Slide 1: Operational Highlights and Financial Results for the Period Ended March 31, 2017
For personal use only Operational Highlights and Financial Results for the Period Ended March 31, 2017 May 2017 |1
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
Slide 3: Agenda
For personal use only Agenda • Financial Results • Operational Update • Timelines |3
Slide 4: Our Mission, Vision and Focus
For personal use only Our Mission, Vision and Focus: Mesoblast is committed to bring to market its disruptive cellular medicines to treat serious and life-threatening illnesses |4
Slide 5: Financial Results
For personal use only Financial Results |5
Slide 6: Q3 FY 2017: Financial Highlights for the Three and Nine Months
Q3 FY 2017: Financial Highlights for the Three and Nine Months For personal use only Ending March 31, 2017 (US$m) Operational streamlining during the first nine months of FY17 enabled absorption of the incremental costs of the MPC-150-IM program in advanced chronic heart failure (CHF) Cash outfl
Slide 7: Operational Streamlining: Nine month update
Operational Streamlining: Nine month update (US$m) For personal use only From August 2016, the Company executed a range of cost reduction initiatives in order to offset the incremental costs of the MPC-150-IM program in FY17. $16.4 million of savings have been generated by these initiatives in the f
Slide 8: Q3 FY 2017: Cash position
Q3 FY 2017:Cash position For personal use only As of March 31, 2017, the Company had cash reserves of US$69.1 million The Company successfully completed a fully underwritten institutional placement of 26.25 million new shares (approx. 6% of issued capital) and raised approx. US$40 million from e
Slide 9: Operational Highlights
For personal use only Operational Highlights |9
Slide 10: Translating our Science into Commercial Reality
Translating our Science into Commercial Reality For personal use only Mesenchymal lineage immuno-selected precursors and Mesenchymal progeny cells (MLCs) Lineage Cells (MLCs) STRO-1/STRO-3 immuno-selection provides a Cytokines homogeneous and potent population of MLCs with receptors Chemokines R
Slide 11: Portfolio of Advanced Product Candidates
Portfolio of Advanced Product Candidates: Ideally Positioned for the 21st Century Cures Act Environment 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
Slide 12: MPC-150-IM Chronic Heart Failure (CHF) Program
For personal use only MPC-150-IM Chronic Heart Failure (CHF) Program Multi-billion dollar blockbuster potential | 12
Slide 13: MPC-150-IM: Targets the Most Serious and Life-Threatening Complications of Heart Failure
MPC-150-IM: Targets the Most Serious and Life-Threatening Complications of Heart Failure For personal use only Significant Burden of Illness and Unmet Need Globally, 17-45% of heart failure patients die within 1 year of hospital admission Majority die within 5 years of admission1 MPC-150-IM to
Slide 14: 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 ef
Slide 15: MPC-150-IM: Therapeutic Benefit on LV Remodeling Enhanced in Subjects with LVESV >100ml
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
Slide 16: MPC-150-IM: Durable 36 Months Protection Against HF-MACE
MPC-150-IM: Durable (36 Months) Protection Against HF-MACE1 in Phase 2 Trial Following Single Dose 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 patients1 treatment in patients with LVESV>100ml2 Over
Slide 17: 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 hosp
Slide 18: MPC-150-IM: Operational Update for Phase 3 Trial in NYHA Class II-III CHF
MPC-150-IM: Operational Update for Phase 3 Trial in NYHA Class II-III Advanced CHF Patients For personal use only Trial is planned to enroll approximately 600 patients In April 2017, a pre-specified interim futility analysis of the efficacy endpoint in the Phase 3 trial's first 270 patients was
Slide 19: MPC-150-IM: Operational Update for Phase 2b Trial in LVAD Patients
MPC-150-IM: Operational Update for Phase 2b Trial in Patients With End-Stage Heart Failure Who Have Received a Left For personal use only Ventricular Assist Device (LVAD) Study is sponsored and funded by the United States National Institutes of Health (NIH), and conducted by the NIH-funded Cardiot
