Slide 1: Financial Results and Operational Update for the Year Ended June 30, 2023
mesoblast For personal use only Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases Financial Results and Operational Update for the Year Ended June 30, 2023 August 2023 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
Our Mission For personal use only Mesoblast is committed to bringing to market innovative cellular medicines to treat serious and life-threatening illnesses
Slide 4: Late-Stage Clinical Pipeline
Late-Stage Clinical Pipeline Based on the Proprietary Allogeneic Mesenchymal Stromal Cell Platform Product Indication Phase 2 Phase 3 Regulatory Filing Approved For personal use only Pediatric Remestemcel-L SR-aGVHD Adult Remestemcel-L SR-aGVHD Rexlemestrocel-L CLBP SR-aGVHD = Steroid-Refractory Acu
Slide 5: Investment Highlights
Investment Highlights Developing off-the-shelf, allogeneic cellular medicines based on proprietary Novel Allogeneic mesenchymal stromal cell (MSC) technology platforms to enable treatment without the Cell Therapy Platform need for donor matching or immunosuppression For personal use only Lead indica
Slide 6: Regulatory Status for Remestemcel-L in Pediatric Patients with SR-aGVHD
Regulatory Status for Remestemcel-L in Pediatric Patients with SR-aGVHD Type A FDA Meeting Scheduled for mid-September During the six-month BLA review we made substantial progress towards bringing this cutting-edge For personal use only product to market with completion of a comprehensive FDA inspec
Slide 7: Regulatory Status for Remestemcel-L in Patients with SR-aGVHD
Regulatory Status for Remestemcel-L in Patients with SR-aGVHD Generating New Clinical Data in Adults In line with our overall commercial strategy to progress to adult patient populations, which For personal use only make up approximately 5-fold larger numbers than children, Mesoblast intends to cond
Slide 8: Manufacturing Remestemcel-L
For personal use only for the Period Ended June 30, 2023 Manufacturing Remestemcel-L 8 © Lonza, reproduced with permission mesoblast
Slide 9: Financial Highlights for the Year
Financial Highlights for the Year Revenue from royalties were US$7.5 million for the year ended June 30, 2023. On a constant currency basis, royalties on sales of TEMCELL® HS Inj.1 in Japan by our licensee were US$8.12 million for the year Royalty Revenue ended June 30, 2023, compared with US$8.7 mi
Slide 10: Reduction in Expenditure on R&D, Improved Loss Before Tax
Reduction in Expenditure on R&D, Improved Loss Before Tax June 30, June 30, Revenue: Revenue predominately from royalties on sales of For personal use only P&L for the quarter ended (US$m) 2023 2022 TEMCELL® HS Inj.1 sold in Japan by our licensee. Total Revenue 7.5 10.2 Reduction in R&D Expenditure:
Slide 11: Cost Containment Plan for Next Months
Cost Containment Plan for Next 12 Months Reduction in Spend on Operational Activities And Payroll Net operating cash usage in FY2023 was a 37% reduction compared with FY2021 and 4% reduction compared with FY2022 For personal use only Further targeted 23% reduction (US$15 million) from US$63.3 millio
Slide 12: Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD)
For personal use only Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD) 12 mesoblast
Slide 13: Acute Graft Versus Host Disease (aGVHD)
Acute Graft Versus Host Disease (aGVHD) 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 Organ by BMT Conditioning & Cytokine Storm Damage Conditioning regimen,
Slide 14: Remestemcel-L: Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD)
Remestemcel-L: Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD) SR-aGVHD is associated with mortality rates as high as 90% Treatment Options Burden of Illness Market Opportunity For personal use only Corticosteroids are first-line Acute GVHD is a life- More than 30,000 therapy for aGVHD
Slide 15: Remestemcel-L for Children with SR-aGVHD
Remestemcel-L for Children with SR-aGVHD Improved Early Survival Across Three Studies involving more than 300 Treated Children Day 100 Survival For personal use only Remestemcel-L Protocol Remestemcel-L Matched Controls Matched Control Protocol First Line Therapy after Steroids Treatment Setting Ped
