Capricor, Inc. KOL Lunch on DMD Key Opinion Leaders Lunch to discuss the emerging clinical paradigm in DMD focusing on gene and cell therapy.

Capricor, Inc. KOL Lunch on DMD Key Opinion Leaders Lunch to discuss the emerging clinical paradigm in DMD focusing on gene and cell therapy. March 9, 2018 NASDAQ: CAPR

Capricor , Inc. KOL Lunch on DMD Forward - Looking Statements 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 trials ; plans regarding regulatory filings, future research and clinical trials ; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market ; plans regarding current and future collaborative activities and the ownership of commercial rights ; scope, duration, validity and enforceability of intellectual property rights ; future royalty streams, expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings, and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward - looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 . Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward - looking statements . There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward - looking statements . More information about these and other risks that may impact Capricor's business is set forth in Capricor's Annual Report on Form 10 - K for the year ended December 31 , 2016 as filed with the Securities and Exchange Commission on March 16 , 2017 , in its Registration Statement on Form S - 3 , as filed with the Securities and Exchange Commission on September 28 , 2015 , together with the prospectus included therein and prospectus supplements thereto, and in its Quarterly Report on Form 10 - Q for the quarter ended September 30 , 2017 , as filed with the Securities and Exchange Commission on November 14 , 2017 . All forward - looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward - looking statements . 2

Capricor, Inc. KOL Lunch on DMD Linda Marban, Ph.D. - Chief Executive Officer, Capricor Therapeutics, Inc. Deborah Ascheim, M.D. - Chief Medical Officer, Capricor Therapeutics, Inc. Craig McDonald, M.D., is professor and chair of the Department of Physical Medicine and Rehabilitation and Director of the Neuromuscular Disease Clinics at the University of California, Davis. Dr. McDonald is an internationally recognized expert in the clinical management and rehabilitation of neuromuscular diseases including DMD. He is the national PI of the Capricor HOPE - 2 Trial. Jeffrey Chamberlain, Ph.D., is professor in the Departments of Neurology, Medicine and Biochemistry and director of the Seattle Wellstone Muscular Dystrophy Center. Dr. Chamberlain is the holder of many of the key patents for microdystrophin and has been a world leader in the development of gene therapies for DMD. Michelle Eagle, Ph.D., the managing director of ATOM International LTD, is one of the creators of, and has published extensively on, the Performance of the Upper Limb (PUL) test, a validated test for skeletal muscle function in Duchenne muscular dystrophy. Pat Furlong, is the founding president and CEO of the Parent Project Muscular Dystrophy (PPMD), the largest non - profit organization in the U.S. focused solely on Duchenne. She has spearheaded the advocacy movement in Duchenne and is a world expert on regulatory strategies for approval of products to treat the disease. Capricor KOL Speakers 3

Capricor , Inc. KOL Lunch on DMD Capricor's Mission Capricor is focused on the discovery, development and commercialization of innovative cell and exosome based therapies for patients with immune - inflammatory rare diseases with a focus on Duchenne muscular dystrophy . 4

Capricor , Inc. KOL Lunch on DMD Treatment Options for DMD are Limited Challenges EXONDYS 51 - 13% of DMD population Gene therapy not always effective due to antibody response Steroids have adverse long term side - effects NF - kB inhibition may not be enough to mediate immune response Exon Skipping Gene therapy Utrophin NF - kB Steroids Immunomodulator y Anti - fibrotic Pro - angiogenic Pro - regenerative Cellular Energy We believe CAP - 1002 may be used synergistically with other therapeutics aimed to treat the underlying genetic mutation with DMD 5

Capricor , Inc. KOL Lunch on DMD 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 EVs (exosomes) Contain non - coding RNAs and proteins Internalized by target cells Stimulate diverse and lasting changes in cellular behavior CAP - 1002 has been investigated in several clinical trials and more than 130 human subjects Capricor's CAP - 1002 Technology 6

Capricor , Inc. KOL Lunch on DMD Physiological Effects of CDCs in mdx Model RESTORED mitochondrial ultrastructure NORMALIZED deficient respiratory capacity of isolated mitochondria Oxidative Stress phospho - Akt Nrf2 (cytoplasmic) Nrf2 (nuclear) HO - 1 catalase SOD - 2 GCLC cat. sub. Inflammation phospho - IkB p65 (nuclear) MCP1 CD68+ macrophages CD3+ T cells NF - kB Fibrosis collagen I collagen III Cellular Energy m itochondrial DNA copy number level of respiratory chain subunits Muscle Cell Generation Ki67 + cardiomyocytes Aurora B cardiomyocytes *CDCs have been the subject of >100 peer - reviewed papers since 2007 Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). 7

