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Enabling Regenerative Medicine Disruptive Stem Cell Technology

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Page 1: Enabling Regenerative Medicinecellect.co/assets/cellect---corporate-presentation-mar... ·  · 2018-03-07Global Transplantation Market ... biobanking o out licensing ... •14 Research

Enabling Regenerative Medicine

Disruptive Stem Cell Technology

Page 2: Enabling Regenerative Medicinecellect.co/assets/cellect---corporate-presentation-mar... ·  · 2018-03-07Global Transplantation Market ... biobanking o out licensing ... •14 Research

This presentation includes statements that are, or may be deemed, ‘‘forward-looking statements.” In some cases, these forward-looking statements can be identified by the use of forward-looking terminology, including the terms “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “approximately,” potential” or, in each case,their negative or other variations thereon or comparable terminology, although not all forward-looking statements contain these words. For example, forward-looking statements are used inthis presentation when we discuss the timing of our clinical trials, the potential for the Company’s technology to be used for various indications and applications, the potential advantages ofthe Company’s technology over its competitors, our future plans which may include licensing and our expected timeline for regulatory approval of our products and studies. We have basedthese forward‐looking statements on assumptions and assessments made by our management in light of their experience and their perception of historical trends, current conditions,expected future developments, and other factors they believe to be appropriate. Because such statements deal with future events and are based on our current expectations, they are subjectto various risks and uncertainties and actual results, performance or achievements could differ materially from those described in or implied by the statements in this presentation. In addition,historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to hereinwould be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in theforward-looking statements: our expectations regarding the timing of commencing clinical trials with respect to our ApoGraft process and our Apotainer selection kit; our expectationsregarding the progress of our clinical trials, including the duration, cost and whether such trials will be conducted at all; our intention to hold meetings with regulators and apply for regulatoryapproval for our product candidates, and the costs and timing of such regulatory approvals; the likelihood of regulatory approvals for our product candidates; the timing and cost of thedevelopments of our prototype Apotainer selection kit; our expectation to obtain a sufficient supply of FasL for our needs in the foreseeable future; the market size and future sales of ourproduct candidates or any other future products or product candidates; that our technology may potentially improve the safety and efficacy of regenerative medicine stem cell therapy andother potential advantages of our selection process for physicians, academics, researchers and others; our intention to expand our product development and build a diversified productportfolio of Apograft products for a broad spectrum of market segments; and our estimates regarding anticipated expenses, capital requirements and our needs for substantial additionalfinancing. By their nature, forward-looking statements involve risks and uncertainties because they relate to events, competitive dynamics, and healthcare, regulatory and scientificdevelopments and depend on the economic circumstances that may or may not occur in the future or may occur on longer or shorter timelines than anticipated. Although we believe that wehave a reasonable basis for each forward-looking statement contained in this presentation, we caution you that forward-looking statements are not guarantees of future performance and thatour actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements containedin this presentation as a result of, among other factors, the factors referenced in the “Risk Factors” section of contained in our most recent Annual Report on Form 20-F filed with theSecurities and Exchange. In addition, even if our results of operations, financial condition and liquidity, and the development of the industry in which we operate are consistent with theforward-looking statements contained in this presentation, they may not be predictive of results or developments in future periods. Any forward-looking statement that we make in thispresentation speaks only as of the date of such statement, and we undertake no obligation to update such statements to reflect events or circumstances after the date of this presentation.More detailed information about the risks and uncertainties affecting the Company is contained under the heading “Risk Factors” in the Annual Report and in the Company’s period filings withthe SEC.

Forward-Looking Statements

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Our Mission

Cellect aims at facilitating Stem Cell technologies to

reach the holy grail of medicine:

• stop fixing by chemicals, poisons, radiation and

massive surgery, and instead

• replace the damaged tissues and organs using

revolutionary Stem Cell technologies.

Cellect offers an enabling platform for Stem Cell

“cellection” and cost-effective mass production of

high quality Stem Cells.

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The Challenge • For Stem Cells to be used for regenerative

medicine, there is a need for large amounts of

Enriched and High Quality Stem Cells.

• Many companies with Stem Cell products

encounter challenges at large scale

manufacturing, leading to

- failures at later stage clinical trials and/or

- unacceptably expensive products.

