Drug Candidate Discovery (3)

Question 1

What phase is this part of?

Answer 1

Pre-clinical R/D

Question 2

Timeline;total years

Answer 2

HTS (6mon-2yrs) –>
Active compound “HIT” compound (3-4 months) –>
HIT TO LEAD compound(3 months) –>
Lead optimization(6-9 mon) –> candidate drugs (2 yrs)

Total years: 3.5-5.5 yrs

Question 3

Target Identification

Answer 3

• understand disease mechanism to identify potential drug targets
• HTS (vivo/vitro)

Question 4

Lead identification (HIT)

Answer 4

• Once disease mechanism + drug targets are identified and validated
• Bio assays can be designed
• Reduce the amount of compounds to be tested from the library (important)
• trying to accurate number of “hits” or possible drug candidates

Question 5

Lead optimization

Answer 5

• In vivo animal
  and in vitro cell-based studies are used to understand the metabolism of
  compounds in the body and to gain some data on toxicity
• This allows for a more effective/efficient compound
• This leads to clinical trials

Question 6

target, lead compound timeline (HTS)

Answer 6

Target identification/validity –> Lead identification –> Lead optimization –> Candidate drug

Question 7

Target Identification: Types of possible biological mechanisms

Answer 7

• Enzyme inhibitor – 38%
• Receptor antagonist (blocking response to receptor) – 24%
• Receptor agonist (enhancing response) – 12%
• Ion channel modulator –8%

Question 8

Bio-assay

Answer 8

• Based on cells or isolated proteins

- assess if compound interacts with target (ie HTS)