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Rodman & Renshaw 13 th Annual Healthcare Conference September 12, 2011

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Page 1: Rodman & Renshaw 13th Annual Healthcare …content.stockpr.com/cbiolabs/media/d8f3658ea8604a40fab3c...• Phase I trial with oral formulation of next generation CBLC137 in solid tumors

Rodman & Renshaw 13th Annual Healthcare Conference

September 12, 2011

Page 2: Rodman & Renshaw 13th Annual Healthcare …content.stockpr.com/cbiolabs/media/d8f3658ea8604a40fab3c...• Phase I trial with oral formulation of next generation CBLC137 in solid tumors

This presentation includes forward-looking statements and predictions,

including statements about potential revenue-bearing transactions, the

market potential of CBLI’s technologies and product candidates, and the

potential value of pipeline products. These statements represent the

Company’s judgment as of the date of this presentation and are subject to

risks and uncertainties that could cause actual results of events to differ

materially from those expressed in such forward-looking statements. In

particular, CBLI faces risks and uncertainties that it may not be able to

sustain its business model, that revenues may be lower or expenses higher

than projected, that product sales may not increase, that development of

product candidates in the Company’s pipeline may not succeed or that

commercial transactions may not go forward as planned.

Safe-Harbor

2

Page 3: Rodman & Renshaw 13th Annual Healthcare …content.stockpr.com/cbiolabs/media/d8f3658ea8604a40fab3c...• Phase I trial with oral formulation of next generation CBLC137 in solid tumors

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CBLI develops drugs against major unmet biodefense and medical needs

Countermeasure against lethal radiation exposure CBLB502

Supportive care drugs against side effects of radiotherapy and chemotherapy

CBLB502, CBLB612

Novel anticancer therapeutics

CBLB502, CBLC102, CBLC137

Mission

3

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• Broad pipeline of drug candidates with multiple applications

• Accelerated commercialization through biodefense

• Strategic partnerships with Cleveland Clinic and Roswell Park Cancer Institute

• Track record of non-dilutive grants and contracts (~$100M,including $30M conditional purchase for CBLB502)

• Patents issued in US, Europe and Asia

• $30M cash and ~ $25M additional committed government and JV funding

Investment Highlights

4

Page 5: Rodman & Renshaw 13th Annual Healthcare …content.stockpr.com/cbiolabs/media/d8f3658ea8604a40fab3c...• Phase I trial with oral formulation of next generation CBLC137 in solid tumors

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CBLB502Radiation Countermeasure

Supportive Care against cancer therapy side effectsImmunotherapy against cancer

5

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Radiation Countermeasure Opportunity

6

• Nuclear attack identified by US and global leaders as number onesecurity threat

• Reauthorization of Pandemic All hazards Preparedness Act includesradiation as top priority

• Terrorist attack with a 10 KT device will kill 400,000 people in NYCmost of them via ARS (Institute of Medicine Report, June 2009)

• Fukushima disaster highlights risk of nuclear industry

• There are no FDA licensed countermeasures for ARS

CBLB502 uniquely positioned as therapeutic against ARS

Page 7: Rodman & Renshaw 13th Annual Healthcare …content.stockpr.com/cbiolabs/media/d8f3658ea8604a40fab3c...• Phase I trial with oral formulation of next generation CBLC137 in solid tumors

CBLB502 as Medical Radiation Countermeasure Origin & Mechanism of Action

• Protein of bacterial origin (flagellin) modified to reduce immunogenicity and toxicity and improve production

• Acts through multiple mechanisms mediated by activation of pro-survival NF-kB signaling pathway

• Selectively protects normal tissues (but not malignant tumors) from radiation

• Increases survival of stem cells and early progenitors of hematopoietic system and stimulated regeneration of different HP lineages

• Reduces radiation damage to and stimulate regeneration of crypts, villi and lamina propria of GI tract

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Page 8: Rodman & Renshaw 13th Annual Healthcare …content.stockpr.com/cbiolabs/media/d8f3658ea8604a40fab3c...• Phase I trial with oral formulation of next generation CBLC137 in solid tumors

