Slide 1: Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases
For personal use only Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases Financial Year Ended June 30, 2020 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 |3
Slide 4: Allogeneic Cellular Medicines for Inflammatory Diseases
Allogeneic Cellular Medicines for Inflammatory Diseases For personal use only Innovative Technology Lead Product Pipeline of Phase 3 Platform Candidate Product Candidates Allogeneic mesenchymal precursor RYONCILTM (remestemcel-L) BLA Lifecycle expansion of cells (MPCs) and their progeny, filed
Slide 5: Platform Technology – Mechanism of Action
Platform Technology – Mechanism of Action Our cellular therapies are activated by multiple inflammatory cytokines through surface receptors, resulting in For personal use only orchestration of an anti-inflammatory cascade IL-10 Breg TH1 Mesenchymal Proliferation Cytokine secretion Proliferation Prec
Slide 6: Pipeline of Phase 3 Product Candidates
Pipeline of Phase 3 Product Candidates For personal use only MESOBLAST PRODUCT THERAPEUTIC PHASE 1/2 PHASE 3 REGISTRATION COMMERCIAL COMMERCIAL CANDIDATE AREA RIGHTS PARTNERS RYONCILTM Acute GVHD - Pediatric (Remestemcel-L) Global * ex-Japan Acute GVHD - Adult Pediatric & adult systemic Chronic GVHD
Slide 7: Commercial Scale Manufacturing Capability
Commercial Scale Manufacturing Capability Manufacturing Remestemcel-L For personal use only Scalable allogeneic “off-the-shelf” cellular platforms Manufacturing meets stringent criteria of international regulatory agencies Robust quality assurance processes ensure final product with batch-to-b
Slide 8: 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 Over 1,100 patents and patent applications (82 patent families) across all major jurisdictions Therapeutic Areas Covers compo
Slide 9: Recent Highlights: Remestemcel-L for Children
Recent Highlights: Remestemcel-L for Children For personal use only Children with Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD) FDA Oncologic Drugs Advisory Committee (ODAC) of the FDA voted overwhelmingly (9-1*) in favor that the available data support the efficacy of RYONCIL in p
Slide 10: Recent Highlights: Remestemcel-L for Adults
Recent Highlights: Remestemcel-L for Adults For personal use only Adults With Acute Respiratory Distress Syndrome (ARDS) due to COVID-19 FDA cleared IND application to treat patients with COVID-19 ARDS, providing a pathway for use under both EAP and in a randomized controlled trial Under emergen
Slide 11: Financials
For personal use only Financials
Slide 12: Financial Highlights
Financial Highlights For personal use only Full Year FY2020 Compared to Full Year FY2019 92% increase in total revenue to US$32.2m 127% increase in milestone revenues from strategic partnerships to US$25.0m 32% growth in commercialization revenue from sales of TEMCELL to US$6.6m 13% (US$11.9
Slide 13: Continued Growth in Revenues from Sales of TEMCELL in Japan
Continued Growth in Revenues from Sales of TEMCELL in Japan for SR-aGVHD For personal use only TEMCELL® HS Inj.1 ANNUAL REVENUE FROM TEMCELL ROYALTIES IN JAPAN ACUTE GRAFT VERSUS HOST DISEASE + OTHER INDICATIONS 7.0 JCR Pharmaceuticals has exclusive rights to Mesoblast’s 6.6 MSC technology for acu
Slide 14: Substantial Increase in Revenues and Reduced Loss After Tax
Substantial Increase in Revenues and Reduced Loss After Tax For personal use only June 30, June 30, Profit and Loss for the full year ending (US$m) 2020 2019 Commercialization revenue 6.6 5.0 Milestone revenue 25.0 11.0 Interest revenue 0.5 0.7 Total Revenue 32.2 16.7 Research and development (56.2)
Slide 15: Strengthened Balance Sheet
Strengthened Balance Sheet For personal use only At June 30, 2020, cash on hand was US$129.3 million Mesoblast completed a US$90 million capital raise from global institutional investors in May 2020 Up to an additional US$67.5 million may be available through existing financing facilities and
Slide 16: RYONCIL (remestemcel-L): Acute Graft Versus Host Disease
For personal use only RYONCIL (remestemcel-L): Acute Graft Versus Host Disease
Slide 17: 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 by BMT Conditioning & Cytokine Storm Organ Damage Conditioning Activation of CD4 regimen, Tissue Mac
Slide 18: 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 US annually1 Despite prophylaxis, ~50% will develop aGVHD2 First-line treatment is cort
Slide 19: Immunomodulatory Activities of Remestemcel-L in Response to Inflammation
Immunomodulatory Activities of Remestemcel-L in Response to Inflammation For personal use only | 19
Slide 20: 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 21: RYONCIL: Anticipated FDA Approval in 2020
RYONCIL: Anticipated FDA Approval in 2020 For personal use only Results from three studies using RYONCIL in children and adults with SR-aGVHD support the FDA BLA filing − RYONCIL was used as salvage therapy in an expanded access program in 241 children with SR-aGVHD (80% Grade C/D) who failed inst
