Lisata Therapeutics, Inc. · NASDAQ:LSTA
Lisata Therapeutics — Caladrius Biosciences — Investor Presentation (August 2015)
38 slides from Lisata Therapeutics's investor presentation, Aug 6, 2015
Slide 1: Delivering Personalized Medicine
Delivering Personalized Medicine Corporate Presentation NASDAQ: CLBS David J. Mazzo, PhD Chief Executive Officer August 2015
Slide 2: Forward-looking statements
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 uncert
Slide 3: Transforming cells into therapies
Transforming cells into therapies 3
Slide 4: PCT, a Caladrius company: premier cell therapy service provider
PCT, a Caladrius company: Industry-recognized single source premier cell therapy service provider Caladrius Biosciences’ Center of Excellence for process development, engineering and manufacturing PRODUCT & PROCESS DEVELOPMENT MANUFACTURING CELL & TISSUE PROCESSING LOGISTICS, STORAGE & DISTRIBUTION
Slide 5: Unmatched experience: >100 clients and 30,000 products over 15 years
Unmatched experience: >100 clients and 30,000 products over 15 years 5
Slide 6: At a glance
At a glance Unifying platform approach yielding a robust, balanced pipeline targeting critical unmet medical needs Proven internal center of excellence (PCT) with bicoastal facilities innovating discovery, development, manufacturing and delivery of cell-based therapies Highly experienced manag
Slide 7: Experienced executive and scientific team
Experienced executive and scientific team David J. Mazzo, PhD Chief Executive Officer 30+ years’ experience - all aspects of large and emerging global biotech, biopharma company operations, successful international drug development Robert S. Vaters, MBA President and Chief Financial Officer 25+ year
Slide 8: Robust and balanced pipeline
Immune modulation Ischemic repair Immuno- oncology Robust and balanced pipeline Fast Track & Orphan Drug Designations with Special Protocol Assessment Granted PRECLINICAL PHASE 1 PHASE 2 PHASE 3 8 PROGRAM CLBS20: metastatic melanoma (AKA NBS20, USAN = eltrapuldencel-T) CLBS14: CHF CLBS12: CLI PRESER
Slide 9: Unifying root approach across platforms
Unifying root approach across platforms Ischemic repair Immuno- oncology Immune modulation 9
Slide 10: Robust and balanced pipeline
Immune modulation Ischemic repair Immuno- oncology Robust and balanced pipeline Fast Track & Orphan Drug Designations with Special Protocol Assessment Granted PRECLINICAL PHASE 1 PHASE 2 PHASE 3 1 0 PROGRAM CLBS20: metastatic melanoma (AKA NBS20, USAN = eltrapuldencel-T) CLBS14: CHF CLBS12: CLI PRES
Slide 11: Immuno-oncology: Turning cancer against itself
Immuno-oncology: Turning cancer against itself Metastatic melanoma: CLBS20 (AKA NBS20, USAN = eltrapuldencel-T) • Fast Track & Orphan Drug designations • Special Protocol Assessment • ATMP (EMA) • $17.7 million CIRM grant award 11
Slide 12: Stage III recurrent/stage IV metastatic melanoma
Stage III recurrent/stage lV metastatic melanoma 1. American Cancer Society, 2014 SEER 2. For Stage IV metastatic melanoma - AJCC Cancer Staging 2010 (based on 17 academic centers) (Five-year data for recently approved melanoma immunotherapies is not yet reflected) 3. GBI Research – 2013 PREVALENCE
Slide 13: CLBS20 Phase 2 data suggests superior survival
0 10 20 30 40 50 60 70 80 Medi an O ve ra ll Su rvi va l ( m o n th s) Lower End 95% CI median overall survival Upper End 95% CI upper CI not estimable Confidence intervals not reported CLBS20 Phase 2 data suggests superior survival ipilimumab (BMS) dacarbazine (generic) vemurafenib (Roche) pembroli
Slide 14: CLBS20: Uniquely targets cancer-initiating cells
CLBS20: Uniquely targets cancer-initiating cells (CICs) Process times are approximate Surgical excision CIC isolation Expansion and irradiation (7-14 weeks) Incubate together and quality control (2-3 weeks) Subcutaneous injection (weekly for 3 wks and monthly for 5 mos) Leukapheresis Monocyte isolat
