FUNCTIONAL STEM CELL SELECTION Corporate Presentation Sept 2015
Forward-Looking Statements This presentation includes information that was included in the Company’s immediate reports published to the public. This presentation does not consist of any offer to purchase or sell Company securities or an invitation to receive such offers. Please be advised that the information and projections provided in this presentation may include forward-looking statements with respect to plans, projections or future performance of the Company, the occurrence of which involves certain risks and uncertainties, some of which may not be under the control of the Company, including, but not limited to, changes in the regulatory environment, the Company’s success in implementing its research, development, sales, marketing and manufacturing plans, protection and validity of patents and other intellectual property rights, the impact of currency exchange rates and the effects of competition.
Additionally, please be advised that certain solutions or applications for the Company’s products, included in this presentation, may not yet be commercially available and / or regulatory cleared for marketing. All trademarks and registered trademarks are the property of their respective owners.
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Snapshot •
Established in 2011, Cellect develops commercial medical applications on the basis of functional cell selection technology
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Technology based on 15 years of academic research and is protected by several patent families in the U.S., Europe, Israel and elsewhere; additional patents are in the approval process
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Enables functional selection of a wide variety of stem cells, with many potential applications
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First product under development is a medical device for the bone marrow transplant market (as well as for other markets in which there is a need for stem cell selection)
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Extensive pre-clinical trials in animals and in human cells; Cellect is currently preparing for first in-human trials
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Business Strategy •
Focused on achieving a relatively quick validation of the technology in a significant market
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Cellect has chosen an application that reduces risk in bone marrow transplant for leukemia patients
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In parallel to validating the technology, Cellect is developing a wide range of applications for a broad spectrum of market segments, up to and including all production and research processes for stem cell based products
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“Cellect Inside” is a strategy that addresses a large market as an enabling technology, similar to the model of Abgenix, Medarex in the area of protein humanization
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What are Stem Cells? • Capable of differentiating into different types of tissues and organs • The basic building block of the healing and regeneration process • A rare cell that is difficult to characterize and identify • The hottest biotechnology field in the coming decades, with potential for a wide variety of applications
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Hematopoietic Stem Cell Applications • Hematopoietic stem cells are blood and lymph-forming cells
• Have been used medically in bone marrow transplants for over 50 years • The primary source of stem cells for a wide variety of medical applications
Blood Cancer
Autoimmune Diseases
Central Nervous System Disorders
Parkinson’s Disease
Hematopoietic Stem Cell Applications
Heart Failure
Vascular Disease
Myocardial Infarction
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Bone Marrow Donation is Life and Death “Bone Marrow” Donation
Large quantity of mature blood cells
Small quantity of stem cells
• Implant Rejection • GvHD (Graft versus Host disease) is a lethal disease in which the donor cells attack the body of the recipient
A new immune system is generated in the body of the recipient
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Cell Selection for Bone Marrow Transplants Laboratory Procedure Morpological Cell Selection Ineffective & non-toxic implant
Donor Sample
Effective & highly toxic implant
Transplantation into patient
• Lab procedure used today aims to lower the frequency of immune response • Procedure is complex and expensive and not sufficiently efficient – with a high risk for GvHD and for tumor recurrence
• There is an unmet medical need for an efficient and safe implant which contains primarily stem cells 8
The Cellect Solution Functional Selection of Cells for Bone Marrow Transplantations
Apoptosis* Signal (e.g. FasL**)
Stem Cells – Resistant to Apoptosis
Mature Cells
* Programmed cell death ** Apoptosis-inducing protein
Apoptotic T Cell
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Functional Stem Cell Selection
Wide variety of research applications
Wide variety of research applications
Bone Marrow Transplantations
Immune Deficiency
Cardiac Diseases
Nervous System Disorders
Mesenchymal Stem Cells
And more …
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CL101: Stem Cell Selection Kit • Off-the-shelf medical device
Current Procedures
CL101
60 – 80%
0 – 10%
• Quick, simple procedure
GvHD
• No need for special lab
Chemotherapy
Wide
Minimal
• Cost-effective
Rate of Infection
High
Low
Procedure and hospitalization
Months
Several Days
Procedure Cost
~ $70,000
Significantly lower
Total cost of procedure
~ $300,000
Significantly lower
• Safe, simple, efficient
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CL101: Product Family for Cell Selection
Various containers (infusion bag, test tube) with an apoptotic environment for cell selection for bone marrow transplantations
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Summary of Pre-clinical Results •
Significant reduction of cells that attack the immune system
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Reduction of GvHD morbidity and mortality (20 – 80% to less than 10%)
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Increase in the percentage of stem and progenitor cells in the sample
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Preservation of successfully engraftment and anti-tumor activity
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Significantly shorter lab procedure time
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Low procedure costs
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Binding Apoptotic Protein to Polymer Process
• Selective apoptosis = engagement improvement
• Successful binding of apoptotic protein (FasL) to plastic surface, without loss of efficacy Product (CL101)
• Direct correlation observed between the quantity of protein that was bound and the success of the apoptotic process
Stem Cell Mature Cell Death Signal
Basis established for developing the offthe-shelf product
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R&D Work Plan • Complete product design and build prototype (Q4, 2015) • Regulatory submissions to initiate clinical trials (Q4, 2015)
• Initiate first in-human trial (H1, 2016) 15
Core Team Dr. Shai Yarkoni (MD, PhD), CEO A senior executive in the biotechnology industry. Founder and CEO of Target-In Ltd., a company in the cancer bio therapeutics field. Founded and until recently, managed Bio-Negev, a biotechnology and life sciences cluster.
Nadir Askenasy (MD, PhD), Chief Scientist Leads research and clinical activity in cellular therapies. Has authored more than 100 peer-reviewed publications, serves on editorial boards of stem cell and medical journals, and has served on the Advisory Committee of medical institutes worldwide. Principal investigator at Frankel Laboratory of Experimental Bone Marrow Transplantation, and Schneider Children’s Medical Center of Israel. Performs clinical work at International Center of Cell Therapy.
Jaron Diament, CFO More than 20 years of financial experience in public and private companies in Israel and abroad, in roles that include VP Finances, CEO, and director. Extensive experience in investments and fundraising in Israel and abroad.
Kasbian Nuriel Chirich, Chairman Businessman with extensive financial and business expertise, and highly experienced with innovative ventures. Leads several ventures in East Africa and Israel and is the Honorary Consul of Tanzania in Israel.
Professor Dov Zipori, Advisory Board Director of the Martin and Helen Kimmel Stem Cell Research Center at the Weizmann Institute. Research was the scientific basis of Pluristem (NASDAQ:PLX).
Dr. Susan Alpert, Advisory Board, External Consultant Previously Head of Medical Device division at the FDA and EVP of Regulatory Affairs at both Medtronics (MDX) and CR BARD. 16
Opportunity Is Well Protected Cellect Biotechnology owns multiple patents that protect its technology platform and products • Granted a license for complementary patents from University of Louisville (USA) • Patent for the concept of using apoptosisinducing agents
• Patent for special-purpose containers and their use • Patents covering the methods of use • Patents for binding methods and structures of polymer + apoptosis compounds 17
In Summary •
Attractive field at the cutting edge of medical science
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Strong intellectual property
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Experienced management team and advisors
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Proof-of-concept for multiple treatments with significant market potential
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Improvement of a recognized procedure with clear unmet medical need
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Strategy focused on fast time-to-market 18