From target to market

Question 1

Goal of drug/development program

Answer 1

Find a new chemical entity that improves health

Question 2

What are Janus Kinases? What is their mechanism of activiation?

Answer 2

Non-receptor tyrosine kinases that are bound to receptors that respond to cyctokines.

When cytokines auto- or trans-phosphorylation of JAKS occur, JAK phosphorylates receptors and causes STATS to bind to phosphorylated recepters. JAK phosphorylate STAT, which dissociates and activates transcription in the nucleus.

Question 3

Why is JAK 2 and JAK 3 drug targets?

Answer 3

JAK 2- Oncology (promotes cell growth, survival, angiogenesis)

JAK3- immunosuppression (deficiency causes immunodeficiency)

Question 4

What type of molecule is c-MET? What does it respond to? Why is it a drug target?

Answer 4

Receptor kinase. Responds to HGF (hepatocyte growth factor). Promotes angiogenesis.

Question 5

What drug targets JAK?

Answer 5

Exel-7019.

Question 6

What drug targets C-Met?

Answer 6

Exel-7184

Cabozantinib (approved for thyroid cancer, investigated for metastatic prostate cancer)

Question 7

What are the four phases of clinical trials?

Answer 7

I-safety

II-general efficacy

III-blinded comparison efficacy

IV-after approval