Phase II Trial Evaluates Tamibarotene-Based Therapy in MDS, AML

In this video, Stéphane de Botton, MD, PhD, discusses research that he presented during the 2023 European Hematology Association Congress in Frankfurt, Germany.

Dr. de Botton presented findings from a study of the molecular and clinical features of patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) who had RARA overexpression and were enrolled in a phase II trial evaluating tamibarotene-based therapy.

The work builds upon research showing a “novel genomically defined subset” of AML or MDS with RARA gene overexpression may be “an actionable target for tamibarotene,” which is an oral and selective RARα agonist, according to Dr. de Botton and colleagues.

“RARA was overexpressed in roughly 50% of MDS and 30% of AML [cases],” he said.

Dr. de Botton highlighted findings of the phase II trial.

“In a phase II trial, they showed that in ineligible patients for intensive chemotherapy overexpressing RARA, treatment with azacitidine plus tamibarotene led to a two-thirds of overall response rate, 50% complete remission,” he said. “In that case, the duration was 18 months, so it was fairly good. The reliability was excellent.”

The research on genetic and mutational features in patients with RARA overexpression is ongoing in the phase II AML-1 trial of tamibarotene plus venetoclax and azacitidine and in the phase III SELECT‑MDS‑1 trial of tamibarotene plus azacitidine.