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 BIOTECH SHOWCASE 2023 January 2023 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
Our Mission For personal use only Mesoblast is committed to bringing to market innovative cellular medicines to treat serious and life-threatening illnesses
Slide 4: Investment Highlights
Investment Highlights First FDA Approval Expected in 2023 For personal use only Potential 2023 Launch for Remestemcel-L BLA to be resubmitted for children with steroid-refractory acute graft Remestemcel-L/SR-aGVHD versus host disease (SR-aGVHD), with potential US approval in mid-CY2023 Rexlemestroce
Slide 5: Remestemcel-L / SR-aGVHD BLA Resubmission Milestones
Remestemcel-L / SR-aGVHD Biologics License Application (BLA) Resubmission Milestones For personal use only Submitted substantial new information on clinical and potency assay items identified in Updated FDA IND File in Oct-22 the CRL received from FDA in 2020 to Investigational New Drug (IND) file f
Slide 6: Late-Stage Clinical Pipeline
Late-Stage Clinical Pipeline Based on the Proprietary Allogeneic Mesenchymal Stromal Cell Platform For personal use only Product Indication Phase 2 Phase 3 Regulatory Filing Approved Status/Next Steps • BLA to be resubmitted Remestemcel-L SR-aGVHD • Planning for a mid-CY2023 launch • Finalizing Phas
Slide 7: Mesoblast's Proprietary Stem Cell Technology
Mesoblast’s Proprietary Stem Cell Technology Based on mesenchymal lineage adult stem cells (MLCs/SCs) For personal use only Mesenchymal Lineage Defined Stem Cells Allogenic Properties Scalable Production MLCs are derived from Biologically-defined, Expanded without Scalable “off-the-shelf” healthy bo
Slide 8: Remestemcel-L: Steroid-Refractory Acute Graft Versus Host Disease
For personal use only Remestemcel-L Steroid-Refractory Acute Graft Versus Host Disease (SR- aGVHD) BLA to be resubmitted. Tracking to a potential mid-CY2023 approval 8 mesoblast
Slide 9: 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% For personal use only Treatment Options Burden of Illness Market Opportunity Corticosteroids are first-line Acute GVHD is a life- More than 30,000 therapy for aGVHD
Slide 10: Improved Early Survival in Children Across Three Studies
Remestemcel-L for SR-aGVHD Improved Early Survival in Children Across Three Studies For personal use only Day 100 Survival Remestemcel-L Protocol Remestemcel-L Matched Controls Matched Control Protocol First Line Therapy after Steroids Treatment Setting Pediatric Subset of Protocol 280: randomized c
Slide 11: Extended Survival Data in Children with SR-aGVHD
Extended Survival Data in Children with SR-aGVHD Remestemcel-L Treatment Resulted in Consistent and Durable Responses Over 4 Years For personal use only Survival Comparison (Remestemcel-L data from the Center for International Blood and Marrow Transplant Research (CIBMTR) dbase) Study GVHD001 MacMil
Slide 12: Survival Outlook in Pediatric Patients with SR-aGVHD
Survival Outlook in Pediatric Patients with SR-aGVHD Remestemcel has the Potential to Improve Long-Term Survival (>2 Years) For personal use only 1.Adapted and redrawn from Figure 2 of MacMillan, M.L. et al. Pediatric acute GVHD: clinical phenotype and response to upfront steroids. Bone Marrow Trans
Slide 13: Greater Responses and Day 180 Survival in Highest-Risk Patients
Remestemcel-L for SR-aGVHD Significantly Greater Day 28 Overall Responses and Day 180 Survival in Highest-Risk Patients (Baseline MAP ≥ 0.29) For personal use only Response by Baseline MAP Survival by Baseline MAP 0.8 MAP > 0.29 0.8 MAP > 0.29 Remestemcel-L vs MAGIC Remestemcel-L vs MAGIC 67% vs 10%
Slide 14: Putting the Pieces in Place for a U.S. Approval
Remestemcel-L / SR-aGVHD Putting the Pieces in Place for a U.S. Approval For personal use only Expanded data Potency Assay Manufacturing readiness Est. mid-CY2023 Approval Survival outcomes have Optimized, validated a In preparation for the BLA to be resubmitted not improved over two potency assay f
Slide 15: Rexlemestrocel-L Chronic Low Back Pain