Development of CBLB502 for Licensure as Countermeasure via FDA’s Animal Rule

• Efficacy in animal models that mimic human disease

• Human safety

• Well understood mechanism of action to justify selection of objective indicators (biomarkers) in humans

Drug candidates, efficacy of which cannot be directly tested in humans due to ethical reasons, are developed according to Animal Rule:

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CBLB502 is efficacious in mice and monkeys in protecting and mitigating regimens

27 studies with non-human primates; >180 studies (with multiple strains of mice, types of irradiation,

survival, HP, GI and other endpoints)

N IH S w is s m ic e , 1 3 G y o f to ta l b o d y ir ra d ia t io n

t im e a fte r ir ra d ia t io n , d a y s

0 2 4 6 8 1 0 1 2 1 4 1 6 1 8 2 0 2 2 2 4 2 6 2 8 3 0

an

ima

l s

urv

iva

l, %

0

2 0

4 0

6 0

8 0

1 0 0 C B L B 5 0 2 (n = 5 0 )

a m ifo s t in (n = 1 3 * )

s a lin e (n = 2 5 )5 -A E D (n = 5 )

Mice

0

10

20

30

40

50

60

70

80

90

100

0 5 10 15 20 25

Days after TBI

Su

rviv

al

(%),

n=

40

7.5 Gy, + 16h, PBS/Tween80

8 Gy, + 16h, PBS/Tween80

7.5 Gy, + 16h, CBLB502 40 µg/kg

8 Gy, + 16h, CBLB502 40 µg/kg =25%

=37%=37%

protection mitigation

Days after 6.5 Gy gamma-TBI0 10 20 30 40

% o

f su

rviv

ors

0

20

40

60

80

100

vehicle (PBS), n=8

CBLB502 @ +16h, n=12

CBLB502 @ +25h, n=10

CBLB502 @ +48h, n=12

Survival of rhesus monkeys after 6.5 Gy -TBI

Days after irradiation

0 10 20 30 40

% o

f s

urv

ivo

rs

0

20

40

60

80

100

120

Vehicle (N=8) at -45'

CBLB502, 0.04 mg/kg (N=11) at -45'

protection mitigationNHPs

Days after 6.5 Gy gamma-TBI0 10 20 30 40

% o

f su

rviv

ors

0

20

40

60

80

100

vehicle (PBS), n=8

CBLB502 @ +16h, n=12

CBLB502 @ +25h, n=10

CBLB502 @ +48h, n=12

Survival of rhesus monkeys after 6.5 Gy -TBI

Days after irradiation

0 10 20 30 40

% o

f s

urv

ivo

rs

0

20

40

60

80

100

120

Vehicle (N=8) at -45'

CBLB502, 0.04 mg/kg (N=11) at -45'

protection mitigationNHPs

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Summary of CBLB502 Efficacy Features in NHPs

• Species: rhesus monkey, Macaca mulatta (best-studied primate model in ARS); both sexes, young adults

• Doses of radiation tested: from LD10/40 to LD75/40 TBI in survival studies and LD90-100 TBI in GI morphology studies

• Efficacious times of treatment: at least from -45’ to >48 hours (treatment at 120 hours is not efficacious)

• Efficacious doses of CBLB502: >=10 ug/kg efficacious at all time points and radiation doses tested, as single intramuscular injection

CBLB502 increases survival (up to 3 times); reduces severity and duration of thrombocytopenia;

reduces severity of neutropenia; reduces morphological damage in BM, GI tract, spleen, thymus and lymph nodes

27 studies with total of 905 non-human primates

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Completed Steps in Production of CBLB502

11

• Full industrial-scale production process based on recombinant DNA technology

• Single fermentation generating hundreds of thousands of doses

• Reproducibility demonstrated in multiple GMP runs

• Stable as a frozen liquid and in lyophilized form

• Release assays validated

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CBLB502 Human Trial Program Summary

12

• Total of 150 human volunteers received range of doses of CBLB502 in 2 studies

• Dose limiting toxicity (DLT) defined (manifested as flu-like syndrome)

