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Nasdaq: CBMG Realizing the Poten/al of Cell-­‐Based Therapy Technology March 25, 2015 Safe Harbor
Statements made in this presenta.on rela.ng to plans, strategies, economic performance and trends, projec.ons of results of specific ac.vi.es or investments, and other statements that are not descrip.ons of historical facts may be forward-­‐looking statements. Forward-­‐looking informa.on is inherently subject to risks and uncertain.es, and actual results could differ materially from those currently an.cipated due to a number of factors, which include, but are not limited to, risk factors inherent in doing business. Forward-­‐looking statements may be iden.fied by terms such as "may," "will," "should," "could," "expects," "plans," "intends," "an.cipates," "believes," "es.mates," "predicts," "forecasts," "poten.al," or "con.nue," or similar terms or the nega.ve of these terms. Although we believe that the expecta.ons reflected in the forward-­‐looking statements are reasonable, we cannot guarantee future results, levels of ac.vity, performance or achievements. The Company has no obliga.on to update these forward-­‐looking statements. This presenta.on is strictly intended to provide general informa.on about our company and business. This presenta.on nor any part hereof cons.tutes an offer of securi.es. March 25, 2015 2 Why China Market Focus 1. 
2. 
3. 
4. 
1.4 billion people Opportunity to serve unmet needs Environmental health threats Capital efficient clinical trial cost Substan.al number of aging popula.on Cancer Osteoarthri/s March 25, 2015 1. 
2. 
3. 
= illion new cases diagnosed each year
•  3.5 m
1 •  6 new cases per minute • 120 million orthopedic pa/ents2 • >57 million are KOA pa/ents3 Na.onal Cancer Registry Center of China Silman AJ, Hochberg MC. Epidemiology of the rheuma.c disease; Oxford Univ Press. 1993:257. Chinese Journal of Clinical Rehabilita.on. October 14, 2005 Vol. 9; Chinese Journal for Clinicians. 2010, 38(7) Interna.onal Journal of Rheuma.c Diseases, 2011 3 Key Investment Highlights Developing biomedicine for safe & effec8ve treatment of degenera8ve and cancerous diseases •  Bifurcated Cancer Immunocell Therapy And Regenera.ve Medicine Technology Pladorms •  Supported With High Barriers To Entry And First Mover Advantage •  Opportunity to Serve Huge China Market o  3.5 million new cancer pa.ents/year o  57 million Knee Osteoarthri.s pa.ents March 25, 2015 4 Product Pipeline Technology
Immune Cell CAR-­‐T CD19 Acute B lymphocy8c leukemia
CD20 2015
2016
2017
2018
2019
2021 Market size TAM annual 0.65±0.08/ 100,000 8,4001 Progressive 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 ReJoinTM for OA (autologous) 57 m5
570,0008
Asthma
30 m6
PD-­‐1 mAb Stem Cell Indica/on /Product
NSCLC lung cancer
haMPC Car.lage Damage
Preclinical Phase I Phase II Phase III March 25, 2015 1. 
2. 
3. 
4. 
5. 
6. 
7. 
8. 
250,0009 (mortality)
0.3 m7