Intramyocardial CDC methods Mouse model of DMD - 10 m/o mdx mice (n=12 per group) - Randomized to CDC vs vehicle - Followed for 6 months or longer - Single or repeat injection at 3 mo as noted CDCs - From background mouse strain (C57BL/10) - Injected intramyocardially (LV; 4 injection sites) Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). 8 Capricor , Inc. KOL Lunch on DMD

Improved cardiac function Increased exercise capacity Increased survival rate Improved function & survival in mouse model Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). 9

Capricor , Inc. KOL Lunch on DMD Physiological Effects of CDCs in mdx Model Oxidative Stress phospho - Akt Nrf2 (cytoplasmic) Nrf2 (nuclear) HO - 1 catalase SOD - 2 GCLC cat. sub. Inflammation phospho - IkB p65 (nuclear) MCP1 CD68+ macrophages CD3+ T cells NF - kB Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). 10

Oxidative stress & inflammation: major players in DMD Inflammatory cell infiltration Oxidative stress Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). 11

Capricor , Inc. KOL Lunch on DMD Physiological Effects of CDCs in mdx Model Oxidative Stress phospho - Akt Nrf2 (cytoplasmic) Nrf2 (nuclear) HO - 1 catalase SOD - 2 GCLC cat. sub. Inflammation phospho - IkB p65 (nuclear) MCP1 CD68+ macrophages CD3+ T cells NF - kB Fibrosis collagen I collagen III *CDCs have been the subject of >100 peer - reviewed papers since 2007 Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). 12

Mdx+Vehicle CTL(WT) Mdx+CDC Reduced cardiac collagen content and fibrosis 0.4 0.45 0.5 0.55 0.6 0.65 0.7 CTL Mdx+Vehicle Mdx+CDC CTL(WT) CDC Vehicle 0.3 0.33 0.36 0.39 0.42 0.45 0.48 0.51 CTL Mdx+Vehicle Mdx+CDC Collagen III A1 CTL(WT) CDC Vehicle Collagen III A1 Collagen I A1 Collagen I A1 Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). 13 Capricor , Inc. KOL Lunch on DMD

Capricor , Inc. KOL Lunch on DMD Physiological Effects of CDCs in mdx Model RESTORED mitochondrial ultrastructure NORMALIZED deficient respiratory capacity of isolated mitochondria Oxidative Stress phospho - Akt Nrf2 (cytoplasmic) Nrf2 (nuclear) HO - 1 catalase SOD - 2 GCLC cat. sub. Inflammation phospho - IkB p65 (nuclear) MCP1 CD68+ macrophages CD3+ T cells NF - kB Fibrosis collagen I collagen III Cellular Energy m itochondrial DNA copy number level of respiratory chain subunits *CDCs have been the subject of >100 peer - reviewed papers since 2007 Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). 14

CTL(WT) Mdx+Vehicle Mdx+CDC Restoration of mitochondrial integrity Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). 15 Capricor , Inc. KOL Lunch on DMD

Capricor , Inc. KOL Lunch on DMD Physiological Effects of CDCs in mdx Model RESTORED mitochondrial ultrastructure NORMALIZED deficient respiratory capacity of isolated mitochondria Oxidative Stress phospho - Akt Nrf2 (cytoplasmic) Nrf2 (nuclear) HO - 1 catalase SOD - 2 GCLC cat. sub. Inflammation phospho - IkB p65 (nuclear) MCP1 CD68+ macrophages CD3+ T cells NF - kB Fibrosis collagen I collagen III Cellular Energy m itochondrial DNA copy number level of respiratory chain subunits Muscle Cell Generation Ki67 + cardiomyocytes Aurora B cardiomyocytes *CDCs have been the subject of >100 peer - reviewed papers since 2007 Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). 16

0 0.001 0.002 0.003 0.004 0.005 0.006 0.007 CTL Mdx+Vehicle Mdx+CDC 0 0.001 0.002 0.003 0.004 0.005 0.006 0.007 CTL Mdx+Vehicle Mdx+CDC WGA Aurora B DAPI WGA Ki67 DAPI Mdx+Vehicle Mdx+CDC CTL(WT) Ki67+ Cardiomyocytes/ Cardiomyocytes in HPF Aurora B+ Cardiomyocytes/ Cardiomyocytes in HPF Cardiomyogenesis Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). 17 Capricor , Inc. KOL Lunch on DMD

20 25 30 35 40 45 50 1 15 30 LVEF (%) Days (after MI) CDC-DMSO CDC-GW4869 * CDC exosomes EF Ibrahim et al., Stem Cell Reports 2014 Exosomes: CDCs MoA Defined 30 - 150 nm particles Present in all body fluids Released by nearly all cell types Loaded with miRs and other bioactive contents Payload very cell - specific Blocking exosome biosynthesis abrogates CDC benefit 20 25 30 35 40 45 50 1 15 30 LVEF (%) Days (after MI) Vehicle CDC-exo Fibroblast-exo * ** 18 Capricor , Inc. KOL Lunch on DMD

