Presentation on theme: "ImmunoCellular Therapeutics"— Presentation transcript:
1ImmunoCellular Therapeutics Industry-leading, next-generation, cancer immunotherapyNovember 2012
2DisclaimerThis presentation contains certain “forward-looking statements” (statements as to matters other than historical facts) as defined in the Private Securities Litigation Reform Act of 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.
3Disruptive Validated Technology Cancer Stem Cell TargetingPotent Immunotherapy+= Effective Cancer Eradication
4Why Cancer Vaccines Previously Failed? ProblemSolutionLate-stage diseaseImmune compromised patientsWeak immune responseMinimal residual diseaseImmune competent patients at diagnosisDendritic cells with persistent T-cell immune responseTumor mutation/escapeFlawed trial endpointsTargeted tumor bulkTarget multiple antigensOverall survival endpointTarget cancer stem cells
5Without killing CSCs, it is like spraying for weeds without killing the roots. The weeds (tumors) come back.
6Immunotherapy Has Advantages in Targeting Cancer Stem Cells Immunotherapy can elicit T-cell mediated rejection of tumorsT cells are the way the body kills cancer cellsImproves specificityTargets intracellular & surface antigensBetter safety profileDifferentiates between CSCs and normal stem cellsCancer cellCytotoxic T-cellCytotoxic T-cells target CSC antigens cancer presented by MHCsAntigenMHCAntibodies only target CSC antigens on the surface of cancer cellsCancer cellAntibodyAntigen
7Product Pipeline Overview Multiple therapies in different cancer indications Active immunotherapiesICT-107Dendritic cell vaccine targeting glioblastoma antigens and CSCsPhase I trial showed compelling clinical outcomesPhase II study results anticipated late 2013ICT-140Dendritic cell vaccine targeting ovarian cancer antigens and CSCsIND filing Q4/2012ICT-121Dendritic cell vaccine targeting CD133 (CSC marker)IND approved; plan enrollment Q4/2012Antibody immunotherapiesLicensed to Caerus Molecular Discovery, funded by BioWa
8GMP Manufacturing Facility ICT-107 Preparation & Manufacturing Multiple doses from only one apheresis procedureGMP Manufacturing FacilityShip overnightCulture with cytokinesApheresisApheresisproductPeripheral blood mononuclear cellsActivated dendritic cellsPulse w/ tumor-associated antigensPatientIntradermal injectionShip back to physicianAliquot & freeze~30 dosesICT-107ICT-107
10ICT-107 Targets Antigens Overexpressed on Glioblastoma Cancer Stem Cells gp100MAGE-1IL-13Rα2HER2/neuAIM2TRP-2EGFRvIIIHSP AgsTumor LysICT-107(IMUC)CDX-110(Celldex)DC Vax(Northwest Bio)Prophage(Agenus)ICT-107 targets six tumor antigens (nine amino acid epitopes that elicit an immune response in HLA-A1/A2 patients)
11Expression of Tumor Antigens in GBM by RT-PCR All GBM patients express three or more antigens75% expressed all sixPt #Pt IDAIMgp100MAGETRP-2Her-2IL-13R1630++wk+++++++<1E51636+>1E51639>1E41640neg>1E31597+++++>1E21587>1E1154415761577155115521562156415401542151915221523152514121466215265135161431715088146891498111539121561131550141547151594161560171578181585191614Patients from ICT-107 Phase I clinical trial
12Phase I Trial with ICT-107Nonrandomized, single-center study at Cedars-Sinai19 GBM patients16 newly diagnosed, 3 recurrent~75% fully resectedPatients received standard of care (surgery and chemo-radiation) followed by three vaccinations of ICT-107 every two weeks.
13Pre- and Post-Operative MRI Scans of Four GBM Patients on ICT-107
14ICT-107 Phase I Results Newly diagnosed GBM patients (efficacy and safety) RT/ChemoVaccineProgressive disease= DeathHistoricalFully-resectedICT-107Six 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, vomiting14.6 historical, 18 fully-resected, 38.4 ICT-107Stupp et al. N Engl J Med Mar 10;352(10): & Stupp et al. Lancet Oncol May;10(5):
15ICT-107 Improves Survival in GBM Progression Free Survival (PFS)Overall Survival (OS)ICT-107ICT-107Historical standard of careHistorical standard of careSignificant increase in median PFS16.9 months for ICT-1076.9 months for historical SoC*Significant increase in median OS38.4 months for ICT-10714.6 months for historical SoC**Surgery followed by radiation and temozolomide (TMZ). Stupp et al. N Engl J Med Mar 10;352(10):
16Correlation of PFS and OS with Antigen Expression
17CD133 Expression (CSC Biomarker) Primary & recurrent tumor samples from the same patient To determine the impact of ICT-107 on CSCs, we looked at CD133 expression from patient samples before and after treatment.CD133 (CSC marker) expression by RT-PCR in primary tumor and samples from subsequent surgeries from newly diagnosed and recurrent patients. Expression is calculated relative to GADPH. The sample for Patient H was negative for tumor.Phuphanich et al. Cancer Immunol Immunother Jul 31.
