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Strong Balance Sheet, Two Active Clinical Development Programs in the U.S. Accelerated Pathways to Commercialization in Japan & Europe
HAIFA, ISRAEL, February 10, 2016 — Pluristem Therapeutics Inc. (NasdaqCM: PSTI, TASE: PLTR), a leading developer of placenta-based cell therapy products, today reported financial highlights for its second quarter ended December 31, 2015, and provided clinical and corporate updates.
“We achieved significant clinical milestones during our second fiscal quarter. PLX-R18, our second product, was cleared by the FDA for clinical studies. The PLX-R18 product is designed to be an entirely new and innovative treatment approach for a wide variety of hematopoietic disorders, and might save the lives of severely ill patients with no alternative treatment options. Receipt of an Orphan Drug Designation from the FDA for PLX-PAD cells in the treatment of severe preeclampsia encourages us to move forward with the development plan of this indication,” stated Pluristem Chairman and CEO Zami Aberman. “In both Europe and Japan, we are working with regulators to expedite commercialization of PLX-PAD in the treatment of critical limb ischemia, and we are very pleased that Japanese regulators approved the protocol for our pivotal Phase 2 trial in that indication.”
As of December 31, 2015, Pluristem had $44 million in cash and cash equivalents, bank deposits, restricted deposits and marketable securities. The Company’s net cash used for operating activities was $ 3.6 million for the second quarter. As a result, Pluristem anticipates being well capitalized to conduct the clinical trials that are planned for initiation in 2016, as well as ongoing R&D efforts to support future products approval.
Two Clinical Development Programs in the U.S.
data analysis. Pluristem intends to pursue early market access for PLX-R18 in the U.S. via a Breakthrough Therapy Designation.
Expedited Path to Commercialization in Japan and Europe
Corporate Collaborations
Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapy products. The Company has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells. The cells release a cocktail of therapeutic proteins in response to inflammation, ischemia, hematological disorders, and radiation damage. PLX cell products are grown using the Company’s proprietary three-dimensional expansion technology. They are off- the-shelf, requiring no tissue matching prior to administration.
Pluristem has a strong intellectual property position; Company-owned and operated, GMP- certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team.
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. For example, we are using forward-looking statements when we discuss the potential of PLX-R18 to be an entirely new and innovative treatment approach for a wide variety of hematopoietic disorders, and to save the lives of severely ill patients with no alternative treatment options, and moving forward with development plan of this indication, when we discuss expediting commercialization of PLX-PAD in the treatment of critical limb ischemia, when we discuss our anticipation to be well capitalized to conduct the clinical trials that are planned for initiation in 2016, as well as ongoing R&D efforts to support future products approval, when we discuss our clinical trials and studies, including timing for initiation, when we discuss pursuing early market access for PLX-R18 in the U.S. via a Breakthrough Therapy Designation, and when we discuss our collaborations with the Berlin-Brandenburg Center for Regenerative Therapy at Charité – University Medicine Berlin and with Fukushima Medical University. 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; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; 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; our patents may not be sufficient; our products may harm recipients; changes in legislation; 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. 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.
HAIFA, ISRAEL, January 26, 2016 — Pluristem Therapeutics Inc. (NasdaqCM: PSTI , TASE: PLTR), a leading developer of placenta-based cell therapy products, today announced that Dr. Karine Kleinhaus, the Company’s Divisional VP, North America, will present at the Bio CEO & Investor Conference on February 8 at 8:30 am ET. The conference will take place on February 8 and 9, 2016 at the Waldorf Astoria in New York City. Investors and media interested in a meeting with Dr. Kleinhaus may contact Pluristem directly or investors may book a one-on-one meeting through the conference organizers.
Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapy products. The Company has reported positive clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells. The cells release a cocktail of therapeutic proteins in response to inflammation, ischemia, hematological disorders, and radiation damage. PLX cell products are grown using the Company’s proprietary three-dimensional expansion technology. They are off- the-shelf, requiring no tissue matching prior to administration.
Pluristem has a strong intellectual property position; Company-owned and operated, GMP- certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team.
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. For example, we are using forward-looking statements when we discuss the findings of the study, the evidence they provide and their potential implications, such as the superiority of placenta-derived cells over those sourced from fat tissue and bone marrow and the potential power of placental cells to address complex diseases without harming the patient, and when we discuss our plans to advance into clinical studies of PLX-PAD cells in treating preeclampsia. 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; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; 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; our patents may not be sufficient; our products may harm recipients; changes in legislation; 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. 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.
