Leeds July 2017 Matthew Durdy Chief Business Officer

Transcription

1 Leeds July 2017 Matthew Durdy Chief Business Officer

2 The Cell and Gene Therapy Catapult 70m Development Facility 1,200m 2 Custom designed cell and gene therapy development facility Prime location in the heart of the London clinical research cluster 120 permanent staff 2 55m large scale manufacture centre 7,200m 2 manufacturing centre designed specifically for cell and gene therapies Located in the Stevenage Biocatalyst Opening 2017

3 Co-ordinated Development Business Uncertainty on reimbursement Poorly understood health economics Business models Manufacturing and supply chain Ability to scale up cost effective, robust and reliable manufacturing Meaningful quality and analytical assays Storage and delivery systems Regulatory and clinical Uncertain, complex regulation Clinical trial site ability to handle live products Cautious hospital research committees 3

4 We collaborate on projects to break down barriers Engineered T cells (blue) Cancer expressing cells (green) Dead Cells (red) CELL VIDEO HERE 24hr time lapse extract from an assay to determine cell killing potential of patients cells post reengineering Product currently in clinical trail 4 Introduction

5 Normalised Cell Index Rapid potency assay of cell killing 4 0:1 1:1 2:1 5: Time (Hours) 2 Potency Assay

6 Cell and Gene Therapy Catapult themes Cost of goods and productivity Supply chain Gene delivery technologies UK The go to place for the development of cell and gene therapies and supply to the global market. Regulatory advantage Rapid clinical adoption and reimbursement Increasing commercialization of research CGT Catapult World leader in technologies for the development, manufacturing and supply of cell and gene therapies. 6

7 C0mmercialisation of research

8 Why commercialise research? Money Fame People

9 Catapult Criteria

10 Catapult Criteria Tractability Mission Catapult fit 1.0 Science 0.0 Ease of Transaction Defensibility Market 10

11 Business Model Framework

12 Business model: not an afterthought Efficacy may dominate discussion in HTA But: speed of sales build up, and market share affected by ability to deliver a product/service, that is easy to buy and competitive

13 The business must meet the needs of many groups Customer layer Key needs Healthcare payers Clinical and cost effective therapy Value for money healthcare Healthcare providers Positive budgetary impact Ease of clinical adoption Clinicians As for patients plus: Adherence to best practice Clinician-patient relationship Patients Increased life span Acceptable quality of life Affordability of treatment Figure 1: Customer pyramid framework for T-cell therapies in cancer. The pyramid represents the healthcare system of a nation, which is the customer for a T-cell therapy and consists of four layers. The size of each layer in the pyramid symbolises the number of people/organisations found in that layer. In terms of access to a T-cell therapy, each customer layer acts as a gatekeeper to the next one down the pyramid, as signified by the door symbol. Each customer layer has a set of key needs that the T-cell cancer therapy must fulfil in order to achieve optimal commercial success.

14 Proposition should be designed to meet all relevant needs Much more that what is in the bag or bottle The Proposition is intertwined with the business model The Proposition is intertwined with clinical development 14

15 The proposition PROPOSITION PRODUCT FORMULATION Cell origin (autologous / allogeneic) Cell concentration Target cancer antigen Cell type Cell design Pharmacologi cal properties Excipient, preservation and packaging Therapeutic indication Techniques and procedures Clinical data package GMP manufacturin g Regulatory approvals/ licenses Manufacturing and supply capability Economic data Pricing, payment and warranty terms Value message communication capability Clinical adoption support Reimbursement support Patient support Long-term patient tracking capability Figure 1: Formulation-Product-Proposition Framework for the commercialisation of T-cell cancer therapies. The therapy can be considered on three levels in terms of what is sold or offered to the customer: (1) formulation (physical manifestation of the therapy); (2) product (formulation plus all the procedures, data and permissions needed for clinical usage of the therapy); and (3) proposition (formulation, product and all additional support offerings/services that should be provided by the manufacturer to the customer to maximise prescribing).

16 Key drivers of the business model Patient population/efficacy relationship Complexity of the clinical interaction Safety profile of the drug Need for strong clinician/patient continuity Complexity of manufacturing process Capital intensity and robustness manufacturing process Supply chain Price and reimbursement mechanism 16

17 Key parameters Key characterist ics Different solutions are emerging: on a spectrum/plane Manufacturing sites for T-cell therapy Cancer centres offering T-cell therapy Single centre co-located manufacturin g BUSINESS MODEL Hub-andspoke Franchise Traditional pharma 1 1 (max 2-3) 1 to many 1 (max 2-3) Advanced therapy treatment centres? 1 Small numbers Moderate numbers Large numbers Autologous approach Challenging safety profile High manufacturing /supply chain complexity Low patient numbers Allogeneic approach Excellent safety profile Low manufacturing /supply chain complexity High patient numbers Figure 1: Business model spectrum for T-cell therapies in immuno-oncology. The diagram shows the spectrum of business models that could potentially be used to commercialise T-cell cancer therapies. The key differentiating characteristics between the business models are considered to be the number of manufacturing sites and treatment facilities located in a given territory (USA or EU). For a specific T-cell therapy, the key parameters described are likely to dictate which business model may be most suitable for commercialisation.

18 Game changers affect the four main drivers Patient population/efficacy relationship Stealth or universal cell Precision medicine Complexity of the clinical interaction Safety switches Complexity of manufacturing process In vivo therapy Expansion free autologous modification Stealth or universal cell Price and reimbursement mechanism Self reporting efficacy 18 Introduction

19 Five Question Framework

20 Accessible Need Is there value enough for everyone?

21 Proposition Does the offering harvest the the need?

22 Defensibility How long will it last?

23 The Team Can I rely on them to make this plan happen?

24 Plan What are we going to do together?

25 Solving industry problems Thaw in clinic device Project: Development of a benchtop thawing system in order to control and monitor final stages of release and use of cryopreserved therapies. Investment: 1.2 million Addressing: Crucial gap in the GMP cryochain No certified medical device on the market for thawing No robust reporting architecture to control and monitor final stages of release of cryopreserved therapies Current system (waterbath) can lead to process variability and inconsistencies in treatments Set up: CGT Catapult issued a challenge call and identified UK SME Asymptote as the best partner to work with on the project. 25

26 Answer all positively, or stop and think again Accessible Need Proposition Defensibility Team Plan

27 Early intervention enhances future value 27 Introduction

28 Cell and Gene Therapy Catapult 12th Floor Tower Wing Guy s Hospital Great Maze Pond London SE1 9RT +44 (0) info@ct.catapult.org.uk ct.catapult.org.uk 28