Mesoblast Limited · ASX:MSB
Mesoblast — Q3 FY2021 Earnings Presentation
40 slides from Mesoblast's earnings presentation, Jun 3, 2021
Slide 1: Operational Highlights & Financial Results for the Period Ended March 31, 2021
For personal use only mesoblast the regenerative medicine company Operational Highlights & Financial Results for the Period Ended March 31, 2021 JUNE 2021 ASX: MSB; Nasdaq: MESO
Slide 2: Cautionary Note Regarding Forward-Looking Statements
For personal use only CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This presentation includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, le
Slide 3: Our Mission
For personal use only Our Mission Mesoblast is committed to bringing to market innovative cellular medicines to treat serious and life-threatening illnesses
Slide 4: Pipeline
Pipeline For personal use only THERAPEUTIC COMMERCIAL PARTNER PLATFORM AREA PHASE 1/2 PHASE 3 REGISTRATION PARTNERS RIGHTS Acute GVHD - Pediatric * Japan Pediatric & adult Acute GVHD - Adult Remestemcel-L systemic (RYONCILTM) inflammatory Acute Respiratory Distress Syndrome # Global diseases COVID-1
Slide 5: Platform Technology – Mechanism of Action
Platform Technology – Mechanism of Action For personal use only Our mesenchymal precursor/stromal cells respond to and are activated by multiple inflammatory cytokines through surface receptors, resulting in orchestration of an anti-inflammatory cascade IL-10 Breg TH1 Proliferation Mesenchymal Cytok
Slide 6: Global IP Estate Provides Substantial Competitive Advantage
Global IP Estate Provides Substantial Competitive Advantage For personal use only ▪ Extensive patent portfolio with protection extending through 2040 in all major markets Therapeutic Areas Core commercial and ▪ Over 1,100 patents and patent applications (82 patent families) across non-core indicatio
Slide 7: Commercial-scale Manufacturing Capabilities
Commercial-scale Manufacturing Capabilities For personal use only ▪ Scalable allogeneic “off-the-shelf” cellular platforms ▪ Manufacturing meets stringent criteria of international regulatory Manufacturing Remestemcel-L agencies ▪ Robust quality assurance processes ensure final product with batch-to
Slide 8: Financial Results
For personal use only Financial Results
Slide 9: Strengthened Balance Sheet After Recent Capital Raise
Strengthened Balance Sheet After Recent Capital Raise For personal use only ▪ Cash on hand as at March 31, 2021 is US$158.3m ▪ Private placement completed in March 2021 was led by principals of SurgCenter Development, one of the largest private operators of ambulatory surgical centers in the US spec
Slide 10: Ongoing Investment in R&D and Manufacturing
Ongoing Investment in R&D and Manufacturing For personal use only March 31, March 31, Profit and Loss for the three months ending (US$m) 2021 2020 Commercialization revenue 2.0 2.1 Milestone revenue - 10.0 Other revenue, including Interest (0.1) 0.1 Total Revenue 1.9 12.2 Research and development (1
Slide 11: Remestemcel-L
For personal use only Remestemcel-L - Acute Graft versus Host Disease - Acute Respiratory Distress Syndrome
Slide 12: Acute GVHD: Serious and Fatal Complication of Allogeneic BMT
Acute GVHD: Serious and Fatal Complication of Allogeneic Bone Marrow Transplantation (BMT) For personal use only PHASE 1 PHASE 2 PHASE 3 Host Tissue Damage Immune Cell Activation Inflammation and End Organ by BMT Conditioning & Cytokine Storm Damage Conditioning Activation of Macrophage regimen, Tis
Slide 13: Children with Steroid-Refractory Acute GVHD at High Risk of Treatment Failure and Death
Children with Steroid-Refractory Acute GVHD at High Risk of Treatment Failure and Death For personal use only Extremely high unmet medical need ▪ More than 2,000 allogeneic BMTs in children and adolescents in US1 ▪ Despite prophylaxis, ~50% will develop aGVHD2 ▪ First-line treatment is corticosteroi
Slide 14: Remestemcel-L: Prior Clinical Data in Children with SR-aGVHD
Remestemcel-L: Prior Clinical Data in Children with SR-aGVHD For personal use only Consistent efficacy and safety outcomes in a total of 309 children from three studies: ▪ Remestemcel-L was used as first-line therapy in a randomized controlled Phase 3 trial of 260 patients, with SR- aGVHD, including
Slide 15: Remestemcel-L Improved Dismal Survival in Children with SR-aGVHD
