Alaunos Therapeutics, Inc. · NASDAQ:TCRT
Alaunos Therapeutics — ZIOPHARM Oncology — Jefferies 2016 Healthcare Conference
36 slides from Alaunos Therapeutics's conference presentation, Jun 9, 2016
Slide 1: ZIOPHARM Oncology Jefferies 2016 Healthcare Conference
ZIOPHARM Oncology Jefferies 2016 Healthcare Conference June 2016 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: Leadership and financial position
Laurence Cooper, MD, PhD Named CEO in May 2015 Developed Sleeping Beauty technology in-licensed by ZIOP/XON in Jan 2015 Previously, Professor of Pediatric Oncology at MD Anderson Cancer Center (MDACC) where he led the bone marrow transplant program and developed significant expertise in immunotherap
Slide 4: Technologies, combinations and manufacturing
Technologies CARs, TCRs, Cytokines, RTS, non-viral gene transfer, T cells, NK cells Combinations Cytokine & CAR, Cytokine & Checkpoint Inhibitor Implementing manufacturing processes for both autologous and allogeneic settings Targeting hematologic malignancies and solid tumors Continued optimization
Slide 5: Addressing unmet medical needs: pipeline
Ad-RTS-IL-12 NK Cells Primary NK cells Combination with Ad-RTS-IL-12 Genetically-engineered TCR Sleeping Beauty TCR Sleeping Beauty TCR and cytokine Other Regulatory T Cells Modified Bacteria (microbiome) GBM Breast Cancer Pediatric GBM + Checkpoint Leukemia/Lymphoma Undisclosed Undisclosed Undisclo
Slide 6: Controlled intra-tumor delivery of IL-12
Controlled intra-tumor delivery of IL-12 Ad-RTS-IL-12 + veledimex IL-12 Pro-inflammatory cytokine can reverse immune escape mechanisms and improve the function of tumor-fighting T cells Ad-RTS-IL-12 + veledimex (V, oral ligand) explores local treatment strategy under the control of the RheoSwitch Th
Slide 7: Ad-RTS-hIL-12 with veledimex in glioblastoma study
A study of Ad-RTS-hIL-12 with veledimex in subjects with glioblastoma or malignant glioma GBM affects approximately 74,000 people worldwide each year Recurrent GBM has one of the lowest 3-year survival rates: 3%, among all cancers. For multiple recurrence, median overall survival (OS) is of 6 to 7 m
Slide 8: RTS switch responds to dose of veledimex in recurrent GBM
RTS® switch responds to the dose of veledimex in patients with recurrent GBM
Slide 9: Early data suggests benefit with favorable survival trend
Early data suggests benefit with a favorable trend in overall survival Too early to determine pseudoprogression versus progression All pseudoprogression / progression are assumed to trigger PD for PFS analysis Clinical benefit including long term survival and tumor regression, can occur after initia
Slide 10: Safety summary from GBM study
Safety summary from GBM study to date (N = 11) Overall Ad-RTS-hIL12 + veledimex was well tolerated Neurotoxicities were manageable and reversible All serious adverse events (SAEs) and Grade 3 related toxicities were found to be rapidly reversible upon discontinuation of veledimex Common related adve
Slide 11: Ad-RTS-hIL-12 with veledimex in breast cancer
A study of Ad-RTS-hIL-12 with veledimex in subjects with breast cancer Phase 1b/2 study to examine the safety, tolerability and preliminary efficacy of one cycle of Ad-RTS-hIL-12 with veledimex following achievement of stable disease (SD) or partial response (PR) on standard first or second-line che
Slide 12: Addressing unmet medical needs: pipeline
Ad-RTS-IL-12 NK Cells Primary NK cells Combination with Ad-RTS-IL-12 Genetically-engineered TCR Sleeping Beauty TCR Sleeping Beauty TCR and cytokine Other Regulatory T Cells Modified Bacteria (microbiome) GBM Breast Cancer Pediatric GBM + Checkpoint Leukemia/Lymphoma Undisclosed Undisclosed Undisclo
