Full Press Release Details
Mesoblast - A Global Leader in Cell
Based Medicines 34th Annual J.P. Morgan Healthcare Conference January 2016 Exhibit 99.1
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
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"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 Teva Pharmaceutical Industries Ltd, 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.
Key Investment Highlights Disruptive
technology platform: proprietary, allogeneic, "off-the-shelf" adult stem cells with predictable therapeutic properties Established late stage portfolio of distinct and advanced product candidates Strategic partnerships delivering
clinical, manufacturing and commercial capabilities, together with financial support Scalable, cost-efficient manufacturing capabilities Intellectual property leadership covering compositions, uses, and manufacturing processes Experienced management
Successfully completed US listing with
access to world's largest sophisticated healthcare investor pool and analysts Financing raised USD $63.5m (net proceeds) which significantly augmented existing cash reserves of USD $77.8m at 30 September 2015 Quarterly cash outflows expected
to be reduced by approximately 20-25% in Q2-4 FY2016 in comparison to Q1 FY2016 (USD$28.1m) and Q4 FY2015 (USD$27.3m) Cash managed to extend runway and achieve Tier 1 value inflexion points Major focus is FDA filing for our first US Product approval
in Acute Graft Versus Host Disease (aGVHD) FDA Approval may be accompanied by a Rare Pediatric Disease Designation / Priority Review Voucher We intend to conclude additional and appropriate strategic partnerships Focused Company With Strong Cash
Reserves to Meet Key Corporate Objectives
Disruptive Technology Platform 1
Multiple, Diverse Mechanisms Mesenchymal Lineage Adult Stem Cells (MLCs), immunoselected precursors and progeny Located around blood vessels in all vascularized tissues Respond to signals associated with tissue damage Secrete diverse variety of
biomolecules responsible for tissue repair and immunomodulation MLCs can be isolated from diverse tissue sources (bone marrow, adipose, dental pulp) MLCs from a single healthy donor can be expanded to thousands of doses in weeks Potential for
commercial large-scale expansion via proprietary manufacturing processes Immunomodulatory properties makes MLCs relatively non-immunogenic Allows for commercially scalable, allogeneic, off-the-shelf products Allogeneic Scalable Use MLC Triggers:
IL-6 LP3, TNF , acidosis
Product Candidates Target Diseases with
High Unmet Needs Three Tier 1 Product Candidates in Phase 3 Programs Update 2
Tier 1 Product Candidate Deliverables
MPC-150-IM: Chronic Heart Failure (CHF)
- Market Opportunity A MPC-150-IM is in development for patients with New York Heart Association Class II-IV CHF MPC-150-IM is positioned to fill the significant treatment gap in patients with advanced CHF AHA statistical Update - Heart
disease and stroke statistics-2015 update Circulation 2015 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. European Heart Journal (2012) 33, 1750-1757 Figure 3 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 Specialists: Targeted physician audience & commercial footprint Heart failure specialists Interventional cardiologists Cardiac
surgeons Gap in treatment options Targeted physician population 5.7m patients (2% of the population) diagnosed with CHF in the US1 870,000 new cases diagnosed in the US each year1 Growing by 2% per annum ~1.9m CHF NYHA Class II-IV patients with low
ejection fraction (LVEF<40%) in the US alone2 Market opportunity
MPC-150-IM: Phase 2 Trial Results in
CHF Identify Optimal Therapeutic Dose A Phase 2, randomised placebo controlled trial in 60 patients with Class II / III CHF and LVEF<40% Placebo vs. 25, 75, 150 M MPCs injected by endomyocardial catheter At 6 months: Dose-dependent effect seen on
left ventricular remodeling, with the 150 M cell dose (MPC-150-IM) showing greatest effect vs. controls 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.
MPC-150-IM: Phase 2 Trial Results in
CHF Identify Optimal Target Patients, Advanced Heart Failure (Baseline LVESV>100ml)1 with LVESV >100ml1 A Change (Entire Cohort) Month 6 minus Baseline Change (LVESV > 100 mL) Month 6 minus Baseline Control (n=15) 150 M MPC (n=15) ,
Control Corrected Control (n=7) 150 M MPC (n=11) , Control Corrected P-values LVESV (ml) +20 -7 -27 +46 -8 -54 <0.02 LVEDV (ml) +20 -10 - 30 +41 -10 -51 <0.03 LVEF (%) -2.3 +0.6 +2.9 -6.4 +1.7 +8.1 <0.05 Intention to treat (ITT)
population, ANCOVA analysis Placebo corrected benefit of single 150M cell dose on cardiac volumes and ejection fraction at 6 months was greatest in patients with more advanced heart failure as defined by baseline LVESV>100ml at baseline
MPC-150-IM: Single Dose Prevents
HF-MACE Over 3 Years A Over 36 months, patients receiving 150M MPC had significantly greater probability of remaining free of a first HF-MACE* vs. controls (0% vs. 33%, p = 0.026 by log-rank) All HF-MACE events occurred in controls with baseline
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 events over 36 months vs. 0 in matched patients receiving 150 M MPCs (p=0.0007) 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. * HF-MACE is defined as a composite of cardiac related death or resuscitated
cardiac death or non-fatal decompensated heart failure events HF-MACE Kaplan-Meier Curve over 36 months following treatment in all patients HF-MACE Kaplan-Meier Curve over 36 months following treatment in patients with LVESV>100ml
Patients with large baseline LVESV
and advanced heart failure are at highest risk of HF-MACE For these patients 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 high risk of HF-MACE through the requirement of a prior hospitalization in the last nine months or high levels of NT-proBNP Trial is recruiting well across North America and is expanding into Europe A
