Mesoblast Limited · ASX:MSB
Mesoblast — 34th Annual J.P. Morgan Healthcare Conference 2016
32 slides from Mesoblast's conference presentation, Jan 19, 2016
Slide 1: Mesoblast - A Global Leader in Cell Based Medicines
Mesoblast - A Global Leader in Cell Based Medicines 34th Annual J.P. Morgan Healthcare Conference January 2016 Exhibit 99.1
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: Key Investment Highlights
Key Investment Highlights Disruptive technology platform: proprietary, allogeneic, “off-the-shelf” adult stem cells with predictable therapeutic properties Established late stage portfolio of distinct and advanced product candidates Strategic partnerships delivering clinical, manufacturing and comme
Slide 4: Focused Company With Strong Cash Reserves to Meet Key Corporate Objectives
Successfully completed US listing with access to world’s largest sophisticated healthcare investor pool and analysts Financing raised USD $63.5m (net proceeds) which significantly augmented existing cash reserves of USD $77.8m at 30 September 2015 Quarterly cash outflows expected to be reduced by ap
Slide 5: Disruptive Technology Platform: Multiple, Diverse Mechanisms
Disruptive Technology Platform 1 Multiple, Diverse Mechanisms Mesenchymal Lineage Adult Stem Cells (MLCs), immunoselected precursors and progeny Located around blood vessels in all vascularized tissues Respond to signals associated with tissue damage Secrete diverse variety of biomolecules responsib
Slide 6: Product Candidates Target Diseases with High Unmet Needs
Product Candidates Target Diseases with High Unmet Needs Three Tier 1 Product Candidates in Phase 3 Programs Update 2
Slide 7: Tier Product Candidate Deliverables
Tier 1 Product Candidate Deliverables 2
Slide 8: MPC-150-IM: Chronic Heart Failure – Market Opportunity
MPC-150-IM: Chronic Heart Failure (CHF) – Market Opportunity A MPC-150-IM is in development for patients with New York Heart Association Class II-IV CHF MPC-150-IM is positioned to fill the significant treatment gap in patients with advanced CHF AHA statistical Update – Heart disease and stroke stat
Slide 9: MPC-150-IM: Phase 2 Trial Results in CHF Identify Optimal Therapeutic Dose
MPC-150-IM: Phase 2 Trial Results in CHF Identify Optimal Therapeutic Dose A Phase 2, randomised placebo controlled trial in 60 patients with Class II / III CHF and LVEF<40% Placebo vs. 25, 75, 150 M MPCs injected by endomyocardial catheter At 6 months: Dose-dependent effect seen on left ventricular
Slide 10: MPC-150-IM: Phase 2 Results Identify Optimal Target Patients
MPC-150-IM: Phase 2 Trial Results in CHF Identify Optimal Target Patients, Advanced Heart Failure (Baseline LVESV>100ml)1 with LVESV >100ml1 A Change (Entire Cohort) Month 6 minus Baseline Change (LVESV > 100 mL) Month 6 minus Baseline Control (n=15) 150 M MPC (n=15) ∆, Control Corrected Control (n=
Slide 11: MPC-150-IM: Single Dose Prevents HF-MACE Over Years
MPC-150-IM: Single Dose Prevents HF-MACE Over 3 Years A Over 36 months, patients receiving 150M MPC had significantly greater probability of remaining free of a first HF-MACE* vs. controls (0% vs. 33%, p = 0.026 by log-rank) All HF-MACE events occurred in controls with baseline LVESV>100ml, where th
Slide 12: MPC-150-IM: Phase 3 Trial Recruiting Well and Targets Advanced Heart Failure
Patients with large baseline LVESV and advanced heart failure are at highest risk of HF-MACE For these patients existing therapies are inadequate and economic burden is greatest To confirm that MPC-150-IM reduces HF-MACE in patients with advanced heart failure, the ongoing Phase 3 trial is designed
Slide 13: MPC-150-IM: Reduced Size of Phase 3 Program Following FDA Discussions
