Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (May 2020)
41 slides from Capricor Therapeutics's investor presentation, May 28, 2020
Slide 1: Capricor Therapeutics Corporate & Investor Presentation, May 2020
eapricor herapeutics” a & Investor Presentation lay 2020 0 NASDAQ: CAPR _— y
Slide 2: Forward-Looking Statements
* Pea ricor Forward-Looking Statements Prtss Statements in this presentation regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trial
Slide 3: Corporate Summary
Corporate Summary &capricor * Cell and exosome-based platform therapeutics company F a NASDAQ + Two products with novel approaches to neuromuscular, infectious, CAPR inflammatory and cardiovascular diseases Proforma Cash (3/31/2 $26.3 million Common sh Targeted Indications 14.3 million CAP-1002 — Du
Slide 4: Capricor's Product Pipeline
Capricor’s Product Pipeline Target Development Phase Candidate Indications Discovery Preclinical Phase! Phase ll _Phase Il Status “Positive Phase Il reported cap-102 Duchenne Muscular ee en allogeneic CDCS 0 -Requeste se es : oeronhy Meeting with FDA CAP-1002 (allogense COC) comp-19 —_— Expanded Acc
Slide 5: Capricor's CAP-1002 Technology
®capricor Saae Capricor’s CAP-1002 Technology CAP-1002 is a biologic consisting of allogeneic cardiosphere-derived cells (CDCs) Manufactured from donated heart muscle Does not act by “stemness” - the cells do not engraft into host tissue MOA: cells secrete exosomes: — Contain miRNAs, non-coding RNAs
Slide 6: Overview of Exosome Technology
Overview of Exosome Technology "capricor — Lipid-bilayer nanoparticles produced by most cells for inter-cellular communication Exosome Release — Contain small fragment nucleic acids and proteins for cellular signaling — Contents readily enter cytoplasm and nucleus — Targeted to specific receptors —
Slide 7: The Field of Exosomes: Increasing Market Opportunity
The Field of Exosomes: Increasing Market Opportunity Sapsicer (Over $375M raised to date in several private ‘companies to date?
Slide 8: Capricor's Multi-Target Approach to COVID-19
Capricor’s Multi-Target Approach to COVID-19 neapaley y COVID-19 Patients treatment using CAP-1002 FDA-granted expanded access study 2 Exosome Vaccine Strategy ad —viP —= Nucleic acid
Slide 9: CAP-1002 Targets Severe Cases of COVID-19
CAP-1002 Targets Severe Cases of COVID-19 "C2pricer Stage Il Stage Il Stage! (Early Infection) = Host inflammatory respo! Severity of lliness Mild constitutional Cad Fever 999.6°F Symptoms ere 1-6, D-dimer, NT-proBNP elevation 1 Low-normal procalctonin
Slide 10: Immunomodulatory Effects of CAP-1002
Immunomodulatory Effects of CAP-1002 Ecapricor CDs: Cardiosphere-derived cells Cardiac inflammation Lung i @ } (Systemic ion | IFN-y, TNFa, IL-1, Multiorgan dysfunction IL-6, IL-8, CXCL10, CCL2, CCL3, CCLS death Cardiac dysfunction Skeletal muscle injury Tissue Fibrosis i, 2 3. 4 5. 6 oe tay precede
Slide 11: CAP-1002: COVID-19 Preliminary Results
CAP-1002: COVID-19 Preliminary Results Si ical COVID-19 patients with ARDS* — Treated at Cedars-Sinai Medical Center in Los Angeles, CA — 5 males and 1 female — Age range: 19 to 75 — 5 of 6 on mechanical ventilation prior to treatment — Received intravenous infusions of 150 million cells of CAP-1002
Slide 12: CAP-1002: COVID-19 Development Plan
CAP-1002: COVID-19 Development Plan “C2Pticer Ongoing: FDA app’ | Jed access p' I to treat up to 20 COVID-19 patients (April 29, 2020) — Emergency use in critical patients — Amendment to expand to randomized, double-blind placebo-controlled Phase II study to treat up to 40 patients under review — Pu
Slide 13: Exosome Platform for COVID-19
Exosome Platform for COVID-19 VLP and mRNA Exosome-based Vaccines
Slide 14: Exosomes Target Cargo Directly to the Cell
Exosomes Target Cargo Directly to the Cell “Capricor —Exosomes serve as a natural signaling system —CD-9, CD-81 and CD-63 are surface markers of exosomes and can serve as targeting molecules for effective delivery to cells and confirmation of exosome loading
Slide 15: Coronavirus Structure
