Alaunos Therapeutics, Inc. · NASDAQ:TCRT
Alaunos Therapeutics — ZIOPHARM Oncology — Barclays Global Healthcare Conference (March 2013)
28 slides from Alaunos Therapeutics's conference presentation, Mar 13, 2013
Slide 1: Better Cancer Medicine
Better Cancer Medicine Barclays Global Healthcare Conference Jonathan Lewis, MD, PhD Chief Executive Officer Exhibit 99.1 1 March 13, 2013
Slide 2: Forward-Looking Statements
2 March 13, 2013 Forward-Looking Statements This presentation contains certain forward-looking statements about ZIOPHARM Oncology that are 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-
Slide 3: Diverse Portfolio of Therapeutics Addressing Unmet Medical Needs in Cancer
Better Cancer Medicine Diverse Portfolio of Therapeutics Addressing Unmet Medical Needs in Cancer Paradigm Shifting Approaches to Impact and Cure Cancer 3 March 13, 2013 Small Molecules Synthetic Biology
Slide 4: Data-Driven Oncology Portfolio
4 March 13, 2013 Compound Preclinical Phase 1 Phase 2 Phase 3 Data-Driven Oncology Portfolio Palifosfamide Soft Tissue Sarcoma Small Cell Lung Cancer Oral Formulation Ad-RTS-IL-12 Melanoma Breast (+palifosfamide) Other Candidates Indibulin Breast Cancer Darinaparsin PTCL
Slide 5: Near-Term Value Drivers
5 March 13, 2013 • Palifosfamide – Two Phase 3 programs • Metastatic soft tissue sarcoma • Small cell lung cancer – Potential in multiple tumor types – Exceptional intellectual property profile (t0 2029) • Synthetic Biology – Two Phase 2 programs • Melanoma • Breast cancer – Exploring multiple oppor
Slide 6: Major Clinical Milestones Over Next 18 Months
6 March 13, 2013 MATISSE PHASE 2 MELANOMA PHASE 2 BREAST CANCER (COMBO) PICASSO 3 Major Clinical Milestones Over Next 18 Months Q1-13 Q2-13 Q3-13 Q4-13 Q1-14 Q2-14 Possible NDA Submission TOP LINE DATA Possible ASCO Late-breaking Interim Analysis Palifosfamide IL-12 DNA Possible Phase 1 Melanoma ASC
Slide 7: Palifosfamide
Palifosfamide 7 March 13, 2013
Slide 8: A Potent Bi-Functional DNA Alkylating Agent
8 March 13, 2013 Broad Application Effect in solid tumors and hematological malignancies Safety, Efficacy and Accessibility Potent bi-functional DNA alkylating agent that has activity in multiple tumors by evading typical resistance pathways (in class with bendamustine, cyclophosphamide, and ifosfam
Slide 9: Soft Tissue Sarcoma
9 March 13, 2013 Soft Tissue Sarcoma • High unmet medical need; no first-line drug approved in ~30 years • First-line metastatic, non-GIST STS: – Approximately 9,000 in U.S. – Approximately 14,000 in Europe • Market in transformation – April 2012 FDA full approval of Votrient ® for second-line thera
Slide 10: Votrient ODAC March 20, 2012
Votrient ® ODAC March 20, 2012
Slide 11: Phase 3 Design (PICASSO 3)
11 March 13, 2013 Phase 3 Design (PICASSO 3) N: 447 subjects; first-line metastatic STS Regimen: Palifosfamide + doxorubicin vs. placebo + doxorubicin Primary Endpoint: PFS for full approval Powering: Powered for both PFS and OS PFS Analysis: Independent blinded radiologic review; Efficacy analysis
Slide 12: Regulatory Strategy: Evolving Landscape of PFS as Primary Endpoint
12 March 13, 2013 • PFS primary endpoint in Phase 3 registration trials and now supportive for certain indications: – In 2011, 4 oncology products approved based on PFS as primary trial endpoint – In 2012, 7 oncology products approved based on PFS as primary trial endpoint, including pazopanib (Votr
Slide 13: Phase 3: Correctly Modeled and Powered for PFS & OS
13 March 13, 2013 Phase 3: Correctly Modeled and Powered for PFS & OS Multicenter, Randomized, Stratified, Balanced Phase 2 Data (PICASSO) Source: Best of ASCO 2010 (PFS Data), internal analysis (OS Data) * Expected 2-yr survival is 25% based on evaluation of randomized data ** Control arm confirmed
Slide 14: Appropriate Risk/Benefit Ratio Is Critical
Appropriate Risk/Benefit Ratio Is Critical 14 March 13, 2013 EORTC 62012 Randomized Phase 3 Trial (ESMO) PICASSO EORTC Pali+Dox Dox Ifos+Dox Dox PFS: median 7.8 mos. 4.4 mos. 7.4 mos. 4.6 mos. PFS: hazard ratio 0.43 0.74 OS: hazard ratio ( with crossover ) 0.78 2yr. N/A (mOS: 0.83) 2-yr survival 40%
