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Caladrius Corporate Presentation - November, 2018

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Caladrius Corporate Presentation - November, 2018

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Caladrius Corporate Presentation - November, 2018

  1. 1. Corporate Presentation David J. Mazzo, PhD President and Chief Executive Officer November 2018 | NASDAQ: CLBS
  2. 2. 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 involve certain risks and uncertainties. All statements other than statements of historical fact contained in this Investor Presentation are forward-looking statements. The Company’s actual results could differ materially from those anticipated in these forward-looking statements as a result of various factors. Factors that could cause future results to differ materially from the recent results or those projected in forward-looking statements include the “Risk Factors” described in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”) on March 22, 2018, as subsequently amended on April 2, 2018, and and in the Company’s other periodic filings with the SEC. The Company’s further development is highly dependent on, among other things, future medical and research developments and market acceptance, which are outside of its control. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date of this Investor Presentation. Caladrius does not intend, and disclaims any obligation, to update or revise any forward- looking information contained in this Investor Presentation or with respect to the matters described herein. 2
  3. 3. Caladrius Biosciences: Uniquely positioned for near-term success ▪ Late-stage therapeutics development company ▪ Four active development programs; two with “breakthrough” designation* ▪ CD34+ cells for ischemic repair (CLBS12*, CLBS14-CMD, CLBS14-RfA*) ▪ T regulatory cells for immune modulation (CLBS03) ▪ Financially stable and debt-free ▪ Strong balance sheet (~$50 million cash as of June 30, 2018) ▪ Low operating cash burn (~$5 million/quarter) ▪ Multiple value creating events within the next 18 months ▪ Key regulatory and data milestones across the pipeline 3
  4. 4. 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); successful international drug development across all therapeutic areas, international capital raising and business transactions; Director and former Chairman of EyePoint Pharma Douglas W. Losordo, MD Executive VP, Global Head of R&D and Chief Medical Officer 25+ years of experience as a leader in cell therapy research and development; renowned clinician with noteworthy academic (Tufts, Northwestern, NYU) and industry (Baxter) credentials; pioneer of CD34+ cell therapy Joseph Talamo, CPA, MBA Senior VP and Chief Financial Officer 25+ years of experience as a versatile finance executive with strong accounting/audit background (KPMG) and leadership roles in publicly traded pharmaceutical development and commercial-stage companies (OSI Pharmaceuticals, BMS) Todd Girolamo, JD, MBA Senior VP, General Counsel and Corporate Secretary 25+ years of legal experience as a practicing attorney (Cahill, Gordon & Reindel; Reid & Priest) as well as finance and biotechnology industry experience (Oppenheimer, CIBC, Leerink Swann) John D. Menditto Executive Director, Investor Relations and Corporate Communications 20+ years of experience as an investor relations and corporate communications professional with a major focus on healthcare and life science (Novartis, Medco Health Solutions, Argos Therapeutics) 4
  5. 5. Ischemic Repair Autologous CD34+ cells 5
