Mesoblast Limited · ASX:MSB
Mesoblast — Earnings Presentation (February 2018)
27 slides from Mesoblast's earnings presentation, Feb 23, 2018
Slide 1: Phase 3 Cell Therapy Trial for aGVHD Successfully Achieves Primary Endpoint
Mesoblast Phase 3 Cell Therapy Trial for Acute Graft Versus Host Disease (aGVHD) Successfully Achieves Primary Endpoint Presented at BMT/Tandem, February 21, 2018 22/23 February 2018 Nasdaq: MESO ASX: MSB |1
Slide 2: Cautionary Note Regarding Forward-Looking Statements
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, levels of activity, perf
Slide 3: Our Mission
Our Mission: Mesoblast is committed to bring to market disruptive cellular medicines to treat serious and life-threatening illnesses |3
Slide 4: Investment Proposition
Investment Proposition: Building a Leading Franchise of Cellular Medicines Disruptive Cellular Technology Platform Commercial Translation Capabilities Advanced Pipeline of Cellular Medicines Targeting Serious or Life-Threatening Conditions with Unmet Needs |4
Slide 5: Disruptive Cellular Medicine Platform
Disruptive Cellular Medicine Platform1-4 Mesenchymal Lineage Cells (MLCs) have unique receptors that respond to activating inflammatory and damaged-tissue signals In response to these signals, MLCs secrete a diverse variety of biomolecules responsible for immunomodulation and tissue repair The
Slide 6: Commercial Translation Capabilities
Commercial Translation Capabilities: Technology Positioned for Scalable, Industrialized Manufacturing Immune privileged nature of MLCs enables allogeneic “off the shelf” product candidates Culture expansion scalable to produce commercial quantities of potent and reproducible therapeutic doses
Slide 7: Portfolio of Advanced Product Candidates
Portfolio of Advanced Product Candidates: Three Tier 1 Product Candidates in Phase 3 Commercialization Product Therapeutic Area Pre-Clinical/ Platform Candidate Pre-IND Phase 2 Phase 3 Approval Partnering1 Advanced (Class 3) HF MPC MPC-150-IM End Stage (Class 4) HF1 MPC MPC-06-ID Chronic Low Back Pa
Slide 8: Acute Graft vs Host Disease
Acute Graft vs Host Disease Remestemcel-L (MSC-100-IV) for Steroid-Refractory aGVHD |8 |8
Slide 9: Acute graft-versus-host disease (aGVHD) Background
Acute graft-versus-host disease (aGVHD) Background Acute graft-versus-host disease (aGVHD) is associated with significant morbidity and is a leading cause of mortality after allogeneic hematopoietic stem cell transplantation Although the incidence of aGVHD varies across transplant type and regim
Slide 10: Immunomodulatory Actions of MSCs Triggered by Inflammatory Cues
The Immunomodulatory Actions of MSCs are Triggered by their Response to Inflammatory cues: Paracrine-mediated alteration of the function of multiple cellular constituents of the innate and adaptive immune systems IL-10 Breg TH1 Proliferation Cytokine Proliferation Antibody Effector MSC IDO, PGE2 sec
Slide 11: Remestemcel-L: Phase 3 Pediatric Trial GVHD001 Completed Enrollment
Remestemcel-L (MSC-100-IV): Phase 3 Pediatric Trial GVHD001 Completed Enrollment as First-line Therapy in aGVHD After Failing Steroids Multi-center, Single-Arm, Open-Label to evaluate efficacy and safety to day 100 (GVHD001) and from day 100 to day 180 (GVHD002) 55 pediatric patients (2 months t
Slide 12: Protocol GVHD001: Demographics
Protocol GVHD001: Demographics Subjects Enrolled 55 Age (years) Mean (SD) 7.8 ( 5.44) Median (minimum, maximum) 7.6 ( 0.6, 17.9) Gender Male 35 ( 63.6%) Female 20 ( 36.4%) Underlying Disease AML 18 ( 32.7%) ALL 12 ( 21.8%) Anemia 5 ( 9.1%) CML 4 ( 7.3%) Sickle Cell 3 ( 5.5%) JML 2 ( 3.6%) MDS 2 ( 3.