Collagen content Cardiomyogenesis Intramyocardial exosomes recap effects of CDCs Functional benefit on heart Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 19 Capricor , Inc. KOL Lunch on DMD

Intravenous CDC methods Mouse model of DMD - 10 - 12 m/o mdx mice - Randomized to CDC vs vehicle - Followed for 3 - 26 weeks - EF, exercise capacity, isolated muscle function CDCs - From background mouse strain (C57BL/10) - 250k cells infused via jugular vein Adapted from Cedars - Sinai Medical Center unpublished data 20 Capricor , Inc. KOL Lunch on DMD

Capricor , Inc. KOL Lunch on DMD Biodistribution and Clearance of IV CAP - 1002 Biodistribution Clearance Mouse model : WT mice CDCs : Human, 150K Route : Jugular vein Mouse model : SCID mice CDCs : Human, 150K Route : Jugular vein Capricor unpublished data 21

Cardiac, skeletal, and isolated muscle function after IV CDCs V e h i c l e C D C 3 w k C D C 6 w k 0 40 50 60 70 E j e c t i o n F r a c t i o n ( % ) Pre-Treatment Post-Treatment * * V e h i c l e C D C 3 w k C D C 6 w k 0 200 250 300 350 400 450 500 E x e r c i s e C a p a c i t y ( m ) * Post-Treatment Pre-Treatment W i l d T y p e V e h i c l e C D C 3 w k C D C 6 w k 0 10 20 30 40 T w i t c h F o r c e ( m N ) * * CDC Vehicle More fusion - competent myoblasts in soleus after IV CDCs Adapted from Cedars - Sinai Medical Center unpublished data 22

Capricor , Inc. KOL Lunch on DMD Phase I / II HOPE - Duchenne Clinical Trial CAP - 1002 (1 x 75M cells) Usual Care n=12 25 patients Ages 12 and older DMD heart disease EF > 35% Stable steroids n=13 6 CAP - 1002 given by intracoronary infusion 12 data presented at AHA 2017 late - breaking session One - time, multi - vessel, intracoronary delivery of cells Safety trial with multiple exploratory efficacy endpoints Three U.S. sites: Cedars - Sinai Medical Center Cincinnati Children's University of Florida https://clinicaltrials.gov/ct2/show/NCT02485938 23

Capricor , Inc. KOL Lunch on DMD Baseline Characteristics Usual Care (n=12) CAP - 1002 (n=13) Age, median years (range) 17.5 (12 - 20) 18 (14 - 25) Wheelchair Use Always (%) 7 (58) 10 (77) Cardiac Scar Size, mean % (SD) 21.4 (10.8) 17.6 (6.8) LV Ejection Fraction, mean % (SD) 48.4 (7.5) 49.6 (6.7) Intracoronary Dose, M cells (SD) n/a 73.7 (3.6) Patients were all male, were all receiving chronic treatment with systemic steroids, and were mostly Caucasian. - HOPE population characterized by advanced disease; majority were non - ambulant - Most DMD clinical development has been conducted in less sick patients 24

Capricor , Inc. KOL Lunch on DMD Heart Muscle: Reduced Myocardial Scarring Assessed by cardiac MRI with blinded analysis by core lab Scar increased in the Usual Care group, but decreased in the CAP - 1002 group 11.9% group difference in change score at Month 12 (p=0.03) Decreased scar is counter to the natural history of DMD p=0.09 p=0.03 Month 6 Month 12 IMPROVEMENT 25

Capricor , Inc. KOL Lunch on DMD Heart Muscle: Increased Regional Systolic Wall Thickening Measurement of cardiac function by MRI allows focus on treated areas Magnitude of scar reduction is consistent with natural history of scar progression in DMD Inferior Anterior Lateral Septal Measure is important indicator of overall cardiac function in DMD p=0.04 p=0.09 INFERIOR WALL ANTERIOR WALL Month 6 Month 12 p=0.10 p=0.54 IMPROVEMENT 26

Capricor , Inc. KOL Lunch on DMD Skeletal Muscle: PUL Results Indicate Functional Benefit Performance of the Upper Limb (PUL) test is a validated instrument in DMD Relates to patients' ability to perform common activities of daily living Trends towards improvement observed throughout follow - up Middle + Distal PUL Score % change from baseline IMPROVEMENT 27

Capricor , Inc. KOL Lunch on DMD - Potential registration trial plan to initiate in 1Q 2018 Randomized, double - blind, placebo - controlled Target enrollment of 84 patients with advanced disease Peripheral intravenous delivery - supported by preclinical studies Repeat - dose design - potential to achieve sustained benefit Primary efficacy endpoint - difference in change in mid - PUL scores at Month 12 Principal Investigator - Craig M. McDonald, M.D. - FDA willing to consider PUL as an efficacy endpoint for registration Type B meeting held in June, following six - month HOPE data - Granted RMAT designation HOPE - 2 Clinical Trial of CAP - 1002 in DMD https://www.clinicaltrials.gov/ct2/show/study/NCT03406780 28

