Drug intelligence / Profile preview

pafuramidine

Development stage
Phase 3
Lead developer
Immtech Pharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

Pafuramidine is an oral small molecule prodrug of furamidine (DB75), developed primarily for the treatment of *Pneumocystis jiroveci* pneumonia (PCP) and first-stage human African trypanosomiasis (sleeping sickness). It was granted orphan drug status by the US FDA for PCP in HIV/AIDS patients. Pafuramidine reached Phase III clinical trials for both PCP and sleeping sickness but development was halted due to concerns about kidney toxicity and drug-induced liver injury. The compound acts as a trypanocidal agent, with its antimicrobial activity believed to involve active uptake by purine transporter systems in trypanosomes and possible interference with DNA-associated enzymes or mitochondrial function, though the precise mechanism remains incompletely understood[1][3][5][6][7].

Brand names
DB289DB-289DB 289
Other names
pafuramidine maleate2,5-bis-(4-amidinophenyl)furan bis-O-methylamidoximepafuramidinaUNII-H1VG379J2XUNII-H-1VG379J2XUNII-H 1VG379J2X
02

Targets

ENT (Equilibrative nucleoside transporter family)

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