Alaunos Therapeutics, Inc. · NASDAQ:TCRT
Alaunos Therapeutics — ZIOPHARM Oncology — 30th Annual J.P. Morgan Healthcare Conference (January 2012)
26 slides from Alaunos Therapeutics's conference presentation, Jan 11, 2012
Slide 1: 30th Annual J.P. Morgan Healthcare Conference
30 th Annual J.P. Morgan Healthcare Conference Jonathan Lewis, MD, PhD Chief Executive Officer Better Cancer Medicine
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 s
Slide 3: Late-stage oncology company overview
• A late - stage oncology company with a compelling product portfolio • Ongoing pivotal Phase 3 study with another pivotal study to initiate in 2012 • A revolutionary technology for drug discovery poised to cure cancers
Slide 4: Integrated, Data Driven Oncology Portfolio
Integrated, Data Driven Oncology Portfolio Late - Stage Opportunity Palifosfamide • Phase 3 study for soft tissue sarcoma (STS) − PFS data expected 2H 2012 • Phase 3 study for small cell lung cancer (SCLC) to initiate 2H 2012 • STS/SCLC market potential of >$1 Bn in sales IL - 12 DNA • Pivotal Phase
Slide 5: Palifosfamide (Zymafos or ZIO-201)
Palifosfamide (Zymafos ® or ZIO - 201)
Slide 6: Palifosfamide: Novel DNA Cross-linker
• More effective, less toxic than in - class agents, improved QOL , ease of administration and flexible pricing • First indication in STS ; second indication in SCLC • Orphan Drug status for STS in U.S. and Europe • U.S. pharmaceutical composition patent rights extending to 2029; other pending appli
Slide 7: Soft Tissue Sarcoma
Soft Tissue Sarcoma $ in Millions • Currently over 100,000 people diagnosed with STS worldwide; estimated metastatic front - line of 23,000 people for U.S. and Europe* – Estimated 9,000 treated for front - line metastatic disease in U.S., target indication – Estimated 14,000 being treated in Europe
Slide 8: Phase 3 Structure (PICASSO 3)
Phase 3 Structure ( PICASSO 3) N : Approximately 424 patients; fully enrolled by end of 1Q12 Front - line metastatic STS Design based on successful Phase 2 study Regimen: Palifosfamide + doxorubicin vs. doxorubicin + placebo Primary Endpoint: PFS for accelerated approval, OS for full approval Powere
Slide 9: Strong Randomized Phase 2 Foundation (PICASSO)
Strong Randomized Phase 2 Foundation (PICASSO) Selected Best of ASCO 2010 Phase 2 (PICASSO) Randomized Outcome • 67 patients randomized, 66 treated, 62 eligible • Palifosfamide administered with doxorubicin compared to single - agent doxorubicin in front - and second - line unresectable or metastati
Slide 10: PFS Kaplan-Meier curve
PFS Receiving/Censored at ≤ 6 Cycles ( w/out ongoing or cross - over palifosfamide ) 0.0 0.1 0.2 0.3 0.4 0.5 0.6 0.7 0.8 0.9 1.0 1 2 3 4 5 6 MONTHS palifosfamide + doxorubicin (30 ) doxorubicin (32) p = 0.023 HR = 0.39
Slide 11: Small Cell Lung Cancer
Small Cell Lung Cancer • Approximately 15% of lung cancers • Estimated 30,000 - 35,000 U.S. annual incidence, 200,000 worldwide 1 ( C hina annual incidence alone growing to >150,000 2 ) • Platinum plus etoposide SOC front - line extensive disease (80 - 90%); only topotecan approved second - line • C
Slide 12: Phase 1b Study: Palifosfamide Plus Carboplatin/Etoposide
Phase 3 study starting 2H 2012 Data of Phase 1b Study: Palifosfamide Plus Carboplatin/Etoposide Presented at AACR NCI EORTC Response Assessment 1 Number of Patients 2 ( 22 enrolled as of 10/26/11) Cancer Types PR 5 Germ Cell Tumor, NSCLC, Ovarian, SCLC (2) SD 4 Ovarian, NSCLC, SCLC, Uterine Leiomyos
Slide 13: Compelling Efficacy: Primary Mediastinal Germ Cell Tumor
Compelling Efficacy: Primary Mediastinal Nonseminomatous Germ Cell Tumor • Normalization of tumor markers: ─ Previously treated ( including ifosfamide containing regimen – developed encephalopathy) ─ β HCG reduction: 2358 to 12 mU/Ml ─ Completed 4 cycles of therapy 0 500 1000 1500 2000 2500 β HCG mU
