Presentation Transcript

1. ImmunoCellular Therapeutics

   Industry-leading, next-generation, cancer immunotherapy November 2012
2. Disclaimer 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 www.sec.gov. 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.
3. Disruptive Validated Technology Cancer Stem Cell Targeting Potent Immunotherapy + = Effective Cancer Eradication
4. Why Cancer Vaccines Previously Failed? Problem Solution 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
5. Without killing CSCs, it is like spraying for weeds without killing the roots. The weeds (tumors) come back.
6. Immunotherapy Has 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 Antibody MHC Antigen Antigen Cancer cell Cancer cell Cytotoxic T-cell
7. Product Pipeline OverviewMultiple 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
8. ICT-107 Preparation & ManufacturingMultiple doses from only one apheresis procedure GMP Manufacturing Facility Shipovernight Culture with cytokines Apheresis  Apheresis product Peripheral blood mononuclear cells Activated dendritic cells Pulse w/ tumor-associated antigens Patient  Intradermal injection Shipback to physician Aliquot & freeze ~30 doses ICT-107 ICT-107
9. ICT-107 targets both tumor cells and CSCs
10. 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)
11. Expression of Tumor Antigens in GBM by RT-PCR All GBM patients express three or more antigens 75% expressed all six Patients from ICT-107 Phase I clinical trial
12. Phase I Trial with ICT-107 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
13. Pre- and Post-Operative MRI Scans of Four GBM Patients on ICT-107
14. ICT-107 Phase I ResultsNewly diagnosed GBM patients (efficacy and safety) RT/Chemo Vaccine Progressive disease Historical Fully-resected ICT-107 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.
15. ICT-107 Improves Survival in GBM Progression Free Survival (PFS) Overall Survival (OS) ICT-107 ICT-107 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.
16. Correlation of PFS and OS with Antigen Expression
17. CD133 Expression (CSC Biomarker)Primary & recurrent tumor samples from the same patient Phuphanich et al. Cancer Immunol Immunother. 2012 Jul 31.
18. ICT-107 Phase II Trial DesignRandomized, 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 + TMZ Newly Diagnosed GBM Patients (n=123) 2:1 randomization Placebo Unloaded DCs + TMZ Surgery Apheresis Vaccinations Radiation TMZ 7 weeks 6 weeks 4 weeks 4 weeks 4 weeks 4 weeks 4 weeks
19. ICT-107 Phase II Trial Enrollment25 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
20. FDA ApprovalsNewly Diagnosed GBM Gliadel Approval in 2003 Double-blind, placebo-controlled, randomized Phase III trial showing 13.8 vs. 11.6 month survival Temozolomide Approval in 2005 Double-blind, placebo-controlled, randomized Phase III trial showing 14.6 vs. 12.1 month survival
21. Projected Costs: ICT-107 vs. ProvengeLower cost of goods, better logistics Source: Quarterly earnings transcripts and public filings.
22. Other Immunotherapy Candidates
23. ICT-140: Ovarian 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
24. ICT-121: CSC-targeted Universal Vaccine 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
25. Inverse Correlation between 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.
26. Strong IP Position 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
27. Experienced Management Team 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
28. Product Pipeline 2011 2012 2013 2014 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Phase II trial ICT-107 New GBM Interim analysis Final results Preclinical Phase I/II trial ICT-140 Ovarian IND Preclinical Phase I trial ICT-121 Recurrent GBM IND
29. Recent & Upcoming Milestones 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
30. Strong Financial Position & Capitalization

31. ImmunoCellular Therapeutics

    Industry-leading, next-generation, cancer immunotherapy November 2012