Slide 1: HOPE-2 Multi-center Randomised Clinical Trial: 1 Year Final Results
HOPE - 2 Multi - center Randomised Clinical Trial of Intravenous Human Cardiosphere - Derived Cells for Late - Stage Duchenne Muscular Dystrophy Data Presentation 1 Year Final Results Craig McDonald, M.D., University of California, Davis Health Eduardo Marbán, MD, PhD, Suzanne Hendrix, PhD, Nathanie
Slide 2: Forward Looking Statements
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 regard
Slide 3: Call Participants
• Linda Marban, Ph.D. – Chief Executive 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 internati
Slide 4: Capricor Therapeutics Company Highlights
Capricor Therapeutics Company Highlights Capricor is a cell and exosome - based platform therapeutics company Publicly Traded (NASDAQ: CAPR) • Research Headquarters: San Diego, CA • Employees: 30 • Founded: 2006, public in 2013 Promising Therapeutic and Vaccine Candidates • Cell Therapy (Duchenne mu
Slide 5: Data Presentation: Dr. Craig McDonald
Data Presentation: Dr. Craig McDonald 1 Year Final Results 5
Slide 6: Disclosure: Dr. McDonald
Disclosure: Dr. McDonald I have the following conflict(s) of interest to declare: • Served as a consultant and advisory board member for Capricor Therapeutics on issues related to DMD drug development • Academic Institution has received research funding from Capricor Therapeutics for the conduct of
Slide 7: Capricor's 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 • Mechanism - cells secrete exosomes: » Contain miRNA, non - coding RNAs and proteins » Internaliz
Slide 8: Targets Multiple Disease Processes in DMD
damage to cell membrane CAP - 1002 dystrophin deficiency cellular [Ca 2+ ] myocyte loss nitrosactive stress inflammation mitochondrial inefficiency fibrosis microvascular ischemia CAP - 1002 Targets Multiple Disease Processes in DMD 8 Exosomes
Slide 9: DMD Progression is Sequential, Non-Linear and Irreversible
DMD Progression is Sequential, Non - Linear and Irreversible 9 0 5 10 15 20 25 30 Years Loss of Ambulation Death Impaired ability to Hop Run Jump Rise from Floor Loss of Rise from Floor Loss of Stair Climb Stages of DMD Disease Progression Loss of Upper Limb Overhead reach Late Ambulatory Stage (Rap
Slide 10: Primary Efficacy Endpoint Mid-Level PUL
Primary Efficacy Endpoint Mid - Level PUL v.1.2 PUL v.2.0: • 3 - point response scale - more robust and reproducible than v1.2 • Compensatory strategies allowed to achieve tasks (not allowed in v1.2) • v2.0: better able to detect change at 12 months at all levels of ability* *Mayhew et al, 2019;
Slide 11: Performance of Upper Limb (Entry Items)
Performance of Upper Limb (Entry Items) 0 1 2 3 4 5 6 No useful function of hands. Can use hands to hold pen or pick up a coin or drive a powered Chair Can raise 1 or 2 hands to mouth but cannot raise a cup with a 200g weight in it to mouth Can raise standardized plastic cup with 200g weight in it t
Slide 12: Clinical Trial Design
HOPE - 2 Clinical Trial Design 12 • Design: Phase II, 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 • Sit
Slide 13: Final Data Safety Results
HOPE - 2 Final Data Safety Results • 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 two hypersensitivity reactions, no safety signals were identified 13
Slide 14: Final Data Efficacy Analysis: PUL 1.2 Mid Level
14 HOPE - 2 Final Data Efficacy Analysis Primary Efficacy Endpoint : PUL 1.2 Mid Level Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as dependent variable and percentile ranked baseline score, treatment, visit, treatment - by - visit int
Slide 15: Final Data Efficacy Analysis: PUL 1.2 Combined Total Dimension
15 HOPE - 2 Final Data Efficacy Analysis PUL 1.2 Combined Total Dimension Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as dependent variable and percentile ranked baseline score, treatment, visit, treatment - by - visit interaction, PUL
Slide 16: Final Data Efficacy Analysis: PUL 2.0 Combined Total Dimension
16 HOPE - 2 Final Data Efficacy Analysis PUL 2.0 Combined Total Dimension Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as dependent variable and percentile ranked baseline score, treatment, visit, treatment - by - visit interaction, PUL
Slide 17: Final Data Efficacy Analysis: All Dimensions of PUL
17 HOPE - 2 Final Data Efficacy Analysis Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as dependent variable and percentile ranked baseline score, treatment, visit, treatment - by - visit interaction, PUL entry - item score at randomizat
Slide 18: Final Data Efficacy Analysis: Left Ventricular Ejection Fraction
18 HOPE - 2 Final Data Efficacy Analysis Left Ventricular Ejection Fraction improvement Δ=4% in LVEF by cardiac MRI Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as dependent variable and percentile ranked baseline score, treatment, visi
Slide 19: Final Data Efficacy Analysis: All Cardiac MRI Measures
19 HOPE - 2 Final Data Efficacy Analysis Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as dependent variable and percentile ranked baseline score, treatment, visit, treatment - by - visit interaction, PUL entry - item score at randomizat
Slide 20: Final Data Efficacy Analysis: Creatine Kinase MB
20 HOPE - 2 Final Data Efficacy Analysis Creatine Kinase MB / Total Creatine Kinase % Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as dependent variable and percentile ranked baseline score, treatment, visit, treatment - by - visit inte
Slide 21: Conclusions
21 Conclusions Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as dependent variable and percentile ranked baseline score, treatment, visit, treatment - by - visit interaction, PUL entry - item score at randomization, and site as model eff
Slide 22: Program Update: Dr. Linda Marban
Program Update: Dr. Linda Marban 22
Slide 23: DMD Program Moving Forward
Developing Transformative Therapies from Bench to Bedside Capricor Therapeutics, Inc. DMD Program Moving Forward • HOPE - 3 : Phase III, randomized, double - blind, placebo - controlled trial • Enrollment: 65 - 75 patients • Number of sites: Est. 20 • Targeting: Non - Ambulant DMD patients • Primary
Slide 24: Acknowledgments
Acknowledgments ▪ All patients and their families who participated in the HOPE - 2 Study ▪ HOPE - 2 Investigators ▪ Craig McDonald, MD (UC Davis) ▪ Cuixia Tian, MD (CCHMC) ▪ Russell Butterfield, MD (University of Utah) ▪ Richard Finkel, MD (Nemours Children’s Hospital) ▪ Joanne Janas, MD (Children’s
Slide 25: Thank you - Question and Answer
Thank you Question and Answer 25