Experimental drug cures advanced leukemia in 18 patients

Researchers at the University of Texas MD Anderson Cancer Center. (USA) have been able to cure advanced leukemia in 18 patients thanks to the administration of the experimental drug revumenib, formerly known as SNDX-5613, according to the results of the phase I study ‘AUGMENT-101’, published in the journal ‘Nature’.

The overall response rate

Specifically.the overall response rate among 60 patients was 53 percent, and the rate of complete remission or complete remission with partial hematologic recovery was 30 percent. In addition, 78 percent of those affected achieved elimination of measurable residual disease.

“These results suggest that revumenib may be an effective oral targeted therapy for patients with leukemia. acute leukemia caused by genetic alterations. These response rates, especially the rates of elimination of residual disease, are the highest we have seen with any monotherapy used for these resistant leukemia subgroups,” said study leader Ghayas Issa.

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First evidence of safety

This is the first evidence showing the safety and clinical activity of such treatment-induced inhibition of menin in acute leukemia, and the data demonstrate the potential for targeting scaffold proteins that have been shown to be vulnerable spots in specific cancers. According to the investigator, targeting menin disrupts the gene transcription machinery and changes gene expression in cancer cells from a leukemia pattern to a normal pattern, ultimately leading to remission.

The trial enrolled 68 patients with a median age of 43 years, including children up to 10 months of age. Disease types included acute myeloid leukemia (82%), acute lymphocytic leukemia (16%), and mixed phenotype acute leukemia (2%). Among the included patients, 67.6 percent had KMT2A rearrangements, 20.6 percent had NPM1 mutations, and 11.8 percent had other genotypes.

Intensive pretreatment

Patients received intensive pretreatment with a median of four prior lines of therapy and 46 percent had a prior allogeneic stem cell transplant. The median duration of response was 9.1 months and the median overall survival was seven months. Twelve patients underwent allogeneic stem cell transplantation after response to revumenib.

“The responses in this trial show that menin inhibitors may be a promising treatment option. that patients tolerate well and could be the newest addition to successful targeted therapies for acute leukemia. I look forward to additional data from this and future trials to inform the potential opportunity to offer this targeted therapy to more patients,” the expert emphasized.

Enrollment for the Phase II cohort of the trial is ongoing. Future trials of revumenib will test combinations with other agents in various settings, including newly diagnosed leukemias, relapsed or refractory disease and maintenance therapy, for leukemias with KMT2A rearrangement or NPM1 mutation and other leukemias susceptible to menin inhibition.