Slide 1: ZIOPHARM / Intrexon Graft-Versus-Host Disease Exclusive Channel Collaboration
ZIOPHARM / Intrexon Graft-Versus-Host Disease Exclusive Channel Collaboration SEPTEMBER 28, 2015
Slide 2: Forward-looking Statements
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 sta
Slide 3: Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT)
Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) Allogeneic HSCT (or bone marrow transplantation) is used to treat various diseases including hematological malignancies, immunological deficiencies as well as non-malignant conditions.
Slide 4: Role of T cells in Recipients of Allogeneic HSCT
Role of T cells in Recipients of Allogeneic HSCT HSCT can be considered as currently the best example of T-cell therapy norDo cipientRe GVHD occurs when immune cells transplanted from a non-identical donor recognize the recipient (the host) as foreign, initiating an immune response that causes disea
Slide 5: GVHD is a multi-system disease
GVHD is a multi-system disease Acute Chronic
Slide 6: Graft-versus-Host Disease (GVHD)
Graft-versus-Host Disease (GVHD) Acute and chronic GVHD are multisystem disorders that are common complications of allogeneic hematopoietic stem-cell transplantation (HSCT) Principal risk factors for non-relapse deaths after allogeneic HSCT are older age and GVHD – J Clin Oncol. 2011 Jun 1;29(16):22
Slide 7: GvHD Market
GvHD Market Rising Number of Allogeneic HSCT Procedures in US and Europe* U.S. Europe 25000 20000 15000 10000 5000 0 2010 2011 2012 2013 There were ~23,000 allogeneic HCST procedures in US and Europe in 2013 About 35% to 50% of allogeneic HSCT recipients will develop acute GVHD From 1995 to 2007 (26
Slide 8: Overcoming Limitations; Expanding Potential Patient Population
Overcoming Limitations; Expanding Potential Patient Population Immunosuppressive agents and systemic steroids routinely used to prevent and treat GvHD have limited efficacy and toxicity, defining the need for safer, more effective therapies. The ECC intends to overcome these limitations through the
Slide 9: IL-2 – Important Immune Modulator
IL-2 – Important Immune Modulator Exclusive collaboration with Intrexon will focus on addressing the underlying pathologies of GvHD through two engineered cell platforms to control the delivery of interleukin-2 (IL-2), a cytokine that modulates the immune system. IL-2 is a pro-survival cytokine take
Slide 10: Current Human Application of Tregs and IL-2
Current Human Application of Tregs and IL-2 Clin Cancer Res. 2014 Apr 15;20(8):2215-25. Blood. 2013 Apr 11;121(15):2864-74. Best Pract Res Clin Haematol. 2011 Sep;24(3):459-66. Sci Transl Med. 2011 May 18;3(83):83ra41. Semin Immunol. 2006 Apr;18(2):78-88. Blood. 2011 Jan 20;117(3):1061-70.
Slide 11: Clinical Application of Gene Control Technologies
Clinical Application of Gene Control Technologies First approach will develop regulatory T cells (Tregs) expressing IL-2 utilizing Intrexon’s proprietary gene control technologies such as its RheoSwitch® (RTS) platform Administer Ad-RTS-hIL12 locally to achieve systemic anti-tumor effects (breast ca
Slide 12: Expression of IL-2 through ActoBiotics® microbes
Expression of IL-2 through ActoBiotics® microbes Second approach is based on genetic engineering of food-grade bacteria Lactococcus lactis - Chromosomal insertion of genes to generate biologically-contained ActoBiotics® therapeutics for in situ expression of proteins and peptides - Exclusive collabo
Slide 13: Targeted ActoBiotics® Delivery to GI Tract
Targeted ActoBiotics® Delivery to GI Tract Oral Administration, Targeted Delivery: - Coated capsules with freeze-dried ActoBiotics® therapeutics are taken by patient and released in gastrointestinal tract to secrete biotherapeutics in situ (locally) at site of disease. - Largest mass of lymphoid tis
Slide 14: Synergies with Existing Programs
Synergies with Existing Programs Virotherapy T-cell therapy Genetic modification Off-the-shelf therapy NK-cell therapy
Slide 15: INTREXON
INTREXON’ " c= ] ‘Acute GVHD Chronic GVHO oN 15
Slide 16: Conclusions
Conclusions Allogeneic HSCT is a widely accepted approach to T-cell therapy that does not rely on aprior knowing the tumor antigens ZIOPHARM is delivering biologics based on genetic modification of the immune system to treat cancer This is based on existing ECC with Intrexon ZIOPHARM will leverage i