Following the preliminary responses from FDA in Dec 2015, the ongoing phase 3 program is planned to be optimized as follows: The current Phase 3 trial size will be reduced from 1,165 to approximately 600 subjects The revised primary endpoint will be a comparison of recurrent HF-MACE between MPC trea
Slide 14: MPC-06-ID: Chronic Low Back Pain – Market Opportunity
MPC-06-ID: Chronic Low Back Pain due to Degenerative Disc Disease – Market Opportunity B MPC-06-ID is in development for the treatment of chronic low back pain (CLBP) lasting >6 months as a result of moderate degenerative intervertebral disc disease LEK & NCI opinion leader interviews, and secondary
Slide 15: MPC-06-ID: Phase 2 Trial Design
MPC-06-ID: 12 Month Phase 2 Trial Design B 100 patients with >6 months of CLBP due to DDD and unresponsive to conservative therapies (including opioids and epidural steroids) were evaluated in a randomized, placebo controlled Phase 2 trial Visual Analog Scale (VAS) scored from 0-100, evaluated at 1,
Slide 16: Greater Proportion of Patients with at Least 50% Pain Reduction Than Controls
12 & 24 Months 50% Reduction in VAS LBP with No Intervention p=0.023 6M MPC vs. saline p=0.118 6M MPC vs. HA p=0.166 18M MPC vs. saline 6 & 12Months 50% Reduction in VAS LBP with No Intervention p=0.013 6M MPC vs. saline P=0.006 6M MPC vs. HA P=0.110 18M MPC vs. saline P=0.060 18M MPC vs. HA B MPCs
Slide 17: Greater Proportion with at Least 15 Point Improvement in Function (ODI)
12 & 24 Months 15pt Improvement in ODI with No Intervention p=0.023 6M MPC vs. saline p=0.012 18M MPC vs. saline 6 & 12 Months 15pt Improvement in ODI with No Intervention p=0.052 6M MPC vs. saline p=0.058 18M MPC vs. saline B MPCs Groups Have a Greater Proportion of Patients Through 24 Months with
Slide 18: MPC-06-ID: Composite Endpoint for Both Pain and Function Over 24 Months
Treatment Success Composite Endpoint 50% VAS back pain reduction AND 15 point ODI improvement AND no intervention at the treated level d. p=0.090 6M MPC vs. saline p=0.044 6M MPC vs. saline p=0.058 6M MPC vs. HA p=0.090 18M MPC vs. saline MPC-06-ID: Composite Endpoint for Both Pain and Function Over
Slide 19: MPC-06-ID: Phase 3 Pathway to Potential Approval
MPC-06-ID: Phase 3 Pathway to Potential Approval in Patients with CLBP due to Moderate Degenerative Intervertebral Disc Disease B Phase 3 trial is enrolling well The study will enroll ~330 patients Primary efficacy endpoint is a composite of ≥ 50% pain improvement (VAS) and ≥ 15 point functional imp
Slide 20: MSC-100-IV / TEMCELL HS Inj.: Acute GVHD – Market Opportunity
MSC-100-IV / TEMCELL® HS Inj. is targeting pediatric and adult patients with acute Graft Versus Host Disease (aGVHD) following allogeneic Bone Marrow Transplant (BMT). Gratwohl A et al Quantitative and qualitative differences in use and trends of hematopoietic stem cell transplantation: a Global Obs
Slide 21: MSC-100-IV: Phase 2 Trial in Children with Steroid Refractory aGVHD
MSC-100-IV: Phase 3 Trial in Children with Steroid Refractory Acute Graft vs Host Disease (SR-aGVHD) C Compared with placebo control patients, MSC-100-IV produced markedly superior overall response at day 28, a clinically meaningful endpoint (p=0.0014). Response at Day 28 Randomized Placebo Controll
Slide 22: MSC-100-IV / TEMCELL HS Inj.: Product Launch Plans in aGVHD
MSC-100-IV / TEMCELL® HS Inj. : Product Launch Plans in aGVHD TEMCELL® HS Inj. is the first allogeneic stem cell product approved in Japan MSC-100-IV has the potential to be the first allogeneic stem cell product approved in US United States (MSC-100-IV): 2017 Potential FDA Approval Open-label Phase
