Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — Caladrius Biosciences — Investor Presentation (January 2016)
28 slides from Lisata Therapeutics's investor presentation, Jan 11, 2016
Slide 1: Caladrius Biosciences Corporate Presentation
Caladrius Biosciences Corporate Presentation David J. Mazzo, PhD, Chief Executive Officer January 2016 NASDAQ: CLBS 1
Slide 2: Forward-looking statements
2 Forward-looking statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management’s current expectations, as of the date of this presentation, and involve certain risks and unce
Slide 3: Investment summary
• PCT: growing business providing development, manufacturing and delivery of cell-based therapies - Averaging 25% year-over-year revenue growth over past two years - Unmatched cell and cell-based gene therapy-specific experience and expertise - Expansive list of noteworthy cell and cell-based gene t
Slide 4: Transforming cells into therapies
Transforming cells into therapies From Concept to Commercial Product 4
Slide 5: PCT: Premier cell therapy development and manufacturing partner
Caladrius Biosciences’ Center of Excellence for process development, engineering and manufacturing cGMP Infrastructure Innovation cGMP Manufacturing Technology Transfer GTP Processing Logistics Process Optimization and Automation Consulting Analytical Development Manufacturing Development Quality PC
Slide 6: Unmatched experience: >120 clients, 20,000 products, 6,000 patients
Unmatched experience: >120 clients, 20,000 products and 6,000 patients over 17 years validate the benefits of PCT Selected Clients 6
Slide 7: PCT is a premier cell therapy service provider
7 PCT is a premier cell therapy service provider • Specializes in cell and cell-based gene therapy development and manufacturing • Proven efficiency enhancement and reduced capital investment for clients vs. internal commitment - From preclinical to commercial • Demonstrated regulatory expertise - 5
Slide 8: Expertise in multiple therapeutic applications and cell types
Expertise in multiple therapeutic applications and cell types • Immunotherapy - T cells / CAR-T cell therapy - Tumor cells - Dendritic cells - Natural killer cells - B cells - Macrophages - Donor lymphocyte infusion • Neuro/endocrine - Neural stem cells - Porcine islets • Hematopoietic replacement -
Slide 9: PCT offers a complete development pathway
9 PCT offers a complete development pathway Clinical manufacturing Process/ manufacturing development Commercial manufacturing Strategic Manufacturing Assessment Cell therapy product concept Client may enter at any stage
Slide 10: Delivering a strategic solution through flexible capacity
10 Delivering a strategic solution through flexible capacity • Supplants expensive, time-consuming facility build-outs and challenges in forecasting capacity use • Provides client-specific dedicated or shared scalable manufacturing solutions • “Partnership” model anticipates and provides prospective
Slide 11: Caladrius base business model
Caladrius base business model 11 Cell Therapy Developers & Academia Expertise Value Creation Revenue
Slide 12: Caladrius enhanced business model
Caladrius enhanced business model 12 Cell Therapy Developers & Academia Expertise Revenue Value Creation
Slide 13: CLBS03: Type 1 Diabetes Mellitus (T1D)
14 • T Cell technology • Strategic collaboration with Sanford Research • Phase 2 proof-of-concept stage • PCT-developed manufacturing process Immune Modulation CLBS03: Type 1 Diabetes Mellitus (T1D)
Slide 14: T Regulatory Cells (Tregs): restoring immune balance and function
15 T Regulatory Cells (Tregs): restoring immune balance and function Normal immune system: immune balance Infusion of Tregs: balance regained Autoimmunity: immune imbalance T regulatory cells T effector cells Natural polyclonal T regulatory cells Simple, cost-effective process with protected intelle
Slide 15: T1D: unmet medical needs create attractive commercial opportunity
16 T1D: unmet medical needs create attractive commercial opportunity • 18,000 children under 20 in US with recent-onset T1D per year1 • 3% annual growth rate worldwide2 • No curative treatments for T1D, only lifelong insulin therapy • Many serious co-morbidities: - Kidney failure - New cases of adul
Slide 16: Treg cell therapy appears durable in humans
