Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (December 2015)
35 slides from Capricor Therapeutics's investor presentation, Dec 10, 2015
Slide 1: Transformative Therapies from Bench to Bedside
NASDAQ: CAPR December 2015 Transformative Therapies from Bench to Bedside
Slide 2: Forward-Looking Statements
2 Forward - Looking Statements This presentation contains forward - looking statements and information that are based on the beliefs of the management of Capricor Therapeutics, Inc. (Capricor) as well as assumptions made by and information currently available to Capricor. All statements other than s
Slide 3: Capricor Financial Highlights
3 Capricor Financial Highlights – Capricor, Inc. founded in 2005 (Baltimore, MD; JHU spinoff) – Completed reverse merger with Nile Therapeutics in November 2013 – Uplisted to NASDAQ in March 2015 – Total Cash as of Sept. 30 , 2015: $17.2M (current cash out: ῀ Q3 2016) – Non - dilutive capital fundin
Slide 4: Capricor Opportunity
4 Capricor Opportunity – Pioneer in Cardiac - Derived Cell (CDC) Technology and CDC - Derived Exosomes – Solid IP position in CDC and E xosome Technology – Diversified platform and therapeutic pipeline – Orphan indication: DMD - associated cardiomyopathy program – Meaningful clinical milestones over
Slide 5: Capricor's Platform & Therapeutic Pipeline
5 Cardiosphere - Derived Cells CAP - 1002 Micro - RNA Platform Natriuretic Peptide therapy Capricor’s Platform & Therapeutic Pipeline
Slide 6: Cardiosphere-Derived Cells (CDCs): Adult Heart disease
6 Cardiosphere - Derived Cells (CDCs): Adult Heart disease
Slide 7: Cell Therapy Platform: CDCs (CAP-1002)
7 Cell Therapy Platform: CDCs (CAP - 1002) Cardiosphere - derived cells (CDCs) Cardiospheres (CSps) Explant - derived cells (EDCs) Explants Cardiac Tissue Features Cardiosphere - Derived Cells (CDCs) Cell Type Human cardiac derived cell Characteristics Unique panel of cellular markers and secreted f
Slide 8: CADUCEUS: Proof of Concept First-in-Man Data
8 CADUCEUS: Proof of Concept First - in - Man Data – Intracoronary delivery of autologous CDCs - 25M cells – Patients with reduced ejection fraction following MI – 25 patients (17 CDCs, 8 Controls) – Sponsored by Cedars - Sinai Medical Center – Results : CDCs reduced the amount of scar in the heart
Slide 9: CADUCEUS: CDC Therapy Reduced Scar Size & Increased Healthy Heart Muscle
9 Makkar et al, Lancet, 2012. CDC p atients had a s ignificant r eduction in i nfarct (scar) s ize and an increase in healthy heart muscle mass. We hypothesize improvement in clinical outcomes . CADUCEUS : CDC Therapy Reduced Scar Size & Increased Healthy Heart Muscle
