MESOBLAST LTD — AGM Information 2016

Nov 21, 2016

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Annual General Meeting

Melbourne 22 November 2016

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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, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this Quarterly Report on Form 6-K are forward-looking statements. Words such as, but not limited to, “believe,” “expect,” “anticipate,” “estimate,” “intend,” “plan,” “targets,” “likely,” “will,” “would,” “could,” and similar expressions or phrases identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and future events and financial trends that we believe may affect our financial condition, results of operation, business strategy and financial needs. These statements may relate to, but are not limited to: expectations regarding the safety or efficacy of, or potential applications for, Mesoblast's adult stem cell technologies; expectations regarding the strength of Mesoblast's intellectual property, the timeline for Mesoblast's regulatory approval process, and the scalability and efficiency of manufacturing processes; expectations about Mesoblast's ability to grow its business and statements regarding its relationships with JCR Pharmaceuticals Co., Ltd, and Lonza and future benefits of those relationships; statements concerning Mesoblast's share price or potential market capitalization; and statements concerning Mesoblast's capital requirements and ability to raise future capital, among others. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. You should read this presentation together with our financial statements and the notes related thereto, as well as the risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast's actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, include, without limitation: risks inherent in the development and commercialization of potential products; uncertainty of clinical trial results or regulatory approvals or clearances; government regulation; the need for future capital; dependence upon collaborators; and protection of our intellectual property rights, among others. Accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

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2

Our Competitive Strengths

Disruptive multi-platform technologies: proprietary, allogeneic, “off-the-shelf” adult stem cells with predictable therapeutic properties

Industry-leading late stage portfolio of distinct and advanced product candidates addressing a significant unmet medical need

Strategic partnerships delivering clinical, manufacturing and commercial capabilities

Scalable, cost-efficient manufacturing capabilities

Intellectual property leadership covering compositions, uses and manufacturing

Experienced management team

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3

FY 2016 and Q1 2017 - Highlights

• Phase 3 trial in acute graft versus host disease (aGVHD) has completed successful pre-specified interim futility analysis and is recruiting towards completion

• Positive Phase 2 trial in biologic refractory rheumatoid arthritis (RA) patients identifies new blockbuster potential market

• Phase 3 trials in advanced chronic heart failure (CHF) and chronic low back pain due to disc degeneration (CLBP) recruiting toward major value inflexion points

• Mesoblast’s licensee in Japan, JCR Pharmaceuticals Co. Ltd., launched the first allogeneic regenerative medicine in Japan, TEMCELL, for aGVHD in children and adults

• Technology platform continues to demonstrate favorable safety profile and efficacy signals

• Prudent use of cash used to advance Tier 1 product candidates (CHF, RA, CLBP and aGVHD)

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4

Extensive Intellectual Property Portfolio – 728 Patents & Patent Applications Across 72 Families[# ]

Composition of matter (COM) and manufacturing process

Specific therapeutic applications

Complementary technologies and additional candidates

147 patents or patent applications Valid through 2024-2035*

429 patents or patent applications Valid through 2035*

152 patents or patent applications Valid through 2024-2032*

• MPCs– 64 patents or patent applications related to MPC composition of matter or methods of isolation, expansion and manufacture of MPCs – start to expire 2020 and extend to 2029

• MSCs – 49 granted patents or patent applications related to MSC composition of matter and manufacture of MSCs –start to expire 2018 and extend to 2035

• DPSCs – 34 patents or patent applications – start to expire 2021 and extend to 2024

In the last financial year we had 35 new patents granted including 7 in the US, 4 in Europe, 5 in Japan and 19 in other jurisdictions

• Immunologic / inflammatory disorders – 130 patents or patent applications – start to expire 2019 and extend to 2035

• Cell-based complementary technologies – 67 patents or patent applications - start to expire 2017 and extend to 2030

• Cardiovascular disorders –

• SDF-1 – 37 patents or patent applications –start to expire 2027 and extend to 2032