Emerging genetic therapies for DMD Jeffrey S. Chamberlain, Ph.D. McCaw Endowed Chair in Muscular Dystrophy Director, Sen. Paul D. Wellstone Muscular Dystrophy Research Center Depts. of Neurology, Medicine and Biochemistry University of Washington Seattle, WA USA Disclosures: JSC is a member of the scientific advisory boards of Solid Biosciences, Ballard Biologics, AAVogen and Akashi Therapeutics | Capricor, Inc. KOL Lunch on DMD 29

Gene Therapy - different types Gene replacement therapy: deliver a new version of a gene to the target tissue (aka gene addition) Gene editing: directly modify a gene to fix or bypass a mutation - CRISPR/Cas9 Gene knockdown: Suppress the activity of a mutant gene - Inhibitory RNA (siRNA, shRNA, etc ) Transcript modification, e.g. exon skipping' Rationale bolstered by multiple recent successes with human gene therapy: X - SCID, Hemophilia b, Liebers congenital amaurosis , Lipoprotein lipase deficiency (Glybera), Metachromatic Leukodystrophy , Wiskott - Aldrich Syndrome Spinal muscular atrophy CAR - T cell modification Muscular dystrophy . | Capricor, Inc. | KOL Lunch on DMD 30

Why gene therapy? Fix the primary cause of genetic disorders Potential for a permanent treatment Genetic manipulation of stem cells Overcome genetic errors that lead to cancer Powerful approach to developing new vaccines | Capricor, Inc. KOL Lunch on DMD 31

Delivering gene therapy to the body Viral' Vectors: modified from viruses, no longer "virus" - Adeno - associated viral vectors (AAV) - best for non - dividing cells - Lentiviral vectors - best for dividing cells (e.g. stem cells) - Adenoviral vectors - mostly now used for vaccines, cancer Non - viral vectors: plasmids, anti - sense oligonucleotides; "nanoparticles" - Short acting , easier to manufacture, high safety - Transcript modification, gene editing Ex vivo gene therapy: Corrected, or normal, Stem Cells - Bring along therapeutic gene(s) or trophic factors | Capricor, Inc. KOL Lunch on DMD 32

Duchenne muscular dystrophy (DMD) Most common form of muscular dystrophy (>300,000 cases) Caused by mutations in the 2.2 MB dystrophin gene X - linked, recessive inheritance 1/3 of cases due to new mutation; thousands of independent mutations | Capricor, Inc. KOL Lunch on DMD 33

Strategies for the development of DMD therapies l Viral vector delivery of genes to muscles Dystrophin replacement Delivery of a surrogate gene ( e.g. Utrophin; GALGT2 ) Transcript modification - bypass mutation Gene editing ( e.g . CRISPR/Cas9) l Antisense oligonucleotide delivery (exon skipping) l Stem cell transplantation ( ex vivo gene therapy) l Pharmacologic therapy - palliative - steroid hormones (prednisone); induce muscle hypertrophy (myostatin inhibitors); reduce inflammation/fibrosis (TGF - b inhibitors), enhance regeneration, stop codon suppression (aminoglycosides) l Combinatorial - future | Capricor, Inc. KOL Lunch on DMD 34

Gene Therapy for DMD/BMD Goal: Develop methods to replace or repair dystrophin gene Pros: - Fixes the primary cause of the disease - lack of dystrophin - Should work for ALL patients Cons: - Requires bodywide (systemic) gene delivery to muscle AAV vectors ? Potential: - DMD (severe) - no dystrophin protein made - BMD (mild) - smaller dystrophins made | Capricor, Inc. KOL Lunch on DMD 35

Challenges for gene therapy for DMD/BMD How can you deliver a new gene to muscles, bodywide , in a safe and effective manner? - Development of delivery VECTORS by manipulating viruses - Remove viral genes, replace with gene of interest ( e.g. dystrophin) Vectors derived from adeno - associated virus are promising - Some serotypes enable systemic gene delivery via the vasculature - AAV vectors have limited carrying capacity; dystrophin is huge gene - Produce smaller, synthetic versions of dystrophin that still work Future/alternate goal: can the mutant gene be edited or repaired? - Gene editing with AAV - CRISPR/Cas9 | Capricor, Inc. KOL Lunch on DMD 36

Assembly of the dystrophin - complex by micro - Dys 14 kb cDNA 3.7 kb cDNA | Capricor, Inc. KOL Lunch on DMD 37

