Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — 24th International Annual Congress of the World Muscle Society (October 2019)
17 slides from Capricor Therapeutics's conference presentation, Oct 7, 2019
Slide 1: HOPE-2 Updated Results from the Interim Analysis
A Study of CAP - 1002 in Ambulatory and Non - Ambulatory Patients with Duchenne Muscular Dystrophy [HOPE - 2] Updated Results from the Interim Analysis Presented at the 24 th International Annual Congress of the World Muscle Society 1 October 7, 2019 Conference Call NASDAQ: CAPR
Slide 2: Forward-Looking Statements
Forward - Looking Statements 2 Statements in this press release 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 r
Slide 3: Stages of DMD Disease Progression: Capricor Target Population
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 3 Late Ambulatory Stage (Rapid Functional decline) Delayed & Impaired Acquisition of Mile
Slide 4: HOPE-2 Interim Analysis Breakdown
HOPE - 2 Interim Analysis Breakdown 4 ▪ Intent - to - Treat population = 20 subjects ▪ Safety population = 20 subjects ▪ Per Protocol population for July analysis = 17 subjects ▪ 3 subjects were excluded due to missed or incomplete infusions ▪ Subject 01 - 0009: incomplete Month 3 infusion due to ac
Slide 5: Performance of the Upper Limb (Entry Items) Target Population
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 to mouth using both hands if necessary Ca
Slide 6: Primary Efficacy Endpoint: PUL Assessment to Assess Skeletal Muscle
Primary Efficacy Endpoint: PUL Assessment to Assess Skeletal Muscle Primary Efficacy Endpoint: Performance of the Upper Limb (PUL: v1.2) to Assess Skeletal Muscle 6 PUL v.2.0: • 3 - point response scale - more robust and reproducible than v1.2 • Compensatory strategies allowed to achieve tasks (no
Slide 7: Improvements in PUL 2.0 Observed Mid-Level (Elbow)
7 -2.5 -2.0 -1.5 -1.0 -0.5 0.0 0.5 1.0 1.5 Month 3 Month 6 M e a n C h a n g e f r o m B a s e l i n e ± S D Timepoints Mid-Level Performance of the Upper Limb 2.0 PLACEBO CAP-1002 p=0.2202 p=0.0612 Comparisons treated vs. placebo using mixed model repeated measures ANOVA with covariates Analysis do
Slide 8: Improvements in PUL 2.0 Observed Shoulder + Mid + Distal
-5 -4 -3 -2 -1 0 1 2 3 Month 3 Month 6 M e a n C h a n g e f r o m B a s e l i n e ± S D Timepoints Performance of the Upper Limb 2.0 PLACEBO CAP-1002 Improvements in PUL 2.0 Observed Shoulder + Mid + Distal ‒ Δ 2.0 difference in CAP - 1002 vs. placebo at 6 - months 8 p= 0.0549 p= 0.0299 Comparisons
Slide 9: Improvements in PUL 2.0 Observed Mid + Distal
-3.0 -2.5 -2.0 -1.5 -1.0 -0.5 0.0 0.5 1.0 1.5 2.0 Month 3 Month 6 M e a n C h a n g e f r o m B a s e l i n e ± S D Timepoints Mid + Distal -Level Performance of the Upper Limb 2.0 PLACEBO CAP-1002 Improvements in PUL 2.0 Observed Mid + Distal ‒ Δ 1.6 difference in CAP - 1002 vs. placebo at 6 - mont
Slide 10: Improvements in Grip Strength and Tip to Tip Pinch Strength
-6 -4 -2 0 2 4 6 Month 3 Month 6 M e a n C h a n g e f r o m B a s e l i n e ± S D Timepoints Grip Strength PLACEBO CAP-1002 -4 -2 0 2 4 6 8 Month 3 Month 6 M e a n C h a n g e f r o m B a s e l i n e ± S D Timepoints Tip to Tip Strength PLACEBO CAP-1002 I mprovements in Grip Strength and in Tip to
Slide 11: Improvements in Pulmonary Function Observed
Improvements in Pulmonary Function Observed 11 -30 -25 -20 -15 -10 -5 0 5 10 15 20 Month 3 Month 6 M e a n C h a n g e f r o m B a s e l i n e ± S D Timepoints PEF Percentage Predicted PLACEBO CAP-1002 -0.30 -0.20 -0.10 0.00 0.10 0.20 0.30 0.40 Month 3 Month 6 M e a n C h a n g e f r o m B a s e l i
Slide 12: Cardiac Function as Measured by MRI
-12 -7 -2 3 8 Month 6 M e a n C h a n g e f r o m B a s e l i n e ± S D Timepoint Systolic Wall Thickening -Anterior PLACEBO CAP-1002 -12 -7 -2 3 8 Month 6 M e a n C h a n g e f r o m B a s e l i n e ± S D Timepoint Systolic Wall Thickening -Lateral PLACEBO CAP-1002 Cardiac Function as measured by M
Slide 13: Increase in Left Ventricle Myocardium Mass
Increase in Left Ventricle Myocardium Mass 13 p=0.2990 Comparisons treated vs. placebo using mixed model repeated measures ANOVA with covariates Analysis done in July Per Protocol Population Colored boxes heights, either positive or negative, represent mean change from baseline Bars represent ± one
Slide 14: HOPE-2 Interim Analysis Data Summary
HOPE - 2 Interim Analysis Data Summary 14 ▪ Skeletal: ▪ Mid - Level PUL 2.0 at 6 months (p=0.0612) (ITT) and (p=0.0389) (July PP) ▪ Shoulder + Mid + Distal Level PUL 2.0 at 6 months (p=0.0299) and strong signal at 3 months (p=0.0549) ▪ Mid + Distal Level PUL 2.0 at 6 months (p=0.0177) ▪ Tip to Tip s
Slide 15: Conclusions and Future Directions
Conclusions and Future Directions Conclusions: ▪ First placebo - controlled trial in DMD to use PUL 2.0 for evaluation of efficacy ▪ First placebo - controlled trial showing upper limb functional improvements in non - ambulant DMD patients ▪ Directionally consistent improvements in function, strengt
Slide 16: Acknowledgements
Acknowledgements ▪ 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 Hospital of Colorado) ▪ Matthew Harmelink, MD (Children’s Hospital of Wisconsin) ▪ Arun Varadha
Slide 17: World-Class DMD Advisory Board
World - Class DMD Advisory Board Craig McDonald, M.D. (National PI) University of California at Davis (USA) Michelle Eagle, Ph.D., M.Sc., MCSP Atom International Ltd (UK) Pat Furlong Parent Project Muscular Dystrophy (USA) Kan Hor, M.D. Nationwide Children's Hospital (USA) Oscar Henry Mayer, M.D. Ch