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Strictly private and confidential information 1. This presentation was prepared for the sake of summary and convenience only and it cannot replace a reviewing.

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Presentation on theme: "Strictly private and confidential information 1. This presentation was prepared for the sake of summary and convenience only and it cannot replace a reviewing."— Presentation transcript:

1 Strictly private and confidential information 1

2 This presentation was prepared for the sake of summary and convenience only and it cannot replace a reviewing of the prospectus and/or the periodic report of Kadimastem Ltd. (hereinafter: “the Company”). This presentation contains projections, estimates and assessments, as are known to the Company at the time of preparation of this presentation, referring to the Company and including, inter alia, forward-looking information as defined in the Securities Law, 5728 – 1968, based on subjective estimates on the part of the Company in respect of its development potential and based on initial information and documents the Company received from professional entities relevant to the Company’s development plans. Forward-looking information is uncertain and mostly is not under the Company’s control and the realization or non-realization of forward-looking information will be affected, among other things, by the risk factors characterizing the Company’s activity, as well as developments in the general environment and external factors affecting the Company’s activity. The Company’s results and achievements in the future may differ materially from those presented in this presentation and the Company makes no undertaking to update or revise such projection or estimate and does not undertake to update this presentation. This presentation does not constitute a proposal to purchase the Company’s securities or an invitation to receive such offers. Investment in securities in general and in the Company in particular bears risks. One should take into account that past performance does not necessarily indicate performance in the future. 2 Disclaimer

3 Strictly private and confidential information Vision Utilize innovative stem cell-based therapies to treat incurable diseases 3

4 Strictly private and confidential information Company Snapshot 4  Established: 2009  Founders: Yossi Ben Yossef and Professor Michel Revel (developer of Merck Serono’s blockbuster drug REBIF ® )  Differentiate stem cells into a range of functioning cells for:  Regenerative Medicine – using differentiated cells to cure diseases  Drug Screening – using differentiated cells to test effects of compounds  Agreement with the Merck Serono for drug screening for multiple sclerosis (MS)

5 Strictly private and confidential information Company Snapshot 5  1,700 m² (18,300 ft²) labs and offices including two clean rooms  30 employees (9 PhD’s)  Experienced Management with extensive biotech experience

6 Strictly private and confidential information Business Model The drug screening engine reduces dependence on outside funding 6

7 Strictly private and confidential information What Are Stem Cells 7 A Cell with a potential to… Self renew Differentiate into a variety of cells

8 Strictly private and confidential information Our Platform Technologies 8 Insulin producing beta-cells Brain supporting (glia) and neuronal cells Accurate and reproducible Large scale production capabilities hESC and iPS cells Reproducible Expansion Differentiation  The basis for both drug screening and regenerative medicine activities  Highly skilled team can transition between activities as needed

9 Strictly private and confidential information Commercial Applications 9 Platform Technologies Regenerative Medicine Drug Screening Existing project Additional diseases Goals ALS (Merck Serono) MS HD Diabetes ALS

10 Strictly private and confidential information Diabetes 10  A group of diseases characterized by excessive levels of blood sugar  Insulin – a hormone secreted from beta cells in the pancreas  The absence of insulin causes an excessive blood glucose level that causes cumulative damage Lack of insulin production or the inability to use it Inability to produce insulin Source:

11 Strictly private and confidential information Diabetes – Target Market m patients worldwide (type 1 diabetes patients +~30% of type 2 diabetes patients - insulin dependent) ~30 million in developed world Global Epidemic (WHO) The global market for diabetes management is expected to reach $114 billion in 2016* Source: the American Diabetes Society (diabetes.org), the World Health Organization (who.int), the International Diabetes Federation (idf.org) and a market study conducted by the MedMarket Diligence company from 2010 )http://www.mediligence.com/rpt/rpt-d510.htm); also care-education/about-diabetes/type-2-diabetes-faqshttp://www.diabetes.ucsf.edu/clinical- care-education/about-diabetes/type-2-diabetes-faqs *

