Mesoblast Limited · ASX:MSB
Mesoblast — Investor Presentation (May 2024)
25 slides from Mesoblast's investor presentation, Jun 3, 2024
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 Corporate Presentation May 2024 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 cellular medicines to treat serious and life-threatening illnesses Our Mission
Slide 4: Global Leader in allogeneic cellular medicines for inflammatory diseases
Global Leader in allogeneic cellular medicines for inflammatory diseases Global ✓ Markets World leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life- For personal use only threatening inflammatory conditions ✓ Locations in Australia, the United Stat
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 Inflammation For personal use only IL-10
Slide 6: Late-Stage Clinical Pipeline based on proprietary allogeneic cell platform
Late-Stage Clinical Pipeline based on proprietary allogeneic mesenchymal precursor / stromal cell platform Product Indication Phase 2 Phase 3 Regulatory Filing Approved For personal use only Pediatric SR-aGVHD RYONCIL® (remestemcel-L) Adult SR-aGVHD RYONCIL® IBD / (remestemcel-L) Crohn’s HFrEF End-s
Slide 7: US patent exclusivity provides a major commercial barrier against competitors
US patent exclusivity for use of mesenchymal precursor / stromal cells for all indications, and for acute GVHD specifically, provides a major commercial barrier against potential competitors “Composition of matter” and “method of treatment” US Composition of Matter For personal use only patents have
Slide 8: Positive regulatory interactions with FDA have been the highlight of 2024
Positive regulatory interactions with FDA have been the highlight of 2024 year-to-date File BLA this quarter for approval of RYONCIL in pediatric acute graft versus host disease FDA informed the company that following additional consideration the available clinical data from its For personal use onl
Slide 9: Mesoblast expects to substantially advance its 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 On-track to submit BLA for approval in pediatric patients this quarter 1 Steroid-Refractory Acute- Graft versus Host D
Slide 10: Financials
Financials Last quarter we completed the entitlement offer and institutional placement raising A$97 million. For personal use only Cash balance at March 31, 2024 was A$117.0 million (US$76.4 million).1 Net operating cash spend of US$11.7 million for the quarter ended March 31, 2024. 28% reduction in
Slide 11: Remestemcel-L: Steroid-Refractory Acute Graft Versus Host Disease
For personal use only Remestemcel-L Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD) 11 mesoblast
Slide 12: Acute GVHD 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 13: SR-aGVHD is associated with mortality rates as high as 90%
Steroid-Refractory Acute Graft Versus Host Disease (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 For personal use only therapy for aGVHD threatening complicatio
Slide 14: Regulatory Status for RYONCIL in pediatric patients with SR-aGVHD
Regulatory Status for RYONCIL in pediatric patients with SR-aGVHD For personal use only FDA informed Mesoblast after its Type C meeting during the quarter that following additional consideration the available clinical data from its Phase 3 study MSB-GVHD001 appears sufficient to support submission o
Slide 15: Potential Pathway to Approval for RYONCIL in adult patients with SR-aGVHD
Potential 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 For personal use only In contrast,
Slide 16: Rexlemestrocel-L: Chronic Heart Failure Reduced Ejection Fraction
For personal use only Rexlemestrocel-L Chronic Heart Failure Reduced Ejection Fraction (HFrEF) 16 mesoblast
Slide 17: HFrEF with underlying ischemia is increasing in prevalence and mortality risk
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 increasing1 Chronic heart failu
Slide 18: REVASCOR potential to improve endothelial dysfunction across HFrEF disease spectrum
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 19: Randomized Trial of Targeted Transendocardial Mesenchymal Precursor Cell Therapy
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 20: REVASCOR results supporting Accelerated Approval
Results of a single intra-myocardial administration of REVASCOR (150 million MPCs) in patients with ischemic HFrEF which support Accelerated Approval LVAD-MPC Study #2, over 12 months of follow-up: For personal use only ❑ Increased proportion of LVAD recipients with ischemic HFrEF etiology successfu
Slide 21: FDA awarded RPDD and ODD to REVASCOR for HLHS
FDA awarded Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) to REVASCOR for pediatric congenital heart disease - hypoplastic left heart syndrome (HLHS) For personal use only FDA granted Mesoblast’s cardiovascular investigational product, REVASCOR, both RPDD and ODD this y
Slide 22: 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) 22 mesoblast
Slide 23: 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 from
Slide 24: 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 25: Thank You
mesoblast For personal use only Thank You 25 mesoblast