Alaunos Therapeutics, Inc. · NASDAQ:TCRT
Alaunos Therapeutics — Ziopharm Oncology — 36th Annual J.P. Morgan Healthcare Conference (January 2018)
26 slides from Alaunos Therapeutics's conference presentation, Jan 11, 2018
Slide 1: The Next Generation of Immunotherapy Platforms
The Next Generation of Immunotherapy Platforms 36th Annual J.P. Morgan Healthcare Conference January 2018 Exhibit 99.1
Slide 2: Forward-Looking Statements
This presentation contains certain forward-looking information about ZIOPHARM Oncology, Inc. that is intended to be covered by the safe harbor for "forward-looking statements" provided by the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that
Slide 3: Two Distinct Platforms, Multiple Opportunities
Two Distinct Platforms, Multiple Opportunities The Next Generation of Immunotherapy Platforms Sleeping Beauty Implement new immune system Most clinically advanced non-viral platform Point-of-care (P-O-C): fraction of cost in a fraction of the time Scalable Control of IL-12 Weaponize existing immune
Slide 4: Two Platforms Drive Next Generation Immunotherapy
Target Indication Preclinical Phase 1 Phase 2 Pivotal Ad-RTS-hIL-12 Multicenter GBM GBM & OPDIVO® (nivolumab) GBM Stereotactic Treatment Pediatric Brain Tumor CAR CD19 2nd Gen shortened manufacture Leukemia/Lymphoma CD19 3rd Gen CAR with mbIL15 (P-O-C) Leukemia/Lymphoma CD33 AML Merck Targets Undisc
Slide 5: Controlled IL-12: A New Paradigm for Recurrent Glioblastoma
CONTROLLED IL-12 First Market: A New Paradigm for Recurrent Glioblastoma
Slide 6: Glioblastoma: Global Opportunity for Controlled IL-12
Glioblastoma: Global Opportunity for Controlled IL-12 The Next Generation of Immunotherapy Platforms ~70,000 new cases world wide each year 12,3901 new cases in the U.S. each year 14,5762 new cases in Europe each year Recurrence = 90% US – 11,1511 EU – 13,1182 Recurrent GBM Average time to death rem
Slide 7: Multicenter Phase 1 Study Evaluates Ad-RTS-hIL-12 Plus Veledimex in Recurrent Glioblastoma
Multicenter Phase 1 Study Evaluates Ad-RTS-hIL-12 Plus Veledimex in Patients with Recurrent Glioblastoma The Next Generation of Immunotherapy Platforms Ad-RTS-hIL-12 A replication-incompetent adenoviral vector administered via a single injection into the tumor and engineered to express and control h
Slide 8: Why Do Patients Benefit?
The Evidence*: MRI evidence: Tumors shrinking after IL-12 immune therapy New emerging biomarker: Ratio of killer-to-suppressor T cells correlates with survival Low-dose steroids: Improved survival likely by maintaining IL-12 immune response; 100%** survival (n=4) Biopsy data: IL-12 recruits T cells
Slide 9: Biopsies Show New Influx of Killer T cells, Upregulation of PD-1
Biopsies Show New Influx of Killer T cells (CD8+), Upregulation of PD-1 The Next Generation of Immunotherapy Platforms Before Ad-RTS-hIL-12 Day 175 after Ad-RTS-hIL-12 CD8 PD-1 3/3 Subjects IFNg: Mean ± SEM (pg/g) Serum (pg/mL) Tumor (pg/g) Baseline 0.0 ± 0.0 4.2 ± 2.6 Days 130-175 0.0 ± 0.0 226 ± 1
Slide 10: Rationale for Combination Therapy for rGBM
Rationale for Combination Therapy for rGBM Tumors remain HOT months after IL-12 therapy T-cells traffic deep into tumor Reassuring safety profile The Next Generation of Immunotherapy Platforms Ad-RTS-IL-12 + veledimex rGBM Turn cold tumors hot IL-12 rGBM
Slide 11: Phase 1 Trial of Ad-RTS-hIL-12 plus Veledimex with Nivolumab
Phase 1 Trial Initiated to Evaluate Ad-RTS-hIL-12 plus Veledimex in Combination with OPDIVO® (nivolumab) to Treat Patients with rGBM The Next Generation of Immunotherapy Platforms Primary objective: Safety and tolerability Secondary: Optimal dose Overall survival Tumor resection Ad-RTS-IL-12 (2 x 10
Slide 12: IL-12 Platform can Combine with Immune Checkpoint Inhibitors Across Oncology
IL-12 Platform can Combine with Immune Checkpoint Inhibitors Across Oncology The Next Generation of Immunotherapy Platforms PD-1 PD-L1 CTLA-4 Lag-3 | Tim-3 | Others IL-12 Turn cold tumors hot Current success of immune checkpoint inhibitors Improved success of immune checkpoint inhibitors 30% 10% Bro
Slide 13: Sleeping Beauty: A New Paradigm for Adoptive Cell Therapy
