Slide 1: Corporate Overview
For personal use only Corporate Overview Dr Silviu Itescu, Chief Executive January 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: Corporate History
Corporate History For personal use only Over a decade of scientific, manufacturing, clinical development and corporate development experience targeted at bringing to market allogeneic, off-the-shelf cellular medicines for inflammatory diseases 2004: 2013: 2015: 2017: 2019: 2019: Mesoblast founded in
Slide 5: Premier Global Cellular Medicines Company
Premier Global Cellular Medicines Company For personal use only Innovative Technology Platform1 Late Stage Pipeline Commercialization Innovative technology targets Initiated rolling filing with US Building US sales force for some of the most severe disease FDA for approval for steroid- potenti
Slide 6: Commercial Scale Manufacturing Capability
Commercial Scale Manufacturing Capability For personal use only Scalable allogeneic “off-the-shelf” cellular medicine platform Manufacturing meets stringent criteria set by international regulatory agencies including FDA and EMA Robust quality assurance processes ensure final product with batc
Slide 7: Global IP Estate Provides Substantial Competitive Advantage
Global IP Estate Provides Substantial Competitive Advantage For personal use only ~1,000 patents and patent applications (68 patent families) across all major jurisdictions Covers composition of matter, manufacturing, and therapeutic applications of mesenchymal lineage cells Diseases Enables l
Slide 8: Commercial and Late-Stage Product Pipeline
Commercial and Late-Stage Product Pipeline For personal use only PLATFORM PRODUCT THERAPEUTIC AREA APPROVAL COMMERCIAL RIGHTS MARKETED TEMCELL® Acute Graft Versus Host MSC 1st allogeneic regen med approved in Japan Japan HS Inj1 Disease MSC Alofisel®2 Perianal Fistula 1st allogeneic regen med appr
Slide 9: Partnerships and License Agreements
Partnerships and License Agreements For personal use only Strategic partnership to develop and commercialize MPC-06-ID for chronic low back pain due to degenerative disc disease in patients who have exhausted conservative treatment options Grünenthal will have exclusive commercialization rights
Slide 10: Overview of Lead Product Candidates
For personal use only Overview of Lead Product Candidates
Slide 11: Acute Graft Versus Host Disease (aGVHD)
Acute Graft Versus Host Disease (aGVHD) Significant market opportunity for remestemcel-L For personal use only aGVHD is a life-threatening complication that occurs in ~50% of patients receiving Burden of allogeneic bone marrow transplants (BMTs)1 Illness Steroid-refractory aGVHD is associated wi
Slide 12: Grade C/D GVHD has Significantly Worse Survival than Grade A/B
Grade C/D GVHD has Significantly Worse Survival than Grade A/B For personal use only IBMTR severity grade Glucksberg severity grade | 12
Slide 13: Remestemcel-L: Expanded Access Program (Protocol 275)
Remestemcel-L: Expanded Access Program (Protocol 275) Remestemcel-L used as salvage therapy in children who have failed steroids and other agents in treatment of aGVHD, including the most severe Grade C/D disease For personal use only Overall Response at Day 28 in 241 pediatric aGVHD patients receiv
Slide 14: Remestemcel-L: Expanded Access Program
Remestemcel-L: Expanded Access Program In children receiving remestemcel-L as salvage therapy in aGVHD, including the most severe Grade C/D disease, Day 28 Overall Response associated with significant Day 100 survival benefit For personal use only Survival of pediatric patients treated with remestem
Slide 15: Remestemcel-L: Pediatric Trial (GVHD001)
Remestemcel-L: Phase 3 Pediatric Trial (GVHD001) - First-line therapy in aGVHD after failing steroids1 For personal use only Multi-center, single-arm, open-label study to evaluate efficacy and safety to day 100 (GVHD001) and from day 100 to day 180(GVHD002) 55 pediatric patients (2 months to 17
