Drug intelligence / Profile preview

Compound 4d

Development stage
Preclinical
Lead developer
Universidad Popular Autónoma del Estado de Puebla
Modality
Small Molecules
Administration
Oral
01

Overview

Compound 4d is a novel steroidal hydrazone derivative, specifically a spirostanic hydrazone, being investigated as a dual-action anabolic and anti-catabolic therapeutic for the treatment of muscle wasting disorders, including cancer-related cachexia and sarcopenia. It exerts its effects through a unique dual mechanism: it acts as an agonist of the Mas receptor (MasR), which activates the PI3K/AKT/mTOR signaling pathway to promote protein synthesis and muscle cell proliferation, while simultaneously functioning as an inhibitor of the activin receptors ACVR1 and ACVR2A (ActRIIA). By blocking these receptors, Compound 4d suppresses the myostatin-mediated SMAD signaling pathway, a primary driver of muscle atrophy. Preclinical studies have demonstrated its ability to increase S-phase cell populations and protein content in skeletal muscle models with low toxicity, positioning it as a promising pharmacological candidate for muscle-preserving interventions.

Other names
steroidal hydrazone compound 4dspirostanic hydrazone 4d
02

Targets

ACVR2B (Activin receptor type IIB)ACVR2A (Activin receptor type 2A)ACVR1 (Activin receptor type I)MAS1 (Mas receptor)

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