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Natalie mount-presentation

  1. 1. Cell Therapy in Big Pharma: The Pfizer Neusentis Approach Natalie Mount PhD Executive Director Neusentis Regenerative Medicine Stem Cells USA and Regenerative Medicine Congress Sept 2011 Neusentis is a wholly owned subsidiary of Pfizer
  2. 2. Presentation overview • Introduction to Neusentis Regenerative Medicine: • Strategy and vision • Selected cell therapy case studies from our portfolio • Challenges and opportunities for cell based therapy in big pharma: • Development • Commercialisation Pfizer Confidential 2
  3. 3. Our Vision & Strategic Themes in Regenerative Medicine Build an industry leading group of scientists and partners to discover, develop and launch innovative cell based therapies to patients • Build knowledge and expertise (clinical, regulatory, safety, logistical) in cell therapy to enable successful development of products • Evaluate allogeneic, autologous and hES cell based approaches • Identify soluble factors that modify cell fate • Build knowledge on business models for a cell based therapy and how to enable a future successful commercial launch • Partner with the Pfizer Business Units to understand key issues in commercialization of a cell based therapy • Partner externally as much as possible to access the best science and accelerate development of the best approaches Science Commercial Partnering Vision 3Pfizer Confidential
  4. 4. The Potential For Cell Based Products Is From Disease Modification To Cure Source: The Frankel Group LLC Biologics Tissue Engineering Cell-Based Products OTC Pharmaceuticals Correct defect, regenerate tissue Relieve first- order symptomsRelieve second-order symptoms Increasing Value of Treatment to Patient OTC=Over The Counter Disease Modification Increasing Revenue / Patient Replace defective organ/tissue Small Molecules 4 Cell Therapy Pfizer Confidential
  5. 5. What different approaches are we taking to regenerative medicine? • Allogeneic adult stem cell therapy • e.g. Athersys MultiStem collaboration • Therapeutic administration of differentiated cells derived from embryonic (or iPS) cells • e.g UCL RPE collaboration • Small molecule & biologic modifiers of endogenous stem cells • Autologous stem cell therapy Pfizer Confidential 5 ‘The development of products (small molecules, biologics, cells) that restore function in damaged or aging tissues and organs’ Underpinned by knowledge of : - stem cell science - developmental pathways
  6. 6. Our Portfolio: hEs Differentiated Cells – University College, London Collaboration Pfizer Confidential  A stem cell derived therapy to replace retinal pigment epithelial (RPE) cells and restore retinal function for patients with macular degeneration  RPE project collaboration drivers:  Highly respected academic and medical partners (University College, London, Institute of Ophthalmology and Moorfields Eye Hospital)  Strong rationale  Non-integrating, well-characterised cells  Low cell number required  Synergy with Pfizer’s Ophthalmology disease area  3 clinical populations may benefit from RPE replacement:  RPE tear: no current treatment, results in blindness in affected eye  Wet Age-related macular degeneration (AMD): choroid pushes through weakened RPE junctions leading to bleed into back of eye – currently treatment is anti-VEGF  Dry AMD: retina detaches from its support due to build-up of retinal debris, slow progression; no major treatment available 6
  7. 7. Our Portfolio: hEs Differentiated Cells - UCL/Pfizer Collaboration Differentiate human embryonic stem cells (hESC) into RPE Seed RPE on immobile support Place matrix + cells behind retina under fovea 1.5 mm Fovea Macula 7 Pfizer Confidential En face view of polyester membrane seeded with RPE cells
  8. 8. Challenges for development of cell based therapies • Cell biology – cell selection, characterization and control • Toxicology program – design and interpretation, cell tracking • Mechanism of action • Clinical trial design – safety; dose and dosing regimen; determination of efficacy • CMC (cells, process, devices) and manufacturing at scale • Trial supply logistics – centralised vs point of care preparation Pfizer Confidential 8
  9. 9. Clinical development program goals Pfizer Confidential Phase 1 Phase 2a Phase 2b Phase 3 (Phase 3b/4) Safety in relevant population Explore dose/dosing regimen Pharmacodynamics / mechanism Robust evidence of efficacy (randomised, double blind, placebo controlled) Explore trial population Build safety Finalise dose / dosing regimen Confirm trial population and background therapy Select endpoints (Regulatory authority and payor) Safety Confirm efficacy and safety to support registration, label claims and reimbursement Initial product manufacturing process Scaled near-final manufacturing process Finalised manufacturing process 9
  10. 10. Our Portfolio: Allogeneic Adult Stem Cell Therapy for IBD - Athersys Collaboration 10 Pfizer Confidential  MultiStem® Adult multipotent progenitor stem cell therapy for Inflammatory Bowel Disease  Collaboration drivers:  Synergy of vision for a product in this area between management teams  MultiStem® previous clinical experience  Strong rationale  Well-characterised and highly expandable cells  Pfizer’s experience in running trials and developing therapies in IBD  Development of MultiStem for IBD has progressed very quickly since the collaboration was signed late December 2009:  IND approval November 2010  Clinical trial to demonstrate safety and efficacy in moderate-severe ulcerative colitis started Feb 2011
  11. 11. FDA has approved ulcerative colitis clinical trial Low dose regimen Cohort 1 (safety) High dose regimen Cohort 2 (safety) High dose regimen Cohort 3 (efficacy) Population: Patients with moderate-severe ulcerative colitis; n=126 Main Objectives: Safety and tolerability Efficacy as measured by effect on disease (rectal bleeding and endoscopic healing) Design: Double blind, randomised, placebo controlled, parallel group, efficacy assessments at 4 & 8 weeks Pfizer confidential 11
  12. 12. Overview of MultiStem® Production Process Lot Release & Product Characterization Testing Sterility Potency Identity and Viability Stable Cytogenetics Absence of ectopic tissue potential in vivo Confidential 12
  13. 13. Pharmacodynamic Markers Cell populations (by FACS) Serum proteins (by Multiplex ELISA) [RNA changes in blood and biopsies] Examine ‘change from baseline’ across the dosing period Link changes in cell population with changes in protein (and potentially RNA) expression Assay panels to examine numbers of and activity of: T-cell (naive & memory subsets, Tregs) NK cell B cell (immature, naïve, isotype switched memory and marginal zone) Analysis will be based on changes observed in FACS, ELISA and/or pre-clinical animal model Measurement of ~90 proteins found in serum - includes cytokines, chemokines and other bioactive proteins 13
  14. 14. Challenges for commercialisation of cell based therapies • Manufacturing requires the most differentiated capabilities and contributes disproportionately to the cost of treatment • Building the business case: • Efficacy, safety and differentiation in chosen disease indication • Supply model • IP • Pricing and reimbursement Pfizer Confidential 14
  15. 15. Summary • Developing cell based therapies brings unique Development and Commercialisation challenges • Excellence in clinical program design can mitigate risk through a framework whereby the goals of each phase are clearly established and integrated with manufacturing investments to deliver a de-risked product • In Neusentis Regenerative Medicine, we have made investments across technology platforms to create a diversified portfolio, to systematically address development and commercialization challenges, and provide flexibility to move quickly as the science breaks 15Pfizer Confidential