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Immunocellular therapeutics

ImmunoCellular Therapeutics

Industry-leading, next-generation, cancer immunotherapy

November 2012


This presentation contains certain “forward-looking statements” (statements as to matters other than historical facts) as defined in the Private Securities Litigation Reform Act of 1995. These statements involve risks and uncertainties that could cause actual events or results to differ materially from the events and include statements about our plans, objectives, expectations and intentions with respect to the potential for success of our scientific approach to cancer immunotherapy, clinical development efforts, operations, financial condition and other statements that are not historical in nature, particularly those that use terms such as “will,” “potential”, “could,” “can,” “believe,” “intends,” “continue,” “plans,” “expects,” “projects,” “estimates” or similar language.

Important factors known to us that could cause actual results to differ materially from those expressed in such forward-looking statements include those set forth in our most recent annual report on Form 10-K, quarterly reports on Form 10-Q and other reports filed with the SEC.

You may obtain these documents for free by visiting EDGAR on the SEC website at The information in this presentation speaks only as of the date hereof, and except as required by law, we disclaim any obligation to update or revise any forward-looking statement.

Disruptive validated technology
Disruptive Validated Technology

Cancer Stem Cell Targeting

Potent Immunotherapy


= Effective Cancer Eradication

Why cancer v accines p reviously f ailed
Why Cancer Vaccines Previously Failed?



Minimal residual disease

Immune competent patients at diagnosis

Dendritic cells with persistent T-cell immune response

  • Late-stage disease

  • Immune compromised patients

  • Weak immune response

  • Target multiple antigens

  • Overall survival endpoint

  • Target cancer stem cells

  • Tumor mutation/escape

  • Flawed trial endpoints

  • Targeted tumor bulk

Immunocellular therapeutics

Without killing CSCs, it is like spraying for weeds without killing the roots. The weeds (tumors) come back.

Immunotherapy has a dvantages in targeting c ancer s tem c ells
Immunotherapy Has killing the roots. The weeds (tumors) come Advantages in Targeting Cancer Stem Cells

  • Immunotherapy can elicit T-cell mediated rejection of tumors

    • T cells are the way the body kills cancer cells

    • Improves specificity

    • Targets intracellular & surface antigens

    • Better safety profile

    • Differentiates between CSCs and normal stem cells

Antibodies only target CSC antigens on the surface of cancer cells

Cytotoxic T-cells target CSC antigens cancer presented by MHCs





Cancer cell

Cancer cell

Cytotoxic T-cell

Product pipeline overview multiple therapies in different cancer indications
Product Pipeline Overview killing the roots. The weeds (tumors) come Multiple therapies in different cancer indications

Active immunotherapies

  • ICT-107

    • Dendritic cell vaccine targeting glioblastoma antigens and CSCs

    • Phase I trial showed compelling clinical outcomes

    • Phase II study results anticipated late 2013

  • ICT-140

    • Dendritic cell vaccine targeting ovarian cancer antigens and CSCs

    • IND filing Q4/2012

  • ICT-121

    • Dendritic cell vaccine targeting CD133 (CSC marker)

    • IND approved; plan enrollment Q4/2012

      Antibody immunotherapies

  • Licensed to Caerus Molecular Discovery, funded by BioWa

Ict 107 preparation manufacturing multiple doses from only one apheresis procedure
ICT-107 Preparation & Manufacturing killing the roots. The weeds (tumors) come Multiple doses from only one apheresis procedure

GMP Manufacturing Facility


Culture with cytokines




Peripheral blood mononuclear cells

Activated dendritic cells

Pulse w/ tumor-associated antigens


Intradermal injection

Shipback to physician

Aliquot & freeze

~30 doses



Immunocellular therapeutics

ICT-107 targets both tumor cells and CSCs killing the roots. The weeds (tumors) come

Ict 107 targets antigens overexpressed on glioblastoma cancer stem cells
ICT-107 Targets Antigens Overexpressed on Glioblastoma Cancer Stem Cells

ICT-107 targets six tumor antigens (nine amino acid epitopes that elicit an immune response in HLA-A1/A2 patients)

Immunocellular therapeutics

Expression of Tumor Antigens in GBM by RT-PCR Cancer Stem Cells

All GBM patients express three or more antigens

75% expressed all six

Patients from ICT-107 Phase I clinical trial

Phase i trial with ict 107
Phase I Trial with ICT-107 Cancer Stem Cells

  • Patients received standard of care (surgery and chemo-radiation) followed by three vaccinations of ICT-107 every two weeks.

