Transcript Drug Screening

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Disclaimer
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.
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Vision
Utilize innovative stem cell-based therapies to
treat incurable diseases
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Company Snapshot
 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)
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Company Snapshot
 1,700 m² (18,300 ft²) labs and offices including two clean rooms
 30 employees (9 PhD’s)
 Experienced Management with extensive biotech experience
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Business Model
The drug screening engine reduces dependence on outside
funding
Drug
Screening
Regenerative
Medicine
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What Are Stem Cells
Self renew
A Cell with a potential
to…
Differentiate into a variety of cells
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Our Platform Technologies
• Large scale production capabilities
• hESC and iPS cells
• Reproducible
Expansion
• Insulin producing beta-cells
• Brain supporting (glia) and neuronal cells
• Accurate and reproducible
Differentiation
 The basis for both drug screening and regenerative medicine activities
 Highly skilled team can transition between activities as needed
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Commercial Applications
Diabetes
Regenerative
Medicine
ALS
Additional
diseases
Platform
Technologies
MS
(Merck Serono)
Drug
Screening
ALS
HD
Existing project
Goals
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Diabetes
 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
Inability to
produce insulin
Source: http://www.idf.org/worlddiabetesday/toolkit/gp/facts-figures
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Lack of insulin production
or the inability to use it
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Diabetes – Target Market
137m 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 http://www.diabetes.ucsf.edu/clinicalcare-education/about-diabetes/type-2-diabetes-faqs
* http://www.transparencymarketresearch.com/diabetes-devices-market.html
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Diabetes – Existing Treatments
Monitoring glucose levels and injecting insulin accordingly
•
Insulin injections do not prevent the cumulative damage of the disease
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Difficulty in balancing diabetic patients
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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
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Diabetes – Our Solution
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
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Diabetes – Our Solution
Stem Cell
Expansion
Differentiation
into Beta Cells
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Sorting of
Beta Cells
Re-aggregation Encapsulation
into Islet-like
structure
Implantation
Commercial Applications
Diabetes
Regenerative
Medicine
ALS
Additional
diseases
Platform
Technologies
MS
(Merck Serono)
Drug
Screening
ALS
(Merck Serono)
HD
Existing project
Goals
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ALS
 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 1903 - 1941
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ALS – Target Market
~150,000 patients ww
~50,000 diagnosed each year
~137 diagnosed each day
The numbers increase as population ages
Annual ALS healthcare cost in US alone
estimated at $6 billion *
Refers to US, Canada, Europe and Australia. http://www.alsa.org/als-care/resources/publications-videos/factsheets/epidemiology.html
* http://alliancerm.org/disease/als-and-regenerative-medicine
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ALS - Existing Treatments
 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
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ALS – Our Approach
 Studies have indicated the involvement of malfunctioning brainsupporting 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
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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
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ALS Development Timeline
Add Strategic Partner
Go/No-Go
(Diabetes or ALS)
In-vivo
Q1 ‘14
IND
Q2 ‘15
$1 M
Clinical Trials
Q2-Q3 ‘16
$5 M
Q2 ‘19
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.
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Diabetes Development Timeline
Add Strategic Partner
In-vivo trials
Clinical
Trials
IND
Q2 ‘16
$1.5 M
Q4 ‘17
$7 M
Q4 ‘20
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.
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Increase in Early-Stage Deals
Early Stage Deal Valuation
Picks Up, But Not Late Stage
$80
$80
Preclinical
$60
$50
$4
5 year
average
$30
2013
$20
$10
$0
$60
$50
$40
5 year
average
2013
$30
$20
$10
$0
$0
$100
$200
$400
Total Deal Value
$500
$100
$200
$400
Total Deal Value
$0
$80
$500
$80
Phase I
$60
$50
$40
5 year
average
$30
Phase III
$70
2013
$20
$10
$0
Average Upfront
$70
Average Upfront
Phase II
$70
Average Upfront
Average Upfront
$70
$60
$50
$40
5 year
average
2013
$30
$20
$10
$0
$0
$100
$200
$400
Total Deal Value
$500
$0
Source: http://www.partnering360.com/insight/showroom/id/429
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$100
$200
$400
Total Deal Value
$500
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Commercial Applications
Diabetes
Regenerative
Medicine
ALS
Additional
diseases
Platform
Technologies
MS
(Merck Serono)
Drug
Screening
ALS
HD
Existing project
Goals
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Drug Screening

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
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Neuro-glia progenitor cells
Differentiated
cells
are
affected (or not) by the drug
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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
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Patents
Stem Cell
Enabling
Technologies
Diabetes
Therapeutics
Drug
Screening
ALS
Therapeutics
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Management Team
Yossi Ben Yossef – CEO
 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
Professor Michel Revel – Chief Scientist
 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)
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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 Q2 2015
 Diabetes - complete in-vivo studies on immune-deficient animals–
Q2 2015
 Diabetes - complete in-vivo studies on immune-competent animals–
Q2 2016
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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
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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®)
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Thank you
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