Summary of Lesson 12 'Biologicals part 1'

Lecture 12: 13/11
Biopharmaceu,cals
Aims
- What are biopharmaceu0cals? defini0on?
- Difference between biopharmaceu0cals and pharmaceu0cals/drugs
- Examples of biopharmaceu0cals
- Advantages of biopharmaceu0cals
Introduc.on
Examples
- EPO (erythropoie0n)
- insulin
- Interferons
- Vaccines
- Interleukins
- Monoclonal an0bodies
o Top of biopharmaceu0cals, easy to produce
- CloMng factors
- Plasminogen ac0vators
- Colony s0mula0ng factors




FC fusion proteins -> very stable, you can add something, easy to produce and easy to purify
Oligonucleo0des become very popular
A lot of proteins are modified with glycosides
Valuable
- Innova0ve therapies for significant unmet medical needs
- Radically new approach
- - > 40 current licensed biopharmaceu0cals
- Over 1/3th of all drugs in development are biopharmaceu0cals
- New molecular en00es
o You see the evolu0on over 0me and at a certain point you get new biological
en00es. They are rapidly increasing -> quite interes0ng
Blockbuster drugs: top 10, situa:on 2010
- Blockbuster = drugs with a sale more than one billion/years
- Blockbuster drugs: top 15, situa0on 2012
- Blockbuster drugs: top 10, situa0on 2015
o 7 of the 10 are biologics-> valuable for companies
- Blockbuster drugs: top 10, situa0on 2018
- Blockbuster drugs: top 5, situa0on 2015 -2019

, o 4 of the 5 are monoclonal an0bodies -> drive a lot of money
- Blockbuster drugs: top 15, 2020
o New vaccine
- Blockbuster drugs: top 15, 2021
o Number one is a vaccine
Advantages
- Inherently highly specific for the target
o Biopharmaceu0cals are more specific because they are designed for a specific
target
o Difference with normal drug that you bring on the market
§ Library screening
• Black box: for example aspecificity, toxicity can not be seen
- Minimum risk for non-mechanism based toxicity and safety
- Capable of increasing growth factors, hormones, enzymes…
- Can modulate protein/protein interac0ons that cannot be targeted by small molecules
o You need a big molecule to block the binding between ligand and receptor
- Applicability in mul0ple therapeu0c areas and for mul0ple targets
o Mainly the ab that interact with the immunesystem
- Value
o Larger profits for companies
o Therapeu0c value
Defini.on
Which one is the biopharmaceu:cal?




- Led
o Biologicals are almost always injectables
§ Why it is difficult to develop them in a pill form
• Swallow protein -> we will digest it
o You can not store them for e long 0me
o Naming: Infliximab = monoclonal ab
What is the difference?




- How do you produce Trexall
o Chemical synthesis