《上海市急性淋巴细胞白血病流行病学调查和分析》 "The inves.ga.on & analysis of epidemiology of acute lymphoblas.c leukemia in Shanghai” 《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” 《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” Interna.onal Journal of Rheuma.c Diseases, 2011 WHO; 2.Epidemiology and Dangerous Factors of Bronchi.c Asthma Market analysis Chinese arthroscopy, July 2014 and “Advances in Orthopedics” Vol 2012, ID 528423 中老年人膝骨性关节炎患病趋势十年前后对照分析, 2013 “Compara.ve analysis of prevalence trend of knee osteoarthri.s in the elderly, 2013” WHO Measurement and Health Information, 2004 5 Differen/ated Technology PlaZorms Cellular Biomedicine Group is the only NASDAQ-­‐listed China-­‐market focused biomedicine developmental company with a bifurcated approach towards cri.cal mass: IMMUNE CELL THERAPY STEM CELL THERAPY Cancers Degenera/ve Diseases • CAR-­‐T cell therapy for cancer • Currently no big pharma has any meaningful development in the pipeline o 
o 
o 
o 
Acute B Lymphocy.c Leukemia Progressive Malignant Lymphoma Hodgkin’s Lymphoma Advanced Lung Cancer • Combina.on immunotherapy • Poten.al faster .me to commercializa.on • CentrixTTM T Cell clonality analysis and Tcm pladorm technology • Poten.al market: Millions of pa.ents in China undergoing cancer therapy • Progenitor cells act as a repair system for the body, replenishing adult .ssue • Transplanta.on of progenitor cells derived from pa.ent or donor’s fat .ssue • CBMG’s proprietary ReJoinTM haMPC therapy applied to osteoarthri.s • Knee OA in Phase IIb trial, completed pa.ent treatment; Posi.ve 12-­‐month results from Phase IIa trial. • Other indica.ons, incl. asthma CBMG in High Growth Phase March 25, 2015 6 CBMG Ver/cally Integrated Cell Therapy •  Sample collec/on -­‐> Processing -­‐> Banking -­‐> Final Prep -­‐> Shipping -­‐> Use •  CBMG Back-­‐End Advantage 24/7 Cloud-­‐Based Temperature Control CAR-­‐T Cell Process* Final Prep Stem Cell Process Shipping Ready to Use • 4~8°C • Liquid format • 72 hours • No thaw, no wash • No poten.al contamina.on • Reduce variability * In process Our No-­‐Thaw SOP Reduces the Common Quality Escape & Logis/cs Challenges March 25, 2015 7 Immune Cell Cancer Therapy Programs CBM-­‐C19.1 and CBM-­‐C20.1
Phase I Trial Results Clinical Stage Immuno-­‐Oncology PlaZorm
CAR-­‐T Func/on Area
CD19-­‐ScFv-­‐
CD8-­‐CD137-­‐
CD3ζ
CD20-­‐ScFv-­‐
CD8-­‐CD137-­‐
CD3ζ
CD30-­‐ScFv-­‐
CD8-­‐CD137-­‐
CD3ζ
Condi/on
Acute B lymphocy.c leukemia with CD19 posi.ve
Progressive Malignant lymphoma with CD20 posi.ve
Hodgkin’s lymphoma with CD30 posi.ve
HER1-­‐ScFv-­‐
Advanced lung CD8-­‐CD137-­‐ cancer with HER1 CD3ζ
posi.ve
Pa/ents Stage of clinical Register No. start and trial
end /mes
Milestone on Data Release 12 I/II
NCT01864889 2013 April -­‐2017 April
March, 2015
10 I/II
NCT01735604 2013 Jan. – 2017 Jan
March, 2015
10
I/II
NCT02259556 2014 OCT -­‐2019 OCT
September, 2015
10
I/II
NCT01869166 2013 May – 2016 May
September, 2015
Reviewing Data to Priori/ze Disease & Ini/ate add’l Trials March 25, 2015 9 CBMG CAR-­‐T Clinical Phase I Results
Products
CAR T
Indication
CBMC20.1
CD20ScFvCD8CD137CD3ζ
Refractory,
Advanced
DLBCL
CBMC19.1
CD19ScFvCD8CD137CD3ζ
Advanced,
Relapsed,
Refractory
B-ALL
Patient
Response
#
7
(5 bulk)
9
(Allo 2)
ORR
2 no bulk:
CR1
(>14m)
PR1 (>6m)
No Bulk:
100%
(2/2)
5 bulk:
PR3 (3-6m)
PD1
NE1
Bulk:
75%
(3/4)
CR
2(3-20w)
PR
4(8-38w)
PD 3
71.4%
(5/7)*
66.7%
(4/6)**
AE/SAE
Publication
Grade 2-3:
3
Grade 4:
1
Clinical
Immunology.
2014,
(155:
160~175)
Grade 2-3:
2
Grade 4:
2 (GVHD)
Onco
immunology.
