Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — Caladrius Biosciences — Investor Presentation (March 2018)
29 slides from Lisata Therapeutics's investor presentation, Mar 14, 2018
Slide 1: Corporate Presentation
Corporate Presentation David J. Mazzo, PhD President and Chief Executive Officer March 2018 | NASDAQ: CLBS
Slide 2: Safe Harbor for forward-looking statements advisory
Safe Harbor for forward-looking statements advisory This Investor Presentation contains forward-looking statements within the meaning of Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management’s current expectations, as of the date of this presentation, and in
Slide 3: Caladrius historical hybrid business model
Caladrius historical hybrid business model 3 Cell Therapeutics Development CDMO PCT
Slide 4: Caladrius recent business model evolution
Caladrius recent business model evolution 4 Cell Therapeutics Development CDMO PCT
Slide 5: Caladrius recent business model evolution
Caladrius recent business model evolution 5 Cell Therapeutics Development CDMO PCT
Slide 6: Caladrius business model evolution – two healthy companies emerge
Therapeutics Development HCATS CDMO Caladrius business model evolution – two healthy companies emerge 6
Slide 7: Caladrius redefined as financially stable with a simplified corporate profile
Therapeutics Development Caladrius redefined as financially stable with a simplified corporate profile 7 Capital for Growth Cell Therapeutics HCATS CDMO
Slide 8: Caladrius Biosciences: Focused, funded and poised for growth
Caladrius Biosciences: Focused, funded and poised for growth • Pure-play clinical stage therapeutics development company with two technology platforms - T regulatory cells for immune modulation - CD34 cells for ischemic repair • Well-funded and debt-free • On-going landmark phase 2 study of CLBS03 i
Slide 9: Immune Modulation: Autologous, polyclonal T regulatory cells
Immune Modulation Autologous, polyclonal T regulatory cells 9
Slide 10: T regulatory cell technology platform built on a strong foundation
T regulatory cell technology platform built on a strong foundation • T cell technology licensed from University of California at San Francisco (Prof. Jeffrey Bluestone – pioneer in T cell biology, et al) and the Centenary Institute • Autologous, ex-vivo expanded and activated, polyclonal T regulator
Slide 11: T Regulatory Cells (Tregs): Restoring immune balance and function
T Regulatory Cells (Tregs): Restoring immune balance and function • Deficiency in number or function of Tregs vs. T effector cells manifests as autoimmune disease • Augmentation of number/potency of Tregs is intended to restore the immune system to its “native” state and reduce/eliminate autoimmune
Slide 12: Polyclonal T regulatory cell therapy potentially applicable across multiple diseases
Polyclonal T regulatory cell therapy is potentially applicable across multiple autoimmune, alloimmune and allergic diseases, many qualifying as orphan 12 Combined markets represent a multi-billion dollar opportunity: 1. Global Patient numbers include total patients from US, EU and Japan only. 2. Ann
Slide 13: Treg therapy offers an attractive medical and commercial opportunity for T1D
T Regulatory Cell (Treg) therapy offers: An attractive medical and commercial opportunity for T1D • Each year >18,000 newly diagnosed patients under 20 years of age in US1; 3% CAGR worldwide2 • No curative treatments, only lifelong insulin therapy (often with serious co- morbidities) • Preserving re
Slide 14: Manufacturing process is scalable and economically viable
Addressing one of the major challenges of autologous cell therapy Manufacturing process is scalable and economically viable • Proprietary and efficient clinical manufacturing process: - Simple, minimally intrusive cell collection process (whole blood or, eventually, apheresis) - Reliable and well-ch
Slide 15: CLBS03: Recent onset Type 1 Diabetes program overview
CLBS03: Recent onset Type 1 Diabetes program overview • Ongoing landmark Phase 2 clinical study in T1D (T-Rex trial) - CIRM grant for up to $12.2 million based on specified milestone achievement - JDRF grant to Benaroya Research Institute to conduct extensive immune profile (study cost offset) - DSM
Slide 16: CLBS03 uniquely positioned in the type 1 diabetes treatment paradigm
