Slide 1: Operational Highlights and Financial Results for the Nine Months and Quarter Ended March 31, 2019
For personal use only Operational Highlights and Financial Results for the Nine Months and Quarter Ended March 31, 2019 May 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 bring to market innovative cellular medicines to treat serious and life-threatening illnesses |3
Slide 4: Premier Global Cellular Medicines Company
Premier Global Cellular Medicines Company For personal use only Innovative Technology Late Stage Pipeline Commercialization Platform1 Upcoming BLA submission for Building focused US sales force Innovative technology targets the steroid-refractory acute GVHD for acute GVHD product launch most s
Slide 5: Recent Corporate Highlights
Recent Corporate Highlights For personal use only Remestemcel-L for Steroid-Refractory Acute Graft Versus Host Disease FDA has agreed to a rolling Biologics Licence Application (BLA) review of remestemcel-L for the treatment of steroid-refractory acute Graft Versus Host Disease (aGVHD) in children
Slide 6: Recent Corporate Highlights (continued)
Recent Corporate Highlights (continued) For personal use only MPC-06-ID for Chronic Lower Back Pain Phase 3 trial in chronic low back pain has completed enrollment with 404 patients randomized to receive MPC-06-ID or placebo. All assessable patients have now completed at least 12-months of safety
Slide 7: Corporate History
Corporate History For personal use only Over a decade of scientific, manufacturing, clinical development and corporate transaction experience targeted at bringing to market, cellular medicines for inflammatory diseases 2004: 2013: 2015: 2017: 2019: Mesoblast is founded in Acquired MSC business from
Slide 8: Partnerships and License Agreements
Partnerships and License Agreements For personal use only JCR has rights to use our MSC technology to treat SR acute GVHD in Japan Its product, TEMCELL ® HS Inj.1, was the first fully approved allogeneic cellular medicine in Japan Royalties and milestones received in last twelve months exceed
Slide 9: Commercial and Late-Stage Product Pipeline
Commercial and Late-Stage Product Pipeline For personal use only |9
Slide 10: Financials
For personal use only Financials | 10
Slide 11: Cash Reserves of US$70.4 million
Cash Reserves of US$70.4 million Significant reduction in operating net cash outflows for the nine months For personal use only March 31, March 31, For the nine months ending (US$m) 2019 2018 Operating net cash outflows (38.7) (54.8) ` Investing net cash outflows (0.2) (0.7) Financing net cash inflo
Slide 12: Revenues – Continued Growth in Royalties and Substantial Milestone Revenues
Revenues – Continued Growth in Royalties and Substantial Milestone Revenues from Corporate Transactions For personal use only March 31, March 31, For the nine months ending (US$m) 2019 2018 Milestone revenue 11.0 12.8 Commercialization revenue 3.3 2.5 Interest revenue 0.5 0.3 Total revenue 14.8 15.6
Slide 13: Increased Loss due to Investment in Manufacturing and Financing
Increased Loss due to Investment in Manufacturing and Financing, and Non-cash Gains in Comparable Period from Revaluation of Tax and Contingent Consideration For personal use only March 31, March 31, Profit and Loss for the nine months ending (US$m) 2019 2018 Total Revenue 14.8 15.6 Research and dev
Slide 14: Operational Highlights
For personal use only Operational Highlights | 14
Slide 15: 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 (BMT)1 Illness Steroid-refractory aGVHD is associated wit
Slide 16: SR-aGVHD Associated with Significant Burden of Illness in Children
Remestemcel-L: SR-aGVHD is Associated with Significant Burden of Illness in Children (US)1 For personal use only Pediatric Average Additional Average Additional Average Additional Average Additional All- Hospitalization LOS: SR aGVHD vs. Outpatient Visits: 30 Prescription Fills: 7 Cause Cost : ~$500
Slide 17: Results from Providers/Payers Qualitative US Market Research
Remestemcel-L: Results from Providers/Payers Qualitative US Market Research1 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 (especially grade C/D) Da
Slide 18: Overview of US Product Reimbursement
Remestemcel-L: Overview of US Product Reimbursement Pricing of Relevant Agents in the Refractory Hematology / Oncology Setting1,2 For personal use only US$50k $100k $200k $375k $475k $500k Branded immune- Combination of Defitelio®2 Yescarta®3 Kymriah®3 suppressants 1 Branded immune- suppressants + E