• 70 patents or patent applications – start to expire 2018 and extend to 2024

• Factors and agents for cardiovascular and other fibrotic indications – 48 patents or patent applications –start to expire 2021 and extend to 2023

• Orthopedic disorders – 66 patents or patent applications – start to expire 2017 and extend to 2032

• Oncology / hematology – 99 patents or patent applications – start to expire 2019 and extend to 2030

• Other therapeutic applications – 64 patents or patent applications – start to expire 2027 and extend to 2032

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• *Excludes possible patent term extension;[#] As at August 2016

5

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 Circulation Research and Diabetes Care respectively

• MSC-100-IV : Data from 241 children presented at annual scientific meetings of the Center for International Blood & Marrow Transplant Research and the American Society of Blood and Marrow Transplantation, and at the 3[rd] International Conference on Regenerative Medicine at The Vatican

• MPC-06-ID : 24-month data presented and received the 2016 Best Basic Science Abstract Award at the 24th Annual Scientific sessions of the Spine Intervention Society

• MPC-75-IA : Phase 2a results in post-traumatic knee injury to the anterior cruciate ligament presented at Osteoarthritis Research Society International World Congress

• Ex vivo fucosylation technology developed at Harvard Medical School and exclusively licensed by Mesoblast published in Stem Cells

• MPC-300-IV : Positive Phase 2 results in patients with diabetic nephropathy published in EBioMedicine

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6

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Tier 1 Product Portfolio Overview

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Diversified Pipeline of Product Candidates for High Unmet Needs

First Product on market - Three Tier 1 Product Candidates in Phase 3

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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

• 5.7m patients (prevalence rate of 2% of the population) diagnosed with CHF in the US[1 ]

Market opportunity

• 915,000 new cases diagnosed in the US each year[1 ]

• Growing by 2% per annum

• ~1.9m CHF NYHA Class II-IV patients with low ejection fraction (LVEF<40%) in the US alone[2 ]

Gap in treatment options

• Class II / III CHF patients with low ejection fraction continue to be at high risk of repeated hospitalizations and mortality, despite standard of care pharmacological treatments[3 ]

• Class III / IV CHF patients only have heart transplant and mechanical support as treatment options

Targeted physician population

• Specialists: Targeted physician audience & commercial footprint

• Heart failure specialists

• Interventional cardiologists

• Cardiac Surgeons

We believe MPC-150-IM is positioned to fill the significant treatment gap in patients with advanced CHF

1. AHA Heart disease and stroke statistics—2016 update: a report from the American Heart Association. Circulation. 2016;133:e38-e360 (P e308).

2. Gurwitz JH, Magid DJ, Smith DH, et al. Contemporary Prevalence and Correlates of Incident Heart Failure with Preserved Ejection Fraction. The American journal of medicine . 2013;126(5):393-400. Derived by applying a HF-REF prevalence rate of 32.6% to the U.S. rate of 5.7m U.S. patients.

3. European Heart Journal (2012) 33, 1750–1757 Figure 3

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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

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Source: Circ Res. 2015; 117:576-584. Perin E et al. A Phase II Dose-Escalation Study of Allogeneic Mesenchymal Precursor Cells in Patients 10 With Ischemic or Non-Ischemic Heart Failure

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MPC-150-IM: Phase 2 Therapeutic Benefit on LV Remodelling is Amplified in Subjects with LVESV >100ml

