Slide 1: Financial Results and Operational Update for the Half Year Ended December 31, 2024
mesoblast For personal use only Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases Financial Results and Operational Update for the Half Year Ended December 31, 2024 February 2025 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
Mesoblast is committed to bringing to market For personal use only innovative off-the-shelf allogeneic cellular medicines to treat serious and life-threatening inflammatory illnesses Our Mission
Slide 4: Global leader in allogeneic cellular medicines for inflammatory diseases
Global leader in allogeneic cellular medicines for inflammatory diseases Global ✓ World leaderMarkets in developing allogeneic (off-the- shelf) cellular medicines for the treatment of For personal use only severe and life-threatening inflammatory conditions ONE product FDA ✓ Locations in Australia,
Slide 5: 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 For personal use only Inflammation IL-10 B
Slide 6: Mesoblast allogeneic Mesenchymal Precursor / Stromal Cell portfolio
Mesoblast allogeneic Mesenchymal Precursor / Stromal Cell portfolio Product Indication Phase 2 Phase 3 Regulatory Filing Approved For personal use only Pediatric RYONCIL® SR-aGvHD remestemcel-L Adult SR-aGvHD RYONCIL® IBD / remestemcel-L Crohn’s Pediatric HLHS REVASCOR® Adult HFrEF rexlemestrocel-L
Slide 7: Financial Results for the Period Ended December 31, 2024
For personal use only Financial Results for the Period Ended December 31, 2024 Manufacturing Remestemcel-L 7 © Lonza, reproduced with permission mesoblast
Slide 8: Financial results
Financial results Cash balance at December 31, 2024 was US$38 million (A$61 million) with pro-forma cash of approximately US$200 million (A$322 million) after successfully completing a global private For personal use only placement primarily to existing major US, UK, and Australian shareholders whic
Slide 9: BLA approval resulting in non-cash balance sheet adjustments, including inventory value write-up
BLA approval resulting in non-cash balance sheet adjustments, including inventory value write-up R&D Expenditure expenses were $20.6m in H1 FY2025, of which $8.2m was due to non-cash share-based Dec 31, Dec 31, P&L for the half year ended (US$m) 2024 2023 payments (SBPs) primarily for STI in lieu of
Slide 10: First mesenchymal stromal cell (MSC) therapy approved by FDA
For personal use only First mesenchymal stromal cell (MSC) therapy approved by FDA 10 mesoblast
Slide 11: RYONCIL is the first FDA-approved off-the-shelf cell therapy for SR-aGvHD in children
For personal use only RYONCIL® is the first FDA-approved, off- the-shelf cell therapy for children aged 2 months and older, including adolescents and teenagers, with steroid-refractory acute graft versus host disease (SR- aGvHD), a life-threatening condition with high mortality rates.1 Press Release
Slide 12: Opportunity to address critical unmet need in children with SR-aGVHD
Opportunity to address critical unmet need in children 2 months and older, including adolescents & teenagers with SR-aGVHD >30,000 allogeneic BMTs ~ 1,500 For personal use only Approx. 10,000 allogeneic performed globally BMTs performed in the US (>20K US/EU) annually, Children & adolescents annuall
Slide 13: Ryoncil delivered high overall response rates at Day 28
Ryoncil® delivered high overall response rates at Day 28, a measure that predicts survival in aGvHD MSB-GVHD0011,2 For personal use only Overall Response Rate (n=54) Single-arm, multi-center Phase 3 at Day 28 95% CI 56.4, 82.0 70% SR-GvHD severity3 at baseline in GVHD001: RYONCIL treatment was not d
Slide 14: High cost of treating child who dies from SR-aGvHD
High cost of treating child who dies from SR-aGvHD For personal use only The cost of treating a child who dies of SR-aGVHD within 12 months of transplant is: ➢ Approximately $2.5M ➢ $1.8M higher than for those with SR aGvHD who remain alive1 1. Grabner M et al. Economic burden of acute steroid-refra
Slide 15: Ryoncil long-term survival free from aGvHD
Ryoncil® long-term survival free from aGvHD Year 2 Survival: For personal use only Long-term follow- Children from up of Ryoncil by the Center for International Blood GVHD001 51% and Marrow N=51 Year 4 Survival: Transplant Research (CIBMTR) 88% Grade C/D 49% Only 14% (N=7) died due to aGvHD through
Slide 16: Value of Ryoncil in treating pediatric patients with SR-aGvHD
Value of Ryoncil® in treating pediatric patients with SR-aGvHD For personal use only Total benefits of patient outcomes using Ryoncil® range $3.2m to $4.1m (comprising long-term survival benefit, cost-offset, and cost-savings) Benefits based on health economic models for lifetime ultra rare disease
Slide 17: Ryoncil for treating pediatric patients with SR-aGvHD
Ryoncil® for treating pediatric patients with SR-aGvHD The recommended dosage of For personal use only Ryoncil® for treatment of pediatric SR-aGvHD is 2×106 MSC/kg body weight per intravenous infusion given twice per week for 4 consecutive weeks The wholesale acquisition cost (WAC) of Ryoncil® is $1
Slide 18: MyMesoblast Mandatory Hub
MyMesoblast Mandatory Hub To assist patients with insurance coverage, financial assistance, and access programs, ensuring that no patient is left behind in receiving this potentially life-saving therapy, a comprehensive patient services hub has been established For personal use only START Patient Jo
Slide 19: Availability of Ryoncil for pediatric SR-aGvHD in the U.S. in March 2025
Availability of Ryoncil® for pediatric SR-aGvHD in the U.S. in March 2025 For personal use only Targeted sales force with experience in bone marrow transplant Staged approach centers based on transplant ------- centers with highest 15 highest volume volume and centers account for experience with ~50
Slide 20: Ryoncil: Expanding the Label
For personal use only Ryoncil® Expanding the Label Expanding the Label 20 mesoblast
Slide 21: Inflammatory Bowel Disease (IBD) – Crohn's Disease & Ulcerative Colitis
Inflammatory Bowel Disease (IBD) – Crohn’s Disease (CD) & Ulcerative Colitis (UC) Potential Treatment for Pediatric Patients with IBD Refractory to Biologics - High Unmet Need For personal use only Unmet Need Burden of Illness Pediatric Population IBD remains a major The age of onset is In the U.S.
