The document provides an overview of drug development and the biotechnology industry. It discusses the traditional and contemporary business models in the industry, which have shifted from closed innovation to more open innovation. It also outlines the services offered by one company, including clinical development support and regulatory consultancy. The document describes the company's research interests in areas like vaccines, proteins, and monoclonal antibodies. It includes organizational charts and provides details about the company's project pipeline across various phases of drug development. Finally, it discusses challenges in the industry like high costs and manufacturing techniques for cost reduction.

1. Drug Development &
Biotechnology
Case Study for PCR901
November 16, 2011
Karen, Nichole & Zinia

2. Outline
• Introduction
• Departmental / Team Structure
– Organogram
• Business Model
• Research Interests
• Services Offered
• Project Team Composition
• The Legendary Case Study
• Challenges & Rewards

3. Introduction: Biologics and Biopharmaceuticals
• Biotechnology
– The use of living organisms and bioprocesses in
engineering, technology, medicine and other fields
requiring bioproducts.
• Biopharmaceutical
– A medical drug derived from biological sources and
produced by biotechnology.

4. THERAPEUTIC
PROTEINS
TISSUES AND MONOCLONAL
CELLS ANTIBODIES
BIOTECH
BLOOD
VACCINES
COMPONENTS
IMMUNO-
THERAPY

5. BUSINESS MODEL

6. “One of the biggest challenges for the
biotechnology and pharmaceutical
companies in the 21st century will be
to develop and deliver drugs that fit
the individual patient’s biology and
pathophysiology”
Jan Trost Jorgensen
principal scientist at Dako

7. Traditional Business Model
• Searching for the “Blockbusters”
• Closed Innovation model
• a.k.a. the FIPCOs (Fully Integrated
Pharmaceutical Companies)
– Majority of research is conducted internally
– Firms conduct basic research, confident that
they have downstream manufacturing and
commercialization capabilities
– Accidental (serendipitous) discoveries that did
not fit with corporate therapeutic interests were
most often cancelled

8. Traditional Business Model
• Open Innovation model
– Expectation is that knowledge is acquired from
external sources
– Licence out knowledge that does not fit the
business model

9. Contemporary Business Model
• Large pharma:
– In house development, manufacturing, marketing and
sales;
– Some with generic/biosimilar subdivisions
• Medium pharma:
– Outsource one or more components, retain in house
marketing and sales
• Small pharma:
– Bring in products researched, developed and
manufactured elsewhere
– Simply register, import / acquire, store and distribute

10. Biotechnology Industry
• Company size by number of employees (%)
Size Percentage
1 – 20 28.1%
21 – 50 19.8%
51 – 100 11.9%
101 – 200 9.9%
201 – 400 4.1%
401+ 6.2%

11. SERVICES OFFERED

12. • Pre-Clinical & Clinical
Development
• Registration
Submissions & Support
• GxP (GMP, GCP, CLP)
& Regulatory
Consultancy
• CRO Qualification
• Preparatory regulatory
audits
• Due Diligence Audits

13. Typical Areas of Product Development
• Vaccines
• Peptides and Proteins
• Follow On Biologics
• Natural Products
• Transdermals
• Radionucleotides
• Osteopathic Devices
• In-vitro Diagnostic Reagents

14. RESEARCH INTERESTS

15. Growing Importance of Biotech
Medicine
• Proven to be safe and effective
• Biotechnology has produced more than 125
medicines
• Treatment for some of the most serious and
intractable diseases
• In 2008:
– 633 biotech medicines in development
– 254 for cancer and related conditions
– 162 for infectious diseases
• Full therapeutic potential can take time to be
realized; new treatment advances are often realized
after product has been on the market for some time

16. Drug Discovery Research

17. On going Research Topics

18. Specific Research
Erythropoeteins:
• The hormone responsible for producing red blood
cells in bonemarrow
• Most common use in people with anemia related
to kidney disfunction
• Drugs Epoitin alpha ( Epogen, Procrit)
• Darbepoetin alpha (Aranesp)