∆ LVESV (Month 6 – Baseline; mL) Baseline LVESV > 100 mL P < 0.02 ∆ LVESV = 54 mL ∆ LVEF (Month 6 – Baseline; %) Baseline LVESV > 100 mL P < 0.05 ∆= 8.1% ∆ LVEDV (Month 6 – Baseline; mL) Baseline LVESV > 100ml P < 0.03 ∆ LVEDV = 51 mL	∆ LVESV (Month 6 – Baseline; mL) Baseline LVESV > 100 mL P < 0.02 ∆ LVESV = 54 mL ∆ LVEF (Month 6 – Baseline; %) Baseline LVESV > 100 mL P < 0.05 ∆= 8.1% ∆ LVEDV (Month 6 – Baseline; mL) Baseline LVESV > 100ml P < 0.03 ∆ LVEDV = 51 mL	∆ LVESV (Month 6 – Baseline; mL) Baseline LVESV > 100 mL P < 0.02 ∆ LVESV = 54 mL ∆ LVEF (Month 6 – Baseline; %) Baseline LVESV > 100 mL P < 0.05 ∆= 8.1% ∆ LVEDV (Month 6 – Baseline; mL) Baseline LVESV > 100ml P < 0.03 ∆ LVEDV = 51 mL	∆ LVESV (Month 6 – Baseline; mL) Baseline LVESV > 100 mL P < 0.02 ∆ LVESV = 54 mL ∆ LVEF (Month 6 – Baseline; %) Baseline LVESV > 100 mL P < 0.05 ∆= 8.1% ∆ LVEDV (Month 6 – Baseline; mL) Baseline LVESV > 100ml P < 0.03 ∆ LVEDV = 51 mL	∆ LVESV (Month 6 – Baseline; mL) Baseline LVESV > 100 mL P < 0.02 ∆ LVESV = 54 mL ∆ LVEF (Month 6 – Baseline; %) Baseline LVESV > 100 mL P < 0.05 ∆= 8.1% ∆ LVEDV (Month 6 – Baseline; mL) Baseline LVESV > 100ml P < 0.03 ∆ LVEDV = 51 mL	∆ LVESV (Month 6 – Baseline; mL) Baseline LVESV > 100 mL P < 0.02 ∆ LVESV = 54 mL ∆ LVEF (Month 6 – Baseline; %) Baseline LVESV > 100 mL P < 0.05 ∆= 8.1% ∆ LVEDV (Month 6 – Baseline; mL) Baseline LVESV > 100ml P < 0.03 ∆ LVEDV = 51 mL	∆ LVESV (Month 6 – Baseline; mL) Baseline LVESV > 100 mL P < 0.02 ∆ LVESV = 54 mL ∆ LVEF (Month 6 – Baseline; %) Baseline LVESV > 100 mL P < 0.05 ∆= 8.1% ∆ LVEDV (Month 6 – Baseline; mL) Baseline LVESV > 100ml P < 0.03 ∆ LVEDV = 51 mL	∆ LVESV (Month 6 – Baseline; mL) Baseline LVESV > 100 mL P < 0.02 ∆ LVESV = 54 mL ∆ LVEF (Month 6 – Baseline; %) Baseline LVESV > 100 mL P < 0.05 ∆= 8.1% ∆ LVEDV (Month 6 – Baseline; mL) Baseline LVESV > 100ml P < 0.03 ∆ LVEDV = 51 mL
	Change (Entire Cohort) Month 6 minus Baseline			Change (LVESV > 100 mL) Month 6 minus Baseline
	PBO (n=15)	150 M MPC (n=15)	∆, PBO Corrected	PBO (n=7)	150 M MPC (n=11)	∆, PBO Corrected	P-values
LVESV	+20	-7	-27	+46	-8	-54	<0.02
LVEDV	+20	-10	- 30	+41	-10	-51	<0.03
LVEF	-2.3	+0.6	+2.9	-6.4	+1.7	+8.1	<0.05

Source : Perin et al., Journal of Cardiac Failure 2015; Vol 21(8): S107; 19[th] Annual Scientific Meeting of the Heart Failure Society of America, Emerson et al.

