Slide 1: Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases
Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases First Quarter Ended September 30, 2019 Nasdaq: MESO ASX: MSB
Slide 2: Cautionary Note Regarding Forward-Looking Statements
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, levels of activity, perf
Slide 3: Our Mission
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 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 Acquired MSC Dual lis
Slide 5: Premier Global Cellular Medicines Company
Premier Global Cellular Medicines Company 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- potential aGVHD product launc
Slide 6: Commercial Scale Manufacturing Capability
Commercial Scale Manufacturing Capability 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 batch-to-batch consistency
Slide 7: Global IP Estate Provides Substantial Competitive Advantage
Global IP Estate Provides Substantial Competitive Advantage ~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 licensing to third part
Slide 8: Commercial and Late-Stage Product Pipeline
Commercial and Late-Stage Product Pipeline PLATFORM PRODUCT THERAPEUTIC AREA APPROVAL COMMERCIAL RIGHTS MARKETED TEMCELL® MSC Acute Graft Versus Host Japan 1st allogeneic regen med approved in Japan (Bone Marrow) HS Inj1 Disease MSC (Adipose) Alofisel®2 Perianal Fistula 1st allogeneic regen med ap
Slide 9: Partnerships and License Agreements
Partnerships and License Agreements 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 for Europe and Latin A
Slide 10: Financials First Quarter FY2020
Financials First Quarter FY2020
Slide 11: Substantial Increase in Revenues
Substantial Increase in Revenues September 30, September 30, September 30, For the quarter ending (US$m) 2019 2018 2017 Upfront/milestone revenue 15.0 10.5 0.5 Commercialization revenue 1.9 1.0 0.6 Interest revenue 0.1 0.1 0.1 Total revenue 17.0 11.6 1.2 Strategic partnerships drive upfront and mi
Slide 12: Loss After Tax Reduced by 72% (US$14.0 million)
Loss After Tax Reduced by 72% (US$14.0 million) September 30, September 30, Profit and Loss for the quarter ending (US$m) 2019 2018 Total Revenue 17.0 11.6 Research and development (12.4) (18.5) Manufacturing (2.7) (4.3) Management & administration (5.4) (5.6) Contingent consideration (0.3) (0.6) Ot
Slide 13: Strong Balance Sheet and 20% Reduction in Operating Net Cash Outflows
Strong Balance Sheet and 20% Reduction in Operating Net Cash Outflows September 30, June 30, As of (US$m) 2019 2019 Cash on Hand 34.5 50.4 ` Pro forma cash on hand 100.0 50.4 Pro forma cash on hand at September 30, 2019 includes a US$15.0 million upfront payment for the strategic partnership with
Slide 14: Operational Highlights First Quarter FY2020
Operational Highlights First Quarter FY2020
Slide 15: Acute Graft Versus Host Disease (aGVHD)
Acute Graft Versus Host Disease (aGVHD) Significant market opportunity for Remestemcel-L 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 with mortality rates as
Slide 16: Remestemcel-L: U.S. Regulatory and Commercial Strategy
Remestemcel-L: U.S. Regulatory and Commercial Strategy US strategy for remestemcel-L informed by TEMCELL sales experience in Japan Rolling BLA submission to FDA Fast Track designation provides eligibility for FDA priority review Commercialization strategy in place for product launch Ramp-u
Slide 17: SR-aGVHD is Associated with Significant Burden of Illness in Children in the U.S.
Remestemcel-L: SR-aGVHD is Associated with Significant Burden of Illness in Children in the U.S.1 Average Average Average Average Additional Pediatric SR Additional Hospitalization Additional Additional All-Cause Cost: aGVHDvs. Outpatient Prescription ~US$500k LOS: 28 days Visits:30 Fills: 7 NoGVHD
Slide 18: Remestemcel-L: Results from Providers/Payers Indicate Near Maximal Rating
Remestemcel-L: Results from Providers/Payers Indicate Near Maximal Rating on Product Attributes1 Median Response Reaction to 0 Tested Target Profile2 6 7 Most Significant Value Drivers for Remestemcel-L Max Rating Product (n=20) Attributes Day 28 overall response rate (especially grade C/D) Day
Slide 19: Remestemcel-L: Life Cycle Strategy
Remestemcel-L: Life Cycle Strategy Mesoblast has over 10 years of experience in hematology-oncology space Remestemcel-L: − Positive Phase 3 Results Q12018 Chronic GVHD − Large US Pediatric GVHD Expanded Access Program (>240 patients) SR-aGVHD pediatric Ex-US launch Label extension 2018 other indicat
Slide 20: Remestemcel-L for Acute GVHD
Remestemcel-L for Acute GVHD 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 remestemcel-L in aGVHD
Slide 21: Common Treatment Pathway in Progressive Heart Failure
Advanced and End-Stage Heart Failure 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 Re-vascularization
Slide 22: Advanced Heart Failure
Advanced Heart Failure Revascor – Commercialopportunity ~ 8 million patients with chronic heart failure by 2030 in US alone1 Burden of 17-45% globally die within 1 year of hospital admission1 Illness Majority of advanced heart failure patients die within 5 years1 Despite recent advances in n
Slide 23: Advanced Heart Failure: Revascor Phase 3 Trial Fully Enrolled
Advanced Heart Failure Revascor - Phase 3 trial fully enrolled Trial design is 1:1 randomized, controlled, double blinded; conducted over 55 sites across North America using 150 million cell dose vs control Events-driven Phase 3 trial completed enrollment of 566 patients in February 2019 Prima
Slide 24: Revascor for Advanced Heart Failure
Revascor for Advanced Heart Failure Key milestones • Full accrual of primary endpoints events in the Phase 3 trial of Revascor for advanced heart failure around the end of CY19 • Data read-out for this Phase 3 trial planned in H1 CY20 • Results will be considered pivotal to support regulatory approv
Slide 25: End-Stage Heart Failure
End-Stage Heart Failure Revascor – Commercial opportunity in reducing GI bleeding in patients with LVADs In the US there are ~ 250,000–300,000 patients annually who suffer from Burdenof advanced systolic heart failure (NYHA Class III–IV)1 Illness Despite optimal medical therapy, mortality exceed
Slide 26: End-Stage Heart Failure in LVAD Patients
End-Stage Heart Failure in LVAD Patients Revascor reduced GI bleeding events causing hospitalizations in two randomized trials MPCs prolong time-to-first major GI bleeding event and reduced cumulative major GI bleeding events in two randomized controlled trials in LVAD patients1,2 Pilot Randomized C
Slide 27: Revascor for End-Stage Heart Failure in LVAD Patients
Revascor for End-Stage Heart Failure in LVAD Patients 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 trial of Revascor • In
Slide 28: 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 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 of opioids in this pa
Slide 29: MPC-06-ID – Development Strategy for US & Europe
MPC-06-ID – Development Strategy for US & Europe 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 commercial manufacturing
Slide 30: Key Terms of the Strategic Partnership with Grünenthal
Key Terms of the Strategic Partnership with Grünenthal 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 receive Up to US$150
Slide 31: Transaction Benefits to Mesoblast
Transaction Benefits to Mesoblast Strong commercial partner 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 (physicians, pharmacists
Slide 32: MPC-06-ID for Chronic Low Back Pain
MPC-06-ID for Chronic Low Back Pain 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 clearance in CY20 from Eur
Slide 33: Questions
Questions