th Annual BIO CEO & Investor Conference February 9,...

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12 th Annual BIO CEO & Investor Conference February 9, 2010

Transcript of th Annual BIO CEO & Investor Conference February 9,...

Page 1: th Annual BIO CEO & Investor Conference February 9, 2010content.stockpr.com/cbiolabs/media/3277457b82af346ee... · 2012-03-05 · 19 studies with over 700 primates tested dose- ...

12th Annual BIO CEO & Investor ConferenceFebruary 9, 2010

Page 2: th Annual BIO CEO & Investor Conference February 9, 2010content.stockpr.com/cbiolabs/media/3277457b82af346ee... · 2012-03-05 · 19 studies with over 700 primates tested dose- ...

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

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CBLI is developing two families of drugs:

Protectans: selectively protect healthy tissues from radiation damage

Curaxins: kill tumor cells

Mission

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CBLI Target Product Market Opportunities

• CBLB502: Protection from Acute Radiation Syndrome (ARS)– >$500 million + annually

• CBLB502: Reduction of cancer treatment side effects~$20 billion market (70% of patients experience regimen-limiting

toxicity )

• CBLB612: Stem cell induction, mitigation of cancer treatment side effects– Potential to compete with G-CSF ($5+ billion drug from

Amgen)

• Curaxins: Broad range anti-cancer drugs– $50 billion growing market

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There is no approved drug which can effectively protect from ARS

The Threat - Nuclear Attack

• A nuclear event has been identified by US Congress as a number one security threat

• A terrorist attack with a 10 KT device will kill 400,000 people in NYC (Institute of Medicine Report, June 2009)

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Recent Milestones – CBLB502

• Phase I ascending-dose safety trial successfully concluded June 2009

• $23 million in development contracts from DoD and BARDA/HHS and NIAID/NIH received in 2008 (~$13 million left)

• $8 million in new funding in September 2009 including $5.3 stimulus GO grant (proposals for ~$30 million in additional funds pending)

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CBLB502: Mechanism of Action

CBLB502 protects from both gastrointestinal and hematopoietic components of radiation

death

NF-kBCBLB502 TLR5IAPs, Bcl-2SOD2, ferritinCytokines

Suppress apoptosisInactivate ROSPromote regeneration

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CBLB502 mobilizes multiple mechanisms of radiation defense, all stemming from TLR5

activation

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TBI, Gy InjectionsN

totalN

survivors% survival

P-value vs. vehicle

6.5 CBLB502, 0.04 mg/kg @+1h-+48h (pooled) 44 30 68.2% 0.002

6.5 Vehicle (PBS) @+1h, +25h (pooled) 18 4 22.2% -

Days after 6.5 Gy gamma-TBI0 10 20 30 40

% o

f sur

vivo

rs

0

20

40

60

80

100

vehicle (PBS), n=8CBLB502 @ +16h, n=12CBLB502 @ +25h, n=10CBLB502 @ +48h, n=12

CBLB502 Efficacy: Survival (Mitigation) after LD70 IR

Effective when injected up to 48 hours after radiat ion

Non-human primates

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Animal Efficacy Rule – Path to FDA licensure

Established FDA pathway to approve drugs where efficacy is unethical to test in humans

Dramatically reduces development time and costs

• Compliance with CMC requirements

• Efficacy in two animal species including rhesus macaques using survival as endpoint

• Safety in healthy humans

• Well understood mechanism of action (to provide biomarkers of efficacy)

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Cell Bank

Test ResidualsQC protocols, stability

Formulation

Bioassay

ManufacturingProduction Strain, DSP

Preclinical & ClinicalStudies

Laboratory Strain

FermentationImprovement

MBF

CBLB502 – ARS: CMC Effort

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GMP process developedDrug released

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TBI Dose ParameterTime relative to TBI (if applicable)

-45' +1h +16h +25h +48h +72h

LD60-70

Survival benefit +40% +45% +45% +40%-60% +45% TBD

Thrombocytopenia reduction +++ +++ +++ +++ +++ +++

Neutropenia reduction + ++ ++ ++ + +

Improved BM, spleen, thymus +++ +++ +++ +++ +++ TBD

Improved GI mucosa ++ +++ Ongoing Ongoing Ongoing TBD

Cytokine release (G-CSF, IL-6, etc.) +++ +++ +++ +++ +++ TBD

Data on dose dependence of efficacy √ √ TBD Ongoing TBD TBD

LD10-20

Thrombocytopenia reduction +++ +++ +++ +++ +++ ++

Neutropenia reduction + ++ ++ ++ + +

Cytokine release (G-CSF, IL-6, etc.) +++ +++ +++ +++ +++ ++

Data on dose dependence of efficacy Ongoing √ TBD TBD TBD TBD

No TBI

Increased platelet levels +++

Increased neutrophil levels ++

Cytokine release (G-CSF, IL-6, etc.) +++

Data on dose dependence of efficacy √

+++: strong effect; ++: moderate effect; +: minor effect; √√√√: data collected

CBLB502 – ARS: Summary of Primate Studies

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19 studies with over 700 primates tested dose-

dependence for the drug, efficacy time window and effects

of various radiation doses

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• 50 human volunteers in groups of 6 received ascending doses of the drug

• Trial completed; database locked mid-July

• Dose limiting toxicity (DLT) defined

• Adverse event profile described; predictable and related to the known pharmacology of CBLB502

• Predicted safe dose in humans exceeds protective dose in primates (based on biomarkers)

