Mesoblast Limited · ASX:MSB
Mesoblast — Q3 FY2023 Earnings Presentation
34 slides from Mesoblast's earnings presentation, May 26, 2023
Slide 1: Operational Highlights and Financial Results for the Quarter Ended March 31, 2023
mesoblast For personal use only Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases Operational Highlights and Financial Results for the Quarter Ended March 31, 2023 May 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 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 FDA review
Slide 5: 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 Status/Next Steps For personal use only • BLA accepted for review Pediatric • PDUFA goal date August 2, Remestemcel-L SR-aGVHD 2023 • FDA P
Slide 6: Manufacturing Remestemcel-L
For personal use only for the Period Ended March 31, 2023 Manufacturing Remestemcel-L 6 © Lonza, reproduced with permission mesoblast
Slide 7: Financial Highlights
Financial Highlights Revenue from royalties on sales of TEMCELL® HS Inj.1 sold in Japan by our licensee were US$1.8 million for the quarter ended March 31, 2023. On a constant currency basis, royalties on sales grew 4% quarter on Royalty Revenue quarter to US$2.02 million for the quarter ended March
Slide 8: Reduction in Expenditure on R&D, Improved Loss Before Tax
Reduction in Expenditure on R&D, Improved Loss Before Tax Mar 31, Mar 31, Revenue: Revenue predominately from royalties on sales of For personal use only P&L for the quarter ended (US$m) 2023 2022 TEMCELL® HS Inj.1 sold in Japan by our licensee. Total Revenue 1.9 2.0 Reduction in R&D Expenditure: re
Slide 9: Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD)
For personal use only Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD) 9 mesoblast
Slide 10: 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 11: 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% 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 12: 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 13: Remestemcel-L Treatment Outcomes
Remestemcel-L Treatment Outcomes Significantly Greater Survival in Highest-Risk Steroid-Refractory Patients with Baseline MAP ≥ 0.29 Kaplan-Meier Estimates of 6-month Overall For personal use only Survival for the Two Patient Cohorts by Baseline MAP Abbreviations: MAP: MAGIC algorithm probability; B
Slide 14: Status of BLA for Remestemcel-L: New Data Under Review
Status of BLA for Remestemcel-L in Pediatric Patients with SR-aGVHD New Data Under Review BLA resubmitted Jan 30, 2023 For personal use only BLA file considered by FDA to be a complete response, accepted for review, with PDUFA goal date August 2, 2023 New data under review shows: o Durable long-term
Slide 15: Status of BLA for Remestemcel-L: Manufacturing Inspection Conducted
Status of BLA for Remestemcel-L in Pediatric Patients with SR-aGVHD Manufacturing Inspection Conducted For personal use only FDA has now conducted the Pre-License Inspection (PLI) of the manufacturing process for remestemcel-L FDA inspection did not result in the issuance of a Form 483, which is pro
Slide 16: Remestemcel-L: Long-Term Survival Data a Cornerstone of BLA Resubmission
Remestemcel-L: Long-Term Survival Data a Cornerstone of BLA Resubmission to FDA for SR-aGVHD Mesoblast provided new results from a four-year observational survival study performed by the For personal use only Center for International Blood and Marrow Transplant Research (CIBMTR) on 51 evaluable pati
Slide 17: 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 18: 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 19: Immunomodulatory Activity of Remestemcel-L Correlates with Survival
The Immunomodulatory Activity of Remestemcel-L on T Cell Activation in vitro is a Direct Measure of Product Potency and Correlates with Survival in Pediatric Patients with SR-aGVHD The clinical benefits of remestemcel-L in SR-aGVHD are likely due to its immunomodulatory effects For personal use only
Slide 20: Correlation of Remestemcel-L (Ryoncil) Lot Potency and 6-Month Survival
Correlation of Remestemcel-L (Ryoncil) Lot Potency and 6-Month Survival Analyses were performed evaluating in vitro/in vivo relationships in relation to inhibition of T-cell activation by product lots administered For personal use only There was an association between higher inhibition of T-cell act
Slide 21: Survival Significantly Improved in EAP aGVHD Patients Receiving Higher Potency Ryoncil
Survival Significantly Improved in EAP 275 aGVHD Patients Receiving Higher Potency Ryoncil Product Made After 2008 Compared with Lower Potency Prochymal Product Made Before 2008 Greatest Effect Seen In Patients With Grade D Disease For personal use only All EAP 275 Day 100 survival (n=205): Grade D
Slide 22: Go to Market Strategy - Pre-Launch
Go to Market Strategy - Remestemcel-L in Pediatric Patients Pre-Launch: Engagement of Highest Transplant Volume Centers with Experience Using Ryoncil Non-promotional activities including profiling centers, educate on disease awareness & unmet needs, For personal use only and support payer engagement
Slide 23: Go to Market Strategy - Post-Approval Launch
Go to Market Strategy - Remestemcel-L in Pediatric Patients Post-Approval Launch: Staged Approach Initially Targeting Highest Transplant Volume Centers For personal use only Staged approach to launch based on centers with highest volume and experience with product Building out efficient, targeted sa
Slide 24: Chronic Low Back Pain due to Degenerative Disc Disease (CLBP)
For personal use only Chronic Low Back Pain due to Degenerative Disc Disease (CLBP) 24 mesoblast
Slide 25: 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 26: Rexlemestrocel-L / CLBP – Program Summary
Rexlemestrocel-L / CLBP – Program Summary For personal use only In Prep for US/EU Commence Pivotal P3 Regulatory Alignment Phase 3 Protocol Submissions Mid-CY2023 Gained alignment with FDA has agreed with The planned Phase 3 RMAT designation for the FDA on the Mesoblast plans for Program will includ
Slide 27: 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 28: 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 29: Chronic Heart Failure Reduced Ejection Fraction (HFrEF)
For personal use only Chronic Heart Failure Reduced Ejection Fraction (HFrEF) 29 mesoblast
Slide 30: 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 31: 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 32: Randomized Trial of Targeted Transendocardial Mesenchymal Precursor Cell Therapy in Heart Failure
ORIGINAL INVESTIGATIONS Precursor Cell Therapy in Patients Randomized Trial of Targeted With Heart Failure Transendocardial Mesenchymal Emerson C. Perin, MD, PHD,a Kenneth M. Borow, MD,b Timothy D. Henry, MD,c Farrell O. Mendelsohn, MD,d Leslie W. Miller, MD,e Elizabeth Swiggum, MD,f Eric D. Adler,
Slide 33: 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 34: Thank You
mesoblast For personal use only Thank You 34 mesoblast