Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Technical Presentation (April 2017)
22 slides from Capricor Therapeutics's technical presentation, Apr 25, 2017
Slide 1: Top-Line Six-Month Results of the HOPE-Duchenne Trial
NASDAQ: CAPR April 25, 2017 a translational m edicine c ompany Top - Line Six - Month Results of the HOPE - Duchenne Trial Conference Call to Discuss
Slide 2: Forward-Looking Statements
2 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 statements of historical fac
Slide 3: Positive Top-Line Six-Month Safety and Exploratory Efficacy Data
3 Positive Top - Line Six - Month Safety and Exploratory Efficacy Data — Statistically - significant i mprovements were observed in exploratory efficacy measures of cardiac and upper limb function in patients who received CAP - 1002 — Clinical results recapitulate pre - clinical data in cardiac and
Slide 4: Duchenne Muscular Dystrophy (DMD) and CAP-1002
4 Duchenne Muscular Dystrophy (DMD) and CAP - 1002 — DMD is a genetic disorder characterized by progressive and debilitating muscle weakness ▪ Results from a lack of functional dystrophin ▪ Affects cardiac muscle and all skeletal muscle ▪ Muscle cell death due to increased fragility ▪ Capacity of mu
Slide 5: About CAP-1002 (allogeneic cardiosphere-derived cells)
5 About CAP - 1002 (allogenic cardiosphere - derived cells) — CAP - 1002 is a cardiac progenitor cell therapeutic candidate ▪ CDCs have been shown to lead to scar regression and generate functional cardiac muscle — Mechanisms of action of CAP - 1002: ▪ A nti - inflammatory, anti - fibrotic , and ant
Slide 6: CAP-1002 Mechanism of Action via Exosomes
6 CAP - 1002: Mechanism of Action via Exosomes ▪ Cells release exosomes – particles comprised of RNAs and proteins ▪ Exosomes act on target cells to epigenetically alter gene expression ▪ Exosomes isolated from CDCs recapitulate their actions
Slide 7: The Randomized Phase I/II HOPE-Duchenne Clinical Trial
7 The Randomized Phase I/II HOPE - Duchenne Clinical Trial — Single dose coronary infusion, once at baseline — Safety trial with multiple exploratory efficacy endpoints — Three U.S. sites: Cedars - Sinai Medical Center Cincinnati Children’s Hospital Medical Center University of Florida Ages 12+ Stab
Slide 8: Baseline Characteristics
8 Baseline Characteristics — Population characterized by advanced disease; majority are non - ambulant — Essentially all DMD clinical development has been conducted in less sick patients Usual Care (n=12) CAP - 1002 (n=13) Age (median, range) 17.5 (12 – 20) 18 (14 – 25) Wheelchair Use Always 7 (58%)
Slide 9: Safety Outcome Measures
9 Safety Outcome Measures Usual Care (n=12) CAP - 1002 (n=13) Peri - procedural TIMI grade flow 0 - 2 0 0 Peri - procedural sudden unexpected death 0 0 Peri - procedural major adverse cardiac events (MACE) 0 0 Sudden unexpected death 0 0 MACE 0 0 CV hospitalization 0 0 Ventricular tachycardia ≥ 30 s
Slide 10: Safety and Tolerability
10 Safety and Tolerability Usual Care (n=12) CAP - 1002 n=13) Any treatment - emergent adverse event (TEAE) 10 (83%) 11 (85%) Severe TEAE 2 (17%) 1 (8%) Serious Adverse Events (SAE) 1 (8%) 3 (23%) Any drug - related TEAE n/a 8 (62%) Any procedure - related TEAE n/a 7 (54%) Discontinuation due to TEA
Slide 11: Exploratory Efficacy Endpoints
11 Exploratory Efficacy Endpoints Baseline Week 6 Month 3 Month 6 Month 12 Imaging Cardiac magnetic resonance ( cMRI ) ● ● ○ Functional Tests Performance of the Upper Limb (PUL) ● ● ● ● ○ 6 - Minute Walk Test ● ● ● ● ○ Spirometry ● ● ● ● ○ Quality of Life Pediatric QL Inventory ● ● ● ● ○ PODCI Adole
Slide 12: Reduced Cardiac Scar in Treated Patients
12 Reduced Cardiac Scar in CAP - 1002 Treated Patients Spontaneous scar reduction is not observed in DMD improvement
Slide 13: Improved Systolic Wall Thickening in Treated Patients
13 Improved Systolic Wall Thickening in CAP - 1002 Treated Patients Systolic thickening is a principal mechanism of cardiac output generation in DMD improvement (ns) (ns)
Slide 14: Results of Ejection Fraction and Cardiac Volumes
14 Results of Ejection Fraction and Cardiac Volumes Patients had near - normal ejection fraction and low volumes at baseline improvement improvement improvement (ns) (ns) (ns)
Slide 15: Performance of the Upper Limb (PUL) Test
15 Performance of the Upper Limb ( PUL) Test — The Performance of the Upper Limb (PUL) test was designed specifically for use in the DMD setting — Evaluates a variety of manual tasks, such as lifting cans, tearing paper, and removing a container lid, which simulate activities of daily living — Boys
Slide 16: Performance of the Upper Limb (PUL) Test
16 Performance of the Upper Limb (PUL ) Test
Slide 17: Mid- and Distal-Level PUL Results
17 Mid - and Distal - Level PUL Results improvement (ns) (ns) (ns)
Slide 18: Responder Analysis on Mid- and Distal-Level PUL
18 Responder Analysis on Mid - and Distal - Level PUL P otential functional improvement in skeletal muscle improvement Responder defined as ≥ 10% or max possible improvement. (ns) (ns)
Slide 19: Cardiac and Skeletal Muscle Effects Seen in the Same Patients
19 Cardiac and Skeletal Muscle Effects Seen in the Same Patients Inferior Wall Thickening + Middle - Level PUL Scar Reduction + Middle - Level PUL Supportive of a treatment effect p - values: * Analysis excluded one subject due to uninterpretable imaging data. *
Slide 20: Quality of Life (PODCI Adolescent Questionnaire)
20 Quality of Life (PODCI Adolescent Questionnaire) improvement (ns) (ns)
Slide 21: Key Conclusions from Six-Month HOPE Data
21 Key Conclusions from Six - Month HOPE Data — Clinical results are in line with preclinical data that illustrate the bioactivity of CAP - 1002 on both cardiac and skeletal muscle in DMD — Exploratory efficacy analyses support the potential benefit of CAP - 1002 to boys and young men with advanced
Slide 22: Next Steps for Capricor's Duchenne Muscular Dystrophy Program
22 Next Steps for Capricor’s Duchenne Muscular Dystrophy Program — Requested FDA meeting to review HOPE data and discuss development toward potential registration — Plan to submit applications for Breakthrough Therapy or Regenerative Medicine Advanced Therapy (RMAT) designations — Repeat dosing via
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