• All biomarkers project similar human dose

Summary of Phase I trial of CBLB502

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- - Human safety is the last remaining developmental milestone: Phase IA study completed in Q3 2009

- - Expect to complete all other remaining requirement s needed for FDA in Q4 2010

Manufacturing&

Clinical/Regulatory

Formulation

Non-ClinicalSafety

Non-ClinicalEfficacy

Manufacturing of Consistency Lots

Reprotox Studies in Rabbits & Rats-Seg. II

Phase II Safety TrialPh IIA (100 subjects) & Ph IIB (~500 subjects)

Development & Stability Studiesof Lyophilized Formulation

Reprotox Studies in Rats-Seg. I&III

GMP manufacturing (>100,000 doses)

INDFiled

Pivotal GLP Mouse& Primate Efficacy Studies to support NDA filing

GLP Acute Mouse & PrimateToxicology Studies

Process validation

Dose EscalationTrial - Phase Ia(50 volunteers)

10/092004 Q4’10

Efficacy in Mice, Rats and Primates

� CompletedHuman Safety Data

BLAFiling

Federal grant support fromDoD and HHS/BARDA

Stability Studies of the Final Product

GLP 2-weekMouse & PrimateToxicology Studies

Mechanism of Action;Markers of Efficacy

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Protectan CBLB502: Development Timeline

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CBLB502 - ARS Market Potential “Back of the Envelope ”

• DoD - 2 million potential doses for US military

• BARDA/HHS - 5-20 million potential doses for protection of US civilians (Strategic National Stockpile)

Primary sales targets : Need understood, concepts of use developed, high degree of financial commitment, relations with CBLI in place

Secondary sales targets : Serious public concern, policies being developed

Projected addressable market ~$500 M/year (w/penetration)

No competing products todayRFP from DoD indicates commitment to procurement

• Israel, UK, Canada, India, China, Japan, S. Korea

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CBLB502: Protection from Local Irradiation

Strong mitigation of radiological damage of healthy tissues shown in mouse model of head-and-neck

damage directly supports first medical trial

3x10 Gy daily with CBLB502 pretreatment

0 5 10 1560

70

80

90

100

110 10 Gy x3 (CBLB502 + 10 Gy) x3

Days

Bod

y w

eigh

t, %

3x10 Gy daily

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CBLB502: Phase I/II Head & Neck Human Trial

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• Open IND

• Funded by $5.3 million stimulus grant received September 2009

• Protocol submitted to IRB

• Planned to start at Roswell Park Cancer Institute 1Q 2010

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Protectan CBLB612Stem Cell Inducing Agent

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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

/ul

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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CBLB612: Product Development Strategy

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

Principle efficacy assessment in Phase I = potential partnering

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Zhejiang Hisun Pharmaceutical license for China signed Sept. 2009

($1.65M upfront development, 10% royalties)

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Curaxin Product LineAnti-cancer drugs

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Curaxins: Overview

• First-in-class broad-spectrum anticancer drugs

• Small molecules suitable for oral administration

• Novel mechanism of action – simultaneous targeting three major pathways deregulated in cancer

• Composition of matter patent applications

• Efficacy in multiple animal models of major cancer types including breast and prostate cancer

• Proof of concept Phase II trial in prostate cancer

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INCURON – JV for Curaxin Development

• 50/50 joint venture with Bioprocess Ventures, Moscow

• ~$18M to reach Phase II for new generation of Curaxins

• CBLI to serve as subcontractor to oversee mechanistic studies and clinical development

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CBLI Summary

IP

• Cleveland Clinic (CCF)• Roswell Park Cancer Institute (RPCI)

Partnerships

• Money raised from capital market $51 million• Federal grants and contracts >$50 million

Funding History

• Incorporated in June 2003 Spin-off from the Cleveland Clinic• HQ - Buffalo, NY 32 employees (majority PhDs & MDs)

• ~16 sets of patent applications filed• First CBLB502 US patent granted

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Financial Summary

• Mcap (at 1/29/10): $78M

• Shares Outstanding:– 20M common

– 36M fully diluted

• Govt. Grants & Contracts committed to support CBLB502 for defense and medical applications (at 9/30/09): $21.8M

– Pending proposals for additional grants and contracts to support all CBLI programs: $37.5M

• CBLI Cash & Rcvbles (at 9/30/09): $5.2M

• Avg. Monthly Burn Rate (CBLI cash):– $200,000

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• Scientist and serial 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, PhDChief 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.SciChief Scientific Officer

• 20 years of financial and accounting experience

• 8 years as a corporate controller of a public company

Jack Marhofer, MBA, CMA, CFMChief Financial Officer

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

• Expert in technical sales and contract negotiations

Yakov Kogan, PhD, MBAChief Operating Officer

CBLI 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, MDChief Medical Officer

• 30 years of Pharma experience

• Former Director of Drug Safety at TAP Pharmaceuticals, Inc.

Farrel Fort, PhD, MBA, DABTVice President, Drug Development

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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 DirectorsBernard L. Kasten, MD

Former CEO, SIGA Technologies

Daniel Perez, MDFormer CEO & President of Berlex Biosciences, a Division of Bayer AG

James Antal, CPA, MBAFormer CFO and CIO of Experian

Paul DiCorleto, PhDChairman, Lerner Research Institute

ManagementMichael Fonstein, PhD

CEO & President, Cleveland BioLabs, Inc.

Andrei Gudkov, PhD, DSciCSO, Cleveland BioLabs, Inc

Yakov Kogan, PhD, MBACOO, Cleveland BioLabs, Inc

CBLI Boards

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