Adeno - associated viral (AAV) vectors Non - integrating vectors, persist as episomes PROS : - Numerous serotypes, many target muscle (AAV6, 8 & 9) - Relatively easy to produce; scalable to bioreactor production - Can be used for bodywide gene delivery , especially to muscles CONS : - Small carrying capacity (~5 kb; Dys cDNA=14 kb) - Generally poor results in stem cells - Rapidly lost from dividing cells ITR Promoter Transgene PolyA + ITR AAV vector: | Capricor, Inc. KOL Lunch on DMD 38

Systemic gene transfer to muscle using AAV6 Single tail vein injection of AAV/alkaline phosphatase into adult mice Gregorevic & Chamberlain, Nature Medicine, 2004 Whole mounts stain - 2 mos pi Histochemical stain - 2 years pi AAV holds 5 kb; dystrophin gene = 2.2 MB 39

Development of micro - Dystrophins "mini" - Dystrophins modified from a very mildly affected BMD patient "Micro - dys" made by systematically removing additional protein domains Many micro - dystrophins made by my lab and some others "H2 - microDys" and "micro - Dys5" entering clinical trials | Capricor, Inc. KOL Lunch on DMD 40

wildtype dko Tdko Forelimb Masseter Heart Diaphragm Quadriceps Tibialis Ant. Gastroc. Expression of micro - Dystrophin one year after AAV infusion into dystrophic mice (IV) Treated gastrocnemius dystrophin Gregorevic, Nat Med, 2006 41

MicroDys stably expressed 2 years after AAV infusion in mdx mice - diaphragm muscles 42

GRMD Biceps Brachii 1E13 vg/kg 2E14 vg/kg Microdystrophin - Sarcoglycan Merge 1E14 vg/kg DGC expression in GRMD dogs following systemic SGT - 001 infusion Kornegay et al | Capricor, Inc. KOL Lunch on DMD 43

SGT - 001 (AAV - Dys) increases force in GRMD dogs following systemic delivery Kornegay et al Analysis of torque produced by extension and flexion of the tibiotarsal joint. *p<0.05 44

AAV - mediated gene therapy for DMD/BMD Micro - dystrophins halt muscle wasting, protect from exercise - injury and improve strength Not all Dys are equivalent Efficient systemic delivery of AAV/ Dys in dogs No reduction in Dys expression after at least 2 years High dose AAV9 delivery has been safely achieved in humans (e.g. SMA - Nationwide Children's ) Clinical trials of AAV/ Dys are beginning by several groups | Capricor, Inc. KOL Lunch on DMD 45

Clinical trials planned / in progress Solid Biosciences - Ganot et al (Chamberlain uDys - 3 rd gen); AAV9 Nationwide Children's - J Mendell et al (Sarepta) (Chamberlain uDys - 1 st gen); AAV - rh74 Genethon - G Dickson (Sarepta) (Chamberlain uDys - 1 st gen); AAV8 Pfizer - Samulski et al (Xiao uDys ; Chamberlain/Hauschka promoter); AAV9 Tentative plans: Systemic (intravascular) delivery of AAV - uDys Solid Biosciences and Nationwide Children's are beginning trials Pfizer: Q2 - Q3 2018?? Genethon: 2019??? Results: Unclear on how long it will take to observe a functional benefit | Capricor, Inc. KOL Lunch on DMD 46

Gene therapy Difficulty of transitioning from academics to clinic Growing, large interest by Biotech and Venture Capitol communities American Society for Gene and Cell Therapy is the premier meeting Future: Gene editing with CRISPR/Cas9? Safety/efficacy? New vectors? Scaling production Combinatorial therapies | Capricor, Inc. KOL Lunch on DMD 47

Gene therapy options/alternatives? Delivery/upregulation of Utrophin (Summit) - AAV/ Utrn : Chamberlain Overexpression of GALGT2 (NCH trial imminent) - Modifies dystroglycan to assemble a Utrophin - complex Stem cells: e.g. Capricor - Modulate muscle environment/enhance regeneration - Provide new myogenic stem cells to replace muscle loss? Combinations : Gene therapy + Stem cell therapy? Add anti - fibrotics and/or anti - inflamatories ? Add enhancers of muscle mass | Capricor, Inc. KOL Lunch on DMD 48

Chamberlain Lab: Katrin Hollinger - 3 rd gen Dys Julian Ramos - 3 rd gen Dys Julie Crudele - Dys/immunology James Allen - AAV Christine Halbert - AAV Matt Karolak - DGC Maja Zavaljevski - DMD/FSHD/DM1 Darren Bisset - DMD/FSHD/DM1 Niclas Bengtsson - Cas9 John Hall - Sat cells/Cas9/FSHD Aisha Mushtaq - DMD/DM1 Andrea Arnett - AAV Glen Banks - Utrn / ringbinden Jane Seto - Dys/FKRP | Capricor, Inc. KOL Lunch on DMD U Washington: Guy Odom - Utrn Steve Hauschka - MCK Joel Chamberlain - FSHD Supported by The National Institutes of Health (NIAMS/NHLBI) The Muscular Dystrophy Association (USA) LGMD2I Fund; Solid Biosciences Wellstone Muscular Dystrophy Research Center - Seattle

