Mesoblast Limited · ASX:MSB

Mesoblast — Shareholder Meeting (November 2016)

32 slides from Mesoblast's shareholder meeting presentation, Nov 22, 2016

  1. Slide 1: Annual General Meeting

    For personal use only Annual General Meeting Melbourne 22 November 2016

  2. 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

  3. Slide 3: Our Competitive Strengths

    Our Competitive Strengths Disruptive multi-platform technologies: proprietary, allogeneic, “off-the-shelf” adult stem cells For personal use only with predictable therapeutic properties Industry-leading late stage portfolio of distinct and advanced product candidates addressing a significant unmet m

  4. Slide 4: FY 2016 and Q1 2017 - Highlights

    FY 2016 and Q1 2017 - Highlights  Phase 3 trial in acute graft versus host disease (aGVHD) has completed For personal use only successful pre-specified interim futility analysis and is recruiting towards completion  Positive Phase 2 trial in biologic refractory rheumatoid arthritis (RA) patients i

  5. Slide 5: Extensive Intellectual Property Portfolio – Patents & Patent Applications Across 72 Families

    Extensive Intellectual Property Portfolio – 728 Patents & Patent Applications Across 72 Families# For personal use only Composition of matter (COM) Specific therapeutic Complementary technologies and manufacturing process applications and additional candidates 147 patents or patent applications 429

  6. Slide 6: FY 2016 and Q1 2017, Peer-Reviewed Articles and Selected Presentations

    FY 2016 and Q1 2017, Peer-Reviewed Articles and Selected Presentations MPC-150-IM and MPC-300-IV: Phase 2 results in CHF and Type 2 Diabetes published in For personal use only  Circulation Research and Diabetes Care respectively  MSC-100-IV: Data from 241 children presented at annual scientific me

  7. Slide 7: Tier Product Portfolio Overview

    For personal use only Tier 1 Product Portfolio Overview 7

  8. Slide 8: Diversified Pipeline of Product Candidates for High Unmet Needs

    Diversified Pipeline of Product Candidates for High Unmet Needs First Product on market - Three Tier 1 Product Candidates in Phase 3 For personal use only 8

  9. Slide 9: MPC-150-IM: Chronic Heart Failure (CHF) – Market Opportunity

    MPC-150-IM: Chronic Heart Failure (CHF) – Market Opportunity MPC-150-IM is in development for patients with New York Heart Association Class II-IV CHF For personal use only  5.7m patients (prevalence rate of 2% of the population) diagnosed with CHF in the US1 Market  915,000 new cases diagnosed in

  10. Slide 10: Change from Baseline in LVESV and LVEDV at Months 6 and 12 in Phase 2 Trial

    Change from Baseline in LV End-Systolic Volume (LVESV) and LV End Diastolic Volume (LVEDV) Obtained Using 2-D Echo at Months 6 and 12 Post Treatment in Phase 2 Trial For personal use only Source: Circ Res. 2015; 117:576-584. Perin E et al. A Phase II Dose-Escalation Study of Allogeneic Mesenchymal P

  11. Slide 11: MPC-150-IM: Therapeutic Benefit on LV Remodelling in Subjects with LVESV >100ml

    MPC-150-IM: Phase 2 Therapeutic Benefit on LV Remodelling is Amplified in Subjects with LVESV >100ml For personal use only Baseline LVESV > 100 mL Baseline LVESV > 100ml Baseline LVESV > 100 mL P < 0.02 (Month 6 – Baseline; mL) (Month 6 – Baseline; mL) P < 0.03 P < 0.05 (Month 6 – Baseline; %) ∆ LVE

  12. Slide 12: MPC-150-IM: Results Show Promise in the Prevention of HF-MACE

    MPC-150-IM: Phase 2 Results Show Promise in the Prevention of HF-MACE* HF-MACE Kaplan-Meier Curve over 36 months HF-MACE Kaplan-Meier Curve over 36 months For personal use only following treatment in all patients1 following treatment in patients with LVESV>100ml2  Over 36 months, patients receiving