Slide 16: Remestemcel-L: Phase 3 Trial Primary Efficacy
Remestemcel-L: Phase 3 Trial Protocol GVHD001 - Primary efficacy overall response at Day 28 was 70%, p=0.00031,2 For personal use only 100% 76% 80% 70% 70% 70% Overall Response rate at Day 28 (30% CR + 41% PR); (95% CI: 56%, Response Rate 60% 82%) 50% p-value calculated from the binomial 40% dis
Slide 17: Remestemcel-L: Protocol GVHD001/002 survival
Remestemcel-L: Protocol GVHD001/002 survival1 In children receiving remestemcel-L as first-line therapy in SR-aGVHD, including Grade C/D disease, Day 28 Overall Response associated with significant Day 100 survival benefit For personal use only Survival in Day 28 Responders Day 100 87% Overall Survi
Slide 18: Remestemcel-L: Phase 3 Trial
Remestemcel-L: Phase 3 Trial1 For personal use only Phase 3 study evaluated remestemcel-L in 55 children to improve overall response rate and survival − 89% of children had grade C/D disease, the most severe form and historically associated with up to 90% mortality2,3 Study successfully met the
Slide 19: Remestemcel-L: Phase 3 Trial compared to MAGIC Database
Remestemcel-L: Phase 3 Trial compared to MAGIC Database Improved Day 28 Overall Response and Day 100 Survival relative to matched controls For personal use only A comparative analysis performed between Mesoblast’s open-label Phase 3 study and contemporaneous controls receiving institutional standa
Slide 20: Remestemcel-L: U.S. Regulatory and Commercial Strategy
Remestemcel-L: U.S. Regulatory and Commercial Strategy For personal use only US strategy for remestemcel-L informed by TEMCELL sales experience in Japan Rolling BLA submission to FDA for treatment of pediatric SR-aGVHD Fast Track designation provides eligibility for FDA priority review Comme
Slide 21: Significant Annual Revenue Growth from Royalties on GVHD Sales in Japan
Significant Annual Revenue Growth from Royalties on GVHD Sales in Japan US$m For personal use only 6.0 5.0 5.0 4.0 3.6 3.0 2.0 1.0 1.5 - 0.4 FY16 FY17 FY18 FY19 37% growth in royalty revenue for the FY2019 year compared to FY2018 from sales of TEMCELL in Japan for SR-aGVHD by Mesoblast licensee JC
Slide 22: Remestemcel-L: Life Cycle Strategy
Remestemcel-L: Life Cycle Strategy Label For personal use only extension Chronic SR-aGVHD GVHD pediatric Ex- US launch Label 2018 Pediatric Phase 3 extension (US) top line data SR-aGVHD Crohns & other adult US/Ex- indications US parallel Product development/ manufacturing launch SR-aGVHD optimizatio
Slide 23: Remestemcel-L for Acute GVHD: Recent Highlights
Remestemcel-L for Acute GVHD For personal use only Recent Highlights Continued growth in revenues from royalties on sales of TEMCELL in Japan for steroid refractory aGVHD Product adoption and reimbursement seen in the Japan GVHD market for TEMCELL informs Mesoblast US commercial strategy for rem
Slide 24: MPC-06-ID: A New Paradigm for Treatment of Chronic Low Back Pain
MPC-06-ID: A New Paradigm for Treatment of Chronic Low Back Pain Due to Degenerative Disc Disease For personal use only Back pain causes more disability than any other condition1 Burden of Inflicts substantial direct and indirect costs on the healthcare Illness system1,2, including excessive use
Slide 25: MPC-06-ID – Development Strategy for US & Europe
MPC-06-ID – Development Strategy for US & Europe For personal use only Phase 3 trial in chronic low back pain completed enrolment in March 2018 with 404 patients randomized to receive MPC-06-ID or placebo Initiate confirmatory Phase 3 trial in Europe in partnership with Grünenthal Complete com
Slide 26: Key Terms of the Strategic Partnership with Grünenthal
