Developing ng R Regene nerat ativ ive Therap apie ies that R Reverse Ch Chroni nic D Dis iseas ase David J J. Ma . Mazzo, PhD hD President and C Chief E f Executive Officer May 14, 2020 | Nasdaq: CLBS
Forward rd-lo looking statement This Investor 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 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 5, 2020 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
Present ntation on contents Investment case summary Management team introduction CD34+ cell therapy platform technology overview Pipeline description and individual program summaries Financial overview Milestone timeline Conclusion 3
Caladri rius investment rationale CD34+ cell therapy company with an advanced clinical pipeline with 2 programs with “breakthrough” designation and 1 targeting COVID-19 induced lung repair Proprietary field-leading technology in multi-billion dollar global indications backed by a strong IP portfolio Multiple potential value creating events in the next 12-18 months based on milestones across the pipeline (timing subject to COVID-19 pandemic influence) Seasoned management team with noteworthy domain expertise along with big pharma and emerging biotech experience Strong balance sheet; ~$34 million in cash & cash equivalents (April 30, 2020) with no debt and cash runway projected to fund operations into 2H 2021 4
Caladri rius management team Da David J J. Mazz azzo, P PhD D Douglas Losor ordo, o, M MD Joseph T h Talamo, C CPA Todd Girol olamo, o, J JD John M Menditt tto President and EVP, Global Head of R&D and Senior VP and Senior VP, General Counsel Vice President, IR and Chief Medical Officer Chief Financial Officer and Corporate Secretary Corporate Communications Chief Executive Officer 5 Note: Select experience is shown above. For a comprehensive bio, please visit: www.caladrius.com
Esteemed scientific advisory board C. Michael Gibson, MD Timothy Henry, MD C. Noel Bairey Merz, MD Cedars-Sinai, L Los Angeles Harvard M Medical S School ool The C Christ H Hospita tal, C Cincinnati ti Thomas Povsic, MD, PhD Richard Schatz, MD Christopher White, MD Duke C Clinical Research I Insti titu tute Scripps Clinic, S San D Diego Ochsner er Health, N New ew Orlea eans Andreas Zeiher, MD Carl Pepine, MD Joseph Wu, MD, PhD Goeth the U Unive versity ty, Frankfurt Unive versity of Florida, G Gainesvi ville Stanford Cardiovascular I Insti titu tute 6
CD34+ cell therapy platform technology overview
CD3 D34+ c 4+ cells ls have a well characterized mechanism of action Augmented microvas asculat ature post-CD CD34+ cells treat atment Normal al microvascu culat ature Compromis ised microvas asculat ature Capil illaries Capil illaries Capil illaries Ne New Capil illaries Artery Vein in Artery Vein in Artery Vein in Naturally occurring endothelial progenitor cells Reestablish blood flow to under-perfused tissues 1,2 Possess pre-programmed tissue repair effects mediated by pro-angiogenic and anti-inflammatory functions 3,4 3 Abd-Allah et al., Cytotherapy 2015, 17: 443-53 1 Mackie, A.R. et al., Tex Heart Inst J 2011, 38(5), 474-485 8 4 Lo , B.C. et al., Am J Respir Cell Mol Biol 2017, 57: 651-61 2 Kocher, A.A. et al., Nat Med 2001, 440-436
CD3 D34+ c 4+ cell th l thera rapy is extensively studied/clinically validated CD34+ cells have been studied clinically in a variety of ischemic disease indications by numerous investigators across many sites and countries CD34+ cells repeatedly have shown evidence of vascular repair in multiple organs Consistent and compelling results of rigorous clinical studies comprising >1,000 patients have been published in peer reviewed journals 1,2,3,4 Single treatment has elicited durable therapeutic effect No cell-related adverse events reported to date 1 Povsic, T. et al. JACC Cardiovasc Interv , 2016, 9 (15) 1576-1585 3 Velagapudi P, et al, Cardiovas Revasc Med , 2018, 20(3):215-219 9 2 Losordo, D.W. et al. Circ Cardiovasc Interv, 2012; 5:821–830 4 Henry T.D., et al, European Heart Jour 2018, 2208–2216