HAIFA, ISRAEL, January 12, 2016 — Pluristem Therapeutics Inc. (NasdaqCM: PSTI , TASE: PLTR), a leading developer of placenta-based cell therapy products, announced today that the U.S. Food and Drug Administration (FDA) cleared the Company’s Investigational New Drug (IND) application to begin its Phase I trial of PLX-R18 cells to treat incomplete hematopoietic recovery following Hematopoietic Cell Transplantation (HCT). The clinical trial is expected to begin in the first half of 2016.
PLX-R18 is Pluristem’s second cell therapy product cleared for clinical studies by the U.S. FDA. It has already been studied in preclinical models of acute radiation syndrome, support of hematopoietic cell transplants, and side effects of radiotherapy and chemotherapies used to treat cancers. Preclinical data from trials conducted by the U.S. National Institutes of Health, Hadassah Medical Center, and other prominent research institutions have shown that PLX-R18 cells secrete a range of specific proteins that trigger the resurgence of progenitor cells, supporting the recovery of blood cell counts. By this mechanism of action, PLX-R18 could potentially treat a broad range of hematologic indications.
“The PLX-R18 product is designed to be an entirely new and innovative treatment approach for a wide variety of hematopoietic disorders, and might save the lives of severely ill patients with no alternative treatment options. We are encouraged by the strong pre-clinical data, and intend to pursue early market access in the U.S. for this important clinical indication,” stated Pluristem Chairman and CEO, Zami Aberman.
The planned study is a Phase 1, multi-center, open-label, dose-escalating study to evaluate the safety of intramuscular injections of PLX-R18 cells in subjects with incomplete hematopoietic recovery following hematopoietic cell transplantation (HCT). This study will be conducted in 30 subjects with incomplete hematopoietic recovery persistent for 6 months or more after HCT. There will be three cohorts: 1) 3 subjects receiving two administrations of 1 million PLX-R18 cells/kg each, separated by a 1 week interval; 2) 12 subjects receiving two administrations of 2 million cells/kg each, separated by a 1 week interval; and 3) 15 subjects receiving two administrations of 4 million cells/kg each, separated by a 1 week interval. The follow up period will be 12 months. The primary endpoints will be safety endpoints and will include adverse events, laboratory values and vital signs. Exploratory endpoints will include changes in platelet and hemoglobin levels, changes in transfusion frequency, a shift from transfusion dependence to transfusion independence, quality of life and changes in the serum immunological parameters.
Bone marrow failure is the inability of bone marrow to produce sufficient numbers of platelets, white or red blood cells. This inability may result in serious illness or death, because these cells are necessary to prevent hemorrhage, infection or severe anemia. Bone marrow failure can be caused either by medical conditions such as aplastic anemia, myelodysplastic syndrome, hematologic malignancies, or as a side effect of radiation or chemotherapy cancer treatment. The incidence of bone marrow failure resulting from these conditions varies widely, but is increasing.
The only cure for bone marrow failure is HCT, although supportive therapies and treatments can reduce symptoms and prolong life for some patients. The hematopoietic cells for HCT can come from a donor or from the patient, and can be harvested from peripheral blood, bone marrow or umbilical cord blood. Transplant patients require extensive care and monitoring, and sometimes need intensive treatment for complications. In cases of incomplete engraftment, blood cell counts are insufficient causing the patient to be at high risk of severe or lethal complications. In severe cases the patient may need to undergo the arduous and dangerous process of a second transplant.
Pluristem Therapeutics is a leading developer of placenta-based cell therapy products.
In 2016, the Company expects to initiate pivotal trials with PLX-PAD cells aimed at conditional marketing approval for the treatment of critical limb ischemia in Europe and Japan.
A global Phase 2 trial is currently being conducted with PLX-PAD cells for the treatment of intermittent claudication. PLX cell products release product-specific cocktails of therapeutic proteins in response to conditions such as inflammation, ischemia or hematological disorders. The Company’s proprietary, three- dimensional expansion technology can grow mass quantities of commercial-grade cells with batch- to-batch consistency at Pluristem’s FDA and EMA-approved, state-of-the-art manufacturing facility.
The cells are off-the-shelf, requiring no tissue matching prior to administration, making the treatment cost effective and readily available in virtually any medical setting. Pluristem has a strong intellectual property position and strategic relationships with major research institutions.
