Vorasidenib, a novel inhibitor of the isocitrate dehydrogenase (IDH) enzyme, has shown promising results in treating recurrent or residual grade 2 IDH-mutant diffuse glioma, according to data from Servier’s Phase 3 INDIGO trial.
Diffuse gliomas are a type of brain tumor that arise from glial cells, which provide support and insulation for neurons. These tumors are classified based on their histological features and genetic mutations, with IDH mutations being a common driver of tumorigenesis.
IDH is an enzyme that plays a key role in cellular metabolism by converting isocitrate to alpha-ketoglutarate. Mutations in the IDH1 or IDH2 genes result in a gain of function that leads to the production of an oncometabolite called 2-hydroxyglutarate (2-HG). This metabolite accumulates in the cells and disrupts normal cellular processes, leading to tumorigenesis.
Vorasidenib is a small molecule inhibitor of mutant IDH1 that has been shown to reduce 2-HG levels and induce differentiation of glioma cells in preclinical studies. The INDIGO trial was designed to evaluate the efficacy and safety of vorasidenib in patients with recurrent or residual grade 2 IDH-mutant diffuse glioma.
The trial enrolled 251 patients who had previously received standard treatment with surgery and/or radiation therapy. Patients were randomized to receive either vorasidenib or placebo, and the primary endpoint was progression-free survival (PFS).
The results of the trial showed that vorasidenib significantly improved PFS compared to placebo, with a median PFS of 15.7 months in the vorasidenib group compared to 9.3 months in the placebo group. The overall response rate (ORR) was also higher in the vorasidenib group, with 56% of patients achieving a partial response or stable disease compared to 31% in the placebo group.
The safety profile of vorasidenib was generally favorable, with the most common adverse events being nausea, vomiting, and fatigue. There were no treatment-related deaths reported in the trial.
These results are significant because there are currently no approved treatments for recurrent or residual grade 2 IDH-mutant diffuse glioma. The standard of care for these patients is watchful waiting or re-resection, which may not be effective in controlling tumor growth.
Vorasidenib represents a new treatment option for these patients that targets the underlying genetic mutation driving tumorigenesis. The INDIGO trial data support the use of vorasidenib as a potential standard of care for recurrent or residual grade 2 IDH-mutant diffuse glioma.
In conclusion, the Phase 3 INDIGO trial data revealed that vorasidenib shows promising results in treating recurrent or residual grade 2 IDH-mutant diffuse glioma. This novel inhibitor of mutant IDH1 has been shown to improve PFS and ORR with a favorable safety profile. Vorasidenib represents a new treatment option for these patients and may become a standard of care in the future.
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- Source: Plato Data Intelligence.