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Proprietary data of Pluristem Therapeutics Inc.1 Advancing cell therapeutic products for clinical use.

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Presentation on theme: "Proprietary data of Pluristem Therapeutics Inc.1 Advancing cell therapeutic products for clinical use."— Presentation transcript:

1 Proprietary data of Pluristem Therapeutics Inc.1 Advancing cell therapeutic products for clinical use

2 Proprietary data of Pluristem Therapeutics Inc.2 This presentation concerning Pluristem Therapeutics may include forward-looking statements which represent Pluristem Therapeutics' expectations or beliefs regarding future events. I caution that such statements involve risks and uncertainties that may cause actual results to differ materially from those in the forward-looking statements. Consequently, all such forward-looking statements are qualified by the cautionary language and risk factors set forth in Pluristem Therapeutics' periodic and other reports filed with the SEC. There can be no assurance that the actual results, events or developments referenced in such forward-looking statements will occur or be realized. Pluristem Therapeutics assumes no obligation to update these forward-looking statements to reflect actual results, changes in assumptions or changes in factors affecting such forward-looking statements. www.pluristem.com Forward Looking Statement

3 Proprietary data of Pluristem Therapeutics Inc.3 Cell therapy (Biotechnology) company. Using off the shelf placental expanded cells to achieve local and systemic therapeutic effect. First in class 3D cell culturing technology allowing for efficient, controlled production of different cell products in commercial quantities. Active with regulators in USA, EU, South Korea, Australia and Israel. Pluristem Corporate Overview

4 Proprietary data of Pluristem Therapeutics Inc.4 Public company, Traded in: Strong balance sheet Market Cap: ~ $100 million Cash and marketable securities: $47 million (September 30, 2015) No debt Net burn: ~ $22 million No royalty liability (other than Israeli government) 165 employees (16 PhD, 4 MD) IP Ownership: over 50 granted patents and ~150 pending applications Financial Glance PSTI PSTI/ PLTR

5 Proprietary data of Pluristem Therapeutics Inc.5 PartnerIndication Deal structure IC, CLI South Korea only Joint Venture following marketing authorization by the South Korean authorities Pluristem keeps IP and manufacturing rights in all collaborations Collaborations Government Partnership Indication Deal structure Acute Radiation Syndrome U.S. National Institutes of Health (NIH) to Support Development of PLX-R18 Acute Radiation Syndrome Pluristem will contribute cells and scientific knowledge, FMU will conduct the studies and provide the required resources.

6 Proprietary data of Pluristem Therapeutics Inc.6 The PLX Technology

7 Proprietary data of Pluristem Therapeutics Inc.7 Placenta derived cells Rich & Diverse Highly potent - pro-angiogenic - immunoregulatory Young donors Unlimited source Easy to collect Ethically accepted Over 20,000 Doses of 300 million cells per placenta The Placenta Project- Launched by the US National Institutes of Health (NIH) to Further explore the role of the placenta in health and disease.

8 Proprietary data of Pluristem Therapeutics Inc.8 3D manufacturing 150,000 doses annually

9 Proprietary data of Pluristem Therapeutics Inc.9 Culture conditions PLX-R18 Hematological PLX-PAD Angiogenesis Culture conditions VEGF IL-10 IL-6 bFGF BDNF TGFβ MMP-9 NGF PLX-CNS Neuronal PLX-IMMUNE Immunological Human Placenta- A platform for cell products Culture conditions

10 Proprietary data of Pluristem Therapeutics Inc.10 Broad platform with tailored products per indication PLX cells can be customized to secrete a specific cytokine secretion profile to target different indications Real off the shelf product requiring no additional manipulation at bedside Unique Immunological Properties No HLA- matching required Low immunogenicity confirmed : PLX does not induce in vivo priming of Th1 responses Repeated IM injections of PLX cells from the same placenta does not induce patients memory T-cells activation. Distinctive MOA Products demonstrate both local and systemic efficacy via intramuscular injection How is PLX Different from the Competition

11 Proprietary data of Pluristem Therapeutics Inc.11 Superior Manufacturing In-house manufacturing which guarantees batch to batch consistency and high margins Near Term Product Approval First product approval expected 2018 for CLI How is PLX Different from the Competition

12 Proprietary data of Pluristem Therapeutics Inc.12 Product 1: PLX-PAD Reduces inflammation Stimulates growth of collateral blood vessels Stimulates repair f damaged muscle

13 Proprietary data of Pluristem Therapeutics Inc.13 Demonstrated clinical safety and significant efficacy in 3 clinical studies (Two Phase I and one Phase I/II study) Muscle Injury following Total Hip Replacement (N=20) PLX-PAD- Strong Clinical Data Improvement of 500% P=0.0067 Injured (operated) Contralateral (non–operated)

14 Proprietary data of Pluristem Therapeutics Inc.14 Demonstrated clinical safety and significant efficacy in 3 clinical studies (Two Phase I and one Phase I/II study) Two Phase I/II Critical Limb Ischemia (N=27) Pre-Treatment 8 Weeks After Treatment Pre-Treatment 8 Weeks After Treatment PLX-PAD- Strong Clinical Data 6-8 month follow-up