Coronavirus Structure Unique Protein Visualization €capricor 4 antigens likely to yield increased protective response OB ID: S108 — Other mRNA vaccines in development are targeting the spike “S” protein solely — Capricor’s exosome platform vaccine addresses all 4 proteins: E,M,N,S (1) VLP & (2) mRNA
Slide 16: Capricor's Exosome-Based Vaccine Approach is Distinctive
Capricor’s Exosome-Based Vaccine Beaori a ae: Capricor Approach is Distinctive Exosome-based approach — Allows for specificity in cellular targeting — Innovative technology - yet is well characterized Unique 4-antigen approach — Potentially confers greater immunity by targeting all 4 antigens ii sup
Slide 17: Exosome VLP: Display Vaccine Approach
(1) Exosome VLP: Display Vaccine Approach* Pricer 1.reate vectors that '3.Grow the cells which produce 4. Purify the VLPs, which carry _§. Immunize with purified ‘express the 4 ‘SARSCOV2 VLPs 2. Transfect Into 293 cals, theS,N,M,andE proteinsin VLPs, inducing protective ‘SARS.COV2 structural native,
Slide 18: Exosome mRNA Vaccine
(2) Exosome mRNA Vaccine "capricor “Sdetwal 20 Seana aymeaze 3 combine aint wiewst Abi) “Sere eyopime "taing oan formulation og) on vo et somal Able reagents BESS <0) Ab esponae Exosome-based mRNA vaccine can have the following potential benefits: — Delivering payload directly to cytoplasm or nucleu
Slide 19: Vaccine Development Planned Timeline
Vaccine Development Planned Timeline Ecapricor SARS-CoV-2 Vaccines — Validation of multi-protein approach (4 viral proteins (S, M, E, N)) - underway — Generate exosome based VLP-based vaccine and mRNA vaccine — underway IND/Clinical Validation — Preclinical testing - underway — Preclinical testing i
Slide 20: CAP-1002 Duchenne Muscular Dystrophy Program
CAP-1002 Duchenne Muscular Dystrophy Program
Slide 21: Lack of Dystrophin Predisposes Muscle to Damage
Lack of Dystrophin Predisposes capri Muscle to Damage Capricor Dystrophin is a structural protein located within the muscle fiber membrane Whole Muscle + Acts both as a cushion and a kind of glue Tissue * Without dystrophin, muscles are unable to function properly, suffer progressive damage and Yase
Slide 22: Mechanism of Action
Mechanism of Action: Peaori Defined in “Stem Cell Reports” apricor * phospho-Akt ‘A Nif2 (cytoplasmic) h A Nrf2 (nuclear) A HO-1 —— #GCLC cat. sub. | Aeatalase A SOD-2 ¥ phospho- kB ‘¥ CD6B+ macrophages ¥ p65 (nuclear) h vcr ¥.cD3+ Tells ‘# mitochondrial DNA copy number | ry RESTORED mitochondrial u
Slide 23: Trajectory of CDCs in DMD (Preclinical Data)
Trajectory of CDCs in DMD (Preclinical Data) Pricer \ . CDCs to treat ie Q + Left ventricular ejection fraction markedly improved vs. control — P<0.05 at all timepoints through 12 weeks of follow-up* + Exercise performance approximately doubled vs. control \ + Hypothesis: CDCs to improve exercise ca
Slide 24: Capricor's Addressable DMD Population
Pcapricor Capricor’s Addressable DMD Population 0 Years 5 10 15 20 25 30 : |_Hop, Run, Jump, Rise . Climb Loss of Stair Descend CAPRICOR's A bese ot Targeted Patient mbulation Population Ventilation
Slide 25: Competitive Landscape for DMD
Competitive Landscape for DMD "capricor a = allenges CAP-1002 Bene SE 83 Wy Exon Skipping — treats a small D portion of the DMI Bron Sep lag) Gene therapy — potential safety Anti-fibrotic Gene therapy risks P ; ro-regenerative | NF-kB inhibition may not be NF-kB enough Cellular Energy Steroids ; . S
Slide 26: Primary Efficacy Endpoint
Primary Efficacy Endpoint Ycapricor Performance of the Upper Limb (PUL: v1.2) to Assess Skeletal Muscle 4items Middle Level Shoulder Level Teg pper_ |_Sarah extremity weigh suid Above shoulder height Hand to mouth w/w/o weights Handto table | [ Stacklight and heavy cans items Distal Level ‘Moving we
Slide 27: Capricor's Regulatory Designations - DMD
Capricor’s Regulatory Designations - DMD GOAL OF FDA'S RMAT DESIGNATION To facilitate efficient development and expedite review of a drug Similar to breakthrough therapy designation: + RMAT provides benefits that include more frequent meetings with FDA to discuss the development plan for the product
Slide 28: HOPE-Duchenne Focused on Older DMD Patients