Slide 10: Effect of Infarct (Scar) Size on Events
10 Effect of Infarct (Scar) Size on Events Wu, E. et al. Heart 94, 730 - 736 (2008 ).
Slide 11: Conclusions from CADUCEUS
11 Conclusions from CADUCEUS – CDCs reduced the amount of scar in the heart caused by a heart attack – Smaller scars may lead to better outcomes – CDCs increase healthy heart muscle mass – Autologous manufacturing is not a viable business model in this indication – Multiple clinical programs underwa
Slide 12: Janssen (J&J) Deal Highlights
12 Janssen (J&J) Deal Highlights Collaboration Agreement and Exclusive License Option with Janssen Biotech for Cell Therapy Program for Cardiovascular Applications – Received upfront payment of $12.5M – Collaboration on elements of cell manufacturing – Exclusive license agreement option for CAP - 10
Slide 13: DYNAMIC: Concordance of Data Suggest Improvement
13 DYNAMIC: Concordance of Data Suggest Improvement AHA: November 2015 NYHA class LV Function & Dimensions (echo) 6 month data for 2 subjects pending Less is better
Slide 14: DYNAMIC: Concordance of Data Suggest Improvement
14 DYNAMIC: Concordance of Data Suggest Improvement AHA: November 2015 6MWT & VO 2 Max Quality of Life 6 month data for 2 subjects pending Less is better
Slide 15: CDCs: Clinical Development
15 ALLSTAR Clinical Trial Collaboration with Janssen Biotech (J&J) ῀ $20M loan award from CIRM Phase II Phase I/II Enrolling Data anticipated: Q1 2017 Phase I/II Validate CADUCEUS data POC with ALLO cells Adult Heart Failure Market (5M HF patients US $32B/annual cost) Orphan Disease Small market/ Bi
Slide 16: Duchenne Muscular Dystrophy
16 Duchenne Muscular Dystrophy Prevalence : 1 in 3,500 male births worldwide ~20,000 male children affected in the US (~275,000 worldwide) DMD is fatal with most deaths due to cardiomyopathy Reference: McNeil et. al, Muscle & Nerve, 2010
Slide 17: Orphan Drug Designation Granted to CAPR
17 Orphan Drug Designation Granted to CAPR – CAP - 1002 can be used in CONJUNCTION with ANY other dystrophin - correcting therapies targeting skeletal muscle ▪ These therapies do not appear effective in cardiac muscle ▪ Very few clinical trials to treat DMD cardiomyopathy – Presents potential billio
Slide 18: Correlation between Scar and LVEF
18 Correlation between Scar and LVEF – Advances in MRI technology have enabled accurate scar measurement – Data shows the difference between “compensated” vs “decompensated” HF is defined by a small amount of muscle mass – Reducing scar could potentially lead to a longer and more productive life Men
Slide 19: mdx Mouse Model of Duchenne Muscular Dystrophy
19 – M imics the target indication, DMD – A ged to the point at which cardiac dysfunction becomes evident: ≥10 months old Reference: Cedars - Sinai Heart Institute mdx Mouse Model of Duchenne Muscular Dystrophy
Slide 20: Global Cardiac Function is improved in mdx Mice
20 * *** * 50 55 60 65 70 75 Baseline Wk3 M2 M3 Mdx+CDC Mdx+Vehicle CTL(WT) Reference: Cedars - Sinai Heart Institute Presented at AHA - November 2014, Chicago, IL * * 30 40 50 60 70 80 Baseline Wk3 M2 M3 Wk3 M2 M3 Mdx+CDC Mdx+Vehicle *** * * 1 st injection 2 nd injection EF(%) Presented at ISEV - A
Slide 21: CDCs Increased Maximal Exercise Capacity in mdx Mice
21 100 160 220 280 340 400 460 520 580 640 700 760 820 wk3 wk4 wk5 wk6 CTL Mdx+CDC Mdx+Vehicle CDCs Increased M aximal E xercise Capacity in mdx Mice Distance(m) ** ** ** ** n = 6 - 11 Reference: Cedars - Sinai Heart Institute Presented at AHA - November 2014, Chicago, IL
Slide 22: Duchenne Cardiomyopathy and CDCs
22 Duchenne Cardiomyopathy and CDCs Injection of CDCs into mdx mouse hearts – Improves global function – D ecreases fibrosis – Improves exercise capacity – Exerts potent anti - oxidant effects – R everses abnormalities in mitochondrial abundance, structure and function – I ncreases cardiomyocyte pro