• Calculated efficacious dose in humans below DLT

• Adverse event profile predictable and directly related to mechanism of action of CBLB502

• Methodology established to determine projected human efficacious dose (based on biomarkers)

• All biomarkers project similar human dose

• Collected information enables start of definitive safety/dose validation trial in healthy volunteers for CBLB502 defense

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CBLB502-Biodefense Path to LicensureRemaining Tasks

13

Completed Remaining steps

CMC

GMP process developed and tested, drug suitable for clinical trials released

Additional consistency runs

Efficacy

Data from ~1,000 primates demonstrates dramatic survival benefits and accelerated

recoveryPivotal animal studies

Human safety

Two trials: 50-subject dose-escalation and 100-subject study completed

Definitive safety study

FDA process

Open IND, Fast Track Status, Orphan Drug Status

Coordinating study protocols, BLA submission

Page 14: Rodman & Renshaw 13th Annual Healthcare …content.stockpr.com/cbiolabs/media/d8f3658ea8604a40fab3c...• Phase I trial with oral formulation of next generation CBLC137 in solid tumors

CBLB502 Federal Contract Funding

14

GRANT/CONTRACT TITLE AMOUNT DATES

DoD /DTRA, Med. Chem. & Biol.

Defense Res. Program

Radioprotective Mechanisms of

CBLB502$1,300,000 3/07-3/10

DoD/CBMS-JPEO

Chemical Biological Medical

Systems Joint Project Mgt.

BAA-07-01- Advanced

Development of a Medical

Radiation Countermeasure

$10,340,000 3/08-10/09

NIAID (NIH)

BioShield Program

CBLB502 mitigation of radiation

induced thrombocytopenia$1,230,000 9/08-3/10

BARDA (HHS)

BioShield Program

BAA-08-08 -Development of

CBLB502 of mitigation of HP

syndrome

$15,800,000 9/08-10/10

NIH/NIAID

Grand Opportunities (GO) GrantProtectan CBLB502 $5,300,000 9/09-9/11

DoD/CBMS-JPEO

Chemical Biological Medical

Systems Joint Project Mgt.

RFP W9113M-09-R-0010

Advanced Development of a

Medical Radiation

Countermeasure

$45,000,000

(15,000,000 +

30,000,000)

9/10-9/13

DoD /DTRA, Med. Chem. & Biol.

Defense Res. Program

Radioprotective Mechanisms of

CBLB502$1,589,106 1/11-4/12

DoD/CBMS-JPEO

Chemical Biological Medical

Systems Joint Project Mgt.

RFP W9113M-09-R-0010

Advanced Development of a

Medical Radiation

Countermeasure

$1,343,759 6/11-9/13

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CBLB502Medical Applications

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CBLB502 in Preclinical Model of Local Irradiation

Toll-like Receptor 5 Agonist Protects Mice from Dermatitis and Oral Mucositis Caused by Local Radiation: Implications for Head

and Neck Cancer Radiotherapy.

(Int. J. Rad. Onc. Biol. Phys., in press)

Result:

• CBLB502 efficacious against radiation-induced mucositis and dermatitis

Significance:

• Strong preclinical support of CBLB502 as radiotherapy adjuvant

• Justification of new application (protection from radiation-induced dermatitis)

Approval of “CBLB502 as supportive care” trial protocol in head and neck cancer patients by Scientific Review Committee of Roswell Park

Head and neck irradiation model in mice

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Extending Indications of CBLB502Mitigation of chemotherapy side effects and direct antitumor action

Irinotecan and CBLB502 against Wart colon tumors in Fisher rats

CBLB502 displays both supportive care and direct antitumor activities in rat model of colon cancer

0 4 8 12 16 20 24 28 32Me

an

Tu

mo

r W

eig

ht

(mg

)

200

500

2000

5000

100

1000

10000

Antitumor activity and toxicity of Irinotecan ± CBLB502

in rats bearingadvanced Ward colorectal carcinoma

Time (Days)

0 4 8 12 16 20 24 28 32

Me

an

Bo

dy

We

igh

t (%

)