Slide 13: Next steps for Ad-RTS-IL-12
Pediatric program Target refractory brain tumors in children Pre-clinical data at Society for Neuro Oncology Annual Meeting (November 2016) Combination program Combining Ad-RTS-hIL-12 with immune checkpoint inhibitors American Society of Cell and Gene Therapy oral presentation May 2016 Survival of m
Slide 14: Addressing unmet medical needs: pipeline
Ad-RTS-IL-12 NK Cells Primary NK cells Combination with Ad-RTS-IL-12 Genetically-engineered TCR Sleeping Beauty TCR Sleeping Beauty TCR and cytokine Other Regulatory T Cells Modified Bacteria (microbiome) GBM Breast Cancer Pediatric GBM + Checkpoint Leukemia/Lymphoma Undisclosed Undisclosed Undisclo
Slide 15: Targeting tumor antigens with CARs and TCRs
Targeting tumor antigens with CARs and TCRs
Slide 16: Sleeping Beauty: first-in-human non-viral gene transfer
Sleeping Beauty: First-in-human study using non-viral gene transfer Long-term follow-up data from 1st generation Sleeping Beauty platform in two trials infusing CAR+ T cells after hematopoietic stem-cell transplantation (HSCT) Showed favorable PFS and OS trends in both autologous and allogeneic coho
Slide 17: Improving CAR+ T cells by co-signaling through IL-15 receptor
Improving CAR+ T cells by co-signaling through IL-15 receptor
Slide 18: Improving CAR+ T cells by co-signaling through IL-15 receptor
Improving CAR+ T cells by co-signaling through IL-15 receptor T-cell survival Tumor only CAR CAR & mIL15
Slide 19: Reducing manufacturing time for CAR+ T cells with Sleeping Beauty
American Society of Cell and Gene Therapy oral presentation (May 2016) Fundamental to advancing SB platform, and any modified cell-based therapy, into a broadly deployed treatment option is a streamlined, simplified, and shortened manufacturing process, with a reduction in the associated cost Decrea
Slide 20: Addressing unmet medical needs: pipeline
Ad-RTS-IL-12 NK Cells Primary NK cells Combination with Ad-RTS-IL-12 Genetically-engineered TCR Sleeping Beauty TCR Sleeping Beauty TCR and cytokine Other Regulatory T Cells Modified Bacteria (microbiome) GBM Breast Cancer Pediatric GBM + Checkpoint Leukemia/Lymphoma Undisclosed Undisclosed Undisclo
Slide 21: CAR+ T-cell platform to target myeloid malignancies
CAR+ T-cell platform to target myeloid malignancies Immunotherapy of tumors with unmet needs outside of the crowded viral CAR T treatment landscape for CD19+ malignancies Rapidly advancing a CAR T target for myeloid malignancies: Encouraging pre-clinical data including CAR expression, cytotoxicity,
Slide 22: Addressing unmet medical needs: pipeline
Ad-RTS-IL-12 NK Cells Primary NK cells Combination with Ad-RTS-IL-12 Genetically-engineered TCR Sleeping Beauty TCR Sleeping Beauty TCR and cytokine Other Regulatory T Cells Modified Bacteria (microbiome) GBM Breast Cancer Pediatric GBM + Checkpoint Leukemia/Lymphoma Undisclosed Undisclosed Undisclo
Slide 23: Intrexon/Merck KGaA agreement in CAR-T
Exclusive agreement to develop and commercialize CAR-T cancer therapies 2 novel CAR T targets nominated Merck KGaA, Darmstadt, Germany to lead IND filing and pre-IND interactions, clinical development and commercialization Intrexon and ZIOPHARM retain ability to explore targets independently, granti
Slide 24: Addressing unmet medical needs: pipeline
Ad-RTS-IL-12 NK Cells Primary NK cells Combination with Ad-RTS-IL-12 Genetically-engineered TCR Sleeping Beauty TCR Sleeping Beauty TCR and cytokine Other Regulatory T Cells Modified Bacteria (microbiome) CAR CD19 1st Generation CD19 2nd Generation CD19 3rd Generation with cytokine Myeloid malignanc
