Mesoblast Limited · ASX:MSB
Mesoblast — Shareholder Meeting (November 2023)
37 slides from Mesoblast's shareholder meeting presentation, Nov 28, 2023
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 Annual General Meeting 2023 November 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: Corporate Vision
Corporate Vision For personal use only To be world’s leading, most innovative, and highly respected cellular medicines company To use our proprietary technologies to develop cellular medicine products that are life-saving and that improve quality of life To establish an organization that attracts mo
Slide 5: Investment Highlights
Investment Highlights Developing off-the-shelf, allogeneic cellular medicines based on proprietary Novel Allogeneic mesenchymal stromal cell (MSC) technology platforms to enable treatment without the Cell Therapy Platform need for donor matching or immunosuppression For personal use only Single-arm
Slide 6: Corporate Level Strategic Options Evaluated and Set
Corporate Level Strategic Options Evaluated and Set Preferred Strategic Options Strategy For personal use only Partner AND 1 Develop All on Own 2 Partner All indications 3 Develop carveouts 4 Sell Indications • Potential immediate financial rewards through partnership • Diminished development and co
Slide 7: Tactical Execution Of Corporate Strategy
Tactical Execution Of Corporate Strategy Intellectual For personal use only Go to market Capital Direct versus Partnership Our Patents & Advancing our our People Clinical Pipeline Building a Manufacturing Strong Brand Strategy for Securing a Product Delineation Robust and Life-Cycle Financial Base 7
Slide 8: Setting Key Strategic Priorities for 2024
Setting Key Strategic Priorities for 2024 ▪ Additional potency assay data, provide to FDA Seeking first regulatory approval in the US ▪ Commence adult SR-aGVHD Phase 3 trial ▪ Continue to seek pediatric SR-aGVHD approval and launch For personal use only Further advancement of ▪ Enrollment of CLBP se
Slide 9: Global Intellectual Property (IP) Estate Provides Substantial Competitive Advantage
Global Intellectual Property (IP) Estate Provides Substantial Competitive Advantage Extensive patent portfolio with protection extending through 2040 Over 1,100 patents and patent applications (82 patent families) For personal use only Markets across all major jurisdictions Global coverage including
Slide 10: Commercial-scale Manufacturing Process and Facilities
Commercial-scale Manufacturing Process and Facilities Scalable allogeneic “off-the-shelf” cellular platforms Manufacturing Remestemcel-L For personal use only Manufacturing meets stringent criteria of international regulatory agencies Robust quality assurance processes ensure final product with batc
Slide 11: 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 IL-10 Breg TH1 Prol
Slide 12: Late-Stage Clinical Pipeline
Late-Stage Clinical Pipeline Based on the Proprietary Allogeneic Mesenchymal Stromal Cell Platform Product Indication Phase 2 Phase 3 Regulatory Filing Approved For personal use only Pediatric Remestemcel-L SR-aGVHD Adult Remestemcel-L SR-aGVHD Crohn’s Rexlemestrocel-L CLBP SR-aGVHD = Steroid-Refrac
Slide 13: Clinical Program Milestones - Next Months
Clinical Program Milestones - Next 12 Months Target Date Status • Currently finalizing additional potency assay data on commercial Q1 CY2024 In progress RYONCIL inventory to provide to FDA Adult & Pediatric • Planned meeting with the FDA regarding potency assay data for the Q1 CY2024 Planned For per
Slide 14: Pathway to Approval for RYONCIL in Pediatric Patients with SR-aGVHD
Pathway to Approval for RYONCIL in Pediatric Patients with SR-aGVHD During the Biologics License Application (BLA) review we made substantial progress towards bringing this cutting-edge product to market with a completed FDA inspection of our manufacturing process. For personal use only In August FD
Slide 15: Pathway to Approval for RYONCIL in Adult Patients with SR-aGVHD
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, 100-day su
Slide 16: Financials
Financials Revenue from royalties, predominantly on sales of TEMCELL® HS Inj.1 sold in Japan by our licensee, were US$7.5 million for the year ended June 30, 2023. For personal use only Cash balance at September 30, 2023 was US$53.2 million, with net operating cash spend of US$14.2 million for the q
Slide 17: Chronic Low Back Pain due to Degenerative Disc Disease (CLBP)
For personal use only Chronic Low Back Pain due to Degenerative Disc Disease (CLBP) 17 mesoblast
Slide 18: 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 Burden of Illness Treatment Options Market Opportunity For personal use only Back pain causes more Minimal treatment options Over 7m patients are d
Slide 19: 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 20: RMAT Designation Granted by FDA for Rexlemestrocel-L in CLBP
Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by FDA for Rexlemestrocel-L in the treatment of CLBP RMAT designation provides all the benefits of Breakthrough and Fast Track designations, including rolling review and eligibility for priority review on filing of a Biologics License
Slide 21: Phase 3 Trial Outcomes based on a Single Injection of Rexlemestrocel-L + HA
Phase 3 Trial Outcomes based on a Single Injection of Rexlemestrocel-L + HA Results in 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 signific
Slide 22: Chronic Heart Failure Reduced Ejection Fraction (HFrEF)
For personal use only Chronic Heart Failure Reduced Ejection Fraction (HFrEF) 22 mesoblast
Slide 23: Rexlemestrocel-L / HFrEF – Program Summary
Rexlemestrocel-L / HFrEF – Program Summary Defining the Regulatory Path to FDA Approval For personal use only Significant Need Promising Data Targeting Inflammation FDA Meeting Cardiovascular disease Recent data from the Effects on LVEF and MACE Mesoblast plans to meet remains the leading DREAM-HF P
Slide 24: 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 Mesoblast’s Development Programs For personal use only DREAM HF-1 Trial LVAD MPC Studies 537 Patients 189 Patients Early Guid
Slide 25: Rexlemestrocel-L – Two Pivotal Studies in Chronic Heart Failure
Rexlemestrocel-L – Two Pivotal Studies in Chronic Heart Failure (CHF) Mesoblast’s Development Programs Assess the Impact of Intra-cardiac Administration of Rexlemestrocel-L Across the Continuum of Disease from Mild/Moderate to End-stage Severity MPC Study Design LVAD-MPC Study #2 DREAM-HF Trial For
Slide 26: 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 27: REVASCOR As Treatment For Severe Congenital Heart Disease
REVASCOR As Treatment For Severe Congenital Heart Disease Filed with FDA For Orphan Drug And Pediatric Rare Disease Designations Hypoplastic left heart syndrome (HLHS) is a severe congenital Anatomy of hypoplastic left heart syndrome heart disease in which the left side of the heart does not For per
Slide 28: REVASCOR As Treatment For Severe Congenital Heart Disease
REVASCOR As Treatment For Severe Congenital Heart Disease Filed with FDA For Orphan Drug And Pediatric Rare Disease Designations In the HLHS randomized controlled single-center US trial in 19 patients, a single intramyocardial administration of REVASCOR at the time of staged surgery resulted in sign
Slide 29: Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD)
For personal use only Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD) 29 mesoblast
Slide 30: Acute Graft Versus Host Disease (aGVHD)
Acute Graft Versus Host Disease (aGVHD) Serious and Fatal Complication of Allogeneic Bone Marrow Transplantation (BMT) For personal use only PHASE 1 PHASE 2 PHASE 3 Host Tissue Damage Immune Cell Activation Inflammation and End Organ by BMT Conditioning & Cytokine Storm Damage Conditioning regimen,
Slide 31: Remestemcel-L: Steroid-Refractory Acute Graft Versus Host Disease
Remestemcel-L: Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD) SR-aGVHD is associated with mortality rates as high as 90% Treatment Options Burden of Illness Market Opportunity For personal use only Corticosteroids are first-line Acute GVHD is a life- More than 30,000 therapy for aGVHD
Slide 32: Remestemcel-L for Children with SR-aGVHD
Remestemcel-L for Children with SR-aGVHD Improved Early Survival Across Three Studies involving more than 300 Treated Children Day 100 Survival For personal use only Remestemcel-L Protocol Remestemcel-L Matched Controls Matched Control Protocol First Line Therapy after Steroids Treatment Setting Ped
Slide 33: Long term Survival in Pediatric Patients with SR-aGVHD Treated with Remestemcel-L
Long term Survival in Pediatric Patients with SR-aGVHD Treated with Remestemcel-L Presented at the 2023 Remestemcel-L Remestemcel-L Tandem has has the Meeting the Potential Potential of ASTCT to Improve to Improve andLong-Term BleakBleak CIBMTR Long-Term Survival Survival (>2 Years) (>2 Years) in in
Slide 34: Extended Survival Data in Children with SR-aGVHD
Extended Survival Data in Children with SR-aGVHD Remestemcel-L Treatment Resulted in Durable Survival Over 4 Years Survival Outcomes in Pediatric & Adult SR-aGVHD For personal use only (Remestemcel-L data from the Center for International Blood and Marrow Transplant Research (CIBMTR) dbase) Study GV
Slide 35: Pathway to Approval for RYONCIL in Pediatric Patients with SR-aGVHD
Pathway to Approval for RYONCIL in Pediatric Patients with SR-aGVHD During the Biologics License Application (BLA) review we made substantial progress towards bringing this cutting-edge product to market with a completed FDA inspection of our manufacturing process. For personal use only In August FD
Slide 36: RYONCIL for Adults with SR-aGVHD
RYONCIL for Adults with SR-aGVHD Commercial strategy is to progress to adults who have failed steroids and a first-line agent, including ruxolitinib Market opportunity approximately five times larger than pediatric For personal use only Approximately 45% of ruxolitinib patients are non-responders 1
Slide 37: Thank You
mesoblast For personal use only Thank You 37 mesoblast