EX-99.1 2 exhibit991.htm EX-99.1 exhibit991
mesoblast Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases March 2025 ASX: MSB; Nasdaq: MESO Presentation at ISCT Ryoncil® - The First FDA Approved Mesenchymal Stromal Cell Therapy Exhibit 99.1


 
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, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward- looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this presentation are forward-looking statements. Words such as, but not limited to, “believe,” “expect,” “anticipate,” “estimate,” “intend,” “plan,” “targets,” “likely,” “will,” “would,” “could,” and similar expressions or phrases identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and future events , recent changes in regulatory laws, and financial trends that we believe may affect our financial condition, results of operation, business strategy and financial needs. These statements may relate to, but are not limited to: expectations regarding the safety or efficacy of, or potential applications for, Mesoblast's adult stem cell technologies; expectations regarding the strength of Mesoblast's intellectual property, the timeline for Mesoblast's regulatory approval process, and the scalability and efficiency of manufacturing processes; expectations about Mesoblast's ability to grow its business and statements regarding its relationships with current and potential future business partners and future benefits of those relationships; statements concerning Mesoblast's share price or potential market capitalization; and statements concerning Mesoblast's capital requirements and ability to raise future capital, among others. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. You should read this presentation together with our financial statements and the notes related thereto, as well as the risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast's actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, include, without limitation: risks inherent in the development and commercialization of potential products; uncertainty of clinical trial results or regulatory approvals or clearances; government regulation; the need for future capital; dependence upon collaborators; and protection of our intellectual property rights, among others. Accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.


 
Our Mission Mesoblast is committed to bringing to market innovative off-the-shelf allogeneic cellular medicines to treat serious and life-threatening inflammatory illnesses


 
4 m e s o b l a s t Global Markets Global leader in allogeneic cellular medicines for inflammatory diseases ✓World leader in developing allogeneic (off-the- shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions ✓ Locations in Australia, the United States and Singapore ✓ Listed on the ASX (MSB) and NASDAQ (MESO) ✓ Developing product candidates for distinct indications based on its remestemcel-L and rexlemestrocel-L stromal cell technology platforms ✓ Extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets ✓ FDA-inspected commercial scale manufacturing process and facilities ONE product FDA approved more than 1,100 patents & applications Phase 3 trials in TWO major indications


 
5 m e s o b l a s t 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 Effector B cell M2 Breg Treg I n f l a m m a t i o n NK Activation Cytotoxicity Maturation Activation Antigen Presentation Proliferation Antibody production IDO, PGE2 TGF𝛽, M-CSF, CCL2 IL-10 TH17 Proliferation Cytokine secretion Cytotoxicity IL-10 TH1 M1 Immature DC IL-1 IL-6 TNFα IL-10 Mesenchymal Precursor / Stromal Cell Polarize M1 to M2 Source: data on file


 
6 m e s o b l a s t Mesoblast allogeneic Mesenchymal Precursor / Stromal Cell portfolio Product Indication Phase 2 Phase 3 Regulatory Filing Approved RYONCIL® remestemcel-L Pediatric SR-aGvHD Adult SR-aGvHD RYONCIL® remestemcel-L IBD / Crohn’s REVASCOR® rexlemestrocel-L (STRO3+) Pediatric HLHS Adult HFrEF End-stage Adult HFrEF Class II/III Rexlemestrocel-L (STRO3+) CLBP SR-aGvHD = Steroid-Refractory Acute Graft versus Host Disease; IBD = Inflammatory Bowel Disease; HLHS = Hypoplastic Left Heart Syndrome HFrEF = Heart Failure with Reduced Ejection Fraction; CLBP = Chronic Low Back Pain; This chart is figurative and does not purport to show individual trial progress within a clinical program Notes: ▪ JCR Pharmaceuticals Co., Ltd. (JCR), has the right to develop mesenchymal stromal cells (MSCs) in certain fields for the Japanese market, including for the treatment of hematological malignancies, such as Graft vs Host Disease, and for hypoxic ischemic encephalopathy (HIE). ▪ Grünenthal has an exclusive license to develop and commercialize rexlemestrocel-L for chronic low back pain in Europe and Latin America/Caribbean. ▪ Tasly Pharmaceuticals has exclusive rights for rexlemestrocel-L for the treatment or prevention of chronic heart failure in China.


 
7 m e s o b l a s t First mesenchymal stromal cell (MSC) therapy approved by FDA


 
8 m e s o b l a s t Acute graft versus host disease (aGvHD) is a serious and potentially fatal complication of allogeneic bone marrow transplantation (BMT) PHASE 1 Host Tissue Damage by BMT Conditioning PHASE 2 Immune Cell Activation & Cytokine Storm PHASE 3 Inflammation and End Organ Damage Conditioning regimen, chemotherapy, or radiation Tissue Damage Activation of CD4 & CD8 T-cells Cytokine Storm TNF, IL-1, IL-6 IFNγ, IL-2, IL-12, IL-21, IL-22, IL-23 Macrophage input to cytokine storm Macrophage Modified from Blazar et al., Nature Reviews Immunology 12: 443 – 458