Slide 22: RYONCIL: U.S. Regulatory and Commercial Strategy
RYONCIL: U.S. Regulatory and Commercial Strategy For personal use only FDA action date set for September 30, 2020 Commercialization strategy in place for product launch Ramp-up for inventory build Building out efficient, targeted sales force - 15 centers account for ~50% of patients Label
Slide 23: Remestemcel-L: Lifecycle Extension Strategy
Remestemcel-L: Lifecycle Extension Strategy For personal use only Post-marketing Study in Adults with SR-aGVHD Utilize remestemcel-L manufactured using optimized process Advisory Board of GVHD experts convened August 2020 Planning underway for randomized controlled trial of remestemcel-L vs st
Slide 24: Remestemcel-L: Lifecycle Extension Strategy
Remestemcel-L: Lifecycle Extension Strategy For personal use only US/Ex-US parallel launch adult SR-aGVHD US launch COVID-19 ARDS / MIS-C Label extensions Pediatric - Influenza ARDS SR-aGVHD Phase - Bacterial ARDS 3 positive results - Chronic GVHD Ex-US launch - Crohn’s COVID-19 ARDS & - HIE Product
Slide 25: Remestemcel-L: Potential New Treatment Paradigm in Adults & Children with COVID-19
For personal use only Remestemcel-L: Potential New Treatment Paradigm in Adults & Children with COVID-19
Slide 26: Overview – Remestemcel-L for ARDS due to COVID-19
Overview – Remestemcel-L for ARDS due to COVID-19 For personal use only COVID-19 is a respiratory virus with a high mortality due to a severe inflammatory condition of the lungs called acute respiratory disease syndrome (ARDS) ARDS is caused by cytokine storm in lungs of patients infected with C
Slide 27: 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) Alveolar Sloughing of bronchial epithelium A major area of unmet medical need airspace Inactivated surfa
Slide 28: Promising Pilot Data in Ventilator-Dependent Patients with COVID-19 ARDS
Promising Pilot Data in Ventilator-Dependent Patients with COVID-19 ARDS For personal use only Compassionate Use Data from Emergency IND 12 patients with moderate or severe ARDS received two infusions of remestemcel-L at Mt. Sinai Hospital in New York City Nine patients (75%) successfully came o
Slide 29: Phase 3 Trial of Patients with ARDS due to COVID-19
Phase 3 Trial of 300 Patients with ARDS due to COVID-19 For personal use only Multi-center, randomized, controlled, blinded study to assess safety and efficacy of remestemcel-L plus standard of care (SOC) versus placebo plus SOC in subjects with moderate/severe ARDS on ventilator due to COVID-19
Slide 30: Key Milestones for Remestemcel-L in COVID-19 ARDS
Key Milestones for Remestemcel-L in COVID-19 ARDS For personal use only Interim analyses planned which could result in stopping the trial early for efficacy or futility First interim analysis is due early September after 30% of patients reach the primary endpoint Seek expedited regulatory appr
Slide 31: Remestemcel-L for Children With COVID-19
Remestemcel-L for Children With COVID-19 For personal use only Children hospitalized with COVID-19 develop both ARDS (22%) and a life-threatening inflammation called Multisystem inflammatory Syndrome (MIS-C) which involves multiple critical organs and their vasculature In approximately 50% of ca
Slide 32: Update on Other Phase 3 Product Candidates
For personal use only Update on Other Phase 3 Product Candidates - Heart Failure - Chronic Low Back Pain
Slide 33: Partnerships and License Agreements
Partnerships and License Agreements Partnerships and License Agreements Phase 3 Product Candidates for Heart Failure and Chronic Low Back Pain Product candidates For personal use only MPC-06-ID CHRONIC LOW BACK PAIN - DEGENERATIVE DISC DISEASE Strategic partnership to develop and commercialize MPC
Slide 34: REVASCOR for Advanced and End-Stage Heart Failure
REVASCOR® for Advanced and End-Stage Heart Failure For personal use only In December 2019, the Phase 3 trial in advanced heart failure surpassed the number of primary endpoint events required for trial completion − Final study visits for all surviving patients have been completed − Ongoing quality
Slide 35: MPC-06-ID for Chronic Low Back Pain
MPC-06-ID for Chronic Low Back Pain For personal use only Phase 3 trial of MPC-06-ID for chronic low back pain in 404 patients: − Final study visits for all patients have been completed − Ongoing quality review of all data is being completed at the study sites − Data readout expected during Q4 CY2
Slide 36: Major Operational Milestones for the Next Months
Major Operational Milestones for the Next 12 Months For personal use only Remestemcel-L for SR-aGVHD RYONCIL Priority Review for pediatric SR-aGVHD underway with PDUFA date set for September 30, 2020 If approved, US launch of RYONCIL planned for 2020 Commence trial in adults with SR-aGVHD to e
Slide 37: Thank You
For personal use only Thank You