Multinational phase 1-2 trial of ODM-208 for heavily pretreated metastatic castration-resistant prostate cancer

Prostate cancer is regulated by steroid hormones, even in castration-resistant disease. ODM-208 is a novel inhibitor of cytochrome P450 11A1, which catalyzes the first step of steroid-hormone biosynthesis. The multinational phase 1-2 CYPIDES trial evaluated ODM-208 for heavily pretreated metastatic castration-resistant prostate cancer (mCRPC). The trial showed that ODM-208 potently inhibited steroid-hormone biosynthesis with the expected toxicity of adrenal insufficiency. Evidence of antitumor activity was seen, especially among patients with androgen receptor gene mutations (PSA decrease of 50% or more in 73.7% of patients).