Nonrandomized, single-center study at Cedars-Sinai

  • 19 GBM patients

  • 16 newly diagnosed, 3 recurrent

  • ~75% fully resected

Pre and post operative mri scans of four gbm patients on ict 107
Pre- and Post-Operative MRI Scans of Cancer Stem CellsFour GBM Patients on ICT-107

Ict 107 phase i results newly diagnosed gbm patients efficacy and safety
ICT-107 Phase I Results Cancer Stem CellsNewly diagnosed GBM patients (efficacy and safety)



Progressive disease




Six patients without recurrence for over 4 years (3 of them over 5 years)

No Grade 3 or 4 toxicities.

Adverse events (Grade 1 or 2) include diarrhea, fatigue, flushing, pruritis, rash, vomiting

= Death

Stupp et al. N Engl J Med. 2005 Mar 10;352(10):987-96 & Stupp et al.LancetOncol. 2009 May;10(5):459-66.

Ict 107 improves s urvival in gbm
ICT-107 Improves Cancer Stem CellsSurvival in GBM

Progression Free Survival (PFS)

Overall Survival (OS)



Historical standard of care

Historical standard of care

  • Significant increase in median PFS

    • 16.9 months for ICT-107

    • 6.9 months for historical SoC*

  • Significant increase in median OS

    • 38.4 months for ICT-107

    • 14.6 months for historical SoC*

*Surgery followed by radiation and temozolomide (TMZ). Stupp et al. N Engl J Med. 2005 Mar 10;352(10):987-96.

Cd133 expression csc biomarker p rimary recurrent tumor samples from the same patient
CD133 Expression (CSC Biomarker) Cancer Stem CellsPrimary & recurrent tumor samples from the same patient

Phuphanich et al. Cancer Immunol Immunother. 2012 Jul 31.

Ict 107 phase ii trial design randomized placebo controlled double blind trial
ICT-107 Phase II Trial Design Cancer Stem CellsRandomized, placebo-controlled, double-blind trial

  • 123 patients treated at 25 centers

    • HLA-A1/A2 50-75% of US population

  • 278 patients enrolled

  • Primary endpoint: OS

  • Secondary endpoints:

    • PFS

    • OS/PFS at various time intervals

    • Immune response (T-cells)

    • Safety

  • Interim analysis (based on 50% events) in Q1/2013

  • Final results in 2H2013

  • Derisked by improving DC function, timing, frequency

  • ICT-107 +


    Newly Diagnosed GBM Patients (n=123)

    2:1 randomization


    Unloaded DCs + TMZ






    7 weeks

    6 weeks

    4 weeks

    4 weeks

    4 weeks

    4 weeks

    4 weeks

    Ict 107 phase ii trial enrollment 25 clinical trial sites 278 patients enrolled
    ICT-107 Phase II Trial Enrollment Cancer Stem Cells25 clinical trial sites – 278 patients enrolled

    • Johns Hopkins University

    • New York University

    • University of Texas at Houston

    • Northwestern University

    • Arizona Cancer Center

    • New Jersey Neuroscience Institute

    • UC San Diego

    • Moffitt Cancer Center

    • Penn State

    • University of Pennsylvania

    • University of Virginia

    • Wake Forest

    • Cornell Presbyterian

    • Massachusetts General

    • Kentuckiana Cancer Institute

    • Cedars-Sinai Medical Center

    • University Hospital Case Medical Center

    • Rush University

    • Overlook Hospital

    • Baylor University

    • Cleveland Clinic

    • University of Alabama

    • Thomas Jefferson

    • Long Island Brain Center

    Fda approvals newly diagnosed gbm
    FDA Approvals Cancer Stem CellsNewly Diagnosed GBM


    • Approval in 2003

    • Double-blind, placebo-controlled, randomized Phase III trial showing 13.8 vs. 11.6 month survival


    • Approval in 2005

    • Double-blind, placebo-controlled, randomized Phase III trial showing 14.6 vs. 12.1 month survival

    Projected costs ict 107 vs provenge lower cost of goods better logistics
    Projected Costs: ICT-107 vs. Cancer Stem CellsProvengeLower cost of goods, better logistics

    Source: Quarterly earnings transcripts and public filings.

