Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Corporate Update (June 2022)
17 slides from Capricor Therapeutics's corporate update, Jun 29, 2022
Slide 1: HOPE-2 Open Label Extension (1-Year Data Results)
HOPE - 2 Open Label Extension (1 - Year Data Results) Trial conducted by Capricor National PI: Craig McDonald, M.D. (UC Davis) NASDAQ:CAPR
Slide 2: Forward Looking Statements
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. Forward Looking Statements Statementsinthispresentationregardingtheefficacy,safety,andintendedutilizationofCapricor'sproductcandidates;theinitiation,conduct,size,timingandresultsofdiscoveryeffortsandclinicaltrials;the
Slide 3: Call Participants
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. Call Participants •Linda Marban, Ph.D. –Chief Executive Officer, Capricor Therapeutics, Inc.•Dan Paulson, M.D. –Vice President of Clinical Development, Capricor Therapeutics, Inc.•AJ Bergmann, M.B.A. –Chief Financial
Slide 4: CAP-1002 Cell Therapy: Mechanism of Action
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. CAP-1002 Cell Therapy Does not act by «stemness»the cells do not engraft into host tissueSourced fromtransplant qualifiedheartsCAP-1002: Mechanism of action Cells secrete exosomes•Contain miRNAs, non-coding RNAs and p
Slide 5: CAP-1002 Infusion Protocol is Easy
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. CAP-1002 Infusion Protocol is Easy •I.V. (intravenous) administration every 3 months –~45 minutes Procedure•Simple oral premedication regimen before infusion •Safety profile: no treatment related SAEs reported through
Slide 6: CAP-1002's Eligible DMD Population
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. CAP-1002’s Eligible DMD Population CAP-1002’s EligiblePatientsEstimated that over 50% of DMD patients in U.S. are non-ambulant Late-stage Disease6
Slide 7: Measure: Performance of Upper Limb (PUL Test)
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. Measure: Performance of Upper Limb (PUL Test) *Mayhew et al, 2019; Pane et al, 2018. 7
Slide 8: HOPE-2 Upper Limb and Cardiac Improvements
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. 8HOPE-2 Upper Limb and Cardiac Improvements Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as dependent variable and percentile ranked baseline score, trea
Slide 9: HOPE-2 Open Label Extension Overview
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. HOPE-2 Open Label Extension Overview Open Label Extension Year 1 Overview •n=13•6 original CAP-1002 patients•7 original placebo patients•1 patient withdrew consent Patient Demographics •Mean age: 13 years•All patients
Slide 10: CAP-1002's Impact on Disease Progression
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. -12-10-8-6 -4 -20061218243036 Placebo Baseline End HOPE-2 OLE Baseline OLE Months 12Mean Change PLACEBO: HOPE-2GAP : OFFCAP-1002OLE: ON CAP-1002*Oneyearchangefrombaselineforaphasereferstoasubject'schangeinoneyeardurin
Slide 11: Impact of CAP-1002 on Rate of PUL Decline
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. -12-10-8 -6-4-20061218243036 CAP-1002 Placebo Gap Baseline End HOPE-2 OLE Baseline OLE Months 12Mean Change HOPE-2GAP : OFF CAP-1002OLE: ON CAP-1002Impact of CAP-1002 on Rate of PUL Decline*Oneyearchangefrombaselinefo
Slide 12: Accelerated Decline in PUL when Off-Treatment
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. Accelerated Decline in PUL when Off-TreatmentMean ChangeHOPE-2GAP : OFF CAP-1002OLE: ON CAP-1002 Difference (PUL 2.0)p-valueOLE vs. Placebo Group from HOPE-22.30.015OLE vs. Placebo GAP2.80.006*Oneyearchangefrombaselin
Slide 13: CAP-1002's Impact on Disease Progression
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. CAP-1002’s Impact on Disease Progression -6-5-4-3 -2-100612 CAP-1002 Placebo HOPE-2 at 12 months4 monthsMean ChangeResults published in The Lancet , March 2022. Slowing of disease progression by ~70%HOPE-213
Slide 14: Time is Muscle for DMD Patients
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. Time is Muscle for DMD Patients•CAP-1002: Potential Disease Modifying Benefit•Patients on therapy experience a slower progression of their disease•Urgency to initiate therapy: while treatment benefits are maintained (
Slide 15: CAP-1002 Opportunity for Backbone Therapy Options
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. CAP-1002 Opportunity for “Backbone Therapy” Options We believe Exon SkippingGene therapySteroidsCAP-1002Potential for “backbone therapy” across non-ambulant population with safety demonstrated in over 200 patients Pot
Slide 16: Acknowledgments
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. Acknowledgments 16 Patients and their families who participated in the HOPE-2 and HOPE-2 OLE Studies•Parent Project Muscular Dystrophy•Craig McDonald, M.D. (UC Davis)•Coalition Duchenne•CureDuchenne•The Jett Foundatio
Slide 17: Q&A
Developing Transformative Therapiesfrom Bench to BedsideCapricorTherapeutics, Inc. Q&A17