Regulation of Advanced Therapies

A Biotech SME Perspective

May 11, 2006

Gil BeyenCEO - TiGenix N.V.www.tigenix.com

Member of the EuropaBio Advanced Therapies Working Groupwww.europabio.org

EuropaBio

EuropaBio is the voice of the biotechnology industry in Europe

EuropaBio represents:+ around 1500 SMEs+ 68 large companies+ 25 national biotechnology associations across Europe + 5 bio-regions

EuropaBio champions the responsible use of biotechnology to ensure that its potential is fully used to the benefit of people and their environment in all fields, including healthcare.

TiGenix – an SME profile

Founded : 2000, spin-off of Universies of Leuven & Ghent

Financing: Private - Venture backed (29 M€)Team: 45 people

Focus: Regenerative medicine for joint surface repair

Science: Biology of stable hyaline cartilage

Approach: Evidence Based Medicine« From procedure to validated medicinal product »

Lead product: ChondroCelect, autologous cell therapy product for cartilage repair in Phase III clinical trial

SME

More than 2 million patients per year with cartilage damage (US + EU) Cartilage damage known to be one of the main causes for OA OA affecting over 10% of the population; one of leading cause of

disability at older age; fast growing; no effective treatments today

Need for biological solutions leading to durable repair(JRC report: market potential cartilage regeneration 2011: 25 bn

€)

Major unmet medical need

Age

Overweight

Cartilage damage

Genetics

Osteoarthritis

Autologous Chondrocyte Implantation (ACI)

3. Implantation

Articular surface

Subchondral bone

1. Biopsy

2. Cell culture

4. Rehab

Full thickness cartilage defect

?

To produce cells that can build stable cartilage in vivo

To develop a consistent, reliable product with proven efficacy

To position the product in the treatment algorithm of joint surface defects

To obtain approval & reimbursement

ChondroCelect

Goal: validated therapeutic product for durable repair

ChondroCelect cell technology

Proprietary potency assay

Extensive preclinical validation

GMP with batch release criteria

Prospective randomised GCP controlled clinical trials

Health economics studies

Key challenges Key responses

Condition: Harmonised and Equitable Regulatory Framework

High standards patient & public confidence

Harmonised requirements

Adapted to specific needs

Data exclusivity & IP protection

Possibility for branding & pricing

Equitable for all parties

The ‘bar can (and should) be high’ as long as at same level for all parties ...

achievable

economic return; condition for innovation

The proposed ATP Regulation provides important step forward (1)

Main positive points:

Single legislation for all Advanced Therapy Medicinal Products

... as a regulation

... utilizing an existing product framework

... tailor made to ATP

Centralised procedure ensuring harmonised standards

Data exclusivity & IP protection foreseen

Financial incentives & administrative help

The proposed ATP Regulation provides important step forward (2)

Remaining concerns:

Equal requirements for all parties (also for hospital-based) ...without hindering academic research

Will there be sufficient expertise and what role? Composition of the CAT and role of CAT advice

Implementation specifics (definitions, adaptation of the Clinical trials and GMP Directives)

Speed of implementation

Conclusions

Cell therapies and tissue engineering will change medical practices profoundly

Harmonized and equitable regulatory framework, ‘conditio sine qua non’ for the necessary innovation and to ensure patients access to valuable new therapies

TiGenix (SME) and EuropaBio welcome the draft proposal ...

... and look forward to contributing to a speedy adoption process

Thank you!

More info:

Gil Beyenwww.tigenix.com

EuropaBiowww.europabio.org