Alaunos Therapeutics, Inc. · NASDAQ:TCRT
Alaunos Therapeutics — Ziopharm Oncology — Investor Presentation (June 2009)
35 slides from Alaunos Therapeutics's investor presentation, Jun 4, 2009
Slide 1: ZIOPHARM Oncology, Inc.
O~ ZIOPHARM Oncology, Inc.
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 and Strategy
Better cancer medicine. Low cost small molecules. Oral and global. Improved quality of life. ZIOPHARM Mission and Strategy
Slide 4: Portfolio of Mid-Stage Development Candidates
Focus of current resources: Zymafos TM (palifosfamide), novel DNA-alkylating molecule in randomized phase II expected to define registration trial 1H 2010; oral form at IND stage To follow: Zybulin TM (indibulin), novel oral tubulin-binding molecule in phase I trials expected to enter phase I/II bre
Slide 5: Zymafos (palifosfamide)
ZYMAFOS TM (palifosfamide)
Slide 6: Palifosfamide
Novel alkylating molecule; patent applications U.S. and internationally Anticipated low cost to manufacture IV form in randomized phase II and oral developed for IND Target indication of soft-tissue sarcoma (front/second-line setting) Palifosfamide Orphan Drug Designation (soft-tissue sarcoma) in U.
Slide 7: Palifosfamide Opportunity
Palifosfamide, novel DNA alkylating agent , related to ifosfamide and cyclophosphamide family Active preclinically in diverse cancers including ifosfamide and cyclophosphamide resistant tumors Development premise : less toxic, more efficacious, enhanced quality of life, easier to administer than rel
Slide 8: Palifosfamide Development Leading to ASCO
Active in Phase I with anticipated toxicity profile Active in Phase II advanced sarcoma Synergistic with doxorubicin in preclinical study US and EU experts recommend randomized phase II (front-/second- line setting, palifosfamide +/- doxorubicin) in soft-tissue sarcoma (PICASSO); trial actively enro
Slide 9: A Phase I Study of Palifosfamide in Combination with Doxorubicin
A Phase I Study of Palifosfamide in Combination with Doxorubicin: Safety and Preliminary Efficacy Luis H. Camacho 1 , Sant Chawla 2 , Victoria Chua 2 , Giovanni Abbadessa 3 , Philip Komarnitsky 3 , Barbara Wallner 3 , Jan Stevens 3 , Jonathan Lewis 3 1Oncology Consultants; 2Sarcoma Oncology Center,
Slide 10: Preliminary Exposure and Efficacy
Preliminary Exposure and Efficacy Exposure 13 treated; 0 ongoing Best Response SD or better in 42% of 12 evaluated patients 3 PR: STS (2) and SCLC (1) SD or better in 75% of STS patients Patients age = 65 (n=4): 2 SD (50%), 1 PR (25%)
Slide 11: Soft-Tissue Sarcomas: Best Response
75 % SD or better Soft-Tissue Sarcomas: Best Response (N=8) LMS (2) MPNS Tumor PD Angiosarcoma Rhabdomyosarcoma (2) % of STS patients: Pleomorphic Sarcoma Endometrial Stromal Cell Sarcoma SD PR
Slide 12: Adverse Events
Adverse events primarily mild to moderate in severity No encephalopathy, no hemorrhagic cystitis, no renal toxicity Most common adverse events include: Neutropenia 6 (46%) Thrombocytopenia 6 (46%) Micro Hematuria 5 (38%) Anemia 5 (38%) Nausea 4 (31%) Vomiting 4 (31%) Adverse Events
Slide 13: Conclusions
Conclusions Palifosfamide 150 mg/m 2 , 3 times per week combined with doxorubicin 75 mg/m 2 once every 3 weeks is a very well tolerated outpatient regimen. There has been no encephalopathy, no hemorrhagic cystitis, no renal toxicity . Adverse events are primarily hematologic and easy to manage. Prel
Slide 14: Palifosfamide Registration Strategy
Palifosfamide / doxorubicin vs. doxorubicin in front- and second-line patients with unresectable or metastatic soft- tissue sarcoma: Phase II randomized trial helps shape registration trial Single registration trial powered for PFS and survival Palifosfamide Registration Strategy
Slide 15: Zybulin (indibulin)
(indibulin) Zybulin TM
Slide 16: Indibulin
Novel oral tubulin binding agent; issued patents and applications Anticipated low cost to manufacture Targets cell mitosis and movement Expected low toxicity (no neurotoxicity) Target indication of subset of breast cancer Indibulin
Slide 17: Indibulin Opportunity
Taxanes widely used and a more efficacious/less toxic oral treatment expected to have billion dollar sales potential Distinct mechanism and Oral dosing Lack of neurotoxicity Potential efficacy in tumors with MDR Treatment approaches, initially in breast cancer : Norton dose density Combination thera
Slide 18: Indibulin Development Leading to ASCO
Single agent activity in phase I in multiple tumor types; confirmatory activity in phase I PET data ; DLT not reached, minimal toxicity and no neurotoxicity Highly synergistic in preclinical study Phase I oral capecitabine with oral indibulin presented at ASCO 2009 Preclinical evaluation (Dr. Norton
