Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — Caladrius Biosciences — Investor Presentation (March 2017)
35 slides from Lisata Therapeutics's investor presentation, Mar 22, 2017
Slide 1: Corporate Presentation
Corporate Presentation David J. Mazzo, PhD Chief Executive Officer March 2017 | NASDAQ: CLBS
Slide 2: Forward-looking statements advisory
Forward-looking statements advisory Additional Information About the Transaction and Where to Find it: Caladrius intends to file with the Securities and Exchange Commission (“SEC”) and mail to its stockholders a proxy statement in connection with, among other things, the proposed sale to Hitachi Che
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 CDMO PCT
Slide 5: Caladrius business model evolution plan
Caladrius business model evolution plan 5 CDMO PCT Cell Therapeutics CLBS03
Slide 6: Caladrius business model evolution plan
Caladrius business model evolution plan 6 CDMO PCT Cell Therapeutics CLBS03
Slide 7: Business model evolution – two healthy companies emerge
Caladrius business model evolution – two healthy companies emerge 7 Cell Therapeutics PCT CDMO
Slide 8: Caladrius unlocks PCT value and preserves working relationship
Caladrius unlocks PCT value and preserves intimate working relationship 8 Capital for Growth Cell Therapeutics PCT CDMO
Slide 9: Focused, funded and poised for growth
Focused, funded and poised for growth1 • Pure-play Cell Therapy therapeutics development company • Two technology platforms on which to build - T regulatory cells for immune modulation - CD34 for ischemic repair • On-going landmark phase 2 study of CLBS03 in recent onset type 1 diabetes - Strategic
Slide 10: Immune Modulation
Immune Modulation 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 (Jeffrey Bluestone – pioneer in T cell biology, et al) and the Centenary Institute • Autologous ex-vivo expanded polyclonal T regulatory cells • Exclusive ri
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 diseases
Polyclonal T regulatory cell therapy is potentially applicable across multiple autoimmune, alloimmune and allergic diseases 13 Combined markets represent a multi-billion dollar opportunity: 1. Pending grant application to fund a Phase 1 study in NMO with decision expected in 1H 2017 2. Global Patien
Slide 14: Simple, cost-effective, proprietary manufacturing process
Simple, cost-effective, proprietary Manufacturing process is scalable and commercially viable • Simple and efficient clinical manufacturing process: - Simple, less intrusive cell collection process (whole blood or, eventually, apheresis) - Reliable and well-characterized cGMP process - Extremely hig
Slide 15: CLBS03: Recent onset type 1 diabetes program overview
CLBS03: Recent onset type 1 diabetes program overview • Ongoing Phase 2 clinical study in T1D (T-Rex trial) - California Institute for Regenerative Medicine up to $12 million grant awarded upon achievement of certain milestones - DSMB satisfactory assessment of safety of initial cohort achieved ahea
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 re
Slide 17: CLBS03 occupies a unique position in the T1D treatment paradigm
CLBS03 occupies a unique position in the type 1 diabetes treatment paradigm 17 Chronic symptom treatment Disease Modification (CLBS03) Function regeneration Approach Treatment of symptoms Reduce or eliminate disease progression; potentially curative Replace ability to produce insulin with new cells/
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 12 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
Ischemic Repair O caladrius
Slide 22: CD34 cell therapy is supported by a strong rationale
CD34 cell therapy is supported by a strong rationale • 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 amputation in crit
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 Study Size • 35 patients enrolled across multiple centers in Japan Treatment • Up to 106 autologous G-CSF-mobilized peripheral blood-deri
Slide 24: Multiple grant opportunities in cardiovascular indications
Multiple grant opportunities in cardiovascular indications 24 Indication Coronary Microvascular Dysfunction Coronary Microvascular Dysfunction Grantor NIH Small Business Innovation Research Department of Defense Decision Expected 2Q17 2H17 Total Award Sought $1.9 million $6.9 million Trial Phase Ear
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 Joseph Talamo, CPA, MBA Senior VP and Chief
Slide 26: 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 27: 2017 Capital Catalysts and Impact
2017 Capital Catalysts and Impact YE2016 Financial Information Hitachi Acquisition of 80.1% of PCT from CLBS CIRM CLBS03 Grant Award (March 2017) Sept. 2016 PIPE – Second Tranche Cash: $14.7m • $5m on signing agreement (received March 17, 2017) • $2m from Sanford (accelerated payment from second tra
Slide 28: 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 29: Investor Relations Contact
Investor Relations Contact LHA Investor Relations Anne Marie Fields, Senior Vice President Phone: 212.838.3777 Email: [email protected] Web: www.caladrius.com 29 NASDAQ: CLBS
Slide 30: PCT: Cell & Cell-Based Gene Therapy CDMO
PCT: Cell & Cell-Based Gene Therapy CDMO 30
Slide 31: PCT: development and manufacturing partner for 18 years
PCT has been: A comprehensive development and manufacturing partner for 18 years • Expertise in multiple cell therapy types and therapeutic applications, including: - CAR-T, TCR, T-cell, NK cell, dendritic cells and CD34+ products, among others 31 cGMP Infrastructure Manufacturing Development cGMP M
Slide 32: PCT's deep experience: extensive client list
PCT’s deep experience is evidenced by: An extensive client list of renown cell therapy companies • Historically: >100 clients, 20,000 products and 6,000 patients • Critical contribution from PCT to development and/or clinical manufacturing • Several clients expected to be among next wave to reach co
Slide 33: PCT growth drivers: expanding cell therapy industry pipeline
465 Phase 2 1. Informa/Alliance for Regenerative Medicine. Regenerative medicine clinical trials are as of December 31, 2014 and December 31, 2016. Note that some Phase 2 trials are pivotal trials. 2. Based on company projections. FDA approvals based on pivotal cell therapy trials and historical rat
Slide 34: PCT's modern cGMP manufacturing facilities
PCT’s modern cGMP manufacturing facilities offer flexibility and mitigate risk • Allendale, NJ (30,000 ft2) - owned - 3 US-compliant cleanrooms - 5 EU and US-compliant cleanrooms (expansion completion in 2017) - Commercial product infrastructure • Mountain View, CA (25,000 ft2) - leased - 7 US-compl
Slide 35: PCT delivers a strategic solution beyond fee-for-service
PCT delivers: A strategic solution that moves well beyond fee-for-service 35 Clinical Manufacturing Process Development Commercial Manufacturing Strategic Manufacturing Assessment Cell therapy product concept Client may enter at any stage
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