12 Strictly private and confidential information Diabetes – Existing Treatments Monitoring glucose levels and injecting insulin accordingly Insulin injections do not prevent the cumulative damage of the disease Difficulty in balancing diabetic patients Dangerous states of diabetes (hyperglycemia) and “sugar level drops” (hypoglycemia) Transplantation of beta cells from cadaveric organ donors  Feasibility proof of concept regarding treatment using beta cells transplant  Shortage in sufficient organ donations  Requires long-term immunosuppression 12

13 Strictly private and confidential information Production and transplantation of stem cell-derived islet-like structures in patients:  Kadimastem produces islet-like clusters that show glucose-stimulated insulin secretion.  Restoring the patient’s ability to produce and release insulin and glucagon  Obviates the need for monitoring blood sugar levels and insulin injections  Eliminates the life threatening consequences of diabetes  Significant improvement of patients’ life expectancy and quality of life 13 Diabetes – Our Solution

14 Strictly private and confidential information Diabetes – Our Solution Stem Cell Expansion Differentiation into Beta Cells Sorting of Beta Cells Re-aggregation into Islet-like structure EncapsulationImplantation

15 Strictly private and confidential information Commercial Applications 15 Platform Technologies Regenerative Medicine Drug Screening Existing project Diabetes Additional diseases Goals ALS (Merck Serono) MS HD (Merck Serono) ALS

16 Strictly private and confidential information ALS 16  A rapidly progressive and fatal neurological disease  Attacks neurons responsible for controlling muscles (motoneurons)  Leads to loss of muscle control  Disease does not impair a person’s mind Lou Gehrig

17 Strictly private and confidential information ALS – Target Market 17 ~150,000 patients ww ~50,000 diagnosed each year ~137 diagnosed each day Annual ALS healthcare cost in US alone estimated at $6 billion * The numbers increase as population ages Refers to US, Canada, Europe and Australia. *

18 Strictly private and confidential information  Medications : Rilutek® - the first and only medication approved by the Food and Drug Administration for slowing ALS  Appears to slow the disease's progression in some people  Physical therapy  Occupational therapy  Speech therapy ALS - Existing Treatments 18

19 Strictly private and confidential information  Studies have indicated the involvement of malfunctioning brain- supporting cells (astrocytes) in ALS  Cells become toxic to Motoneurons  Reduced levels of brain’s “well-being”  Our therapeutic approach:  Increase neuronal survival by transplanting functional human astrocytes into ALS patients ALS – Our Approach 19

20 Strictly private and confidential information Therapeutics Development Strategy Advance ALS until end of in-vivo trials, in parallel to Diabetes Go/No-Go decision point - Q2 2015: – Positive in-vivo results in ALS  continue to advance program and slow down Diabetes – Negative in-vivo results in ALS  continue to advance Diabetes and slow down ALS

21 Strictly private and confidential information ALS Development Timeline In-vivoIND Clinical Trials Q2 ‘15 $1 M Q2 ‘19 Q2-Q3 ‘16 $5 M Add Strategic Partner Go/No-Go (Diabetes or ALS) Q1 ‘14 It is hereby clarified, that the aforementioned assessments, regarding the expected progress of the Company's R&D in the field of ALS is considered prospective information, as defined in the Securities Law, which is based, inter alia, on current quotations, Company's working plan and acquaintance with the regulatory requirements to this date. This information might not realize and the actual results might be substantially different, inter alia, as a result of lack of proper funding for progress in the desired scope of activity, failure of pre-clinical and/or clinical experiments, delay of schedule, regulatory changes in countries where the Company plans to conduct the experiments, etc.

