Mesoblast Limited · ASX:MSB
Mesoblast — ISCT North America 2025
20 slides from Mesoblast's conference presentation, Mar 13, 2025
Slide 1: Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases
mesoblast For personal use only Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases Presentation at ISCT Ryoncil® - The First FDA Approved Mesenchymal Stromal Cell Therapy March 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: First mesenchymal stromal cell (MSC) therapy approved by FDA
For personal use only First mesenchymal stromal cell (MSC) therapy approved by FDA 7 mesoblast
Slide 8: Acute graft versus host disease is a serious and potentially fatal complication of BMT
Acute graft versus host disease (aGvHD) is a serious and potentially fatal complication of allogeneic bone marrow transplantation (BMT) PHASE 1 PHASE 2 PHASE 3 For personal use only Host Tissue Damage Immune Cell Activation Inflammation and End Organ by BMT Conditioning & Cytokine Storm Damage Condi
Slide 9: 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 ~1,500 For personal use only >30,000 allogeneic BMTs Approx. 10,000 allogeneic performed globally Children & adolescents BMTs performed in the US (>20K US/EU) annually, undergo
Slide 10: Ryoncil for treating pediatric patients with SR-aGvHD
Ryoncil® for treating pediatric patients with SR-aGvHD For personal use only The recommended dosage of 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 10 mesoblast
Slide 11: Improvements in manufacturing process give rise to a product with greater potency
Improvements in manufacturing process give rise to - a product with greater potency Potency for Drug Product Lots used in EAP275 For personal use only Prochymal Percent DCB1 process Ryoncil DCB2 process Percent Mean % IL-2Rα Inhibition 100% of Ryoncil lots released meet FDA approved potency release
Slide 12: Improvements in manufacturing process give rise to improved survival in SR-aGvHD
Improvements in manufacturing process give rise to - a product associated with improved survival in SR-aGvHD Day 100 survival for Grade D EAP275 patients (n=106) For personal use only Ryoncil 72% DCB2 process Survival Probability Prochymal 44% DCB1 process Log-rank p value = 0.005 12 Mesoblast data
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 aGvHD1 MSB-GVHD0012,3 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 severity4 at baseline in GVHD001: RYONCIL treatment was not
Slide 14: 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 15: Ryoncil lot potency correlates with reduction in activated T cells
Ryoncil® lot potency, measured by IL-2Rα Inhibition Assay, correlates with reduction in circulating levels of activated T cells in patients with SR-aGVHD: evidence for immunomodulatory mechanism of action (MOA) For personal use only Supporting evidence for Ryoncil immunomodulatory MOA from pharmacod
Slide 16: RYONCIL is the first FDA-approved off-the-shelf cell therapy for SR-aGvHD
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 17: 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 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: Thank You
mesoblast For personal use only Thank You 20 mesoblast