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. For example, we are using forward-looking statements when we discuss our expected Phase 1 trial of PLX-R18 cells to treat incomplete hematopoietic recovery following HCT, including the recruitment process and its timing; when we discuss the possibility of PLX-R18, if successful, to be used to support the recovery of the hematological system and restore production of normal levels of blood cells by the transplanted hematopoietic cells; when we discuss PLX-R18’s potential to become novel innovative product with the ability to support the recovery of all three blood lines; when we discuss PLX-R18’s potential to treat a broad range of indications related to abnormal bone marrow function; when we discuss the possibility of PLX-R18 cells to be an entirely new and innovative treatment for incomplete engraftment of HCT, and to save the lives of severely ill patients with no remaining treatment options; when we discuss our intention to pursue early market access in the U.S. for PLX-R18; and when we discuss our plan to initiate pivotal trials with PLX- PAD cells aimed at conditional marketing approval for the treatment of critical limb ischemia in Europe and Japan in 2016. 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; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; 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; our patents may not be sufficient; our products may harm recipients; changes in legislation; 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. 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.
First published study comparing therapeutic outcome of placenta-derived cells as compared to cells
derived from bone marrow or fat tissue
– Data describes the mechanism of action of PLX-PAD cells in preeclampsia
– Study independently conducted by scientists at Texas A&M Health Science Center
– PLX-PAD Cells recently received Orphan Drug Designation for Severe Preeclampsia
HAIFA, ISRAEL, January 6, 2016 — Pluristem Therapeutics Inc. (NasdaqCM: PSTI , TASE: PLTR), a leading developer of placenta-based cell therapy products, today announced the publication of a scientific study of PLacental eXpanded (PLX) cells in the prominent peer- reviewed journal Clinical Science. The paper, titled “Human Placenta-Derived Stromal Cells Decrease Inflammation, Placental Injury, and Blood Pressure in Hypertensive Pregnant Mice,” describes the positive findings of a recent preclinical study of PLX-PAD cells for the treatment of preeclampsia. This marks the first published study indicating the superiority of placenta-derived mesenchymal cells in a therapeutic outcome as compared to cells derived from bone marrow or fat tissue, thus having implications beyond preeclampsia. Scientists also described the potential mechanism of action by which PLX-PAD cells treated symptoms of preeclampsia. Conducted by independent scientists at the Texas A&M Health Science Center/Baylor Scott & White Health, the paper was co-authored by scientists from the Health Science Center and Pluristem Therapeutics.
Preeclampsia occurs in approximately 6-8% of pregnancies worldwide, and greatly increases the risks of serious illness and death for a pregnant woman and her baby. There is no cure other than delivery, which may be necessary even if the baby will be born prematurely, in some cases even before a viable gestational age is reached. Pluristem’s PLX-PAD cells recently received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of severe preeclampsia.
“This study is extremely important at this time because it provides data which demonstrate two critical outcomes. First, study findings indicated the potential mechanism of action to explain the effectiveness of PLX-PAD cells in the treatment of symptoms of preeclampsia following intramuscular injection. Second, the data have implications beyond preeclampsia by suggesting the superiority of placenta-derived cells over those sourced from fat tissue and bone marrow,” stated Pluristem Chairman and CEO Zami Aberman. “The study confirms the power of placental cells to address complex diseases without harming the patient, through a clear mechanism of action and multifactorial response. Specific to preeclampsia, with the support of the FDA’s Orphan Drug Designation, we look forward to advancing into clinical studies in this severe unmet need that causes so many serious consequences for pregnant women and their babies.”
Data from the study showed that PLX-PAD cells, which are derived from placental cells, were able to normalize or significantly lower all the key symptoms of preeclampsia in two mouse models of the disease with only one intramuscular dose. PLX-PAD cells normalized blood pressure, marker levels of kidney function, and blood vessel function, all of which are considered to be key physiologic features contributing to preeclampsia. PLX-PAD also reduced levels of markers of inflammation and placental injury, thought to be central to the disease process as well.
Another important finding was that PLX-PAD cells did not affect healthy pregnant mice or their fetuses, consistent with prior independent studies conducted at Charles River. The trial data provide further evidence that administration of PLX cells into muscle generates a systemic effect, without any cell migration or engraftment into the mother, the placenta or her fetus. The article cites an earlier animal study, in which PLX-PAD cells were shown to stay in the muscle in which they were injected, and that they were no longer detectable after several weeks, i.e., there was no migration or engraftment of PLX cells.
