Mesoblast Limited · ASX:MSB
Mesoblast — UBS Healthcare Conference 2008
20 slides from Mesoblast's conference presentation, Nov 7, 2008
Slide 1: Commercialization Of Stem Cell Technology
For personal use only Commercialization Of Stem Cell Technology UBS Healthcare Conference Sydney, November 2008
Slide 2: Mesoblast Company Overview
For personal use only Mesoblast Company Overview • listed Dec. ‘04, raised $21m • strong board and management • major milestones accomplished >6 months ahead of schedule • further capital raised $17m July ’06, $13m Dec ’07 • sufficient cash to accomplish major milestones next 12 months • current mar
Slide 3: Achieving Institutional Shareholder Support
For personal use only Achieving Institutional Shareholder Support 1. transparent corporate structure and governance 2. identifying value inflexion points as corporate drivers (a) commercial (b) technical (c) clinical 3. risk mitigation strategies (a) seasoned management (b) multiple programs (c) ear
Slide 4: Commercialising Stem Cells: What is the Right Business Model?
Commercialising Stem Cells: What is the Right Business Model? For personal use only Embryonic Stem Cells NO near-term clinical products (ethics, cancer risk) Adult Stem Cells Autologous: low margin, high cost variability of source material not applicable for emergency use cord blood (e.g. banking) h
Slide 5: Proprietary MPC Isolation
Proprietary MPC Isolation For personal use only Bone Marrow (BM) BM + stem cell-binding antibody magnetic beads Competitive Advantages: Precise identification, magnet ease of isolation and scale-up fresh, • purer initial stem cell pool quiescent • homogeneous population MPC • efficient large-scale e
Slide 6: Highly Expandable Commercial Stem Cell Production
Highly Expandable Commercial Stem Cell Production For personal use only bone marrow + antibody Orthopaedic magnetic bone beads cartilage NON-IMMUNOGENIC magnet other cell types Isolated Cultured heart muscle cells cells Cardiac smooth muscle Stem cell Differentiated cell arteriole
Slide 7: For personal use only
For personal use only
Slide 8: Our products combine speed to market with a high margin business model
For personal use only Our products combine speed to market with a high margin business model MPC products are biologic drugs in a bottle • safer than small molecules • greater predictability from pre-clinical to human trial end-points • more rapid regulatory approval • one donor -- thousands of pati
Slide 9: Road To FDA Approvals For An Allogeneic Stem Cell Product
For personal use only Road To FDA Approvals For An Allogeneic Stem Cell Product Preclinical 9 characterize stem cell population 9 proof-of-principle small animal studies 9 optimize ex vivo culture process in GMP facility 9 safety/dose-ranging studies in large animal models Clinical • phase 2 trials
Slide 10: Major Value Inflexion Points Since 2005
For personal use only Major Value Inflexion Points Since 2005 Technical 1. Demonstrated scale-up of manufacturing process 2. Demonstrated safety of manufacturing and cells in patients 3. Shown that allogeneic (“off-the-shelf”) stem cells are effective Clinical 4. IND submissions for four Phase 2a tr
Slide 11: Lead Clinical Programs
Lead Clinical Programs For personal use only 1. Spinal Fusion 9 single-center, FDA-cleared, dose-escalating Phase 2a study • expansion to randomised multi-center Phase 2a trial • Phase 3 trial 2009 for intervertebral fusion 2. Long bone repair 9 Phase 1b clinical trial long bone non-union completed
Slide 12: Clinical Programs In Partnership With US-Based Angioblast Systems, Inc.
Clinical Programs In Partnership With US-Based For personal use only Angioblast Systems, Inc.
Slide 13: Lead Preclinical Programs
For personal use only Lead Preclinical Programs 7. Age-Related Macular Degeneration (AMD) and Diabetic Retinopathy efficacy demonstrated in rodent study; 42-animal non-human GLP primate study completed optimal dose identified for monotherapy significant synergy in combination with anti-VEGF therapy
Slide 14: Treatment Of Non-Union Long Bone Fractures
For personal use only Phase 1b Trial InOf Treatment Non-Union Non LongLong -Union BoneBone Fracture Repair Fractures Treatment Of Non-Union Long Bone Fractures Study background • open label pilot trial at Royal Melbourne Hospital • autologous, immunoselected culture-expanded MPC • 10 patients with n
Slide 15: MPCs Induce New Bone Growth And Union In A Patient With Femoral Fracture
For personal use only MPCs Induce New Bone Growth And Union In A Patient With Femoral Fracture And Non-Union After 9 Months baseline 6 wks post-MPC 12 wks post-MPC implantation implantation
Slide 16: MPC repair of distal tibia fracture non-union
MPC repair of distal tibia fracture non-union For personal use only Pre-Implant 12 weeks Post-Implant
Slide 17: Commercial Strategies For Biologicals In Regenerative Medicine
For personal use only Commercial Strategies For Biologicals In Regenerative Medicine
Slide 18: Number of Spinal Fusion Procedures in the US
There were c. 360 thousand spinal fusion procedures in 2005 in the US, of which posterior lumbar and anterior cervical comprised over 75% of total procedures For personal use only Number of Spinal Fusion Procedures in the US US Spinal Fusions (2005) Thousands of Procedures Percent CAGR% 358k 400 Not
Slide 19: Impact of BMP-2 on Medtronic's Spinal Division revenues
The introduction of BMP-2 had a dramatic impact on the revenues of Medtronic’s Spinal Division and helped pull through significant instrumentation sales For personal use only Medtronic Spinal Division Revenue CAGR% CAGR% (2001-07) (2005-07) Millions of US dollars 3,000 INFUSE Total 24.8 16.5 2,500 l
Slide 20: Genzyme-Osiris Strategic Alliance Nov. 4/08
For personal use only Genzyme-Osiris Strategic Alliance Nov. 4/08 First Major Pharma Deal In Stem Cells 1. Osiris allogeneic adult stem cell platform, in Phase 3 for 2 clinical indications, in Phase 2 for four clinical indications 2. Genzyme to commercialize stem cell products outside US, Osiris in