Mesoblast Limited · ASX:MSB
Mesoblast — Alliance for Regenerative Medicine Investor Day (March 2014)
18 slides from Mesoblast's conference presentation, Mar 27, 2014
Slide 1: Building out a multi-product regenerative medicine company
For personal use only Building out a multi-product regenerative medicine company Keynote presentation to Alliance for Regenerative Medicine Investor Day New York, 26 March 2014
Slide 2: Cautionary note regarding forward-looking statements
For personal use only 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 quantifi
Slide 3: Successful products built on traditional Pharma fundamentals
Successful products built on traditional Pharma fundamentals Commercialization strategy Manufacturing capabilities For personal use only 1 Proprietary platform technology 1 Defined regulatory path adult vs embryonic vs iPS autologous vs allogeneic 2 Intellectual property 2 Control of manufacturi
Slide 4: Proprietary platform technology
1 Proprietary platform technology – must carry inherent success factors to support product development and business For personal use only model Mesoblast developing proprietary adult mesenchymal lineage cells, including Mesenchymal Precursor Cells (MPCs), Mesenchymal Stem Cells (MSCs), Dental Pulp
Slide 5: Intellectual property (IP) – protects commercial interests
2 Intellectual property (IP) – protects commercial interests For personal use only Mesoblast owns or has exclusive rights to more than 60 patent families covering mesenchymal lineage cells, which provide commercial advantages and long-term protection across its technology platforms Acquisition o
Slide 6: Strong cash position – allows multiple product development
3 Strong cash position – allows multiple product development For personal use only Commercial success of platform technology developer is critically dependent on having access to substantial sources of funds, in order to – ensure timely, optimal product clinical development – enable multiple clinica
Slide 7: Strategic partnerships
4 Strategic partnerships – additional sources of funding, validation of technology, access to product commercialization For personal use only and other areas of partners’ expertise Mesoblast’s alliances include: – cardiovascular and neurological MPC programs being developed in partnership with Tev
Slide 8: Manufacturing capabilities – define ability to commercialize cell-based products
Manufacturing capabilities – define ability to commercialize cell-based products For personal use only Manufacturing strategy must incorporate regulatory, commercial, and R&D strategies and consider: – Control of manufacturing – Regulatory compliance with best practice – Commercial scale-up with c
Slide 9: Concurrent development of multiple products
65 Concurrent development of multiple products For personal use only Increases risk-adjusted probability of success Leverages safety data across technology platform Allows re-focusing of clinical strategy as results become available Leverages IP protection maximally Early success may valid
Slide 10: Defined product regulatory path
Defined product regulatory path – continues to evolve for cell-based products, combining traditional approval process with unique features For personal use only Discover; proof of concept; cell product potential; therapeutic mechanism and pathway; cell and disease interaction Basic Research PACT Web
Slide 11: Cell variability drives regulatory paradigm
Cell variability drives regulatory paradigm For personal use only Cell variability at a number of levels Donor - allogeneic/autologous, gender, age, health? Tissue source – bone marrow, adipose, cardiac, neural? How are the cells isolated/processed? Manual or device? Are the cells cultured
Slide 12: Ongoing regulatory interactions essential for successful product launch
Ongoing regulatory interactions essential for successful product launch MOA hypotheses (re-evaluated) For personal use only In vivo correlation Qualitative based on MOA Assay matrix – some are quantifiable Best candidate(s) – set quantitative values Finalize potency Commercial manufacturing Product
Slide 13: Mechanisms of Action (MOA) drive product development
Mechanisms of Action (MOA) drive product development For personal use only Must understand well the unique MOAs and scientific advantages attributable to the particular cell therapy being developed Is the proposed MOA replacement of damaged tissue by engraftment of differentiated or undifferenti
Slide 14: Mesenchymal lineage cells secrete multiple paracrine factors
Mesenchymal lineage cells secrete multiple paracrine factors, giving rise to complex MOAs For personal use only Mesoblast's strategic product focus is guided by specific MOAs of our cellular therapies The mesenchymal lineage stem cells respond to signals from inflammation and /or tissue damage by re
Slide 15: Mesoblast's pipeline in key therapeutic areas
Mesoblast’s pipeline in key therapeutic areas For personal use only 14
Slide 16: How is the regenerative medicine sector performing?
How is the regenerative medicine sector performing? For personal use only A sample of company sponsored stem cell products in clinical trials 2014 14 12 Phase 3 Phase 2 10 Phase 1 8 6 4 2 0 15
Slide 17: Leveraging strengths
Leveraging strengths For personal use only Probability of success enhanced by: – proprietary technology offering unique scientific and clinical advantages – strong IP position – products specifically targeting major medical conditions – strong cash position enabling simultaneous product developmen
Slide 18: Leading the development of adult stem cell therapies
For personal use only Leading the development of adult stem cell therapies
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