Key Terms of the Strategic Partnership with Grünenthal For personal use only Grünenthal has obtained An exclusive license for Europe and Latin America to develop and commercialize MPC-06-ID in the treatment of chronic low back pain due to degenerative disc disease In consideration, Mesoblast will
Slide 27: Transaction Benefits to Mesoblast
Transaction Benefits to Mesoblast Strong commercial partner For personal use only Delivers commercialization, distribution, sales & marketing Field force comprises around 1,600 people across Europe, Latin America & US – overall focus is on pain – visited nearly 300,000 stakeholders in 2018 (ph
Slide 28: MPC-06-ID for Chronic Low Back Pain: Key Milestones
MPC-06-ID for Chronic Low Back Pain For personal use only Key Milestones Last patient last visit at 24-months of follow up in the Phase 3 trial of MPC-06-ID for chronic low back pain H1 CY20, with the primary endpoint being a composite outcome of pain and function at 12 and 24 months Obtain clea
Slide 29: Advanced and End-Stage Heart Failure
Advanced and End-Stage Heart Failure For personal use only Common Treatment Pathway in Progressive Heart Failure1 Class I Progressive Vascular (Endothelial) Dysfunction and Heart Failure ClassIV Mesoblast Target Market: Early ACEI orARB Advanced Statins and End-Stage HF patients3 Beta blockers
Slide 30: Advanced & End-Stage Heart Failure
Advanced & End-Stage Heart Failure Rising incidence of HF1 together with high mortality rates highlight large clinical unmet need For personal use only More than 26 million people worldwide living with heart failure1 Burden of 8.5 million patients in the US alone, expected to be living with hear
Slide 31: Revascor Phase 2: Maximal Therapeutic Benefit on Left Ventricular Volumes
Revascor Phase 2: Maximal Therapeutic Benefit on Left Ventricular Volumes Seen in Subjects with LVESV >100ml1 For personal use only Placebo (PBO) corrected benefit of 150MM cell dose on cardiac volumes and ejection fraction at 6 months was greatest in patients with more advanced heart failure as def
Slide 32: Revascor Phase 2: Single Dose Prevented HF-MACE for 36 Months
Revascor Phase 2: A Single Dose Prevented Any Heart Failure-Related Major Adverse Cardiac Event (HF-MACE) for 36 Months in High Risk Patients For personal use only % HF-MACE Kaplan-Meier Curve over HF-MACE Kaplan-Meier Curve over 36 monthsfollowing 36 months following treatment in allpatients1 treat
Slide 33: Advanced Heart Failure: Revascor Phase 3 Trial
Advanced Heart Failure Revascor - Phase 3 trial For personal use only Trial design: 1:1 randomized, controlled, double blinded; conducted over 55 sites across North America using 150 million cell dose vs control Target patient population enriched for those with LVESV>100ml, at highest risk for e
Slide 34: Revascor for Advanced Stage Heart Failure
Revascor for Advanced Stage Heart Failure For personal use only Key milestones Initiated final study visits for all surviving patients in Phase 3 trial of Revascor in Advanced Heart Failure, with a target of last patient/last visit at the end of January 2020 Data read-out for this Phase 3 trial
Slide 35: Revascor for End-Stage Heart Failure in LVAD Patients
Revascor for End-Stage Heart Failure in LVAD Patients For personal use only Recent Highlights Mesoblast and the International Center for Health Outcomes Innovation Research (InCHOIR) at the Icahn School of Medicine at Mount Sinai in New York have agreed on the protocol for a confirmatory Phase 3 t
Slide 36: Anticipated Major Milestones – Next 6 Months
Anticipated Major Milestones – Next 12 Months For personal use only Remestemcel-L for Steroid-Refractory Acute Graft Versus Host Disease Final module of rolling Biologic License Application (BLA) submission scheduled for filing with the US Food and Drug Administration (FDA) in January 2020 MPC-06-
Slide 37: Thank you
For personal use only Thank you