Cimdelirsen was evaluated in a 4-month double-blind, placebo-controlled phase 2 study in uncontrolled acromegaly patients (IGF-1 between 1.3 to 5 x upper limit of normal) treated with long-acting somatostatin receptor ligands (NCT03548415) and in an open-label extension safety study (NCT03967249). The gene discussed is IGF1; the disease is acromegaly.