Slide 1: Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases
mesoblast Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases Financial Results and Operational Update for the Half-Year Ended December 31, 2023 February 2024 ASX: MSB; Nasdaq: MESO
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
Slide 4: 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. Single-arm pivotal Phase 3 trial
Slide 5: Global Intellectual Property (IP) Estate
Global Intellectual Property (IP) Estate Provides Substantial Competitive Advantage Extensive patent portfolio with protection extending through 2040 Over 1,100 patents and patent applications (82 patent families) Markets across all major jurisdictions Global coverage including U.S., Europe, China,
Slide 6: Commercial-scale Manufacturing Process and Facilities
Commercial-scale Manufacturing Process and Facilities Scalable allogeneic “off-the-shelf” cellular platforms Manufacturing Remestemcel-L Manufacturing meets stringent criteria of international regulatory agencies Robust quality assurance processes ensure final product with batch-to-batch consistency
Slide 7: Platform Technology – Shared Mechanism of Action Across Our Products
Platform Technology – Shared Mechanism of Action Across Our Products Our mesenchymal precursor/stromal cells respond to and are activated by multiple inflammatory cytokines through surface receptors, resulting in orchestration of an anti-inflammatory cascade Inflammation IL-10 Breg TH1 Proliferation
Slide 8: 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 Pediatric Remestemcel-L SR-aGVHD Adult Remestemcel-L SR-aGVHD Crohn’s Rexlemestrocel-L HFrEF SR-aGVHD = Steroid-Refractory Acute Graft Vers
Slide 9: Clinical Program Milestones - 2024
Clinical Program Milestones - 2024 Target Date Status • Additional potency assay data provided to FDA Q1 CY2024 Achieved RYONCIL Adult & Pediatric • FDA meeting regarding potency assay data for the pediatric BLA Q1 CY2024 Scheduled SR-aGVHD (remestemcel-L) • Completion and submission to FDA of proto
Slide 10: Regulatory Status for RYONCIL in Pediatric Patients with SR-aGVHD
Regulatory Status for RYONCIL in Pediatric Patients with SR-aGVHD FDA Meeting Scheduled for March Mesoblast has an upcoming meeting scheduled for March with the United States Food and Drug Administration (FDA). Mesoblast has provided the agency with new data from a second potency assay for its produ
Slide 11: Pathway to Approval for RYONCIL in Adult Patients with SR-aGVHD
Pathway to Approval for RYONCIL in Adult Patients with SR-aGVHD Survival in adults with SR-aGVHD who have failed at least one additional agent, such as ruxolitinib, remains as low as 20-30% by 100 days, a patient population with no approved therapies.1,2 In contrast, 100-day survival was 67% after R
Slide 12: REVASCOR in Adults with Chronic Heart Failure with Reduced Ejection Fraction
REVASCOR in Adults with Chronic Heart Failure with Reduced Ejection Fraction (HFrEF), Including End-Stage Patients with a Left Ventricular Assist Device (LVAD) FDA Meeting Regarding Regulatory Path to Approval REVASCOR has shown the potential to reduce major adverse cardiac events (MACE) such as hea
Slide 13: Pediatric Congenital Heart Disease - Hypoplastic Left Heart Syndrome (HLHS)
Pediatric Congenital Heart Disease - Hypoplastic Left Heart Syndrome (HLHS) Awarded FDA Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) During the quarter FDA granted Mesoblast’s cardiovascular product, REVASCOR, both RPDD and ODD. This followed submission of results from
Slide 14: Financial Results
Financial Results for the 6 months ended December 31, 2023 Manufacturing Remestemcel-L 14 © Lonza, reproduced with permission mesoblast
Slide 15: Financial Highlights
Financial Highlights At December 31, 2023, cash-on-hand was US$77.6 million (A$113.5 million), after completing Cash Reserves Institutional Placement and Entitlement Offer of A$60.3 million. Strengthened Balance Sheet through delivering on cost containment strategies. Reduction in net cash usage for
Slide 16: Reduction in Manufacturing, R&D and Management Administration; Improved Loss Before Tax
Reduction in Manufacturing, R&D and Management Administration; Improved Loss Before Tax Dec 31, Dec 31, P&L for the six months ended (US$m) 2023 2022 Total Revenue 3.4 3.4 Revenue: Revenue predominately from royalties on sales of TEMCELL® HS Inj.1 sold in Japan by our licensee. Research and developm
Slide 17: Remestemcel-L
Remestemcel-L Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD) 17 mesoblast