Slide 16: Extended Survival Data in Children with SR-aGVHD
Extended Survival Data in Children with SR-aGVHD Remestemcel-L Treatment Resulted in Durable Survival Over 4 Years Survival Outcomes in Pediatric & Adult SR-aGVHD For personal use only (Remestemcel-L data from the Center for International Blood and Marrow Transplant Research (CIBMTR) dbase) Study GV
Slide 17: Remestemcel-L for Adults with SR-aGVHD
Remestemcel-L for Adults with SR-aGVHD Commercial strategy is to progress to adults who have failed steroids and a first-line agent, including ruxolitinib For personal use only Market opportunity approximately five times larger than pediatric Approximately 45% of ruxolitinib patients are non-respond
Slide 18: Chronic Low Back Pain due to Degenerative Disc Disease (CLBP)
For personal use only Chronic Low Back Pain due to Degenerative Disc Disease (CLBP) 18 mesoblast
Slide 19: Chronic Low Back Pain Due to Degenerative Disc Disease (CLBP) Impacts 7M+
Chronic Low Back Pain Due to Degenerative Disc Disease (CLBP) Impacts 7M+ Rexlemestrocel-L represents a potential new paradigm for the treatment of CLBP Burden of Illness Treatment Options Market Opportunity For personal use only Back pain causes more Minimal treatment options Over 7m patients are d
Slide 20: Rexlemestrocel-L / CLBP – Program Summary
Rexlemestrocel-L / CLBP – Program Summary For personal use only Regulatory Alignment Phase 3 Protocol Product Manufacturing Pivotal P3 Trial Gained alignment with FDA has agreed with Product has been RMAT designation for the FDA on the Mesoblast plans for manufactured for use in CLBP received from F
Slide 21: RMAT Designation Granted by FDA for Rexlemestrocel-L in CLBP
Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by FDA for Rexlemestrocel-L in the treatment of CLBP RMAT designation provides all the benefits of Breakthrough and Fast Track designations, including rolling review and eligibility for priority review on filing of a Biologics License
Slide 22: Phase Trial Outcomes based on a Single Injection of Rexlemestrocel-L + HA
Phase 3 Trial Outcomes based on a Single Injection of Rexlemestrocel-L + HA Results in More than Three Years of Pain Reduction Greatest pain reduction was observed in the pre-specified population of subjects with CLBP duration shorter than the baseline study median of 68 months (n=202) with signific
Slide 23: Chronic Heart Failure Reduced Ejection Fraction (HFrEF)
For personal use only Chronic Heart Failure Reduced Ejection Fraction (HFrEF) 23 mesoblast
Slide 24: Rexlemestrocel-L / HFrEF – Program Summary
Rexlemestrocel-L / HFrEF – Program Summary Defining the Regulatory Path to FDA Approval For personal use only Significant Need Promising Data Targeting Inflammation FDA Meeting Cardiovascular disease Recent data from the Effects on LVEF and MACE Mesoblast plans to meet remains the leading DREAM-HF P
Slide 25: Patients Experience Progressive Vascular Dysfunction and Heart Failure
Patients Experience Progressive Vascular Dysfunction and Heart Failure Rexlemestrocel-L has the potential to improve endothelial dysfunction in patients from Class II thru IV Mesoblast’s Development Programs For personal use only DREAM HF-1 Trial LVAD MPC Studies 537 Patients 189 Patients Early Guid
Slide 26: Randomized Trial of Targeted Transendocardial Mesenchymal Precursor Cell Therapy
ORIGINAL INVESTIGATIONS Randomized Trial of Targeted Transendocardial Mesenchymal Emerson C. Perin, MD, PHD,a Kenneth M. Borow, MD,b Timothy D. Henry, MD,c Farrell O. Mendelsohn, MD,d Leslie W. Miller, MD,e Elizabeth Swiggum, MD,f Eric D. Adler, MD,g David H. Chang, MD,h R. David Fish, MD,a Precurso
Slide 27: Rexlemestrocel-L – Two Pivotal Studies in Chronic Heart Failure
Rexlemestrocel-L – Two Pivotal Studies in Chronic Heart Failure (CHF) Mesoblast’s Development Programs Assess the Impact of Intra-cardiac Administration of Rexlemestrocel-L Across the Continuum of Disease from Mild/Moderate to End-stage Severity MPC Study Design LVAD-MPC Study #2 DREAM-HF Trial For
Slide 28: Thank You
mesoblast For personal use only Thank You 28 mesoblast