19. Specific Research
Monoclonal antibodies
• The newest one approved is Rituximab (Rituxan)
• Several monoclonal anibodies are being
researched for treating rheumatoid arthritis (RA)
and other conditions
• Anticancer and immunological therapies using
monoclonal antibodies are being a focus of
reseach

20. Current Research Focus
Long Term
conditions
Genetic
Engineering Oncology
Biotechnology
current
Gene therapy Infectious
diseases

21. PROJECT TEAM COMPOSITION

22. Project Team Composition
• Clinicians
• Pharmaceutical
Scientists
• Regulatory Strategists
• Business
Development Experts
• Market Analysts

23. DEPARTMENTAL / TEAM
STRUCTURE

24. R&D Organizational Structure
Chairman of
the Board and
CEO
EVP Research
& Development
SVP SVP Regulatory SVP Executive
SVP Discovery SVP Clinical VP Program Mgt
Translational Affairs and International Director
Research Development & Strategic Ops
Sciences Safety R&D Compliance
VP Comparative Exec Dir
VP Research VP Scientific VP Regulatory Exec Medical Dir Director Quality
Biology & Safety Strategic
(Chemistry) Affairs Affairs (EU) Compliance
(Preclinical) Operations
VP PK & Drug Exec Dir Director
VP Research VP Development Exec Medical Dir
Metabolism VP Safety Program Mgt Compliance
(TA) (TA) (North America)
(Preclnical) (TA) Auditing
VP Process & Exec Dir Director
VP Research VP Development VP Intl RA & Exec Medical Dir
Product Program Mgt Systems
(TA) (TA) Safety (Asia/Pacific)
Development (TA) Compliance
Exec Dir RA & Exec Medical Dir Exec Dir
VP Research VP Medical VP Development Director Training
Safety (Emerging Program Mgt
(TA) Sciences (TA) & Development
Operations Countries) (TA)

25. R&D Organization Structure
Level 9 Vice President
Level 8 Executive Director
Level 7 Director
Level 6 Principal/Senior Manager
Level 5 Specialist/Manager
Level 4 Senior Associate
Level 3 Associate

26. CASE STUDY

27. New Product Development
Drug
Drug Discovery Commercialization
Development

28. New Product Development
Drug
Drug Discovery Commercialization
Development

29. Drug Discovery
• Project Procurement
– Identify unmet medical need
– What is known about the disease?
– What are the current treatment options, if any?
– Does the company have the expertise, technology
and financial resources?
– Potential competitors and regulatory constraints?

30. Drug Discovery
Top 5 Death Causing Diseases in 2005 (NCHS)

31. Therapeutic Areas (TA)
• Oncology
• Inflammation
• Hematology/Nephrology
• Metabolic Disorders
• Neuroscience
• Cardiovascular

32. Drug Discovery
Research Interest
Target Discovery
Target Validation
Screening
Drug Design

33. New Product Development
Drug
Drug Discovery Commercialization
Development

34. Drug Development
Preclinical
studies
Clinical
Study: Phase
I
Post-Market Clinical
Surveillance: Study: Phase
Phase IV II
Clinical
Study:
Phase III

35. Product Pipeline: Phase I
Product Name Product Modality Therapeutic Area
1 AMG139 Antibody Inflammatory Diseases
2 AMG145 Antibody Hypercholesterolemia
3 AMG151 Oral/Small Molecule Type II Diabetes
4 AMG157 Antibody Asthma
5 AMG167 Antibody Bone-Related Conditions
6 AMG181 Antibody Ulcerative Colitis
7 AMG191 Antibody Inflammatory Diseases
8 AMG208 Oral/Small Molecule Various Cancer Types
9 AMG319 Oral/Small Molecule Hematologic Malignancies
10 AMG337 Oral/Small Molecule Various Cancer Types