HOPE-Duchenne Focused on Older DMD Patients + Phase I/II study: 25 patients, randomized and open-label One-time, multi-vessel, intracoronary delivery of cells HOPE population were all on stable corticosteroids Very limited options for this patient population RESULTS + Reduction in cardiac scar at 6
Slide 29: HOPE-Duchenne: Reduced Cardiac Scar and Improved PUL
HOPE-Duchenne: Papi Reduced Cardiac Scar and Improved PUL Sapsicer Midedistal PUL Scar size HB Usual caro (N= 14) EE CAP-1002 (N= 13)
Slide 30: HOPE-2 Phase II Clinical Study
HOPE-2 Phase II Clinical Study
Slide 31: HOPE-2 Clinical Trial
HOPE-2 Clinical Trial Scapricor + Design: Phase Il, randomized, double-blind, placebo-controlled trial in participants with DMD and reduced skeletal muscle function Objective: Evaluate safety and efficacy of CAP-1002 + Dosing Regimen: 150M cells delivered intravenously every 3 months Sites: 9 sites
Slide 32: Clinically Meaningful Changes Observed in PUL 2.0
Clinically Meaningful Changes Observed in PUL 2.0 €e . (Shoulder + Mid + Distal) apricor = MONTH 12/ET = MONTH 12/ET i ® 9 ma cap-1002 = * ¥ mm PLACEBO g 2 . ° & £4 3 3 3 £ improvement = ve ——— & :* fe " ar 2 = H 3b p=0,0532 = st ea g° & a = ( ‘02.4 points in CAP-1002 vs. placebo at 12-months ]
Slide 33: Clinically Meaningful Changes in Mid-Level PUL 1.2
Clinically Meaningful Changes in Mid-Level PUL 1.2 Similar changes shown in HOPE-Duchenne Mean Change from Baseline +/- SEM MONTH 12/ET 0. Wm CAP-1002 mi PLACEBO -2: 6 p=0.0818 8: Mean Change from Baseline +/- SEM ( ‘28 point difference in CAP-1002 vs. placebo at 12-months } 3 Pcapricor MONTH 12/ET
Slide 34: Improvements in LV Ejection Fraction Observed
Improvements in LV Ejection Fraction (%) Observed Scapricor (Potential for long-term preservation of cardiac function | s MONTH 12/ET a mm CAP-1002 $ = PLACEBO 2 5 0 7 % a 5-4 improvement £ S © -2 S & p=0.0042 § -3 8 =
Slide 35: Improvements in LV End-Systolic and End-Diastolic Volume
Improvements in LV End-Systolic Volume Pann & LV End-Diastolic Volume Observed Capricor LV ES Volume, Indexed, ml/m? LV ED Volume, Indexed, ml/m? = MONTH 12/ET = MONTH 12/ET a mm CAP-1002 B 5. mm CAP-1002 + ‘ ml PLACEBO ? ml PLACEBO ° 2 = £ a @ 3 2 3 5 E 0 5 6 & rs 3 3 2 improvement g -10 5 6 p=0.06
Slide 36: Improvements in Creatine Kinase MB / Total Observed
Improvements in Creatine Kinase MB / Total Ree sricas Creatine Kinase (%) Observed R (enzyme associated with breakdown of cardiac muscle cells | 4 ‘ p=0.006 wm CAP-1002 mm Placebo m 2: = wa oO Ro A -2: Month 12 Visit
Slide 37: HOPE-2 Safety Results
HOPE-2 Safety Results "capricor * A total of 69 infusions (CAP-1002 or placebo) were performed in HOPE-2 * Generally safe and well tolerated throughout the study * With the exception of hypersensitivity reactions, no safety signals were identified
Slide 38: Conclusions and Future Directions
Conclusions and Future Directions Scapricor Conclusions: Moving Forwar — First placebo-controlled trial — Requested End-of Phase 2 Meeting with showing upper limb functional FDA to discuss next steps and pathway improvements in non-ambulant to approval DMD patients — Engaged global CMO for scale-up
Slide 39: World-Class DMD Advisory Board
World-Class DMD Advisory Board Seapricor Craig McDonald, M.D. (National Pl) University of California at Davis (USA) Michelle Eagle, Ph.D, M.Sc, MCSP. Atom International Ltd (UK) Richard Finkel, M.D. Nemours Children's Hospital (USA) Pat Furlong Parent Project Muscular Dystrophy (USA) Kan Hor, M.D. N
Slide 40: Recent and Targeted Milestones in 2020
Recent and Targeted Milestones in 2020 Scapricor Gell Therapy (CAP-1002) Technology Feb. 2019: HOPE-Duchenne (Phase |/I}) study published in Journal of Neurology Oct. 2019: Reported positive six-month results of HOPE-2 trial at World Muscle Society Intemational Conference (Oct. 2019: Met with FDA to
Slide 41: Senior Leadership Team
Senior Leadership Team a Linda Marbin, Ph.D. Chief Executive Officer, Co-founder and Director Ds Marin has ove 25 years of experience Inthe biotechnology 1+ Been th Caprio sine 205 and CEO since 2010, + Drearbin eared a PRD om Case Wester Reserve Univesity incardae physlogy Karen Krasney, 1D. Execut
Capricor Therapeutics — Investor Presentation (May 2020) on 6ix