Mesoblast Limited · ASX:MSB

Mesoblast — Q1 FY2018 Earnings Presentation

45 slides from Mesoblast's earnings presentation, Nov 15, 2017

  1. Slide 1: Cellular Medicines for Intractable Serious and Life-Threatening Diseases

    For personal use only Cellular Medicines for Intractable Serious and Life-Threatening Diseases November 2017 ASX: MSB Nasdaq: MESO

  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 Mission

    For personal use only Our Mission: Mesoblast is committed to bring to market disruptive cellular medicines to treat serious and life-threatening illnesses |3

  4. Slide 4: Investment Proposition: Building a Leading Franchise of Cellular Medicines

    Investment Proposition: Building a Leading Franchise of Cellular Medicines For personal use only  Disruptive Cellular Technology Platform  Commercial Translation Capabilities  Advanced Pipeline of Cellular Medicines  Targeting Serious or Life-Threatening Conditions with Unmet Needs |4

  5. Slide 5: Disruptive Cellular Medicine Platform

    Disruptive Cellular Medicine Platform1-4 For personal use only  STRO-1+ Mesenchymal Precursor Cells (MPCs) are at the apex of the hierarchy of Mesenchymal Lineage cells  STRO-1/STRO-3 immuno-selection provides a homogeneous population of MPCs with unique receptors that respond to activating inflam

  6. Slide 6: Commercial Translation Capabilities: Scalable, Industrialized Manufacturing

    Commercial Translation Capabilities: Technology Positioned for Scalable, Industrialized Manufacturing For personal use only  Immune privileged nature of STRO-1+ MPCs enables allogeneic “off the shelf” product candidates  Culture expansion scalable to produce commercial quantities of potent and rep

  7. Slide 7: Portfolio of Advanced Product Candidates

    Portfolio of Advanced Product Candidates: Three Tier 1 Product Candidates in Phase 3 For personal use only 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-

  8. Slide 8: The 21st Century Cures Act: Expedited Approval Path (RMAT)

    The 21st Century Cures Act (“Cures Act”): Legislation for An Expedited Approval Path for Cellular Medicines Designated as For personal use only Regenerative Medicine Advanced Therapies (RMAT)  Cellular medicines may be designated as regenerative advanced therapies, if they are intended to treat, mo

  9. Slide 9: Intellectual Property

    Intellectual Property: An Extensive Portfolio Covering Composition of Matter, Manufacturing, and Therapeutic For personal use only Applications of Potent Immuno-selected Mesenchymal Lineage Precursors and Progeny ~ 800 Patents and patent applications across 69 Patent Families. Protection across majo

  10. Slide 10: Diverse Pipeline of Cellular Medicines

    For personal use only Diverse Pipeline of Cellular Medicines | 11

  11. Slide 11: Acute Graft vs Host Disease (aGVHD)

    For personal use only Acute Graft vs Host Disease (aGVHD) MSC-100-IV for Steroid-Refractory aGVHD | 11

  12. Slide 12: MSC-100-IV: Market Opportunity for aGVHD

    MSC-100-IV: Market Opportunity for aGVHD For personal use only  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 patient

  13. Slide 13: MSC-100-IV for aGVHD: Product Development Strategy

    MSC-100-IV for aGVHD: Product Development Strategy 1. Target pediatric patients with SR-aGVHD first For personal use only  Extensive safety and efficacy data generated and published with MSC-100-IV in children with SR-aGVHD1  High economic burden in treatment of children with SR-aGVHD  Fast-track

  14. Slide 14: MSC-100-IV: Expanded Access Program

    MSC-100-IV: Expanded Access Program Overall Day 28 Response in Pediatric aGVHD Patients Receiving MSC-100-IV as First-line or Salvage Therapy After Failing Steroids For personal use only Population: steroid-refractory 100% aGVHD pediatric patients  241 pediatric patients undergoing HSCT were 80% 73