CLBS03 to be uniquely positioned in the type 1 diabetes treatment paradigm 16 Chronic blood glucose management Disease Modification (CLBS03) Function regeneration Approach Symptom management Reduce or eliminate disease progression; potentially “curative” Replace depleted cells/organs producing insul
Slide 17: Published Phase 1 studies of Treg cell therapy
US Study1 EU Study2 Dose 4-dose escalation cohorts (0.05 x 108 to 26 x 108 cells) 1 infusion (10 or 20 million cells/kg) or 2 infusions (30 million cells/kg total) Patients 14 adult patients with established T1D 22 patients aged 5-18 with T1D Results • Demonstrated safety/tolerance - No cytokine rel
Slide 18: T-Rex Study: Phase 2 trial in adolescents with T1D
T-Rex Study: Phase 2 trial in adolescents with T1D initiated in March 2016 18 Rigorous Design • Double-blind, placebo-controlled, randomized (1:1:1) trial • Adolescent patients ages 8 to <18 with recent-onset (diagnosed within last 100 days) T1D Standard Endpoints • Preservation of C-peptide level,
Slide 19: T-Rex study timeline to primary endpoint analysis
2019 T-Rex study timeline to primary endpoint analysis after 12 months of follow-up 19 • Last patient randomized • 6-month follow-up of 50% of subjects • 50% of subjects treated • 70th subject enrolled Interim Analysis of Safety and Early Therapeutic Effect (futility analysis)* Topline Data: Primary
Slide 20: Ischemic Repair: CD34 cells
Ischemic Repair CD34 cells 20
Slide 21: CD34 cell therapy supported by a substantial body of clinical evidence
CD34 cell therapy is supported by a substantial body of clinical evidence • CD34 cells have been investigated in clinical studies with >400 patients exposed - Pre-clinical studies document improved microcirculation1 - Phase 2 clinical studies provide consistent evidence of benefit in safety and func
Slide 22: Japanese development program for critical limb ischemia (CLBS12)
22 Design • Prospective, open label, controlled, randomized trial • Patients with no-option CLI Advantageous Primary Endpoint • Time to continuous CLI-free status (2 consecutive monthly visits, adjudicated independently) Study Size • 35 patients to be enrolled across multiple centers in Japan Treatm
Slide 23: US development program for Syndrome X (CLBS14-CMD)
US development program for “Syndrome X” (CLBS14-CMD) Phase 1/2 proof-of-concept study in Coronary Microvascular Dysfunction 23 Design • Interventional, open label, exploratory (proof-of-concept) trial Primary Endpoint • Safety and the evaluation of adverse events Secondary Endpoints • Changes from b
Slide 24: US development program in Refractory Angina (CLBS14-RfA)
US development program in Refractory Angina (CLBS14-RfA) Late Stage Clinical Development Program Data Exclusively Licensed from Shire • Robust data included in exclusive license - Phases 1, 2, & 3 clinical studies1,2,3 (combined n=304) o Meta-analysis results using all three trials (published in the
Slide 25: Multi-Product Pipeline Based on Proprietary Technology Platforms
Grant Partner: NIH SBIR Country: USA Pre-Clinical Phase 1 Phase 2 Phase 3 Multi-Product Pipeline Based on Proprietary Technology Platforms 25 Collaborator: Sanford Research Grant Partners: CIRM & JDRF Country: USA Active trial Trial to be initiated Clinical development plan pending FDA discussions C
Slide 26: Experienced executive team with broad domain-specific expertise
Experienced executive team with broad domain-specific expertise David J. Mazzo, PhD President and Chief Executive Officer 30+ years of experience in all aspects of large pharma (Merck, Baxter, RPR, HMR, Schering-Plough) and emerging biopharma (Chugai USA, Regado) company operations, successful inter
Slide 27: Key Financials Figures
Key Financials Figures1 27 Current Cash2: $64.5m 2017 Average Quarterly Operating Cash Burn3: $5.4m Long-term Debt: $0 Common Shares Outstanding: 9.4m shares Options Outstanding: 1.1m shares (average exercise price $33.30) Warrants Outstanding: 0.3m shares (average exercise price $54.80) 1 As of Sep
Slide 28: Caladrius offers multiple potential near-term value creating milestones
Timeframe Treg Platform • T-rex interim analysis 6 months post treatment of 50% patients 1Q2018 (completed) • Analysis of 12 month data (primary efficacy endpoint) Early 2019 • 2-year follow-up complete Late 2019 CD34 Platform • Initiate 35 patient Phase 2 trial of CLBS12 in Japan for critical limb
Slide 29: Investor Relations Contact
Investor Relations Contact John D. Menditto Phone: 908.842.0084 Email: jmenditto@caladrius.com Web: www.caladrius.com 29 NASDAQ: CLBS
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