Drug intelligence / Profile preview

CGX-926

Development stage
Preclinical
Lead developer
Congruence Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

**CGX-926** is an investigational, orally administered small molecule pharmacological corrector developed by Congruence Therapeutics using its proprietary Revenir™ computational platform. It targets **MC4R-deficient genetic obesity**, a rare early-onset condition caused by mutations in the melanocortin 4 receptor (**MC4R**) gene, affecting protein trafficking and function, with no approved therapies. CGX-926 stabilizes mutant MC4R proteins, corrects trafficking deficiencies to the cell surface, and restores receptor signaling, as shown in preclinical models where it reduced body weight gain, fat mass, and hyperphagia in MC4R-deficient mice. The company plans a Phase 1 trial in healthy volunteers and Phase 1b proof-of-concept in patients starting early 2026, following CTA filing in late 2025.[1][2][3][7][9]

Other names
CGX926CGX-926CGX 926
02

Targets

MC4R (Melanocortin 4 receptor)

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