Remestemcel-L Improved Dismal Survival in Children with SR-aGVHD For personal use only 2 Steroid Refractory – Remestemcel-L Steroid Refractory – Best Available Therapies 1. Adapted and redrawn from Figure 2 of MacMillan, M.L. et al. Pediatric acute GVHD: clinical phenotype and response to upfront st
Slide 16: Remestemcel-L: Regulatory & Commercial Update for SR-aGVHD
Remestemcel-L: Regulatory & Commercial Update for SR-aGVHD For personal use only ▪ On August 13, 2020, results from 309 children with SR-aGVHD treated with remestemcel-L were presented to the Oncologic Drugs Advisory Committee (ODAC) of the United States Food and Drug Administration (FDA) ▪ The ODAC
Slide 17: ARDS due to COVID-19, Influenza & Bacterial Infection – Major Unmet Need
ARDS due to COVID-19, Influenza & Bacterial Infection – Major Unmet Need For personal use only Normal alveolus Injured alveolus during the acute phase Acute respiratory distress syndrome (ARDS) ▪ A major area of unmet medical need Sloughing of bronchial epithelium Alveolar airspace ▪ Multiple trigge
Slide 18: Clinical Experience with Remestemcel-L in COVID-19 ARDS
Clinical Experience with Remestemcel-L in COVID-19 ARDS For personal use only Emergency IND in Ventilator-Dependent COVID-19 ARDS ▪ 11 patients (10/11 were < 65 years) with moderate or severe ARDS on ventilators, received two infusions of remestemcel-L 2 million cells/kg within five days at Mt. Sina
Slide 19: Dynamic Changes in the Treatment Regimes and Population Enrolled During the Trial
Dynamic Changes in the Treatment Regimes and Population Enrolled During the Trial For personal use only ▪ Recent experience suggests that mortality benefits in ARDS patients are most likely to be seen when using anti-inflammatory therapy early and prior to mechanical ventilation1 ▪ During the conduc
Slide 20: Meta-Analysis of Case Fatality Rates for COVID-19 Patients on Ventilation
Meta-Analysis of Case Fatality Rates (CFR) for COVID-19 Patients on Invasive Mechanical Ventilation (IMV): Mortality Significantly Increases with Age For personal use only 100% IMV and CFR Stratified by Age Alive Dead Unknown Age n (%, 95% CI) n (%, 95% CI) n (%, 95% CI) 90% 84.40% ≤40* (N=4,145) 1,
Slide 21: Viral ARDS ICU Admissions Are Predominantly Patients < 65 Years of Age
Viral ARDS ICU Admissions Are Predominantly Comprised Of Patients < 65 Years of Age For personal use only Severe Influenza Admissions To ICU Severe Influenza Admissions To ICU In Spain During and Post Pandemic Period In France Over Time2 1. Martin-Loeches et al. Pandemic and post-pandemic Influenza
Slide 22: Greater Mortality in Control Patients Older than 65
Greater Mortality through Day 60 in Control Patients Older than 65, Consistent with other Trials For personal use only Controls Age < 65 vs ≥ 65 (n=110) | 22
Slide 23: Remestemcel-L vs Controls with COVID-19 ARDS: Mortality through 60 Days
Remestemcel-L vs Controls with COVID-19 ARDS: Mortality through 60 Days in Treated Patients For personal use only All Modified Intent to Treat Patients (n=217), REM vs Control | 23
Slide 24: Pre-Specified Mortality Analysis through 60 Days by Age
Remestemcel-L vs Controls: Pre-Specified Mortality Analysis through 60 Days < or ≥ 65 Years Old For personal use only Modified Intent to Treat Patients < 65 years old (n=123) Modified Intent to Treat Patients ≥ 65 years old (n=94) REM vs Control REM vs Control | 24
Slide 25: Remestemcel-L plus Dexamethasone Synergistic in Reducing Mortality
Remestemcel-L plus Dexamethasone Synergistic in Reducing Mortality in Exploratory Population < 65 years old For personal use only All Treated Patients < 65 years old on Dexamethasone (n=73) | 25
Slide 26: Conclusions and Next Steps for Remestemcel-L in ARDS Due to COVID-19
Conclusions and Next Steps for Remestemcel-L in ARDS Due to COVID-19 For personal use only ▪ Remestemcel-L did not significantly reduce overall mortality ▪ Remestemcel-L reduced mortality and increased ventilator-free days through 60 Days in pre-specified patient population < 65 years old ▪ Addition
Slide 27: Rexlemestrocel-L in Cardiac Disease: DREAM HF Phase Trial
For personal use only Rexlemestrocel-L in Cardiac Disease: DREAM HF Phase 3 Trial
Slide 28: Chronic Heart Failure: Rising Incidence & High Mortality
