Milademetan is an oral, selective, small molecule inhibitor that is currently in clinical development for the treatment of patients with TP53 wildtype cancers.1
When a cell’s DNA is damaged beyond repair, tumor suppressor protein p53 triggers cell death to prevent tumor development. If TP53 is mutated or suppressed, this protection is lost.2,3
MDM2 is a key negative regulator of p53 responsible for controlling and limiting p53 levels. If MDM2 levels are high, p53 levels may be significantly suppressed, aiding in tumor growth.3,4 Milademetan may reactivate p53, restoring the protection it provides.1
One of the historic challenges in developing a drug that inhibits the MDM2-p53 complex has been to deliver a benefit to patients while reducing toxicity.1,5 Rain is hopeful that milademetan’s pharmacological characteristics may support a dosing schedule that will reduce toxicity while allowing patients to experience potential benefits from the drug.
Rain is using in vitro diagnostics to confirm the presence of predictive biomarkers to help identify patients who may benefit from milademetan treatment.
