The factors discussed herein, including those risks described in Item 1A. "Risk Factors", and expressed from time to time in our filings with the Securities and Exchange Commission could cause actual results and developments to be materially different from those expressed in or implied by such statements. The forward-looking statements are made only as of the date of this filing, and except as required by law we undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.
PART I
Our Current Business
We are a bio-therapeutics company developing standardized cell therapy products for the treatment of life threatening diseases. We are developing a pipeline of products, stored ready-to-use, derived from human placenta, a non-controversial, non-embryonic, adult cell source. Placental-derived adherent stromal cells are grown using our proprietary PluriX™ three-dimensional process that allows cells to grow in a natural environment and enables us to produce large quantities of clinical grade cells. We refer to the cells that are grown in the PluriX™ as our PLacental eXpanded cells, or PLX cells. We are expanding our in-house manufacturing capacity so that we will be able to grow large scale quantities of our cells efficiently and without reliance on outside vendors.
We were incorporated as a Nevada corporation in 2001. We have a wholly owned research and development subsidiary in Israel called Pluristem Ltd. We operate in one segment, namely, the research, development, and commercialization of cell therapeutics and related technologies.
Our strategy is to develop and manufacture cell therapy products for the treatment of multiple disorders using several methods of administration. We plan to execute this strategy independently, using our own personnel, and through relationships with research and clinical institutions or in collaboration with other companies, such as United Therapeutics Corporation, or United. We plan to have in-house manufacturing capacity to grow clinical grade PLX cells in commercial quantities and to control all of our proprietary manufacturing processes in order to assist in executing this strategy.
We believe that intramuscular administration, or IM, which means that the cells are administrated locally to the muscle and not systemically, may be suited for a number of different clinical indications. Such indications include peripheral artery disease, or PAD, critical limb ischemia, or CLI, intermittent claudication, or IC, muscle injuries, thromboangiitis obliterans, or Buerger's disease, neuropathic pain, wound healing, orthopedic injuries, bone marrow diseases and acute radiation syndrome. In addition, we have reported the results of animal and other pre-clinical studies that demonstrated the potential utility of our PLX cells, using other several administrating methods, for the treatment of multiple sclerosis, ischemic stroke, inflammatory bowel disease, acute myocardial infraction, diabetic diastolic heart failure, interstitial lung disease and radiation exposure. Under our exclusive license agreement with United, or the United Agreement, we plan to participate in the development and commercialization of a PLX cell-based product for the treatment of pulmonary arterial hypertension, or PAH.
Our first product in development, called PLX-PAD, is intended to improve the quality of life of millions of people suffering from PAD.
Recent Developments
Clinical Activities
A seven year old girl suffering from aplastic bone marrow disease was given two doses of PLX cells intramuscularly in a compassionate use situation. Approximately 10 days following the last administration of PLX cells, which took place on March 28, 2012, the patient's hematological parameters began to significantly improve. We believe that this clinical result suggests that PLX cells may be effective in supporting bone marrow transplantation and in treating bone marrow suppression from radiation and chemotherapy. Additionally, this case suggests that PLX cells administered locally into muscle tissue may have systemic effects. The treatment was made under the Israeli government's compassionate use program.
In August 2012, we announced that the injection of PLX cells in a patient suffering from bone marrow failure in which there was a dangerous reduction in the number of red blood cells, white blood cells, and platelets (pancytopenia) caused a meaningful improvement in her clinical condition leading to her discharge from the hospital. The treatment was made under the Israeli government's compassionate use program.
In September 2012, we announced that our PLX cells were administered to a third patient suffering from Acute Myeloid Leukemia in Hadassah Medical Center and his clinical condition and wellbeing significantly improved, resulting in his release from the hospital. The treatment was made under the Israeli government's compassionate use program.
In April 2012, we received clearance from the U.S. Food and Drug Administration, or the FDA, for our investigational new drug application for the use of PLX-PAD in a phase II clinical trial in IC. The trial will evaluate the safety and efficacy of two different doses of PLX-PAD compared to a placebo administered by one or two intramuscular injections. The trial is expected to be conducted at several leading U.S. clinical sites.
In August 2012, we announced that we received clearance from the Paul-Ehrlich-Institute, the medical regulatory body in Germany, to commence a Phase I/II randomized, double blind, placebo controlled study to assess the safety and efficacy of PLX cells, through intramuscular injections, for the regeneration of injured gluteal musculature following total hip replacement.
2
Pre-Clinical Activities
We recently announced the results of two pre-clinical studies using our PLX cells in animals for the treatment of acute myocardial infarction and diabetic diastolic heart failure. In the heart attack study, PLX cells reduced the area of infarction and improved cardiac hemodynamic parameters. In the diabetic diastolic heart failure study, conducted under the European Commission's Seventh Framework Program (FP7) Collaboration, data suggest that cardiac function in animals with diabetic-induced diastolic dysfunctional heart failure improved following the administration of PLX cells.
In June 2012, we announced the completion of a pre-clinical study measuring the effectiveness of our PLX cells when administered intramuscularly in animals whose bone marrow had been previously irradiated. This approach may produce systemic benefits for a number of hematological disorders, as well as primary and secondary bone marrow failure, such as in radiation sickness and possibly for some complications from chemotherapy and radiotherapy.
In July 2012, we announced an invitation from the U.S. National Institute of Allergy and Infectious Diseases to submit our PLX cells to the Institute for evaluation in models of hematopoietic and gastrointestinal acute radiation syndrome.
In July 2012, we also announced the results of pre-clinical studies in animals that show that PLX cells may be effective in reducing pulmonary fibrosis and improving lung function in a group of diseases collectively called interstitial lung disease.
Scientific Background
Cell therapy is an emerging and promising field within the regenerative medicine area. The characteristics and properties of cells vary as a function of tissue source and growth conditions. The human placenta provides a unique, renewable, uncontroversial source of non-embryonic, adult cells and represents a new approach in the cell therapy field.
The use of our PLX cells for human therapy does not require tissue matching prior to administration. Thus, it allows for the development of a ready-to-use "off-the-shelf" product.
Our Technology
We develop and intend to commercialize cell therapy production technologies and products. We are expanding non-controversial, placental-derived Adherent Stromal Cells, or ASCs, by a proprietary three dimensional (3D) process, termed PluriX™, and using the cells in therapeutics for a variety of degenerative, ischemic, inflammatory and autoimmune disorders.
PluriX™ uses a system of stromal cell cultures and substrates to create an artificial three dimensional environment where placental-derived stromal cells (obtained after birth) can grow. Our three-dimensional process enables the large scale production of reproducible, high quality cell products, and is capable of manufacturing large numbers of PLX doses originating from different placentas. Additionally, our manufacturing process has demonstrated batch-to-batch consistency, an important manufacturing challenge for biological products.