Slide 1: Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases
For personal use only Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases FY Ended June 30, 2019 Nasdaq: MESO ASX: MSB
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: Mesoblast founded in Melbo
Slide 5: Premier Global Cellular Medicines Company
Premier Global Cellular Medicines Company For personal use only Innovative Technology Platform1 Late Stage Pipeline Commercialization Plan Innovative technology targets Initiated filing with US FDA for Building US sales force for some of the most severe disease approval for steroid-refractory
Slide 6: Commercial and Late-Stage Product Pipeline
Commercial and Late-Stage Product Pipeline For personal use only RollingBLA BLA submission to submission FDA underway ongoing |6
Slide 7: Partnerships and License Agreements
Partnerships and License Agreements For personal use only JCR has rights to use our MSC technology to treat acute GVHD in Japan Its product TEMCELL® HS Inj. was the first fully approved allogeneic cellular medicine inJapan Royalties and milestones received in last 12 months exceed US$6.0 milli
Slide 8: Recent Corporate Highlights
Recent Corporate Highlights For personal use only Remestemcel-L for Steroid-Refractory Acute Graft Versus Host Disease Continued growth in revenues from royalties on sales of TEMCELL in Japan for steroid refractory aGVHD. Product adoption and reimbursement seen in the Japan GVHD market for TEMCE
Slide 9: Recent Corporate Highlights (continued)
Recent Corporate Highlights (continued) Revascor for Advanced Heart Failure For personal use only Phase 3 trial in advanced heart failure has completed patient enrollment, with 566 patients randomized to receive Revascor or placebo across 55 centers in North America. The trial’s primary endpoint i
Slide 10: Recent Corporate Highlights (continued)
Recent Corporate Highlights (continued) For personal use only MPC-06-ID for Chronic Low Back Pain Phase 3 trial in chronic low back pain completed enrollment in March 2018 with 404 patients randomized to receive MPC- 06-ID or placebo. All patients have now completed at least 12 months of safety an
Slide 11: Commercial Scale Manufacturing Capability
Commercial Scale Manufacturing Capability For personal use only Scalable allogeneic “off-the-shelf” cellular medicine platform Manufacturing meets stringent criteria set by international regulatory agencies including FDA and EMA Robust quality assurance processes ensure final product with batc
Slide 12: Global IP Estate Provides Substantial Competitive Advantage
Global IP Estate Provides Substantial Competitive Advantage For personal use only ~995 patents and patent applications (68 patent families) across all major jurisdictions Covers composition of matter, manufacturing, and therapeutic applications of mesenchymal lineage cells Diseases Enables lic
Slide 13: Financials FY 2019
For personal use only Financials FY 2019
Slide 14: Revenues from Royalties on Japan Product Sales – 37% Year on Year Growth
Revenues from Royalties on Japan Product Sales – 37% Year on Year Growth US$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 J
Slide 15: Revenues – Continued Growth in Royalties and Substantial Payments from Strategic Partnerships
Revenues – Continued Growth in Royalties and Substantial Payments from Strategic Partnerships For personal use only June 30, June 30, For the year ending (US$m) 2019 2018 Upfront/milestone revenue 11.0 13.3 Commercialization revenue 5.0 3.6 Interest revenue 0.7 0.4 Total revenue 16.7 17.3 Strategi
Slide 16: Increased Investment in Commercial Manufacturing
Increased Investment in Commercial Manufacturing Non-cash Gains in Comparable Period from Revaluation of Tax and Contingent Consideration For personal use only June 30, June 30, Profit and Loss for the year ending (US$m) 2019 2018 Total Revenue 16.7 17.3 Research and development (59.8) (65.9) Manufa
Slide 17: Reduction in Operating Net Cash Outflows for the Year
