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 showed that vorasidenib significantly improved PFS compared to placebo, with a median PFS of 15.9 months versus 9.3 months, respectively. The overall response rate (ORR) was also higher in the vorasidenib group, with 56% of patients achieving a partial response or better compared to 14% in the placebo group.
In addition, vorasidenib was well-tolerated, with no unexpected safety signals. The most common adverse events were mild to moderate in severity and included nausea, fatigue, and headache.
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 is watchful waiting or re-resection, which may not be effective in delaying disease progression.
Vorasidenib represents a new treatment option for these patients and has the potential to improve outcomes and quality of life. The data from the INDIGO trial support the continued development of vorasidenib as a targeted therapy for IDH-mutant gliomas.
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 demonstrated significant improvements in PFS and ORR, with a favorable safety profile. These findings highlight the potential of vorasidenib as a targeted therapy for IDH-mutant gliomas and provide hope for patients with this rare and challenging disease.
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- Source: Plato Data Intelligence.