Slide 14: Worldwide Estimated Sales: STS & SCLC
Worldwide Estimated Sales: STS & SCLC Blended EU/US Price Per Patient SCLC Patients Treated $ 30,000 $ 40,000 $ 50,000 14,000 $ 420 $ 560 $ 700 16,000 $ 480 $ 640 $ 800 18,000 $ 540 $ 720 $ 900 Blended EU/US Price Per Patient STS Patients Treated $ 30,000 $ 40,000 $ 50,000 9,000 $ 270 $ 360 $ 450 10
Slide 15: IL-12 DNA
1L-12 DNA Semcary 16, 201
Slide 16: Exclusive Channel Partnership with Intrexon
Exclusive channel partnership (ECP) formed 1/6/11 ZIOPHARM Exclusive Channel Collaboration Pre - Discovery Lead Generation Lead Optimization Pre - clinical Studies Clinical Trials Market Pre - clinical and clinical product development and commercialization Technology development, product discovery,
Slide 17: ZIOPHARM-Intrexon Partnership: DNA Therapeutics
• Synthetic biology discovery platform for the development of complex transgenes (DNA therapeutics) • Product candidate optimization at an industry leading pace – Minimize immunogenicity/toxicity – Maximize yield/therapeutic profile • Novel in vivo control of biologic drug dosing • Control over biol
Slide 18: IL-12 DNA
• Interleukin - 12 (IL - 12) is a potent, naturally occurring anti - cancer protein central to initiation and regulation of cellular anti - cancer immune responses – Use as recombinant protein therapy limited by toxicity • Two product candidates in Phase 1 (melanoma) – 1. Autologous dendritic cells
Slide 19: ZIOPHARM Advances Lead Candidate for Pivotal Study
• DC - IL - 12 clinical POC data presented ASCO 2011 • AD - IL - 12 mid - Phase 1 • Pivotal Phase 2 study to initiate end of 2012 • Additional multi - indication Phase 2; second pivotal to follow 2013 ZIOPHARM Advances Lead Candidate for Pivotal Study Late 2012
Slide 20: ASCO 2011 First Proof of Concept in Humans
ASCO 2011 First Proof of Concept in Humans Clinical Responses No. of evaluable patients Partial response (PR) 1 Stable disease (SD) 3 Progressive disease (PD) 4 Disease control rate (CR,PR, and SD): 50% Adverse Events were mild to moderate with nausea, vomiting, anorexia, arthralgia, fever, chills b
Slide 21: Early-Stage Clinical Development
Early - Stage Clinical Development
Slide 22: Early-Stage Clinical Development
DNA therapeutics • INDs next 12 - 24 months from preclinical work in progress • Pre - clinical and discovery continuing to advance multiple antibody, immunotoxin , and protein decoy candidates • Pre - clinical programs underway for next generation RheoSwitch Therapeutic System ® and systemic deliver
Slide 23: Financial Highlights
• Primary shares outstanding: approximately 68.5MM • Cash: approximately $119MM @ 9/30/11 • Current cash resources expected to support operations into early 2013 Financial Highlights
Slide 24: 2012 Anticipated Key Clinical Milestones
2012 Anticipated Key Clinical Milestones Palifosfamide IL - 12 DNA Early Stage Development Full Enrollment 1Q 2012 STS Pivotal PFS Data 2H 2012 SCLC Adaptive Phase 3 Initiation 2H 2012 IL - 12 DNA Program Phase 1 Data 2012 Pivotal Phase 2 2H 2012 Additional multi - indication Phase 2 2H 2012 • Secon
Slide 25: Key Investment Highlights
• An intelligent portfolio of therapeutics addressing unmet medical needs in cancer – Near - term, Phase 3 study results with palifosfamide in STS • Phase 3 for SCLC in 2H 2012 • >$1 Bn total market potential – DNA therapeutics targeting established treatment pathway into pivotal study in 2012 – Sma
Slide 26: 30th Annual J.P. Morgan Healthcare Conference
30 th Annual J.P. Morgan Healthcare Conference Jonathan Lewis, MD, PhD Chief Executive Officer Better Cancer Medicine
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