Chronic Heart Failure: Rising Incidence & High Mortality For personal use only ▪ Cardiovascular disease (CVD) remains the leading cause of death in the United States1 ▪ Heart failure affects 6.5 million patients in the US and 26 million patients globally. As populations age, the prevalence is increa
Slide 29: Proposed Mechanism of Action of Intra-Cardiac MPC Administration
Proposed Mechanism of Action of Intra-Cardiac MPC Administration in Treatment of both Heart Failure & Large Vessel Atherosclerosis For personal use only Mesenchymal precursor cells (MPC) key mechanisms of action thought to beneficially Cardiomyocyte Survival impact the heart and the NFkB systemic va
Slide 30: Ischemic MACE: Rexlemestrocel-L Significantly Reduced Incidence of MI & Stroke
Ischemic MACE: Rexlemestrocel-L Significantly Reduced Incidence of MI & Stroke by 60% Relative to Controls (n=537 Patients) For personal use only Absence of Ischemic MACE All Patients (n=537) REX 5% vs Controls 13%, p=0.002 REX Effect Size: ↓60% Days from Day 0 Treatment | 30
Slide 31: 3P-MACE: Rexlemestrocel-L Reduced Composite of Cardiac Death, MI or Stroke
3P-MACE: Rexlemestrocel-L Significantly Reduced Composite of Cardiac Death, MI or Stroke Compared to Controls Across All 537 Patients For personal use only Absence of Cardiac Death or Non-fatal Ischemic MACE All Patients (n=537) REX 20.6% vs Controls 30%, p=0.027 REX effect size: ↓30% Days from Day
Slide 32: 3P-MACE Reduced by 55% in NYHA Class II Patients
3P-MACE: Rexlemestrocel-L Significantly Reduced Composite of Cardiac Death, MI or Stroke by 55% in 206 NYHA Class II Patients For personal use only Absence of Cardiac Death or Non- fatal Ischemic MACE NYHA II (n=206) REX 13% vs Controls 29%, p=0.009 REX effect size: ↓ 55% Days from Day 0 Treatment |
Slide 33: Rexlemestrocel-L for Chronic Heart Failure
Rexlemestrocel-L for Chronic Heart Failure For personal use only Treatment Algorithm in Progressive Heart Failure Progressive Vascular (Endothelial) Dysfunction and Heart Failure Early ▪ ACEI or ARB Mesoblast Target Market ▪ Statins Chronic Heart Failure1 ▪ Beta blockers ▪ Re-vascularization or valv
Slide 34: Rexlemestrocel-L in Chronic Low Back Pain: Phase Trial
For personal use only Rexlemestrocel-L in Chronic Low Back Pain: Phase 3 Trial
Slide 35: Single Injection of Rexlemestrocel-L + HA Results in at Least 2 Years of Pain Reduction
Single Injection of Rexlemestrocel-L + HA in Phase 3 Trial Results in at Least 2 Years of Pain Reduction with Opioid Sparing Activity in Patients with CLBP For personal use only ▪ Achievement of significant and durable reductions in CLBP through 24 months across the entire evaluable study population
Slide 36: LS Mean VAS Low Back Pain Change from Baseline – Entire Study
LS Mean VAS Low Back Pain Change from Baseline VAS of 60.4 - Entire Study (n=391) For personal use only LS Mean of Change from Baseline (95% CI) -10 P = MPC + HA vs. Placebo Absence of Non-fatal Ischemic MACE -15 -20 -25 -30 Month 1 Month 3 Month 6 Month 12 Month 18 Month 24 Visit Treatment Group Re
Slide 37: LS Mean VAS Low Back Pain Change – Duration CLBP < 68 Month Median
LS Mean VAS Low Back Pain Change from Baseline VAS - Duration CLBP < 68 Month Median (n=194) For personal use only P = MPC + HA vs. Placebo -10 Absence of Non-fatal Ischemic MACE LS Mean of Change from Baseline (95% CI) -20 -30 -40 Month 1 Month 3 Month 6 Month 12 Month 18 Month 24 Visit Treatment G
Slide 38: LS Mean VAS Low Back Pain Change – Opioid Users
LS Mean VAS Low Back Pain Change from Baseline VAS - Opioid Users (n=168) For personal use only P = MPC + HA vs. Placebo 0 Absence of Non-fatal Ischemic MACE LS Mean of Change from Baseline (95% CI) -10 -20 -30 Month 1 Month 3 Month 6 Month 12 Month 18 Month 24 Visit Treatment Group Rexlemestrocel-L
Slide 39: Key Initiatives and Upcoming Milestones for the Next Two Quarters
Key initiatives and Upcoming Milestones for the Next Two Quarters For personal use only Remestemcel-L ▪ In the treatment of SR-GVHD in children, Mesoblast plans to discuss with CBER’s review team at the OTAT our approach to address certain outstanding CMC items, including potency assay validation ▪
Slide 40: Mesoblast: the regenerative medicine company
For personal use only mesoblast the regenerative medicine company