Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (December 2019)
23 slides from Capricor Therapeutics's investor presentation, Dec 17, 2019
Slide 1: Corporate & Investor Presentation
NASDAQ : CAPR Corporate & Investor Presentation December 2019
Slide 2: Forward-Looking Statements
Forward - Looking Statements 2 Statements in this press release 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 r
Slide 3: Developing Cell and Exosome-Based Therapies for Rare Diseases
Developing Cell and Exosome - Based Therapies for Rare Diseases RARE DISEASE FOCUS STRATEGIC COLLBORATIONS DOMAIN EXPERTISE FINANCIAL • Preclinical Stage: Exosome platform technology in active development • Advanced Clinical Program: Cell Therapy (CAP - 1002) for Duchenne muscular dystrophy (DMD) •
Slide 4: The Field of Exosomes: Increasing Market Opportunity
The Field of Exosomes: Increasing Market Opportunity ▪ Global market size projected to grow at high rates ▪ Increasing financing activity among private companies: ▪ Over $300M raised to date in several private companies ▪ M&A activity continuing to increase: ▪ Delivery ▪ Roche collaboration with Pur
Slide 5: Delivery of Gene-based Therapies to Treat Disease
Delivery of Gene - based Therapies to Treat Disease ▪ Gene therapy using viral delivery (AAV) ▪ Immune response ▪ Delivery of RNAs ▪ Uptake to render biologic relevance ▪ Therapeutic development very slow ▪ Synthetic nanoparticles have not been successful as delivery vehicles 5 Exosomes : ▪ nature’s
Slide 6: Why Engineer Exosomes?
‒ Engineering exosomes loaded with bioactive molecules potentially will: ▪ increase potency ▪ reduce variability ▪ eventually help during product development ‒ Different modifications are now under evaluation: ▪ Cargo – Nucleic acids (mRNA or miRNAs) ▪ Membrane modifications – Tropism change – Coati
Slide 7: Targeted Exosome Platform Allows for Broad Applicability
Targeted Exosome Platform Allows for Broad Applicability Regenerative Medicine 2.0 • Immunomodulation • Tissue regeneration CDC - XO (Exosomes) Specific Tropism • Modify XO membrane • Targeted delivery Surface - Modified Exosomes Loaded XOs • XO loaded with therapeutic cargo Engineered Exosomes 7
Slide 8: Loading miRNAs into Exosomes
Loading miRNAs into Exosomes ▪ MicroRNAs (miRNAs) are a class of small, noncoding RNAs involved in regulation of gene expression ▪ Loading XOs with miRNAs may allow for tunable regulation of gene expression in target cells 8
Slide 9: Capricor's Exosome Product Pipeline
Capricor’s Exosome Product Pipeline 9 Candidate Target Indications Development Phase Status Discovery Preclinical Phase I EC - XO (expanded culture XOs) Inflammatory diseases Target IND submission in 2020 Engineered XOs Small RNA Loading (siRNAs, miRs , sgRNAs) Evaluating Target IND submission Engin
Slide 10: EC-XOs: Mechanism of Action
EC - XOs: Mechanism of Action Immunomodulator y Anti - inflammatory Anti - fibrotic Anti - apoptotic Angiogenic Regenerative 10
Slide 11: CDC-XOs Immunogenicity
CDC - XOs Immunogenicity ▪ Allogeneic CDCs have a low immunogenicity (20 times lower than xeno - transplant) ▪ In pre - clinical studies, CDC - XOs show a reduced immunogenicity when compared to CDCs, allowing multiple administration without significant immune response Humoral Immune Response CDC_1C
Slide 12: Comparison of CDC-XO vs MSC-XOs Reveal Different Cargo
