Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Technical Presentation (May 2020)
34 slides from Capricor Therapeutics's technical presentation, May 13, 2020
Slide 1: HOPE-2 12-month Top-Line Final Study Results
A Study of CAP - 1002 in Ambulatory and Non - Ambulatory Patients with Duchenne Muscular Dystrophy [HOPE - 2] 12 - month Top - Line Final Study Results 1 May 13, 2020 Conference Call NASDAQ: CAPR
Slide 2: Forward-Looking Statements
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 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 discov
Slide 3: Call Participants
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside • Linda Marban, Ph.D. – Chief Executive Officer, Capricor Therapeutics, Inc. • Craig McDonald, M.D., Professor and Chair of the Department of Physical Medicine and Rehabilitation and Director of the Neuromuscular D
Slide 4: Capricor's Regulatory Designations - DMD
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Capricor’s Regulatory Designations - DMD Similar to breakthrough therapy designation: • RMAT provides benefits that include more frequent meetings with FDA to discuss the development plan for the product candidate
Slide 5: CAP-1002 Mechanism of Action
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside CAP - 1002 Mechanism of Action Immunomodulation ‒ Allogeneic cardiosphere - derived cells (CDCs) ‒ MOA: cells secrete exosomes: ‒ Contain miRNAs, other non - coding RNAs and proteins ‒ Internalized by target cells
Slide 6: Immunomodulatory Effects of CAP-1002
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Immunomodulatory Effects of CAP - 1002 CDCs: Cardiosphere - derived cells Macrophages Effector T - cells Proinflammatory Cytokines IFN - γ , TNF α , IL - 1 β , IL - 6, IL - 8, CXCL10, CCL2, CCL3, CCL5 Systemic Infl
Slide 7: Trajectory of CDCs in DMD (Preclinical Data)
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Trajectory of CDCs in DMD (Preclinical Data) • Hypothesis: CDCs to treat cardiomyopathy • Left ventricular ejection fraction markedly improved vs. control – P <0.05 at all timepoints through 12 weeks of follow - up
Slide 8: HOPE-Duchenne (Phase I/II Results)
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE - Duchenne (Phase I/II Results) Reduced Cardiac Scar and Improved PUL R.G. Victor et al., AHA LBCT 2017; M. Taylor et al., submitted *p - values are based on absolute change from baseline INFERIOR WALL IMPROVE
Slide 9: HOPE-2 12-Month Top-Line Final Data
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE - 2 12 - Month Top - Line Final Data Dr. Craig McDonald National PI 9
Slide 10: DMD Progression is Sequential, Non-Linear and Irreversible
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 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 Lat
Slide 11: HOPE-2 Clinical Trial
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE - 2 Clinical Trial • 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 - 10
Slide 12: Performance of the Upper Limb (Entry Items)
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 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 sta
Slide 13: Performance of the Upper Limb (PUL) to Assess Skeletal Muscle
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 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 le
Slide 14: Upper-Limb Skeletal Muscle Measured by PUL
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Upper - Limb Skeletal Muscle Measured by: Performance of the Upper Limb (PUL) 2.0 Performance of the Upper Limb (PUL) 1.2 14
Slide 15: Clinically Meaningful Changes Observed in PUL 2.0
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside PUL 2.0 (full) MONTH 12/ET -5 -4 -3 -2 -1 0 CAP-1002 PLACEBO p=0.0532 M e a n C h a n g e f r o m B a s e l i n e + / - S E M PUL 2.0 (full) MONTH 12/ET C A P - 1 0 0 2 P L A C E B O -8 -6 -4 -2 0 2 4 p=0.0201 (t t
Slide 16: Individual Patient Data: PUL 2.0
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside PUL 2.0: Combined High-, Mid-, and Distal-Level CAP-1002 3 6 9 12 -8 -6 -4 -2 0 2 4 MONTH C h a n g e f r o m b a s e l i n e PUL 2.0: Combined High-, Mid-, and Distal-Level PLACEBO 3 6 9 12 -8 -6 -4 -2 0 2 4 MONTH
Slide 17: Clinically Meaningful Changes in Mid-Level PUL 1.2
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside PUL 1.2 (mid) MONTH 12/ET -8 -6 -4 -2 0 CAP-1002 PLACEBO p=0.0818 M e a n C h a n g e f r o m B a s e l i n e + / - S E M PUL 1.2 (mid) MONTH 12/ET C A P - 1 0 0 2 P L A C E B O -15 -10 -5 0 5 M e a n C h a n g e f