15 Proprietary data of Pluristem Therapeutics Inc.15 PLX PAD in CLI via new regulatory pathways European initiative intended to grant earlier access to drugs meant to treat debilitating and/or life-threatening diseases with unmet medical need. May 2015 - PLX-PAD cells have been selected for the project. Manufacturing facility approved and inspected by European Qualified Person for Phase III and marketing. Earlier entry in a multi-billion market (2018)- Conditional marketing approval in selected population of CLI patient following Initial Phase II study, then post- marketing extension to more CLI patients. Pluristem intends to expand development of PLX-PAD to additional indications via the Adaptive Pathway project Pluristem is committed to provide early access of PLX-PAD to Critical limb ischemia (CLI) patients all over the world Europe - Adaptive Pathways pilot project 1.7 M patients in the major pharmaceutical markets*. Expected to reach 2M in 2020. Two Phase I data with PLX-PAD in CLI patients showing good safety profile and efficacy. Safety data from 150 patients enrolled into IC study to support initial approval in CLI. *U.S., France, Germany, U.K., Italy, Spain and Japan. Phase II Trial* Conditional MA* 2016 2017 20182019 20202021 Collection of real-world Data from treated patients** Full MA Phase II Trial Two Phase III Trials Full MA Adaptive Pathways Traditional Pathway MA = marketing authorization *Can start marketing product in restricted, niche population **Additional studies in broader population of CLI patients Target 2018 Product Launch 2021 Traditional Product Launch

16 Proprietary data of Pluristem Therapeutics Inc.16 New Japanese regulations to accelerate development of drugs in the field of regenerative therapy. Conditional, time-limited approval for marketing and reimbursement upon proof of safety and a signal of effectiveness. No formal Phase III trial is expected to be required. April 2015- PLX-PAD quality standards and large-scale manufacturing methods accepted by PMDA. August 2015- PLX-PAD cells cleared by PMDA for use in clinical trials in Japan. Pending approval of clinical study design to initiate a Phase II study during H1 2016. Japan - Accelerated Pathway for Regenerative Medicine PLX PAD in CLI via new regulatory pathways Pluristem is committed to provide early access of PLX-PAD to Critical limb ischemia (CLI) patients all over the world 1.7 M patients in the major pharmaceutical markets*. Expected to reach 2M in 2020. Two Phase I data with PLX-PAD in CLI patients showing good safety profile and efficacy. Safety data from 150 patients enrolled into IC study to support initial approval in CLI. Conditional MA** Collection of data from treated patients*** Full MA Additional Trials to Confirm Safety & Efficacy Full MA Pathway for Regenerative Medicine Traditional Pathway Initial Clinical Study Exploratory Study* MA = marketing authorization * If results likely to predict efficacy and confirm safety then conditional, time-limited marketing authorization granted; no confirmation of efficacy needed. ** Time limit of 7 years till full MA or withdrawal ***Post-marketing safety-monitoring, surveillance and studies Initial Clinical Study Target Early Product Launch Traditional Product Launch

17 Proprietary data of Pluristem Therapeutics Inc.17 Product 2: PLX-R18 Stimulates regeneration of damaged bone marrow to produce blood cells (white, red and platelets) PLX-R18 Pipeline:

18 Proprietary data of Pluristem Therapeutics Inc.18 PLX-R18 - Targeting Bone Marrow Failure Main clinical manifestations of low blood counts:  Neutropenia: recurrent or severe infections  Anemia: fatigue, shortness of breath, pallor, tachycardia  Thrombocytopenia: bruising, purpura, petechiae, bleeding Bone Marrow Transplant Failure / Incomplete Engraftment  FDA IND application approved for Phase I study in the US Acute Radiation Syndrome (ARS) – Supported by the NIH (NIAID)  Animal Rule pathway  Two significant animal trials announced  Preparing to submit protocol for large animal trial Potential indications include- Chemotherapy, Aplastic Anemia (AA), Acute myeloid leukemia (AML), Myelodysplastic syndrome (MDS)

19 Proprietary data of Pluristem Therapeutics Inc.19 U.S. National Institutes of Health to Support Development of Pluristem's PLX-R18 Collaboration for ARS with US Government

20 Proprietary data of Pluristem Therapeutics Inc.20 CMC & Manufacturing Facility for PLX-PAD approved by FDA, German, EU, South Korean, Japanese & Israeli Regulatory Agencies for 3D culturing for Phase II, III trials and marketing

21 Proprietary data of Pluristem Therapeutics Inc.21 IC CLI- Europe CLI- Japan Orthopedic Indication PAH Preeclampsia Hematology ARS End of enrollment Proprietary data of Pluristem Therapeutics Inc.21 Company milestones H1-2016H2-2016H1-2017H2-2017H1-2018H2-2018 indication End of enrollment Data Readout Licensing deal Study initiation Initial approval Data Readout End of enrollment Data Readout Study initiation Conditional approval We will seek to include the indication under the Adaptive licensing pathway- based on discussion with EMA Pivotal study Data Readout BLA application Conducting additional pre-clinical studies based on discussions with FDA Product PLX- PAD IND Study initiation End of enrollment Data Readout Adaptive or phase II/III PLX-R18 End of follow up Cohort 2

22 Proprietary data of Pluristem Therapeutics Inc.22 Zami Aberman Chairman & CEO Efrat Livne-Hadass VP Human Resources Racheli Ofir, Ph.D. VP Research & Intellectual Property Sagi Moran VP Operations Erez Egozi VP Finance Karine Kleinhaus, M.D., MPH Divisional VP, North America Yaky Yanay President & COO Hillit Mannor Shachar, M.D., M.B.A. VP Business Development Lior Raviv Director of Development Esther Lukasiewicz Hagai, M.D., Ph.D. VP Clinical & Medical Affairs Orly Amiran VP Quality Assurance Management Team

23 Proprietary data of Pluristem Therapeutics Inc.23


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