Slide 15: Small Cell Lung Cancer
15 March 13, 2013 Small Cell Lung Cancer • High unmet medical need; no innovation in decades • Worldwide market opportunity: 250,000+patients – 30,000-35,000 patients in the U.S. • Rationale: ifosfamide only first-line therapy to show benefit added to SOC (platinum plus etoposide) - excessive toxici
Slide 16: MATISSE Adaptive Trial Design
16 March 13, 2013 MATISSE Adaptive Trial Design Single pre-planned evaluation by the IDMC • • • Events Continue trial with decreased sample size Complete trial as planned Continue trial with increased event size Optimize chances of success Stop trial for efficacy/futility Complete trial as planned o
Slide 17: MATISSE Trial Progress
17 March 13, 2013 • MATISSE trial enrollment ahead of projection • Successful IDMC safety review of initial 20 patients • Interim analysis of 125 events expected end of 2013 MATISSE Trial Progress
Slide 18: Market Potential
Palifosfamide Price Per Patient Per Year Patients Treated $ 54,000 $ 72,000 $ 90,000 18 March 13, 2013 • Recent, U.S. orphan drug launches in oncology priced at $9K - $23K per cycle range – Examples of products: Adcetris ™ , Caprelsa ® , Zelboraf ® Market Potential Scenarios of U.S. Only Peak Gross
Slide 19: Synthetic Biology
Synthetic Biology 19 March 13, 2013
Slide 20: Synthetic Biology
20 March 13, 2013 • Revolutionary technology for precise, controlled delivery of DNA expressing therapeutic proteins in vivo • Lead Ad-RTS-IL-12 candidate in melanoma, breast cancer • Next generation of therapeutic approaches in research pipeline – Modular inducible cancer immunotherapy (MICI) – Ant
Slide 21: Using Natural Cell Biology to Produce Proteins: Adenoviral Approach
21 March 13, 2013 Using Natural Cell Biology to Produce Proteins: Adenoviral Approach Oral ligand activates DNA transcription, protein production begins Target Cell mRNA Translation Therapeutic proteins Adaptable Controlled Precision
Slide 22: Phase 1/2 Ad-RTS-IL-12 Candidate
22 March 13, 2013 Phase 1/2 Ad-RTS-IL-12 Candidate • Ad-RTS-IL-12 Phase 1b in metastatic melanoma – Biologically effective dose was determined – Clinical activity observed in 71% of patients (n=7), two highest dose cohorts – No dose-limiting toxicity reported to date • Ad-RTS-IL-12 Phase 2 in metast
Slide 23: Phase 2 Breast Cancer Ad-RTS-IL-12 + palifosfamide
23 March 13, 2013 Design Randomized, open-label study of Ad-RTS-IL-12 monotherapy or combination with palifosfamide in patients with recurrent/metastatic breast cancer and accessible lesions N Up to 68 subjects Population Recurrent/metastatic breast cancer Primary Endpoint 16-week PFS rate Select Se
Slide 24: Using Synthetic and Natural Cell Biology: Intramuscular Plasmid DNA
Sustained protein production and therapeutic anti-tumor efficacy drawing from only a single intramuscular administration of plasmid DNA. 24 March 13, 2013 Using Synthetic and Natural Cell Biology to Produce Proteins: Intramuscular Plasmid DNA EORTC-NCI-AACR International Conference on Molecular Targ
Slide 25: Next Generation: Multigenic Approach
IP1: LacZ rLUC Stuffer Stuffer Stuffer SP-RTS IP2: fLuc rLUC Stuffer Stuffer Stuffer SP-RTS IP3: SEAP rLUC Stuffer Stuffer Stuffer SP-RTS IP4: GUS rLUC Stuffer Stuffer Stuffer SP-RTS IP4: GUS rLUC SP-RTS IP1: LacZ IP2: fLuc IP3: SEAP 25 March 13, 2013 Next Generation: Multigenic Approach IP1: LacZ r
Slide 26: Financial Highlights
26 March 13, 2013 • Primary shares outstanding: approximately 83.2M • Cash: approximately $95.3M @ 9/30/12 • Current cash resources expected to support operations into 2H 2013 Financial Highlights
Slide 27: Major Clinical Milestones Over Next 18 Months
27 March 13, 2013 PHASE 2 BREAST CANCER (COMBO) MATISSE PICASSO 3 PHASE 2 MELANOMA Major Clinical Milestones Over Next 18 Months Q1-13 Q2-13 Q3-13 Q4-13 Q1-14 Q2-14 Possible NDA Submission TOP LINE DATA Possible ASCO Late-breaking Interim Analysis Palifosfamide IL-12 DNA Possible Phase 1 Melanoma AS
Slide 28: Better Cancer Medicine
Better Cancer Medicine Barclays Global Healthcare Conference Jonathan Lewis, MD, PhD Chief Executive Officer 28 March 13, 2013
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