Slide 22: Label expansion strategy for Ryoncil in biologic-refractory IBD
Label expansion strategy for Ryoncil® in pediatric & adult patients with biologic-refractory IBD For personal use only A recent pilot study in adults demonstrated positive outcomes (rapid mucosal healing and disease remission) in biologic refractory patients receiving remestemcel-L by direct endosco
Slide 23: Label extension strategy for Ryoncil in adult patients with SR-aGvHD
Label extension strategy for Ryoncil® in adult patients with SR-aGvHD Continued unmet need in adults with SR-aGVHD who fail ruxolitinib (>40% of treated patients). For personal use only Survival in these patients who fail ruxolitinib remains a dismal 20-30% by 100 days, a patient population with no
Slide 24: Rexlemestrocel-L Chronic Low Back Pain due to Degenerative Disc Disease
For personal use only Rexlemestrocel-L Chronic Low Back Pain due to Degenerative Disc Disease (CLBP) 24 mesoblast
Slide 25: Chronic low back pain due to degenerative disc disease impacts 7M+
Chronic low back pain due to degenerative disc disease (CLBP) impacts 7M+ Burden of Illness Treatment Options Market Opportunity For personal use only Back pain causes more Minimal treatment options Over 7m patients are disability than any other for patients with chronic low estimated to suffer cond
Slide 26: Patients with CLBP refractory to standard treatment have minimal options
Patients with CLBP refractory to standard treatment have minimal options Rexlemestrocel-L has potential to be first-line treatment for patients with moderate to severe CLBP, refractory to conservative treatment For personal use only Rexlemestrocel-L targeting moderate-to-severe CLBP Conservative Opi
Slide 27: Single injection of rexlemestrocel-L + HA showed more than three years of pain reduction
Phase 3 trial outcomes based on a single injection of rexlemestrocel-L + HA showed 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 significantl
Slide 28: Rexlemestrocel-L Heart Failure
For personal use only Rexlemestrocel-L Heart Failure 28 mesoblast
Slide 29: Adult HFrEF with underlying ischemia: increasing prevalence and high mortality risk
Adult: Heart failure with low ejection fraction (HFrEF) and underlying ischemia is increasing in prevalence and associated with high risk of mortality, heart attacks and strokes For personal use only Heart failure affects 6.5 million patients in the US alone, with prevalence increasing.1 Chronic hea
Slide 30: REVASCOR potential across the spectrum of HFrEF disease
REVASCOR has the potential to improve endothelial dysfunction in HFrEF patients across the spectrum of disease from mild-moderate to end-stage patients with a left ventricular assist device (LVAD) Mesoblast’s Programs for REVASCOR (150 million MPCs) For personal use only DREAM HF-1 Trial LVAD MPC St
Slide 31: MPCs reduce CV mortality by 60-80% in HFrEF with baseline inflammation
MPCs Compared to Controls Reduce CV Mortality by 60-80% in Patients with HFrEF and Baseline Inflammation (hsCRP >2mg/ml or IL-6 >3.4pg/ml) For personal use only Perin EC, Borow KM, Henry TD, et al. Mesenchymal precursor cells reduce mortality and major 31 mesoblast morbidity in ischaemic heart failu
Slide 32: Greatest treatment effect on MACE in chronic HFrEF with ischemia plus inflammation
MPCs Compared to Controls had the Greatest Beneficial Treatment Effect on 2-Point MACE and 3-Point MACE in the Sub-group of Chronic HFrEF Patients with Baseline Ischemia plus Inflammation For personal use only Perin EC, et al Mesenchymal precursor cells reduce mortality and major morbidity in ischae
Slide 33: Pathway to accelerated approval for REVASCOR in adults with HFrEF
Pathway to accelerated approval for REVASCOR in adults with HFrEF DREAM-HF Trial over a mean follow-up of 30 months showed significant reduction in 3-Point MACE in ischemic HFrEF patients (n=158). For personal use only LVAD-MPC Study #2, over 12 months of follow-up, showed significant increase in pr
Slide 34: FDA awarded designations to REVASCOR for hypoplastic left heart syndrome
FDA awarded Rare Pediatric Disease Designation, RMAT and Orphan Drug designation to REVASCOR for hypoplastic left heart syndrome For personal use only FDA granted REVASCOR, Rare Pediatric Disease Designation (RPDD), Regenerative Medicine Advanced Therapy (RMAT) designation, and Orphan Drug Designati
Slide 35: Key Corporate Milestones
For personal use only Key Corporate Milestones 35 mesoblast
Slide 36: Expected advances of multiple product pipeline toward FDA approvals
Mesoblast expects to substantially advance its multiple product pipeline toward FDA approvals over the next six to twelve months Program Key Objectives For personal use only RYONCIL 1 Ryoncil® available for use in SR-aGvHD at U.S. hospitals this quarter Pediatric & Adult Inflammatory Diseases Studie
Slide 37: Thank You
mesoblast For personal use only Thank You 37 mesoblast