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11

MPC-150-IM: Phase 2 Results Show Promise in the Prevention of HF-MACE*

HF-MACE Kaplan-Meier Curve over 36 months following treatment in all patients[1 ]

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HF-MACE Kaplan-Meier Curve over 36 months following treatment in patients with LVESV>100ml[2 ]

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• Over 36 months, patients receiving 150M MPC had significantly greater probability of remaining free of a first HFMACE vs. controls (0% vs. 33%, p = 0.026 by log-rank)

• All HF-MACE events occurred in controls with baseline Left Ventricular End Systolic Volume (LVESV)>100ml, where the treatment effect size was even greater (0% vs. 71%, p = 0.0007 by log rank)

• Controls with baseline LVESV>100ml had 11 total/recurrent HF-MACE events over 36 months vs. 0 in matched patients receiving 150 M MPCs (p=0.0007)

• HF-MACE is defined as a composite of cardiac related death or non-fatal heart failure hospitalisations.

• Circ Res. 2015; 117:576-584. Perin E et al. A Phase II Dose-Escalation Study of Allogeneic Mesenchymal Precursor Cells in Patients With Ischemic or Non-Ischemic Heart Failure

• Journal of Cardiac Failure 2015; Vol 21(8): S107; 19[th] Annual Scientific Meeting of the Heart Failure Society of America, Emerson et al.

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

• 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 greatest

• To confirm that MPC-150-IM reduces HF-MACE in patients with advanced heart failure, the ongoing Phase 3 trial is designed to enrich for patients with advanced heart failure and high risk of HF-MACE

• Enrichment for these patients based on heart failure hospitalization in the past 9 months and/or significantly elevated baseline NT-proBNP

• The trial’s primary endpoint is a comparison of recurrent HF-MACE between cell-treated patients and controls

• Terminal events are also being analysed as they relate to non-fatal recurrent HF-MACE

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13

MPC-150-IM: Phase 3 Trial Operational Update

• 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 recommendation that the study should continue as planned

• The trial’s primary endpoint is a comparison of recurrent heart failurerelated major adverse cardiovascular events (HF-MACE) in advanced CHF patients receiving either MPC-150-IM by catheter injection into the left ventricular heart muscle, or control

• Based on observed HF-MACE event rates in the trial to date, the Company has decided to bring forward to Q1 CY2017 a previously planned Interim Analysis to assess the trial’s primary endpoint

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14

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

Ongoing randomized, controlled Phase 2 Trial in 48 patients with biologic refractory rheumatoid arthritis, comparing two doses of MPC-300-IV against placebo

• There are approximately 5.3 million prevalent cases in the US, Japan, and EU5, of which there were 2.4 million in the US alone in 2014[1 ]

• Incidence increases with age – 8.7 per 100,000 for ages 18-34 vs. 89 per 100,000 for ages 65-74[2 ]

Market opportunity

• RA treatment is a approximately $15 billion global market in 2014 projected to grow to over $18 billion in 2024. primarily due to sales of the anti-TNF agents[1]

• In the US, the anti-TNF refractory population is the fastest growing branded market segment, projected to increase by approximately 8% annually and potentially higher with the expected market entry and greater availability of anti-TNF biosimilars[1]

• One third of RA patients do not respond or cannot tolerate current biologic therapies[3]

• Sustained remission defined by ACR 70 only occurs in 5-15% of patients on biologics[5 ]

Gap in treatment

options

• Biologics are associated with increased incidence of opportunistic infections and malignancies[4 ]

• Indications for currently approved biologics target either a single cytokine or immune cell pathway even though RA involves multiple signals / pathways[4 ]

• Need for disease-modifying therapies that are well tolerated and induce remission in a greater percentage of patients (ACR 70) as early as possible in the disease management[3 ]

• Decision Resources Rheumatoid Arthritis – Disease Landscape & Forecast - January 2016; Market Forecast Methodology; Access & Reimbursement – August 2016.