MSC CDC MSC CDC Comparison of CDC - XO vs MSC - XOs Reveal Different Cargo CDC - exosomes MSC - exosomes miRNA clustering analysis (RNA - Seq) Exosomes RNA - Seq Read counts (type) High Low Exosomes surface expression profile ( MACSplex ) CDC #1.1 CDC #1.2 CDC #2.1 CDC #2.2 CDC #3.1 CDC #3.2 MSC #1
Slide 13: Exosomes: POC Established in Multiple Indications
13 Exosomes: POC Established in Multiple Indications GVHD LUPUS NEPHRITIS SEPSIS DUCHENNE MUSCULAR DYSTROPHY* F4/80 + CD11b + Macrophage: 46.5% Macrophage: 14.5% 21 28 35 42 49 56 2 4 6 8 Day G V H D S c o r e Vehicle Low dose High Dose 21 28 35 42 49 56 Days 8 6 4 2 GVHD Score GVHD Score Vehicle Lo
Slide 14: Exosomes Lyophilization
Exosomes Lyophilization Particle size miRNA stability Lyophilized CDC - XO show : • Good conservation of miRs 146a and 210 • Similar particle size and concentration post lyophilization • Similar results in - vitro and in - vivo to UFC product In vitro bioactivity In vivo bioactivity Macrophage activ
Slide 15: World-Class Facilities and Infrastructure
World - Class Facilities and Infrastructure • Capricor’s Research, Development and Manufacturing facilities are located in the Cedars - Sinai Medical Center in Los Angeles, CA • Capricor has access to core research facilities 15
Slide 16: CAP-1002 Duchenne Muscular Dystrophy Program
CAP - 1002 Duchenne Muscular Dystrophy Program 16
Slide 17: Conclusions and Future Direction – CAP-1002 for DMD
Conclusions and Future Direction – CAP - 1002 for DMD ‒ First placebo - controlled trial showing upper limb functional improvements in non - ambulant DMD patients ‒ Directionally consistent improvements in function, strength, pulmonary and cardiac endpoints 17 ‒ Phase III clinical trial in pre - FDA
Slide 18: Senior Leadership Team
Senior Leadership Team Linda Marbán , Ph.D. Chief Executive Officer, Co - founder and Director Under her direction, Capricor has secured over $45 million in non - dilutive funding and over $50 million in equity capital. Earlier in her career, Dr. Marbán was with Excigen, Inc. where she was responsib
Slide 19: Appendices
capricor Appendices
Slide 20: CDC-XOs miR Profile: Identity, Purity & Potency
CDC - XOs miR Profile: Identity, Purity & Potency Potent Non - Potent Potency ( miR - #3) CDC 1 CDC 2 CDC 3 CDC 4 CDC 5 CDC 6 CDC 7 CDC 8 CDC 10 CDC 9 CDC 1 CDC 2 CDC 3 CDC 4 CDC 5 CDC 6 CDC 7 CDC 8 CDC 9 CDC 10 MSC vs CDC - EVs CDC - EVs CDC - EVs MSC - EVs Potent Non - Potent MSC - XO CDC - XO Pur
Slide 21: Exosomes Landscape
Exosomes Landscape 21 Information from public sources Exosomes Field Platform for therapeutics - Engineered - Wild - type CAPRICOR Oncology - Engineered - Drug - loaded Diagnostics Wild - Type for therapeutics
Slide 22: Functional capacity after oral exosome delivery in MDX mice
0 50 100 150 200 250 300 Exercise capacity CTL C X+C X * Distance(meter) Distance(m) Ejection fraction 0 500 1000 1500 2000 0 25 50 75 100 30 40 50 60 70 WT CTL C X X+C EF(%) * R 2 =0.71 P<0.001 EF week3 5 10 15 20 25 WT CTL C X X+C Soleus specific force wk3 Po(N/cm 2 ) * 0 20 40 60 80 0 250 500 750
Slide 23: CDC-exosomes are taken up via endocytosis in duodenal epithelial cells
3 2 1 1 3 2 CDC - exosomes are taken up via endocytosis in duodenal epithelial cells and released into blood after fusion of their membrane or MVB membrane* *Unpublished data, Marbán lab, Smidt Heart Institute, CSMC, 12/2019 23
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