Alaunos Therapeutics, Inc. · NASDAQ:TCRT
Alaunos Therapeutics — ZIOPHARM Oncology — Deutsche Bank 38th Annual Healthcare Conference (May 2013)
29 slides from Alaunos Therapeutics's conference presentation, May 29, 2013
Slide 1: Better Cancer Medicine
Better Cancer Medicine Deutsche Bank 38 th Annual Healthcare Conference Jonathan Lewis, MD, PhD Chief Executive Officer Exhibit 99.1 May 29, 2013
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains certain forward-looking information about ZIOPHARM Oncology 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 statement
Slide 3: DNA Therapeutics: Using the power of DNA to treat and prevent cancer
DNA Therapeutics Using the power of DNA to treat and prevent cancer May 29, 2013
Slide 4: DNA-Based Therapeutics
May 29, 2013 • Paradigm-shifting, synthetic biology technology for precise, controlled delivery of therapeutic proteins in vivo • Engineered approach to product design allows us to rapidly develop new genetically-based treatments for cancer with multiple effectors • Focused, disciplined and iterativ
Slide 5: Molecular and Cellular Oncology DNA-Coded Toolset
May 29, 2013 Cells DC T Cell B Cell Macrophage NK cell Treg Other immune cells Direct tumor lysis ADCC Complement cytotoxicity Innate immunity stimulation Adaptive immunity stimulation Immune evasion inhibition Pro-apoptosis Necrosis Anti-angiogenesis Growth inhibition Anti-tumor metabolism EMT bloc
Slide 6: A Platform System for Rapidly Developing Controllable DNA Therapies
May 29, 2013 A Platform System for Rapidly Developing Controllable DNA Therapies Inventoried DNA modules Monogenic Multigenic Viral delivery vector RheoSwitch Therapeutic System ® 3’ Target Gene (s) for Expression Intramuscular Cell Plasmid REG
Slide 7: Using Natural Cell Biology to Regulate Proteins
May 29, 2013 Using Natural Cell Biology to Regulate Proteins Target Cell Controlled Therapeutic proteins Adaptable Precision mRNA Oral ligand activates DNA, protein production begins Translation
Slide 8: Science-Driven Oncology Portfolio
Compound Pre Clinical Phase 1 Phase 2 May 29, 2013 Ad IL-12 Melanoma Breast GBM DC IL-12 Melanoma GBM Cell signal targeting Multigenic platforms Immunotherapy Programs Palifosfamide (SCLC) Science-Driven Oncology Portfolio IND
Slide 9: IL-12 Program Ad-RTS-IL-12 DC-RTS-IL-12
IL-12 Program Ad-RTS-IL-12 DC-RTS-IL-12 May 29, 2013
Slide 10: Ad-RTS-IL-12
May 29, 2013 Ad-RTS-IL-12 • Interleukin-12 (IL-12) is a potent, naturally occurring anti-cancer protein central to initiation and regulation of cellular anti-cancer immune responses • Regulated intratumoral expression of IL-12 promotes activation of TIL’s to drive a cytotoxic immune response against
Slide 11: Increases CD8+ TILs in the 4T1 Syngeneic Mouse Model
May 29, 2013 Increases CD8 + TILs in the 4T1 Syngeneic Mouse Model Vehicle INXN-1001 mg/m + Ad-RTS-mIL-12 1x10 vp
Slide 12: Systemic Tumor Response in B16 Melanoma Model
May 29, 2013 Systemic Tumor Response in B16 Melanoma Model Group untreated; Group AL in food ~675 mg/m /day; Group Ad-RTS-mIL-12 1x10 vp Days 12 & 19; Group 4 AL + Ad-RTS-mIL-12. Arrows = administration of Ad-RTS-mIL-12 Treated Tumor on the Right Flank Untreated Tumor on the Left Flank
Slide 13: Phase I/II Open Label Study of Ad-RTS-hIL-12 + AL in Melanoma
May 29, 2013 A Phase I/II, Open Label Study of Ad-RTS-hIL-12 + AL in Subjects with Unresectable Stage III or IV Melanoma • 3 + 3, single-arm design • Primary endpoint – Safety and tolerability of intratumoral injections of 1012 vp Ad-RTS-hIL-12 in combination with escalating doses of INXN-1001 • Sec
Slide 14: Prominent Inflammatory Response Correlates with High Levels of IFN-gamma
Prominent Inflammatory Response Correlates with High levels of IFN- • Initial increase in lesion size due to inflammatory response seen at Cycle 1 Day 16 • Lesion was undetectable at Cycle 2 Day 1 • Subject ultimately progressed and was taken off study C1D1 C1D1 C1D16 C1D16 C2D1 C2D1 May 29, 2013
Slide 15: Summary of Immunological and Clinical Activity