Slide 25: Sleeping Beauty non-viral approach for targeting intracellular antigens by TCRs
Non-viral Sleeping Beauty Viral delivery Target tumor antigens via multiple TCRs Limited appeal for targeting multiple intracellular antigens via TCRs Cost-effective approach High cost approach Rapid manufacture Labor intensive, slow manufacture Express multiple genes by combining DNA plasmids Packa
Slide 26: Targeting intracellular antigens: key to T-cell therapy for solid tumors
Targeting intracellular antigens: The key to implementing T-cell therapy for solid tumors
Slide 27: Targeting shared intracellular tumor antigens using a library of TCRs
Targeting “shared” intracellular tumor antigens using a library of TCRs
Slide 28: Targeting private intracellular tumor antigens (neoantigens)
“Clinical evidence supports the hypothesis that immunogenic products of somatic mutations unique to each patient’s cancer—so-called neoantigens—are the relevant targets for successful immunotherapies” “Success for cell-based immunotherapies may come from the arduous task of targeting the unique set
Slide 29: Targeting neoantigens with personalized TCR-modified T cells
Tumor antigen is not known before the patient arrives Tumor and normal cells are interrogated to determine the neoantigen TCRs against known tumor antigen are prepared in real time TCR expressed in autologous T cells using SB platform Targeting neoantigens deploying personalized TCR-modified T cells
Slide 30: Neoantigen-specific TCRs expressed using SB system
Neoantigen-specific TCRs expressed using SB system to target solid tumors Molecular Therapy 05 March 2016.
Slide 31: Addressing unmet medical needs: pipeline
Ad-RTS-IL-12 NK Cells Primary NK cells Combination with Ad-RTS-IL-12 Genetically-engineered TCR Sleeping Beauty TCR Sleeping Beauty TCR and cytokine Other Regulatory T Cells Modified Bacteria (microbiome) GBM Breast Cancer Pediatric GBM + Checkpoint Leukemia/Lymphoma Undisclosed Undisclosed Undisclo
Slide 32: Natural Killer Cells: Beyond CAR+ T cells
Natural killer (NK) cells Target tumors such as AML so do not require CAR Killing is independent of a specific (known) target antigen Do not have T-cell receptor (TCR), so do not require genetic editing to eliminate TCR May be used as an off-the-shelf therapeutic Cytokines, e.g., IL-12 are “fuel” fo
Slide 33: Addressing unmet medical needs: pipeline
Ad-RTS-IL-12 NK Cells Primary NK cells Combination with Ad-RTS-IL-12 Genetically-engineered TCR Sleeping Beauty TCR Sleeping Beauty TCR and cytokine Other Regulatory T Cells Modified Bacteria (microbiome) GBM Breast Cancer Pediatric GBM + Checkpoint Leukemia/Lymphoma Undisclosed Undisclosed Undisclo
Slide 34: Regulatory T cells and microbiome for GvHD
Regulatory T cells and manipulating the microbiome for graft-versus-host-disease (GvHD) Regulatory T cells Genetic modification L. lactis IL - 2 Genetic modification Donor Anti - inflammatory Gut health Sensors Regulatory T cells Prevention and/or treatment Recipient Acute GVHD Chronic GVHD
Slide 35: Multiple immunotherapies and combinations are needed
Multiple immunotherapies and combination immunotherapies are needed and being administered We stand alone in our ability to control the delivery of IL-12 We stand alone in being able to harness non-viral DNA as a method to genetically control T cells We are launching multiple immunotherapy trials Tr
Slide 36: ZIOPHARM Oncology Jefferies 2016 Healthcare Conference
ZIOPHARM Oncology Jefferies 2016 Healthcare Conference June 2016
ZIOPHARM Oncology — Jefferies 2016 Healthcare Conference on 6ix