Slide 15: Phase 2: two trials; consistent, compelling data
0% 20% 40% 60% 80% Control Group Irradiated Tumor Cells Treatment Group 0% 20% 40% 60% 80% 100% 0 10 20 30 40 50 60 Pe rc en t Su rviv in g Months Phase 2: two trials; consistent, compelling data Dillman, et al. Journal Immunotherapy 2012 Historical control for distant metastases 2-YEAR OVERALL SURV
Slide 16: The Intus study: Phase 3 with SPA
The Intus study: Phase 3 with SPA and orphan drug and fast track designations DESIGN • Randomized (2:1), double blind, placebo controlled trial • Stage III recurrent or stage IV metastatic melanoma • Single trial for registration assuming positive outcome ENDPOINT • Overall survival POWERING • 80% p
Slide 17: Timeline to BLA
17 1Q 2015 Screening Initiated 4Q 2017 Interim Results after 99 Deaths 2Q 2015 First Patient Randomized 2Q 2018 Projected BLA Submission based on successful interim analysis 2Q 2019 Final Study Results 4Q 2019 Projected BLA Submission 4Q 2016 Enrollment Completed 2015 2016 2017 2018 2019 Study conti
Slide 18: Potential application for multiple solid tumor types
Potential application for multiple solid tumor types Multi-billion dollar lifecycle opportunity LUNG CANCER (Feasibility of cell lines from biopsies initiated) COLON CANCER (Feasibility of cell lines planned) OVARIAN CANCER (US FDA-cleared phase 2 protocol, cell line feasibility established) HEPATOC
Slide 19: Robust and balanced pipeline
Immune modulation Ischemic repair Immuno- oncology Robust and balanced pipeline Fast Track & Orphan Drug Designations with Special Protocol Assessment Granted PRECLINICAL PHASE 1 PHASE 2 PHASE 3 1 9 PROGRAM CLBS20: metastatic melanoma (AKA NBS20, USAN = eltrapuldencel-T) CLBS14: CHF CLBS12: CLI PRES
Slide 20: Ischemic repair
Ischemic repair Critical Limb Ischemia: CLBS12 Chronic Heart Failure: CLBS14 20
Slide 21: Ischemic repair: leveraging the body's natural repair mechanism
Ischemic repair: Leveraging the body’s natural repair mechanism to develop new blood vessels CD34/CXCR4 from peripheral blood • Ischemic events – caused by restriction of blood to tissue, e.g., stroke, acute myocardial infarction and claudication • Results of ischemia include chronic heart failure,
Slide 22: PreSERVE study
PreSERVE study: enrolled Phase 2 study in follow-up 22 DESIGN • Randomized (1:1), Phase 2, double blind, placebo controlled trial • Post-AMI (STEMI) patients PRIMARY ENDPOINTS AND KEY SECONDARY ENDPOINT • Change in cardiac perfusion from baseline to 6 months (exploratory endpoint) • Incidence rates
Slide 23: PreSERVE interim conclusions emphasize cell dose-dependent trends
PreSERVE interim conclusions emphasize cell dose-dependent trends* CD34 cell dose-dependent trend in reduction of MACE — Signal for a mortality benefit (12 month data) Signal for reduction in frequency of SAEs in higher dose groups (12 month data) CD34 cell dose-dependent trend in improvement
Slide 24: Significant Unmet Need for CLI and CHF
Significant Unmet Need for CLI and CHF NO-OPTION CRITICAL LIMB ISCHEMIA • If options for surgical or endovascular therapies are exhausted, patients only receive pain management, wound care or amputation • Even with surgical options over 50% lead to amputation or death within one year1 • Prevalence (
Slide 25: CLBS12 CLI Japan Program Update
25 CLBS12 CLI Japan Program Update Objective: file J-IND approval and consummate partnership to allow for clinical study execution Could take advantage of new Japanese regulatory path to early conditional approval Clinical background: Previous studies of autologous CD34+ cells in no-option CLI patie
Slide 26: Phase 3 study in Japan, targeting conditional approval
Phase 2 study in Japan, targeting conditional approval Study designed in consultation with PMDA 26 DESCRIPTION • Prospective, open label controlled, randomized, multicenter study in patients with no-option CLI DOSAGE • Up to 106 autologous G-CSF-mobilized peripheral blood-derived CD34+ cells/kg per