18Newly Diagnosed GBM Patients (n=123) ICT-107 Phase II Trial Design Randomized, placebo-controlled, double-blind trial123 patients treated at 25 centersHLA-A1/A % of US population278 patients enrolledPrimary endpoint: OSSecondary endpoints:PFSOS/PFS at various time intervalsImmune response (T-cells)SafetyInterim analysis (based on 50% events) in Q1/2013Final results in 2H2013Derisked by improving DC function, timing, frequencyICT-107 +TMZNewly Diagnosed GBM Patients (n=123)2:1 randomizationPlaceboUnloaded DCs + TMZ7 weeks6 weeks4 weeksTMZRadiationSurgeryApheresisVaccinations
19ICT-107 Phase II Trial Enrollment 25 clinical trial sites – 278 patients enrolled Johns Hopkins UniversityNew York UniversityUniversity of Texas at HoustonNorthwestern UniversityArizona Cancer CenterNew Jersey Neuroscience InstituteUC San DiegoMoffitt Cancer CenterPenn StateUniversity of PennsylvaniaUniversity of VirginiaWake ForestCornell PresbyterianMassachusetts GeneralKentuckiana Cancer InstituteCedars-Sinai Medical CenterUniversity Hospital Case Medical CenterRush UniversityOverlook HospitalBaylor UniversityCleveland ClinicUniversity of AlabamaThomas JeffersonLong Island Brain Center
20FDA Approvals Newly Diagnosed GBM GliadelApproval in 2003Double-blind, placebo-controlled, randomized Phase III trial showing 13.8 vs month survivalTemozolomideApproval in 2005Double-blind, placebo-controlled, randomized Phase III trial showing 14.6 vs month survival
21Intradermal injection Projected Costs: ICT-107 vs. Provenge Lower cost of goods, better logisticsICT-107Provenge% DCs/APC60%-90%15%-20%Interleukin-12YesNoTarget antigensSixOneDoses/apheresis~301StorageLiquid nitrogenN/AAdministrationIntradermal injectionIV infusionCost of Goods5%-10%70%Source: Quarterly earnings transcripts and public filings.21
23ICT-140: Ovarian Cancer Vaccine Ovarian cancer is similar to GBMMinimal residual disease after surgeryImmuno-responsiveDendritic cell vaccine targeting CSCsSeven antigens over-expressed in ovarian cancer, including three antigens used in ICT-107HER2/neu, IL-13Rα2, MAGE1, mesothelin, EphA2, & two more antigensFile IND by Q4/2012
24ICT-121: CSC-targeted Universal Vaccine Dendritic cell vaccine loaded with two CD133 peptidesCD133 is highly expressed on CSCsCD133 is expressed on most solid tumors, including brain, colon, non-small cell lung, melanoma, pancreatic, and breast cancerInitial indication in recurrent GBMPI-sponsored Phase I trial at Cedars-Sinai Medical Center20 patientsIND approved; planenrollment Q4/2012
25Inverse 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
26Strong IP Position 28+ patents and patent applications 10 patents issued or allowed18+ patents pendingVaccine patents and applications includeMethod of use for six antigen vaccine (ICT-107)Manufacturing process for production of ICT-107Use of dendritic cells with chemotherapy for neural cancersImmunotherapy targeting IL-13Rα2Immunotherapy targeting CD133Issued patents on monoclonal antibodies cover composition of matter, therapeutic treatments and diagnostics
27Experienced Management Team John Yu, MD, Chairman & CSO, Interim CEONeurosurgeon at Cedars-Sinai, Mass General Hospital, Harvard Medical SchoolElma Hawkins, PhD, Head of Clinical DevelopmentAntigenics, Genzyme, Warner Lambert/Parke DavisJim Bender, PhD, MPH, VP of Manufacturing & Product DevelopmentIDM Pharma, Baxter HealthcareDavid Fractor, CPA, CFOHemaCare, Andwin, Deloitte & TouchePeter Ho, PhD, Director of Business DevelopmentGrey Healthcare Group, Prudential Equity Group, Allergan, D.E. ShawExperience in developing over 20 products in cell & gene therapy and vaccines
29Recent & Upcoming Milestones May 2012NYSE MKT listingJuly 2012ICT-121 INDOctober 2013SITC abstractOctober 2012SITC abstractDecember 2012ICT-121 Ph 1Q3 2013ICT-140 Ph 1/22H 2013ICT-107 Ph 2 finalJune 2013ASCO abstractNovember 2013SNO abstractQ1 2013ICT-107 Ph 2 interimICT-140 INDContinue to have discussions with potential global and regional partners.November 2012SNO abstractAugust 2012ICT-107 Ph 2 enrollment
30Strong Financial Position & Capitalization Cash (as of 9/30/2012)$10 million (A)Burn rate$3 million per quarterOutstanding debtNoneShares outstanding (as of 9/30/12)41.1 million (A)Market capitalization(as of 11/6/2012)$98 millionWarrants outstanding (as of 9/30/12)8.9 million (A)Options outstanding10.4 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.
31ImmunoCellular Therapeutics Industry-leading, next-generation, cancer immunotherapyNovember 2012