  6. 6. CD34+ cells promote angiogenesis of the microvasculature ▪ >700 subjects studied in randomized double-blind placebo-controlled trials provide consistent evidence of therapeutic benefit and tolerance ▪ Improved mortality, reduced chest pain and increased exercise tolerance in refractory angina1 ▪ Reduced amputation in critical limb ischemia2 ▪ Improved function in claudication3 6 1. Losordo et al. Circ Res 2011.; Povsic et al. JACC Cardiovasc Interv. 2016. 2. Losordo et al. Circ Cardiovasc Interv 2012. 3. From US study (n=17); Not yet published Normal microvasculature Augmented microvasculature post-CD34+ cells introduction Compromised microvasculature
  7. 7. Simple, scalable and economical autologous cell therapy process 7 Cells returned to same patient Maximum of 4 days from donation to injection Day -3: Patient dosed with GCSF to mobilize CD34+ cells from bone marrow to peripheral blood Day 1: Sample collection via apheresis; shipment to processing center Day 2: CD34+ cells isolated, preparation for patient injection and shipment to clinic Day 3-4: Cells returned to patient through intramuscular injection, intracoronary injection or intramyocardial injection, depending on indication Isolation of CD34+ cells Sample collection ShipmentShipment
  8. 8. CLBS12 Critical Limb Ischemia (CLI) (Japan) 8
  9. 9. 9 CLI Represents a Multi-billion Dollar Global Market Opportunity Japan USA Europe* No-option CLI patients eligible for CLBS12 (not eligible for revascularization) ~51,000 ~300,000 ~560,000 CLI represents an expedited commercial opportunity in Japan *Europe: Source: Independent Third-party analysis; Full third-party report available upon request ▪ CLBS12 eligible for early conditional approval based on current phase 2 study ▪ Estimated >$100M initial commercial opportunity with significant pharmaco-eco benefits ▪ Successful outcome in Japan could lead to expedited development in other major markets
  10. 10. 10 Week 12 Post-treatment Before Treatment Kawamoto et al, Atherosclerosis 2012 Single CD34+ cell therapy administration results in permanent reversal of CLI Actual CLI Patient Laser Doppler Imagery
  11. 11. 11 Design • Prospective, open label, controlled, randomized trial (1:1 w/SOC) CLI patients Primary Endpoint • Time to continuous CLI-free status (2 consecutive monthly visits, adjudicated independently) Study Size • 30 patients with no-option CLI plus 5 patients with Buerger’s Disease; ~10 centers in Japan Dose • Up to 106 autologous G-CSF-mobilized peripheral blood-derived CD34+ cells/kg per affected limb Control/ comparator • Standard of Care drugs approved in Japan (e.g., antiplatelets, anticoagulants and vasodilators) • Choice of pharmacotherapy will be made by the investigators according to protocol Mode of administration • Intramuscular, 20 injections in affected lower limb in single administration Timing/Cost • First patient enrolled in December 2017 with final results expected early 2020 • ~$7 million costs remain to study completion (fully funded) CLBS12 pivotal phase 2 study in Japan Awarded SAKIGAKE (“breakthrough”) designation with priority review Eligible for early conditional approval
  12. 12. CLBS14-CMD Coronary Microvascular Dysfunction (CMD) (USA) 12
  13. 13. 13 CMD is an unmet medical need with significant market potential 1Cleveland Clinic/AHA (American Heart Association) 2Townsend, N, et al.: Cardiovascular disease in Europe: epidemiological update 2016, European Heart Journal, Volume 37, Issue 42, 7 November 2016, Pages 3232–3245 3Kita, T; Coronary heart disease risk in Japan – an East/West divide?, European Heart Journal Supplements, Volume 6, Issue suppl_A, 1 March 2004, Pages A8–A11 4Ueshima, H, et al.; Cardiovascular Disease and Risk Factors in Asia, AHA Journal, December 16/23, 2008, Volume 118, Issue 25 ▪ Nearly 50% of patients with Coronary Artery Disease (CAD) have CMD ▪ Multi-billion dollar global opportunity based on significant pharmaco-eco benefits USA1 Europe2 Japan3,4 ~8,300,000 ~6,000,000 ~1,000,000 CMD Patients Eligible for CLBS14-CMD Europe:
  14. 14. CLBS14-CMD Phase 1b/2a proof-of-concept study (ESCaPE-CMD) Currently enrolling patients in USA 14 Design • Interventional, open label, proof-of-concept trial Primary Endpoint • Safety and the evaluation of adverse events Secondary Endpoints • Changes from baseline to 6 months for coronary flow reserve, endothelial-dependent microvascular function, time to angina; other cardiovascular metrics Study Size • 20 patients at 2 centers in the USA (Cedars Sinai, LA & Mayo Clinic) Dose • Up to 300 x 106 CD34+ cells Control • No control arm Mode of administration • Single intracoronary infusion Timing/Cost • First patient enrolled April 2018 with final results expected by end of 2019 • NIH grant covers majority of costs (CLBS to contribute ~$0.7 million – fully funded)
  15. 15. CLBS14-RfA Refractory Angina (RfA) (USA) 15
  16. 16. RfA Patients Eligible for CLBS14-RfA CLBS14-RfA presents a multi-billion dollar global market opportunity 16 Europe: USA Europe Japan ~200K ~100K ~100K ▪ Patients with RfA often have multiple costly comorbidities Source: Independent Third-party analysis; Full third-party report available upon request
  17. 17. US development program in Refractory Angina (CLBS14-RfA) ▪ Phases 1, 2, & partial 3 clinical studies1,2,3 completed (combined n=304) ▪ Patient-level pooled-analysis compelling results published in European Heart Journal, 1/2018 17 1 Losordo, D.W., et al, Intramyocardial transplantation of autologous CD34+++ stem cells for intractable angina: a phase I/Iia double-blind, randomized controlled trial. Circluation, 2007. 115(25): p. 3165-3172 2 Losordo, D.W., et al., Intramyocardial, autologous CD34+++ cell therapy for refractory angina. Circ Res, 2011. 109(4): p. 428-36. 3 Povsic, T.J., et al., The RENEW Trial: Efficacy and Safety of Intramyocardial Autologous CD34++(+) Cell Administration in Patients With Refractory Angina. JACC Cardiovasc Interv, 2016. 9(15): p. 1576-85. IMPROVED total exercise time throughout the 3– 12 month period on treadmill stress test Significant DECREASE in all-cause mortality at 24 months LOWER relative frequency of angina throughout the 3–12 month period
  18. 18. US development program in Refractory Angina (CLBS14-RfA) ▪ Phases 1, 2, & partial 3 clinical studies1,2,3 completed (combined n=304) ▪ Patient-level pooled-analysis compelling results published in European Heart Journal, 1/2018 18 1 Losordo, D.W., et al, Intramyocardial transplantation of autologous CD34+++ stem cells for intractable angina: a phase I/Iia double-blind, randomized controlled trial. Circluation, 2007. 115(25): p. 3165-3172 2 Losordo, D.W., et al., Intramyocardial, autologous CD34+++ cell therapy for refractory angina. Circ Res, 2011. 109(4): p. 428-36. 3 Povsic, T.J., et al., The RENEW Trial: Efficacy and Safety of Intramyocardial Autologous CD34++(+) Cell Administration in Patients With Refractory Angina. JACC Cardiovasc Interv, 2016. 9(15): p. 1576-85. Total exercise time (TET) and angina frequency results Auto-CD34+ Cells Placebo
  19. 19. US development program in Refractory Angina (CLBS14-RfA) ▪ Phases 1, 2, & partial 3 clinical studies1,2,3 completed (combined n=304) ▪ Patient-level pooled-analysis compelling results published in European Heart Journal, 1/2018 19 1 Losordo, D.W., et al, Intramyocardial transplantation of autologous CD34+++ stem cells for intractable angina: a phase I/Iia double-blind, randomized controlled trial. Circluation, 2007. 115(25): p. 3165-3172 2 Losordo, D.W., et al., Intramyocardial, autologous CD34+++ cell therapy for refractory angina. Circ Res, 2011. 109(4): p. 428-36. 3 Povsic, T.J., et al., The RENEW Trial: Efficacy and Safety of Intramyocardial Autologous CD34++(+) Cell Administration in Patients With Refractory Angina. JACC Cardiovasc Interv, 2016. 9(15): p. 1576-85. Major cardiac events (MACE) results