The study also compared PLX-PAD cells, which are placenta derived, to cells derived from bone marrow and human adipose (fat) tissue, which were found to have no therapeutic effect. Data showed that PLX-PAD cells successfully treated symptoms that are present in diseases beyond preeclampsia, including immune system disturbances, excessive inflammation, and vascular dysfunction. Cells from bone marrow and fat tissue administered in these studies did not improve outcomes.
There were three study arms. On gestational days 13, 15 and 17, pregnant C57BL/6J mice were given intraperitoneal injections of a control (saline), TLR3 agonist or TLR7 agonist. Administration of TLR3 or TLR7 agonists generated two parallel animal models of preeclampsia. On gestational day 14, mice within each group received an intramuscular injection of either a control (PlasmaLyte) or PLX-PAD cells. Selected groups of TLR3 and TLR7 agonist-exposed mice received either adipose cells or bone marrow cells on day 14, administered intramuscularly as well.
Mice exposed to either TLR3 or TLR7 agonists developed preeclampsia-like symptoms; these included hypertension, signs of kidney dysfunction (abnormal urinary protein/creatinine ratio), and signs of vascular dysfunction (abnormal endothelium-dependent relaxation responses). Those treated with intramuscular injection of PLX-PAD cells on day 14 of gestation exhibited significantly decreased systolic blood pressure by day 17. In both mouse models of preeclampsia, each decrease was statistically significant (p<0.05) for PLX-PAD treated mice as compared to control. By day 17, PLX-PAD treatment normalized the elevated levels of urinary protein/creatinine ratio in both models, and both decreases were statistically significant (p<.05) as compared to control. On gestational day 17, aortic endothelium-dependent relaxation responses improved significantly in PLX-PAD treated mice both in the TLR3 and the TLR7 models of preeclampsia, and the improvements were statistically significant for both (p<.05) as compared to control. Treatment with PLX-PAD cells also reduced the levels of markers of systemic inflammation and placental injury in these models.
In both mouse models of preeclampsia, single doses of PLX-PAD cells were able to reduce all of the measured preeclampsia symptoms in these mice. In contrast, human adipose or bone marrow cells were not able to reduce the inflammation, hypertension, or proteinuria in these preeclamptic mice.
The cells were shown to secrete multiple chemical factors thought to address pathological processes central to the disease, including hMMP-1, hMMP-2, hMMP-3, and hTIMP-1. Also observed in treated mice were increased levels of mEGF and mVEGF. These findings suggest that PLX-PAD cells exert multifactorial therapeutic mechanism to address various features of a complex disease process.
Preeclampsia is one of the most common medical complications of pregnancy, and one of the leading known causes of premature births, stillbirths and early neonatal and maternal deaths. The disease occurs after the 20th week of pregnancy, and is characterized by high blood pressure and significant amounts of protein in the urine or end-organ dysfunction. The disease may lead to liver and renal failure, central nervous system (CNS) abnormalities including seizures, and disseminated intravascular coagulopathy. The only definitive treatment for preeclampsia is delivery.
Severe preeclampsia, which occurs in 1% of pregnancies in the U.S., is defined by the presence of at least one additional symptom in a patient meeting the criteria for preeclampsia; these additional symptoms include severe high blood pressure, signs of severe kidney malfunction, low platelets, persistent headaches, and pulmonary edema.
Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapy products. The Company has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells. The cells release a cocktail of therapeutic proteins in response to inflammation, ischemia, hematological disorders, and radiation damage. PLX cell products are grown using the Company’s proprietary three-dimensional expansion technology. They are off- the-shelf, requiring no tissue matching prior to administration.
Pluristem has a strong intellectual property position; Company-owned and operated, GMP- certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team.
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. For example, we are using forward-looking statements when we discuss the findings of the study, the evidence they provide and their potential implications, such as the superiority of placenta-derived cells over those sourced from fat tissue and bone marrow and the potential power of placental cells to address complex diseases without harming the patient, and when we discuss our plans to advance into clinical studies of PLX-PAD cells in treating preeclampsia. 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; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; 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; our patents may not be sufficient; our products may harm recipients; changes in legislation; 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. 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.
