Mesoblast Limited · ASX:MSB

Mesoblast — J.P. Morgan 2018 Healthcare Conference

43 slides from Mesoblast's conference presentation, Jan 12, 2018

  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 J.P. Morgan 2018 Healthcare Conference January 2018 Nasdaq: MESO ASX: MSB

  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: December 2017 FDA RMAT Designation for Heart Failure Patients With LVADs

    December 2017 Mesoblast Received FDA RMAT Designation For Its Cell Therapy In Heart Failure Patients With Left Ventricular Assist Devices (LVADs) For personal use only  RMAT designation grant was based on the completed study data set and related analyses of a 30-patient randomized, blinded, placebo

  10. Slide 10: Intellectual Property: Extensive Portfolio

    Intellectual Property: An Extensive Portfolio Covering Mesenchymal Lineage Precursors and Progeny For personal use only • Composition of Matter, Manufacturing, and Therapeutic Applications of Potent Immuno-selected mesenchymal lineage precursor and stem cells • 800 Patents and patent applications ac

  11. Slide 11: Patent Settlement and License Agreement With TiGenix

    December 2017 Mesoblast Concluded Patent Settlement and License Agreement With TiGenix For personal use only  Mesoblast granted TiGenix exclusive access to certain of its patents to support global commercialization of the adipose-derived mesenchymal stem cell (MSC) product Cx601 limited to the loca

  12. Slide 12: Diverse Pipeline of Cellular Medicines

    For personal use only Diverse Pipeline of Cellular Medicines | 12

  13. Slide 13: Acute Graft vs Host Disease (aGVHD)

    For personal use only Acute Graft vs Host Disease (aGVHD) MSC-100-IV for Steroid-Refractory aGVHD | 13

  14. Slide 14: 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

  15. Slide 15: 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

  16. Slide 16: 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

  17. Slide 17: 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

  18. Slide 18: MSC-100-IV: Pediatric Phase 3 Trial Completed Enrollment

    MSC-100-IV: Phase 3 Pediatric Trial Completed Enrollment 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

  19. Slide 19: MPC-150-IM Chronic Heart Failure (CHF) Program

    For personal use only MPC-150-IM Chronic Heart Failure (CHF) Program | 19 | 19

  20. Slide 20: 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

  21. Slide 21: MPC-150-IM: Product Development Strategy Following RMAT Designation

    MPC-150-IM: Product Development Strategy Following RMAT Designation for Heart Failure Patients With Left Ventricular Assist Devices (LVADs) For personal use only 1. Leverage data for potential near term market entry opportunity for MPC- 150-IM in end-stage heart failure patients with LVADs 1. Broade

  22. Slide 22: 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

  23. Slide 23: MPC-150-IM Proof of Concept Trial: Trial Design

    MPC-150-IM Proof of Concept Randomized Controlled Phase 2 Trial Which Successfully Supported RMAT Designation: Trial Design For personal use only Assessed for eligibility (N=81)  57.4 yrs (±13.6)  83% Male Excluded (n=51)  LVEF 18.1% (± 4.3) Randomized (n=30)  37% Ischemic, 63% Non-ischemic  67

  24. Slide 24: MPC-150-IM Proof of Concept Trial: Trial Results

    MPC-150-IM Proof of Concept Randomized Controlled Phase 2 Trial Which Successfully Supported RMAT Designation: Trial Results For personal use only  25M cell dose, no cell-related safety events observed  Median time to first hospitalization was 91 days in the MPC group vs 51 days in the control gro

  25. Slide 25: 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  The 159-patient, double-blind, placebo-controlled 2:1 randomized trial, is evaluating the safety and For personal use only efficacy of injecting MPC-150-IM into the native myocardium of LVAD recipients 

  26. Slide 26: 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

  27. Slide 27: MPC-150-IM: Phase 2 Randomized Placebo Controlled Trial

    MPC-150-IM: Phase 2 Randomized Placebo Controlled Trial in 60 Patients HF Class II/III and LVEF<40% For personal use only Objectives LVESV Month 6 - Baseline LVEDV Month 6 - Baseline  Identify a dose response and an optimal therapeutic dose  Identify optimal target population for therapeutic effec

  28. Slide 28: MPC-150-IM: Therapeutic Benefit on LV Remodeling

    MPC-150-IM: Therapeutic Benefit on LV Remodeling Enhanced in Phase 2 Subjects with LVESV >100ml For personal use only  Placebo corrected benefit of 150M cell dose on cardiac volumes and ejection fraction at 6 months was greatest in patients with more advanced heart failure as defined by ∆ ∆ baselin

  29. Slide 29: MPC-150-IM: Durable 36-Month Protection Against HF-MACE

    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

  30. Slide 30: 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

  31. Slide 31: 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

  32. Slide 32: Chronic Low Back Pain (CLBP) Due to Disc Degeneration

    For personal use only Chronic Low Back Pain (CLBP) Due to Disc Degeneration | 32 | 32

  33. Slide 33: 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

  34. Slide 34: 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

  35. Slide 35: 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 Composite Responders at both 12 & 24 Months - PP1  100 patients with >6 months of CLBP due to DDD and unresponsive to conservative 50.0% therapies (incl. opioids and epidural 45.0% 40.7% 40.0% steroids) were evaluated in

  36. Slide 36: MPC-06-ID: Phase 2 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…

  37. Slide 37: 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…

  38. Slide 38: 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

  39. Slide 39: Our Inflammatory Diseases Portfolio (MPC-300-IV)

    For personal use only Our inflammatory diseases portfolio (MPC-300-IV) ||39 39

  40. Slide 40: MPC-300-IV: Evaluated in Immune Mediated Diseases

    MPC-300-IV: Being evaluated in immune mediated diseases where the cellular product candidate responds to multiple inflammatory signals by releasing factors that modulate the immune response For personal use only Phase 2 Clinical Data in Immune Mediated Diseases  60 patients, type 2 diabetes with in

  41. Slide 41: 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

  42. Slide 42: Milestones

    For personal use only Milestones | 42

  43. Slide 43: Targeted Upcoming Milestones and Catalysts

    Targeted Upcoming Milestones and Catalysts For personal use only  MSC-100-IV for Pediatric Acute GVHD − 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 2B Class IV trial six-month primary endpoint reache

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