May 29, 2013 • Clinical activity at 100 mg and 160 mg doses coincided with the highest serum levels of IL-12 and IFN- Summary of Immunological and Clinical Activity – Prominent inflammatory responses in injected and non-injected lesions – Decrease in size of injected and non-injected lesions • Clini
Slide 16: Summary of Safety
May 29, 2013 Summary of Safety • Controlled expression of IL-12 limits systemic toxicity while inducing biological and clinical activity in a dose-dependent fashion – Toxicity reversible on stopping oral activator ligand • The most common related TEAEs ( 20% of subjects): • Chills and Pyrexia (73% e
Slide 17: Advanced Breast Cancer
May 29, 2013 • Preclinical – Intratumoral administration of Ad-RTS-mIL-12 (Ad) in 4T1 BALB/c mouse breast carcinoma model: dose-related decrease in tumor growth rate. (AACR 2013) – Therapeutic strategy appears to be well tolerated • Phase 2 – Multi-center, randomized, open-label – Ad-RTS IL-12 in co
Slide 18: Dose-dependent Anti-Tumor Activity in Murine 4T1 (Breast Cancer) Model
May 29, 2013 Dose-dependent Anti-Tumor Activity of in Murine 4T1 (Breast Cancer) Model Time (Days) Vehicle Control Ad-RTS-mIL12 (AD) INXN-1001 (AL) 15 mg/m2 AD + AL 15 mg/m2 AD + AL 30 mg/m2 AD + AL 75 mg/m2 AD + AL 150 mg/m2 IPM 40 mg/m2 IPM 80 mg/m2 IPM 120 mg/m2 AD + AL 30 mg/m2 + IPM 40mg/m2 AD
Slide 19: Glioblastoma Multiforme: Promising Preclinical Activity
May 29, 2013 Glioblastoma Multiforme: Promising Preclinical Activity 100 % survival observed with Ad-RTS-IL-12 + AL or DC-RTS-IL-12 + AL INXN-1001 dosing Day 4 to EOS at ~ 675 mg/m ² /day in chow; DC –RTS-IL12 or Ad-RTS-IL12 on Day 5 Kaplan Meier Survival in GL261 Orthotopic Syngeneic Mouse Glioma M
Slide 20: Preclinical and Discovery Programs
May 29, 2013 Preclinical and Discovery Programs
Slide 21: Platform System for Rapidly Developing Controllable DNA Therapies
May 29, 2013 A Platform System for Rapidly Developing Controllable DNA Therapies Inventoried DNA modules Monogenic Multigenic Viral delivery vector RheoSwitch Therapeutic System® 3’ REG Target Gene (s) for Expression Intramuscular Cell Plasmid
Slide 22: Using Natural Cell Biology to Regulate Proteins
May 29, 2013 Using Natural Cell Biology to Regulate Proteins Precision Oral ligand activates DNA, protein production begins Controlled Therapeutic proteins Adaptable Translation mRNA Target Cell
Slide 23: Next Generation: Multigenic Approach
IP1: LacZ rLUC Stuffer Stuffer Stuffer SP-RTS IP2: fLuc rLUC Stuffer Stuffer Stuffer SP-RTS IP3: SEAP rLUC Stuffer Stuffer Stuffer SP-RTS IP4: GUS rLUC Stuffer Stuffer Stuffer SP-RTS IP4: GUS rLUC SP-RTS IP1: LacZ IP2: fLuc IP3: SEAP May 29, 2013 IP1: LacZ rLUC Stuffer Stuffer Stuffer SP-RTS IP2: fL
Slide 24: Small Molecule Programs
Small Molecule Programs May 29, 2013
Slide 25: Small Molecule Programs
May 29, 2013 Small Molecule Programs Palifosfamide Bi-functional DNA alkylating agent that has activity in multiple tumors by evading typical resistance pathways, less toxic and ease of administration Phase soft tissue sarcoma program terminated, ongoing adaptive Phase in small cell lung Indibulin N
Slide 26: Program Milestone Timing 2013 2014
Program Milestone Timing 2013 2014 May 29, 2013 Expected Milestones IL-12 Melanoma preliminary Phase 2 data 4Q Breast cancer preliminary Phase 2 data 4Q GBM preclinical proof of concept 4Q Multigenic platforms Preclinical data 4Q Immunotherapy Programs Preclinical data 4Q Palifosfamide Interim SCLC
Slide 27: Financial Highlights
May 29, 2013 • Primary shares outstanding: approximately 82.9M • Cash: approximately $55.7M @ 3/31/13 • Current cash resources expected to support operations into 1Q 2014 Financial Highlights
Slide 28: Conclusion
May 29, 2013 • Paradigm-shifting, synthetic biology technology for precise, controlled delivery of therapeutic proteins in vivo • Engineered approach to product design allows us to rapidly develop new genetically-based treatments for cancer with multiple effectors • Focused, disciplined and iterativ
Slide 29: Better Cancer Medicine
Better Cancer Medicine Deutsche Bank 38 Annual Healthcare Conference Jonathan Lewis, MD, PhD Chief Executive Officer th May 29, 2013
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