80

85

90

95

100

105

110

Control

CBLB502 0.2 mg/kg x 5

Irinotecan 200 mg/kg x 3

CBLB502 0.2 mg/kg(5) + Irinotecan 200 mg/kg

CBLB502 0.2 mg/kg(3) + Irinotecan 200 mg/kg

Irinotecan 200 mg/kg + CBLB502 0.2 mg/kg

"Toxicity"

"Antitumor Activity"

Irinotecan daily x3, 200 mg/kg i.v. +/- CBLB502

0 4 8 12 16 20 24 28 32 36 40 44 48 52Me

an

Tu

mo

r W

eig

ht

(mg

)

200

500

2000

5000

100

1000

10000

Indivadual rat bearing advanced Ward colorectal carcinoma

response to CBLB502 0.2 mg/kg/day by i.p. daily x 5

Time (Days)

0 4 8 12 16 20 24 28 32 36 40 44 48 52

Me

an

Bo

dy

We

igh

t (%

)

90

95

100

105

110

115

Rat # 1

Rat # 2

Rat # 3

Rat # 4

"Toxicity"

"Antitumor Activity"

0

CBLB502, x3 daily, 0.2 mg/kg

Irinitecan+CBLB502

placebo

Irinitecan alone

(all dead from GI toxicity)

CBLB502 rescues animals from Irinotecan toxicity with no

interference with its antitumor activity

CBLB502 caused complete regression of tumors in part

of the animals

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Many of these trials enable assessment of both supportive care and direct anti-tumor activity of CBLB502

Prospective Clinical Trials of CBLB502 in Cancer Patients

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• Reducing severity of mucositis and enhancing efficacy of radiotherapy of H&N

cancer

• Reducing severity of bowel toxicity and enhancing efficacy of radiotherapy of

pancreatic cancer

• Reducing severity of diarrhea in colon cancer patients treated with Irinotecan

• Treating primary hepatocellular carcinoma (liver cancer)

• Treating liver metastasis of colon cancer

• Treating liver metastasis of breast cancer

• Pre-operational treatment of prostate cancer

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CBLB612Stem Cell Inducing Agent

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CBLB612 is 6x more efficacious than G-CSF and induces both early and late progenitor cells

Effects of CBLB612 and G- CSF are synergistic

CBLB612 Induces Propagation of HSCs

CBLB612 or

G-CSF

CBLB612 or

G-CSF

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Dramatic improvement of blood recovery during Cyclophosphamide treatment in mice

CBLB612 Supportive Care During Chemotherapy

WBC

0.00

5.00

10.00

15.00

20.00

25.00

30.00

10x3/u

l

CBLB612 10.33 2.66 3.09 14.73

PBS 9.65 1.98 0.43 7.21

day -5 day7 day14 day22

White Blood Cells

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Principle efficacy assessment in Phase I = potential partnering

CBLB612 Product Development Strategy

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• 6-month Phase I safety study in healthy volunteers enables accurate estimate of induction and mobilization of stem cells in peripheral blood, a direct predictor of efficacy of the drug

• 2.5 years from today to critical human data

• Hisun licensing deal of 2009 provides additional data and possibility of synergistic development

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CuraxinsAnticancer drugs

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Page 24: Rodman & Renshaw 13th Annual Healthcare …content.stockpr.com/cbiolabs/media/d8f3658ea8604a40fab3c...• Phase I trial with oral formulation of next generation CBLC137 in solid tumors

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Curaxins

• Synthetic small molecules with proprietary structure

• Unique mechanism of action: simultaneously affect multiple molecular targets in cancer cell

• Efficacious in a broad spectrum of preclinical tumor models

• Mechanism of action enables additional clinical indications beyond cancer treatment (anti-inflammatory, anti-infective)

• Recent peer review publications:• Science Translational Medicine (2011)• Journal of Virology (2010)• Cell Cycle (2009)• Oncogene (2009)

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Incuron – JV for Curaxin Development

• 50/50 joint venture with Bioprocess Ventures, Moscow

• ~$18M to reach inflection points for primary molecules

• CBLI oversees mechanistic studies and formal development

• Phase Ib trial for prototype CBLC102 in liver metastases started October 2010 in Russia

