Drug development & biotechnology


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  • Immunotheraphy = can be suppression immunotherapy (allergy products) or activation immunotherapy (cancer products)Tissues and cells = transplantationBlood and blood components = e.g. clotting factorsHuman cells or tissue intended for implantation, transplantation, infusion, or transfer into a human recipient = e.g. bone, skin, corneas, ligaments, tendons, dura mater, heart valves, hematopoietic stem/progenitor cells derived from peripheral and cord blood, oocytes and semen.
  • Dako – leading the way in cancer diagnostics
  • Small biotech companies usually derived from universitiesLarge companies such as Genentech, Amgen, Biogen Idec
  • Pre-Clinical & Clinical Development provide Legal strategies and patent development, Clinical protocol development and study designs for phases I through IV, BA and BE clinical approaches and protocol development, PK / PD modeling and simulation, Medical writing, Biostatistical and statistical approaches, Formulation and process evaluation.Registration Submissions & Support provide support with many regulatory submissions. Including IND/NDS, DMF, Annual Reports, PSUR, NDA, BLA, PMA, ANDA, eCTD etc.GxP (GMP, GCP, GLP) & Regulatory Consultancy provide advice to clients on a range of regulatory & technical issues. As well as implementation of quality management systems, Advice on legal and regulatory issues including Patent challenges, Citizen’s petitions, Litigation, and assisting with responses to regulatory inspectionsCRO QualificationHelp acquire an appropriate contract research organization (CRO) or by performing audits to specified GxP standard. Assess potential contract services for Clinical supplies, Production, Packaging, Testing etc.Preparatory regulatory auditsHelp prepare for regulatory inspection by conducting one or more “practice” regulatory audits in advance of the pending “actual” Health Canada, FDA, EMEA, etc. regulatory visit.Due Diligence Audits Assist pharmaceutical companies, investment banks & private venture capitalists in the process of due diligence for potential acquisitions, buy-ins, joint ventures & other investment projects. We perform in-depth assessment of the regulatory compliance status of companies or projects.
  • Get an example of one product that may have multiple indications – Enbrel?
  • Identifying, cutting out and pasting in various genes to create an “expression vector”The expression vector creates RNA from the genetically engineered DNA of the expression vector The protein(s) of interest is made from RNAThe proteins are biopharmaceuticals.
  • Oncology studies is the biggest focus in this sector due to severity of cancers in modern societyInsulin therapies are the second focus in this sector
  • Biopharmaceutical companies are changing their long term focus on long term conditions, such as, cardiovascular disease, diabetes and asthmaGene therapy and genetic engineering development would influence the potential of biopharmaceuticals.
  • Expertise?
  • NCHS = National Center for Health Sciences
  • 21 Products
  • 21
  • 14
  • 9
  • AS = Ankylosing Spondylitis
  • Drug development & biotechnology

    1. 1. Drug Development & Biotechnology Case Study for PCR901 November 16, 2011 Karen, Nichole & Zinia
    2. 2. Outline • Introduction • Departmental / Team Structure – Organogram • Business Model • Research Interests • Services Offered • Project Team Composition • The Legendary Case Study • Challenges & Rewards
    3. 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.
    6. 6. “One of the biggest challenges for thebiotechnology and pharmaceuticalcompanies in the 21st century will beto develop and deliver drugs that fitthe individual patient’s biology andpathophysiology” Jan Trost Jorgensen principal scientist at Dako
    7. 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. 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. 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. 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%
    12. 12. • Pre-Clinical & Clinical Development• Registration Submissions & Support• GxP (GMP, GCP, CLP) & Regulatory Consultancy• CRO Qualification• Preparatory regulatory audits• Due Diligence Audits
    13. 13. Typical Areas of Product Development • Vaccines • Peptides and Proteins • Follow On Biologics • Natural Products • Transdermals • Radionucleotides • Osteopathic Devices • In-vitro Diagnostic Reagents
    15. 15. Growing Importance of BiotechMedicine • 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. 16. Drug Discovery Research
    17. 17. On going Research Topics
    18. 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. 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. 20. Current Research Focus Long Term conditions Genetic Engineering Oncology Biotechnology current Gene therapy Infectious diseases
    22. 22. Project Team Composition • Clinicians • Pharmaceutical Scientists • Regulatory Strategists • Business Development Experts • Market Analysts
    24. 24. R&D Organizational Structure Chairman of the Board and CEO EVP Research & Development SVP SVP Regulatory SVP ExecutiveSVP 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. 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. 26. CASE STUDY
    27. 27. New Product Development DrugDrug Discovery Commercialization Development
    28. 28. New Product Development DrugDrug Discovery Commercialization Development
    29. 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. 30. Drug DiscoveryTop 5 Death Causing Diseases in 2005 (NCHS)
    31. 31. Therapeutic Areas (TA)• Oncology• Inflammation• Hematology/Nephrology• Metabolic Disorders• Neuroscience• Cardiovascular
    32. 32. Drug DiscoveryResearch Interest Target Discovery Target Validation Screening Drug Design
    33. 33. New Product Development DrugDrug Discovery Commercialization Development
    34. 34. Drug Development Preclinical studies Clinical Study: Phase I Post-Market Clinical Surveillance: Study: Phase Phase IV II Clinical Study: Phase III
    35. 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. 36. Product Pipeline: Phase I (contd..) 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. 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. 38. Product Pipeline: Phase II (contd..) 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. 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. 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)
    42. 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. 43. Manufacturing techniques for costreduction • 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. 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. 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. 46. Questions & Comments?