4

Our Vision• Develop Cellect-owned safe, and cost-effective

products for specific Stem Cell treatments

• Propose Stem Cell companies an efficient, cost

effective and standardized selection process for Stem

Cells, enabling reproducible mass production of raw

material from any source for any indication.

“STEM CELL INDUSTRY INFLECTION POINT IS STANDARDIZATION AND CELLECT HAS THE SOLUTION” (Ray Dirks)http://www.raydirks.com/2017/10/18/stem-cell-industry-inflection-point-is-standardization-and-cellect-has-the-solution/

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Technology

• Cellect develops a technology enabling mass production of Stem Cells for pharma,

medical and research uses

• Strong IP portfolio with 7 patent families; 4 of which already granted in US

• Phase I/II clinical trial: First cohort successfully completed one month– no related adverse

effects

Strategy

• Does not compete with Stem Cell companies, but proposes enabling and standardization

of more effective, safer and less costly transplant products manufacturing.

• Seeking to enter into multiple licensing deals - initial revenues expected already in 2018

Management

• Seasoned professional management team

• KOLs on BOD and advisory board

Healthy Finances

• Sufficient cash to support current Phase I/II clinical trial and pre-clinical pipeline

development

5

Cellect –Investment OverviewNASDAQ:APOP

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Program I: Hematopoietic Stem Cells (HSCs) Bone Marrow Transplantations

Challenge Existing Product

and Process

Trade Off

(selection)

Cellect’s Solution – ApoGraftTM Technology

in the ApoTainerTM

Acute Graft vs Host

Disease (aGvHD)

in 30 - 50% of

patients

Stem Cell purification using

specific markers

- Low GvHD

- Failures of transplant

- Recurrence of cancer

(low GvL)

Design:

- Cellect’s ApoTainer comprising FasL induces cell death

(Apoptosis) of mature cells

- Results in significant GvHD reduction

- Full preservation of transplant

- Preserves anti-cancer activity (high GvL)

- Fast, safe and cost-effective

Non-toxic

&

ineffective

Effective

&

highly toxic

Cellect’s ApoTainerTM comprising

apoptosis-inducing molecules:

Negative Selection of GvHD-

inducing cells

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• The clinical score measures the severity of GvHD at the indicated times in irradiated transplanted NSG mice.• Each symbol represents the mean value ± SEM for 10 mice in each group.• MPBC = human mobilized peripheral blood cells• ApoGraft – MBPCs after incubation according to ApoGraft protocol with indicated FasL concentration

Efficacy Study:ApoGraft Improves GvHD Clinical Score

0 2 0 4 0 6 0 8 0 1 0 0

0

2

4

6

8

T im e (d a y s )

Cli

nic

al

sc

ore

M P B C s c o n t r o l

A p o G r a f t 2 5 n g / m l F a s L

A p o G r a f t 1 0 0 n g / m l F a s L

**

***

• Severe GvHD in mice injected with human

MBPCs, including high mortality

• Negligible GvHD detected with human

MBPCs injected after ApoGraft process

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Cellect’s ApoTainerTM vs. Alternatives

Current Procedures ApoTainerTM **

Risk of GvHD 30% - 50% Negligible

Chemotherapy Wide Reduced

Rate of Infection High Low

Procedure and

hospitalizationMonths Days

Cell Selection Cost ~ $70,000 < $10,000

Total cost of procedure ~ $300,000 < $100,000

** Company Estimates

• 2 hours simple bed-side procedure• No major change in SOC

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Program II: Mesenchymal Stem Cells (MSCs)Broad Applications in Stem Cell Therapies

Challenges Existing

Process

Trade Off

(expansion)

Cellect’s Solution - ApoGraftTM

Technology in the ApoTainerTM

- Fat-derived MSCs (ASCs)

expansion lengthy

- Major cell source for esthetic,

orthopedic and other indications

(> 500 non - regulated clinics in

USA) with recurrent failures due to

lack of standardization

- Few approved products –

challenging prices

- No selection

- Lengthy expansion

time (weeks)

- High cell numbers, but

also high cost

- Often loss of

“stemness” and

therefore reduced

efficacy

Design:

- Results in significantly higher cell numbers with

enhanced “stemness” in early passages

- Shorter expansion times

- Safe and cost-effective production

High

“stemness”

&

low yield

Low

“stemness”

&

high yield

Cellect’s ApoTainerTM

comprising apoptosis-inducing

molecules: death of mature

cells – Positive Selection of

Stem Cells

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Adipose Stem Cells (ASCs) – Expansion with FasL

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• FasL selects EARLY progenitor cells

• FasL induces proliferation of ASC

0

0.1

0.2

0.3

0.4

0.5

Control Fas-L

Ce

ll N

o. x

10

6

ASC Expansion

Large colony Small colony

Early

progenitor cellsprogenitor cells

0

10

20

30

40

50

60

Control Fas-L

No

. of

larg

e c

olo

nie

s

CFU-F Assay of Stem Cell Progenitor Cells

Dual effect drives improved cost-benefit for product manufacturing

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Cellect in The Stem Cells Value Chain

Raw

Material

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Highly effective

selection -

Reduced cost

Positive

Selection

HSC

MSC

Negative

Selection

Time Cost

Reduced

GvHD

Safety

Highly effective

expansion-

Reduced cost

Standardization

Efficacy

Quality

~2 hours

No change

in SOC

Few days

Shorter

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Stem Cells Enabling Technology – Fast Growing Market Potential

Yen-Shun et al. Int J Stem Cell Res Ther 2016, 3:029

Mesenchymal Stem Cells –

Clinical Trials

• Number of clinical trials with MSCs fast growing

• Large number of possible indications

• Largest number of trials for GvHD, Heart

Disease and Orthopedics, fields with Cellect

involvement

• All procedures starting with similar MSCs, all

potential candidates for Cellection

Hematopoietic Stem Cells –

Global Transplantation Market

• Fast growing market with estimated WW CAGR of

almost 12.8% until 2025 (TMR)

• Currently almost exclusive procedure in malignant

diseases

• Large number of potential non-malignant indications

(severe auto-immune diseases, metabolic diseases,

immune deficiencies etc.)

• Main challenge limiting outcome and broader use of

HSCT: cytotoxicity - GVHD12

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Scalable Versatile Product Platform

Research Clinic Industry

• Combination product (biological and device)

• From test tube to industrial-level bioreactor size

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Business Model & Strategy• Medical proof of concept – launch a consumable medical device with first indication –

substantially reduce GvHD

• Build a brand of quality (“Cellected”) to establish leadership position

• Leverage growing IP portfolio (7 patent families)

• Planned additional internal programs – development to product launch:

o POC indications

o in-licensing

• Technology Platform – Enter into strategic deals with pharma, biotech, research and

biobanking

o out licensing: upfront + milestone payments + royalties

o spin-offs

• Research collaborations for other cell selection technologies14

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Clinical Program

2016 2017 2018 2019 2020

Ph I/II GvHD (ISR)

ApoTainer Optimization

Non-interventional

MSC Preclinical

GvHD US study

ApoTainer Pivotal Study

Ph I/II MSC

ApoGraft

ApoTainer

MSC

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ApoTainer Comparability

Ph I/II GvHD (ISR) Follow-up Study

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ApoGraft Phase I/II Clinical Trial

Rambam Medical Center Haifa (Israel)

Hadassah Medical Center Jerusalem (Israel)2 sites

An Open-Label Phase I/II, Pilot, Staggered Four-Cohort Safety and Proof-of-Concept Study

of ApoGraft in the Prevention of Acute Graft Versus Host Disease Trial

12 matched related donor-recipient pairsTrial Group

Safety and preliminary efficacyTrial Purpose

180 daysDuration

Three patients (first cohort) completed 1 month follow up

- full engraftment and no related adverse effects

- dose escalation approved by DSMB

- patient enrollment continues

Current Status

(Jan 2018)

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Financial Data

• Solid balance sheet with $~9.5 M of cash as of 9/30/2017; $~4.0 M raised in Jan 2018; no loans

• Sufficient cash to support current Phase I/II clinical trial and pre-clinical pipeline development

• Ordinary shares outstanding - ~130 million (equivalent to ~6.5 million ADSs)

