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Japan : Victoria Business Forum Silviu Itescu
Chief Executive 25 August 2015
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CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This presentation, including any comments made during or following the presentation, may contain forward-looking statements that are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. 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. Actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Factors 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.
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Mesoblast Competitive Strengths
1 Disruptive platform based on allogeneic, “off-the-shelf” adult stem cells
2 Substantial intellectual property covering products, uses, and manufacturing
3 Five product candidates that are in active Phase 3 programs or are Phase 3-ready
4 Scalable manufacturing capabilities
5 Strategic collaborations with industry leaders
6 Experienced management team
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• Traditional biological and chemical based therapies generally target single pathophysiological pathways.
• MLCs impact multiple downstream pathways and have the potential attributes for regeneration and repair: • Detect injury and inflammation • Respond to local stimuli and signals from the injured tissue • Release a wide range of biomolecules (growth factors, chemokines, enzymes etc.)
MLCs release multiple factors involved in:
Cell survival and proliferation Cell and tissue repair Stimulation of mature blood vessel growth and reversal of endothelial dysfunction Inhibition or modulation of damaging inflammatory and immune responses Tissue remodelling by reduction in scar formation and fibrosis
*MLCs comprise Mesenchymal Precursor Cells (MPCs ) and culture-expanded Mesenchymal Stem Cells (MSCs)
Paracrine Effects of Mesenchymal Lineage Cells Rationale for Developing Our Mesenchymal Lineage Adult Stem Cells (MLCs)*
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MLC Product Development Strategy
Multiple mechanisms of action may facilitate therapeutic effects in diseases
with high unmet needs
Proprietary manufacturing processes utilized to create distinct product candidates with specific technical
attributes
Initial focus on advanced disease populations with commensurate
pricing and potential for label expansion
Allows for distinct product candidate reimbursement, commercialization and
partnering strategies
Independent, Distinct Product Candidates
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Five Product Candidates in Active Phase 3 Programs or Phase 3-ready
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Legislative framework*
The Pharmaceuticals, Medical Devices and Other Therapeutic Products Act (PMD Act) came into effect 25 November 2014 in Japan
Established a framework for expedited approval in Japan for regenerative medical products
Key Takeaways Conditional product approvals will be based on existing Phase 2 trial results demonstrating probable efficacy
and safety with bridging studies in Japanese patients Conditional approvals will allow sales of each product candidate for up to 7 years Conditionally approved products will be covered by health insurance Conditional approvals will cover allogeneic cell therapy product candidates manufactured under GMP outside
of Japan; and Full approval is expected to require further confirmation of safety and efficacy in a larger population.
New framework for Expedited Japanese Approval of Regenerative Medicines
With late-stage clinical product candidates in multiple indications, we believe we are well positioned to take advantage of this new legislative framework in Japan
*PMDA slide presentation - 5th Asian Cellular Therapy Organization Conference, Osaka; 9 November 2014
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Population demographics changing - 40% of Japanese will be 65+ by 2050 Objectives To capitalize on clinical data generated to date, our strong intellectual property
portfolio, and manufacturing know how to achieve accelerated product regulatory approvals in Japan
To generate nearer term revenues in Japan, the world’s second most established healthcare market
In order to achieve our corporate objectives in Japan, we: Have completed prioritization of lead product candidates based on assessment of
commercial opportunity and feasibility in Japan Will leverage existing Phase 2 clinical trial results from our lead product candidates Have engaged with product development and commercialization experts in Japan Have initiated dialog with the Japanese regulatory authority, the PMDA Are in active discussions with existing and potential commercialization partners for
our product candidates in Japan
Our Strategy for Japan
We believe the PMD Act will enable our cell therapy products to be made available sooner to patients with unmet medical needs, and to achieve nearer term revenues in
Japan, ahead of other jurisdictions
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MPC-150-IM: Congestive Heart Failure (CHF) – Market Opportunity
MPC-150-IM is in development for patients with New York Heart Association Class II-IV CHF
We believe MPC-150-IM is positioned to fill the significant treatment gap in patients with advanced CHF
1. AHA statistical Update – Heart disease and stroke statistics-2014 update Circulation 2014
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.