17 Treg cell therapy appears durable in humans1 1. Gitelman et al, American Diabetes Association Abstract, 2014. 2. Dr. Jeffrey Bluestone Lab Study Leadership Jeffrey Bluestone, PhD of UCSF, leader in field of Tregs Design US UCSF/Yale open-label Phase 1 study, 4-dose escalation cohorts Patients 14
Slide 17: Treg cell therapy preserves beta cell function in children
Treg cell therapy preserves beta cell function in children1 Marek-Trzonkowska, N t al. Clinical Immunology 2014 1. Children aged 5-18 administered 1 (10 or 20 mil cells/kg) or 2 doses (total 30 mil cells/kg) of Tregs 0 0.2 0.4 0.6 0.8 1 1.2 Day O Month 4 One Year Fasting C-peptide levels C -p e p ti
Slide 18: TRex Study: Phase 2 proof-of-concept in adolescents with T1D
TRex Study: Phase 2 proof-of-concept in adolescents with T1D1 19 Design • Double-blind, placebo-controlled, randomized (1:1:1) trial • Adolescent patients ages 12 to 18 with recent-onset T1D Key Endpoints • Preservation of C-peptide level, insulin use, hypoglycemic episodes, Hemoglobin A1 C level (a
Slide 19: TRex Study: efficient asset de-risking study design
Interim blinded analysis when ~50% of subjects complete six-month follow up TRex Study: efficient asset de-risking study design 18 52 Initial 18 patients evaluated for safety (regulatory go/no-go) and immunological markers through three months Patient Enrollment 111 Sanford Research will: • Provide
Slide 20: TRex study timeline
Year 1 Part 1 N = 18 Start-up 1st patient randomized Enrollment & Treatment 3-Month F/U Year 2 Year 1 Part 2 N = 93 Start-up Enrollment & Treatment 19th patient randomized Year 2 Regulatory go/no-go Trex study timeline 21
Slide 21: Potential application across multiple autoimmune and allergic diseases
Potential application across multiple autoimmune and allergic diseases 22 Multibillion-dollar lifecycle opportunity Steroid-resistant asthma Multiple sclerosis (MS) Chronic obstructive pulmonary disease (COPD) Inflammatory bowel disease Graft vs. Host disease Rheumatoid arthritis Lupus
Slide 22: Additional Technology Platforms for Partnering
23 • Phase 2 data for both platforms • Applicability to multiple indications Additional Technology Platforms for Partnering • Tumor cell/dendritic cell technology for immuno-oncology • CD34 cell technology for ischemic repair
Slide 23: Tumor cell/dendritic cell technology for immuno-oncology
24 Tumor cell/dendritic cell technology for immuno-oncology • Uniquely targets cancer-initiating cells, applicability to multiple indications • Checkpoint inhibitors reduce impediments to an existing path - CLBS technology may open entirely new paths to multiple-antigen recognition • Promising Phase
Slide 24: CD34 cell technology for ischemic repair
25 CD34 cell technology for ischemic repair • CD34 cells shown to induce the development of new blood vessels, preventing tissue death by improving blood flow • Significant unmet need for critical limb ischemia (CLI) and chronic heart failure (CHF) • Seeking partnership for Japanese development for
Slide 25: Experienced executive team with broad domain-specific expertise
Experienced executive team with broad domain-specific expertise David J. Mazzo, PhD Chief Executive Officer 30+ years of experience in all aspects of large and emerging global biotech, biopharma company operations, successful international drug development Robert A. Preti, PhD Senior VP, Dev. and Te
Slide 26: Select financial information
27 Select financial information • Cash, cash equivalents and marketable securities*: $29.4 million • Long-term debt*: $15 million * As of September 30, 2015 0 5 10 15 20 25 30 35 Revenue in millions $ 2014 (Actual) 2015 (Guidance) 2016 (Guidance) $23m ↑28% $30m+ ↑30% $18m
Slide 27: Investment summary
Investment summary 28 • PCT: growing business providing development, manufacturing and delivery of cell-based therapies - Averaging 25% year-over-year revenue growth over past two years - Unmatched cell and cell-based gene therapy-specific experience and expertise - Expansive list of noteworthy cell
Slide 28: Investor Relations Contact
Investor Relations Contact: LHA Investor Relations Anne Marie Fields, Senior Vice President Phone: 212.838.3777 Email: afields@lhai.com Web: www.caladrius.com NASDAQ: CLBS 29
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