Slide 18: Individual Patient Data: PUL 1.2 (Mid Level)
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside PUL 1.2: Mid-Level Elbow Dimension CAP-1002 3 6 9 12 -15 -10 -5 0 5 10 MONTH C h a n g e f r o m b a s e l i n e PUL 1.2: Mid-Level Elbow Dimension PLACEBO 3 6 9 12 -15 -10 -5 0 5 10 MONTH C h a n g e f r o m b a s
Slide 19: Cardiac Function Measured by MRI
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Cardiac Function Measured by MRI: LV Ejection Fraction (%) LV End - Systolic Volume & LV End - Diastolic Volume CK - MB (% of total CK) 19
Slide 20: Improvements in LV Ejection Fraction
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside LV EF (%) MONTH 12/ET -3 -2 -1 0 1 CAP-1002 PLACEBO p=0.0042 M e a n C h a n g e f r o m B a s e l i n e + / - S E M 20 Improvements in LV Ejection Fraction (%) Observed improvement Comparisons treated vs. placebo
Slide 21: Individual Patient Data: LV Ejection Fraction
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 6 12 -6 -4 -2 0 2 4 Cardiac MRI: LV Ejection Fraction (%) CAP-1002 MONTH C h a n g e f r o m b a s e l i n e 6 12 -6 -4 -2 0 2 4 Cardiac MRI: LV Ejection Fraction (%) PLACEBO MONTH C h a n g e f r o m b a s e l i n
Slide 22: Improvements in LV End-Systolic and End-Diastolic Volume
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside LV ES Volume, Indexed, ml/m 2 MONTH 12/ET -4 -2 0 2 4 CAP-1002 PLACEBO p=0.0122 M e a n C h a n g e f r o m B a s e l i n e + / - S E M Has been used as a surrogate endpoint for approval in adult heart failure 22 L
Slide 23: Improvements in Creatine Kinase MB / Total Creatine Kinase
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 23 Improvements in Creatine Kinase MB / Total Creatine Kinase (%) Observed Month 12 -2 -1 0 1 2 3 4 Creatine Kinase MB/Total Creatine Kinase (%) Change From Baseline Visit % C K - M B CAP-1002 Placebo ✱✱✱ Compariso
Slide 24: Individual Patient Data: CK-MB / Total CK
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 3 6 9 12 -4 -2 0 2 4 Serum Chemistry: Creatine Kinase MB/Total Creatine Kinase (%) CAP-1002 C h a n g e f r o m b a s e l i n e (MONTH) 3 6 9 12 -4 -2 0 2 4 Serum Chemistry: Creatine Kinase MB/Total Creatine Kinase
Slide 25: Respiratory Function Measured by Inspiratory Flow Reserve and PEF
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Respiratory Function Measured by: Inspiratory Flow Reserve Peak Expiratory Flow (% predicted) 25
Slide 26: Improvements Observed in Inspiratory Flow Reserve
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Pulmonary Function Testing: Inspiratory Flow Reserve CAP-1002 3 6 9 12 -0.4 -0.2 0.0 0.2 0.4 0.6 0.8 MONTH C h a n g e f r o m b a s e l i n e Pulmonary Function Testing: Inspiratory Flow Reserve PLACEBO 3 6 9 12 -
Slide 27: Improvements Observed in Peak Expiratory Flow
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Pulmonary Function Testing: PEF (% of Predicted) CAP-1002 3 6 9 12 -60 -40 -20 0 20 40 MONTH C h a n g e f r o m b a s e l i n e Pulmonary Function Testing: PEF (% of Predicted) PLACEBO 3 6 9 12 -60 -40 -20 0 20 40
Slide 28: Safety Summary
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Safety Summary 28
Slide 29: HOPE-2 Safety Results
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE - 2 Safety Results 29 ▪ 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 hypersensitivity reactions, n
Slide 30: HOPE-2 Safety Mitigation Efforts
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE - 2 Safety Mitigation Efforts 30 ▪ To reduce the risk of such future adverse events, Capricor initiated a commonly used pre - medication strategy including intravenous steroids and antihistamines to prevent or
Slide 31: Conclusions and Future Directions
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Conclusions and Future Directions Conclusions: ‒ First placebo - controlled trial showing upper limb functional improvements in non - ambulant DMD patients ‒ Directionally consistent improvements in strength, respi
Slide 32: DMD Advisory Board
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 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) Richard Finkel, M.D. Nemours Children's Hospital (USA) Pa
Slide 33: Acknowledgements
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 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
Slide 34: Thank you - Questions and Answer
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Thank you Questions and Answer 34
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