Slide 13: Protocol GVHD001: Transplant Characteristics
Protocol GVHD001: Transplant Characteristics reflect aGVHD risk factors Conditioning Regimen Myeloablative Reduced Intensity Non-Myeoablative ● 87% of subjects received myeloablative conditioning ● 51% of subjects received an HLA-mismatched regimen transplant Graft Source Bone Marrow PBSC Cord Blood
Slide 14: Protocol GVHD001: Disease Characteristics
Protocol GVHD001: Disease Characteristics reflect aGVHD severity GVHD Grade at Baseline ● 47% of subjects had Grade D disease at baseline Grade B Grade C ● 89% of subjects had Grade C/D disease at baseline Grade D ● 26% of subjects had Skin involvement only Baseline Organ Involvement ● all had stage
Slide 15: Protocol GVHD001: Disease Characteristics (risk)
Protocol GVHD001: Disease Characteristics (risk) Disease Risk at Study Baseline ● 73% of subjects had high-risk disease at baseline10 ● stage 3 (n=6) or stage 4 (n=10) Lower GI only ● stage 4 Skin only (n=4) ● any stage Lower GI + any stage Liver (n=4) Standard ● stage 2-4 Lower GI + any stage Skin
Slide 16: Protocol GVHD001: Primary Efficacy Outcome
Protocol GVHD001: Primary Efficacy Outcome Overall Response at Day 28 was 69%, p=0.0003 80% 70% 60% 50% 40% 30% 20% 10% 0% 69% Overall Response rate at Day 28 (29% CR + 40% PR) p-value calculated from the binomial distribution, under the assumption of a 0.45 success rate under the null hypothesi
Slide 17: Protocol GVHD001: Results of Safety and Mortality
Protocol GVHD001: Results of Safety and Mortality Remestemcel-L (MSC-100-IV) infusions were well tolerated The incidence of adverse events in the trial was consistent with that expected from the underlying disease state and in line with previous use of Remestemcel-L (MSC-100-IV) Eleven subject
Slide 18: Protocol GVHD001: Summary and Conclusions
Protocol GVHD001: Summary and Conclusions This Phase 3 study evaluated allogeneic mesenchymal stem cells (MSCs), Remestemcel-L (MSC-100-IV), for the treatment of steroid-refractory acute graft-versus-host disease intended to improve overall response rate in pediatric subjects Study successfully
Slide 19: Protocol GVHD001: Authors
Protocol GVHD001: Authors • Chaudhury S; Lurie Children’s Hospital of Chicago, Chicago, IL • Nemecek E; Oregon Health and Science University, Portland, OR • Mahadeo K; MD Anderson Cancer Center, Houston, TX • Prockop S; Memorial Sloan Kettering Cancer Center, New York, NY • Horn B; University of Flo
Slide 20: Remestemcel-L: Expanded Access Program
Remestemcel-L (MSC-100-IV): Expanded Access Program Overall Day 28 Response in Pediatric aGVHD Patients Receiving Remestemcel-L (MSC-100-IV) as First-line or Salvage Therapy After Failing Steroids Population: steroid-refractory 100% aGVHD pediatric patients 241 pediatric patients undergoing HSCT w
Slide 21: Remestemcel-L: Expanded Access Program
Remestemcel-L (MSC-100-IV): Expanded Access Program Correlation of Day 28 Overall Response with Day 100 Survival, Using Remestemcel-L (MSC- 100-IV) as First-line or Salvage Therapy After Failing Steroids and/or Additional Treatments Remestemcel-L (MSC-100-IV) in Children with SR-aGVHD who failed mul
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Slide 23: Remestemcel-L: Market Opportunity for aGVHD
Remestemcel-L (MSC-100-IV): Market Opportunity for aGVHD Steroid-refractory aGVHD patients have mortality rates as high as 95%1 Burden of Refractory aGVHD is associated with significant extended hospital stay costs2 Illness aGVHD - a severe immunological reaction occurring in BMT patients Is
Slide 24: Remestemcel-L for aGVHD: Product Development Strategy
Remestemcel-L for aGVHD: Product Development Strategy 1. Target pediatric patients with SR-aGVHD first Extensive safety and efficacy data generated and published in children with SR-aGVHD1 High economic burden in treatment of children with SR-aGVHD Fast-track designation provides pathway for p
Slide 25: Milestones
Milestones | 25 | 25
Slide 26: Targeted Upcoming Milestones and Catalysts
Targeted Upcoming Milestones and Catalysts Remestemcel-L (MSC-100-IV): for Pediatric Acute GVHD − Day 28 primary endpoint data read-out (Q1 CY18) COMPLETE − Day 100 survival data (Q2 CY18) − Day 180 safety data (Q3 CY18) MPC-06-ID for Chronic Low Back Pain − Phase 3 trial expected to complete en
Slide 27: Questions?
Questions? | 27 | 27