Drug intelligence / Profile preview

AZD4017

Development stage
Phase 2
Lead developer
AstraZeneca
Modality
Small Molecules
Administration
Oral, Topical, Ophthalmic
01

Overview

AZD4017 is a selective, orally bioavailable small molecule inhibitor of 11β-hydroxysteroid dehydrogenase type 1 (11β-HSD1), developed by AstraZeneca. The drug was investigated for the treatment of obesity, raised intraocular pressure (IOP), type 2 diabetes mellitus, idiopathic intracranial hypertension (IIH; also known as pseudotumor cerebri), and Cushing syndrome. By inhibiting 11β-HSD1, AZD4017 blocks the conversion of inactive cortisone to active cortisol in metabolic tissues such as liver, skeletal muscle, and adipose tissue. This mechanism aims to reduce local glucocorticoid activation and mitigate adverse metabolic effects associated with excess glucocorticoids or exogenous steroid therapy. Clinical trials included monotherapy and combination therapy with prednisolone to reduce steroid-induced side effects[1][3][5][6][8]. Development for some indications was discontinued due to safety or efficacy concerns.

Other names
2-[(3S)-1-[5-(cyclohexylcarbamoyl)-6-propylsulfanylpyridin-2-yl]piperidin-3-yl]acetic acidUNII-3JL137394YUNII3JL137394YUNII 3JL137394Y
02

Targets

HSD11B1 (11-beta-hydroxysteroid dehydrogenase type 1)

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