36. Product Pipeline: Phase I (cont'd..)
Product Name Product Modality Therapeutic Area
11 AMG557 Antibody Lupus
12 AMG745 Protein Muscle-Wasting Disorder
13 AMG747 Oral/Small Molecule Neuroscience
14 AMG761 Antibody Asthma
15 AMG780 Antibody Various Cancer Types
16 AMG811 Antibody Lupus
17 AMG820 Antibody Various Cancer Types
18 AMG888 Antibody Various Cancer Types
19 AMG900 Oral/Small Molecule Various Cancer Types
20 Dulanermin Protein Various Cancer Types

37. Product Pipeline: Phase II
Product Name Product Modality Therapeutic Area
1 AMG386 Protein Various Cancer Types
2 AMG785 Antibody Bone-Related Conditions
3 AMG827 Antibody Inflammatory Diseases
4 AMG853 Oral/Small Molecule Asthma
5 Conatumumab Antibody Various Cancer Types
6 Denosumab Antibody Rheumatoid Arthritis
7 Ganitumab Antibody Various Cancer Types
8 Motesanib Oral/Small Molecule Breast Cancer
9 Nplate® Protein Thrombocytopenia
10 Nplate® Protein Myelodysplastic
Syndromes

38. Product Pipeline: Phase II (cont'd..)
Product Name Product Modality Therapeutic Area
11 Omecamtiv mercarbil Oral/Small Molecule Heart Failure
12 Rilotumumab Antibody Various Cancer Types
13 Sensipar® Oral/Small Molecule Post Renal Transplant
14 Vectibix® Antibody Head and Neck Cancer

39. Product Pipeline: Phase III
Product Name Product Modality Therapeutic Area
1 AMG386 Protein Ovarian Cancer
2 Anaresp® Protein Anemia in Heart Failure
3 Ganitumab Antibody Pancreatic Cancer
4 Motesanib Oral/Small Molecules Lung Cancer
5 Prolia® Antibody Male Osteoporosis
6 Sensipar® Oral/Small Molecules Cardiovascular Disease
and Chronic Kidney
Disease
7 Vectibix® Antibody Colorectal Cancer
8 XGEVA™ Antbody Bone Metastases in
Breast Cancer
9 XGEVA™ Antibody Bone Metastases in
Prostate Canacer

40. Marketed Products: Phase IV
Product Name
1 Anaresp® (darbepoetin alfa)
2 Enbrel® (etanercept)
3 EPOGEN® (Epoetin alfa)
4 Neulasta® (pegfilgrastin)
5 NEUPOGEN® (Filgrastim)
6 Nplate® (romiplostim)
7 Prolia™ (denosumab)
8 Sensipar® (cinacalcet)
9 Vectibix® (denosumab)
10 XGEVA™ (denosumab)

41. CHALLENGES & REWARDS

42. Challenges and Resolutions
• More Expensive
• Biosimilar Verification
• Small molecule vs Large molecule therapies
• Required comprehensive immunogenecity testing
• Extended post market surveillance
• Sterile Environment requirement
• Batch-to-batch release – Section 12 of the ACT

43. Manufacturing techniques for cost
reduction
• Portable clean room technology
• The use of cell culture perfusion for production of
antibodies
• Sequential micro column chromatography
• Extensive use of inline dilution of buffer
concentrates

44. Advantages
• Stem cell therapies have the ability to eliminate
concerns over viral or prion contamination prior to
organ replacement
• Biopharmaceuticals have fewer side effects and
more potent effects on target cells
• Pharmaceutical biotechnology is being further
developed to fight cancer, viral infections,
diabetes and hepatitis
• Safer and more effective antibiotics, insulins,
interferons, estrogen and human growth
hormones are being developed

45. Conclusions
• Globally, Canada ranks 2nd in number of biotech
companies and spends the most on R&D per
employee1
• Some slowdown in venture capital since decline
of Harvard oncomouse patent
• Significant growth in Quebec, Ontario and BC;
biotechnology is the place to be for up-and-
coming innovators
1Statistics Canada; http://www.nature.com/bioent/2004/040501/full/bioent810.html

46. Questions & Comments?