Presentation on theme: "November 2012 ImmunoCellular Therapeutics Industry-leading, next-generation, cancer immunotherapy."— Presentation transcript:
November 2012 ImmunoCellular Therapeutics Industry-leading, next-generation, cancer immunotherapy
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Disruptive Validated Technology Cancer Stem Cell TargetingPotent Immunotherapy 3 = Effective Cancer Eradication +
Without killing CSCs, it is like spraying for weeds without killing the roots. The weeds (tumors) come back.
Immunotherapy Has Advantages in Targeting Cancer Stem Cells 6 Cancer cellCytotoxic T-cell Cytotoxic T-cells target CSC antigens cancer presented by MHCs Antigen MHC 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 Cancer cell Antibody Antigen
Product Pipeline Overview 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 7
ICT-107 Preparation & Manufacturing Multiple doses from only one apheresis procedure GMP Manufacturing Facility Apheresis Patient Apheresis product Ship back to physician ICT-107 ~30 doses Peripheral blood mononuclear cells Activated dendritic cells Culture with cytokines Intradermal injection Pulse w/ tumor- associated antigens Aliquot & freeze Ship overnight 8
9 ICT-107 targets both tumor cells and CSCs
ICT-107 Targets Antigens Overexpressed on Glioblastoma Cancer Stem Cells gp100 MAGE-1 IL-13R α 2 HER2/neu AIM2 TRP-2 EGFRvIII HSP Ags Tumor Lys ICT-107 (IMUC) CDX-110 (Celldex) DC Vax (Northwest Bio) Prophage (Agenus) 10 ICT-107 targets six tumor antigens (nine amino acid epitopes that elicit an immune response in HLA-A1/A2 patients)
Expression of Tumor Antigens in GBM by RT-PCR All GBM patients express three or more antigens 75% expressed all six 11 Pt #Pt IDAIMgp100MAGETRP-2Her-2IL-13R wk wk<1E wk >1E wk >1E wk neg+++>1E >1E wk >1E wk wkneg wk wk wk wk wk wk wk wk neg neg++++neg wk wk wk neg neg wk negwkneg+++neg wk wk wk Patients from ICT-107 Phase I clinical trial
Phase I Trial with ICT-107 Nonrandomized, single-center study at Cedars-Sinai 19 GBM patients 16 newly diagnosed, 3 recurrent ~75% fully resected 12 Patients received standard of care (surgery and chemo-radiation) followed by three vaccinations of ICT-107 every two weeks.
Pre- and Post-Operative MRI Scans of Four GBM Patients on ICT
ICT-107 Phase I Results Newly diagnosed GBM patients (efficacy and safety) 14 = Death HistoricalFully-resected RT/ChemoVaccineProgressive disease 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 Stupp et al. N Engl J Med Mar 10;352(10): & Stupp et al. Lancet Oncol May;10(5):
ICT-107 Improves Survival in GBM *Surgery followed by radiation and temozolomide (TMZ). Stupp et al. N Engl J Med Mar 10;352(10): Historical standard of care ICT-107 Significant increase in median PFS 16.9 months for ICT-107 6.9 months for historical SoC* Progression Free Survival (PFS) Significant increase in median OS 38.4 months for ICT-107 14.6 months for historical SoC* Historical standard of care ICT-107 Overall Survival (OS) 15
Correlation of PFS and OS with Antigen Expression 16
CD133 Expression (CSC Biomarker) P rimary & recurrent tumor samples from the same patient 17 Phuphanich et al. Cancer Immunol Immunother Jul 31.
ICT-107 Phase II Trial Design Randomized, placebo-controlled, double-blind trial Newly Diagnosed GBM Patients (n=123) ICT TMZ Placebo Unloaded DCs + TMZ 2:1 randomization 123 patients treated at 25 centers HLA-A1/A % 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 7 weeks6 weeks4 weeks TMZ Radiation 4 weeks SurgeryApheresisVaccinations 18
ICT-107 Phase II Trial Enrollment 25 clinical trial sites – 278 patients enrolled 19 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 Gliadel Approval in 2003 Double-blind, placebo-controlled, randomized Phase III trial showing 13.8 vs month survival Temozolomide Approval in 2005 Double-blind, placebo-controlled, randomized Phase III trial showing 14.6 vs month survival 20
Projected Costs: ICT-107 vs. Provenge Lower cost of goods, better logistics ICT-107Provenge % DCs/APC60%-90%15%-20% Interleukin-12YesNo Target antigensSixOne Doses/apheresis~301 StorageLiquid nitrogenN/A AdministrationIntradermal injectionIV infusion Cost of Goods5%-10%70% 21 Source: Quarterly earnings transcripts and public filings.
Other Immunotherapy Candidates 22
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/
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 breast cancer Initial indication in recurrent GBM PI-sponsored Phase I trial at Cedars-Sinai Medical Center 20 patients IND approved; plan enrollment Q4/
Inverse Correlation between CD133 Expression with Survival on Gliomas Source: Rebetz et al. PLoS ONE 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.
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 26
Experienced Management Team 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 27 Experience in developing over 20 products in cell & gene therapy and vaccines
Product Pipeline Q1Q2Q3Q4Q1Q2Q3Q Q1Q2Q3Q Q1Q2Q3Q ICT-107 New GBM Phase II trial Phase I/II trial Phase I trial Preclinical ICT-140 Ovarian ICT-121 Recurrent GBM Interim analysis Final results IND
Recent & Upcoming Milestones 29 July 2012 ICT-121 IND October 2012 SITC abstract November 2012 SNO abstract August 2012 ICT-107 Ph 2 enrollment Q ICT-107 Ph 2 interim ICT-140 IND 2H 2013 ICT-107 Ph 2 final Q ICT-140 Ph 1/2 October 2013 SITC abstract November 2013 SNO abstract May 2012 NYSE MKT listing June 2013 ASCO abstract December 2012 ICT-121 Ph 1
Strong Financial Position & Capitalization Cash (as of 9/30/2012)$10 million (A) Burn rate$3 million per quarter Outstanding debtNone Shares outstanding (as of 9/30/12) 41.1 million (A) Market capitalization (as of 11/6/2012) $98 million Warrants outstanding (as of 9/30/12) 8.9 million (A) Options outstanding 10.4 million (average weighted exercise price of $1.15) (A) In October 2012, we raised $19.3 million from the issuance of 10 million shares of common stock and 4.5 million warrants. 30
November 2012 ImmunoCellular Therapeutics Industry-leading, next-generation, cancer immunotherapy