development and expedited marketing approvals, however there is no assurance that such designations will ever be obtained.
Our development efforts
to date have primarily culminated in two studies performed on human HSCT grafts. The first study was performed during 2015-2016.
In this study we used small portions received under ethical committee approval from human donors to validate and optimize the process,
and show robustness and repeatability of the process. More than 100 ApoGraft samples were analyzed for the different effects on
the various groups of cells (stem and mature immune) as well as their functional capabilities (such as migration, colony formation
and anti-cancer activity). The samples represented 5% of a graft used for transplantation into patients. The grafts were processed
in vitro and in vivo (mice) allowing stem cell production for transplantation using ApoGraft. The use of the ApoGraft resulted
in a significant increase in the death of certain mature immune cells, primarily unique subsets of T Lymphocytes, without compromising
the quantity and quality of stem cells.
The second study, which
was initiated in the first quarter of 2017, is a Phase I/II, dose escalating, 4-cohort, open label clinical trial of up to twelve
patients designed to evaluate the safety, tolerability and efficacy of functionally selected donor derived mobilized peripheral
blood cells that underwent our ApoGraft process and were transplanted into patients with hematological malignancies in an allogeneic
hematopoietic stem cell transplantation. The primary endpoint of the study is overall incidence, frequency and severity of adverse
events potentially related to ApoGraft at 180 days from transplantation. The first patient was recruited for this trial in February,
2017 and in October 2018, we announced that the first six patients finished first month follow up and all these patients have shown
100% engraftment with no procedure related adverse events and that the first three patients of the trial (cohort I) completed the
180-day study period with full safety and tolerability. We expect to report topline results from the trial in late 2019 or early
Patients who complete
the Phase I/II study are given the option to enroll in a non-interventional long-term follow-up study for up to two years post-transplantation
to assess incidence, grade and stage of acute GvHD and chronic GvHD, non-relapse related mortality, disease relapse/recurrence
and overall survival.
We aim to commence a
second human ApoGraft trial in the United States for patients with hematological malignancies in an allogeneic HSCT by the end
of 2019. To this end, we plan to enter into a collaboration agreement with a leading academic institution to initiate the trial
and are currently in advanced discussions with a leading academic institution. There can be no assurance that we will enter into
any agreement with such institution. Previously, in May 2017, we announced that the FDA provided us with pre-Investigational New
Drug (IND) meeting minutes supporting an IND submission for ApoGraft.
We are also conducting
studies on mesenchymal stem cells derived from fat tissues. In October 2017, we announced positive results from a more than 20-patient
trial on the use of our selection platform technology on stem cells derived from fat tissues. The study comprised samples obtained
via liposuction from over 20 adult patients and was conducted in collaboration with the Plastic Surgery Department and the Microsurgery
and Plastic Surgery Laboratory of the Tel-Aviv Medical Center (Ichilov Hospital). Fat-derived stem cells were treated according
to our protocols and have shown that our selection platform technology led to both an expansion of cells and an improvement in
their unique cell activity and attributes. The ability of those cells to create colonies and differentiate into bone was enhanced
significantly after only a short incubation. In addition, in October 2018, we announced that we achieved positive results on the
use of human fat derived stem cells treated with the ApoGraft process in orthopedic treatments of animals.
We expect to announce
in the first half of 2019 pre-clinical results for the use of human fat derived stem cells treated with ApoGraft in orthopaedic
treatments of animals. Furthermore, we plan on submitting an IND for the initiation of a Phase I/II trial of ApoGraft for anti-inflammatory