Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — Caladrius Biosciences — Investor Presentation (June 2017)
30 slides from Lisata Therapeutics's investor presentation, Jun 5, 2017
Slide 1: Corporate Presentation
Corporate Presentation David J. Mazzo, PhD Chief Executive Officer June 2017 | 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 transforms cells into therapies
Caladrius transforms cells into therapies 3
Slide 4: Caladrius historical hybrid business model
Caladrius historical hybrid business model 4 Cell Therapeutics CLBS03/CLBS12 CDMO PCT
Slide 5: Caladrius recent business model evolution
Caladrius recent business model evolution 5 Cell Therapeutics CLBS03/CLBS12 CDMO PCT
Slide 6: Caladrius recent business model evolution
Caladrius recent business model evolution 6 Cell Therapeutics CLBS03/CLBS12 CDMO PCT
Slide 7: Business model evolution – two healthy companies emerge
Cell Therapeutics PCT CDMO Caladrius business model evolution – two healthy companies emerge 7
Slide 8: Caladrius unlocks PCT value and preserves working relationship
Cell Therapeutics Caladrius unlocks PCT value and preserves intimate working relationship 8 Capital for Growth Cell Therapeutics PCT CDMO
Slide 9: Caladrius Biosciences: Focused, funded and poised for growth
Caladrius Biosciences: Focused, funded and poised for growth • Pure-play clinical stage therapeutics development company • Two technology platforms on which to build - T regulatory cells for immune modulation - CD34+ cells for ischemic repair • Well-funded with cash of ~$70 million • Debt-free • On-
Slide 10: Immune Modulation: Autologous, polyclonal T regulatory cells
Immune Modulation Autologous, polyclonal T regulatory cells 10
Slide 11: 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 12: 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 13: Polyclonal Treg therapy applicable across multiple autoimmune diseases
Polyclonal T regulatory cell therapy is potentially applicable across multiple autoimmune, alloimmune and allergic diseases, many qualifying as orphan 13 Combined markets represent a multi-billion dollar opportunity: 1. Global Patient numbers include total patients from US, EU and Japan only. 2. Ann
Slide 14: Simple, cost-effective, proprietary manufacturing process
Simple, cost-effective, proprietary Manufacturing process is scalable and economically viable • Simple and efficient clinical manufacturing process: - Simple, minimally intrusive cell collection process (whole blood or, eventually, apheresis) - Reliable and well-characterized cGMP process - Extremel
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) - C.I.R.M. 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) -
Slide 16: Treg therapy: attractive medical and commercial opportunity for T1D
T Regulatory Cell (Tregs) 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 r
Slide 17: CLBS03 unique position in type 1 diabetes treatment paradigm
CLBS03 occupies a unique position in the type 1 diabetes treatment paradigm 17 Chronic blood glucose management Disease Modification (CLBS03) Function regeneration Approach Symptom management Reduce or eliminate disease progression; potentially “curative” Replace depleted cells/organs producing insu
Slide 18: 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 19: T-Rex Study: Phase 2 trial in adolescents with T1D
T-Rex Study: Phase 2 trial in adolescents with T1D initiated in March 2016 19 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 20: T-Rex Study: Timeline including near-term milestones
2019 T-Rex Study: Timeline including near-term milestones 20 • Last (111th) patient randomized • 6-month follow-up of 50% of subjects • 50% of subjects treated • 70th subject enrolled Interim Analysis of Early Therapeutic Effect Topline Data: Primary Efficacy Endpoint 1 year Analysis 2016 Study init
Slide 21: Ischemic Repair: CD34+ cells
Ischemic Repair CD34+ cells 21
Slide 22: CD34 cell therapy supported by a body of clinical evidence
CD34 cell therapy is supported by a profound body of clinical evidence • CD34 cells have been investigated in clinical studies encompassing >700 patients - Pre-clinical studies document improved microcirculation1 - Phase 2 clinical studies consistently show benefits in safety and function o Reduced
Slide 23: Japanese development program for critical limb ischemia
23 Design • Prospective, open label, controlled, randomized trial • Patients with no-option CLI Advantageous Primary Endpoint • Time to continuous CLI-free status (defined as 2 consecutive monthly visits) Study Size • 35 patients enrolled across multiple centers in Japan Treatment • Up to 106 autolo
Slide 24: Grant opportunity in additional cardiovascular indication
Grant opportunity in additional cardiovascular indication 24 Indication Coronary Microvascular Dysfunction Grantor NIH Small Business Innovation Research Decision Expected Mid-2017 Total Award Sought $1.9 million Trial Phase Early Phase 2 Number of Subjects 20 Study Initiation Within 5 months of awa
Slide 25: Multi-Product Pipeline Based on Proprietary Technology Platforms
Pre-Clinical Phase 1 Phase 2 Phase 3 Multi-Product Pipeline Based on Proprietary Technology Platforms 25 Collaborator: Sanford Research Grant Partner: CIRM Country: USA Active trial Trial to be initiated Active trial: Investigator sponsored (Bluestone) Country: Japan CLBS14 CMD CD34 Cell Platform (I
Slide 26: 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 pharma (RPR, HMR, Schering- Plough) and emerging biopharma (Chugai USA, Regado) company operations, successful international drug development ac
Slide 27: 2016 Goals and Results
2016 Goals Results in 2016 Grow and expand the PCT business on all fronts • 57% annual revenue growth to annual revenue of $35.3 million • Initiated global collaboration and license agreement with Hitachi Chemical • Began 5-year agreement with Adaptimmune for late-stage clinical supply Advance CLBS0
Slide 28: 2017 Capital Catalysts and Impact
2017 Capital Catalysts and Impact March 31, 2017 Financial Information Hitachi Acquisition of 80.1% of PCT from CLBS for $75m Cash (closed on May 18, 2017) CIRM CLBS03 Grant Award Sept. 2016 PIPE – Second Tranche Cash: $12.0m $75m Transaction • $5m received as advance payment in 1Q17 • $65m received
Slide 29: Expected Timeframe
Expected Timeframe CLBS03 • DSMB safety assessment on 1st patient cohort Completed 2016 • Initiation of enrollment of 2nd patient cohort Completed 2016 • 50% of patients treated: starts clock to 6-mos. follow-up interim analysis Mid-2017 • 70th patient enrolled: triggers capital infusion Mid-2017 •
Slide 30: 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 30 NASDAQ: CLBS
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