3. 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
5.1m patients (2% of the population) diagnosed with CHF in the U.S.1
825,000 new cases diagnosed in the U.S. each year1
– Growing by 2% per annum
~1.7m CHF NYHA Class II-IV patients with low ejection fraction (<40%) in the US alone2
Market opportunity
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MPC-06-ID: Chronic Discogenic Low Back Pain (CDLBP) – Market Opportunity
MPC-06-ID is in development for the treatment of CDLBP lasting >6 months as a result of moderate degenerative intervertebral disc disease
1. LEK & NCI opinion leader interviews, and secondary analysis 2. Shapiro CM Phys Med Rehabil Clin N Am 2014
For patients who fail conservative treatment (rest and analgesia) 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 2
Specialists: Targeted physician audience & commercial footprint – Pain management specialists and anaesthesiologists – Orthopedic / spine surgeons
Gap in treatment
options
Targeted physician
population
Prevalent CLBP population to grow to 21.9m patients in the U.S. by 20221
55% of CLBP population seek treatment1
~40% of CLBP patients have a discogenic cause
Market opportunity
We believe MPC-06-ID is positioned to fill the significant treatment gap in patients with moderate to severe CDLBP after conservative treatment options have failed
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MSC-100-IV /JR-031 is in development for pediatric and adult patients with acute GVHD following allogeneic hematopoietic stem cell transplant (HSCT) who have failed to respond to steroid treatment
Targeted physician population
Highly targeted physician audience & commercial footprint for lead launch in pediatrics
~ 70 centers in the US conduct pediatric allogeneic HSCTs
• ~ 50% of all pediatric transplants
concentrated in 12 centers & key metropolitan areas
1. Gratwohl T el al, Quantitative and qualitative differences in use and trends of hematopoetic stem cell transplantation : a Global Observation study. Haematollogica 213;98(8) 2. CIBMTR, Decision resources GVHD Epi Nov 2012. 3. Decision resources Niche Markets and Rare diseases: GVHD Nov 2012 4. Cahn et al prospective evaluation of 2 aGVHD grading systems Blood Aug 2005 5. Svahn BM et al, Increased costs after allogeneic haematopoetic SCT are associated with major complications and
re-transplantation BMT 2012; 47:706-715
MSC-100-IV: Acute Graft Versus Host Disease (GVHD) – Market Opportunity
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 lead launch in pediatrics
~ 75 centers in the US conduct pediatric allogeneic HSCTs
~ 50% of all pediatric transplants concentrated in 15 centers & key metropolitan areas
No approved treatment
options
Targeted physician
population
~30,000 allogeneic HSCTs performed globally each year, 25% pediatric1,2
~50% of all patients develop GVHD (Grades II-IV)3
~50% steroid-refractory acute (SR-a) GVHD4
European study showed cost of treating patients with SR-a GVHD was approx US $325,000 5
Market opportunity
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MSC-100-IV / JR-031 : Product Launch Plans in GVHD
MSC-100-IV/JR-031 have potential to be first allogeneic stem cell products approved in USA, Japan “Halo” effect for Mesoblast’s commercial go-to-market capability
USA:
Positive FDA meeting July 2014 clarified pathway for accelerated approval – 60 patient open-label Phase 3 pediatric trial initiated – Confirmatory Phase 3 adult trial design in liver / gut subset
Manufacturing and commercial readiness
BLA filing for pediatric registration on track
Japan:
• JCR Pharmaceuticals Co Ltd., our GVHD partner in Japan, filed for regulatory approval for JR-031 in Japan in September 2014 and was granted orphan drug priority review
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Portfolio Targeting Inflammatory Diseases – A Major Emerging Opportunity
MLCs have receptors that respond to pro-inflammatory signals, inducing release of multiple anti inflammatory mediators
MLCs thereby target multiple immune pathways concurrently
This should position MLC product candidates as ideal therapeutics for immune mediated diseases where multiple pathways are associated with disease activity for which there are no alternatives and/or resistance to other therapies
MLCs have demonstrated favorable risk: benefit profiles in development programs, with potential to be differentiated from other biologics that have infectious or neoplastic complications
Mesoblast is developing MLC product candidates to target
– Diabetic kidney disease
– Biologic-refractory rheumatoid arthritis
– Biologic-refractory Crohn’s disease
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Extensive Intellectual Property Portfolio – 652 patents across 67 families
Specific therapeutic applications
378 patents or patent applications Valid through 2035*
Immunologic / inflammatory disorders – 99 patents or patent applications – start to expire 2019* and extend to 2035*
Cardiovascular disorders – 67 patents or patent applications – start to expire 2018* and extend to 2024*
Orthopedic disorders – 63 patents or patent applications – start to expire 2017* and extend to 2032*
Oncology / hematology – 96 patents or patent applications – start to expire 2019* and extend to 2030*
Other therapeutic applications – 53 patents or patent applications – start to expire 2027* and extend to 2032*
Composition of matter (COM) and manufacturing process
138 patents or patent applications Valid through 2024-2035*
MPCs– 58 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*
MSC’s – 48 granted patents or patent applications related to MSC composition of matter and manufacture of MSCs –start to expire 2018* and extend to 2035*
DPSCs – 32 patents or patent applications – start to expire 2021* and extend to 2024*
Complementary technologies and additional candidates
136 patents or patent applications Valid through 2024-2032*
Cell-based complementary technologies – 62 patents or patent applications - start to expire 2017* and extend to 2030*
SDF-1 – 26 patents or patent applications –start to expire 2027* and extend to 2032*
Factors and agents for cardiovascular and other fibrotic indications – 48 patents or patent applications –start to expire 2021* and extend to 2024*
*Excludes possible patent term extension
In the last 12 months we have had 33 new patents granted including 9 in the US, 6 in Japan, 5 in China and 13 in other jurisdictions
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Scalable Manufacturing Capabilities: Partnership with Lonza
Manufacturing objectives
Distinct manufacturing processes for each product
Commercial scale processes with batch-to-batch consistency and reproducible release criteria
Ensure commercial product supply is aligned with projected market needs
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Strategic Collaborations
Manufacturing Development Commercialization
Manufacturing R&D
Manufacturing
Clinical research
CHF clinical research
CHF program – MPC-150-IM
Global rights Additional CV & CNS
programs
Global rights
Japan GVHD rights
Heart failure
Back pain partner
options
GVHD Japan
Thank You www.mesoblast.com