Slide 23: Anti-Inflammatory Portfolio - a Major Emerging Opportunity
MLCs have receptors that respond to pro-inflammatory signals, resulting in release of anti inflammatory mediators Mesoblast is developing MLC product candidates to target immune mediated diseases where multiple pathways are associated with treatment resistant disease: Biologic refractory rheumatoid
Slide 24: Anti-Inflammatory Portfolio Clinical Studies
Biologic-refractory rheumatoid arthritis, 48 patients – Ongoing Randomized, placebo controlled, dose-escalating study First dose / cohort 1 is fully enrolled Topline data expected Q1 2016 Clinical objective is sustained remission Type 2 Diabetes with inadequately controlled glucose, 61 patients – Pu
Slide 25: MPC-300-IV: Biologic Refractory Rheumatoid Arthritis – Market Opportunity
MPC-300-IV: Biologic Refractory Rheumatoid Arthritis (RA) – Market Opportunity D GlobalData©: EPiCast Models / PharmaeTrack GlobalData©: Rheumatoid Arthritis Therapeutic – Pipeline Oct 2011 Alivernini, S et. al. Arthritis Research & Therapy 2009, 11:R163 Ongoing randomized, controlled Phase 2 Trial
Slide 26: MPC-300-IV: Diabetic Nephropathy – Market Opportunity
MPC-300-IV: Diabetic Nephropathy (DN) – Market Opportunity D 1. EpiCast Model Diabetic Nephropathy – Epidemiology Forecast to 2022 PharmaeTrack 2. Global Data PharmETrack; Solicited Analysis 3.US Renal Data System Annual Data Report 2012,2013,2014, 4. Levey A and Coresh J. Lancet 2012; 379:165-180,
Slide 27: Strategic Collaborations with Industry Leaders
Strategic Collaborations with Industry Leaders 3 Manufacturing Development Commercialization Manufacturing R&D Manufacturing Clinical development CHF clinical research CHF program – MPC-150-IM Global rights Additional CV & CNS programs R&D Collaborations Commercial partnerships Licensing / JV Japan
Slide 28: Scalable Manufacturing Capabilities: Partnership with Lonza
Scalable Manufacturing Capabilities: Partnership with Lonza Manufacturing objectives Distinct manufacturing processes for each product Commercial scale processes with batch-to-batch consistency and reproducible release criteria Substantial advances made in development of consistent high yield manufa
Slide 29: Extensive Intellectual Property Portfolio
Extensive Intellectual Property Portfolio – 661 Patents Across 72 Families# Specific therapeutic applications 382 patents or patent applications Valid through 2035* Immunologic / inflammatory disorders – 100 patents or patent applications – start to expire 2019* and extend to 2035* Cardiovascular di
Slide 30: Experienced Management Team
Experienced Management Team 6 Dr John McMannis – PhD Head Manufacturing 27+ years in clinical cellular therapy trials in both academic and commercial environments. Previously: Professor of Medicine, Director of the Cell Therapy Laboratory and Technical Director of the Cord Blood Bank, University of
Slide 31: Experienced Board of Directors
Experienced Board of Directors 6 Brian Jamieson, FCA Non-Executive Chairman Former Chief Executive of a major Australian Law Firm Minter Ellison, Chief Executive Officer at KPMG Australia, KPMG Board Member in Australia and a member of the USA Management Committee. Brian has over 30 years of experie
Slide 32: Key Investment Highlights
Key Investment Highlights Disruptive technology platform: proprietary, allogeneic, “off-the-shelf” adult stem cells with predictable therapeutic properties Established late stage portfolio of distinct and advanced product candidates Strategic partnerships delivering clinical, manufacturing and comme
Mesoblast — 34th Annual J.P. Morgan Healthcare Conference 2016 on 6ix