  15. Slide 15: MSC-100-IV: Expanded Access Program Survival Correlation

    MSC-100-IV: Expanded Access Program Correlation of Day 28 Overall Response with Day 100 Survival, Using MSC-100-IV as First-line or Salvage Therapy After Failing Steroids and/or Additional Treatments For personal use only MSC-100-IV in Children with SR-aGVHD who failed multiple other modalities - Su

  16. Slide 16: MSC-100-IV: Prior Clinical Results Support Ongoing Phase 3 Trial

    MSC-100-IV: Prior Clinical Results1 Support Ongoing Phase 3 Trial in Children with Steroid For personal use only Refractory Acute GVHD (SR-aGVHD) MSC-100-IV as first line therapy in children with SR-aGVHD  These combined study results demonstrate that compared with placebo control patients, MSC-100

  17. Slide 17: MSC-100-IV: Phase 3 Pediatric Trial as First-line Therapy

    MSC-100-IV: Phase 3 Pediatric Trial as First-line Therapy in aGVHD After Failing Steroids For personal use only  Multi-center, Single-Arm, Open-Label to evaluate efficacy and safety to day 100 (001) and from day 100 to day 180 (002)  Up to 60 pediatric patients (2 months to 17 years)  aGVHD follo

  18. Slide 18: MPC-150-IM Chronic Heart Failure (CHF) Program

    For personal use only MPC-150-IM Chronic Heart Failure (CHF) Program | 18 | 18

  19. Slide 19: MPC-150-IM: Targeting Patients with Worsening HF

    MPC-150-IM: Targeting Patients with Worsening HF Despite Optimal Standard of Care For personal use only Common Treatment Pathway in CHF1 Early  ACEI or ARB MPC-150-IM targets HFrEF  Statins NYHA Advanced-stage HF  Beta blockers and End-stage HF patients3  Re-vascularization or valvular surgery P

  20. Slide 20: MPC-150-IM: Class IV Market Opportunity

    MPC-150-IM: Class IV Market Opportunity For personal use only  250K – 300K patients/yr suffer from advanced systolic HF (NYHA Class IV)1 Burden of  50k patients/yr have end-stage heart failure  Despite optimal medical therapy, 1-year mortality exceeds 50% in end-stage Illness heart failure patien

  21. Slide 21: LVAD MPC Pilot Trial Proof of Concept

    LVAD MPC Pilot Trial Proof of Concept: Evaluating 25M MPCs as Adjunct to LVAD1-2 For personal use only Assessed for eligibility (N=81)  57.4 yrs (±13.6) Excluded (n=51)  83% Male Randomized (n=30)  LVEF 18.1% (± 4.3)  37% Ischemic, 63% Non-ischemic  67% Destination, 33% MPC (n=20) Control (n=10

  22. Slide 22: LVAD MPC Pilot Trial Outcomes

    LVAD MPC Pilot Trial Outcomes: 25M MPCs Increased Ability to be Weaned off LVADs and Increased Short- For personal use only Term Survival1  No cell-related safety events observed LVAD MPC Pilot Trial: 12 Month Survival  Median time to first hospitalization was 91 days in the MPC group vs 51 days i

  23. Slide 23: MPC-150-IM: Phase 2b Trial in End-Stage Heart Failure with LVADs

    MPC-150-IM: Phase 2b Trial Evaluating 150M MPCs in End-Stage Heart Failure Patients with LVADs For personal use only  The 159-patient, double-blind, placebo-controlled 2:1 randomized trial, is evaluating the safety and efficacy of injecting MPC-150-IM into the native myocardium of LVAD recipients 

  24. Slide 24: MPC-150-IM: Commercial Strategy

    MPC-150-IM: Commercial Strategy For personal use only  Leverage data for potential earlier market entry opportunity for MPC-150-IM in end-stage heart failure patients (more than 5K/yr)  evaluate potential to reduce LVAD morbidity, increase survival and increase LVAD use as destination therapy (DT)

