3 People are part of an astonishing variety of life.
We’re all made of the same material. Typical Cell Protein Sugar Fat
Chronic Kidney Disease People with kidney disease do not make enough of the EPO protein, produced by the kidneys to stimulate production of red blood cells. Erythropoietin (EPO) Protein When complex processes are all working precisely, we are healthy… But for many people, something goes wrong. Disease is the result.
Different Treatments ______ ________ ____________ ________ Different Causes of Disease, Biotech Medicines:Live,& ComplexLarge
Biologics Differ From Traditional Drugs in Both Complexity and Sensitivity Manufacturing Unique bank of living cells;Unlikely to achieve identical copy Predictable chemical & reagent reaction; Identical copy can be made Characterization Many products well-characterized;Correlation of structure-function elusive Easy to fully characterize Stability Sensitive to storage andhandling conditions More stable Immunogenicity Higher potential Lower potential Structure Complex Simple Stability Complex Degradation Stable Variability Heterogeneous ‘product’ Single defined structure Biologics (Protein-based medicines) Pharmaceuticals (Chemically-based drugs) Pharmaceuticals (Chemically-based drugs) Size Large Small Properties
Typical Protein Production Process Fermentation units. Host cells thrive in these vats (also called bioreactors) filled with a life- sustaining bath of nutrients. Fermentation is the science of growing host cells in large quantities, so that the gene inside them produces the protein that we want in large quantities. Typical Protein Production Process Put gene into DNA vector Identify and isolate gene that will produce a medically useful protein Transfer into Host Cell Expression Screening/Selection DNA vectors are used to move genes into new organisms. They are generally plasmids (pieces of bacterial DNA that can live and replicate outside the chromosome). Chinese hamster ovary (CHO) cells were introduced in the early 1960s as an ideal cell line for genetic studies. Genes won’t produce proteins outside of a living cell, so we have to insert our gene into a living cell (a “host cell”). Biotech Medicine: The final protein must have precisely the right amino acid sequence, folded into precisely the right three-dimensional shape. Downstream Extraction/ Purification/ Dilution We must validate that downstream processing continues to remove viruses, DNA and toxins from the protein product stream. Validation testing may be defined as “the act of proving that any procedure, process, equipment, material, activity or system leads to the expected results.”
Biocomparables: Similar, not Identical to Original Products Different cell lines Different manufacturing processes....but not identicalBiocomparables are similar... Impact of small differences in either biological or manufacturing process could lead to different clinical efficacy and safety for patients No therapeutic substitution Immunogenicity differences
Amgen: A Biotechnology Pioneer Founded in 1980, Amgen was one of the first biotechnology companies to successfully discover, develop, and make protein- based medicines Today, we’re leaders in the next wave of innovation: –Developing therapies in multiple modalities –Driving cutting-edge research and development –Continuing to advance the science of biotechnological manufacturing
Our Worldwide Presence and a Leader In Biotechnology Manufacturing The Netherlands Belgium Ireland Spain Switzerland Juncos, Puerto Rico Washington, DC Louisville, KY West Greenwich, RI Cambridge, MA Toronto, ON Thousand Oaks, CA Seattle, WA Bothell, WA Hungary United Kingdom Denmark Portugal Italy Germany Norway France Czech Republic Sweden Austria Poland Australia Boulder, CO Longmont, CO Finland South San Francisco, CA Slovenia Slovakia Lithuania Latvia Estonia Luxembourg Burnaby, BC United Arab Emirates Mexico City, Mexico Hong Kong India Russia Greece Brazil Japan Bulgaria Romania Turkey Woburn, MA 54 Countries by 2011 75 Countries by 2015 35% of worldwide manufacturing capacity
Our Products Oncology, Nephrology, Bone, Metabolism, Inflammation… For additional information about Amgen products, including important safety information, please visit www.amgen.com
Research and Development at Amgen Guiding Principles Focus on serious illness Be modality independent Assess efficacy in patients Seamless integration from research through commercialization
Pioneering Science Delivers Vital Medicines For more information, visit www.amgen.com Our Mission : To serve Patients