OUTCOME MEASURES IN DMD CLINICAL AND TRIAL CONSIDERATIONS PRESENTED BY : MICHELLE EAGLE | Capricor, Inc. KOL Lunch on DMD

OUTCOME MEASURES IN DMD Tools/assessments used to: Evaluate the baseline status of a patient Evaluate the effect of an intervention M onitor disease progression Inform clinical decision making regarding initiation/change of therapies Clinic or clinical trial context 51 | Capricor, Inc. KOL Lunch on DMD

WHAT MAKES A GOOD OUTCOME MEASURE? Reliable Excellent inter - rater and intra - rater reliability Low measurement error Valid The degree to which an assessment measures what it intends to Sensitive Responsive in the ability to detect meaningful, real changes 52 | Capricor, Inc. KOL Lunch on DMD

QUALITIES OF GOOD OUTCOME MEASURES Meaningful Clinical meaningfulness Cost efficient Easy to administer Standardised 53 | Capricor, Inc. KOL Lunch on DMD

54 | PRESENTATION TITLE SECOND LINE PROBLEMS ASSESSMENT Functional Scales - North Star, PUL EK Scale timed tests - 10 metre, rise from floor, 6 MWT muscle strength - MMT, myometry , QMT Range of movement Respiratory - FVC, MIP/MEP, PCF Loss of function Weakness Contractures Immobility 54 | Capricor, Inc. KOL Lunch on DMD

CURRENT OUTCOME MEASURES USED IN DMD North Star Ambulatory Assessment PUL (Performance upper limb) Egen Klassification (EK) 9 Hole peg test 6MWT Timed functional tests Timed up and go Rise from floor Climb/descend stairs Run/walk 10 metres ACTIVE seated Pulmonary Function Tests FVC/PEF/PCF/MIP/MEP/SNIP Strength measures Manual Muscle Testing Quantitative Muscle Testing myometry Grip strength Patient reported OMs (PROM) QOL measures 55 | Capricor, Inc. KOL Lunch on DMD

PROGRESSION OF DISEASE OVER TIME 56 | Capricor, Inc. KOL Lunch on DMD

OM'S A single outcome is unlikely to cover the breadth of the disease from highly ambulant to wheelchair dependent with limited hand function Rates of change may vary for upper and lower limb function Careful balance between asking the impossible and the ultra easy Some OM have been developed that are specifically for ambulant and others for non - ambulant people, others are more general and can be used across all ages but tend to be less senstivie 57 | Capricor, Inc. KOL Lunch on DMD

PROGRESSIVE IMPROVEMENT IN UNDERSTANDING OM'S We understand more about the relationship between strength and function That the individual may not represent the population That OMs do not always correlate highly throughout the disease progression and that some OMs are more useful at different stages of disease Some OMs may be more relevant to measure function and others may be more sensitive to change and that the two are not necessarily the same but both may be required in a clinical trial That a tool box is required - with the right tool for the right job Don't put all your eggs in one basket! 58 | Capricor, Inc. KOL Lunch on DMD

The table shows the application of outcome measures relevant for DMD in relation to age and validation status ( ). To be validated (?). Lynn et al. Neuromuscul Dis 2014;25:96 - 105 DECISION FRAMEWORK FOR INCLUSION OF CLINICAL OUTCOME MEASURES IN PHASE II/III TRIALS 59

Fig. 2 Total PUL scores in ambulant (blue) and non ambulant (red) DMD patients plotted according to age. Pane et al. 2014 http://dx.doi.org/10.1016/j.nmd.2013.11.014 60 | Capricor, Inc. KOL Lunch on DMD

AMBULATORY ASSESSMENTS NSAA/ 6 MWT/ TIMED FUNCTION TESTS | Capricor, Inc. KOL Lunch on DMD

LOSS OF AMBULATORY FUNCTION OCCURS IN A PREDICTABLE WAY Unable to jump, hop and run Gowers' sign with standing Loss of standing from the floor Loss of lie to sit Loss of stair climbing Loss of ability to stand from a chair Loss of ability to walk independently (10 meter walk/run; 6MWD) Loss of standing in place North Star Ambulatory Assessment Developed taking into consideration all the functional milestones and activities that are clinically meaningful in DMD A 10 - point change in the linearised scale is clinically meaningful, e.g. 90 - 80 = can no longer hop 50 - 40 = inability to rise independently from the floor 21 - 11 = loss of ability to stand still and upright 6MWD, 6 - minute walk distance | Capricor, Inc. KOL Lunch on DMD 62

