Pluristem Issues Responses from CEO Aberman in Q&A, Says No Effect on PLX-PAD Other Trials Outside US

PluristemTherapeutics Inc. (NASDAQ: PSTI), a leading developer ofplacenta-based cell therapies, recently announced that it hasreceived notification from the U.S. Food and Drug Administration(FDA) that its United States phase II Intermittent Claudication (IC)study (IND 15038) has been placed on clinical hold due to a seriousallergic reaction in a case which required hospitalization. Out of 74 patients enrolled in Pluristem's trials to date, this isthe first case of a serious allergic event. This event occurred in apatient with multiple diseases which may have influenced the severityof the event. The patient was discharged from the hospital thefollowing day, following the resolution of all her symptoms. The FDA will issue a letter within the next 30 days detailing a listof questions and requests for information from Pluristem. Zami Aberman, Pluristem's Chairman and CEO, took time to answer questionsabout the company. Q: What do you mean by the word "serious"? Aberman: According to the FDA a serious adverse event (SAE) in humandrug trials is defined as any untoward medical occurrence that, atany dose, results in death, is life-threatening or requires inpatienthospitalization. Q: So did the patient have anaphylactic shock? Aberman: The patient did not go into anaphylactic shock and her vitalsigns were stable from the time she was first seen by medicalprofessionals and during her short hospitalization. Q: Does this mean that most likely you have to run an additionalsafety study in US? Aberman: No, we will do the risk assessment based on the FDA'squestions, investigate what happened and continue the phase II studyonce the FDA is satisfied with our responses. Q: What exactly is involved with a clinical hold? Does this affectany of your other trials? Aberman: A clinical hold means that we will not recruit additionalpatients into the US study until the FDA lifts the hold. This doesnot affect the other trials, namely our muscle injury trial orintermittent trial, both in Germany. What are the principal goals of the IC study (I have theclinicaltrials.gov description) and what is the proposed timetable? Aberman: The principal goals of the IC Phase 2b, dose escalationstudy is to evaluate the safety and efficacy of three different doses of our PLacental Expanded (PLX) cells in the treatment ofIntermittent Claudication (IC), a subset of peripheral artery disease(PAD). We anticipate that enrollment to the study will be completedduring H1/2014. Q: Any thoughts on how the clinical hold might affect that timetable? Aberman: We were notified by our regulator representative workingwith the FDA about the clinical hold in the US due to a seriousallergic reaction. According the FDA guidelines, within 30 days wewill receive written notification that will include all the questionsand clarifications needed. We have enjoyed an excellent workingrelationship with the FDA for several years and have receivedvaluable assistance on more than once occasion as we work together tomake cell therapy a reality. We will work continue to fullycooperation with the FDA, as we have in the past, to resume theclinical trial. We hope that in the upcoming weeks we will clarify with the FDA all their issues in order to continue our trial in theUS as planned. Hopefully there will be a minimal, if at all,influence on the trials' timetable. Q: Given the extensive exclusion criteria and the fact that thecompany has not previously experienced a serious allergic event withPLX, any thoughts as to what the company will examine first in thiscase? Aberman: The patient suffered from serious background diseases andwas chronically oxygen dependent. Approximately 12 hours followingthe intramuscular treatment with our PLX cells, the patient noted arash and experienced shortness of breath. She was treated in thehospital and released approximately 12 hours after admissionfollowing the resolution of all her symptoms. We are investigating tosee if there are connections between her reaction and our treatment. Q: Also, what is the immediate impact, if any, of the clinical hold on the company's other development activities? Aberman: The only trial effected by the hold is the PLX-PAD phase IIIntermittent Claudication (IC) study and the CLI study in the U.S.There is no impact on the PLX-PAD other clinical trials outside theUS underway or planned at this time. Q: Finally, is there any additional information/background that wouldhelp our readers understand the PLX technology, the IC study or theeffect of the clinical hold? Aberman: Pluristem's PLX cells are mesenchymal-like adherent stromalcells (ASCs) derived from full term human placentas. These cells areexpanded in the company's proprietary bioreactor system that createsa three-dimensional (3D) microenvironment. This 3D technology allowsfor the controlled, large-scale growth of cells implementing anoptimized, standardized, scaled-up and "hands-off" operation. Thisallows PLX cells to be mass-produced with batch-to-batch consistency in an effective and controllable process. Furthermore, Pluristem's 3Dexpansion technology allows for the production of specific PLX cellproducts for each of the Company's targeted indications.