1. GlobalData[©] : Rheumatoid Arthritis Therapeutic – Pipeline Oct 2011

2. Decision Resources Rheumatoid Arthritis – Unmet Need – April 2016.

1. Information listed in the package insert of anti-TNF-α therapies such as Enbrel® (etanercept), Rituxan® (rituximab), Remicade® (infliximab), and Humira® (adalimumab). 5. Alivernini, S et. al. Arthritis Research & Therapy 2009, 11:R163

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15

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

• 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 at least 1 anti-TNF +/- other biologics

• The primary objective of the study was to evaluate safety and tolerability of a single intravenous MPC infusion in biologic refractory RA patients through a 12 week primary endpoint

• Additional objectives were to evaluate pre-specified clinical efficacy endpoints at the primary 12 week time-point, as well as to assess the onset and time course of effect within the first 12 weeks and subsequent duration of responses and safety profile through the full 52 week study

• The American College of Rheumatology (ACR) response, a validated measure of clinical symptoms and signs, and DAS28, a validated measure of RA disease activity index, were assessed at baseline and weeks 1, 4, 8 and 12. The health assessment questionnaire disability index (HAQ-DI), a validated measure of function, was assessed at baseline and weeks 4 and 12

• All analyses and test methods for the trial’s efficacy endpoints were pre-specified in the trial’s Statistical Analysis Plan (SAP) and submitted to the FDA before any analyses were conducted. The SAP included a pre-specified analysis on the key subgroup based on use of 1-2 prior biologics. No post hoc analyses were conducted

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16

MPC-300-IV: Operational Update

• The Phase 2 trial of Mesoblast’s intravenous product candidate, MPC-300-IV, in biologic refractory rheumatoid arthritis has completed enrollment

• A single intravenous MPC infusion in biologic refractory RA patients was without serious adverse events over 12 weeks, and resulted in dose-related improvements in clinical symptoms, function, and disease activity

• The responses to date in this Phase 2 trial provide support for the potential of Mesoblast’s allogeneic MPCs to be positioned early as a treatment option in RA patients who have previously received a prior anti-TNF or other biologic agent

• Durability of response will be evaluated at 6 and 9 months

• Given the large market opportunity, the Company believes that MPC-300-IV is wellpositioned to advance through a strategic partnership into Phase 3 development for biologic refractory rheumatoid arthritis

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MPC-300-IV: Key Efficacy Responses at Week 12 in Biologic Refractory RA

		All Subjects	All Subjects	Subgroup with Prior Use of 1-2 Biologics	Subgroup with Prior Use of 1-2 Biologics	Subgroup with Prior Use of 1-2 Biologics	Subgroup with Prior Use of 1-2 Biologics
	Placebo	1M/kg	2M/kg	p-value 2M/kg vs placebo **Placebo **	1M/kg	2M/kg	p-value 2M/kg vs placebo
	N=16	N=16	N=16	N=9	N=10	N=11
ACR70	0%	20%	27%	0.04 0%	20%	36%	0.09
ACR50	19%	27%	31%	>0.1 11%	30%	55%	0.07
ACR20	50%	47%	50%	>0.1 33%	60%	55%	>0.1
HAQ-DI<-0.22	38%	53%	93%	0.003 33%	60%	91%	0.02
HAQ-DI LS mean change frombaseline	-0.2	-0.3	-0.6	0.02 -0.1	-0.4	-0.7	0.03
DAS28-CRP LS mean change frombaseline	-1.4	-1.3	-2.0	>0.1 -1.1	-1.8	-2.4	0.06
DAS28- CRP<3.2	19%	27%	36%	>0.1 22%	30%	40%	>0.1

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MPC-300-IV: One Dose Provides Compelling Efficacy at 12 Weeks in the Biologic Refractory Population

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ACR70 @ 3 Months
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40%
Marketed Pipeline 36%
35%
30%
27%
25%
20%
22%
15%
14%
10%
11%
5%
6%
0%
Inhibitors of IL-6, Jak Inhibitors [2] MPC 2m/kg MPC 2m/kg
CD80/CD86, CD20, JAK [1]
(1-2 biologics)
ACR50 @ 3 Months
60%
55%
50%
40%
40% 42%
30%
26% 28%
20%
18%
10%
0%
Inhibitors of IL-6, Jak Inhibitors [2] MPC 2m/kg MPC 2m/kg
CD80/CD86, CD20, JAK [1] (1-2 biologics)
----- End of picture text -----

