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MED310 COPY > Drug Discovery > Flashcards

Drug Discovery Flashcards

1
Q

General principle of drug design?

A

Design on Molecular scale with particular therapeutic outcome

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2
Q

Main approaches

A
  • Small molecule
  • Large molecule
  • Gene therapy
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3
Q

What is the cost of goods and CNS penetrance in small molecules ?

A

Low Cost and there is CNS penetrance

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4
Q

Are small molecules validated in MND?

A

Yes

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5
Q

What are the biological targets of small molecules ??

A

Enzymes
Kinases
GPCRs
Ion channels

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6
Q

What is the cost of goods and CNS penetrance in Large molecules ??

A
  • High cost

- No penetrance

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7
Q

What is the target of Large molecule drugs ?

A
  • Cytokines
  • Cell surface R
  • Extracellular proteins
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8
Q

What is the cost of goods and CNS penetrance in Gene therapy ??

A
  • High Cost

- CNS Penetrance

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9
Q

What is the target of gene therapy ??

A
  • Gene Knockdown

- Gene replacement

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10
Q

What is the issue with CNS penetrance ??

A

BBB restrictions

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11
Q

What type of approach is used in small molecules?

A

Multidisciplinary

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12
Q

What are the preliminary stages of drug development?

A
  • Target identification
  • Hit Identification
  • Hit to lead
  • Lead optimisation
  • Candidate drug Pre nomination
  • Clinical development
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13
Q

Target identification

A

Build a case around particular target disease
Target must be validated – must be significant
Monogenic disorders favoured
- Target must be ‘druggable’
- Must be possible to modulate the target with small molecule drug

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14
Q

Possible Drug targets?

A

Targets

  • Receptors
  • Enzyme’s
  • Hormones& Factors
  • DNA
  • Nuclear Receptors
  • Ion channels
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15
Q

Issue with drug development ?

A

Difficult to match up chemistry with biology

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16
Q

Hit identification

A
  • Biochemical screening
  • Hit conformation
  • Test in more biological relevant assays
  • Iterative cycles of making similar compounds, screening those and feeding that information into new syntheses
  • Design/test cycles
  • Multidisciplinary
17
Q

Hit to lead

A
  • Additional in vitro tests
  • hERG channel assays – cardiac safety screening
  • in vivo testing – Distribution, metabolism, pharmacokinetics
  • select several families for lead optimisation and further evaluation
18
Q

Lead optimisation

A
  • Refine activity via further synthesis
  • Extensive in vivo testing for PK and efficacy
  • Preliminary toxicity in vivo
  • Clinical candidate selection
19
Q

Along side the drug development process…

A 
	market analysis 
	formulation,
	manufacturing scale up, 
	patenting 
	Regulatory – ethics panel
20
Q

Phase 1

A

aim- safety, toxicity, does ranging
Endpoint- Pharmacokinetic profiles, Adverse event profile
Nimber- 20-100
Type of study - Single, multiple does with healthy volunteers closely monitored
Success rate- &)%

21
Q

Phase 2a/2b

A 
Aim- Preliminary Effective ness data 
Endpoint- Biomarkers study, identify common short term side effects
Number- Several hundred
Type - Place controlled dose escalation 
Success- 33%
22
Q

Phase 3

A

Aim - Confirmation of effectiveness, evaluation and risk benefit profile
Endpoint- Primary effectiveness
Number - hundreds of thousands

Type- Large placebo controlled
Success - 25-30%

23
Q

After what phase is approval given ?

A

Phase 3

24
Q

Phase 4

A 
Aim- Safety 
Endpoint- Safety profile in patient population 
Numbers- Many thousands 
Type - Comparative 
Success rate- 20%
25
Q

Drivers of Drug discovery

A 
Applied research with profit motive 
Academic drug discovery 
Open innovation in pharma biotech 
Research excellent framework 
Academic entrepreneurship
26
Q

What is Repurposing?

A

used already but can it be applied somewhere else \

27
Q

What is the benefit of repurposing?

A
  • Reduced cost development
  • Rapid translation
  • Hits likely
28
Q

What is the disadvantage of repurposing?

A
  • Existing safety data might not support new use
  • Unwanted- on target affects – existing use of drug might not be wanted
  • Low/no activity at novel targets due to limited chemical diversity

MED310 COPY (18 decks)

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