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Drug Development & Biotechnology
Case Study for PCR901
November 16, 2011
Karen, Nichole & Zinia
Outline
• Introduction• Departmental / Team Structure
– Organogram• Business Model• Research Interests• Services Offered• Project Team Composition• The Legendary Case Study• Challenges & Rewards
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.
BIOTECH
THERAPEUTIC PROTEINS
MONOCLONAL ANTIBODIES
VACCINES
IMMUNO- THERAPY
BLOOD COMPONENTS
TISSUES AND CELLS
BUSINESS MODEL
“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
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
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
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
• Company size by number of employees (%)
Biotechnology Industry
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%
SERVICES OFFERED
• Pre-Clinical & Clinical Development
• Registration Submissions & Support
• GxP (GMP, GCP, CLP) & Regulatory Consultancy
• CRO Qualification• Preparatory regulatory
audits• Due Diligence Audits
Typical Areas of Product Development
• Vaccines• Peptides and Proteins• Follow On Biologics• Natural Products• Transdermals• Radionucleotides• Osteopathic Devices• In-vitro Diagnostic Reagents
RESEARCH INTERESTS
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
Drug Discovery Research
On going Research Topics
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)
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
Current Research Focus
Biotechnology current
Long Term conditions
Oncology
Infectious diseasesGene therapy
Genetic Engineering
PROJECT TEAM COMPOSITION
Project Team Composition
• Clinicians• Pharmaceutical
Scientists• Regulatory Strategists• Business
Development Experts• Market Analysts
DEPARTMENTAL / TEAM STRUCTURE
R&D Organizational StructureChairman of
the Board and CEO
EVP Research & Development
SVP Discovery Research
VP Research (Chemistry)
VP Research (TA)
VP Research (TA)
VP Research (TA)
SVP Translational
Sciences
VP Comparative Biology & Safety
(Preclinical)
VP PK & Drug Metabolism (Preclnical)
VP Process & Product
Development
VP Medical Sciences
SVP Clinical Development
VP Scientific Affairs
VP Development (TA)
VP Development (TA)
VP Development (TA)
SVP Regulatory Affairs and
Safety
VP Regulatory Affairs
VP Safety
VP Intl RA & Safety
Exec Dir RA & Safety
Operations
SVP International
R&D
Exec Medical Dir (EU)
Exec Medical Dir (North America)
Exec Medical Dir (Asia/Pacific)
Exec Medical Dir (Emerging Countries)
VP Program Mgt & Strategic Ops
Exec Dir Strategic
Operations
Exec Dir Program Mgt
(TA)
Exec Dir Program Mgt
(TA)
Exec Dir Program Mgt
(TA)
Executive Director
Compliance
Director Quality Compliance
Director Compliance
Auditing
Director Systems
Compliance
Director Training & Development
R&D Organization Structure
Level 3
Level 4
Level 5
Level 6
Level 7
Level 8
Level 9 Vice President
Executive Director
Director
Principal/Senior Manager
Specialist/Manager
Senior Associate
Associate
CASE STUDY
Drug Discovery Drug Development Commercialization
New Product Development
Drug Discovery Drug Development Commercialization
New Product Development
• 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?
Drug Discovery
Top 5 Death Causing Diseases in 2005 (NCHS)
Drug Discovery
Therapeutic Areas (TA)
• Oncology• Inflammation• Hematology/Nephrology• Metabolic Disorders• Neuroscience• Cardiovascular
Research Interest
Target Discovery
Target Validation
Screening
Drug Design
Drug Discovery
Drug Discovery Drug Development Commercialization
New Product Development
Clinical Study: Phase I
Clinical Study: Phase II
Clinical Study: Phase
III
Post-Market Surveillance:
Phase IV
Drug Development
Preclinical studies
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
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
Product Pipeline: Phase I (cont'd..)
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
Product Pipeline: Phase II
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
Product Pipeline: Phase II (cont'd..)
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
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)
CHALLENGES & REWARDS
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
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
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
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
Questions & Comments?
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