• 1Inhibitors of Il-6 /CD80-CD86/CD-20/ JAK; Biologic Refractory Studies: Orencia (BMS) – ATTAIN, Rituxan (Roche) – REFLEX, Actemra /8mg/kg (Roche)- RADIATE, Xeljanz /5mg bid (Phase III)

• 2 Jak Inhibitors Biologic Refractory Studies: baricitinib/4mg (Eli Lilly/Incyte) – RA – BEACON, ABT-494 /12mg (AbbVie) – Phase IIB

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1919

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 months as a result of moderate degenerative intervertebral disc disease

Market opportunity

• Over approximately 7m patients in the US are estimated to suffer from CLBP due to degenerative disc disease (DDD) in 2016[1,2,5]

• Current MPC-06-ID development program targets over approximately 3.2m patients

Gap in treatment options

• For patients who fail conservative treatment (rest, analgesia, opioids, and epidural steroids), treatment options are limited to highly invasive therapies such as spinal fusion or artificial disc replacement[3]

• Surgeons report ~40% of patients ultimately fail back surgery[3]

Targeted physician population

• Specialists: Targeted physician audience & commercial footprint[4]

• Pain management specialists and anesthesiologists

• Orthopedic / spine surgeons

We believe MPC-06-ID is positioned to fill the significant treatment gap in patients with moderate to severe CLBP after conservative treatment options have failed

1. Decision Resources: Chronic Pain December 2015

2. LEK & NCI opinion leader interviews, and secondary analysis

3. Simon et al – Discogenic Low Back Pain Phys Med Rehabil Clin N Am 25 (2014) 305–317

4. Shapiro CM Phys Med Rehabil Clin N Am 2014

5. Navigant: Commercial Assessment for a Proprietary Cell-Based Therapy for DDD in the U.S. and the EU3 – August 2014.

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MPC-06-ID: Phase 2 Trial

• 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 1,3,6,12 and 24 months – Minimally clinical important difference (MCID) in VAS is defined as >30% improvement[1]

• Guidance from key opinion leaders and payers requires > 50% in pain reduction at a distinct time point

• Oswestry Disability Index (ODI) is a standardized measure of function and was evaluated at 1,3,6,12 and 24 months

• Minimally clinical important difference (MCID) in ODI is defined as >30% or 10 point improvement[1 ]

• 15 point improvement has been used as the MCID for surgical devices to support FDA and EU marketing authorization[2]

1. Ostelo RWJ, Deyo RA, Stratford P, et al. Interpreting change scores for pain and functional status in low back pain. Spine 2008; 33(1):90-94. 2. EMA-SEED 2015.

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MPC-06-ID: Phase 2 Trial Results Presented At Spine Intervention Society 2016 Annual Meeting

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----- Start of picture text -----

% 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 vs. saline
40.0% b) p=0.058 6M MPC vs. HA c) p=0.090 18M MPC vs. saline 37.9% [c] 40.0% 38.5% [d]
34.6%
35.0%
35.0%
30.0%
30.0%
25.0%
25.0%
20.0% 17.7%
20.0%
15.8% [b]
15.0% 12.5% [d]
15.0%
11.8% [a,c]
10.0%
10.0%
5.0%
5.0%
0.0%
0.0%
Saline HA 6M MPCs 18M MPCs Saline HA 6M MPCs 18M MPCs
% Responder Rate
----- End of picture text -----*

*** 50% VAS back pain reduction AND 15 point ODI improvement AND no intervention at the treated level**

22

MPC-06-ID: Phase 3 Trial Operational Update

• 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-06ID for treatment of chronic low back pain were presented at the 24th Annual Scientific Meeting of the Spine Intervention Society and received the 2016 Best Basic Science Abstract award

• FDA has provided written guidance:

• Use of a composite primary endpoint is acceptable for approval

• Agreed thresholds for pain (50% decrease in VAS) and function (15 point improvement in ODI)