HAIFA, ISRAEL, December 21, 2015 — Pluristem Therapeutics Inc. (NasdaqCM / TASE: PSTI), a leading developer of placenta-based cell therapy products, today announced that it has reached an agreement with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) on the design of the final trial needed to apply for conditional approval of PLX-PAD cells in the treatment of critical limb ischemia (CLI). The approval of the protocol for the 75-patient trial was part of a larger agreement on the development of PLX-PAD via Japan’s new accelerated regulatory pathway for regenerative medicine.
“With this achievement we have advanced our strategy to expedite commercialization of our cell products. Pluristem is now positioned favorably to accelerate negotiations with those Japanese pharma companies interested in becoming dominant players in the expanding regenerative medicine market in Japan,” stated Pluristem Chairman and CEO Zami Aberman.
The trial will collect data on 75 patients suffering from CLI. These patients will be randomized into three groups of 25. Group one will receive an initial 150 million PLX- PAD cell dose followed eight weeks later by a second 150 million cell dose; group two will be treated with an initial 300 million PLX-PAD cells followed eight weeks later by a second dose of 300 million cells; group three will receive two doses of placebo. The cells will be injected into a leg muscle using a standard syringe. Efficacy and safety will be determined from outcomes measured nine months after administration of the first dose. The primary efficacy endpoint will be diagnosis of a patient as CLI-free for 60 days. Pluristem expects to submit the formal Clinical Trial Notification (CTN) to the PDMA, based on the agreement reached with the regulatory body, in early 2016. The PMDA is expected to respond officially within 30 days. Earlier in 2015, the PMDA cleared PLX- PAD cells for use in clinical studies in Japan, a prerequisite to conducting this clinical trial.
Japan’s Act on the Safety of Regenerative Medicines went into effect in November 2014. Its purpose is to facilitate faster approval of cellular therapies and other regenerative medicine treatments for marketing. According to the law, these therapies can receive conditional, time-limited approval for marketing, and be eligible for reimbursement, upon proof of safety and a signal of effectiveness but prior to verification of efficacy. Safety and efficacy need to be confirmed via collection of observational data after the conditional approval.
Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapy products. The Company has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells. The cells release a cocktail of therapeutic proteins in response to inflammation, ischemia, hematological disorders, and radiation damage. PLX cell products are grown using the Company’s proprietary three-dimensional expansion technology. They are off-the-shelf, requiring no tissue matching prior to administration.
Pluristem has a strong intellectual property position; Company-owned, GMP-certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team.
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. For example, we are using forward-looking statements when we discuss our expectation that the agreement with the PDMA will accelerate discussions with potential Japanese partners, when we discuss our plan to conduct clinical trial of PLX- PAD cells in the treatment of CLI and the trial’s design, and when we discuss our expectation to submit the formal CTN to the PDMA by the end of Q1, 2016 and the expected timing of response by the PDMA. 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; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; 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; our patents may not be sufficient; our products may harm recipients; changes in legislation; 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. 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.
Yaky Yanay to deliver two presentations regarding accelerated clinical development pathways in Europe and Japan
HAIFA, ISRAEL, December 17, 2015 — Pluristem Therapeutics Inc. (NasdaqCM / TASE: PSTI), a leading developer of placenta-based cell therapy products, today announced that the Company’s President and COO Yaky Yanay will deliver two presentations at the 12th Annual Phacilitate Cell & Gene Therapy World Conference which takes place January 25-27, 2016 in Washington D.C.
On Monday, January 25 at 3:00pm Mr. Yanay will present “Pursuing clinical development as an in house manufacturer under the new regulations in Japan” during the session titled “Sharing Experiences to Date of Western Industry Trailblazers Seeking to Access the Japanese Regenerative Medicine Marketplace.”
On Tuesday, January 26 at 3:15pm Mr. Yanay’s presentation titled “Industry perspective on the EMA Adaptive Pathway” will be part of Focus Session 2 “Accelerated clinical development pathways across the globe – what evidence of benefits to date for cell & gene therapy?”
This year’s conference, titled “From Promise to Products,” features a one-day Japan Executive Summit which will focus on the novel regulatory pathways that have opened up in the Japanese regenerative medicine arena.
Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has cleared Pluristem’s PLX-PAD cells for use in clinical trials in Japan. Pluristem is pursuing approval to conduct a pivotal Phase II clinical trial for conditional marketing approval of PLX-PAD in critical limb ischemia (CLI) through Japan’s accelerated regulatory pathway for regenerative medicine.