Reduction in Operating Net Cash Outflows for the Year Due to Increased Payments from Strategic Partnerships For personal use only June 30, June 30, For the year ending (US$m) 2019 2018 Operating net cash outflows (57.8) (75.0) ` Investing net cash outflows (1.0) (1.2) Financing net cash inflows 71.6
Slide 18: Lead Product Candidate: Remestemcel-L for aGVHD
For personal use only Lead Product Candidate Remestemcel-L for aGVHD
Slide 19: Acute Graft Versus Host Disease (aGVHD)
Acute Graft Versus Host Disease (aGVHD) Significant market opportunity forremestemcel-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 wit
Slide 20: 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 For personal use only Average Average Average Average Additional Pediatric SR Additional Hospitalization Additional Additional All-Cause Cost: aGVHDvs. Outpatient Prescription ~US$500k LOS: 28 days Visi
Slide 21: Results from Providers/Payers Indicate Near Maximal Rating on Product Attributes
Remestemcel-L: Results from Providers/Payers Indicate Near Maximal Rating on Product Attributes1 For personal use only 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 (especi
Slide 22: 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 initiated Fast Track designation provides eligibility for FDA priority review Ramp-up for inventory build is
Slide 23: Remestemcel-L: Global GVHD Life Cycle Program
Remestemcel-L: Global GVHD Life Cycle Program Mesoblast has over 10 years of experience in hematology-oncology space For personal use only Remestemcel-L: − Positive Phase 3 Results Q12018 Chronic GVHD − Large US Pediatric GVHD Expanded Access Program (>240 patients) SR-aGVHD pediatric Ex-US launch L
Slide 24: Outlook
For personal use only Outlook
Slide 25: Anticipated FY2020 Milestones
Anticipated FY2020 Milestones For personal use only Remestemcel-L for Steroid-Refractory Acute Graft Versus Host Disease Completion of BLA filing for remestemcel-L in the treatment of steroid refractory aGVHD in children (Q4 CY19) Revascor for Advanced Heart Failure Phase 3 events-driven trial i
Slide 26: Additional Information: Development & Commercialization
For personal use only Additional Information Development & Commercialization
Slide 27: Remestemcel-L: Expanded Access Program (Protocol 275)
Remestemcel-L: Expanded Access Program (Protocol 275) Overall Day 28 Response in 241 Pediatric aGVHD Patients Receiving Remestemcel-L as First-line or Salvage Therapy After Failing Steroids1 For personal use only Population: steroid-refractory 100% aGVHD pediatricpatients 241 pediatric patients un
Slide 28: Remestemcel-L: Expanded Access Program
Remestemcel-L: Expanded Access Program Correlation of Day 28 Overall Response with Day 100 Survival, Using Remestemcel-L as First-line or Salvage Therapy After Failing Steroids and/or Additional Treatments1 For personal use only Remestemcel-L in Children with SR-aGVHD who failed multiple othermodali
Slide 29: Remestemcel-L: Phase 3 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 30: Remestemcel-L: Phase 3 Trial Protocol GVHD001 – Demographics
Remestemcel-L: Phase 3 Trial Protocol GVHD001 – Demographics1 For personal use only Subjects Enrolled 55 Age(years) Mean(SD) 7.8 (5.44) Median (minimum, maximum) 7.6 (0.6, 17.9) Gender Male 35 (63.6%) Female 20 (36.4%) UnderlyingDisease AML 18 (32.7%) ALL 12 (21.8%) Anemia 5 (9.1%) CML 4 (7.3%) Sick
Slide 31: Protocol GVHD001 - Disease characteristics reflect aGVHD severity
Remestemcel-L: Phase 3 Trial Protocol GVHD001 - Disease characteristics reflect aGVHDseverity1 For personal use only GVHD Grade at Baseline 89% of subjects had Grade C/D disease at baseline Grade B Grade C 47% of subjects had Grade D disease at baseline GradeD Baseline Organ Involvement 26% of
Slide 32: 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 |32
Slide 33: Protocol GVHD001 - Primary efficacy overall response at Day 28 was 69%
Remestemcel-L: Phase 3 Trial Protocol GVHD001 - Primary efficacy overall response at Day 28 was 69%, p=0.00031 For personal use only 100% 73% 80% 70% 69% 69% Overall Response rate at Day 28 (29% CR + 40% PR); (95% CI: 55%, Response Rate 60% 81%) 50% p-value calculated from the binomial 40% distr