    Ict 140 ovarian cancer vaccine
    ICT-140: Cancer Stem CellsOvarian Cancer Vaccine

    Ovarian cancer is similar to GBM

    • Minimal residual disease after surgery

    • Immuno-responsive

      Dendritic cell vaccine targeting CSCs

    • Seven antigens over-expressed in ovarian cancer, including three antigens used in ICT-107

      • HER2/neu, IL-13Rα2, MAGE1, mesothelin, EphA2, & two more antigens

    • File IND by Q4/2012

    Ict 121 csc targeted universal vaccine
    ICT-121: CSC-targeted Universal Vaccine Cancer Stem Cells

    • Dendritic cell vaccine loaded with two CD133 peptides

    • CD133 is highly expressed on CSCs

    • CD133 is expressed on most solid tumors, including brain, colon, non-small cell lung, melanoma, pancreatic, and breastcancer

    • Initial indication in recurrent GBM

    • PI-sponsored Phase I trial at Cedars-Sinai Medical Center

      • 20 patients

      • IND approved; plan

        enrollment Q4/2012

    Inverse correlation between cd133 expression with survival on gliomas
    Inverse Correlation Cancer Stem Cellsbetween CD133 Expression with Survival on Gliomas

    CD133 expression correlates inversely with grade II to IV glioma patient survival time. The survival time calculated from the day of operation was plotted against the percentage of CD133+ cells in the CD45-cell fraction from the specimens of each patient. UD: undetectable CD133 expression. Bold black bars indicate the median survival time for patients in groups with CD133+ cells either lower or higher than 30% of total CD45-cells.

    Source: Rebetz et al. PLoS ONE. 2008.

    Strong ip position
    Strong IP Position Cancer Stem Cells

    28+ patents and patent applications

    • 10 patents issued or allowed

    • 18+ patents pending

      Vaccine patents and applications include

    • Method of use for six antigen vaccine (ICT-107)

    • Manufacturing process for production of ICT-107

    • Use of dendritic cells with chemotherapy for neural cancers

    • Immunotherapy targeting IL-13Rα2

    • Immunotherapy targeting CD133

      Issued patents on monoclonal antibodies cover composition of matter, therapeutic treatments and diagnostics

    Experienced management team
    Experienced Management Team Cancer Stem Cells

    Experience in developing over 20 products in cell & gene therapy and vaccines

    John Yu, MD, Chairman & CSO, Interim CEO

    • Neurosurgeon at Cedars-Sinai, Mass General Hospital, Harvard Medical School

      Elma Hawkins, PhD, Head of Clinical Development

    • Antigenics, Genzyme, Warner Lambert/Parke Davis

      Jim Bender, PhD, MPH, VP of Manufacturing & Product Development

    • IDM Pharma, Baxter Healthcare

      David Fractor, CPA, CFO

    • HemaCare, Andwin, Deloitte & Touche

      Peter Ho, PhD, Director of Business Development

    • Grey Healthcare Group, Prudential Equity Group, Allergan, D.E. Shaw

    Product pipeline
    Product Pipeline Cancer Stem Cells





















    Phase II trial


    New GBM

    Interim analysis

    Final results


    Phase I/II trial





    Phase I trial


    Recurrent GBM


    Recent upcoming milestones
    Recent & Upcoming Milestones Cancer Stem Cells

    May 2012

    NYSE MKT listing

    July 2012

    ICT-121 IND

    October 2013

    SITC abstract

    October 2012

    SITC abstract

    2H 2013

    ICT-107 Ph 2 final

    December 2012

    ICT-121 Ph 1

    Q3 2013

    ICT-140 Ph 1/2

    June 2013

    ASCO abstract

    November 2013

    SNO abstract

    Q1 2013

    ICT-107 Ph 2 interim

    ICT-140 IND

    November 2012

    SNO abstract

    August 2012

    ICT-107 Ph 2 enrollment

    Immunocellular therapeutics1

    ImmunoCellular Therapeutics Cancer Stem Cells

    Industry-leading, next-generation, cancer immunotherapy

    November 2012