Slide 23: HOPE: Halt cardiomyOPathy progrEssion in Duchenne
23 H alt cardiomy OP athy progr E ssion in Duchenne (HOPE) – FDA approved Phase I/II clinical trial (orphan designation) – R andomized , open label multi - center study ( ῀ 3 - 4 sites) ▪ 12 boys randomized to CDC (CAP - 1002) infusion ▪ 12 boys randomized to ‘usual care’ – Triple vessel intra - cor
Slide 24: Exosomes: Next Generation Regenerative Medicine Therapeutic Platform
24 Exosomes: Next Generation Regenerative Medicine Therapeutic Platform
Slide 25: Exosomes: Cell Free Regenerative Medicine
25 Exosomes: Cell Free Regenerative Medicine – Nanometer - sized lipid - bilayer vesicles – Rich in RNAs and proteins – Secreted by nearly all cell types – Cell signaling modality – Potential for broad therapeutic applicability – IP : Exclusive world - wide license agreement with Cedars - Sinai Medi
Slide 26: Exosomes: Paracrine Signals Suggest Cells MOA
26 Exosomes: Paracrine Signals Suggest C ells MOA – Autologous and allogeneic cells show a similar potency – Delivered cells are cleared shortly after administration (< 1 month) – The therapeutic benefit is maintained after delivered cells are gone – Administered cells promote endogenous regenerativ
Slide 27: CDC Exosomes Improve Cardiac Function and Preserve Muscle Mass
27 CDC E xosomes Improve Cardiac Function and Preserve M uscle Mass 0 2 4 6 8 10 12 14 16 18 CTRL NHDF-XO MSC-XO CDC-XO Scar Mass (mg) ** ** * CTRL NHDF - Exosomes MSC - Exosomes CDC - Exosomes 25 30 35 40 45 50 1 15 30 EF (%) Days post MI Control CDC-XO NHDF-XO MSC-XO ** * Ibrahim et al, Stem Cell
Slide 28: Exosomes: Targeted Milestones
28 – Targeting announcing First - in - Man clinical indication: Q1 2016 – Indications under consideration: ▪ Eyes ▪ Skin ▪ Cancer – Meet with FDA: Q1 2016 – Targeting IND submission: 2016 Exosomes: Targeted Milestones
Slide 29: Natriuretic Peptide Technology
29 NATRIURETIC PEPTIDE TECHNOLOGY
Slide 30: The Post-Acute Hospitalization Period
30 The Post - Acute Hospitalization Period ( 90 days): When the Rate of Re - hospitalization and Death are Highest 0% 20% 40% 60% - 60 120 180 240 300 360 Percent of Patients Days After Hospital Admission for Heart Failure % of Patients with Death or Re - hospitalization after admission for Acute HF
Slide 31: Mayo Designed CD-NP/Cenderitide: Only Dual NP Receptor Activator
31 Mayo Designed CD - NP/Cenderitide: Only Dual NP Receptor Activator - S - S - K L L D R I G S M S G L G G F C C K G S L G pGC - B agonist Anti - fibrotic Endothelial regenerating Anti - inflammatory P S L R D CD - NP Cenderitide P R P N A P S T S A pGC - A agonist Enhances renal function Suppresse
Slide 32: Cenderitide: The Opportunity
32 - S - S - K L L D R I G S M S G L G G F C C K G S L G N H 2 P S L R D P R P N A P S T S A Cenderitide The Opportunity + 1 st arm of Phase II complete
Slide 33: Cenderitide for Outpatient and Ambulatory Heart Failure
33 Cenderitide for Outpatient and Ambulatory Heart Failure Target Indication Prevention of re - hospitalization in patients with a recent acute heart failure admission as well as other potential indications – Phase IIa PK/PD Trial ▪ 1 st arm - 14 patients treated, enrollment complete ▪ Patients with
Slide 34: Senior Management, Board of Directors and Scientific Advisory Board
34 Senior Management – Chief Executive Officer Linda Marbán, Ph.D. (Founder, JHU, Cleveland Clinic) – Chief Medical Officer Deborah Ascheim, M.D. (Mount - Sinai, Columbia University) – EVP & General Counsel Karen Krasney, J.D. – Acting VP Clinical Operations: Jeff Rudy, (Amgen, Celladon) – VP of New
Slide 35: Transformative Therapies from Bench to Bedside
NASDAQ: CAPR December 2015 Transformative Therapies from Bench to Bedside www.capricor.com
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