Slide 18: Acute Graft Versus Host Disease (aGVHD)
Acute Graft Versus Host Disease (aGVHD) Serious and Fatal Complication of Allogeneic Bone Marrow Transplantation (BMT) PHASE 1 PHASE 2 PHASE 3 Host Tissue Damage Immune Cell Activation Inflammation and End Organ by BMT Conditioning & Cytokine Storm Damage Conditioning regimen, Activation of CD4 & CD
Slide 19: 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 Corticosteroids are first-line Acute GVHD is a life- More than 30,000 therapy for aGVHD threatening complicat
Slide 20: 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 Remestemcel-L Protocol Remestemcel-L Matched Controls Matched Control Protocol First Line Therapy after Steroids Treatment Setting 1. Pediatric Subset of Pr
Slide 21: Long term Survival in Pediatric Patients with SR-aGVHD Treated with Remestemcel-L
Long term Survival in Pediatric Patients with SR-aGVHD Treated with Remestemcel-L Presented at the 2023 Remestemcel-L Remestemcel-L Tandem has has the Meeting the Potential Potential of ASTCT to Improve to Improve andLong-Term BleakBleak CIBMTR Long-Term Survival Survival (>2 Years) (>2 Years) in in
Slide 22: 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 (Remestemcel-L data from the Center for International Blood and Marrow Transplant Research (CIBMTR) dbase) Study GVHD001 MacMillan et al1
Slide 23: Regulatory Status for RYONCIL in Pediatric Patients with SR-aGVHD
Regulatory Status for RYONCIL in Pediatric Patients with SR-aGVHD FDA Meeting Scheduled for March Mesoblast has an upcoming meeting scheduled for March with the United States Food and Drug Administration (FDA). Mesoblast has provided the agency with new data from a second potency assay for its produ
Slide 24: RYONCIL for Adults with SR-aGVHD
RYONCIL for Adults with SR-aGVHD Commercial strategy is to progress to adults who have failed steroids and a first-line agent, including ruxolitinib. Market opportunity approximately five times larger than pediatric. Approximately 45% of ruxolitinib patients are non-responders.1 Survival in adults w
Slide 25: Rexlemestrocel-L: Chronic Heart Failure Reduced Ejection Fraction (HFrEF)
Rexlemestrocel-L Chronic Heart Failure Reduced Ejection Fraction (HFrEF) 25 mesoblast
Slide 26: Rexlemestrocel-L / HFrEF – Program Summary
Rexlemestrocel-L / HFrEF – Program Summary Defining the Regulatory Path to FDA Approval Significant Need Promising Data Targeting Inflammation FDA Meeting Cardiovascular disease Recent data from the Effects on LVEF and MACE Mesoblast met with the remains the leading DREAM-HF P3 trial outcomes are en
Slide 27: 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 DREAM HF-1 Trial LVAD MPC Studies 537 Patients 189 Patients Early Guideline Directed Medical
Slide 28: Randomized Trial of Targeted Transendocardial Mesenchymal Precursor Cell Therapy in Patients
ORIGINAL INVESTIGATIONS Randomized Trial of Targeted Transendocardial Mesenchymal Perin EC, Borow KM, Henry TD, et al. Randomized Trial of Targeted Transendocardial Mesenchymal Precursor Cell Therapy in Patients With Heart Failure. Journal of the Precursor Cell Therapy in Patients American College o
Slide 29: REVASCOR As Treatment For Severe Congenital Heart Disease
REVASCOR As Treatment For Severe Congenital Heart Disease Awarded FDA Rare Pediatric Disease Designation and Orphan Drug Designation REVASCOR has multiple mechanisms-of-action that may be beneficial Anatomy of hypoplastic left heart syndrome to children with congenital heart disease including neovas
Slide 30: REVASCOR As Treatment For Severe Congenital Heart Disease
REVASCOR As Treatment For Severe Congenital Heart Disease Awarded FDA Rare Pediatric Disease Designation and Orphan Drug Designation In the HLHS randomized controlled single-center US trial in 19 patients, a single intramyocardial administration of REVASCOR at the time of staged surgery resulted in
Slide 31: Rexlemestrocel-L: Chronic Low Back Pain due to Degenerative Disc Disease (CLBP)
Rexlemestrocel-L Chronic Low Back Pain due to Degenerative Disc Disease (CLBP) 31 mesoblast
Slide 32: 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 Back pain causes more Minimal treatment options Over 7m patients are disability than any oth
Slide 33: Rexlemestrocel-L / CLBP – Program Summary
Rexlemestrocel-L / CLBP – Program Summary 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 FDA appropriate pivotal
Slide 34: 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 35: Phase 3 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 36: Thank You
mesoblast Thank You 36 mesoblast