Slide 27: Potential application across several indications
Potential application across several indications Multi-billion dollar lifecycle opportunity STROKE CLAUDICATION 27 N-STEMI REFRACTORY ANGINA SYNDROME X Platform supported by independent preclinical and early clinical data, as well as positive suggestion of safety and therapeutic activity seen to dat
Slide 28: Robust and balanced pipeline
Immune modulation Ischemic repair Immuno- oncology Robust and balanced pipeline Fast Track & Orphan Drug Designations with Special Protocol Assessment Granted PRECLINICAL PHASE 1 PHASE 2 PHASE 3 2 8 PROGRAM CLBS20: metastatic melanoma (AKA NBS20, USAN = eltrapuldencel-T) CLBS14: CHF CLBS12: CLI PRES
Slide 29: Immune Modulation: Diabetes Mellitus Type-1 (T1D): CLBS03
Immune Modulation Diabetes Mellitus Type-1 (T1D): CLBS03 29
Slide 30: T Regulatory Cells (Tregs): restoring immune balance and function
30 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 PROCESS WITH PROTECTED INTELLECTUAL PROPERTY
Slide 31: Diabetes Mellitus Type-1 (T1D): an autoimmune disease
Diabetes Mellitus Type-1 (T1D): an autoimmune disease 1. Hamman RF, et al. Diabetes Care. 2014; Sosenko JM, et al. Diabetes Care. 2008; Palmer JP. Diabetes/metabolism research and reviews. 2009 2. The DIAMOND Project Group. Diabetic Medicine. 2006;23:857-866. PREVALENCE AND UNMET MEDICAL NEED • 18,0
Slide 32: Treg cell therapy appears durable in humans
Treg cell therapy appears durable in humans1 1. Gitelman et al, American Diabetes Association Abstract, 2014 2. Dr. Jeffrey Bluestone Lab PI Jeffrey Bluestone, PhD, of UCSF – leader in field of Tregs DESIGN U.S. UCSF/Yale open label Phase 1 study, 4-dose escalation cohorts PATIENTS 14 adult patients
Slide 33: Treg cell therapy preserves beta cell function in children
0 0.2 0.4 0.6 0.8 1 1.2 Day O Month 4 One Year 0% 20% 40% 60% 80% 100% Control Group Treatment Group Treg cell therapy preserves beta cell function in children1 33 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 mi
Slide 34: The Trutina study: Phase 2 in adolescents with T1D
The Trutina study: Phase 2 in adolescents with T1D1 34 DESIGN • Double blind, placebo controlled, randomized (1:1:1) • Adolescent patients with recent onset T1D ages 12 to 18 PRIMARY ENDPOINT • Preservation of C-peptide at 52 weeks in comparison to placebo POWERING • 80% power to detect 50% attenuat
Slide 35: Trutina study: Efficient asset de-risking study design
Trutina study: Efficient asset de-risking study design 35 18 51 Initial 18 patients evaluated for safety through 3-6 months (~$3 million cost to these results) PATIENT ENROLLMENT Interim blinded analysis when ~50% of subjects complete 12-month follow up Expected cost of trial: ~$22.5 million 111
Slide 36: Potential application across multiple autoimmune and allergic diseases
Potential application across multiple autoimmune and allergic diseases Multi-billion dollar lifecycle opportunity 36 STEROID RESISTANT ASTHMA MULTIPLE SCLEROSIS (MS) CHRONIC OBSTRUCTIVE PULMONARY DISEASE (COPD) INFLAMMATORY BOWEL DISEASE GRAFT VS. HOST DISEASE RHEUMATOID ARTHRITIS LUPUS
Slide 37: Investment summary
Investment summary Lead immuno-oncology program (Phase 3 study in metastatic melanoma) with Fast Track and Orphan Drug designations and a SPA Additional platforms for autoimmune disorders (FDA-cleared Phase 2 study in adolescents with type I diabetes) and multiple cardiovascular indications In
Slide 38: Delivering Personalized Medicine - Contact
Delivering Personalized Medicine NASDAQ: CLBS Contact: Eric Powers, Manager of Communications & Marketing Phone: 212.584.4173 Email: epowers@caladrius.com Web: www.caladrius.com
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