Alaunos Therapeutics, Inc. · NASDAQ:TCRT
Alaunos Therapeutics — Ziopharm Oncology — Investor Presentation (April 2008)
39 slides from Alaunos Therapeutics's investor presentation, Apr 23, 2008
Slide 1: Jonathan Lewis, MD, PhD Chief Executive Officer
Jonathan Lewis, MD, PhD Chief Executive Officer
Slide 2: Forward-Looking Statements
Some of the statements made in this presentation are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of and commercialization of
Slide 3: ZIOPHARM Mission
Advance patient treatment in cancer Apply new insights from molecular and cancer biology to understand how to improve the efficacy and safety of approved and developmental cancer therapies Develop and commercialize a diverse, risk-sensitive portfolio of cancer drugs to address unmet medical needs ZI
Slide 4: 2007 Year in Review
Three product portfolio in phase I/II trials; multiple registration pathways for both niche and broad-based indications Multiple data points in all programs for phase II randomized trials – palifosfamide expected to initiate Q3 2008 Significant portfolio sales potential remains validated Intellectua
Slide 5: Leadership Team
EPIX, Sandoz (Novartis) 17 John Amedio, PhD VP, Manufacturing & Process Development EPIX, Theseus, Dupont, Genzyme, PAREXEL 22 Bob Morgan, JD VP, Regulatory Affairs & Quality Biogen (Amevive®), ImmuLogic, Point Therapeutics, BioTransplant 26 Barbara Wallner, PhD Chief Technology Officer Bayer (Nexav
Slide 6: ZIOPHARM Pipeline
INDIBULIN (301) PALIFOSFAMIDE (201) DARINAPARSIN (101) ZIOPHARM PIPELINE PRE-CLINICAL PHASE I PHASE II PHASE III Oral Single Agent Combination (erlotinib) Tarceva® Second Combination Trial Sarcoma Combination (doxorubicin) Adriamycin® Oral RCT Myeloma / Single Agent Leukemia / Lymphoma Hepatocellula
Slide 7: Product Status
Indibulin – most commercial promise (major solid tumors) Palifosfamide – most advanced (sarcoma) Darinaparsin – best “tumor response” (lymphoma) Product Status
Slide 8: Strategy analysis results in 2008 adjustments
Strategy analysis results in 2008 adjustments Palifosfamide – most advanced program (sarcoma) IV randomized phase II initiating Q3 Oral additional preclinical study Indibulin – most commercial promise (major solid tumors) Tarceva ® combination study in progress Xeloda ® combination trial in Q3 Darin
Slide 9: ZIO-301 Indibulin
ZIO-301 INDIBULIN
Slide 10: Indibulin
Novel, oral tubulin binding agent Targets cancer cell mitosis and movement Potent preclinical synergy with approved therapies (Tarceva ® , 5-FU, Taxotere ® ) Low toxicity profile Strong intellectual property Indibulin
Slide 11: Indibulin Mechanism of Action
Tubulins are involved in cell migration – “seeding” Indibulin inhibits cell migration of MO4 cells in concentration dependent manner Anti-invasive and anti-metastatic activity Indibulin is also anti-mitotic Cell Mitosis Cell Migration “Seeding” Indibulin Mechanism of Action
Slide 12: Indibulin and Tarceva
Indibulin and Tarceva ® 70.65
Slide 13: Indibulin Synergizes With 5-FU
Days post tumor implant 0 1000 2000 3000 4000 5000 6000 0 10 20 30 40 50 60 Vehicle Indibulin, 30 mg/kg 75 mg/kg 5-FU Combination Human Breast Cancer, MX-1, Xenograft Second Phase I/II Combination Trial To Follow Indibulin Synergizes With 5-FU
Slide 14: Preliminary Efficacy Seen in Phase I Studies
Prostate Rapidly rising PSA prior to starting study NSCLC Prior gem/cis CRC 2 heavily pretreated patients H & N 2 pretreated patients Ovarian Heavily pretreated patient with brain metastases Reduction in abdominal tumor size and markers Papillary thyroid Reduction in tumor size Reduction in thyroglo
Slide 15: Indibulin Phase I Preliminary PET Data
Change In Maximum SUV Of Target Lesions From Baseline* Unverified data. Cutoff date: 20 FEB 2008 Ca=carcinoma; SUV=standardized uptake value (sum) * Data represent maximum percent change in SUV of target lesions from baseline as assessed by Investigator from PET on Week 7 of treatment. Patient has g
Slide 16: Indibulin: Development Strategy
Taxanes are standard of care Lung, breast, prostate, ovarian and gastric cancer Indibulin may garner significant market share Oral dosing Lack of neurotoxicity Potential efficacy in MDR tumors Potential initial indications Lung, head & neck, breast, gastric, prostate, other Label expansion following
Slide 17: Indibulin: Clinical Development Plan
Oral Single Agent Phase I/II (erlotinib) Tarceva ® combination Second Combination Trial Potential Registration Phase Other POC Studies Indibulin: Clinical Development Plan Preclinical Phase I Phase II Phase III Q3 ‘09 Final Composite Data 1H08
Slide 18: ZIO-201 Palifosfamide
ZIO-201 PALIFOSFAMIDE
Slide 19: Palifosfamide (ZIO-201)
Stabilized active metabolite of ifosfamide (IFOS); related to cyclophosphamide - CPA Novel IV alkylating agent; oral form developed Niche and large market potential Potent preclinical synergy with Adriamycin ® Activity, convenience and safety profile over ifosfamide Patent applications filed in U.S.