22 Strictly private and confidential information Diabetes Development Timeline In-vivo trialsIND Clinical Trials Q4 ‘20 Q4 ‘17 $7 M Q2 ‘16 $1.5 M Add Strategic Partner It is hereby clarified, that the aforementioned assessments, regarding the expected progress of the Company's R&D in the field of diabetes is considered prospective information, as defined in the Securities Law, which is based, inter alia, on current quotations, Company's working plan and acquaintance with the regulatory requirements to this date. This information might not realize and the actual results might be substantially different, inter alia, as a result of lack of proper funding for progress in the desired scope of activity, failure of pre-clinical and/or clinical experiments, delay of schedule, regulatory changes in countries where the Company plans to conduct the experiments, etc.

23 Strictly private and confidential information Increase in Early-Stage Deals 23 Early Stage Deal Valuation Picks Up, But Not Late Stage $80 $60 $50 $4 $30 $20 $70 $10 $0 $100$200 $400 $500 5 year average 2013 Preclinical Average Upfront Total Deal Value $80 $60 $50 $40 $30 $20 $70 $10 $0 $100$200 $400 $500 5 year average 2013 Phase I Average Upfront Total Deal Value $80 $60 $50 $40 $30 $20 $70 $10 $0 $100$200 $400 $500 5 year average 2013 Phase III Average Upfront Total Deal Value $80 $60 $50 $40 $30 $20 $70 $10 $0 $100$200 $400 $500 5 year average 2013 Phase II Average Upfront Total Deal Value Source:

24 Strictly private and confidential information Commercial Applications 24 Platform Technologies Regenerative Medicine Drug Screening Existing project Diabetes ALS Additional diseases Goals (Merck Serono) MS HD ALS

25 Strictly private and confidential information  Production of specific, relevant and functional cells from stem cells  Creating simplified models for drug activity, automatically quantified by imaging based system  Testing how potential drugs affect the cell functionality A stem cell colony Neuro-glia progenitor cells Drug Screening Differentiated cells are affected (or not) by the drug 25

26 Strictly private and confidential information Collaboration with Merck Serono  Goal: Using Kadimastem’s specialized cells for MS drug discovery  The agreement: 1) A non-exclusive collaboration 2) Merck Serono provides potential drugs  4/2012 – Signed drug screening agreement focusing on MS  11/2013 – Signed MOU to advance collaboration to include:  $ 1M grant from Merck Serono  Optimizing screening capabilities for MS drugs  Development of screening capabilities for ALS and Huntington’s Diseases 26

27 Strictly private and confidential information Patents 27 Diabetes Therapeutics ALS Therapeutics Drug Screening Stem Cell Enabling Technologies

28 Strictly private and confidential information 28  Founded Kadimastem in 2009  12 years of experience in leading investments and building start-up companies in the field of life sciences  Investment and active involvement in Evogene, VBL, Radvision, Rosetta Genomics, BiondVax, Comfi, Wisecom Yossi Ben Yossef – CEO  Founded Kadimastem in 2009  Professor Emeritus of molecular genetics at the Weizmann Institute  Israel Prize laureate  Over 30 years of experience in development and global commercialization of advanced biotechnological products  Developed Merck’s REBIF® drug for multiple sclerosis ($2.2B USD in sales in 2011) Professor Michel Revel – Chief Scientist Management Team

29 Strictly private and confidential information Milestones  Milestones  Initiate in-vivo studies in a diabetic model  Initiate in-vivo studies in a SOD1 ALS animal model  Sign agreements with Pharma companies (MOU Merck Serono )  ALS – Complete animal testing Q  Diabetes - complete in-vivo studies on immune-deficient animals– Q  Diabetes - complete in-vivo studies on immune-competent animals– Q √ √ √

30 Strictly private and confidential information Strategy  Strategy  Shorten time and cost by licensing and mergers  Adding strategic partner for the development of diabetes and ALS therapies  Drug screening agreements with additional drug companies 30

31 Strictly private and confidential information Take home messages  Developing drugs to improve patients’ life quality & expectancy  Therapeutic programs with blockbuster potential (Diabetes, ALS)  Commercial agreement with Merck-Serono  Highly experienced executive team (CSO developer of Rebif ® ) 31

32 Strictly private and confidential information 32 Thank you


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