Mesoblast Limited · ASX:MSB
Mesoblast — Investor Presentation (June 2009)
21 slides from Mesoblast's investor presentation, Jun 25, 2009
Slide 1: Commercialisation Of Stem Cell Technology
For personal use only Commercialisation Of Stem Cell Technology Lodge Partners June 2009
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 $11m March ’09 • sufficient cash to accomplish major milestones next 12 months
Slide 3: 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 4: 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 5: Mesenchymal Precursor Cell Technology Platform
For personal use only Mesenchymal Precursor Cell Technology Platform Assignment of MPC IMVS/Hanson Institute Intellectual Property ANGIOBLAST (Australia) Systems, Inc. (USA) all other applications Approx. 5% Shareholding MPC License for Based on Licensed IP Orthopaedic Applications Approx. 4% Shareh
Slide 6: 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 7: Proprietary MPC Isolation
Proprietary MPC Isolation Bone Marrow (BM) For personal use only 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 8: 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 9: 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 10: 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 characterize stem cell population proof-of-principle small animal studies optimize ex vivo culture process in GMP facility safety/dose-ranging studies in large animal models Clinical • phase 2 trials
Slide 11: Lead Orthopaedic Programs
Lead Orthopaedic Programs For personal use only
Slide 12: Programs In Partnership With US-Based Angioblast Systems, Inc.
Programs In Partnership With US-Based For personal use only Angioblast Systems, Inc.
Slide 13: Programs In Partnership With US-Based Angioblast Systems, Inc. (c'td)
Programs In Partnership With US-Based For personal use only Angioblast Systems, Inc. (c’td) 4. 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 sy
Slide 14: United States Market Opportunity For CHF Product
For personal use only United States Market Opportunity For CHF Product Class II-IV EF < 35% is 40% of existing 5.5M pool plus 550,000 new-onset annually CHF post-AMI…20% of 800,000 new cases/annually projected annual growth in cell therapy uptake 45% peak penetration Year 8 post-launch, pred
Slide 15: Congestive Heart Failure (CHF) Program
Congestive Heart Failure (CHF) Program For personal use only Pre-Clinical positive results of allogeneic MPC in sheep with established CHF … effects dose-related, independent of mode of delivery (similar results with surgical and endoventricular catheter delivery) positive results of allogeneic
Slide 16: Phase 2a Congestive Heart Failure (CHF) Trial
For personal use only Phase 2a Congestive Heart Failure (CHF) Trial 60 patient multi-center, randomized, controlled trial class II-IV CHF three cohorts of 20 patients, each cohort testing progressively increasing allogeneic cell dose cells injected by JNJ NOGA Myostar catheter 20 patients
Slide 17: Allogeneic MPCs Increase EF In CHF Patients
Over 3 Months, Allogeneic MPCs Increase EF In CHF Allogeneic MPCs Increase EF Most Effectively In CHF Patients (EF < 40), Whereas Controls Deteriorate Patients With Worst Baseline Function (EF <30%) 45 40 40 35 For personal use only Abolute Ejection Fraction (%) Absolute Ejection Fraction % 35 30 30
Slide 18: Bone Marrow Transplantation By Expanding Cord Blood HSC
Bone Marrow Transplantation By Expanding Cord Blood HSC For personal use only • Pre-clinical data shows >20-fold expansion of Hematopoietic Stem Cells (HSC) by MPC co-culture • Orphan Drug designation received for increasing HSC in cancer patients needing allogeneic BMT • Investigator-led IND progra
Slide 19: Clinical Trial Results
Clinical Trial Results For personal use only • First 5 patients transplanted have all engrafted successfully, no safety issues • All have demonstrated >20-fold expansion of HSC by MPC co-culture with allogeneic cord • Median time to neutrophil engraftment 15 days (historic controls approx. 4 weeks)
Slide 20: Eye Program For AMD and/or Diabetic Retinopathy
For personal use only Eye Program For AMD and/or Diabetic Retinopathy positive results of MPC in rodent model of AMD positive results in primate study for IND submission synergistic efficacy with Lucentis IND submission for Phase 2a clinical trial in patients with AMD and/or diabetic retinop
Slide 21: Commercial Strategies For Our Biologicals
Commercial Strategies For Our Biologicals For personal use only