For personal use only Rexlemestrocel-L Chronic Low Back Pain associated with Degenerative Disc Disease (CLBP) Commence Pivotal Phase 3 Study in CY2023 with Reduction in Pain Primary Endpoint 15 mesoblast
Slide 16: Chronic Low Back Pain Due to Degenerative Disc Disease 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 For personal use only Burden of Illness Treatment Options Market Opportunity Back pain causes more Minimal treatment options Over 7m patients are d
Slide 17: 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 1L Treatment for Patients with Moderate to Severe CLBP, Refractory to Conservative Treatment For personal use only Rexlemestrocel-L targeting moderate-to-severe CLBP Conservative Opioid Inte
Slide 18: Phase 3 Trial Outcomes: More than Three Years of Pain Reduction
Phase 3 Trial Outcomes based on a Single Injection of Rexlemestrocel-L + HA Results in More than Three Years of Pain Reduction For personal use only Greatest pain reduction was observed in the pre-specified population of subjects with CLBP duration shorter than the baseline study median of 68 months
Slide 19: Pricing Landscape for Pain Management to Disease Modifying Therapies
Pricing Landscape for Pain Management to Disease Modifying Therapies (DMTs) Reference Pricing Analysis Suggests Higher US Price Points for DMTs For personal use only ~$1.2k - $2.5k ~$9k - $15k+ ~$32.2k ~$45.2k ~$77.5k Annual Cost of Branded Disease Modifying Annual Cost of Branded Musculoskeletal Ag
Slide 20: Preparing to Initiate the Pivotal Phase 3 Program
Rexlemestrocel-L/DCLBP Preparing to Initiate the Pivotal Phase 3 Program For personal use only In Prep for US/EU Commence Pivotal P3 Regulatory Alignment Phase 3 Protocol Submissions H1 CY2023 Gained alignment with FDA has agreed with The planned Phase 3 Awaiting clearance from the FDA on the Mesobl
Slide 21: Rexlemestrocel-L Chronic Heart Failure Reduced Ejection Fraction
For personal use only Rexlemestrocel-L Chronic Heart Failure Reduced Ejection Fraction (HFrEF) Tracking to a potential H1 CY2023 FDA meeting to map out pivotal trial plans and regulatory pathway 21 mesoblast
Slide 22: Chronic Heart Failure: Rising Incidence and High Mortality
Chronic Heart Failure (CHF): Rising Incidence and High Mortality New therapies reduce recurrent hospitalization but do not materially improve mortality or major ischemic event rates For personal use only Cardiovascular disease (CVD) remains the leading cause of death in the United States1 Heart fail
Slide 23: 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 For personal use only Treatment Algorithm in Progressive Heart Failure (HF) Progressive Vascular (Endothelial) Dysfunction an
Slide 24: DREAM Phase Trial in Heart Failure (HFrEF)
Rexlemestrocel-L: DREAM Phase 3 Trial in Heart Failure (HFrEF) Improvement in Left Ventricular Systolic Function, as Measured by Left Ventricular Ejection Fraction (LVEF) at 12 Months represents a potential early surrogate endpoint For personal use only In all treated patients (n=537), rexlemestroce
Slide 25: DREAM-HF Phase Trial in HFrEF
DREAM-HF Phase 3 Trial in HFrEF Rexlemestrocel-L reduced incidence of 3-Point Composite MACE v. controls across all treated patients, with enhanced effect in those with active inflammation as measured by CRP >2 For personal use only All Treated Patients (n=537) All Treated Patients with hsCRP ≥2 (n=
Slide 26: Defining the Regulatory Path to FDA Approval
Rexlemestrocel-L / HFrEF Defining the Regulatory Path to FDA Approval For personal use only Significant Need Promising Data Targeting Inflammation H1 CY2023 FDA Meeting Cardiovascular disease Recent data from the Effects on LVEF and MACE Mesoblast plans to meet remains the leading DREAM P3 trial sho
Slide 27: The Makings of a Potential Banner Year for Mesoblast
The Makings of a Potential Banner Year for Mesoblast For personal use only January 2023 H1 CY2023 H2 CY2023 BLA resubmission for Initiate pivotal Phase 3 Estimated product launch for remestemcel-L/SR-aGVHD study rexlemestrocel-L remestemcel-L/SR-aGVHD /CLBP H1 CY2023 Mid-CY2023 FDA meeting for PDUFA
Slide 28: Thank You
mesoblast For personal use only Thank You 28 mesoblast