  20. 20. US development program in Refractory Angina (CLBS14-RfA) 20 ▪ Data license obtained from Shire in 1Q18 ▪ CLBS owns IP for product ▪ IND reactivated with CLBS as sponsor ▪ RMAT (Regen Medicine Advanced Therapy) designation – awarded 2Q2018 ▪ Granted by the FDA for therapies intended to treat serious conditions ▪ Therapy must show preliminary evidence of addressing unmet medical need ▪ Similar to breakthrough therapy designation, it includes increased agency interaction and potential for accelerated approval ▪ FDA meeting to finalize BLA development plan arranged for 4Q2018 ▪ Preliminary discussions indicate grant funding likely to contribute to any remaining clinical studies
  21. 21. Immune Modulation Autologous, ex vivo expanded & activated polyclonal T regulatory cells CLBS03 (USA) 21
  22. 22. T regulatory cells (Tregs) control immune balance and function ▪ Deficiency in number or function of Tregs manifests as autoimmune disease ▪ Augmentation of Tregs is intended to restore the immune system to its “native” state and reduce/eliminate autoimmune disease symptoms and progression 22 1 Normal immune system: immune balance 2 Autoimmunity: immune imbalance 3 Infusion of Tregs: Immune balance restored T regulatory cells T effector cells Natural polyclonal T regulatory cells
  23. 23. Immune modulation critical to curtailing disease progression 23 Chronic blood glucose management Disease Modification (CLBS03) Function regeneration Approach Symptom management Reduce or eliminate disease progression; potentially “curative” Replace depleted cells/organs producing insulin; does not address underlying autoimmune disease Insulin Impact Improve therapeutic effect and/or efficiency of delivery of insulin/analogs Avoid or reduce need for insulin by preserving active beta cells Provides new source of insulin producing cells Availability Currently available with more in development Currently in Phase 2 trial Many years of development remaining
  24. 24. 24 USA4 Europe5 Japan6 New onset T1D patients eligible for CLBS03 19,000 54,500 3,000 T1D is a >$1 billion worldwide market opportunity 5Europe: ▪ Each year ~20,000 newly diagnosed patients <20 years of age in USA1 ▪ 3% CAGR worldwide2 ▪ No curative treatments, only lifelong insulin therapy ▪ Frequent serious, costly co-morbidities ▪ Preserving remaining beta cell function should slow/stop disease progression ▪ Leading to long-term pharmaco-economic benefits3 1. National Diabetes Statistic Report, 2014 2. Maahs DM, et al. Endocrinol Metab Clin North Am. 2010 3. Nathan DM, et al. Arch Intern Med. 2009 4. Thunander M et al, Diabetes Res Clin Pract. 2008:82:247-255 5. Haller MJ et al, Pediatr Clin North Am. 2005;52:1553-157 6. Kawasaki E., Matsuura N., Eguchi K., Diabetologia, 2006:49(5):828-36
  25. 25. Reliable, scalable & economically viable autologous cell therapy process ▪ Proprietary and efficient clinical manufacturing process: ▪ Simple, minimally intrusive cell collection process (whole blood or, eventually, apheresis) ▪ Reliable and well-characterized cGMP process ▪ High Phase 2 manufacturing success rate (>93%) ▪ Discounted development and manufacturing services rates from HCATS through 2024 1 Day 1: Patient whole blood donation 3 Day ~14: Infusion of Treg therapy to same patient Collection & Shipment Processing & Return Shipment Infusion 2 Days 2-13: Treg isolation, activation & expansion 25
  26. 26. CLBS03: Recent onset Type 1 Diabetes program overview ▪ International regulatory recognition ▪ FDA Fast Track designation (first time granted to a T1D program) and Orphan designation ▪ EU ATMP (Advanced Therapeutic Medicinal Product) classification ▪ Enrollment completed for landmark Phase 2 clinical study in T1D (T-Rex trial) ▪ T-rex trial based on published clinical studies showing the T regulatory cell therapy is well tolerated1,2, durable1 and preserving of beta cell function in children2 ▪ CIRM and JDRF grants of ~$10 million combine to offset study costs ▪ Strategic collaboration with Sanford Research (Sioux Falls, SD) providing $5 million in equity investment plus operating support for trial and clinical sites ▪ Planned interim analysis completed: therapy is well tolerated and non-futile for therapeutic effect ▪ Primary endpoint analysis expected in early 2019 26 1. Bluestone, et al. Science Translational Medicine 2015 2. Marek-Trzonkowska, N et al. Clinical Immunology 2014 3. Remission Definition: Daily dose of insulin ≤ 0.5 UI/kg body weight & fasting c-peptide > 0.5 ng/ml at 12 months after recruitment
