binimetinib

Binimetinib is a small-molecule, orally administered inhibitor of mitogen-activated protein kinase kinase (MEK) 1 and 2, enzymes in the MAPK/ERK signaling pathway. By blocking MEK1/2 activity, binimetinib reduces the phosphorylation of ERK, leading to decreased cell proliferation and increased tumor cell death in cancers dependent on MAPK signaling.

Clinical development has focused on melanoma and other solid tumors. In melanoma, binimetinib has been evaluated in patients with NRAS mutations and in combination regimens with other targeted therapies or immunotherapies. It has also been studied in various other tumor types in phase II and III trials, with mixed results across different genetic contexts and treatment combinations. The agent has been investigated as both a monotherapy and a component of combination strategies intended to enhance efficacy or overcome resistance to other targeted therapies.

Safety and tolerability observed in trials align with patterns seen with MEK inhibitors. Reported adverse events commonly include skin rash, diarrhea, nausea, fatigue, and edema, as well as laboratory abnormalities such as elevated liver enzymes. Ocular toxicity, including visual changes or retinal effects, has been associated with MEK inhibitors in some patients and may require monitoring. Additional drug-related toxicities, such as cardiac or pulmonary effects, have been noted in broader MEK inhibitor experiences and warrant appropriate assessment during treatment.

Regulatory status for binimetinib has varied by jurisdiction, and it has not achieved broad regulatory approval for cancer treatment. It remains under investigation in clinical trials, with the potential for future development depending on ongoing study results.