Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Corporate Update (July 2019)
24 slides from Capricor Therapeutics's corporate update, Jul 16, 2019
Slide 1: HOPE-2 Data Update Conference Call July 15, 2019
NASDAQ : CAPR HOPE - 2 Data Update Conference Call July 15, 2019
Slide 2: Forward-Looking Statements
Forward - Looking Statements 2 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 re
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: 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 5 Candidate Indication Development Phase Status Preclinica l Phase I Phase II Phase III CAP - 1002 (allogeneic CDCs) Duchenne Muscular Dystrophy ▪ HOPE - 2 trial ▪ Improvement in skeletal and cardiac muscle function seen in randomized clinical trial in advanced DMD (HOPE
Slide 6: Duchenne Muscular Dystrophy Program
CAP - 1002: Duchenne Muscular Dystrophy Program 6
Slide 7: Clinical Trial Design
Aim: 84 pts. Ages 10+ upper limb strength Stable steroid regimen EF & FVC > 35% CAP - 1002 (150M cells) Placebo Endpoints Safety 1. Respiratory Fx 2. Immune Response 3. Adverse Events 4. Death Efficacy 1. PUL 2. Cardiac MRI 3. Myometry 4. PFTs 5. Biomarkers 6. QOL Over 12 months • 9 Visits • 4 Inf
Slide 8: 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 ▪ MOA: cells secrete exosomes » Contain non - coding RNAs and proteins » Internalized by target ce
Slide 9: Lack of Dystrophin Predisposes Muscle to Damage
Lack of Dystrophin Predisposes Muscle to Damage 9 — 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 dystroph
Slide 10: Targets Multiple Disease Processes in DMD
damage to cell membrane CAP - 1002 Targets Multiple Disease Processes in DMD CAP - 1002 dystrophin deficiency c ellular [Ca 2+ ] myocyte loss nitrosactive stress inflammation mitochondrial inefficiency fibrosis microvascular ischemia 10
Slide 11: Interim Analysis Efficacy Results
CAP - 1002: HOPE - 2 Interim Analysis Efficacy Results 11
Slide 12: Interim Analysis Breakdown
HOPE - 2 Interim Analysis Breakdown 12 • 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 - 100
Slide 13: 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/m 2 — PUL entry scores were either in the 2 - 3 range or the 4 - 5 range (stratified) — DMD medical history and dis
Slide 14: Efficacy Endpoints – Interim Analysis
HOPE - 2 Efficacy Endpoints – Interim Analysis 14 • 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 •
Slide 15: Mid-Level Performance of the Upper Limb (PUL 2.0)
Mid - Level Performance of the Upper Limb (PUL 2.0) 15 p=0.0591 p=0.0389* 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 — A signi
Slide 16: Independent Skeletal Muscle: Grip Strength and Tip to Tip Pinch Strength
Independent Skeletal Muscle: Grip Strength and Tip to Tip Pinch Strength 16 p=0.5837 p=0.0389* * Statistically significant p=0.9057 p=0.0674 — 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
Slide 17: PFTs: Peak Expiratory Flow (PEF) - % Predicted
-30 -25 -20 -15 -10 -5 0 5 10 15 20 Month 3 Month 6 Mean Change from Baseline ± SD Timepoints Pulmonary Function Testing - PEF % predicted PLACEBO CAP-1002 PFTs: Peak Expiratory Flow (PEF) - % Predicted 17 p=0.1892 p=0.6477 — An improvement in Peak Expiratory Flow was observed at 3 and 6 months in C
Slide 18: PFTs: Inspiratory Flow Reserve (IFR) - Absolute
PFTs: Inspiratory Flow Reserve (IFR) - Absolute 18 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
Slide 19: Cardiac: Systolic Wall Thickening – Anterior & Lateral
Cardiac: Systolic Wall Thickening – Anterior & Lateral 19 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
Slide 20: Cardiac: LV Myocardium Mass
Cardiac: LV Myocardium Mass 20 p=0.2990 — An increase in left ventricle myocardium mass was observed at 6 months in CAP - 1002 group when compared with placebo group
Slide 21: Interim Analysis Safety Results
HOPE - 2 Interim Analysis Safety Results 21 • A total of 56 infusions were performed in HOPE - 2 to date • With the exception of two serious adverse events 1 in the form of immediate immune reactions, no safety signals were identified • To reduce the risk of future adverse events, Capricor initiated
Slide 22: Interim Analysis Data Summary
HOPE - 2 Interim Analysis Data Summary 22 • 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 month
Slide 23: Comments by Craig McDonald, M.D.
Comments by Craig McDonald, M.D. 23
Slide 24: Thank you / Questions and Answers
Thank you Questions and Answer 24