• Phase I trial with oral formulation of next generation CBLC137 in solid tumors planned for 1Q12 in Russia

• Optimization of IV formulation of next generation CBLC137 for future trial in US ongoing

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Milestones

• Start of pivotal animal efficacy studies for CBLB502 defense

• Start of definitive safety/dose validation trial in healthy volunteers for CBLB502 defense

• CBLB502 trial as single agent in advanced cancer patients

• CBLB502 trial as supportive care in head and neck cancer patients

• Completion of CBLC102 trial in liver metastases patients in Russia

• Phase I trial next generation Curaxin CBLC137

• High profile peer reviewed publications

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Financial Summary

• Shares Outstanding: 35M common, 52M fully diluted

• Government Grants & Contracts support CBLB502 for defense and limited medical applications: $14.8M unspent as of 6/30/11 (excl. $30M option for first purchase)

• CBLI subsidiary Incuron funds Curaxin development for next 2-3 years: $12.1M left

• CBLI Cash & Receivables (at 6/30/11): $29.5M (CBLI only), $2.3M (Incuron – first tranch)

• Avg. Monthly Burn Rate (on CBLI cash): ~ $1-1.4M (CBLI only)

Page 28: Rodman & Renshaw 13th Annual Healthcare …content.stockpr.com/cbiolabs/media/d8f3658ea8604a40fab3c...• Phase I trial with oral formulation of next generation CBLC137 in solid tumors

• Scientist and entrepreneur

• Founder of Dia-M and The Fellowship for Interpretation of Genomes (FIG)

• Founder and Former CEO of Integrated Genomics, Inc. (‘97-03)

Michael Fonstein, PhD

Chief Executive Officer & President

• SVP of Basic Science, Roswell Park Cancer Institute

• Former Chair, Dept. Molecular Biology at Cleveland Clinic

• 30+ issued patents

• 150+ research publications

Andrei Gudkov, PhD, D.Sci

Chief Scientific Officer

• 30 years of financial and operations management and accounting experience

• 6 years as CFO of a public biotech company

• 15 years experience in federal contracting

Neil Lyons, CPA

Chief Financial Officer

• Former Director of Business Development at Integrated Genomics, Inc.

• Expert in technical sales and contract negotiations

Yakov Kogan, PhD, MBA

Chief Operating Officer

Senior Management Team

• 25 years global oncology drug development experience

• Senior positions in clinical operations at CROs

• Led clinical development in several publicly traded biotech companies

Michael Kurman, MD

Chief Medical Officer

• Over 20 years of regulatory experience at large and small pharma

• Multiple successful NDAs, MAAs, sNDAs, advisory committees

Ann Hards, PhD

Executive Vice President, Regulatory Affairs and Quality Assurance

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Scientific Advisory Board

George R. Stark, PhDChairman of SAB, Member of NAS, Former director of LRI, Scientific Advisor to Amersham and Genentech, pioneered numerous major research technologies

Inder Verma, PhDMember of NAS, Professor of Salk Institute, Founder and Scientific Advisor to Cell Genesys, Signal Pharmaceuticals, UroGenesys, Ventana Pharmaceuticals, Quark Biotech. Internationally recognized leader in cancer biology and inflammation

Bruce Blazar, MDProfessor, Chair in Transplantation Immunology of University of Minnesota. Member of the FDA Advisory Committee, SAB member of BioMarin Pharmaceutical, Seattle Genetics, etc.

Board of Directors

Independent Directors

Bernard L. Kasten, MDFormer CEO, SIGA Technologies

David Hohn, MDFormer President of Roswell Park Cancer Institute

James Antal, CPA, MBAFormer CFO and CIO of Experian

Paul DiCorleto, PhDChairman, Lerner Research Institute

Management

Michael Fonstein, PhDCEO & President, Cleveland BioLabs, Inc.

Andrei Gudkov, PhD, DSciCSO, Cleveland BioLabs, Inc

Yakov Kogan, PhD, MBACOO, Cleveland BioLabs, Inc

Boards

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