Alaunos Therapeutics, Inc. · NASDAQ:TCRT
Alaunos Therapeutics — ZIOPHARM Oncology — 19th Annual NewsMakers in the Biotech Industry Conference (September 2012)
27 slides from Alaunos Therapeutics's conference presentation, Sep 7, 2012
Slide 1: Better Cancer Medicine
Better Cancer Medicine 19 th Annual NewsMakers in the Biotech Industry Conference
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
Slide 3: Our Approach: Better Cancer Medicine
Early to Late - Stage Small Molecules DNA - Based Therapeutics Revolutionary Discovery Technology Poised to Cure Cancers Our Approach Better Cancer Medicine Diverse Portfolio of Therapeutics Addressing Unmet Medical Needs in Cancer
Slide 4: 2012 Anticipated Key Clinical Milestones
2012 Anticipated Key Clinical Milestones Indibulin: Oral Phase 1/2 Data Darinaparsin: Ongoing Studies with Solasia Early Stage Small Molecule Pipeline IL - 12 DNA Program Phase 1 Data 2012 Phase 2 Melanoma 2H 2012 New DNA Candidates • Preclinical Data 2012 Palifosfamide DNA Therapeutics PICASSO 3 ST
Slide 5: Data-Driven Oncology Portfolio
Compound Preclinical Phase 1 Phase 2 Phase 3 Palifosfamide Soft Tissue Sarcoma Small Cell Lung Cancer Other Phase 2 Study Oral DNA Therapeutics Ad IL - 12 DC IL - 12 Other Candidates Indibulin Breast Cancer Darinaparsin Oral Data - Driven Oncology Portfolio
Slide 6: Near-Term Value Drivers
• Palifosfamide – Wholly - owned asset – Two Phase 3 programs • Metastatic soft tissue sarcoma • S mall cell lung cancer – Potential in multiple solid tumor types • DNA therapeutics – Targeting established pathways – Approaching Phase 2 for melanoma – Exploring multiple avenues for revolutionary tre
Slide 7: Palifosfamide
Palifosfamide Septem
Slide 8: A Novel DNA-Targeted Drug
Broad Application Effect in solid tumors and hematological malignancies Safety, Efficacy and Accessibility Active in ALDH hi cells, rapid on - off kinetics, less toxic and ease of administration Addressing Unmet Needs Orphan Drug status for STS in U.S. and Europe Long Commercial and Development Runw
Slide 9: Soft Tissue Sarcoma
Soft Tissue Sarcoma $ in Millions • High unmet medical need; no first - line drug approved in ~30 years • First - line metastatic, non - GIST STS: 1 – Approximately 9,000 in U.S. 2 – Approximately 14,000 in Europe • Market in transformation – April 2012 FDA full approval of Votrient ® for second - l
Slide 10: Phase 3 Structure (PICASSO 3)
Phase 3 Structure ( PICASSO 3) N : Fully Enrolled - Approximately 424 patients; first - line metastatic STS Regimen: Palifosfamide + doxorubicin vs. doxorubicin + placebo Primary Endpoint: PFS for accelerated approval; OS for full approval Powered for PFS & OS PFS Power: 85% power to detect 0.60 HR,
Slide 11: Small Cell Lung Cancer
Small Cell Lung Cancer • High unmet medical need; little innovation in decades • Worldwide market opportunity: 250,000+ patients 1 – 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 toxicity 2 •
Slide 12: Phase 3 in SCLC (MATISSE)
Design Multinational, multi - center, randomized controlled, open - label, adaptive trial N Up to 548 subjects Population Males and females, age ≥18 years, with extensive - stage small cell lung cancer Primary Endpoint Overall survival (OS) Secondary Endpoints Progression free survival (PFS) Objecti
Slide 13: MATISSE Adaptive Trial Design
MATISSE Adaptive Trial Design Single pre - planned evaluation by the IDMC • Optimize chances of success • Stop trial for efficacy/futility • Complete trial as planned or resize study 125 Events Continue trial with decreased sample size Complete trial as planned Continue trial with increased event si