SLEEPING BEAUTY A New Paradigm for Adoptive Cell Therapy
Slide 14: Two Platforms Drive Next Generation Immunotherapy
Target Indication Preclinical Phase 1 Phase 2 Pivotal Ad-RTS-hIL-12 Multicenter GBM GBM & OPDIVO® (nivolumab) GBM Stereotactic Treatment Pediatric Brain Tumor CAR CD19 2nd Gen shortened manufacture Leukemia/Lymphoma CD19 3rd Gen CAR with mbIL15 (P-O-C) Leukemia/Lymphoma CD33 AML Merck Targets Undisc
Slide 15: Sleeping Beauty under P-O-C
Sleeping Beauty under P-O-C Very rapid manufacture Scalable process Avoids use of virus Removes need to grow T cells outside the body Reduces costs ZIOP’s Vision – Scalable, Lower Cost, High-throughput Platform The Next Generation of Immunotherapy Platforms Non-viral gene transfer 1 mbIL-15 improves
Slide 16: Eliminate the Complexity – P-O-C Trial to be Initiated 2018
Eliminate the Complexity – P-O-C Trial to be Initiated 2018 The Next Generation of Immunotherapy Platforms Apheresis (blood draw) Isolate, genetically modify T cells with electroporation Infuse CAR+ T cells for in vivo expansion Control <2 days CAR Target mbIL15 for T-cell survival benefit after inf
Slide 17: First Point-of-Care – CD19-Specific CAR+ T cells
First Point-of-Care – CD19-Specific CAR+ T cells The Next Generation of Immunotherapy Platforms Implementing P-O-C 1, 2, 3 2nd generation ongoing: CAR+ T cells 1st generation complete: CAR+ T cells Demonstrated Sleeping Beauty (SB) genetically modifies T cells safely ASH 2017: Long-term survival dat
Slide 18: CAR+ T Cells Targeting CD33 on AML
CAR+ T Cells Targeting CD33 on AML The Next Generation of Immunotherapy Platforms Follow-up visits Adult & pediatric Relapsed/Refractory AML Lymphodepletion Manufacturing T-cell infusion Trial open at MD Anderson Cancer Center to establish CD33 as a target for CAR+ T cells Technology employs lentivi
Slide 19: NCI Progressing Sleeping Beauty to Target Neoantigens in Solid Tumors with TCRs
NCI Progressing Sleeping Beauty to Target Neoantigens in Solid Tumors with TCRs The Next Generation of Immunotherapy Platforms Clinical 2018 Completed Clinical Protocol Validation Preparations underway for process validation Preclinical Data JANUARY 2017 - ZIOPHARM and Intrexon Announce CRADA with N
Slide 20: Sleeping Beauty Platform's Versatility, Multiple Targets
Sleeping Beauty Platform’s Versatility, Multiple Targets The Next Generation of Immunotherapy Platforms CD19 CD33 Merck KGaA: two targets Plus others….. Neoantigens Sleeping Beauty reprograms T cells to target the personalized neoantigens within solid tumors DNA Plasmid CARs CARs CARs DNA Plasmid TC
Slide 21: Changing the Paradigm for Manufacturing Genetically Modified T Cells
Changing the Paradigm for Manufacturing Genetically Modified T Cells The Next Generation of Immunotherapy Platforms Centralized Manufacturing Hospital-based Manufacturing Centralized production is necessary due to the use of viral-based gene transfer which is tied to the need for ex vivo numeric exp
Slide 22: Rolling out Point-of-Care at Hospitals – First Hospital Signed Up
Rolling out Point-of-Care at Hospitals – First Hospital Signed Up The Next Generation of Immunotherapy Platforms <2 days onsite Patient Hospital system holds exclusive regional license Intrexon and Ziopharm receive licensing fees, royalties First Hospital Signed Up
Slide 23: The Year Ahead
The Year Ahead Ziopharm ONCOLOGY
Slide 24: Two Platforms with Multiple Milestones in 2018
Two Platforms with Multiple Milestones in 2018 Off to a great start Combination trial with nivolumab Stereotactic adult & pediatric trials Controlled IL-12 with RTS® Sleeping Beauty The Next Generation of Immunotherapy Platforms To be initiated Pivotal, randomized control trial 2nd Gen. Phase 1 CAR+
Slide 25: Financial Update as of Sept. 30, 2017
Financial Update as of Sept. 30, 2017 Condensed Consolidated Balance Sheet Cash, cash equivalents and short term investments $84.4M At MD Anderson Cancer Center from prepayment for programs to be conducted by the Company $29.4M Third Quarter 2017 Financial Results and Updates on Recent Activities Cu
Slide 26: Ziopharm Oncology
Ziopharm ON C © IL OLN January 2018
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