Slide 19: 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 pediatric patients 241 pediatric patients
Slide 20: Expanded Access Program: Response and Day 100 Survival
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 other modal
Slide 21: 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 22: Phase 3 Trial Protocol GVHD001 – Demographics
Remestemcel-L: Phase 3 Trial Protocol GVHD001 – Demographics1 Subjects Enrolled 55 For personal use only 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%) Underlying Disease AML 18 (32.7%) ALL 12 (21.8%) Anemia 5 (9.1%) CML 4 (7.3%) S
Slide 23: Protocol GVHD001 - Disease Characteristics Reflect aGVHD Severity
Remestemcel-L: Phase 3 Trial Protocol GVHD001 - Disease characteristics reflect aGVHD severity1 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 Grade D Baseline Organ Involvement 26%
Slide 24: Grade C/D GVHD has Significantly Worse Survival than Grade A/B
Grade C/D GVHD has Significantly Worse Survival than Grade A/B1 For personal use only | 24
Slide 25: 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%, 60% 81%) 50% p-value calculated from the binomial 40% distribution, under
Slide 26: Overall Response at Day 28 Predicts Survival Through Six Months
Remestemcel-L: Phase 3 Trial Overall response at Day 28 predicts survival through six months1 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 asso
Slide 27: 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. | 27
Slide 28: Regulatory and Commercial Strategy Overview
Remestemcel-L: Regulatory and Commercial Strategy Overview For personal use only FDA agreed to rolling review of BLA submission Fast Track designation provides eligibility for FDA priority review Ramp-up for inventory build is underway Commercialization strategy in place for product launch
Slide 29: Comprehensive Global GVHD Program
Remestemcel-L: Comprehensive Global GVHD Program Mesoblast has over 10 years of experience in hematology-oncology space For personal use only Remestemcel-L: − Positive Phase 3 Results Q12018 − Large US Pediatric GVHD Expanded Access Program Chronic GVHD (>240 patients) SR-aGVHD Pediatric Ex-US L
Slide 30: 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 or ARB Advanced Statins and End-Stage HF patients3 Beta blocke
Slide 31: Advanced Heart Failure: Revascor Commercial Opportunity
Advanced Heart Failure Revascor – Commercial opportunity 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 32: Revascor: Phase 2 Trial Identified Optimal 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 populat
Slide 33: Revascor: Therapeutic Benefit on LV Remodeling in Phase 2
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 34: Revascor: Single Dose Prevented Any HF-MACE in Phase 2
Revascor: A 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 36 monthsfollowing 36 months following treatment in allpat
Slide 35: 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 150MM cell dose vs control Events-driven Phase 3 trial completed enrollment of 566 patients in Febru
Slide 36: End-Stage Heart Failure: Commercial Opportunity in Reducing GI Bleeding
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 suffer from Burden of advanced systolic heart failure (NYHA Class III–IV)1 Illness Despite optimal medi
Slide 37: 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 Control
Slide 38: 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. | 38
Slide 39: Revascor – Benefits from an Expedited Approval Path under RMAT
End-Stage Heart Failure Revascor – Benefits from an expediated approval path under RMAT For personal use only Revascor Has Received RMAT Designation for Use in End-Stage Heart Failure Patients with LVADs Key benefits of the Regenerative Medicine Advanced Therapies (RMAT) designation include: − Pot
Slide 40: 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 than any other condition1 Burden of Illness Inflicts substantial direct and indirect costs on the healthcaresystem,1 including excessive use of opio
Slide 41: 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% | 41 45.0% 46.6% 45.0% p = 0.032 p = 0.0
Slide 42: 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
Slide 43: Anticipated CY2019 Milestones
Anticipated CY2019 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 Revascor for Advanced and End-Stage Heart Failure Phase 3 trial in advanced