North Star Ambulatory Assessment 63

NORTH STAR AMBULATORY ASSESSMENT Sit to stand Descend step | Capricor, Inc. KOL Lunch on DMD 64

Jump Gets to sit | Capricor, Inc. KOL Lunch on DMD 65

6MWT Adapted/validated for DMD (McDonald 2010) Regulator approved Minimally clinically important difference considered 30 meters Correlates with: Knee extension strength 10 minutes continuous step activity Number of challenges conducting test, especially with younger and more cognitively impaired patients Shorter fixed distance tests under development | Capricor, Inc. KOL Lunch on DMD 66

6 - MINUTE WALK TEST 6MWT: popular primary outcome measure Other primary outcome measures exist, including 4 - stair climb E Mercuri showed that if the distance walked in 6 minutes is at least 330 metres at baseline, the risk of losing ambulation within 2 years is significantly reduced ( Mazzone et al. PLOS One 2013;8:e52512) And that for every 30 metre incremental decrease in the baseline 6MWT, the percentage of patients who remain ambulatory over the following 2 years decreases substantially However, a $50 incentive can significantly improve the test result ( Alfano et al. Neuromuscul Dis 2014; 24:860 ) 6MWT, 6 - minute walk test | Capricor, Inc. KOL Lunch on DMD 67

STRENGTH TESTING | Capricor, Inc. KOL Lunch on DMD

MANUAL MUSCLE TESTING (MMT) MRC scale Reliable in patients with NMD when conducted by experienced evaluators Issues with differentiating between higher grades: Importance of practice and experience Cheap/easy to conduct 69 | Capricor, Inc. KOL Lunch on DMD

HAND HELD MYOMETRY Variety of myometers available: Microfet Make test Explanation/demonstration/prac tice Key muscle groups Standardised testing positions and myometer placement Best of 3"valid" efforts Repeat if patient moves out of position or doesn't gives a submaximal effort Encouragement +++ As you would for any effort dependent test 70 | Capricor, Inc. KOL Lunch on DMD

QUANTITATIVE MUSCLE TESTING SYSTEMS CINRG CQMS 71 | Capricor, Inc. KOL Lunch on DMD

ASSESSMENTS FOR NON - AMBULANT | Capricor, Inc. KOL Lunch on DMD

NON - AMBULATORY MILESTONES - EGEN KLASSIFIKATION SCALE The EK scale is a good clinical tool - each question can help to formulate requirements for treatment or adaptations Highly clinically meaningful but less sensitive to change over the short term Loss of ability to turn in bed Loss of head control Loss of trunk mobility Loss of ability to control wheelchair EK, Egen Klassifikation 73 | Capricor, Inc. KOL Lunch on DMD

Prior to this publication in 2012 there were numerous workshops and discussions on existing measures Need for a tool that could assess upper limb function across the lifespan 74 | Capricor, Inc. KOL Lunch on DMD

NON - AMBULATORY MILESTONES Loss of ability to reach over head Loss of ability to reach the scalp Loss of ability to self - feed without adaptations (hand to mouth) Loss of ability to place hands to table top Loss of ability to use a computer (distal hand function) Performance of Upper Limb Developed using Rasch Analysis and clinical sensibility 75 | Capricor, Inc. KOL Lunch on DMD

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CONCEPTUAL FRAMEWORK | Capricor, Inc. KOL Lunch on DMD 77

DIMENSIONS+ ITEMS | Capricor, Inc. KOL Lunch on DMD 78

Version 1.1, 1.2 and now 2.0 79 | Capricor, Inc. KOL Lunch on DMD

PUL ITEMS Shoulder domain mid level - domain | Capricor, Inc. KOL Lunch on DMD 80

PUL ITEMS Bilateral activity Distal domain | Capricor, Inc. KOL Lunch on DMD 81

RESPIRATORY FUNCTION TESTS | Capricor, Inc. KOL Lunch on DMD

RESPIRATORY FUNCTION Respiratory function (RF) and survival are strongly associated Most frequently, both in clinics and in trials, forced vital capacity is measured Milestones include time to ventilation and death Other commonly used measures are maximal inspiratory pressure, maximal expiratory pressure, and maximal sniff nasal inspiratory pressure , Peak cough flow, peak expiratory flow Measurement in non - ambulant boys is particularly relevant but improving RF in ambulant boys is a goal for therapy 84 | Capricor, Inc. KOL Lunch on DMD