first interim analysis will be performed during Q1 2016 (results available Q2 2016) focused on safety and efficacy based on secondary surrogate volume measurements MPC-150-IM: Phase 3 Trial Recruiting Well and Targets Advanced Heart Failure where
Medical Need is Greatest A
Following the preliminary responses
from FDA in Dec 2015, the ongoing phase 3 program is planned to be optimized as follows: The current Phase 3 trial size will be reduced from 1,165 to approximately 600 subjects The revised primary endpoint will be a comparison of recurrent HF-MACE
between MPC treated patients and controls The proposal to use of recurrent HF-MACE as a primary endpoint is based on having successfully achieved this endpoint in the Phase 2 trial A second confirmatory study, will be conducted in parallel in an
identical patient population of up to 600 patients using the same primary endpoint In Q2 2016, Teva and Mesoblast will provide updated timelines for program completion MPC-150-IM: Reduced Size of Phase 3 Program Following FDA Discussions
MPC-06-ID: Chronic Low Back Pain due
to Degenerative Disc Disease - Market Opportunity B 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 LEK & NCI opinion
leader interviews, and secondary analysis Shapiro CM Phys Med Rehabil Clin N Am 2014 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 Surgeons report ~40% of patients ultimately fail back surgery Specialists: Targeted physician audience & commercial footprint Pain management specialists and anesthesiologists Orthopedic / spine
surgeons Gap in treatment options Targeted physician population Over 5.7m patients in the US suffer from CLBP due to degenerative disc disease (DDD) MPC-06-ID is being developed to target 4.0m patients with Moderate and Severe CLBP due to DDD Market
opportunity MPC-06-ID is positioned to fill the significant treatment gap in patients with moderate to severe CLBP after conservative treatment options have failed
MPC-06-ID: 12 Month Phase 2 Trial
Design B 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% improvement1 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 improvement1 15 point improvement has been used as the
MCID for surgical devices to support FDA and EU marketing authorization 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.
12 & 24 Months 50% Reduction in
VAS LBP with No Intervention p=0.023 6M MPC vs. saline p=0.118 6M MPC vs. HA p=0.166 18M MPC vs. saline 6 & 12Months 50% Reduction in VAS LBP with No Intervention p=0.013 6M MPC vs. saline P=0.006 6M MPC vs. HA P=0.110 18M MPC vs. saline P=0.060
18M MPC vs. HA B MPCs Groups Have a Greater Proportion of Patients Through 24 Months with at Least 50% Pain Reduction Than Controls
12 & 24 Months 15pt Improvement
in ODI with No Intervention p=0.023 6M MPC vs. saline p=0.012 18M MPC vs. saline 6 & 12 Months 15pt Improvement in ODI with No Intervention p=0.052 6M MPC vs. saline p=0.058 18M MPC vs. saline B MPCs Groups Have a Greater Proportion of Patients
Through 24 Months with at Least 15 Point Improvement in Function (ODI) With No Intervention Compared to Controls
Treatment Success Composite Endpoint
50% VAS back pain reduction AND 15 point ODI improvement AND no intervention at the treated level d. p=0.090 6M MPC vs. saline p=0.044 6M MPC vs. saline p=0.058 6M MPC vs. HA p=0.090 18M MPC vs. saline MPC-06-ID: Composite Endpoint for Both Pain and
Function Over 24 Months - Phase 2 Data B
MPC-06-ID: Phase 3 Pathway to
Potential Approval in Patients with CLBP due to Moderate Degenerative Intervertebral Disc Disease B Phase 3 trial is enrolling well The study will enroll ~330 patients Primary efficacy endpoint is a composite of 50% pain improvement (VAS)
and 15 point functional improvement (ODI) over 12 and 24 months The Phase 3 endpoint is consistent with the approach for approval of spinal device technologies An interim analysis for efficacy will be performed during Q4 2016
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). 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. CIBMTR, Decision resources GVHD Epi Nov 2012. APBMT Annual Report Dec 2012; Assumes a growth rate of approximately 3% per year Decision resources Niche Markets and
Rare diseases: GVHD Nov 2012 MSC-100-IV / TEMCELL HS Inj. : Acute Graft vs Host Disease - Market Opportunity C Mortality can reach 85% in patients with liver & gut complications No currently approved therapies for steroid refractory
patients Off-label options have mixed efficacy with high toxicity Significant need for a new treatment with a favorable risk / benefit profile Highly targeted physician audience & commercial footprint for pediatric launch in US ~ 75 centers in
the US conduct pediatric allogeneic BMTs ~ 50% of all US pediatric transplants concentrated in 15 centers & key metropolitan areas No approved treatment options Targeted physician population ~30,000 allogeneic BMTs performed globally each year,
25% pediatric1,2 ~3,700 allogeneic BMTs performed in Japan each year3 ~50% of all patients develop aGVHD (Grades II-IV)4 Market opportunity
MSC-100-IV: Phase 3 Trial in
Children with Steroid Refractory Acute Graft vs Host Disease (SR-aGVHD) C Compared with placebo control patients, MSC-100-IV produced markedly superior overall response at day 28, a clinically meaningful endpoint (p=0.0014). Response at Day 28
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%) 25/32 (78.1%) Non-responder 11/14 (78.6%) 5/14 (35.7%) 7/32 (21.9%) p-value = 0.0014
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 (n = 102) (n = 58) Survival of Pediatric Patients Treated with MSC-100-IV 28-Day Responders vs Non-responders MSC-100-IV in Children with SR-aGVHD who failed multiple other modalities MSC-100-IV as first line
therapy in children with SR-aGVHD
MSC-100-IV / TEMCELL HS Inj. :
Product Launch Plans in aGVHD TEMCELL HS Inj. is the first allogeneic stem cell product approved in Japan MSC-100-IV has the potential to be the first allogeneic stem cell product approved in US United States (MSC-100-IV): 2017 Potential FDA