Regulation of Advanced Therapies

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• Regulation of Advanced Therapies
• A Biotech SME Perspective
• May 11, 2006

Gil Beyen CEO - TiGenix N.V. www.tigenix.com Memb
er of the EuropaBio Advanced Therapies Working
Group www.europabio.org
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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.

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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
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Major unmet medical need
Osteoarthritis
Age
Overweight
Genetics
Cartilage damage

• 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 )

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Autologous Chondrocyte Implantation (ACI)
Full thickness cartilage defect
1. Biopsy
2. Cell culture


3. Implantation

Articular surface Subchondral bone
?
4. Rehab
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ChondroCelectGoal validated therapeutic
product for durable repair
Key challenges
Key responses

• 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 cell technology
• Proprietary potency assay
• Extensive preclinical validation
• GMP with batch release criteria
• Prospective randomised GCP controlled clinical
  trials
• Health economics studies

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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

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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

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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

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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

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Thank you!More infoGil Beyenwww.tigenix.c
omEuropaBiowww.europabio.org