IND 123 MIDTERM FINAL STUDY PAPER 2026
COMPLETE QUESTIONS AND ANSWERS
◉ What is the main focus during the hit identification phase?
Answer: To find compounds that show activity against the target in
assays.
◉ What is the outcome of successful proof of concept studies?
Answer: Guidance for the development of targeted therapies for
specific diseases.
◉ What is the role of in silico screening in drug discovery? Answer:
To use computer models to predict the binding of compounds to
targets.
◉ What is the goal of lead refinement in drug development? Answer:
To modify chemical structure to improve potency, selectivity, and
safety.
◉ What are the selection criteria for drug candidates? Answer:
Strong, consistent activity in assays and models, and reduced
toxicity.
, ◉ What is Trikafta? Answer: A next-gen corrector that is a triple-
combination therapy targeting the defective CFTR protein,
benefiting ~90% of CF patients.
◉ What are the early leads in CFTR modulator development?
Answer: Ivacaftor (potentiator) and tezacaftor (corrector).
◉ What is the average time and cost for drug development? Answer:
10-15+ years and approximately $2.6 billion.
◉ What is the difference between preclinical and nonclinical
studies? Answer: Preclinical studies assess safety and efficacy in
non-human subjects, while nonclinical studies are conducted under
regulated conditions to determine safety without human subjects.
◉ What are the goals of preclinical studies? Answer: Regulatory
compliance, safety assessment, efficacy evaluation, and data
production for regulatory approval.
◉ What does pharmacokinetics refer to? Answer: What the body
does to a drug, including parameters like T1/2, AUC, Tmax, Cmax,
Vd, and Clearance.
COMPLETE QUESTIONS AND ANSWERS
◉ What is the main focus during the hit identification phase?
Answer: To find compounds that show activity against the target in
assays.
◉ What is the outcome of successful proof of concept studies?
Answer: Guidance for the development of targeted therapies for
specific diseases.
◉ What is the role of in silico screening in drug discovery? Answer:
To use computer models to predict the binding of compounds to
targets.
◉ What is the goal of lead refinement in drug development? Answer:
To modify chemical structure to improve potency, selectivity, and
safety.
◉ What are the selection criteria for drug candidates? Answer:
Strong, consistent activity in assays and models, and reduced
toxicity.
, ◉ What is Trikafta? Answer: A next-gen corrector that is a triple-
combination therapy targeting the defective CFTR protein,
benefiting ~90% of CF patients.
◉ What are the early leads in CFTR modulator development?
Answer: Ivacaftor (potentiator) and tezacaftor (corrector).
◉ What is the average time and cost for drug development? Answer:
10-15+ years and approximately $2.6 billion.
◉ What is the difference between preclinical and nonclinical
studies? Answer: Preclinical studies assess safety and efficacy in
non-human subjects, while nonclinical studies are conducted under
regulated conditions to determine safety without human subjects.
◉ What are the goals of preclinical studies? Answer: Regulatory
compliance, safety assessment, efficacy evaluation, and data
production for regulatory approval.
◉ What does pharmacokinetics refer to? Answer: What the body
does to a drug, including parameters like T1/2, AUC, Tmax, Cmax,
Vd, and Clearance.
