Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Technical Presentation (July 2019)
24 slides from Capricor Therapeutics's technical presentation, Jul 15, 2019
Slide 1: HOPE-2 Data Update Conference Call
HOPE-2 Data Update Conference Call July 15, 2019 NASDAQ: CAPR
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 regarding
Slide 3: Call Participants
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. McDona
Slide 4: Our Mission
Our 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
Slide 5: Capricor's Product Pipeline
Capricor’s Product Pipeline Development Phase Candidate Indication Status Preclinical Phase I Phase II Phase III HOPE-2 trial Improvement in skeletal and cardiac muscle function seen Duchenne in randomized clinical trial in CAP-1002 Muscular advanced DMD (HOPE- (allogeneic CDCs) Dystrophy Duchen
Slide 6: CAP-1002: Duchenne Muscular Dystrophy Program
CAP-1002: Duchenne Muscular Dystrophy Program 6
Slide 7: HOPE-2 Clinical Trial Design
HOPE-2 Clinical Trial Design Endpoints CAP-1002 (150M cells) Aim: 84 pts. Safety Efficacy Ages 10+ Over 12 1. Respiratory 1. PUL upper limb strength months Fx 2. Cardiac • 9 Visits Stable steroid regimen 2. Immune MRI • 4 Infusions EF & FVC > 35% Response 3. Myometry Placebo 3. Adverse 4. PFTs Eve
Slide 8: Capricor's CAP-1002 Technology
Capricor’s CAP-1002 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 MOA: cells secrete exosomes Contain non-coding RNAs and proteins
Slide 9: Lack of Dystrophin Predisposes Muscle to Damage
Lack of Dystrophin Predisposes Muscle to Damage — Dystrophin is a structural protein in muscle — Acts both as a cushion and a kind of glue — Without dystrophin, muscles are unable to function properly, suffer progressive damage and eventually die — Much of the muscle injury that occurs in dystrophin
Slide 10: CAP-1002 Targets Multiple Disease Processes in DMD
CAP-1002 Targets Multiple Disease Processes in DMD dystrophin deficiency damage to myocyte loss cell membrane CAP-1002 nitrosactive stress cellular [Ca2+] inflammation mitochondrial inefficiency microvascular ischemia fibrosis 10
Slide 11: CAP-1002: HOPE-2 Interim Analysis Efficacy Results
CAP-1002: HOPE-2 Interim Analysis Efficacy Results 11
Slide 12: HOPE-2 Interim Analysis Breakdown
HOPE-2 Interim Analysis Breakdown • Intent-to-Treat population = 20 subjects • Safety population = 20 subjects • Per Protocol population = 17 subjects • 3 subjects were excluded due to missed infusions • Safety population data available for interim analysis by visit: Visit Placebo CAP-1002 Total Day
Slide 13: HOPE-2 Patient Demographics
HOPE-2 Patient Demographics — CAP-1002 and Placebo groups had similar demographics and baseline characteristics — Mean (SD) age = 14.3 (3.11 years) — Mean (SD) BMI = 22.6 (4.88) kg/m2 — PUL entry scores were either in the 2-3 range or the 4-5 range (stratified) — DMD medical history and disease prog
Slide 14: HOPE-2 Efficacy Endpoints – Interim Analysis
HOPE-2 Efficacy Endpoints – Interim Analysis • Skeletal • PUL 2.0 and PUL 1.2 • Grip Strength • Tip to tip pinch strength • Additional skeletal measures • Pulmonary • Peak Expiratory Flow • Inspiratory Flow Reserve • Forced Vital Capacity • Cardiac • Myocardium mass • Systolic wall thickening • Addi
Slide 15: Mid-Level Performance of the Upper Limb (PUL 2.0)
Mid-Level Performance of the Upper Limb (PUL 2.0) Legend: Colored boxes heights either positive or negative represent mean change from baseline Bars represent ± one standard deviation from the mean Analysis done in Per Protocol Population * Statistically significant p=0.0591 p=0.0389* — A significan
Slide 16: Tip to Tip Pinch Strength
Independent Skeletal Muscle: Grip Strength and Tip to Tip Pinch Strength p=0.5837 p=0.0389* p=0.9057 p=0.0674 * Statistically significant — An improvement in Grip Strength (statistically significant) and in Tip to Tip Pinch Strength was observed at 6 months in CAP-1002 group when compared with place
Slide 17: PFTs: Peak Expiratory Flow (PEF) - % Predicted
PFTs: Peak Expiratory Flow (PEF) - % Predicted Pulmonary Function Testing - PEF % predicted 20 15 Mean Change from Baseline ± SD 10 5 0 -5 PLACEBO -10 CAP-1002 -15 -20 -25 -30 Month 3 Month 6 p=0.1892 Timepoints p=0.6477 — An improvement in Peak Expiratory Flow was observed at 3 and 6 months in CAP-
Slide 18: PFTs: Inspiratory Flow Reserve (IFR) - Absolute
PFTs: Inspiratory Flow Reserve (IFR) - Absolute p=0.0473* p=0.6303 * Statistically significant — A significant improvement in Inspiratory Flow Reserve was observed at 3 months in CAP-1002 group when compared with placebo group 18
Slide 19: Cardiac: Systolic Wall Thickening – Anterior & Lateral
Cardiac: Systolic Wall Thickening – Anterior & Lateral p=0.4238 p=0.2041 — An improvement in anterior and lateral systolic wall thickening was observed at 6 months in CAP-1002 group when compared with placebo group 19
Slide 20: Cardiac: LV Myocardium Mass
Cardiac: LV Myocardium Mass p=0.2990 — An increase in left ventricle myocardium mass was observed at 6 months in CAP-1002 group when compared with placebo group 20
Slide 21: HOPE-2 Interim Analysis Safety Results
HOPE-2 Interim Analysis Safety Results • A total of 56 infusions were performed in HOPE-2 to date • With the exception of two serious adverse events1 in the form of immediate immune reactions, no safety signals were identified • To reduce the risk of future adverse events, Capricor initiated a commo
Slide 22: HOPE-2 Interim Analysis Data Summary
HOPE-2 Interim Analysis Data Summary • Statistical Significance in PUL 2.0 at 6 months (p=0.0389) and strong signal at 3 months (p=0.0591) • Statistical Significance in grip strength (independent skeletal measure) at 6 months (p=0.0389) and a strong trend in tip to tip pinch strength at 6 months. •
Slide 23: Comments by Craig McDonald, M.D.
Comments by Craig McDonald, M.D. 23
Slide 24: Thank you Questions and Answer
Thank you Questions and Answer 24
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