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xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015
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Page 1: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

xxx

Realizing the Potential ofCell-Based Therapy Technology

Nasdaq: CBMG

Copyright CBMG, April 30, 2015

Page 2: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

Company Overview

Addressing unmet medical needs of the world’s largest population, Cellular Biomedicine Group is a biomedicine firm engaged in the development of effective cellular therapies for degenerative joint diseases and airway diseases, immuno-oncology drugs for various cancers, as well as providing biomedical and cellular technical services to healthcare facilities in China.

Led by a seasoned management team and board with more than 150 years of relevant experience in China, Europe, and the USA, it is the only pure-play NASDAQ-listed China market-focused biomedicine developmental company.

April 30, 2015

Page 3: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

China Market Focus

1. 1.4 billion people2. Environmental health threats3. Substantial number of aging population4. Capital efficient clinical trial cost

• 120 million orthopedic patients2

• >57 million are KOA patients3

• 3.5 million new cases diagnosed each year1

• 6 new cases per minute

Osteoarthritis

Cancer

Opportunity to serve unmet needs =

1. National Cancer Registry Center of China2. Silman AJ, Hochberg MC. Epidemiology of the rheumatic disease; Oxford Univ Press. 1993:257. Chinese Journal of Clinical Rehabilitation. October 14, 2005 Vol. 9; Chinese Journal for Clinicians. 2010, 38(7)3. International Journal of Rheumatic Diseases, 2011 3April 30, 2015

Page 4: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

Key Investment Highlights

Developing biomedicine for safe & effective treatment of degenerative and cancerous diseases

• Opportunity to Serve Huge China Marketo 3.5 million new cancer patients/yearo 57 million Knee Osteoarthritis patients

• Opportunity to Serve Huge China Marketo 3.5 million new cancer patients/yearo 57 million Knee Osteoarthritis patients

• Bifurcated Cancer Immunocell Therapy And Regenerative Medicine Technology Platforms

• Supported With High Barriers To Entry And First Mover Advantage

• Bifurcated Cancer Immunocell Therapy And Regenerative Medicine Technology Platforms

• Supported With High Barriers To Entry And First Mover Advantage

April 30, 2015 4

Page 5: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

Differentiated Technology Platforms

Cellular Biomedicine Group is the only NASDAQ-listed China-market focused biomedicine developmental company with a bifurcated approach towards critical mass:

Degenerative Diseases•Currently no big pharma has any meaningful development in the pipeline

•Progenitor cells act as a repair system for the body, replenishing adult tissue

•Transplantation of progenitor cells derived from patient or donor’s fat tissue

•CBMG’s proprietary ReJoinTM haMPC therapy applied to osteoarthritis

•Knee OA in Phase IIb trial, completed patient treatment, positive 6-month results; Positive 12-month results from Phase IIa trial.

•Other indications, incl. asthma

Cancers•CAR-T cell therapy for cancer

o Acute B Lymphocytic Leukemiao Progressive Malignant Lymphomao Hodgkin’s Lymphomao Advanced Lung Cancer

•Combination immunotherapy•Potential faster time to commercialization•CentrixTTM T Cell clonality analysis and Tcm platform technology•Potential market: Millions of patients in China undergoing cancer therapy

IMMUNE CELL THERAPY STEM CELL THERAPY

CBMG in High Growth PhaseCBMG in High Growth PhaseApril 30, 2015 5

Page 6: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

TechnologyIndication/Product

2015 2016 2017 2018 2019 2021 Market size TAMannual

Immune Cell

CAR-T

CD19 Acute B lymphocytic leukemia

0.65±0.08/100,000 8,4001

CD20Progressive malignant lymphoma

8.09/100,000 105,0002

CD30 Hodgkin’s lymphoma 0.4/100,000 5,2003

EGFR (HER1)

Advance lung cancer

53.57/100,000( 2012 ) 728,0004

PD-1 mAb

NSCLC lung cancer

Stem Cell

haMPC

ReJoinTM for OA (autologous) 57 m5 570,0008

Asthma 30 m6 250,0009 (mortality)