Slide 14: Palifosfamide Market Opportunity
Palifosfamide Market Opportunity
Slide 15: STS Market Position
• No uniform standard of care for first - line in U.S. market 1 – Opportunity to consolidate the market within 24 months after launch 2 • EU market primarily ifosfamide and/or doxorubicin 3 • Palifosfamide could become new standard of care in first - line metastatic, non - GIST STS 2 – Better effica
Slide 16: SCLC Market Position
• Etoposide plus carboplatin represents ~70% of current SOC 1 • Goal to establish palifosfamide in combination with etoposide and carboplatin as first - line standard of care in metastatic SCLC SCLC Market Position 1 Source : U.S.: IntrinsiQ Data, © Copyright 2012, IntrinsiQ, LLC an AmerisourceBerge
Slide 17: Market Potential
• Recent, U.S. orphan drug launches in oncology priced at $9K - $23K per cycle range – Examples of products: Adcetris ™ , Caprelsa ® , Istodax ® , Zelboraf ® • >$ 1 Bn total global market potential for palifosfamide in STS and SCLC Market Potential Scenarios of U.S. Only Peak Gross Revenues in $Mill
Slide 18: IL-12 DNA
IL-12 DNA On ZOPHARM Oncology en September? 2018 18
Slide 19: DNA Therapeutics
• R evolutionary technology for precise, controlled delivery of therapeutic proteins in vivo • Focused, disciplined approach to development – Minimize expense – Drive value through near - term proof - of - concept studies • Lead therapeutic in melanoma for early validation of target and platform • “
Slide 20: Using Natural Cell Biology to Produce Proteins
Using Natural Cell Biology to Produce Proteins Controlled Oral ligand activates DNA, protein production begins Target Cell mRNA Translation Precision Therapeutic proteins Adaptable
Slide 21: Two Clinical Stage Product Candidates
Two Clinical Stage Product Candidates • DC IL - 12 Phase 1b in metastatic melanoma – Safety profile predictable – 2 PRs, 2 SDs, biomarker effects • Ad IL - 12 Phase 1b in metastatic melanoma – S afe to date – Preliminary activity – Significant data expected 2H 2012 P ost - treatment Pre - treatment
Slide 22: Phase 1/2 of Ad-IL12 + AL in Melanoma
Next Phase of Development: • Remains a significant unmet need in metastatic melanoma despite evolving landscape • Phase 2 to begin at confirmation of biologically effective dose • Phase 2 modifications to include additional patient - selection criteria to maximize signal detection • Planned to be co
Slide 23: Early-Stage Development
Early - Stage Development
Slide 24: Early-Stage Clinical Development
DNA Therapeutics • Preclinical and discovery continuing to advance multiple antibody, immunotoxin, and protein decoy candidates • INDs next 12 to 24 months from preclinical work in progress Indibulin • Novel oral tubulin binding agent; expected low toxicity and neurotoxicity not seen • Ongoing Phase
Slide 25: Financial Highlights
• Primary shares outstanding: approximately 79.6M • Cash: approximately $ 110.4M @6/30/12 • Current cash resources expected to support operations into 2H 2013 Financial Highlights
Slide 26: 2012 Anticipated Key Clinical Milestones
2012 Anticipated Key Clinical Milestones Indibulin: Oral Phase 1/2 Data Darinaparsin: Ongoing Studies with Solasia Early Stage Small Molecule Pipeline IL - 12 DNA Program Phase 1 Data 2012 Phase 2 Melanoma 2H 2012 New DNA Candidates • Preclinical Data 2012 Palifosfamide DNA Therapeutics PICASSO 3 ST
Slide 27: Better Cancer Medicine
Better Cancer Medicine 19 th Annual NewsMakers in the Biotech Industry Conference