Mesoblast Limited · ASX:MSB
Mesoblast — Shareholder Meeting (November 2024)
38 slides from Mesoblast's shareholder meeting presentation, Nov 15, 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 Annual General Meeting 2024 November 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 off-the-shelf allogeneic cellular medicines to treat serious and life-threatening inflammatory illnesses Our Mission
Slide 4: Corporate Vision
Corporate Vision For personal use only To be the 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 attract
Slide 5: 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 6: 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 7: 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 8: 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 9: Late-Stage Clinical Pipeline based on proprietary allogeneic 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 RYONCIL® SR-aGVHD remestemcel-L Adult SR-aGVHD RYONCIL® IBD / remestemcel-L Crohn’s Pediatric HLHS
Slide 10: 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 Resubmitted BLA for approval in pediatric patients with FDA accepting 1 Steroid-Refractory Acute- Graft versus Host Di
Slide 11: Financials
Financials Cash balance at September 30, 2024 is US$51.1 million, with additional US$60.0 million available from existing financing facilities on FDA approval of RYONCIL. For personal use only Net operating cash spend of US$10.5 million for the quarter ended September 30, 2024. 26% reduction in net
Slide 12: Remestemcel-L: Steroid-Refractory Acute Graft Versus Host Disease
For personal use only Remestemcel-L Steroid-Refractory Acute Graft Versus Host Disease (SR-aGVHD) 12 mesoblast
Slide 13: RYONCIL for steroid-refractory acute graft versus host disease
RYONCIL for steroid-refractory acute graft versus host disease (SR-aGVHD) For personal use only Product Indication Phase 2 Phase 3 Regulatory Filing Approved Pediatric RYONCIL® SR-aGVHD remestemcel-L Adult SR-aGVHD This chart is figurative and does not purport to show individual trial progress withi
Slide 14: 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 15: 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 16: 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 1.
Slide 17: Long term Survival in Pediatric Patients with SR-aGVHD
Long term Survival in Pediatric Patients with SR-aGVHD Treated with Remestemcel-L Remestemcel-L Remestemcel-L hasPotential has the the Potential to Improve to Improve BleakBleak Long-Term Long-Term Survival Survival (>2 Years) (>2 Years) in in Pediatric Pediatric SR-aGVHD SR-aGVHD For personal use o
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: Potential FDA approval of RYONCIL for pediatric patients with SR-aGVHD
Potential FDA approval of RYONCIL for pediatric patients with SR-aGVHD Mesoblast resubmitted its Biologics License Application (BLA) to FDA for approval of RYONCIL on July 8, For personal use only 2024 and anticipates a decision prior to or on the FDA’s Prescription Drug User Fee Act (PDUFA) goal da
Slide 20: Pre-Launch Activities For Go to Market Strategy
Pre-Launch Activities For Go to Market Strategy - RYONCIL in Pediatric Patients Hiring of select senior positions to build targeted commercial team has commenced For personal use only Key Activities: o Market Access initiates payer outreach o Medical provides education to payers o Corporate leadersh
Slide 21: Post-Launch Activities For Go to Market Strategy
Post-Launch Activities For Go to Market Strategy - RYONCIL in Pediatric Patients For personal use only Post-launch - Staged approach based on centers with highest volume and experience with product. Targeted sales force with experience in bone marrow transplant centers - 15 highest volume centers ac
Slide 22: Label extension strategy for RYONCIL in adult patients
Label extension strategy for RYONCIL in adult patients with SR-aGVHD Continued unmet need in adults with SR-aGVHD who fail ruxolitinib (>40% of treated patients). For personal use only Survival in these patients who fail ruxolitinib remains a dismal 20-30% by 100 days, a patient population with no a
Slide 23: Rexlemestrocel-L: Chronic Low Back Pain
For personal use only Rexlemestrocel-L Chronic Low Back Pain due to Degenerative Disc Disease (CLBP) 23 mesoblast
Slide 24: Rexlemestrocel-L for chronic low back pain
Rexlemestrocel-L for chronic low back pain For personal use only Product Indication Phase 2 Phase 3 Regulatory Filing Approved Rexlemestrocel-L CLBP (STRO3+) This chart is figurative and does not purport to show individual trial progress within a clinical program Notes: ▪ Grünenthal has an exclusive
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+ 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 cond
Slide 26: 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 first-line treatment for patients with moderate to severe CLBP, refractory to conservative treatment For personal use only Rexlemestrocel-L targeting moderate-to-severe CLBP Conservative Opi
Slide 27: Single injection showed more than three years of pain reduction
Phase 3 trial outcomes based on a single injection of rexlemestrocel-L + HA showed 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 significantl
Slide 28: 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 29: Rexlemestrocel-L: Heart Failure
For personal use only Rexlemestrocel-L Heart Failure 29 mesoblast
Slide 30: REVASCOR for pediatric congenital heart disease and adults with ischemic HFrEF
REVASCOR for pediatric congenital heart disease and adults with ischemic HFrEF For personal use only Product Indication Phase 2 Phase 3 Regulatory Filing Approved Pediatric HLHS REVASCOR® Adult HFrEF rexlemestrocel-L End-stage (STRO3+) Adult HFrEF Class II/III This chart is figurative and does not p
Slide 31: Pediatric: REVASCOR as treatment for severe congenital heart disease
Pediatric: REVASCOR As treatment for severe congenital heart disease REVASCOR has multiple mechanisms-of-action that may be beneficial to children with congenital heart disease including Anatomy of hypoplastic left heart syndrome neovascularization, anti-fibrosis, and reduction in inflammation. For
Slide 32: Pediatric: REVASCOR HLHS randomized controlled trial results
Pediatric: REVASCOR as treatment for severe congenital heart disease 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 significantly increased LV systolic For personal use only and di
Slide 33: FDA awarded Rare Pediatric Disease and Orphan Drug designation to REVASCOR
Pediatric: FDA awarded Rare Pediatric Disease designation and Orphan Drug designation to REVASCOR for hypoplastic left heart syndrome For personal use only FDA granted Mesoblast’s cardiovascular investigational product, REVASCOR, both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designa
Slide 34: Adult: HFrEF is increasing in prevalence with high mortality risk
Adult: 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 increasing.1 Chronic hea
Slide 35: REVASCOR potential to improve endothelial dysfunction in HFrEF patients
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 36: 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 37: Pathway to accelerated approval for REVASCOR in adults with HFrEF
Pathway to accelerated approval for REVASCOR in adults with HFrEF DREAM-HF Trial over a mean follow-up of 30 months showed significant reduction in 3-Point MACE in ischemic HFrEF patients (n=158). For personal use only LVAD-MPC Study #2, over 12 months of follow-up, showed significant increase in pr
Slide 38: Thank You
mesoblast For personal use only Thank You 38 mesoblast