Cartilage Damage 0.3 m7

Phase I

Phase II

Phase III

Preclinical

Product Pipeline

April 30, 2015 6

1. 《上海市急性淋巴细胞白血病流行病学调查和分析 》 "The investigation & analysis of epidemiology of acute lymphoblastic leukemia in Shanghai”2. 《中国 2009年恶性淋巴瘤发病与死亡分析 “》 Analysis of China in 2009 and the death of malignant lymphoma incidence”3. 《 2003-2007年中国恶性淋巴瘤发病与死亡分析 》 2012年肿瘤年报 "2003-2007 China annual tumor report; incidence & mortality of malignant lymphoma”4. International Journal of Rheumatic Diseases, 2011 5. WHO; 2.Epidemiology and Dangerous Factors of Bronchitic Asthma6. Market analysis Chinese arthroscopy, July 2014 and “Advances in Orthopedics” Vol 2012, ID 528423 7. 中老年人膝骨性关节炎患病趋势十年前后对照分析 , 2013 “Comparative analysis of prevalence trend of knee osteoarthritis in the elderly, 2013”8. WHO Measurement and Health Information, 20049. WHO Measurement and Health Information, 2004

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ReJoinTM KOA

Stem Cell Therapy Programs

Page 8: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

3D SPGR MRI Quantitative AssessmentIncrease in cartilage volume of whole joint as early as 12 weeks after the therapy, and confirmed significant increase (P<0.002) at 48 weeks after the therapy; reduction of bone marrow lesions in some patients

Western Ontario and McMaster Universities Arthritis Index (WOMAC)Decreased by 9.92 from the baseline (P<0.01) at 48 weeks after the therapy; improved knee mobility

Numeric Rating Scale for Pain (NRS-11)2.75 (P<0.01) and 2.35 (P<0.01) at 12 weeks and 24 weeks, respectively, 0.92 (P>0.05) at 48 weeks from the baseline after the therapy

Short Form Health Survey (SF-36)Decreased by 6.31 from the baseline (P>0.05) at 48 weeks after the therapy

Leading Program: ReJoinTM haMPC Proof-of-ConceptPhase IIa 48-week follow-up data

n=13

8April 30, 2015

Page 9: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

ARTZ RejoinTM-100

-50

0

50

100T

he

pe

rce

nta

ge

of

ch

ang

e f

or

WO

MA

C(%

)

n=12 n=12

* P=0.017

The Percentage of Change for WOMAC

• WOMAC: The Western Ontario and McMaster Universities Arthritis Index • WOMAC Improvement (%)= [WOMAC0W - WOMAC24W] / WOMAC0W × 100%• ReJoinTM : Human Adipose Tissue Derived Mesenchymal Progenitor Cells, CBMG• ARTZ@ : Sodium Hyaluronate, LG Chemical, Kabi Pharmacia (日本生物化学工业公司 ), 1987• 24th week after therapy, the WOMAC score significantly decreased from 33.42 to 17.00 ( 47.12%

improvement, ) in ReJoinTM group, and from 31.83 to 25.00 (16.32% improvement ) in ARTZ@ group. The results showed a significant difference ( p=0.017) in the percentage of changes between two groups at 24th week follow up

Phase IIb ReJoinTM 24th Week - WOMAC Score

April 30, 2015 9

The percentage of WOMAC change

The

perc

enta

ge o

f WO

MA

C ch

ange

Page 10: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

ARTZ RejoinTM-150

-100

-50

0

50

100

150

Th

e p

erc

en

tag

e o

f c

ha

ng

e f

or

VA

S(%

)

n=12 n=12

P=0.119

The Percentage of Change for VAS (Left)

ARTZ RejoinTM-150

-100

-50

0

50

100

150

Th

e p

erc

en

tag

e o

f c

ha

ng

e f

or

VA

S(%

)

n=12 n=12

P=0.229

The Percentage of Change for VAS (Right)

• VAS: Visual Analogue Scale Score• VAS improvement (%)=[VAS0W -VAS24W] / VAS 0W ×100%• ARTZ@: Sodium Hyaluronate, LG Chemical, Kabi Pharmacia (日本生物化学工业公司 ), 1987• ReJoinTM : Human Adipose Tissue Derived Mesenchymal Progenitor Cells, CBMG• 24th week after therapy, the VAS of Left knee significantly decreased from 4.96 to 2.21 (45.14%

improvement) in ReJoinTM group, and from 5 to 3.24 (19.22% improvement) in ARTZ@ group. The same result appeared for the right knee ,from 5.67 to 2.29 ( 49.52% improvement) in ReJoinTM group ,and from 4.83 to 3.33 (26.92% improvement) in ARTZ@ group . But not a significant difference (left knee p=0.019, right knee p=0.229) between two groups at 24th week follow up.