Caladri rius’ CD34 process is simple/fast/economical/scaled Sample collection on via apheresis after CD34+ + Isolat atio ion, conce centrat atio ion and Cells returned to same patient by cell d drug ug-in induced mobil iliz izat atio ion inject ctio ion; site indic icat atio ion dependent formulat atio ion of CD34+/CX CXCR CR4+ cells Day 1 1 Day 2 2 Day 3 3 or 4 4 or or Shipme ment Shipme ment Drug induced mobilization eliminates need for surgical bone marrow aspiration No genetic manipulation or ex vivo expansion of cells Four days or less from donation to treatment Cost-of-goods an order of magnitude less expensive than CAR-T therapies 10
Caladri rius’ CD34 technology has robust intellectual property Fundamental l pro rotection to 2031+ 27 9 Foreig ign p patents U.S. p patent nts grant nted grant nted Key Cl Claim aims Pharmaceutical composition of non-expanded CD34+/CXCR4+ stem cells Therapeutic concentration range Stabilizing serum Repair of injury caused by vascular insufficiency 11
Caladri rius’ innovative CD34+ cell therapy pipeline* Commercialization Development Stage Product Indication Target 1 COV OVID-19 19 CLB CLBS119 Phase 2 2 (USA; start target 3Q20) TBD BD Lung ung D Dama mage CLBS12 CLB CLI CLI Regis istrat atio ion elig igib ible t trial ial (Japan; ongoing) 4Q202 2021 CLB CLBS16 CM CMD Phase 2 2b (USA; start target 2H 2020) TBD BD CLB CLBS14 NORD RDA Phase 3 3 confirmato tory (USA; initiation pending funding) TBD BD *Products are distinct and not interchangeable CLI LI = Critical Limb Ischemia CMD MD = Coronary Microvascular Dysfunction 1 Timing subject to COVID-19 pandemic influence NORDA DA = No Option Refractory Disabling Angina 12
CLBS11 119 COV OVID-19 Induced L Lung g Damage ge (USA)
Indication: COVID-19 induced lung damage Human coronavirus disease-19 (COVID-19) is caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) 2,3 that severe lung damage due to COVID-19 leads to long term Evidence suggests 1, 1,2, disability and possibly death as a result of inflammation and vascular damage Early data shows that almost all patients who survive COVID-19 pneumonia have some level of lung damage visible in CT scans taken before hospital discharge 4 Prior data from the SARS epidemic suggest that CD34+ cells in the lung could be a target of SARS-CoV-2 infection and that destruction of lung CD34+ progenitors could account for the inability of patients with severe lung damage to recover fully 5 1 Varga Z, et al., Lancet . 2020;395(10234):1417 ‐ 1418 4 Yuhui Wang, et al, Radiology , March 19, 2020 2 Lo., et al , Am J Respir Cell Mol Biol , 2017. 57(6): p. 651-661 5 Chen Y, et al. J Exp Med . 2007;204(11):2529 ‐ 2536. 14 3 Abd-Allah SH., et al, Cytotherapy , 2015. 17(4): p. 443-453
Our so r solu luti tion: CLBS119 The FDA has authorized Caladrius’ IND for the study of CLBS119, a CD34+ cell therapy for the repair of COVID-19 induced lung damage CLBS119 will be studied in a Phase 2 open-label proof-of-concept clinical trial to evaluate the safety and efficacy of autologous peripheral blood derived CD34+ cells delivered by intravenous infusion Efficacy endpoints include clinical measures such as biomarkers of inflammation, pulmonary function testing and resolution of lung infiltrates Safety endpoints include examination of adverse events, laboratory investigations, physical examinations, vital signs and occurrence of death Study initiation target: 3Q20 15
CLBS BS12 12 Critica cal L Limb b Ischemia (Japan) SAKIGAKE designated – Japan Advanced Therapeutic Medicinal Product (ATMP) designated - EU
Recommend
More recommend