• IPO on NASDAQ in 2016. De-listed from the TASE (Sep 2017)

Average Volume (*)

* Based on 90 days average

ADSs US$

NASDAQ 115,620 ~ 897,200~

TASE – De-listed 0 0

Total 115,620 ~ 897,200~

(*) Holdings and Average volume as of February 9th, 2018

Founders, 33.99%

>10% Investor, 11.05%

Public, 54.96%

Holdings (*)

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Experienced Management Team / AdvisorsDr. Shai Yarkoni MD, PhD, Co- Founder & CEOSenior executive in the biotechnology industry. Founder and CEO of Target-In Ltd., a cancer biotech therapeutics company. Founded and, until recently, managed Bio-Negev.

Eyal Leibovitz, CFOMore than 25 years of experience in senior management, finance, IR, M&A and business development in international companies. Mr. Leibovitz has served as CFO of Evogene, N-trig Ltd., Kamada Ltd., and New Media Communications.

Dr. Ronit Bakimer-Kleiner PhD, Chief Development OfficerMore then 20 years of experience as a scientist and executive manager in the biotechnological industrial environment, has amassed comprehensive knowledge in cell therapy and GMP manufacturing and has an extensive background in immunology, autoimmunity and cell biology, and in management of GMP facilities and tech-transfer.

Dr. Tamara Byk-Tennenbaum, PhD, MBA, VP OperationsMore than 25 years experience of scientific management in the fields of hematopoietic stem cells, hemostasis, neurobiology and gene therapy. Served as VP Technology Platform at Evogene, R&D Associate Director at Omrix (Ethicon Biosurgery - JnJ) and Scientific Director at Teva. Extensive experience in product development from early stages to launch, including scale up and GMP manufacturing.

BOARDsKasbian Nuriel Chirich, Co-Founder & Chairman of the Board Businessman with extensive financial and business expertise. Leads several business ventures in East Africa and Israel and is the Honorary Consul of Tanzania in Israel.

Dr. Michael Berelowitz MD, Board MemberPreviously Sr. VP, Head of Clinical Development and Medical Affairs, Pfizer Specialty Care Business Unit and Head of Global Medical at Pfizer.

David Braun, Board MemberHead of Merck Group’s Medical Device Business

Dr. Susan Alpert, Advisory BoardPreviously Head of Medical Device division at the FDA and EVP of Regulatory Affairs at both Medtronic (NYSE:MDT) and CR Bard (NASDAQ:BCR).

Dr. Corey Cutler MD, Advisory Board Head of bone marrow transplantation at world-renowned Dana-Farber cancer institute, an Associate Professor of Medicine at Harvard Medical School.

Prof. John F. DiPersioDeputy Director, Alvin J. Siteman Cancer Center and Chief of the Division of Oncology at Washington University School of Medicine in St. Louis and the Virginia E. and Samuel J. Golman Professor of Medicine.

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Strong IP Protection 7 families of global patent applications (4 already issued)

• Patent for the concept of using apoptosis-inducing agents for Stem Cells selection - issued

• Patents for composition of matter of ApoTainers™- issued

• Patents covering methods of use - issued

• Patents covering method of manufacturing ApoTainers™

• Patent for Mesenchymal Stem Cells selection

• Strong IP validated by proof-of-concept studies

• Expiration of concept patent - 2029

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Page 20: Enabling Regenerative Medicinecellect.co/assets/cellect---corporate-presentation-mar... ·  · 2018-03-07Global Transplantation Market ... biobanking o out licensing ... •14 Research

SummaryNASDAQ:APOP

• Cellect technology aims to enable standardized mass production of Stem Cells for

research, clinics and pharma.

• Using the transformational Cellect Platform, Stem Cell products are intended to be safer

and more cost effective.

• Cellect platform uses are expected to drive initial revenues already in 2018

• Strong IP portfolio with 7 patent families; 4 of which already granted in US

• US orphan drug designation granted (2017)

• Clinical trial Phase I/II started and first cohort completed one month with no related

adverse effects (Jan 2018). Dose escalation approved by DSMB.

• Seasoned management team and Advisory Board

• Healthy finances

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Thank You

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