Survival in Duchenne muscular dystrophy: 1967 - 2002 0 5 10 15 20 25 30 0.0 0.2 0.4 0.6 0.8 1.0 Died in 60s Died in 70s Died in 80s Died in 90s not ventilated ventilated in 90s age at death %survival Mean age of death vent + SS vent 1990s 1980s 1970s 1960s CM 10 11 12 13 14 15 16 17 18 19 20 21 22 23 24 25 26 27 28 29 30 ventilated in 1990s Died in 1990s Died in 1980s Died in 1970s Died in 1960s Cardiomyopathy vent + SS age at death 85 | Capricor, Inc. KOL Lunch on DMD

NORTH STAR AMBULATORY ASSESSMENT 6 MINUTE WALK TEST TIMED FUNCTION TESTING Performance of upper limb Performance of upper limb/EK SCALE MUSCLE STRENGTH TESTING RESPIRATORY FUNCTION TESTING AMBULANT TRANSITIONAL NON AMBULANT YOUNGER LIFETIME OLDER 86 | Capricor, Inc. KOL Lunch on DMD

Thank you | Capricor, Inc. KOL Lunch on DMD

Duchenne Muscular Dystrophy: Overview, Natural History, and Unmet Need Craig McDonald, MD Professor of PM&R and Pediatrics Study Chair CINRG Duchenne Natural History Study University of California Davis Medical Center Sacramento, CA | Capricor, Inc. KOL Lunch on DMD 8 8

Disclosures Consulting work on Duchenne muscular dystrophy clinical trials for - Capricor Therapeutics, Inc. - Catabasis Pharmaceuticals, Inc. - PTC Therapeutics - Sarepta - Prosensa - Pfizer - Eli Lilly - Bristol Myers Squib - Italfarmaco - Mitobridge - Cardero Therapeutics | Capricor, Inc. KOL Lunch on DMD 8 9

Duchenne Muscular Dystrophy Is a Devastating Progressive Disease with Significant Unmet Need Rare recessive x - linked disorder caused by mutation in the DMD gene Leads to dystrophin deficiency in muscle tissue and subsequently chronic activation of NF - kB Progressive disease that leads to devastating deteriorating muscle strength and early death Only supportive treatments are available - Physical therapy - Orthopedic Surgery for contractures and scoliosis - Assisted ventilation - Heart failure management (e.g., afterload reduction) - Off - label / labeled use of corticosteroids - Eteplirsen in the US for exon - 51 mutations - Ataluren in the EU for nonsense mutations | Capricor, Inc. KOL Lunch on DMD 9 0

Disease Progression Is Characterized by Muscle Damage and Replacement of Muscle Fibers with Fat Infiltration and Sclerosis, Resulting in Loss of Function Normal muscle tissue Muscle tissue 19 year old DMD patient Post - Mortem Fat infiltration, sclerotic changes and loss of muscle fibers Lack of dystrophin cause shearing of the sarcolemma and activation of NF - kB, increasing cellular damage and muscle fiber loss Progressive loss of muscle fibers and replacement of functional muscle units by fat infiltration and sclerosis Loss of function; walking capacity preserved in spite of significant loss of muscle strength due to Reserve capacity in muscle function Biomechanical compensations | Capricor, Inc. KOL Lunch on DMD 9 1

Loss of Muscle Fiber in DMD: Normal 3 year old 9 year old 19 year old (Post - Mortem in Year 1990) Post - Mortem Serum CK | Capricor, Inc. KOL Lunch on DMD 9 2

DMD pathomechanism Adapted from Engvall & Wewer (2003) FASEB 17:1579 Structural defect Membrane instability Apoptosis / Necrosis Inflammation Fibrosis Fiber Death | Capricor, Inc. KOL Lunch on DMD 9 3

Motor and Cognitive Assessment of Infants and Young Boys with Duchenne Muscular Dystrophy: Results from the Muscular Dystrophy Association DMD Clinical Research Network. Connolly et al. (n=25; 1.8 0.8 years) | Capricor, Inc. KOL Lunch on DMD 9 4

Neck Flexion Weakness one of earliest clinical signs 2 years, 10 months | Capricor, Inc. KOL Lunch on DMD 9 5

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Early Gower ' s Sign (2 yr 10 mo) | Capricor, Inc. KOL Lunch on DMD 9 7

9 Year Old Boy: Baseline Assessment Rise From Floor 7 Sec; 6MWD 414 Meters | Capricor, Inc. KOL Lunch on DMD 9 8

Case Study: 9 Year Old Boy: Assessment in 2007 Rise From Floor 13 Sec; 6MWD 330 Meters | Capricor, Inc. KOL Lunch on DMD 9 9

DMD Patient Age 9.5 10 - meter Walk/Run = 6.5 s 6MWD = 330 Meters Case Study: 9 Year Old Boy: Assessment in 2007 10 meter run / walk = 6.5 seconds; 6MWD 330 Meters | Capricor, Inc. KOL Lunch on DMD 100

Case Study: Burden of Disease Same Young man aged 17 Assessment 2015 | Capricor, Inc. KOL Lunch on DMD 101