Key clinical point : A novel retinoic acid receptor alpha agonist appeared safe in a phase 1 study of patients with relapsed and refractory high-grade myelodysplastic syndrome or acute myelogenous leukemia.
Major finding : A total of 4 of 11 patients (36%) had stable disease or better after two 28-day treatment cycles; the study ended early because of an inadequate supply of the drug.
Study details : The data come from a phase 1 dose-escalation study of the novel retinoic acid receptor alpha agonist IRX195183; 11 adults aged 18 to 60 years with relapsed or refractory myelodysplastic syndrome or acute myelogenous leukemia received the drug for two 28-day cycles with dosage starting at 50 mg daily or 75 mg daily.
Disclosures: The study was supported by the National Heart, Lung, and Blood Institute, the National Cancer Institute, the Leukemia and Lymphoma Society, and the Augustine Fellowship. IRX195183 was provided by Io Therapeutics, Inc. Lead author Dr. Ambinder had no financial conflicts to disclose. Several coauthors are authors on a patent for the use of IRX195183.
Source: Ambinder AJ et al. Front Oncol. 2020 Oct 23. doi: 10.3389/fonc.2020.587062.