Question

Please answer both questions.

1. Briefly compare and contrast the development of biological therapeutics versus small molecule drug discovery. What are the advantages and liabilities of biological therapeutics?

2. Report on an FDA approved biological therapeutic, briefly describe its structure and mechanism of action. Would you expect any off-target interactions? why or why not?

Answer 1

Small molecule drugs can be taken orally, and tend to work in the body within cells. biologics are larger in size, they are typically injected and interact within the body in the bloodstream or on the surfaces of cells, rather than within the cells.

Advantages and liabilities:-

Small molecules are generally synthesized through chemical reactions, but biologics are synthesized in specially Engineered cells.so biologics are harder to synthesize as compared to small molecules.

Small molecules are well-characterized, and can be easily purified and analyzed, but biologics are the mixture of different compound which make them harder to characterize and analyze.

FDA approved biological therepeutic:-

One of the recent FDA approved drug is ISTURISA. The active ingredient of the drug is OSILODROSTAT.

Structure:-

IUPAC Name- 4-[(5R)-6,7-dihydro-5H-pyrrolo[1,2-c]imidazol-5-yl]-3-fluorobenzonitrile

Mechanism:-

osilodrostat leads to normalisation of cortisol levels in the majority of patients by inhibiting 11β-hydroxylaseand aldosterone synthase .the primary objective is the treatment of Cushing’s patients