  25. Slide 25: MPC-150-IM: Class III Heart Failure Market Opportunity

    MPC-150-IM: Class III Heart Failure Market Opportunity  Globally, 17-45% of heart failure patients die within 1 year of hospital admission For personal use only Burden of  Majority die within 5 years of admission1 Illness  MPC-150-IM to target advanced HFrEF NYHA Class II-III with the objective o

  26. Slide 26: MPC-150-IM: Durable Protection Against HF-MACE in Phase 2 Trial

    MPC-150-IM: Durable (36 Months) Protection Against HF-MACE1 in Phase 2 Trial Following Single Dose in NYHA Class II/III With Reduced Ejection Fraction For personal use only % HF-MACE Kaplan-Meier Curve over HF-MACE Kaplan-Meier Curve over 36 months following 36 months following treatment in all pati

  27. Slide 27: MPC-150-IM: Phase 3 Trial Targets Advanced Heart Failure

    MPC-150-IM: Phase 3 Trial Targets Advanced Heart Failure For personal use only NYHA class II/III patients with large baseline LVESV and advanced heart failure are at highest risk of heart failure-related major adverse cardiac events (HF-MACE)  Have increased likelihood of having recurrent HF hospit

  28. Slide 28: MPC-150-IM: Operational Update for Phase 3 Trial

    MPC-150-IM: Operational Update for Phase 3 Trial in NYHA Class II-III Advanced CHF Patients For personal use only  Trial has enrolled more than 400 of approximately 600 patients  In April 2017, a pre-specified interim futility analysis of the efficacy endpoint in the Phase 3 trial's first 270 pati

  29. Slide 29: Chronic Low Back Pain (CLBP) Due to Disc Degeneration

    For personal use only Chronic Low Back Pain (CLBP) Due to Disc Degeneration | 29 | 29

  30. Slide 30: MPC-06-ID: Non-Opioid Alternative for Chronic Low Back Pain

    MPC-06-ID: a non-Opioid alternative for Chronic Low Back Pain due to Degenerative Disc Disease For personal use only  Back pain causes more disability than any other condition1 Burden of  Inflicts substantial direct and indirect costs on the healthcare system1, Illness including excessive use of o

  31. Slide 31: The Opioid Epidemic

    The Opioid Epidemic For personal use only • 50% of opioid prescriptions are for chronic low back pain (CLBP) • Over 1,000 people are treated in U.S. emergency departments everyday for misusing prescription opioids • Over 33,000 people in the U.S. died of prescription opioid related overdoses in 2016

  32. Slide 32: MPC-06-ID: Phase 2 Trial Results Support Phase 3 Program

    MPC-06-ID: Phase 2 Trial Results Support Phase 3 Program For personal use only  100 patients with >6 months of CLBP due to Composite Responders at DDD and unresponsive to conservative both 12 & 24 Months - PP1 therapies (incl. opioids and epidural steroids) were evaluated in a blinded, 50.0% random

  33. Slide 33: MPC-06-ID: Phase 2 Results: Reduction in Low Back Pain Through 36 Months

    MPC-06-ID: Phase 2 Clinical Trial Results: Substantial Reduction in Low Back Pain Through 36 Months After Single Dose For personal use only 𝝓𝝓 𝝓𝝓 * 𝝓𝝓 Minimal * * Difference2 § # § Substantial Clinical Improvement2 # p ≤ 0.05 *𝝓𝝓 MPC MPC 6M vs. Saline 18M vs. Saline Primary # MPC 6M vs. HA…

  34. Slide 34: MPC-06-ID: Reduction in Functional Disability Through 36 Months

    MPC-06-ID: Phase 2 Clinical Trial Results Reduction in Functional Disability Through 36 Months After Single Dose For personal use only p ≤ 0.05 𝝓𝝓 *𝝓𝝓 MPC MPC 6M vs. Saline 18M vs. Saline 𝝓𝝓 𝝓𝝓 * * § MPC 18M vs. HA p = 0.054 § * Minimal Difference2 FDA Functional Improvement Threshold…