 
9 m e s o b l a s t Opportunity to address critical unmet need in children 2 months and older, including adolescents & teenagers with SR-aGVHD ~1,500 Children & adolescents undergo allogeneic BMT in US annually Approx. 10,000 allogeneic BMTs performed in the US annually Acute GvHD occurs in ~50% of patients1 with approx. half failing to respond to steroids SR-aGvHD has high mortality1,4 and significant extended hospital stay costs2 ~50% Incidence of acute GvHD ~375 pts/year with SR-aGvHD 1. Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory acute GVHD: predictors and outcomes. Advances in Hematology. 2. Niederwieser D, Baldomero H, Szer J. (2016) Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey. 3. HRSA Transplant Activity Report, CIBMTR, 2020 4. Axt L, Naumann A, Toennies J (2019) Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation. >30,000 allogeneic BMTs performed globally (>20K US/EU) annually, ~20% pediatric2,3 Corticosteroids are first-line therapy for aGvHD RYONCIL is the only approved therapy for SR-aGvHD in children 2 months and older


 
10 m e s o b l a s t Ryoncil® for treating pediatric patients with SR-aGvHD 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


 
11 m e s o b l a s t Improvements in manufacturing process give rise to - a product with greater potency Prochymal DCB1 process Ryoncil DCB2 process P e rc e n t P e rc e n t Potency for Drug Product Lots used in EAP275 Mean % IL-2Rα Inhibition 100% of Ryoncil lots released meet FDA approved potency release criteria, compared with less than two thirds of Prochymal lots used in adult GvHD trial 280 Mesoblast data unpublished


 
12 m e s o b l a s t Ryoncil DCB2 process Prochymal DCB1 process 72% 44% Log-rank p value = 0.005 Follow-Up Duration (Days) S u rv iv a l P ro b a b ili ty Day 100 survival for Grade D EAP275 patients (n=106) Improvements in manufacturing process give rise to - a product associated with improved survival in SR-aGvHD Mesoblast data unpublished


 
13 m e s o b l a s t Ryoncil® delivered high overall response rates at Day 28, a measure that predicts survival in aGvHD1 Overall Response Rate at Day 28 95% CI 56.4, 82.0 MSB-GVHD0012,3 (n=54) Single-arm, multi-center Phase 3 70% Full Prescribing Information at www.ryoncil.com 1. MacMillan ML, Holtan SG, Rashidi A, et al. “Pediatric acute GVHD: clinical phenotype and response to upfront steroids.” Bone marrow transplantation vol. 55,1 (2020): 165- 171; 2.NCT02336230; 3. Kurtzberg, J. et al. A Phase 3, Single-Arm, Prospective Study of Remestemcel-L, Ex Vivo Culture-Expanded Adult Human Mesenchymal Stromal Cells for the Treatment of Pediatric Patients Who Failed to Respond to Steroid Treatment for Acute Graft-versus-Host Disease. Biol Blood Marrow Transplant 26 (2020) 845-854 https://doi.org/10.1016/j.bbmt.2020.01.018; 4. International Blood and Marrow Transplantation Registry Severity Index Criteria (IBMTR) Abbreviations: CI = confidence interval SR-GvHD severity4 at baseline in GVHD001: Grade B: 11% Grade C: 43% Grade D: 46% RYONCIL treatment was not discontinued or interrupted in any patient for any laboratory abnormality, and the full course was completed without interruption in more than 85% of patients


 
14 m e s o b l a s t Ryoncil® long-term survival free from aGvHD Year 2 Survival: Long-term follow- up of Ryoncil by the Center for International Blood and Marrow Transplant Research (CIBMTR) 51% Year 4 Survival: 49% Kurtzberg phase III paper and Tandem poster Kurtzburg JS. Chaudhury S, Nemecek E , et al. Long-term Survival in Children Treated With Remestemcel-L for Steroid Refractory-Acute GVHD, Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, 2023; Mesoblast data on file Children from GVHD001 N=51 88% Grade C/D Only 14% (N=7) died due to aGvHD through 4 years


 
15 m e s o b l a s t 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) Supporting evidence for Ryoncil immunomodulatory MOA from pharmacodynamic analysis of blood samples in 40 subjects in GVHD001: • Circulating levels of CD3+CD4+CD25+HLA-DR+ activated T cells were reduced by 64% at Day 180 as compared to baseline. • Tumor necrosis factor receptor 1 (TNFR1) levels were reduced by 79% at Day 180 as compared to baseline. • Suppressor of tumorigenesis 2 (ST2) levels, a biomarker of gut inflammation, were reduced by 75% at Day 180 as compared to baseline. Mesoblast data unpublished


 
16 m e s o b l a s t Press Release available at www.mesoblast.com 1. Please see the full Prescribing Information at www.ryoncil.com 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


 
17 m e s o b l a s t High cost of treating child who dies from SR-aGvHD 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-refractory graft-versus-host disease in commercially insured pediatric patients. J Manag Care Spec Pharm.2021;27(5):607-14


 
18 m e s o b l a s t MyMesoblast Mandatory Hub START Patient Journey SR-aGVHD post-BMT, Ryoncil plan of treatment established MyMesoblast Patient enrollment Review treatment plan Assign patient ID for order authorization Order intake / coverage research Ryoncil prepared for shipment Outpatient Treatment / Follow up after therapy initiated Provide order status confirmation Patient Access Consultant Alignment call to confirm therapy started / potential outpatient treatment transition 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


 
19 m e s o b l a s t Availability of Ryoncil® for pediatric SR-aGvHD in the U.S. in March 2025 Staged approach based on transplant centers with highest volume and experience with Ryoncil® product Targeted sales force with experience in bone marrow transplant centers ------- 15 highest volume centers account for ~50% of patients ------- Targeting 45 highest volume centers / 80% of patients


 
20 m e s o b l a s t mesoblast Thank You