Slide 20: Ifosfamide (IFOS)
IFOS approved for testicular cancer; standard of care for sarcoma; as part of ICE regimen for lymphoma In Sarcoma: 5.5 – 8.4 % front-line response rate in multicenter, randomized study (n=326)* Response does not translate into survival benefit Median PFS 2.16 – 3.0 months Ifosfamide-based therapy as
Slide 21: Palifosfamide Advantages
Palifosfamide advantages over IFOS include: Active, no pro-drug No hemorrhagic cystitis or CNS toxicity No Mesna Active in IFOS and CPA resistant xenografts IV and oral Palifosfamide Advantages
Slide 22: Palifosfamide: Phase II Sarcoma
Phase II trial includes diverse sarcoma subtypes 50 patients, fully enrolled and treatment completed Median number of prior chemotherapies was 5 Adverse events primarily mild to moderate in severity and gastrointestinal or renal related No reports of CNS or bladder toxicities and no significant bone
Slide 23: Palifosfamide: Phase II Sarcoma Trial
N Median PFS (weeks) % Progression-free at 3 months All 44 10 40.1 IFOS-naïve 11 Not reached 52.1 Over age 65 11 17 53.0 Note: PFS was calculated using time interval of first study drug dose until date of documented disease progression (clinical or radiological symptomatology or death). Patients wer
Slide 24: Retroperitoneal Sarcoma: Multiple Prior Recurrences
13.9 × 11.2 cm Baseline 12 weeks 9.0 × 7.0 cm Retroperitoneal Sarcoma: Multiple Prior Recurrences
Slide 25: Palifosfamide: Clinical Development Plan
Sarcoma Sarcoma Combination (doxorubicin) Adriamycin ® Phase I/II RCT Oral Palifosfamide : Clinical Development Plan Preclinical Phase I Phase II Phase III Final Data 2H08 Q3 IND ‘08
Slide 26: Palifosfamide: Synergistic With Adriamycin
Time since tumor implantation, days Palifosfamide : Synergistic With Adriamycin ® (MX-1 Xenograft Model)
Slide 27: Palifosfamide Registration Options
Registration strategy Data and expert opinion suggest: palifosfamide / Adriamycin ® vs. Adriamycin ® in front and second-line patients; PFS as primary endpoint Do single study for approval Window of opportunity for studies in sarcoma Palifosfamide Registration Options
Slide 28: ZIO-101 Darinaparsin
ZIO-101 DARINAPARSIN
Slide 29: Darinaparsin (ZIO-101) Organic Arsenic
Organic Arsenic: first in a new class of molecules Novel IV multifunctional agent; phase I oral accelerated Potentially safer and more active for cancer treatment than approved inorganic arsenic Early activity in NHL and liver; Active in myeloma, other heme indications Issued U.S. patents, applicati
Slide 30: Darinaparsin: Mechanism of Action
Darinaparsin: Mechanism of Action Darinaparsin Death receptor NADPH oxidase Active NADPH oxidase ROS Cytochrome C Apoptosome Caspase 9 Caspase 3 Caspase 8 Bax Bcl2 Bclxl Nucleus PARP Cell cycle arrest Apoptosis Anti-angiogenesis Mitochondrial perturbation
Slide 31: Darinaparsin: Phase I/II Trials
Hematological Cancers Results reported in hematology phase II trial In 6 of 14 leukemia patients SD Solid Tumors Phase I solid tumor results reported (37/40 patients) 2/3 renal with stable disease; 4/18 colorectal with stable disease; one pancreas, H&N, spinal tumor Multiple Myeloma Phase I myeloma
Slide 32: Darinaparsin: NH Lymphoma CR
Baseline 12 weeks Darinaparsin: NH Lymphoma CR
Slide 33: Darinaparsin: Early Activity Results
Baseline Post treatment Darinaparsin: Early Activity Results Pancreas Cancer – Liver Metastases
Slide 34: Darinaparsin: Safety Summary
Well tolerated No clinically relevant QTc prolongation, bone marrow suppression or peripheral neuropathy DLT of transient confusion, ataxia All toxicities reversible Darinaparsin: Safety Summary
Slide 35: Darinaparsin: Clinical Development Plan
Myeloma/Single Agent Oral Hepatocellular Carcinoma Additional IV phase II data and oral results form basis for future study Leukemia/Lymphoma Darinaparsin: Clinical Development Plan Preclinical Phase I Phase II Phase III Final Data 1H08 Interim Data 1H08 Preliminary Data 1H08 Final Data 1H08
Slide 36: Business Strategy: Progressive Label Expansion
Head & Neck (3 rd ) Oral Single Agent IV Breast(3 rd ) Colorectal(3 rd ) Prostate (3 rd ) NSCL(2 nd ) Ovarian (3 rd ) Oral Combination Secondary Indications Target Indications INDIBULIN Breast (3 rd ) Colorectal (3 rd ) Prostate (3 rd ) Ovarian (3 rd ) Oral Combination Lymphoma (ICE, RICE) Ovarian (
Slide 37: Upcoming Milestones
Compound Goal Target Upcoming Milestones 2H Ph II INDIBULIN (301) Initiate ph Ib/IIa combination Tarceva ® trial Q1 Initiate second ph II combination trial Q3 Final composite data from three ph I trials 1H Potential registration phase ‘09 PALIFOSFAMIDE (201) Initiate IV ph I/II combination Adriamyci
Slide 38: Financial Highlights
Cash at 12/31/07: $35.0 MM Current Cash Burn: $2.1 MM / mth Primary Shares: ~ 21 MM Cash financial guidance: Strategy review decisions impact cash burn by extending into Q3 ’09 (update of 10KSB) Financial Highlights
Slide 39: ZIOPHARM Oncology, Inc.
O~ ZIOPHARM Oncology, Inc.
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