Slide 19: Indibulin in Combination with Capecitabine, Suitable for Mathematically-Optimized Dose-Scheduling
Jonathan J. Lewis 1 , Matthew D. Galsky 2,6 , Luis H. Camacho 3 , David M. Loesch 4,6 , Philip B. Komarnitsky 1 , Barbara Wallner 1 , Jan Stevens Larry Norton 5 1 ZIOPHARM Oncology, New York, NY; 2 Comprehensive Cancer Centers of Nevada, Las Vegas, NV; 3 Oncology Consultants P.A., Houston, TX; 4 Cen
Slide 20: Preliminary Clinical Activity
Preliminary Clinical Activity Median SD 6 Cycles Breast and colon cancer SD for 6 Cycles Bladder cancer SD for 9 Cycles Prostate cancer SD for 9 Cycles Breast and Colon Cancer 6 Cycles Bladder and Prostate Cancer 9 Cycles
Slide 21: Preliminary Safety
AEs that are Related and occurred in 2 or more pts ( = 29 %) Frequency, % Grade 1/2 AEs that were related • Fatigue 4 (57) • Anorexia 2 (29) • Dyspnea 2 (29) • Hand Foot Syndrome 2 (29) • Mucositis 2 (29) • Vomiting 2 (29) Grade 3/4 AEs that were related • Hypophosphatemia 1 (14) Preliminary Safety
Slide 22: Indibulin: Optimization of Dosing Schedules
Indibulin: Optimization of Dosing Schedules
Slide 23: Indibulin: Optimization of Dosing Schedules
Days of Growth MX-1 Xenograft 0 500 1000 1500 2000 2500 0 5 10 15 20 25 30 35 Vehicle Control daily Indibulin 22 mg/kg daily Indibulin 29 mg/kg 5 days on, 5 days off Therapy Starts (Day 8) Indibulin: Optimization of Dosing Schedules
Slide 24: Conclusions
Oral indibulin in combination with capecitabine is very well tolerated with no neurotoxicity. Early activity in breast, colon, bladder, and prostate cancers. Formal analyses of preclinical data utilizing Norton- Simon Modeling reveals that the major effect of therapy occurs in five days of exposure
Slide 25: Zinapar (darinaparsin)
(darinaparsin) Zinapar TM
Slide 26: Darinaparsin
Novel mitochondrial-targeted agent (organic arsenic); first in a new class of molecules IV formulation in multiple phase I/II studies with confirmed activity in lymphoma Oral form ongoing in Phase I Families of compounds covered by issued patents with further applications pending in U.S. and interna
Slide 27: Darinaparsin Opportunity
Inorganic arsenic use limited by cardiotoxcity (“Black Box” warning) IV darinaparsin (organic arsenic) in several phase I/II studies with no cardiotoxicity , well tolerated; MTD not yet reached with oral form IV phase II data in hematological malignancies presented at ASCO 2009 -- advisors believe d
Slide 28: Darinaparsin: Development of IV and Oral Forms
Novel Organic Arsenic Molecule Darinaparsin: Development of IV and Oral Forms I. S. Lossos 1 , M. D. Craig 2 , M. S. Tallman 3 , R. V. Boccia 4 , P. R. Conkling 5 , C. Becerra 6 , P. B. Komarnitsky 7 , B. Wallner 7 , J.J.Lewis 7 , W. H. Miller 8 1 Miller School Of Medicine, University of Miami, Miam
Slide 29: IV Darinaparsin Efficacy
IV Darinaparsin Efficacy Complete Responses: 3 66 year old female, PTCL (3 + months*) 3 prior treatment regimens: CHOP x 6, ICE and EPOCH x 2 Patient taken off study for autologous BMT 73 year old female, PTCL + senile EBV-associated, B-cell lymphoproliferative disorder (5 + months) 6 prior regimens
Slide 30: IV Darinaparsin Efficacy
IV Darinaparsin Efficacy 1 Marginal Zone Non-Hodgkin Lymphoma transformed to DLBCL (14 + months) * 5 prior treatment regimens: chlorambucil, RCHOPx5, RICEx3, RT and BMT 1 Marginal Zone Non-Hodgkin Lymphoma (3 months) Rituximab x 8, RCVP x 1, and gemcitabine x 1 1 PTCL (4 months) EPOCH x 2, dox, cyto
Slide 31: IV Darinaparsin Related Adverse Events
IV Darinaparsin Related Adverse Events Events that were grade 3 N % Fatigue Alk. Phos Increased Events that were considered SAEs N % Fall Neutropenic Fever No QT prolongation
Slide 32: Oral Efficacy
Oral Efficacy MTD not reached 1 PR (H&N) duration 5+ months 15 prolonged SD (H&N, lymphoma, colon, pancreas) Duration 3 – 6+ months
Slide 33: Summary
Summary IV Lymphoma 7 / 19 objective responses (3 CRs, 4 PRs) 3 / 5 objective responses PTCL (2 CRs, 1 PR) 4 prolonged SD PO All comers not yet at MTD 1 PR, 15 prolonged SD
Slide 34: Portfolio Highlights
Palifosfamide pivotal trial design expected from IV phase II randomized study to initiate as early as 1H 2010 and focus of current resources Indibulin phase I and dose scheduling oral data highlight potential benefit over widely used tubulin targeted agents with phase I/II novel dose schedule in bre
Slide 35: ZIOPHARM Oncology, Inc.
O~ ZIOPHARM Oncology, Inc.
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