Pluristem’s PLX cell program in critical limb ischemia (CLI) was one of a handful of programs that were selected for the European Medicines Agency’s (EMA) Adaptive Pathways pilot project whose goal is to improve timely access for patients to innovative new medicines. Pending a single successful Phase II trial, PLX-PAD could enter the European market in 2018 with conditional approval to treat CLI with PLX-PAD.
About Pluristem Therapeutics
Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapy products. The Company has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells. The cells release a cocktail of therapeutic proteins in response to inflammation, ischemia, hematological disorders, and radiation damage. PLX cell products are grown using the Company’s proprietary three-dimensional expansion technology. They are off-the-shelf, requiring no tissue matching prior to administration.
Pluristem has a strong intellectual property position; Company-owned, GMP-certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team.
PLX cells were demonstrated to be safe for both the mother and fetus and improved several parameters of preeclampsia in several animal studies
HAIFA, ISRAEL, December 31, 2015 — Pluristem Therapeutics Inc. (NasdaqCM, TASE: PSTI), a leading developer of placenta-based cell therapy products, today announced that the U.S. Food and Drug Administration has granted the Company’s PLX- PAD cells Orphan Drug Designation in the treatment of severe preeclampsia. Preeclampsia is among the most common medical complications of pregnancy and a leading cause of premature births, stillbirths and neonatal and maternal deaths. There is no cure except delivery. Due to high risks to the mother, women diagnosed with severe preeclampsia are usually delivered promptly, even if the baby will be born prematurely and may suffer permanent disabilities as a result. Severe preeclampsia occurs in approximately 1% of pregnancies in Western countries.
Benefits of Orphan Drug Designation for PLX-PAD cells include close guidance that may accelerate time to marketing approval, orphan drug grants, tax credits, and a 7-year market exclusivity upon marketing approval. It is estimated by different sources that preeclampsia costs the global health care system about $3 billion annually.
“Attainment of Orphan Drug Designation for our cells in severe preeclampsia exemplifies our global strategy of bringing cell therapies to patients through accelerated approval pathways,” stated Pluristem Chairman and CEO Zami Aberman. “We are encouraged by the US FDA designation that demonstrates Pluristem’s commitment to the program and the potential promise it holds to address a serious, unmet medical need faced by pregnant women every year”
PLX-PAD cells improved several parameters of preeclampsia in animal models in a study conducted in collaboration with Brett Mitchel PhD, Associate Professor of Internal Medicine at the Cardiovascular Research Institute (CVRI) of the Texas A&M College of Medicine. In a different animal study conducted a Charles River Laboratories, PLX-PAD cells were demonstrated to be safe for both the mother and fetus. As previously requested by the FDA, study in additional animal model (over expression of sFLT-1) to confirm the efficacy of PLX-PAD is on-going to test the efficacy of PLX-PAD in an additional therapeutic pathway. Data is expected in H1 of 2016.
Preeclampsia is one of the most common medical complications of pregnancy, and one of the leading known causes of premature births, stillbirths and early neonatal and maternal deaths. The disease occurs after the 20th week of pregnancy, and is characterized by high blood pressure and significant amounts of protein in the urine or end-organ dysfunction. The disease may lead to liver and renal failure, central nervous system (CNS) abnormalities including seizures, and disseminated intravascular coagulopathy. The only definitive treatment for preeclampsia is delivery. Severe preeclampsia, which occurs in 1% of pregnancies in the U.S., is defined by the presence of at least one additional symptom in a patient meeting the criteria for preeclampsia; these additional symptoms include severe high blood pressure, signs of severe kidney malfunction, low platelets, persistent headaches, and pulmonary edema.
Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapy products. The Company has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells. The cells release a cocktail of therapeutic proteins in response to inflammation, ischemia, hematological disorders, and radiation damage. PLX cell products are grown using the Company’s proprietary three-dimensional expansion technology. They are off-the-shelf, requiring no tissue matching prior to administration.
Pluristem has a strong intellectual property position; Company-owned, GMP-certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team.
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. For example, we are using forward-looking statements when we discuss benefits of Orphan Drug Designation for PLX cells, or when we discuss our global strategy of bringing cell therapies to patients through accelerated approval pathways, or when we discuss our commitment to the severe preeclampsia program and the potential promise it holds to address a serious, unmet medical need faced by pregnant women every year. 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; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; 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; our patents may not be sufficient; our products may harm recipients; changes in legislation; 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. 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.