• Two time points (12 and 24 months) for meeting pain and functional improvement criteria

• No intervention at the treated level through 24 months

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23

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)

• ~30,000 allogeneic BMTs performed globally (~20K US/EU5) each year, ~20% pediatric[1,2 ]

Market opportunity

• ~3,700 allogeneic BMTs performed in Japan each year[3 ]

• ~50% of all US patients develop aGVHD (Grades II-IV)[4 ]

• Steroid-resistant acute GVHD have a dismal prognosis, with mortality rates in excess of 85%

Unmet

Need

• No currently approved therapies for steroid refractory patients (ex Japan)

• Off-label options have mixed efficacy with high toxicity

• Significant need for a new treatment with a favorable risk / benefit profile

• Japan product (TEMCELL® HS Inj.) launched; reimbursed up to ~$195K for full treatment course[5 ]

Path to

market

• US product candidate (MSC-100-IV) currently in 60 patient open-label Phase 3 registration pediatric trial

• Highly targeted physician audience and commercial footprint for pediatric launch in US

• High risk adult population identified for Phase 3 trial

• Successful Interim Analysis Q4 2016, enrollment complete 1H 2017

We believe MSC-100-IV has potential to be first allogeneic non-hematopoietic stem cell product approved in USA, triggering a “halo” effect for Mesoblast’s other Tier 1 products

1. Gratwohl A et al Quantitative and qualitative differences in use and trends of hematopoietic stem cell transplantation: a Global Observational Study. Haematologica. 2013 Aug;98(8):1282-90. 2. CIBMTR, Decision resources GVHD Epi Nov 2012.

2. APBMT Annual Report Dec 2012; Assumes a growth rate of approximately 3% per year

3. Jagasia et al Risk factors for acute GVHD and survival after hematopoietic cell transplantation. 2012 January vol 119 (1).

.

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1. Based on a ¥JPY = $USD 0.009375 spot exchange rate on as of the market close on November 11, 2016. Amounts are rounded. Source: Bloomberg.

24

MSC-100-IV: Product Development Strategy

 Manufacturing

• 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 development and access work in parallel

• Launch pediatric product in 2018 in US

 Adults

• Complete targeted Phase 3 study in high - risk subset of adult patients with aGVHD (with liver and gut disease)

• Market development and access work in parallel

• Launch adult product in 2021 in major markets

 Life cycle management and label expansion

• Prophylaxis

• Acute GVHD, first-line

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MSC-100-IV North America aGVHD Market Opportunity

	Target Population	2015	Source
	Allogeneic Hematopoietic Stem Cell Transplants Steroid Refractory Acute GVHD Grades II-IV 5,400 2,900 10,200 Epidemiology Acute GVHD Grades II-IV
			• US: estimated from 2013 CIBMTR (table 6) • Canada: estimate based on general population size and US transplant activity per 10m • Estimated 53% Rate: Jagasia, M., Arora, M., Flowers, M. (2012) Risk Factors for acute GVHD and Survival after Hematopoietic Cell Transplantation. Blood, 5 January (119):296-307 • Estimated 54% Rate: Westin, J., Saliba, RM., Alousi, A. (2011) Steroid Refractory Acute GVHD: Predictors and Outcomes. Advances in Hematology (2011); 1-8
	First-Line High Risk Adults with aGVHD Target Population 1,900 Pediatric SR aGHVD Grades II-IV 600 Total North America aGHVD Target Population ~2,500
			• Pediatric: estimated from Center for International Blood and Marrow Transplant Research -Transplant Activity Report Covering 2009-2013 • Adult High Risk: • MacMillan, ML., Robin, M., Harris, AC. (2015) A refined risk score for acute graft-versus-host disease that predicts response to initial therapy, survival, and transplant-related mortality. Biol Blood Marrow Transplant. Apr;21(4):761-7 • Jagasia, M., Arora, M., Flowers, M. (2012) Risk Factors for acute GVHD and Survival after Hematopoietic Cell Transplantation. Blood, 5 January (119):296-307 • CIBMTR: Current uses and outcomes of hematopoietic stem cell transplantation 2015 summary slides