April 30, 2015 10

Phase IIb ReJoinTM 24th Week - VAS Score

The percentage of VAS change (left) The percentage of VAS change (right)

The

perc

enta

ge o

f VA

S ch

ange

(lef

t)

The

perc

enta

ge o

f VA

S ch

ange

(rig

ht)

Page 11: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

ARTZ RejoinTM-1000

-500

0

500

1000

1500

2000

2500

Change of Left Knee Cartilage Volume

Ch

an

ge

of

Ca

rtil

ag

e V

olu

me

(mm

3)

n=12 n=11ARTZ RejoinTM

-1000

-500

0

500

1000

1500

2000

2500

Change of Right Knee Cartilage Volume

Ch

an

ge

of

Ca

rtil

ag

e V

olu

me

(mm

3)

n=12 n=11

• + Mean;--- Median• Increased volume of knee cartilage = Cartilage Volume ( 24w ) – Cartilage Volume ( 0w )• Normality test / Wilcoxon test• The knee cartilage volume were measured by using a semi-automated segmentation method(ITK-SNAP) by

three different blinded researchers.• ReJoinTM : Human Adipose Tissue Derived Mesenchymal Progenitor Cells, CBMG• ARTZ@ : Sodium Hyaluronate, LG Chemical, Kabi Pharmacia (日本生物化学工业公司 ), 1987• 24th week after therapy, the left knee cartilage volume increased 229.3 mm3 in ReJoinTM group, but

only increased 29.9 mm3 in ARTZ@ group, the right knee cartilage volume significantly increased 585.8mm3 (p=0.0084) in ReJoinTM group, and increased 502.8 mm3 (p=0.014) in ARTZ@ group. But not still significant difference between two groups at 24th week follow up.

April 30, 2015 11

Phase IIb ReJoinTM 24th Week – Cartilage MRI

Page 12: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

CBM-C19.1 and CBM-C20.1Phase I Trial Results

Immune Cell Cancer Therapy Programs

Page 13: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

Clinical Stage Immuno-Oncology Platform

Reviewing Data to Prioritize Disease & Initiate add’l Trials Reviewing Data to Prioritize Disease & Initiate add’l Trials April 30, 2015 13

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CAR-T: Promising Clinical Results*

10

* Very stringent patient selection: advanced, relapsed and refractory; no conditioning therapy nor add’l allo-HSCT therapy; **Seven CD19 patients with and without extramedullary involvement and treated with autologous CAR-CD19 T cell therapy;***Six CD19 patients with extramedullary leukemia involvement or bulky adenopathy;Note: Two CD19 patients treated with allogeneic CAR-CD19 T cell therapy; one died from GVHD and one survived with PR

April 30, 2015

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CBM-C20.1 Case Report

CBM-C20.1 infusion CBM-C20.1 infusion CBM-C20.1 infusionApril 30, 2015

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16

+32d +42d +50d +88d

-15d +70d

Patient 9: 23 year old female, common-ALL, relapsed 13m after donor-HSCT

GVHD--Skin Damage

CR-Bone Marrow

CBM-C19.1 Case Report

aggravated regressed PET-CT scan

April 30, 2015

Page 17: Xxx Realizing the Potential of Cell-Based Therapy Technology Nasdaq: CBMG Copyright CBMG, April 30, 2015.

CBMG Vertically Integrated Cell Therapy

17

Sample Collection & Shipment

Cell Isolation Culture & Expansion

Cell Banking & Storage

Shipment Final Product & Formulation

Tissue Transport Kit 201220200402.2 201220056183.5 201420660845.9

Tissue Preservation Solution

201210137981.5 PCT/CN2013/075111 CFDA Class I Medical

Device Registration:

20150021

A-Stromal Extraction Kit

201220200403.7201420673999.1201410785126.4201330527554.3

CFDA Class I Medical Device Registration:

苏锡食药监械(准)字

2013 第 1400047 号

Cell Purification 201110154507.9

MIISF Culture Media TRADE SECRET

XISF Dissociation Media TRADE SECRET

T-Cell/CAR-T Expansion 201520033059.0 201510016959.9 201510061635.7

PATENTS

CYSF Serum-Free Cryopreservation

Media

201110227449.8 PCT/CN2012/079737

US 14369448 EP 12821828.6

C1S Biopreservation Solution

201210187672.9 201330385774.7

CFDA Class I Medical Device

Registration: 20150022

MSC Usage 201310044272.7

PCT/CN2014/071716 201210466717.6

PCT/CN2013/087284 ReJoinTM Kit

201310289978.X PCT/CN2014/081849

201320422962.7 T-Cell Analysis

US 7375211 ZL02131070X

REAGENTS & KITS

KEY PATENTS & CERTIFICATES

April 30, 2015

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•One of the few cell therapy companies in China with facilities certified by internationally recognized ISO accreditation institution SGS

•6 independent production lines

•Capacity for over 5,000 patients for autologous cell therapies per year

•Two cell banks in different cities with storage capacity to host more than 200,000 individual cell sources

•The most advanced cold-chain temperature control systems (IT cloud system), monitoring the process of cell culture, cell storage and transportation

Facilities

Shanghai GMP facility

CBMG has GMP facilities in the PRC designed to U.S. FDA standards and Chinese regulatory bodies’ standards

April 30, 2015

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History

• Cellular Biomedicine Group founded in China

• CBMG international-standard R&D and manufacturing facility opened in Shanghai, China

• Released positive results from TC-DC Phase I Clinical Trial for Liver Cancer (HCC)

• June 18 – Commenced trading on Nasdaq Stock Exchange: CBMG

• Completed Patient Treatment, ReJoinTM Phase IIb Clinical Trial for Knee Osteoarthritis (KOA)

• Acquired Tcm immune cell therapy technology

• Launched cartilage damage (CD) stem cell clinical study

• Released positive 12 month follow-up data from ReJoinTM Phase I/IIa Clinical Trial

• Acquired 3rd generation CAR-T, CD19 and PD-1 cancer immunotherapy patents

• Acquisition of CD19,CD20,CD30,HER1 Patients and Clinical Data I/II

• Released positive interim 6 month data from ReJoinTM Phase IIb Clinical Trial

• Released positive CAR-T Phase I Clinical Data for CD19 & CD20April 30, 2015

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Upcoming Major Milestones

• Publish CAR-T Phase I Clinical Data - CBM-C30.1, Hodgkin’s lymphoma•Fortify science leadership with key appointments

• Publish CAR-T Phase I Clinical Data - CBM-HER1.1, Advanced lung cancer• Prioritize cancer disease plan to commercialization• Expansion of GMP facilities 57,000 ft2

• Release 48-week follow-up data from ReJoinTM Phase IIb Clinical Trial

20April 30, 2015

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Seasoned Management

Wei (William) Cao, PhD, BM / CEO & DirectorPost-doctorate in Pathology, Harvard Medical SchoolPost-doctorate in Cardiothoracic Surgery, Stanford University Medical School Former GM, Affymetrix Greater ChinaElected member of Expert Committee, 3rd Medical Technology Approval Office, Chinese Medical Doctor Association, Ministry of Health, PRC

Bizuo (Tony) Liu, BA in PhysicsChief Financial Officer, Board Secretary & DirectorFormer Corporate VP, Alibaba Group; CFO, HiChinaFormer GM, Corporate Strategy, Microsoft Former Corporate Accounting Director, Microsoft

Andy Chan, JD, MBASenior Vice President of Business Development Former CBMG CFO and Board SecretaryFormer VP, BD & China Operations, Jazz SemiconductorFormer Senior Director, Strategic Business Development, AlliedSignal Aerospace

Helen Zhang, BMVP China Operation and TechnologyFormer Associate Director, Harvard Gene Therapy Initiative, Harvard Medical SchoolFormer Max-Delbruck Research Fellow, National Research Center for Molecular Medicine, Berlin, Germany

Maxwell Wang, MD, PhD / Director of Medical ResearchOrthopedic Surgeon Over 10 years of research focused on mesenchymal stem cells and bone cells in Germany, UK and USA. The first to find the paracrine and autocrine effects of CCN family genes in bone cell differentiationMD degree, Wuerzburg University in GermanyPhD degree, University of Aberdeen, UK

Chase Dai, D.M.Sc.VP and GM of Cell Therapy Business Unit. Former CEO, MedGene Biotechnology Former Deputy Chief Physician of Cardiology Dept, Beijing 301 PLA Hospital