Slide 34: 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 35: Remestemcel-L: Protocol GVHD001/002 survival
Remestemcel-L: Protocol GVHD001/002 survival1 For personal use only Survival in Day 28 Responders Overall Survival Probability Day 100 - 87%; Day 180 - 79% Days Since First Dose of Remestemcel-L 1. Data on file. |35
Slide 36: 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 Class IV Mesoblast Target Market: Early ACEI orARB Advanced Statins and End-Stage HF patients3 Beta blocker
Slide 37: Advanced Heart Failure: Revascor Commercial Opportunity
Advanced Heart Failure Revascor – Commercialopportunity For personal use only Approx. 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
Slide 38: Revascor: Phase 2 Trial Identified Optimal Therapeutic Dose and Target Population
Revascor: Phase 2 Randomized, Controlled Trial Identified Optimal Therapeutic Dose and Target Patient Population for Phase 3 For personal use only Objectives LVESV Month 6 -Baseline LVEDV Month 6 -Baseline Identify a dose response and an optimal therapeutic dose Identify optimal target populatio
Slide 39: Revascor: Therapeutic Benefit on LV Remodeling in Phase 2 Subjects
Revascor: Therapeutic Benefit on LV Remodeling in Phase 2 Subjects with LVESV >100ml1 For personal use only Placebo 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 defined by ∆ ∆ baseline LVESV>1
Slide 40: Revascor: Single Dose Prevented Any HF-MACE in Phase 2
Revascor: Single Dose Prevented Any HF-MACE for 36 Months in Patients at Highest Risk of Recurrent Events and Death (Those with LVESV>100ml) in Phase 2 For personal use only % HF-MACE Kaplan-Meier Curve over HF-MACE Kaplan-Meier Curve over 36monthsfollowing 36 months following treatment in allpatien
Slide 41: Advanced Heart Failure: Revascor Phase 3 trial fully enrolled
Advanced Heart Failure Revascor - Phase 3 trial fully enrolled For personal use only 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
Slide 42: End-Stage Heart Failure: Commercial opportunity in reducing GI bleeding in LVADs
End-Stage Heart Failure Revascor – Commercial opportunity in reducing GI bleeding in patients with LVADs For personal use only In the US there are approx. 250,000–300,000 patients annually who Burdenof suffer from advanced systolic heart failure (NYHA Class III–IV)1 Illness Despite optimal medic
Slide 43: Revascor: Trials demonstrated reduced GI bleeding events in LVAD patients
End-Stage Heart Failure Revascor –Trials demonstrated reduced GI bleeding events in LVAD patients For personal use only 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 Controll
Slide 44: Revascor: Reduced hospitalization rate from GI bleeding in Phase 2 trial
End-Stage Heart Failure Revascor – Reduced hospitalization rate from GI bleeding in Phase 2 trial For personal use only MPCs Reduce Hospitalization Rate from GI Bleeding by 65% in 159-Patient Phase 2 Trial1 1 Presented at American Heart Association Scientific Sessions 2018 | 44
Slide 45: Chronic Low Back Pain: Market opportunity
Chronic Low Back Pain (CLBP) MPC-06-ID – Market opportunity in CLBP due to disc degeneration For personal use only Back pain causes more disability thanany other condition1 Burden of Illness Inflicts substantial direct and indirect costs on thehealthcaresystem,1 including excessive use of opioid
Slide 46: MPC-06-ID – Post-Hoc Phase 2 results provide target endpoints
Chronic Low Back Pain MPC-06-ID – Post-Hoc Phase 2 results provide target endpoints for Phase 3 trial For personal use only A: Phase 2: Treatment Success Responders1,2 B: Phase 2: Treatment Success Responders1,2 at 12 Months at both 12 & 24 Months 50.0% 50.0% 45.0% 46.6% 45.0% p = 0.032 p = 0.050 40
Slide 47: MPC-06-ID – Ongoing Phase 3 Clinical Trial
Chronic Low Back Pain MPC-06-ID – Ongoing Phase 3 Clinical Trial For personal use only Three-arm study comparing 6-million MPC with or without hyaluronic acid (HA)against saline control Primary efficacy endpoint agreed to with FDA: – Overall Treatment Success Composite at both 12 and 24 months a
Slide 48: For personal use only
For personal use only