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MSC-100-IV: Phase 3 Trial in Children with Steroid Refractory Acute Graft vs Host Disease (SR-aGVHD)

MSC-100-IV as first line therapy in children with SR-aGVHD (Day 28 response)

MSC-100-IV in children with SR-aGVHD who failed multiple other modalities (Day 100 survival)

Response at Day 28	Randomized Placebo Controlled Trial	Randomized Placebo Controlled Trial	Open-label Expanded Access Program
	Placebo	MSC-100-IV	MSC-100-IV
Responder	3/14 (21.4%)	9/14 (64.3%)	29/36 (81%)
Non- responder	11/14 (78.6%)	5/14 (35.7%)	7/36 (19%)
	p-value = 0.0014

Compared with placebo control patients, MSC-100-IV produced markedly superior overall response at day 28, a clinically meaningful endpoint (p=0.0014).

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82%
39%
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Survival at Day 100 of pediatric patients treated with MSC-100-IV stratified by 28-Day responders vs nonresponders, n=241

• Evidence that MSC-100-IV is effective when used as first line therapy in children with SR-aGVHD

• FDA agreement on 60 patient open label Phase 3 trial for accelerated US approval pathway

• Enrollment criteria: MSC-100-IV offered as first line therapy in children with SR-aGVHD

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MSC-100-IV: Graft vs Host Disease: Pediatric GVHD001/GVHD002 Phase 3 Study

 Phase 3 study ongoing, ~ 40 sites planned[1 ]

• 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 patients (2 months to 17 years inclusive)

• aGVHD following allogeneic HSCT failing systemic corticosteroid therapy

• Grades C and D aGVHD involving skin, liver and/or GI tract

• Grade B aGVHD involving liver and/or GI tract with or without concomitant skin disease

: Endpoints[1]

• Primary endpoint: Overall response at Day 28

• Key secondary endpoint: Survival at Day 100 in responders at Day 28

• Subjects evaluated at Days 28, 56 and 100 in study 001, and out to Day 180 in study 002

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1. Clinicaltrials.gov identifier: NCT02652130.

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GVHD001 Interim Futility Analysis Method and Results - November 2016

• 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 significant treatment effect at study completion, conditional on the data observed at this interim time point

• DSMB notified Mesoblast that analysis was successful

• Interim analysis outcome is consistent with what has previously been demonstrated for the product used in this indication under both expanded access protocol and earlier placebo-controlled trial

• Enrollment in the study is ongoing across multiple sites in the United States and will continue

• Completion is expected in mid-2017

• Commercial launch activities are underway

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MSC-100-IV: Concentrated High Volume Centers Provide Compelling Low Cost Commercial Structure[1 ]

Top Pediatric Transplant Centers

Top Adult Transplant Centers

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• There are 65 centers in the U.S. that conduct allogeneic transplants in pediatrics

• ~ 50% of all of these transplants happen at 13 influential centers

• There are 108 centers in the U.S. that conduct adult allogeneic transplants

• ~50% of all of these transplants happen at 19 influential centers

• Broad overlap between high volume pediatric centers and influential adult centers

• Source: Center for International Blood & Marrow Transplant Research.

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Tier 1 Product Candidate Deliverables (Calendar Year)

Product Candidate Programs

Milestones

2016 2017 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 line results first cohort Rheumatoid Arthritis (Biologic MPC-300-IV Full trial results Refractory) 6/9 month trial results

Chronic Low Back Pain Due to MPC-06-ID Degenerative Disc Disease

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MSC-100-IV / Acute Graft Versus Host Disease Temcell® HS Inj.

Phase 3 Enrollment Complete Phase 3 Interim Analysis

TEMCELL® HS Inj. Launched in Japan Interim Results Phase 3 Enrollment Complete

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Milestone Milestone
Achieved Target
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Thank You and Questions

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