  35. Slide 35: MPC-06-ID: Phase 3 Trial Update

    MPC-06-ID: Phase 3 Trial Update For personal use only  A 360-patient Phase 3 trial across U.S. and Australian sites  Targeted to complete recruitment early Q1 CY18  FDA has provided written guidance:  Use of a composite primary endpoint at 12 and 24 months is acceptable  Agreed thresholds for p

  36. Slide 36: Our inflammatory diseases portfolio (MPC-300-IV)

    For personal use only Our inflammatory diseases portfolio (MPC-300-IV) ||36 36

  37. Slide 37: MPC-300-IV: Cellular product candidate responding to multiple inflammatory signals

    MPC-300-IV: Cellular product candidate that responds to multiple inflammatory signals For personal use only Phase 2 Clinical Data in Immune Mediated Diseases  60 patients, type 2 diabetes with inadequately controlled glucose:  Randomized, placebo controlled dose-ranging study completed  Positive

  38. Slide 38: MPC-300-IV: Biological Refractory Rheumatoid Arthritis Market Opportunity

    MPC-300-IV: Biological Refractory Rheumatoid Arthritis (RA) Market Opportunity For personal use only  There are approx 6.0 million cases in the US, Japan, and EU5, with 2.9 million Burden of in the US alone in 20161,2 illness  ~1/3 of RA patients do not respond or cannot tolerate TNF inhibitors4 T

  39. Slide 39: MPC-300-IV: Phase 2 Study Design

    MPC-300-IV: Phase 2 Study Design For personal use only  Inclusion Criteria  Inadequate response to at least 1 anti-TNF +/- other biologics  On a stable regimen methotrexate for >4 months +/- DMARDs for >3 months  Randomisation Scheme  MPC 1 million cells/kg (N=16 )  MPC 2 x million cells/kg (N

  40. Slide 40: MPC-300-IV: Phase 2 Trial in Biologic Refractory Rheumatoid Arthritis

    MPC-300-IV: Phase 2 trial in biologic refractory Rheumatoid Arthritis shows early and durable effects after single dose For personal use only  Infusions were well-tolerated and there were no treatment-related serious adverse events reported, with the safety profile comparable among the placebo and

  41. Slide 41: Financials & Milestones

    For personal use only Financials & Milestones | 41

  42. Slide 42: Q1 FY18 Cash Position and Cash Flows

    Q1 FY18 Cash Position and Cash Flows for three months ending 30 Sep 2017 (US$m) For personal use only 30 Sep 2017 30 Sep 2016 $Change Operating cash outflows (20.3) (20.8) 0.5 Investing cash outflows (0.6) (0.3) (0.3) Financing cash inflows/(outflows) 38.4 (0.1) 38.5 Forex (0.3) 0.6 (0.9) Net increa

  43. Slide 43: Q1 FY18 - Profit and Loss for the three months ending Sep 2017

    Q1 FY18 - Profit and Loss for the three months ending 30 Sep 2017 (US$m) For personal use only For the three months ending 30 Sep 2017 30 Sep 2016 $ Change % Revenue 1.2 0.4 0.8 197% Research and Development (15.4) (14.0) (1.4) (10%) Manufacturing Commercialization (0.9) (3.3) 2.4 73% Management & A

  44. Slide 44: Q1 FY18 - Profit and Loss for the three months ending Sep 2017

    Q1 FY18 - Profit and Loss for the three months ending 30 Sep 2017 (US$m) For personal use only For the three months ending 30 Sep 2017 30 Sep 2016 $ Change % Revenue 1.2 0.4 0.8 197% Research and Development (15.4) (14.0) (1.4) (10%) Manufacturing Commercialization (0.9) (3.3) 2.4 73% Management & A

  45. Slide 45: Targeted Upcoming Milestones and Catalysts

    Targeted Upcoming Milestones and Catalysts For personal use only  MSC-100-IV for Pediatric Acute GVHD  Phase 3 expected to complete enrollment (Q4 CY17)  Day 28 primary endpoint data read-out (Q1 CY18)  Day 100 survival data (Q2 CY18)  MPC-150-IM for Advanced and End-Stage Heart Failure  Phase

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