Slide 20: Inflammatory diseases portfolio (MPC-300-IV)
For personal use only Our inflammatory diseases portfolio (MPC-300-IV) is a highly attractive emerging opportunity Multi-billion dollar blockbuster potential | 20
Slide 21: MPC-300-IV: Biological Refractory Rheumatoid Arthritis Market Landscape
MPC-300-IV: Biological Refractory Rheumatoid Arthritis (RA) Market Landscape For personal use only Market Size There are approx 6.0 million prevalent cases in the US, Japan, and EU5, with 2.9 million in the US alone in 20161,2 In 2016, sales of RA agents, predominantly TNF inhibitors exceeded $1
Slide 22: MPC-300-IV: Phase 2 Study Design
MPC-300-IV: Phase 2 Study Design For personal use only Phase 2 double-blind, randomized, placebo-controlled, dose-escalating study comparing a single infusion of two MPC doses (1M/kg and 2M/kg) with placebo in patients with active RA – Inadequate response to at least 1 anti-TNF +/- other biologics
Slide 23: MPC-300-IV: Phase 2 Trial 39-Week Results
MPC-300-IV: Phase 2 Trial 39-Week Results For personal use only The safety profile over 39 weeks was comparable among the placebo and both MPC treatment groups, with no cell-related serious adverse events Both MPC doses outperformed placebo at week 39 in each of ACR20/50/70 responses, as well as
Slide 24: Chronic Low Back Pain (CLBP) Due to Disc Degeneration
For personal use only Chronic Low Back Pain (CLBP) Due to Disc Degeneration Multi-billion dollar blockbuster potential | 24
Slide 25: MPC-06-ID: Potential Alternative to Invasive Surgery and Opioid Use
MPC-06-ID: Potential Alternative to Invasive Surgery and Opioid Use for Chronic Low Back Pain Patients For personal use only Significant Burden of Illness and Unmet Need Back pain causes more disability than any other condition1 Inflicts substantial direct and indirect costs on the healthcare sy
Slide 26: 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 DDD and unresponsive to conservative therapies (incl. opioids and epidural steroids) were evaluated in a blinded, randomized, placebo controlled Phase 2 trial Composite Respond
Slide 27: MPC-06-ID: Durable 36-Month Outcomes in Phase 2
MPC-06-ID: Durable 36-Month Outcomes in Phase 2 For personal use only Using the Intent to Treat (ITT) analysis, in line with FDA guidance for the ongoing Phase 3 trial, similar results were seen for the primary endpoint composite over 24 months, with 38% of the 6 million MPC group achieving this o
Slide 28: MPC-06-ID: Phase 3 Trial Update
MPC-06-ID: Phase 3 Trial Update For personal use only A 360-patient Phase 3 trial is recruiting well across sites in US and Australia On track to complete recruitment in 2017 FDA has provided written guidance: - Use of a composite primary endpoint is acceptable for approval - Agreed thresholds
Slide 29: Acute Graft vs Host Disease (aGVHD)
For personal use only Acute Graft vs Host Disease (aGVHD) Our nearest-term revenue product candidate: MSC-100-IV for steroid-refractory aGVHD | 29
Slide 30: MSC-100-IV: Acute Graft vs Host Disease (aGVHD)
MSC-100-IV: Acute Graft vs Host Disease (aGVHD) Serious and Life-Threatening Complication of Bone Marrow Transplants For personal use only Significant Burden of Illness and Unmet Need aGVHD - a severe immunological reaction occurring in BMT patients Steroid-refractory aGVHD (SR-aGVHD) patients h
Slide 31: MSC-100-IV for aGVHD: Product Development Strategy
MSC-100-IV for aGVHD: Product Development Strategy For personal use only Pediatric aGVHD Multi-center, single-arm, open-label, ongoing Phase 3 study in 60 pediatric patients with steroid-refractory aGVHD The pre-specified interim futility analysis of the trial’s primary endpoint was successfully
Slide 32: Timelines
For personal use only Timelines | 32
Slide 33: Upcoming Milestones and Catalysts for 2017
Upcoming Milestones and Catalysts for 2017 For personal use only MPC-150-IM − Phase 2B complete trial enrollment for Class IV (Mid-17) − Phase 2B data read-out Class IV (4Q CY17) MSC-100-IV − Phase 3 complete trial enrollment and top-line data read-out (2H CY17) MPC-06-ID − Phase 3 complete tr
Slide 34: Thank you
For personal use only Thank you | 34