european regenerative medicine firms & their strategic approaches michael morrison university of...

European Regenerative Medicine Firms & Their Strategic Approaches

Michael Morrison

University of York

OVERVIEW

Creating the European RM ‘Universe’ of firms

Characterizing the European RM Universe

Strategic Analysis

CREATING THE EUROPEAN REGENERATIVE MEDICINE ‘UNIVERSE’

DEFINING REGENERATIVE MEDICINE

Regenerative medicine is a heterogeneous domain, incorporating multiple technological avenues of investigation.

United by a shared goal of stimulating, directing or augmenting the body’s capacity for self-repair and regeneration.

Our approach concentrates on technologies using novel biomaterials – living cells, genes and bioscaffolds.

DEFINING REGENERATIVE MEDICINE

Excludes small molecules and biologicals (e.g. antibodies) that aim to stimulate in vivo regenerative action.

Large pharmaceutical companies with an interest in aspects of regenerative medicine such as Pfizer, Sanofi-Aventis, Smith and Nephew etc not included on the list

Basic cell culture tools/media suppliers not included unless have specialist focus.

DEFINING REGENERATIVE MEDICINE

‘European’ companies must be based entirely in a European state, or have their headquarters there.

Subsidiaries of non-European firms are not included.

Company must have some publicly available details (usually in the form of a website).

CHARACTERIZING THE EUROPEAN REGENERATIVE MEDICINE FIRM UNIVERSE

FIRM TYPE BY PRIMARY ORIENTATION

N = 59

GEOGRAPHIC DISTRIBUTION OF FIRMS

N =59

FIRMS BY SIZE (EMPLOYEE NOS)

FIRMS BY AGE (YEAR FOUNDED)

SUMMARY OF FIRM CHARACTERISTICS

Cell therapy is the largest single sector Significant no. of bioscaffold and service

firms Most firms are small (< 99 employees) Wide range of company ages – many

remaining from 1990s and pre 2002 ‘crash’ UK and Germany are the main geographic

hubs of commercial RM activity

STRATEGIC ANALYSIS OF EUROPEAN REGENERATIVE MEDICINE FIRMS

AUTOLOGOUS VS ALLOGENIC CELL THERAPY

STEM CELL VS SOMATIC CELL THERAPY

THERAPEUTIC FOCUS OF CELL THERAPY FIRMS

SALEABLE THERAPEUTIC PRODUCTS

RM PRODUCTS IN DEVELOPMENT

GENE THERAPY COMPANIES

Name Location

Size Found’d Disease focus

Notes

Amsterdam Molecular Therapeutics (AMT)

NL Small 1998 Metabolic disorder

Lead product Glybera submitted for EMA MAA in 2009

Ark Therapeutics UK Medium 1997 Vascular occlusion. Cancer

Cerepro submitted to EMA for MAA in 2008, received rejection 2009. Trinam in phase III trials

Arthrogen BV NL Small 2005 Rheumatoid Arthritis

Lead product still at preclinical stage

Diamyd Medical AB

Sweden Small 1976 Chronic pain, cancer

Main therapeutic programme is therapeutic vaccines for diabetes.

Oxford BioMedica UK Small 1996 Neurodegenera-tive, Ocular

Lead product in phase I/II

Transgene France Small 1979 Cancer Several cancer vaccines in development

Mol Med Italy Small 1996 Graft vs. Host disease

Lead product in Phase III trials

Mologen Germany Small 1998 Cancer Developing non- RM anti-cancer therapeutics

GENE THERAPY LANDSCAPE

Engagement with Advanced Therapy Medicinal Products regulation:

Ark’s Cerepro rejected in 2009 but has named-patient approval in France and Finland

Amsterdam Molecular Therapeutics’ Glybera submitted for MAA in 2010

MolMed (Italy) have cell-based ex-vivo gene therapy in phase III trials

Service Total Stem cell?

Cell and tissue based drug discovery / screening services / toxicity testing

4 2

Cell and tissue supply including research and clinical grade material

4 2

Contract manufacture of cells / cell line expansion, cell culturing and scale up

1 -

Specialist tools and reagents 3 -Distribution of bioscaffolds for tissue repair, wound healing etc

1 -

TOTAL 13 4

FOCUS OF DEDICATED SERVICE PROVISION FIRMS

CONCLUSIONS

CONCLUSIONS

Companies pursuing newer regenerative medicine models appear to co-exist with older tissue engineering and gene therapy firms.

Strong industry preference for less risky technologies – autologous cell therapy with somatic cells or adult stem cells.

Little commercial investment in iPS and embryonic stem cell technologies.

CONCLUSIONS

Commercial development concentrated in Western Europe, and concentrated in hubs.

Importance of biotechnology infrastructure and financial support.

Regulatory environment is also an important shaping factor – as with hESCs

Can be illustrated by Germany – UK RM firm breakdown

CONCLUSIONS

RM Sub-sector Germany UK

Cell Therapy 9 6

Gene Therapy 1 2

Bioscaffolds 2 4

Service/ other 3 9

TOTAL 15 21

http://www.york.ac.uk/res/remedie