HAIFA, ISRAEL, December 8, 2015 — Pluristem Therapeutics Inc. (NasdaqCM / TASE: PSTI), a leading developer of placenta-based cell therapy products, announced that it received a notice today from United Therapeutics Corporation (“United”) ending its licensing agreement for the development of PLX-PAD for the treatment of pulmonary arterial hypertension (PAH). According to the licensing agreement Pluristem will regain full rights to PLX-PAD in this indication, as well as all clinical data and regulatory submissions, allowing Pluristem to move forward with the clinical development of the program and seek other licensing partners.
Data from the first cohort of three patients given PLX-PAD in the single-arm Phase I trial conducted by United in September 2014 which has now become available for publication, demonstrated a good safety profile and encouraging, albeit limited, efficacy trend. In addition, initial findings from cohort 1 suggest an average improvement of 21 meters from base line in the six-minute walk distance (6MWD) at 3-month follow up. The two patients with Grade 2 (moderate) PAH had an average improvement of 40 meters in 6MWD. The first cohort of patients received a single IV injection of 0.5 million cells/kg patient weight. Enrollment of the second cohort of patients is complete and follow up is ongoing. Those patients received 1 million cells/kg patient weight.
“We thank United for the work they have completed on this project and believe PLX- PAD can make a significant contribution to the health of patients suffering from PAH. The data generated by United provides a good foundation to suggest that our cells can be safely administered intravenously, with potential broad application, and can improve the quality of life for PAH patients. We have previously announced results from several studies that demonstrate the safety of intramuscular administration, and with the new intravenous data we can now expand the potential indications for which PLX cells can be used,” stated Pluristem Chairman and CEO Zami Aberman.
Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapy products. The Company has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells. The cells release a cocktail of therapeutic proteins in response to inflammation, ischemia, hematological disorders, and radiation damage. PLX cell products are grown using the Company’s proprietary three-dimensional expansion technology. They are off-the-shelf, requiring no tissue matching prior to administration.
Pluristem has a strong intellectual property position; Company-owned, GMP-certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team.
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. For example, we are using forward-looking statements when we discuss our plans to move forward with the clinical development of the PLX-PAD for treatment of PAH program and seek other licensing partners, when we discuss PLX-PAD’s potential to make a significant contribution to the health of patients suffering from PAH, when we discuss the potential for intravenous use of PLX cells across a broad range of 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; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; 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; our patents may not be sufficient; our products may harm recipients; changes in legislation; 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.
Pluristem Therapeutics Inc. Karine Kleinhaus, MD, MPH Divisional VP, North America 1-914-512-4109
Effects of PLX-R18 will be studied in the lungs, gastrointestinal tract, skin, and bone marrow following exposure to catastrophic levels of radiation
HAIFA, ISRAEL, December 3, 2015 — Pluristem Therapeutics Inc. (NasdaqCM/ TASE: PSTI), a leading developer of placenta-based cell therapy products, today announced that it has signed a Memorandum of Understanding (MOU) for a collaboration with Fukushima Medical University, Fukushima Global Medical Science Center. The purpose of the collaboration is to develop Pluristem’s PLX-R18 cells for the treatment of Acute Radiation Syndrome (ARS), and for morbidities following radiotherapy in cancer patients.
ARS is caused by exposure to dangerously high levels of radiation, such as could occur in a nuclear catastrophe, and incorporates potentially lethal damage to the gastrointestinal tract, lung, skin and bone marrow, as well as other systems. In this new collaboration PLX-R18 cells will be studied primarily as a potential treatment for radiation-induced damage to the skin, lungs and gastrointestinal tract. The parties intend to develop preclinical models of radiation damage in these tissues, and then use them in trials. Pluristem will contribute PLX-R18 cells and scientific knowledge, while Fukushima Medical University will conduct the studies, and provide the required resources.
The collaboration will proceed alongside research supported by the U.S. National Institutes of Health (NIH), which is studying PLX-R18 as a potential treatment for the hematologic component of ARS. Insufficient blood cell production by the bone marrow, which may be caused by various reasons including ARS and cancer treatments, can be life threatening because it may lead to hemorrhage, the inability to fight infection, and anemia.
Data from a preclinical study, published in the peer-reviewed scientific journal PLOS One, suggest that PLX-R18 cells, administered intramuscularly, may be a highly effective off-the-shelf therapy for bone marrow failure following total body exposure to high doses of radiation. The work with Fukushima Medical University, Fukushima Global Medical Science Center will further evaluate the efficacy of intramuscular administration for systemic treatment of ARS.
