Regulation of Advanced Therapies

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• Regulation of Advanced Therapies
• An SME Perspective
• April 11, 2006

Gil Beyen CEO TiGenix N.V. www.tigenix.com
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Profile

• Founded 2000, spin-off of Universies of Leuven
  Ghent
• Private - Venture backed (29 MM)
• Team 45
• 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 (medicinal product) 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
• Limited life span of prosthesis, combined with
  ageing of the population
• ? Need for biological solutions leading to
  durable repair

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


3. Implantation Injection of cell
suspension under membrane
Durable repair

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

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

• To produce cells that can build stable cartilage
  in vivo
• To develop a consistent, reliable cellular
  therapy with proven efficacy
• To position the product in the treatment
  algorithm of joint surface defects
• To obtain approval reimbursement

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Condition Harmonized and Equitable Regulatory
Framework

• What is important for Industry (and other
  organizations) to make these investments?
• Clear and harmonized requirements, adapted to
  specific needs
• Data exclusivity IP protections
• Possibility for branding,
• Possibility for pricing reimbursement
• Equitable regulation for all parties
• ?The bar can (and should) be high, as long as
  at same level for all parties

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The proposed ATP Regulation provides important
step forward

• Main positive points
• Regulation
• As medicinal product
• Specificity of ATP products recognized
• Data exclusivity IP protection
• Centralized procedure
• Financial incentives
• ...
• Remaining concerns
• Equal requirements for all parties (also for
  hospital-based)
• Implementation specifics (definitions, GMP, ...)
• Implementation timeline

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Thank you!More infoGil Beyen32 16
396060www.tigenix.com