2015,
(Accepted
March 2015)
*7 CD19 pa.ents with and without extramedullary involvement and treated with autologous CAR-­‐CD19 T cell therapy; **6 CD19 pa.ents with extramedullary leukemia involvement or bulky adenopathy; Note: Two CD19 pa.ents treated with allogeneic CAR-­‐CD19 T cell therapy; one died from GVHD and one survived with RR March 25, 2015
10 CBM-­‐C20.1 Case Report
CBM-­‐C20.1 infusion March 25, 2015
CBM-­‐C20.1 infusion CBM-­‐C20.1 infusion 11 CBM-­‐C19.1 Case Report
Pa/ent 9: 23 year old female, common-­‐ALL, relapsed 13m axer donor-­‐HSCT
CR-­‐Bone Marrow
GVHD-­‐-­‐Skin Damage +32d
+42d
+50d
+88d
aggravated regressed
March 25, 2015
-15d
+70d
PET-­‐CT scan
12 Stem Cell Therapy Programs ReJoinTM KOA Phase IIb Trial Interim Data
ReJoinTM 24th Week -­‐ WOMAC Score
The percentage of WOMAC change T h e p e rc e n ta g e o f c h a n g e fo r W O M A C (% )
The T h epercentage P e r c e n t aogf eWoOMAC f C h acnhange g e fo r
*
P = 0 .0 1 7
100
50
0
-5 0
-1 0 0
WOMAC (%)
• 
• 
• 
• 
• 
WOMAC
AR T Z
R e jo in
n=12
n=12
16.32
TM
47.12
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
March 25, 2015
14
ReJoinTM 24th Week -­‐ VAS Score
100
50
0
-5 0
-1 0 0
-1 5 0
VAS (%)
• 
• 
• 
• 
• 
P = 0 .1 1 9
150
AR T Z
R e jo in
n=12
n=12
19.22
45.14
TM
(L e ft)
ercentage TThe h e Ppe
r c e n t a g e oof fVCAS h acnhange g e f o r(right) V A S ( R ig h t )
The percentage of VAS change (right) T h e p e rc e n ta g e o f c h a n g e fo r V A S (% )
The percentage of VAS change (lek) T h e p e rc e n ta g e o f c h a n g e fo r V A S (% )
The T h e pPercentage e r c e n t a g eof oV
f AS C hc
ahange n g e f o(rlek) VAS
P = 0 .2 2 9
150
100
50
0
-5 0
-1 0 0
-1 5 0
VAS (%)
AR T Z
R e jo in
n=12
n=12
26.92
TM
49.52
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.
March 25, 2015
15
ReJoinTM 24th Week – Car/lage MRI
C h a n g e o f R ig h t K n e e C a r t ila g e V o lu m e
C h a n g e o f C a r tila g e V o lu m e (m m )
3
3
C h a n g e o f C a r tila g e V o lu m e (m m )
C h a n g e o f L e ft K n e e C a r t ila g e V o lu m e
2500
2000
1500
1000
500
0
-5 0 0
-1 0 0 0
AR T Z
R e jo in
n=12
n=11
TM
2500
2000
1500
1000
500
0
-5 0 0
-1 0 0 0
AR T Z
R e jo in
n=12
n=11
TM
Mean
29.89
Mean
229.3
Mean
502.9
Mean
585.8
Median
-­‐307.5
Median
403.1
Median
445.6
Median
329.8
•  + 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.
March 25, 2015
16
2015 Major Milestones •  Acquire CAR-­‐T CD19, CD20, CD30, and CD HER1, 3rd CAR-­‐T, an.-­‐PD1 •  Release 24-­‐week interim data from ReJoinTM Phase IIb Clinical Trial •  Publish CAR-­‐T Phase I Clinical Data •  March: CBM-­‐C19.1, Acute B lymphocy.c leukemia CBM-­‐C20.1, Progressive malignant lymphoma •  Publish CAR-­‐T Phase I Clinical Data •  September: CBM-­‐C30.1, Hodgkin’s lymphoma CBM-­‐HER1.1, Advanced lung cancer •  Priori.ze cancer disease plan to commercializa.on •  Expansion of GMP facili.es à 47,000 x2 •  Release 48-­‐week follow-­‐up data from ReJoinTM Phase IIb Clinical Trial Credible Execu/on & Trac/on in Deliverables March 25, 2015
17
Summary §  Clean balance sheet: No debt, no overhang, used common shares only §  Raised ~$55m to date; $14.6m on hand at end of 2014 §  Shares out: 11.3 million; fully diluted: 13.2 million §  Focusing on China, the 2nd largest and fastest growing medical market with huge aging popula.on §  First mover advantage CBMG is an.cipated to be the first to pursue interna.onal compliance in China, thereby seyng the clinical pathways to several treatments §  Cancer Immuno-­‐oncology: Achieving leading business posi.on by amalgama.ng technology leaders in China §  CAR-­‐T technology – clinical phase §  Tcm technology service – budding revenue §  Leading Regenera/ve Medicine Program (KOA) where there are currently no big pharma alterna.ves §  Interna/onally experienced team A culturally savvy team of professional managers with proven track record in mul.-­‐billion dollar mul.na.onal companies §  Mul/ple inflexion points to grow shareholder value March 25, 2015 18 xxx
Addressing unmet medical needs of the world’s largest popula/on THANK YOU www.CellBioMedGroup.com