“The researchers at Fukushima have a strong interest in cell therapies that may offer a treatment for conditions caused by exposure to ionizing radiation. We are honored to work with the thought leaders in the field at Fukushima Medical University, Fukushima Global Medical Science Center to further develop PLX-R18 for ARS-related indications,” stated Pluristem CEO Zami Aberman.
“We anticipate that our work with Fukushima Medical University, Fukushima Global Medical Science Center will help to maintain the health of those involved in decommissioning the Fukushima reactors; the decommissioning process is estimated to take about 40 years. We hope that this collaboration can advance efforts to safeguard populations around the world from illness related to radiation exposure,” concluded Mr. Aberman.
PLX-R18 is Pluristem’s second cell therapy product in development. It is already being studied as a treatment for bone marrow that is unable to produce blood cells because of a variety of causes including: ARS; unsuccessful hematopoietic cell transplants; certain cancers; radiotherapy and certain chemotherapies used to treat some cancers; and immune-mediated bone marrow failure. Pluristem is applying for FDA approval of a Phase I trial of PLX-R18 in incomplete bone marrow recovery following hematopoietic cell transplantation. Pluristem will also be submitting a protocol for late-stage trials of PLX-R18 in the treatment of the hematological component of ARS, targeting the FDA’s Animal Rule regulatory pathway. With its capabilities, PLX-R18 could potentially treat a broad range of indications related to bone marrow function, which together constitute a substantial global market.
Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapy products. The Company has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells. The cells release a cocktail of therapeutic proteins in response to inflammation, ischemia, hematological disorders, and radiation damage. PLX cell products are grown using the Company’s proprietary three-dimensional expansion technology. They are off-the-shelf, requiring no tissue matching prior to administration.
Pluristem has a strong intellectual property position; Company-owned, GMP-certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team.
This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. For example, we are using forward-looking statements when we discuss our expected collaboration with Fukushima Medical University Hospital, when we discuss our expectation to initiate an animal model to study the potential of PLX-R18 cells to treat high-dose radiation therapy, when we discuss our hope that the collaboration with Fukushima Medical University Hospital will advance efforts to safeguard populations around the world from illness related to radiation exposure, when we discuss our plan to submit a protocol for late-stage trials of PLX-R18 in ARS, targeting the FDA’s Animal Rule regulatory pathway, and when we discuss PLX-R18’s potential to treat a broad range of indications related to bone marrow function, which together constitute a substantial global market. 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.
HAIFA, ISRAEL, November 24, 2015 — Pluristem Therapeutics Inc. (NasdaqCM/ TASE: PSTI), a leading developer of placenta-based cell therapy products, today announced that members of its executive and scientific teams will present at the following conferences during the month of December:
Florida Wealth Management & Family Office Conference 2015 Date: December 1, 2015
Location: Miami Beach, Florida
Yaky Yanay, Pluristem’s President, CFO and COO will deliver a corporate presentation.
8th International Conference on Cachexia, Sarcopenia and Muscle Wasting Date: December 5, 2015
Location: Paris
Dr. Tobias Winkler of the Center for Musculoskeletal Surgery & Julius Wolff Institute Berlin, Charité, who served as Senior Scientist on Pluristem’s completed Phase I/II orthopedic study, will present data from the trial. His presentation titled, “First in human allogeneic cell therapy after muscle trauma improves functional regeneration,” will be delivered as part of the ‘Late Breaking Clinical Trials & Trial Updates’ session.
Oppenheimer 26th Annual Healthcare Conference Date: December 8, 2015
Location: New York City
Karine Kleinhaus, MD, MPH, Pluristem’s Divisional VP, North Americas, will deliver a corporate presentation.
Israel Society for Biotechnology Engineering Date: December 13, 2015
Location: Tel Aviv
The following three poster presentations will be delivered:
Pluristem previously announced that on December 6, 2015, the Company’s Vice President of Research & Intellectual Property, Dr. Racheli Ofir, will present new data on PLX-R18 at the American Society of Hematology’s Annual Meeting in Orlando, Florida.
About Pluristem Therapeutics
Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapy products. The Company has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells. The cells release a cocktail of therapeutic proteins in response to inflammation, ischemia, hematological disorders, and radiation damage. PLX cell products are grown using the Company’s proprietary three-dimensional expansion technology. They are off-the-shelf, requiring no tissue matching prior to administration.
Pluristem has a strong intellectual property position; Company-owned, GMP-certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team.
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