  13. Slide 13: MPC-150-IM: Phase 3 Trial Targets Advanced Heart Failure

    MPC-150-IM: Phase 3 Trial Targets Advanced Heart Failure For personal use only  Patients with large baseline LVESV and advanced heart failure are at highest risk of HF-MACE – Have increased likelihood of having recurrent HF hospitalizations – Existing therapies are inadequate and economic burden is

  14. Slide 14: MPC-150-IM: Phase 3 Trial Operational Update

    MPC-150-IM: Phase 3 Trial Operational Update For personal use only  Phase 3 trial for 600 patients with advanced heart failure is recruiting well across North American sites; over 300 patients enrolled  After reviewing patient data in April and October 2016, the trial’s DSMB has maintained its rec

  15. Slide 15: MPC-300-IV: Biologic Refractory Rheumatoid Arthritis (RA) – Market Opportunity

    MPC-300-IV: Biologic Refractory Rheumatoid Arthritis (RA) – Market Opportunity Ongoing randomized, controlled Phase 2 Trial in 48 patients with biologic refractory rheumatoid For personal use only arthritis, comparing two doses of MPC-300-IV against placebo  There are approximately 5.3 million prev

  16. Slide 16: MPC-300-IV: Study Design for Phase 2 Trial in Biologic Refractory RA

    MPC-300-IV: Study Design for Phase 2 Trial in Biologic Refractory RA For personal use only  Phase 2 double-blind, randomized, placebo-controlled, dose-escalating study comparing a single MPC infusion of either 1M/kg or 2M/kg against placebo in 48 patients with active RA and inadequate response to a

  17. Slide 17: MPC-300-IV: Operational Update

    MPC-300-IV: Operational Update  The Phase 2 trial of Mesoblast’s intravenous product candidate, MPC-300-IV, in For personal use only biologic refractory rheumatoid arthritis has completed enrollment  A single intravenous MPC infusion in biologic refractory RA patients was without serious adverse e

  18. Slide 18: MPC-300-IV: Key Efficacy Responses in Biologic Refractory RA

    MPC-300-IV: Key Efficacy Responses at Week 12 in Biologic Refractory RA All Subjects Subgroup with Prior Use of 1-2 For personal use only Biologics Placebo 1M/kg 2M/kg p-value Placebo 1M/kg 2M/kg p-value 2M/kg vs 2M/kg vs placebo placebo N=16 N=16 N=16 N=9 N=10 N=11 ACR70 0% 20% 27% 0.04 0% 20% 36%

  19. Slide 19: MPC-300-IV: One Dose Provides Efficacy in the Biologic Refractory Population

    MPC-300-IV: One Dose Provides Compelling Efficacy at 12 Weeks in the Biologic Refractory Population ACR70 @ 3 Months For personal use only 40% Marketed Pipeline 36% 35% 30% 27% 25% 20% 22% 15% 14% 10% 5% 11% 6% 0% Inhibitors of IL-6, MPC 2m/kg CD80/CD86, CD20, JAK 1 Jak Inhibitors2 MPC 2m/kg (1-2 bi

  20. Slide 20: MPC-06-ID: CLBP Due to Degenerative Disc Disease – Market Opportunity

    MPC-06-ID: CLBP Due to Degenerative Disc Disease – Market Opportunity MPC-06-ID is in development for the treatment of chronic low back pain (CLBP) lasting >6 For personal use only months as a result of moderate degenerative intervertebral disc disease  Over approximately 7m patients in the US are

  21. Slide 21: MPC-06-ID: Phase 2 Trial

    MPC-06-ID: Phase 2 Trial  100 patients with >6 months of CLBP due to DDD and unresponsive to conservative For personal use only therapies (including opioids and epidural steroids) were evaluated in a randomized, placebo controlled Phase 2 trial  Visual Analog Scale (VAS) scored from 0-100, evaluat