Professor Zhong Chen Kou / Chief Scientist, Immunology Research fellowships at Chinese Academy of Medical Science, Peking Union Medical College, and Harvard Medical School Research assistant professor at the University of Florida Currently, Chief Scientist, Tampa Bay technology center, BioMed Immunotech, University of South Florida

21

Edward Li , MD, PhD / Senior Director, ImmunologyFormer Technical director, Agreen BiotechnologyFormer Deputy Researcher of Neurobiology Dept, Beijing Institute of Geriatrics, Xuanwu Hospital, Capital Medical UniversityFormer Visiting scholar, Osaka University Hospital, in JapanPhD degree, Shiga University of Medical Science, in Japan

April 30, 2015

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Scientific Advisory Board

Scott J. Antonia, MD, PhDScientific Advisor, Immuno-OncologyPrincipal investigator of multiple high profile immuno-oncology clinical trials, including anti-PD-1 therapy for advanced lung cancerDepartment chair & program leader, Thoracic Oncology, Moffitt Cancer CenterProgram leader Immunology Program, Moffitt Cancer CenterProfessor of oncology, University of South Florida College of Medicine (uSF)Medical degree & doctorate in immunology from the University of Connecticut Health CenterInternal medicine residency, medical oncology fellowship & post-doctoral fellowship in immunobiology at Yale University School of MedicinePublished over 90 peer-reviewed publications

Guoping Fan, PhDScientific Advisor, Stem Cell Technology & ApplicationsProfessor, Department of Human Genetics, David Geffen School of Medicine at University of Los Angeles (UCLA)Member, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell ResearchFormer postdoctoral fellow at the Whitehead Institute for Biomedical InstituteB.S. in Biochemistry at Nanjing University, ChinaPh.D. in Neuroscience at Case Western Reserve UniversityPublished over 80 peer-reviewed publications

22April 30, 2015

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Non-Management Board of Directors

Hon. Nadir Patel, MBA / Independent DirectorMBA, NYU Stern School of Business; London School of Economics & Political Science; HEC Paris School of MgmtCanada’s High Commissioner to IndiaFormer Assistant Deputy Minister, Corporate Planning, Finance & Information Technology, and Chief Financial Officer, Canada’s Department of Foreign Affairs, Trade & DevelopmentFormer Canadian Consul General in Shanghai

Wen Tao (Steve) Liu, PhD/ Executive ChairmanPhD, Physical Chemistry, Rensselaer Polytechnic Institute, NYCFormer President & CEO, Shanghai Hua Hong NEC Electronics Company Former President & CEO, Seeo Inc.

Terry A. Belmont / Vice ChairmanOver 20 years of experience in leading major academic and non-academic medical centers and healthcare entitiesFormer CEO of Long Beach Memorial Medical Center and Miller Children’s HospitalCEO, UC Irvine Medical Center since 2009

Guo-Tong Xu, M.D., PhD / Independent DirectorProfessor of Ophthalmology and Regenerative Medicine, Dean of Tongji University School of MedicineDirector of Stem Cell Bank of TUSMPhD in pharmacology from University of North Texas Health Science CenterMD degree from Peking Union Medical CollegeMD degree from Chinese Academy of Medical Sciences

David Bolocan / Independent DirectorOver 20 years in retail banking and paymentsMgng Director for Argus Information & Advisory ServicesFormerly held senior executive roles at SunTrust, JPM Chase and MBNA/Bank of America

Gerardus A. Hoogland, MD, MBA / DirectorOver 20 years of experience managing international pharmaceutical companiesDirector of Cytespace Pvt, Ltd, a clinical research site solution organization Chief Executive Officer of HealthCrest AG, an investment and consulting companyMD, University of Amsterdam; MBA, Institute d’Administration des Affaries (INSEAD)

Alan Au/ Independent DirectorOver 15 years of experience in cross boarder M&A and financing transactions for pharmaceutical and healthcare companies Senior adviser to the Board of Directors of Simcere Pharmaceutical GroupCertified Public Accountant in the U.S., holds the Chartered Financial Analyst (CFA) designation

23April 30, 2015

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xxx

Addressing unmet medical needsof the world’s largest population

THANK YOUwww.CellBioMedGroup.com


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