Drug intelligence / Profile preview

GB004

Development stage
Discontinued
Lead developer
Gossamer Bio
Modality
Small Molecules
Administration
Oral, Topical
01

Overview

GB004 is an orally administered, small molecule prolyl hydroxylase inhibitor designed to stabilize hypoxia-inducible factor 1 alpha (HIF-1α), a transcription factor involved in protective cellular responses at the intersection of hypoxia and inflammation. It is gut-targeted to promote mucosal healing and resolve local inflammation in the gastrointestinal tract without systemic immunosuppression. The drug was developed for inflammatory bowel diseases, particularly ulcerative colitis. Clinical studies have shown that GB004 achieves higher concentrations in colonic tissue than plasma and engages HIF pathway target genes relevant to epithelial barrier function. While early-phase trials indicated safety and some efficacy signals, a phase II trial did not meet its primary or secondary endpoints for ulcerative colitis[2][3][4][5][6][7][8].

Other names
AKB-4924AKB4924AKB 4924GB-004GB004GB 004
02

Targets

PHD1 (Prolyl hydroxylase domain-containing protein 1)

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