  27. 27. Phase 2 (T-rex) trial in adolescents with T1D initiated in March 2016 27 Design • Double-blind, placebo-controlled, randomized (1:1:1) trial • Adolescent patients ages 8 to <18 with recent-onset T1D (diagnosed within 100 days) Standard Endpoints • Preservation of C-peptide level, insulin use, severe hypoglycemic episodes, glucose and hemoglobin A1c levels Study Size • 110 patients enrolled across 15 study sites in the USA (enrollment completed in Dec. 2017) Power • 80% power to detect a 0.2 pmol/mL difference in AUC mean C-peptide (active vs. placebo) Dose • CLBS03 dose cohorts of 2.5 or 20 million cells/kg body weight Control • Placebo (standard of care including insulin) Mode of Administration • Single infusion Timing/Cost • Top-line data in early 2019 • <$2 million in study costs remaining (fully funded)
  28. 28. Country: USA Pre-Clinical Phase 1 Phase 2 Phase 3 Country: Japan Multi-product pipeline based on proprietary technology platforms 28 Active trial Development plan to BLA pending FDA meeting in 4Q18 CLBS14-CMD Coronary Microvascular Dysfunction CD34+ Cell Therapy Platform (Ischemic Repair) T Regulatory Cell Therapy Platform (Immune Modulation) CLBS12 Critical Limb Ischemia * CLBS03 Recent Onset Type 1 Diabetes CLBS14-RfA Refractory Angina Country: USA *Eligible for early conditional approval Country: USA
  29. 29. FDA Meeting Program 2018(Oct-Dec) 2019 2020 Key dates and upcoming potential value creating events 29 CLBS03 (T1D)---------------------------------------------------------------------------------------------------------------------------------- CLBS12 (Critical Limb Ischemia)--------------------------------------------------------------------------------------------------------------- CLBS14-RfA (Refractory Angina)------------------------------------------------------------------------------------------------------------- Phase 2 Topline Data Expected Phase 2 Topline Data Expected Phase 2 Topline Data Expected CLBS14-CMD (Coronary Microvascular Dysfunction)----------------------------------------------------------------------------------------
  30. 30. CLBS key financial information1 30 Current Cash2: $50.3m 2018 Actual (Jan-Jun) Operating Cash Burn3: $12.2m 2018 Projected (Jul-Dec) Operating Cash Burn4: $10m Cash Runway Based on Current Plan4: 2020 Long-Term Debt: $0 Common Shares Outstanding: 9.7m shares Options Outstanding: Options Outstanding with Exercise Price < $5.00 = 551,000 shares Options Outstanding with Exercise Price < $25.00 = 200,000 shares Options Outstanding with Exercise Price > $25.00 = 393,000 shares 1.1m shares (average exercise price $33.50) 1 As of June 30, 2018 2 Cash, cash equivalents and marketable securities 3 Includes approximately $2m in final retention payments related to the PCT sales transaction of 2017 4 Excludes CLBS14-RfA development costs - to be determined
  31. 31. Caladrius Biosciences: Uniquely positioned for near-term success ▪ Late-stage therapeutics development company ▪ Four active development programs; two with “breakthrough” designation* ▪ CD34+ cells for ischemic repair (CLBS12*, CLBS14-CMD, CLBS14-RfA*) ▪ T regulatory cells for immune modulation (CLBS03) ▪ Financially stable and debt-free ▪ Strong balance sheet (~$50 million cash as of June 30, 2018) ▪ Low operating cash burn (~$5 million/quarter) ▪ Multiple value creating events within the next 18 months ▪ Key regulatory and data milestones across the pipeline 31
  32. 32. Investor Relations Contact John D. Menditto Phone: 908.842.0084 Email: jmenditto@caladrius.com www.caladrius.com 32 NASDAQ: CLBS

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