  22. Slide 22: MPC-06-ID: Phase 2 Trial Results Presented At Spine Intervention Society 2016

    MPC-06-ID: Phase 2 Trial Results Presented At Spine Intervention Society 2016 Annual Meeting For personal use only % Patients with Treatment Response % Patients with Treatment Response at 6 & 12 Months at 12 & 24 Months 50.0% 50.0% 44.4%a,b 45.0% 45.0% a) p=0.044 6M MPC vs. saline d) p=0.090 6M MPC

  23. Slide 23: MPC-06-ID: Phase 3 Trial Operational Update

    MPC-06-ID: Phase 3 Trial Operational Update For personal use only  Current 360 patient Phase 3 trial is recruiting well across US sites • The 24-month results from the Company’s 100-patient Phase 2 trial of MPC-06- ID for treatment of chronic low back pain were presented at the 24th Annual Scientif

  24. Slide 24: MSC-100-IV / TEMCELL HS Inj.: Acute Graft vs Host Disease

    MSC-100-IV / TEMCELL® HS Inj. : Acute Graft vs Host Disease 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) For personal use only  ~30,000 allogeneic BMTs performed globally (~20K

  25. Slide 25: MSC-100-IV: Product Development Strategy

    MSC-100-IV: Product Development Strategy  Manufacturing For personal use only – Optimised process – modernized and harmonized – Commercial readiness for launch  Pediatrics – Complete targeted Phase 3, 60 patient open label clinical trial in SR-aGVHD for accelerated approval pathway (US) – Market d

  26. Slide 26: MSC-100-IV North America aGVHD Market Opportunity

    MSC-100-IV North America aGVHD Market Opportunity Target Population 2015 Source For personal use only • US: estimated from 2013 CIBMTR (table 6) Allogeneic Hematopoietic Stem Cell Transplants 10,200 • Canada: estimate based on general population size and US transplant activity per 10m Epidemiology A

  27. Slide 27: MSC-100-IV: Trial in Children with Steroid Refractory Acute GVHD

    MSC-100-IV: Phase 3 Trial in Children with Steroid Refractory Acute Graft vs Host Disease (SR-aGVHD) For personal use only MSC-100-IV as first line therapy in children with MSC-100-IV in children with SR-aGVHD who failed SR-aGVHD (Day 28 response) multiple other modalities (Day 100 survival) Open-la

  28. Slide 28: MSC-100-IV: Pediatric GVHD001/GVHD002 Phase 3 Study

    MSC-100-IV: Graft vs Host Disease: Pediatric GVHD001/GVHD002 Phase 3 Study  Phase 3 study ongoing, ~ 40 sites planned1 For personal use only – Multi-center, Single-Arm, Open-Label to evaluate efficacy and safety to day 100 (study 001) and from day 100 to day 180 (study 002) – At least 60 pediatric

  29. Slide 29: GVHD001 Interim Futility Analysis Method and Results - November 2016

    GVHD001 Interim Futility Analysis Method and Results - November 2016 For personal use only  Predefined Bayesian futility rule that determined the predictive probability of success using the primary endpoint of Day 28 overall response  Method determined the likelihood of obtaining a statistically s

  30. Slide 30: MSC-100-IV: Concentrated High Volume Centers Provide Low Cost Commercial Structure

    MSC-100-IV: Concentrated High Volume Centers Provide Compelling Low Cost Commercial Structure1 For personal use only Top Pediatric Transplant Centers Top Adult Transplant Centers  There are 65 centers in the U.S. that conduct  There are 108 centers in the U.S. that allogeneic transplants in pediat

  31. Slide 31: Tier 1 Product Candidate Deliverables (Calendar Year)

    Tier 1 Product Candidate Deliverables (Calendar Year) 2016 2017 For personal use only Product Candidate Programs Milestones Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Phase 3 Enrollment Complete Class II and III Heart Failure MPC-150-IM Interim Results Class IV Heart Failure Requiring Phase 2b trial results LVAD Top l

  32. Slide 32: Thank You and Questions

    For personal use only Thank You and Questions

Mesoblast — Shareholder Meeting (November 2016) on 6ix